MeiraGTx Annual Report 2018

Plain-text annual report

Table of Contents UNITED STATESSECURITIES AND EXCHANGE COMMISSIONWashington, D.C. 20549 FORM 10-K ☒ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934For the fiscal year ended December 31, 2018OR ☐TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934For the transition period from to Commission file number: 001-38520 MEIRAGTX HOLDINGS PLC(Exact name of registrant as specified in its charter) Cayman Islands Not applicable(State or other jurisdiction ofincorporation or organization) (I.R.S. EmployerIdentification No.)430 East 29th Street, 10th FloorNew York, NY 10016(Address of principal executive offices) (Zip Code)(646) 490 2965(Registrant’s telephone number, including area code)Securities registered pursuant to Section 12(b) of the Act: Title of each class Name of exchange on which registeredOrdinary Shares, $0.00003881 Nominal value NasdaqSecurities registered pursuant to Section 12(g) of the Act: None Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes ☐ No ☒Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Exchange Act. Yes ☐ No ☒Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90days. Yes ☒ No ☐Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T(§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐Indicate by check mark if disclosure of delinquent filers pursuant to Item 405 of Regulation S-K (§229.405 of this chapter) is not contained herein, and will not be contained,to the best of registrant’s knowledge, in definitive proxy or information statements incorporated by reference in Part III of this Form 10-K or any amendment to this Form10-K. ☒Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, smaller reporting company, or an emerging growthcompany. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the ExchangeAct. Large accelerated filer ☐ Accelerated filer ☐Non-accelerated filer ☒ Smaller reporting company ☒ Emerging growth company ☒If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financialaccounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒As of June 30, 2018, the aggregate market value of the registrant’s ordinary shares held by non-affiliates of the registrant was approximately $154.6 million (based upon theclosing sale price of the registrant’s ordinary shares on that date on the Nasdaq Global Select Market).As of March 15, 2019, the registrant had 33,183,734 ordinary shares outstanding.DOCUMENTS INCORPORATED BY REFERENCEPortions of the registrant’s definitive Proxy Statement relating to its 2019 annual shareholder meeting to be filed with the SEC within 120 days after the end of the fiscal yearended December 31, 2018 are incorporated herein by reference in Part III. Table of ContentsCONTENTS Page PART I 3 Item 1A. Risk Factors 65 Item 1B. Unresolved Staff Comments 120 Item 2. Properties 120 Item 3. Legal Proceedings 120 Item 4. Mine Safety Disclosures 120 PART II 121 Item 5. Market For Registrant’s Common Equity, Related Stockholder Matters And Issuer Purchases Of Securities 121 Item 6. Selected Financial Data 121 Item 7. Management’s Discussion And Analysis Of Financial Condition And Results Of Operations 122 Item 7A. Quantitative And Qualitative Disclosures About Market Risk. 134 Item 8. Financial Statements And Supplementary Data 135 Item 9. Changes In And Disagreements With Accountants On Accounting And Financial Disclosure 174 Item 9A. Controls And Procedures 174 Item 9B. Other Information 174 PART III 175 Item 10. Directors, Executive Officers And Corporate Governance 175 Item 11. Executive Compensation 175 Item 12. Security Ownership Of Certain Beneficial Owners And Management And Related Stockholder Matters 175 Item 13. Certain Relationships And Related Transactions, And Director Independence 176 Item 14. Principal Accountant Fees And Services 176 PART IV 177 Item 15. Exhibits 177 Item 16. Form 10-K Summary 180 Table of ContentsSPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTSThis Annual Report on Form 10-K (the “Form 10-K”) contains forward-looking statements that can involve substantial risks and uncertainties. Allstatements other than statements of historical facts contained in this Form 10-K, including statements regarding our future results of operations andfinancial position, business strategy, prospective products, product approvals, research and development costs, future revenue, timing and likelihoodof success, plans and objectives of management for future operations, future results of anticipated products and prospects, plans and objectives ofmanagement are forward-looking statements. These statements involve known and unknown risks, uncertainties and other important factors that maycause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed orimplied by the forward-looking statements.In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,”“intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “would” or “continue” or the negative of these terms orother similar expressions. The forward-looking statements in this Form 10-K are only predictions. We have based these forward-looking statementslargely on our current expectations and projections about future events and financial trends that we believe may affect our business, financialcondition and results of operations. These forward-looking statements speak only as of the date of this Form 10-K and are subject to a number ofrisks, uncertainties and assumptions described under the sections in this Form 10-K entitled “Item 1A. Risk Factors” and “Item 7. Management’sDiscussion and Analysis of Financial Condition and Results of Operations” and elsewhere in this Form 10-K. Because forward-looking statementsare inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond our control, youshould not rely on these forward-looking statements as predictions of future events. The events and circumstances reflected in our forward-lookingstatements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Moreover,we operate in an evolving environment. New risk factors and uncertainties may emerge from time to time, and it is not possible for management topredict all risk factors and uncertainties. Except as required by applicable law, we do not plan to publicly update or revise any forward-lookingstatements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.You should read this Form 10-K and the documents that we reference in this Form 10-K and have filed as exhibits to this Form 10-K, completely andwith the understanding that our actual future results may be materially different from what we expect.In addition, statements that “we believe” and similar statements reflect our beliefs and opinions on the relevant subject. These statements are basedupon information available to us as of the date of this Form 10-K, and while we believe such information forms a reasonable basis for suchstatements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustiveinquiry into, or review of, all relevant information. These statements are inherently uncertain and investors are cautioned not to unduly rely uponthese statements. 1Table of ContentsBASIS OF PRESENTATIONOn June 7, 2018, in connection with its initial public offering (the “IPO”), MeiraGTx Holdings plc, an exempted company incorporated under the lawsof the Cayman Islands, acquired all the issued and outstanding ordinary shares of MeiraGTx Limited pursuant to a series of reorganization transactions.We refer to these events in this Form 10-K as the “Reorganization Transactions.” Prior to the Reorganization Transactions, MeiraGTx Holdings plc hadnot conducted any operations and had nominal assets and liabilities.Unless the context otherwise requires, references in this Form 10-K to “Meira,” “we,” “us”, “our” or “the Company” refer to (i) MeiraGTx Limited andits subsidiaries prior to the Reorganization Transactions and (ii) MeiraGTx Holdings plc and its subsidiaries upon completion of the ReorganizationTransactions, as applicable.We have proprietary rights to trademarks, trade names and service marks appearing in this Form 10-K that are important to our business. Solely forconvenience, the trademarks, trade names and service marks may appear in this Form 10-K without the ® and TM symbols, but any such references arenot intended to indicate, in any way, that we forgo or will not assert, to the fullest extent under applicable law, our rights or the rights of the applicablelicensors to these trademarks, trade names and service marks. All trademarks, trade names and service marks appearing in this Form 10-K are theproperty of their respective owners. 2Table of ContentsINDUSTRY AND OTHER DATAWe obtained the industry, market and competitive position data in this Form 10-K from our own internal estimates and research as well asfrom industry and general publications and research, surveys and studies conducted by third parties. Industry publications, studies and surveysgenerally state that they have been obtained from sources believed to be reliable, although they do not guarantee the accuracy or completeness of suchinformation. While we believe that each of these studies and publications is reliable, we have not independently verified market and industry data fromthird-party sources. While we believe our internal company research as to such matters is reliable and the market definitions are appropriate, neithersuch research nor these definitions have been verified by any independent source.PART IITEM 1. BUSINESSOverviewWe are a vertically integrated, clinical stage gene therapy company with five programs in clinical development and a broad pipeline ofpreclinical and research programs. We have core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as apotentially transformative gene regulation technology. Led by an experienced management team, we have taken a portfolio approach by licensing,acquiring and developing technologies that give us depth across both product candidates and indications. Though initially focusing on the eye,salivary gland and central nervous system, we intend to expand our focus in the future to develop additional gene therapy treatments for patientssuffering from a range of serious diseases.We own and operate a flexible and scalable viral vector manufacturing facility that we expect can supply our current clinical andpreclinical programs through regulatory approval and, should they be approved, provide sufficient capacity for commercial production. Completed inearly 2018 and designed to meet global regulatory requirements, including the current good manufacturing practices, or cGMP, required by the U.S.Food and Drug Administration, or FDA, our 29,000 square foot facility has two cell production suites, three independent viral vector production suitesproviding multi-product and multi-viral vector manufacturing capabilities and an integrated, flexible fill-and-finish suite. In May 2018, we weregranted a license to manufacture gene therapy product candidates in our cGMP compliant manufacturing facility by the UK Medicines and Healthcareproducts Regulatory Agency. On December 14, 2018, we acquired from Moorfields Eye Hospital NHS Foundation Trust of Moorfields Eye Hospital along leasehold interest, (the “Head Lease”), in the site of our manufacturing facility, 92 Britannia Walk, London N1 7LU, for a purchase price of£5,250,000 (approximately $6,615,000 assuming a rate of $1.26 per GBP on the date of the acquisition. As a result of this transaction, we are now thetenant under the Head Lease, which has a remaining term of 108 years, with no facility rent dueWe have also established a comprehensive platform for the efficient clinical development of the next generation of gene therapies andmanufacturing in accordance with cGMP. Our deep understanding of disease models informs our development of potency assays for the cGMPproduction of our product candidates, and our teams experienced in viral vector design and optimization work closely with our process developmentteam to design viral vectors and develop proprietary production cell lines for efficient scaling of manufacturing processes.We are also developing a potentially transformative technology to enable the use of small molecules to turn gene therapy productcandidates on and off. The aim of this gene regulation platform is to convert gene therapy into a generalizable delivery mechanism for biologic drugsusing a small molecule “switch” for temporal control. We believe the capacity for temporal control of gene therapy products has the potential totransform the gene therapy landscape by opening up new treatment possibilities. 3Table of ContentsOur PipelineOur initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, or IRDs, severe forms of xerostomia andneurodegenerative diseases. Utilizing our product development platform, we have assembled a pipeline of gene therapies to treat these serious diseases.Our criteria for selecting our initial product candidates included: • unmet medical need; • high potential for meaningful clinical benefit; • promising preclinical data using multiple animal models as well as human stem cell derived organoids; • compartmentalized anatomy of target tissue and the partially immune protected nature of target tissue; and • understanding of the disease state from natural history studies and detailed long-term characterization of patients prior to entry intogene therapy treatment studies.A summary of our product candidates and the status of such product candidates as of March 15, 2019 is described below. We retainworldwide development and commercialization rights to all of our product candidates, with the exception of AAV-CNGB3, AAV-CNGA3 andAAV-RPGR, which are subject to a strategic Collaboration, Option and License Agreement (the “Collaboration Agreement”) that we executed withJanssen Pharmaceuticals, Inc. (“Janssen”), one of the Janssen Pharmaceutical Companies of Johnson & Johnson that was executed on January 30, 2019. 4Table of ContentsIn addition to these clinical and preclinical programs, we have preclinical and research programs in other indications and novel moleculartechnologies that we aim to advance into clinical development, including: • neovascular age related macular degeneration, or wet AMD – use of a gene therapy product to deliver an antibody targeting thevascular endothelial growth factor receptor 2, or anti-VEGFR2, with the aim of blocking disease related vascular formation in theeye; • geographic atrophy age related macular degeneration, or dry AMD – use of gene therapy technology to introduce light sensitivemolecules into rod photoreceptors in order to restore some aspects of vision lost in this disease; • amyotrophic lateral sclerosis, or ALS—targeting dysregulation of neuronal RNA, processing, which we believe may lead to thedegeneration of motor neurons that occurs in ALS; • Alzheimer’s disease—targeting endosomal trafficking, which is a central mechanism that we believe underlies Alzheimer’s disease;and • gene regulation—use of our proprietary RNA shape regulation cassette to switch gene therapy product candidates on and off withsmall molecules, potentially transforming gene therapy technology into a delivery mechanism for a broad array of biologic drugs. 5Table of ContentsOur Ophthalmology ProgramsEye diseases are our first area of clinical focus and we aim to provide treatments with durable, long-term clinical benefit that will haltvision loss in patients. We currently have three ongoing Phase 1/2 clinical programs in IRDs, with an additional program expected to initiate aPhase 1/2 clinical trial in 2019. The targets of our three ongoing Phase 1/2 ophthalmology programs include achromatopsia, or ACHM, related tomutations in CNGB3, X-linked retinitis pigmentosa related to mutations in RPGR, or XLRP-RPGR, and inherited retinal dystrophy caused bymutations in RPE65, or RPE65-deficiency. We also have a product candidate that was manufactured and released for compassionate use under aspecials license in the United Kingdom to treat patients with Leber congenital amaurosis 4, or LCA4, caused by mutations in AIPL1. For each of ourPhase 1/2 clinical programs we have a prospectively designed natural history study ongoing, which includes the same endpoints as our correspondinggene therapy treatment trial. We believe use of these natural history studies differentiates our programs by providing patient populations to facilitatethe efficient execution of our clinical trials and offering insight into the appropriate endpoints for regulatory approval of our gene therapy productcandidates. In addition to these clinical programs in IRDs, we have preclinical programs that apply novel approaches to both wet and dry AMD.The FDA and EMA have granted orphan drug designation to each product candidate in our ongoing clinical programs, including thosetreating mutations in CNGB3, RPGR and RPE65, as well our product candidates for the treatment of achromatopsia due to autosomal-recessive CNGA3gene mutations and to treat mutations in AIPL1. The FDA has also granted rare pediatric disease designation for our clinical programs treatingmutations in RPE65 and CNGB3 and our product candidate for the treatment of autosomal-recessive CNGA3 gene mutations, and Fast Trackdesignation to our clinical programs for the treatment of achromatopsia caused by CNGB3 gene mutations to improve visual function and for thetreatment of XLRP caused by mutations in RPGR. We have also received PRIME designation from the EMA for our clinical program treating mutationsin CNGB3.The deep scientific and clinical understanding of IRDs driving our approach to gene therapy development helps us to optimize our productcandidates for each specific genetic mutation and phenotype. We develop our viral vectors by selecting and modifying proprietary cell specificpromoters, selecting appropriate capsids for transfection of target cells and refining the vector for efficient production and scalable manufacturing. Notonly does this allow us to synergistically target a portfolio of inherited eye conditions, we also believe it has potential to be applied to thedevelopment of gene therapies for other diseases.Our longstanding relationships with leading institutions in retinal disease treatment, including the Moorfields Eye Hospital in London, theUniversity of Michigan Kellogg Eye Center, Massachusetts Eye and Ear, the Medical College of Wisconsin & Froedtert Hospital and the Casey EyeInstitute at the Oregon Health & Science University, provide us with access to experts whose guidance and insight informs our development strategy,as well potential patients for our clinical trials.Our Salivary Gland ProgramsOur second area of clinical focus is xerostomia, a chronic and debilitating disorder of the salivary glands in which saliva production isimpaired. Xerostomia may be caused by a number of different insults to the salivary glands, including radiation therapy for head and neck cancer andcertain autoimmune diseases. A Phase 1 clinical trial of our gene therapy product candidate, AAV-AQP1, is ongoing in patients who have survivedcancer free for five or more years following treatment for head and neck cancer and are suffering from grade 2 or 3 radiation induced late xerostomia, orRIX. There are approximately 170,000 grade 2 or 3 RIX patients who have survived two or more years after radiation treatment for head and neckcancer in the United States, with approximately 10,000 new cases each year. We also intend to initiate a Phase 1/2 clinical trial of AAV-AQP1 for thetreatment of patients with chronic xerostomia caused by Sjogren’s syndrome, an autoimmune disease affecting more than two million people in theUnited States.The FDA has granted orphan drug designation to AAV-AQP1. 6Table of ContentsOur Neurodegenerative Disease ProgramsNeurodegenerative diseases are our third area of focus. Our first target indication is Parkinson’s disease, where we have Phase 2 clinicaldata from a successful randomized, double-blind, sham-controlled trial. Our product candidate targeting Parkinson’s disease, AAV-GAD, is designed todeliver the glutamic acid decarboxylase, or GAD, gene to the subthalamic nucleus in order to increase production of GABA, the primary inhibitoryneurotransmitter in the human brain. GAD is the rate-limiting enzyme in the synthesis of GABA, therefore we believe that increasing subthalamicnucleus GAD expression through gene therapy has the potential to result in normalization of motor circuits and improve symptoms in Parkinson’sdisease patients without affecting other brain regions, which can be responsible for complications of existing therapies. To date, we have not had anyformal meetings with regulatory agencies nor determined the regulatory pathway and any potential related development costs for our recently acquiredAAV-GAD gene therapy program for Parkinson’s disease.Our second target indication is ALS, where we currently have IND-enabling studies ongoing. We believe our approach to treating ALSpatients is differentiated because, rather than targeting a specific genetic defect that defines a small subset of ALS patients, we aim to target theunderlying cell biology driving motor neuron death in ALS, potentially enabling us to treat a broader patient population that includes both sporadicand inherited forms of the disease. Increasing evidence suggests a critical role of RNA metabolism in neuronal cells, in particular in motor neurons thatare specifically affected in ALS. We believe that dysregulation of neuronal RNA processes results in the degeneration of motor neurons that leads toALS. Using our viral vector product candidate, AAV-UPF1, we target the central quality control system regulating RNA in motor neurons with the aimof enhancing motor neuron survival in ALS patients.We have an Alzheimer’s disease program that is likewise directed towards the underlying cell biology of the disease, in this caseendosomal trafficking, a mechanism cells use to cycle proteins to the cell surface. Over the past decade, evidence has emerged supporting endosomaltrafficking dysfunction in neurons as a central process in the early etiology of Alzheimer’s disease. In particular, a master regulator of trafficking out ofthe endosomes, called retromer, has been implicated. We are in the process of identifying what we believe to be the optimal approach to restoringnormal endosomal function to the neurons that are the first to be affected in Alzheimer’s. In parallel, we are developing and validating biomarkers ofendosomal dysfunction and pre-symptomatic Alzheimer’s disease. We believe this approach may also provide a framework for treating certain forms ofParkinson’s disease that are also associated with endosomal dysfunction.Our StrengthsIn addition to our four ongoing clinical programs, we have a broad pipeline of preclinical programs, core capabilities in viral vector designand optimization, gene therapy manufacturing and a potentially transformative gene regulation technology. Utilizing the following key strengths, weaim to develop, commercialize and expand our portfolio of product candidates. • Deep Expertise in Gene Therapy Development: We believe our expertise in viral vector design, optimization and processdevelopment allows us to efficiently advance gene therapy products candidates from preclinical development to cGMPmanufacturing and clinical development through commercialization. • Potentially Transformative Gene Regulation Technology Platform: We are developing proprietary technology to enableinnovative gene therapy treatments whose expression can be turned on and off with an easily administered small molecule. Webelieve the capacity for temporal control of gene therapy products has the potential to transform the gene therapy landscape byopening up new treatment possibilities. • Manufacturing Capabilities and Capacity: We have a flexible and scalable cGMP manufacturing facility and production process,which we expect can supply all of our current clinical and preclinical programs through regulatory approval and, should they beapproved, provide sufficient capacity for their commercial production. 7Table of Contents • Robust and Diverse Clinical and Preclinical Pipeline: Applying our portfolio approach to gene therapy product development, ourinitial focus is on treatments for IRDs, salivary glands disorders and neurodegenerative diseases with potential for acceleratedapproval and has produced five programs in clinical development and multiple preclinical development programs. • Relationships with Leading Institutions: Our longstanding relationships with leading institutions and experts provides us withguidance on development strategy and access to potential patients for our clinical trials. • Natural History Study Data: We sponsor ongoing prospective long-term natural history studies in IRDs that facilitate our abilityto efficiently enroll our treatment studies, potentially reducing clinical trial timelines and providing insight into the appropriateendpoints for regulatory approval.Our StrategyOur goal is to develop and commercialize innovative gene therapy products to treat serious disorders and broaden the scope of indicationsthat may be treatable by our gene therapies. Our strategy to achieve this goal is to: • successfully complete clinical development, obtain regulatory approval and commercialize our pipeline of gene therapy productcandidates to treat disorders of the eye and salivary gland; • continue to advance the development of our pipeline of product candidates for the treatment of neurodegenerative disorders; • utilize our viral vector design and optimization capabilities to identify and develop new gene therapies for other serious diseases; • advance the development of our potentially transformative proprietary technology for regulating the activity of gene therapyproducts using small molecules and initiate clinical trials of new product candidates; and • continue to pursue and evaluate further strategic collaborations with additional biotechnology and pharmaceutical companies toleverage our capabilities, manufacturing capacity and proprietary gene regulation technology. 8Table of ContentsThe figure below depicts the steps in our product engine, including disease selection, design, development/testing, clinical andmanufacturing: Gene Therapy OverviewGene therapy uses a delivery vehicle, referred to as a vector, to insert a functionally active gene into cells in the body. The gene encodes atherapeutic protein that may block disease pathways or may enhance a deficient pathway. Gene therapy has been studied for over 50 years, with avariety of different viral vectors employed to deliver therapeutic genes. Since the first clinical study of therapeutic gene transfer in humans in 1990,more than 2,300 gene therapy studies covering a broad range of disease targets have been initiated. Recently, the first gene therapies have receivedregulatory approval, including approval by the FDA of Luxturna for an ophthalmology condition, resulting in a growing acceptance of gene therapytechnology as a potentially safe and effective therapeutic approach.Our current programs use the adeno-associated virus, or AAV, as the vector for delivering gene sequences into a patient’s cells. The keycomponents of an AAV vector include: (i) the capsid, or the outer viral protein shell that encloses the target DNA, which is responsible for binding tothe cell surface and allowing the therapeutic gene that it is carrying to enter the cell; (ii) the therapeutic gene, or transgene, that encodes the therapeuticprotein; and (iii) the promoter, or the DNA sequence that drives the expression of the transgene. AAV is a good vector for gene therapy deliverybecause of its relative safety and broad applicability. AAV is less immunogenic, or less prone to causing an immune reaction, than previousgenerations of gene therapy vectors, such as adenoviral vectors and AAV does not readily integrate into the genome of the target cell, reducing thepotential for oncogenesis, or the induction of cancer. AAV vectors can transfer a therapeutic gene into, or transduce, numerous cell types. Slightdifferences in capsid proteins can modulate the efficiency with which 9Table of Contentsdifferent capsids deliver genes to different cells, thus allowing different AAV capsids to be selected to most effectively target particular cell types.The therapeutic gene sequence that enters the targeted cell includes both the protein coding region and an engineered promoter sequencethat is used to drive functional gene expression. These engineered promoters may be designed to drive different levels of gene expression or to limitgene expression to specific cell types. Additional aspects of the transgene sequence may be engineered for optimal gene expression, such as codonusage and synthetic introns, which may enhance levels of therapeutic protein expression.Gene therapy can be used to address monogenic diseases, which result mutations in a single gene in a patient’s genome. In such cases, theviral vector is used to deliver a normal copy of the gene to the cells that are defective due to the lack of the gene function. The normal gene then drivesproduction of the missing protein and offers a therapeutic benefit in patients with the disease. This gene replacement approach underlies all of ourclinical IRD programs.Rather than replacing a gene that is defective or missing in a monogenic disease, gene therapy can also provide a therapeutic impact byadding a particular new gene function to cells and thereby change cell behavior and function. This is the aim of our salivary gland programs, where ourtreatment is designed to promote water to flow through otherwise impermeable cells in damaged salivary glands and increase saliva flow into themouth. Additionally, gene therapy may be used to deliver a therapeutic protein that may block a disease pathway or enhance a deficient cellularpathway in multifactorial diseases such as wet AMD and neurodegenerative diseases, including ALS and Alzheimer’s disease.Importantly, AAV vectors enable targeting of therapeutic genes to non-dividing cells, in which they are thought to remain for the rest ofthe cell’s life. This means that a single treatment may offer patients a durable effect and long-term benefit. The specific cells of the eye, salivary glandand the neurons that we target in our current gene therapy programs are largely non-dividing cells and preclinical evidence has shown that they can beeffectively targeted by the specific AAV capsids that we use, enabling us to potentially achieve a durable impact on each of the diseases that we treat.Our Ophthalmology ProgramsOverview and StrategyWe currently have three ongoing Phase 1/2 clinical programs targeting IRDs, including ACHM related to mutations in CNGB3, XLRP-RPGR and RPE65-deficiency, with an additional program expected to enter a Phase 1/2 clinical trial in 2019 in patients with ACHM related tomutations in CNGA3. We also have a product candidate that was manufactured and released for compassionate use under a specials license in the UK totreat LCA4 patients. We chose diseases of the eye as our first area of clinical focus because we believe the eye is ideally suited for gene therapy for thefollowing reasons. • The eye is easily accessible and has highly compartmentalized anatomy, which allows for accurate delivery of vectors to specifictissues using direct visualization and microsurgical techniques. • The structure of the eye allows for efficient delivery to specific cell subtypes with small volumes of vector, making the dose perpatient much lower than for systemic treatment. • Anatomical barriers and unique structure of the eye make the immune response to the intraocular administration of vectors moreattenuated than systemic administration. • Largely non-dividing cell populations in the eye make good targets for potentially stable, long-term gene delivery and expression. • The retina, a structure in the back of the eye, is visible and there are many well validated structural and functional readoutsallowing the detailed assessment of the therapeutic impact of the gene therapy treatment. 10Table of ContentsOur strategy for developing gene therapies targeting eye diseases is to begin with a number of monogenic IRDs that are good candidatesfor gene replacement therapies and expand to more common eye diseases over time. We have taken a portfolio approach to the development of IRDsbecause, while some of these genetic defects are rare, IRDs as a class are one of the most common causes of blindness in working age adults and thereare multiple synergies at the clinical, regulatory and commercial levels between many of these diseases caused by different gene mutations.Two of our clinical-stage product candidates are targeting IRD indications in achromatopsia caused by mutations in the CNGB3 gene andinherited retinal dystrophy caused by mutations in the RPE65 gene. The primary defect in these particular genetic disorders is the absence of functionof one of the two different types of photoreceptors that constitute the light sensing part of the retina, rods or cones. In achromatopsia, the conescompletely lack function, while in RPE65-deficiency it is the rods that lack function. In these indications, we anticipate that restoring a normal copy ofthe mutant gene to the retina of patients with these genetic disorders may restore function to the particular type of photoreceptor that is defective ineach of these indications. Although cone photoreceptors are generally preserved during childhood in RPE65-deficient patients, the lack of healthyactive rods eventually affects the cones that they support and over time both rods and cones die, the retina degenerates and RPE65-deficient patientsbecome totally blind.We also have a clinical-stage product candidate for XLRP-RPGR, in which all photoreceptors, both rods and cones, function poorly,leading to degeneration of the retina and total blindness. We have used data and learnings from our RPE65 studies to inform the design and techniquesused in our XLRP-RPGR program. The aim of our XLRP-RPGR treatment is to slow the degeneration of the retina, which we measure using validatedsurrogate structural endpoints and changes in the detailed structural maps of these patients’ retinas over time.In order to expand our gene therapy pipeline for retinal diseases, we are also developing treatments for certain multifactorial eye diseases,which are diseases caused by multiple genetic or environmental factors. We have a preclinical program using an anti-VEGFR2 antibody targeting wetAMD and we are currently conducting IND-enabling studites for this program. We are also working to integrate our gene regulation technology withour wet AMD program. Our ultimate aim is to activate the anti-VEGFR2 antibody gene that we have installed in the eye with a small molecule appliedtopically in an eye drop. This could potentially allow for intermittent dosing of the antibody using an eye drop rather than injection. Additionally, weare developing a novel approach to treat advanced dry AMD patients who have lost central vision through our innovative “rod-to-cone” technology.By genetically engineering rods with molecules that will improve their speed of response to light, we aim to effectively transform a patch of rodphotoreceptors in the outer part of the retina to behave more like cone photoreceptors, thus improving vision.We intend to leverage our platform to take advantage of the many synergies across our ophthalmology programs, including identification,diagnosis and characterization of patients, specialized surgical techniques, clinical and regulatory process, vector production and cGMPmanufacturing, as well as commercial synergies, if these product candidates are approved by the FDA and other regulatory authorities.Our Competitive Advantage: Natural History Studies, Relationships with Leading Institutions and Our cGMP Manufacturing FacilityIRDs as a class are the most common cause of blindness in the working age population worldwide and a leading cause of impaired vision inchildren in developed countries. There are approximately 200,000 people in each of the United States and European Union affected by IRDs. However,IRDs may be caused by mutations in over 200 identified genes, and in many cases each genetically defined IRD may be a small patient population.Meaningful clinical trials for these sorts of rare indications are especially challenging because they require access to sufficient patients and baselinedata on each patient in order to secure clear indicators of efficacy as a result of intervention. We seek to address this problem by sponsoringprospectively designed natural history studies in each of the indications that we are treating in our Phase 1/2 trials. 11Table of ContentsThe lead investigator for the natural history studies is Michel Michaelides, Professor of Ophthalmology at Moorfields Eye Hospital inLondon. For each of the natural history studies, baseline assessments are made upon enrollment, with follow up assessments at six-month intervals fortwo years and annual assessments for up to an additional three years. A broad range of assessments are used, including functional tests, retinal imagingand electrophysiological assessments. The same assessments used for each natural history study are used in our corresponding clinical trial targetingthe same indication, allowing us to compare the impact of our product candidates on the progression of these diseases on a population, as well asindividual patient basis.We expect the natural history studies will enhance our understanding of disease progression for each indication that we are targeting andallow us to identify optimal windows for intervention, provide specific functional and structural parameters to quantify treatment effects and defineclinical endpoints. These studies also provide us with a source of potential patients for our treatment studies and have facilitated efficient enrollment ofthese studies. These patients are not only genotyped, but also have up to five years of detailed functional and structural assessment data prior toenrollment into an appropriate treatment study.We also have longstanding active relationships and clinical site agreements with leading institutions in retinal disorder treatments,including Moorfields Eye Hospital in London, the University of Michigan Kellogg Eye Center, Massachusetts Eye and Ear, the Medical College ofWisconsin & Froedtert Hospital and the Casey Eye Institute at the Oregon Health & Science University. Our relationships with these institutions, inmost cases, precede the date on which we entered into a clinical site agreement with the applicable institution. Professor Robin Ali, Ph.D., our Head ofPreclinical Ophthalmology and one of our founders, is Professor of Human Molecular Genetics at UCL Institute of Ophthalmology and Theme Leaderfor Gene Therapy at NIHR Biomedical Research Centre for Opthalmology, Moorfields Eye Hospital in London, and a Visiting Professor at theUniversity of Michigan Kellogg Eye Center. These institutions are among the premier treatment centers for the indications that we are pursuing andprovide us with access to potential patients for our clinical trials and experts in IRDs who offer strategic guidance and expertise for our developmentstrategy. These institutions provide services with respect to our preclinical and clinical studies. For example, all of our IRD clinical trials are beingconducted at Moorfields Eye Hospital in London, University of Michigan Kellogg Eye Center and Massachusetts Eye and Ear Hospital in Boston.Participants enrolled at the University of Michigan Kellogg Eye Center and Massachusetts Eye and Ear Hospital may travel to the Medical College ofWisconsin & Froedtert Hospital for adaptive optic assessments. The Casey Eye Institute at the Oregon Health & Science University provides certainreading center and other clinical services with respect to our clinical trials. AchromatopsiaDisease Background and Market OpportunityAchromatopsia, or ACHM, is an IRD that specifically prevents cone photoreceptors from functioning. ACHM patients are legally blindfrom birth and usually suffer from severely reduced visual acuity of 20/200 or worse, a disabling sensitivity to light, or photoaversion, total colorblindness and involuntary back and forth eye movements, or nystagmus.Cones and rods are the two kinds of photoreceptors in the human eye. Photoreceptors are light-sensitive cells that absorb light and convertit into an electrical signal that is transmitted to the brain for the perception of light, or vision. Rods can detect very low levels of light and are quicklyinactivated in higher light levels, enabling vision in dim light. Cones, on the other hand, remain active in high light levels and support daytime visionand the perception of color. Even though only 5% of the photoreceptors in the eye are cones, they are concentrated in the central part of the retina, themacula, where they are tightly packed, thus enabling high-acuity daytime vision. The highest cone density is in the centermost spot of the macula,called the fovea. Since cones are essential for central visual acuity, color vision, and most daily visual activities, ACHM patients suffer significantvision loss due to the complete lack of cone function.ACHM occurs in approximately one in 30,000 people in the United States. To date, mutations of any one of six genes encodingcomponents of the light sensing machinery of cone photoreceptors have been 12Table of Contentsidentified as causing ACHM. The CNGB3 and CNGA3 genes are the two most common of these genes, together accounting for up to 92% of ACHMcases, with CNGB3 slightly more common than CNGA3 in most geographic territories. Together, the proteins encoded by the CNGB3 and CNGA3genes make up the cone-specific cyclic nucleotide gated, or CNG, channel, which is essential for cones to produce an electrical signal in response tolight. Mutations in either of these genes prevent the formation of the CNG channel.There are estimated to be approximately 12,000 patients with ACHM caused by mutations in CNGB3 in the United States, Japan andGermany, France, Spain, Italy and the United Kingdom, or the EU5, with about 25% of those patients being under the age of 18 and approximately 125new cases being diagnosed annually. We believe the availability of a therapeutic option may increase patient identification and the estimatedprevalence of ACHM.Our Gene Therapy ProgramWe have designed specifically optimized gene therapy viral vector candidates to treat ACHM caused by mutations in each of CNGB3 andCNGA3, with which we aim to address the majority of patients suffering from ACHM. Our product candidates are delivered via subretinal injectioncovering the central macula region of the eye, where most of the cones in the retina are located.ACHM is predominantly a stationary disease, which means that ACHM patients’ retinas contain non-functioning cones that survive intactfor many decades. This is in contrast to many IRDs in which the entire retina slowly degenerates over a patient’s life. This extended survival of coneswith their potential for light sensitivity presents a wide window of opportunity to introduce a normal copy of the mutated gene via a gene therapyproduct candidate and thereby restore cone function. While the stationary nature of ACHM means that cones remain present for decades, the functionalconnections between active cones and the visual cortex in the brain are thought to become fixed in teenage years. Therefore, we believe that youngerindividuals are likely to benefit most from gene therapy treatment for ACHM because of their greater visual plasticity. Another debilitating symptomof ACHM, which lasts throughout life, is photoaversion. A disabling and ubiquitous symptom of ACHM, photoaversion is the avoidance of light dueto discomfort in the presence of levels of light equivalent to a normally lit room or daylight. ACHM patients often avoid light and wear dark glasses,which further diminishes their already very poor vision. We believe it is possible that restoration of cone function in adult patients might have animpact on photoaversion even if brain plasticity is limited.We believe that gene therapy treatment for ACHM in which we aim to restore cone function via a gene replacement strategy may offerbenefits across a range of ages, which we aim to define in our clinical development programs.ACHM Caused by Mutations in CNGB3With our collaborators at the University College of London’s Institute of Ophthalmology, or UCL IO, led by Professor Robin Ali, we havedeveloped a product candidate to treat ACHM caused by mutations in the CNGB3 gene. Mutations in the CNGB3 gene prevent cone photoreceptorsfrom functioning because CNGB3’s gene product is integral to the formation of a specific membrane channel that enables cones’ electrical response tolight. CNGB3 is a gene exclusively expressed in cones and our aim is to replace the absent function of the mutant CNGB3 gene with a normal copy ofthe gene in cones of IRD patients and thereby restore cone function. In order to drive expression of the functional gene specifically in cones and not inother cells of the retina, we use the cone specific human cone arrestin, or CAR, promoter to drive the expression of a codon optimized CNGB3 cDNA.Codon optimization improves protein expression by increasing translation efficiency. To transfect cone photoreceptors, we use the AAV8 capsid,which enables the efficient delivery of the CNGB3 gene cargo to those photoreceptors. As the vast majority of the cones in the eye are located centrallyand concentrated in the macula, we treat this central region of the retina through subretinal injection to deliver the viral vector product candidate to thephotoreceptors in which its activity is required. 13Table of ContentsPreclinical studies in mice lacking the Cngb3 gene were carried out by our collaborators at UCL IO and led by Professor Robin Ali in 2010and 2011 and the data was published in Human Molecular Genetics in 2011. In these studies, it was observed that delivery of our CNGB3 productcandidate, AAV-CNGB3, via subretinal injection at doses between 6E9 and 8E9 viral genomes per eye, or vg/eye, was associated with a restoration ofcone function to near normal levels.In one of these studies, retinal response to light was measured in three groups of mice: Cngb3 mutant mice treated with AAV-CNGB3,untreated Cngb3 mutant mice and normal mice. Mice were treated with AAV-CNGB3 vector dosed at 2E12 vg/mL (8E9 vg/eye) via subretinalinjection. One eye was treated per mouse. Vector was administered when mice were 30 days old, and electrical responses, or ERGs, of the retina weremeasured 90 days following administration of the vector.Figure 1 shows representative ERG responses of the retina to different light stimuli: a single flash of bright light (1A; 1B; 1C) and repeatedpulses, or flickers, of bright light at different frequencies to assess the refresh rate of the photoreceptors (1A’; 1B’; 1C’). Retinas from three differentgroups of mice were tested: retinas of normal mice (Figure 1A), retinas of mutant mice lacking the Cngb3 gene (Figure 1B) and retinas of mutant micelacking the Cngb3 gene, but treated with AAV-CNGB3 (Figure 1C). It was observed that the response to a bright light pulse was largely absent in theretinas of Cngb3 mutant mice, as this response is largely mediated by the cones and is therefore severely impacted by the Cngb3 mutation (Figure 1B).It was also observed that treatment of Cngb3 mutant mice with AAV-CNGB3 was associated with a high-degree of restored cone function in thesemutant mouse retinas in response to a single flash of bright light (Figure 1C). In addition, we tested the response of cones to rapid flickers of brightlight (Figure 1A1) because only cones can register multiple sequential signals from rapid flickers. It was observed that registering rapid flickers wasalso impacted by the Cngb3 mutation (Figure 1B1) and the cone flicker response was nearly completely restored in the retinas of Cngb3 mutant micefollowing treatment with AAV-CNGB3 (Figure 1C1). Figure 1. Electrical responses of the retina reacting to two different light stimuli: a single flash of bright light and repeat pulses of brightlight at increasing frequency, or flickers.Figure 1A and 1A1. The electrical responses of the retina from a normal mouse reacting to a flash of bright light and flickers.Figure 1B and 1B1. The electrical responses of the retina from a mutant mouse lacking Cngb3 reacting to a flash of bright light andflickers. 14Table of ContentsFigure 1C and 1C1. The electrical responses of the retina from a mutant mouse lacking Cngb3 treated with AAV-CNGB3 reacting to aflash of bright light and flickers. Treatment with AAV-CNGB3 was associated with a high degree of restoration of cone function, with the electricalresponse to a bright flash of light and rapid flickers nearly matching the response observed in the normal mouse retina.In another one of the preclinical studies carried out by our collaborators at UCL IO, it was assessed whether functional improvement at theretinal level of Cngb3 mutant mice was impacted by the age of the mice at the time treatment with the gene therapy product candidate. In this study,Cngb3-mutant mice were treated with AAV-CNGB3 at 30 days old, the P30 group, or 180 days old, the P180 group. One eye was treated in each mousevia subretinal injection dosed at 2E12 vg/mL (8E9 vg/eye). Optomotor measurements were taken 60 days after treatment of the P30 group of the Cngb3mutant mice treated with AAV-CNGB3 (n=11), untreated Cngb3 mutant mice (n=11) and normal, age-matched untreated mice (n=5). Optomotormeasurements were also taken 60 days after the treatment of the P180 group of the Cngb3 mutant mice treated with AAV-CNGB3 (n=6), untreatedCngb3 mutant mice (n=6) and normal, age-matched untreated mice (n=5). Optomoter assessments were made by placing each mouse on a pedestallocated in the center of four inward facing computer screens, that projected a rotating image. The head-tracking responses of each mouse was observedusing an overhead infrared video camera. Visual acuity was measured by involuntary reflex head-tracking responses driven by the left (clockwiserotations, black arrow) and right (counter-clockwise rotations, white arrow) eyes, respectively (Figure 2A).Figure 2B is a graph comparing the visual acuity as measured by the average optomotor assessment of the normal eyes, eyes from Cngb3mutant mice treated with AAV-CNGB3 and eyes from Cngb3-mutant mice that were untreated. It was observed that Cngb3 mutant mice had asignificantly lower visual acuity than normal mice. However, after treatment with AAV-CNGB3, visual acuity in the Cngb3 mutant mice eyes treated at30 days old was observed to be restored to a similar level as that observed in normal mice. In contrast, no significant difference in visual acuitybetween the treated and untreated eyes was observed when treatment was given at 180 days old. Data from this study suggests that treatment receivedat an older age is less capable of restoring visual acuity. Figure 2A. Depiction of how optomotor assessments were taken. Each mouse was placed on a pedestal located in the center of four inwardfacing screens and was observed by an overhead infrared video camera. The assessments were made 60 days following treatment of the Cngb3 mutantmice with AAV-CNGB3 at 30 days old and 180 days old.Figure 2B. Graph showing averaged optomotor assessment of each group. Visual acuity, as measured by the optomotor assessment, wasobserved to be restored to normal levels in the mouse treated at 30 days old (left panel) but not in the mouse treated at 180 days old (right panel).Between October 2016 and December 2016, we carried out a dose-ranging preclinical study at UCL IO to determine the efficacy ofdifferent doses of AAV-CNGB3 in rescuing cone response to a single pulse of bright 15Table of Contentslight in Cngb3 mutant mice as measured by electrical responses across the retina, or ERG. The study was conducted to support a planned Phase 1/2clinical trial in patients with CNGB3 mutations following the FDA’s request for additional rescue data at a range of different doses. While long-termfunctional rescue was observed in prior tests, there was no dosage titration tested in those studies. The data from this study has not been published.We assessed cone-mediated ERG responses in Cngb3 mutant mice at three doses and compared these responses to baseline recordings ofuntreated eyes at two different time-points following treatment. Cngb3 mutant mice were administered AAV-CNGB3 via subretinal injection of 4uL ofvector at each dose. Mice were treated at four weeks of age and ERG responses were measured in these mice at four weeks and eight weeks followingtreatment. Three eyes were treated at each of the three doses, and three eyes were untreated. The reviewer analyzing the ERG traces was masked towhich eye received treatment.Figure 3 shows the photopic ERG amplitudes measured to assess cone photoreceptor function in Cngb3 mutant mice treated withAAV-CNGB3 at 1E11 vg/mL (4E8 vg/eye; n=3), 3E11 vg/mL (1.2E9 vg/eye; n=3) and 1E12 vg/mL (4E9 vg/eye; n=3), and untreated eyes (n=3) at fourand eight weeks post administration. A photopic ERG is the electrical response of the retina corresponding to cone electrical activity following asingle pulse of bright light (single ERG recordings are shown in Figure 1). The higher the ERG amplitude, the greater the response of the retina tobright light. These data show a statistically significant increase in cone ERG responses for all three doses of AAV-CNGB3 and for both time-pointsfollowing vector administration except for low dose at four weeks post injection (p=0.051). These data indicate that AAV-CNGB3 is associated withthe rescue of the cone electrical response in the retina of Cngb3 mutant mice in a dose-responsive fashion. While initial responses were observed inmice four weeks after subretinal injection of the vector, at all three doses significantly stronger ERG responses were observed eight weeks after vectoradministration, with the maximum ERG response observed at the highest dose eight weeks after administration.In this study, a dose response in ERG amplitudes from treated eyes was observed, suggesting that increasing amounts of AAV-CNGB3 isassociated with greater rescue of responses in Cngb3 mutant mice. The three doses tested in this study are the same as those doses administered in ourAAV-CNGB3 Phase 1/2 clinical trial on a vg/mL of viral vector titer basis. 16Table of ContentsFigure 3. Graph of averaged photopic ERG amplitudes at four and eight week time-points post administration of AAV-CNGB in Cngb3mutant mice treated at four weeks of age. The blue line indicates the electrical response amplitudes observed following administration of differentdoses of AAV-CNGB3 measured at four weeks after treatment. The red line shows the increased level of electrical response observed in the same micetreated with AAV-CNGB3 four weeks later, at eight weeks following treatment. This is compared to the ERG response in untreated eyes, whichremained unchanged at both time points.During 2016, we conducted three preclinical toxicology studies of AAV-CNGB3 at UCL IO. We conducted a nine-month study in normalmice in which four eyes were dosed with saline as a control, nine eyes were dosed at 2E9 vg/eye, and nine eyes were dosed at 4E9 vg/eye, with atoxicology assessment at one, three, six and nine months post-administration. We also conducted an eight week study in normal mice, in which 15mice were dosed with saline as a control, 15 mice were dosed at 2E9 vg/eye, and 15 mice were dosed at 4E9 vg/eye, providing five mice from eachgroup for studying local and systemic effects at each time-point of one, four and eight weeks after treatment. We also conducted an eight week rabbitstudy in which the right eyes of nine animals were dosed with saline as a control, the right eyes of nine animals were dosed at 0.8E11 vg/eye and theright eyes of nine animals were dosed at 2.4E11 vg/eye, providing three animals from each dose group for studying local and systemic effects at eachtime-point of one, four and eight weeks after treatment. Biodistribution was examined in the eight-week mouse and rabbit studies. No harmful effectson the retina or systemically were observed at the time-points listed after treatment. The data from these studies have not been published. We usedthese data to support our clinical trial application, or CTA, and IND for treatment of patients with ACHM related to CNGB3 mutations withAAV-CNGB3.Clinical Development of AAV-CNGB3We have an ongoing natural history study in ACHM including over 90 patients that allows us to collect structural and functional data forup to five years on prospectively defined endpoints, including functional tests (visual acuity, contrast sensitivity, mobility maze and photoaversionassessments), retinal imaging (color fundus photography, fundus autofluoresence imaging, spectral domain optical coherence tomography and visualfield 17Table of Contentstesting) and electrophysiological assessments. The study center is the Moorfields Eye Hospital in London. We believe access to these ACHM patientshas enabled us to efficiently enroll the most appropriate patients into our CNGB3 Phase 1/2 clinical trial. We intend to present this natural history datato regulatory agencies for discussion of our pivotal trial design and path to regulatory approval. In addition to giving us access to patients andpotentially accelerated enrollment in our treatment studies, we believe the prospective natural history data on each treated patient allow us to gatherrobust data from our Phase 1/2 clinical trial in a condensed timeframe.We are conducting a Phase 1/2 clinical trial of AAV-CNGB3 in both adult and pediatric patients. In the dose escalation phase of the trial,up to 18 adult patients (18 years or older) may be administered one of three different doses of vector in dose escalating cohorts of three to six patientsat a time. We have treated patients in all three dose cohorts. We have also treated one pediatric patient in the expansion cohort of the trial. We willclose out this trial six months after the last pediatric patient has been treated. Six months following treatment, each patient moves onto a long termfollow up study in which they are followed for safety and indication of benefit for an additional four and a half years.The primary endpoint of this open-label, dose-escalation clinical trial is the safety of a one-time subretinal administration of AAV-CNGB3in patients with ACHM caused by CNGB3 mutations. Secondary endpoints include the outcomes of a range of functional assessments and detailedstructural analysis of the retina, including imaging of individual photoreceptors.This trial is open in the United Kingdom and the United States. We have treated 11 adult patients, including three patients at the highestproposed dose, and nine pediatric patients.We submitted our IND for AAV-CNGB3 to the FDA in the fourth quarter of 2017. The IND was initially not opened because we received aquestion from the FDA regarding our injection device compatibility assay, thus putting the IND on clinical hold until we satisfactorily answered theFDA’s question and the clinical hold was lifted by the FDA. In the device compatibility assay the FDA noted a disparity between the target titer for theintended low dose dilution and the actual titer obtained on polymerase chain reaction, or PCR, analysis. The FDA requested clarification on whetherthis was an imprecise dilution scheme for the low dose or a PCR assay issue. We submitted a response to the FDA on May 2, 2018 providing data thatidentified the issue as a PCR assay artifact and also showing data that we believe supports that this has now been addressed. On May 31, 2018 the FDAreleased the clinical hold on our IND for AAV-CNGB3.In this trial, AAV-CNGB3 viral vector is delivered via subretinal injection of up to 0.5mL targeting the central region of the retina,including the macula and fovea, where most of the cones are located. One eye is treated in each patient, which is the eye assessed to be the worst eye atbaseline. We completed the dose escalation phase of our Phase 1/2 clinical trial, with 11 adult patients treated. These 11 patients were dosed in threecohorts with escalating doses of 1E11 vg/mL (cohort one), 3E11 vg/mL (cohort two) and 1E12 vg/mL (cohort three) of AAV-CNGB3. In addition, ninepediatric patients have now been treated in the extension phase of the study.In each of the 11 adult patients treated and nine pediatric patients treated, the macula, including the fovea, was covered by the subretinalinjection. Subretinal injections have been administered by three different surgeons at the Moorfields Eye Hospital in London and the University ofMichigan Kellogg Eye Center in the United States.In adult and pediatric CNGB3 patients, treatment of the central retina with our AAV-CNGB3 product candidate via subretinal injection hasbeen generally well tolerated. Data from the first adult treatment cohort along with our preclinical data of AAV-CNGB3 was used to support our PRIMEdesignation that was awarded by the EMA in February 2018.As of December 31, 2018, we had completed dosing of eleven adult patients and nine pediatric patients. We may treat up to four additionalpatients in the United States during the first quarter of 2019. We 18Table of Contentsexpect to release data from the adult dose escalation cohorts along with preliminary six-month data from the pediatric patients in the second half of2019.Our CNGB3 gene therapy product candidate, AAV-CNGB3, was granted orphan drug designation by the FDA and EMA, rare pediatricdisease designation by the FDA for the treatment of achromatopsia caused by mutations in the CNGB3 gene, and Fast Track designation by the FDA forthe treatment of achromatopsia caused by CNGB3 mutations to improve visual function. We have also been granted PRIME designation by the EMA.ACHM Caused by Mutations in CNGA3We are also developing AAV-CNGA3 to treat ACHM caused by mutations in the CNGA3 gene. While the mechanism for treating CNGA3related ACHM is similar to that for CNGB3, we believe a larger amount of CNGA3 protein is required to restore cone function compared to CNGB3. Webelieve this may reflect the 1:3 ratio in which CNGB3: CNGA3 subunits associate to form the cone CNG channel. We have designed a syntheticpromoter to drive high levels of CNGA3 expression specifically in cones. In preclinical studies, our novel cone specific promoter has been associatedwith strong gene expression in all human cone subtypes in human retinas grown in vitro using human pluripotent stem cells, or hPSC. AAV-CNGA3utilizes this proprietary pan cone promoter to drive a codon optimized CNGA3 gene sequence. We believe this novel promoter can drive sufficientexpression of CNGA3 in cones to restore light sensitivity to these cones in CNGA3 deficient patients. We use the AAV8 capsid to transfect conephotoreceptors in the back of the eye and we target the cones concentrated in the central region of the retina via a subretinal injection that covers themacula.A key aspect of developing an effective treatment for ACHM caused by CNGA3 mutations is a promoter that drives robust gene expressionin all of the three different cone subtypes, S/blue, L/red and M/green. In particular, it is important to evaluate the activity of the promoter in humanphotoreceptors, which can be achieved prior to treating patients using human stem cell derived retinas. Our relationship with UCL provides access towhat we believe is one of the few centers in the world that can generate human “mini retinas,” or HMRs, which are human retinas grown in vitro fromhPSCs. These HMRs develop from stem cells over several months to form primary photoreceptors arrayed in a spherical structure that resembles manyaspects of the human retina and includes all the rod and cone subtypes, allowing us to assess and predict the utility of many aspects of gene therapyvectors for human clinical application.In vitro studies to measure the gene expression driven by our cone specific promoter in human cones using HMRs were conducted at UCLIO during 2017. Figure 4A shows a fully formed HMR that has been transfected with a construct in which our cone specific promoter was associatedwith the expression of green fluorescent protein, or GFP, a green marker gene. The expression of GFP is seen specifically in cone photoreceptorsthroughout this HMR, indicating that our promoter is driving robust gene expression in human cones. To support that this promoter drives geneexpression in all cone subtypes, GFP expression driven by our promoter was overlaid with markers specific to each of the cone types. Figure 4B is animage of an S/blue cone and Figure 4C is an image of an L/red cone and an M/green cone, with the cone-specific marker labeled in red in each case.The multiple colors in Figures 4B’ and 4C’ show the overlay of these cone-specific markers with cone specific GFP expression, indicating that ourpromoter drives gene expression in all cone subtypes in this HMR. The data from these studies have not been published. 19Table of ContentsFigure 4A. An hPSC derived HMR showing GFP expression (green) driven by our promoter in human cones throughout the HMR.Figure 4B. The red marker identifies this cone as an S/blue cone.Figure 4B’. An overlay of GFP expression from our promotor on top of the S/blue cone specific marker. The overlap between the conemarker and GFP expression indicates that our promoter drives gene expression in S/blue cones.Figure 4C. The red marker identifies M/red and L/green cones.Figure 4C’. An overlay of GFP expression from our promotor on top of the M/Red and L/green cone specific marker. The overlap betweenthe cone marker and GFP expression indicates that our promoter drives gene expression in M/red and L/green cones.In preclinical studies of mutant mice lacking the Cnga3 gene conducted at UCL IO during 2017 and 2018, treatment of retinas of Cnga3mutant mice with AAV-CNGA3 was associated with restoration of the cone electrical response following subretinal delivery. Figure 5 and Figure 6show the rescue of cone function that was observed in two of these studies with doses spanning those proposed in the clinical trial and in a dosedependent manner. We believe this supports the evaluation of AAV-CNGA3 to treat patients with ACHM caused by mutations in CNGA3 in a Phase1/2 clinical trial. The data from these studies have not been published.Figure 5 shows the electrical response of retinas to a single flash of bright light. A retina from three different mice were tested: a normalmouse (Figure 5A), a mutant mouse lacking the Cnga3 gene (Figure 5B) and a mutant mouse lacking the Cnga3 gene, but treated with 3uL of1E12 vg/mL AAV-CNGA3 (Figure 5C). A high degree of restoration of function of mutant cones was observed in the retina from the Cnga3 mutantmouse treated with AAV-CNGA3, with approximately 60% of the electrical response to a bright flash of light of the normal retina observed in the retinafrom the Cnga3 mutant mouse that were treated with AAV-CNGA3. The mice were injected with AAV-CNGA3 at four weeks of age and assessed usingERG four weeks post injection. Figure 5. Cone electrical response in mice retinas reacting to a single flash of bright light, measured by ERG. 20Table of ContentsFigure 5A. The electrical response of the retina from a normal mouse reacting to a flash of bright light.Figure 5B. The electrical response of the retina from a mutant mouse lacking Cnga3 reacting to a flash of bright light.Figure 5C. The electrical response of the retina from a mutant mouse lacking Cnga3 treated with AAV-CNGA3 reacting to a flash of brightlight.Figure 6 shows the cone-mediated ERG responses measured in Cnga3 mutant mice treated with three different doses of AAV-CNGA3. 3uLof AAV-CNGA3 was administered via subretinal injection in each eye at 1E11 vg/mL (n=5), 3E11 vg/mL (n=5) and 1E12 vg/mL (n=5). The mice weretreated at 15 days old and the ERG was measured four weeks after treatment. The reviewer analyzing the ERG responses was masked to which eyereceived treatment. Doses tested in this study span the doses of AAV-CNGA3 proposed for our Phase 1/2 clinical trial. Figure 6 shows the ERGresponses that were observed at each of the three doses. Figure 6. The photopic ERG amplitudes measured to assess cone photoreceptor function in Cnga3 mutant mice treated with AAV-CNGA3at doses of 1E11 vg/mL (n=5), 3E11 vg/mL (n=5) and 1E12 vg/mL (n=5). Mice were treated at 15 days post-natal and ERGs were assessed four weeksfollowing treatment. The retinal ERGs are the electrical recordings corresponding to cone activity following a single pulse of bright light. A doseresponse was observed in retinal sensitivity obtained using the three tested doses and a treatment effect was also observed in the lowest dose tested.In 2018, we completed a toxicology package similar to that used with the AAV-CNGB3 and AAV-RPGR programs using cGMPAAV-CNGA3 material manufactured at our cGMP facility. We conducted an approximately six-month toxicology study in mice and two, eight-weekacute toxicology and biodistribution studies in mice and rabbits.Clinical development of AAV-CNGA3We have an ongoing natural history study in ACHM including over 90 patients that allows us to collect structural and functional data forup to five years on prospectively defined endpoints, including functional tests 21Table of Contents(visual acuity, contrast sensitivity, mobility maze and photoaversion assessments), retinal imaging (color fundus photography, fundus autofluoresenceimaging, spectral domain optical coherence tomography and visual field testing) and electrophysiological assessments. The study center is theMoorfields Eye Hospital in London. We believe access to these ACHM patients will enable us to efficiently enroll appropriate patients into ourCNGA3 Phase 1/2 clinical trial. In addition to giving us access to patients and potentially accelerated enrollment in our treatment study, we believe theprospective natural history data on each treated patient will allow us to gather robust data from our Phase 1/2 clinical trial in a condensed timeframe.We aim to initiate a Phase 1/2 dose escalating trial of AAV-CNGA3 in ACHM patients with mutations in CNGA3. AAV-CNGA3 clinicalmaterial is currently being manufactured at our cGMP facility and we expect to release material for this trial in the first half of 2019 and open a Phase1/2 dose escalation trial in pediatric patients shortly thereafter.We will present pre-clinical data describing our AAV-CNGA3 vector optimization work in a scientific forum in the first half of 2019.X-Linked Retinitis PigmentosaDisease Background and Market OpportunityRetinitis pigmentosa, or RP, is a group of IRDs which represent the most common genetic cause of blindness. The condition ischaracterized by progressive retinal degeneration and vision loss that ends in complete blindness. There are currently no approved treatments for RP.We believe gene therapy is a promising therapeutic approach. RP initially presents as nighttime blindness during childhood or early adulthood,progressing to peripheral visual field loss and “tunnel vision,” central visual impairment, reduced visual acuity and, ultimately, complete blindness.RP may be caused by mutations in any of over 100 different genes. The most severe forms of RP are X-linked, or XLRP, with onset in earlychildhood and rapid progression to blindness generally by the time patients reach 30 to 40 years old. RP has an estimated prevalence of one in 3,500 inthe United States, and between 10% and 15% of RP patients are estimated to suffer from XLRP. The most frequent mutation causing XLRP is in theretinitis pigmentosa GTPase regulator gene, or RPGR. XLRP associated with a mutation in RPGR, or XLRP-RPGR, accounts for more than 70% of casesof XLRP. There are estimated to be approximately 20,000 XLRP-RPGR patients in the United States, Japan and EU5, with a little less than 50% thosepatients being under the age of 40 and approximately 200 new cases being diagnosed annually. We believe the availability of a therapeutic optionmay increase patient identification and the estimated prevalence of XLRP-RPGR.The RPGR protein has an essential role in the maintenance of the structure and function of both rod and cone photoreceptors. The RPGRprotein is tightly localized in the photoreceptors at the junction of the inner and outer segments, or cilium, of the photoreceptors. Correct localizationof the RPGR protein to this site is important for the transport of light sensing proteins to the outer part of the photoreceptors, where these proteinsconvert light signals into electrical signals. In the absence of the RPGR protein the movement of light sensing protein within both rods and cones isimpaired, leading to dysfunction and death of photoreceptors, resulting in retinal degeneration and ultimately complete blindness.Our Gene Therapy ProgramOur product candidate for the treatment of XLRP, AAV-RPGR, is designed to treat XLRP caused by mutations in RPGR, the most commonform of XLRP. The eye specific form of RPGR is encoded by a nucleotide sequence called RPGR open reading frame 15, or RPGR-ORF 15. Both rodsand cones photoreceptors require RPGR-ORF 15 to function. The protein coding message RPGR-ORF 15 contains a region of repeated 22Table of Contentssequences that make the RPGR-ORF 15 nucleotide sequence unstable in the cell. We have engineered a small deletion in RPGR-ORF 15 that weobserved was associated with a stable sequence that rescued RPGR protein levels, localization and function in mouse and human photoreceptors inpreclinical studies. Our novel AAV-RPGR viral vector utilizes the human rhodopsin kinase, or RK, promoter to specifically drive the expression of ourstabilized RPGR-ORF 15 in both rods and cones. We selected the AAV5 capsid because of its efficient transfection into both of these types ofphotoreceptors.In preclinical studies, performed at UCL IO between 2009 and 2015 and discussed in Gene Therapy in 2016, it was observed that treatmentwith AAV-RPGR containing our stabilized RPGR-ORF15 was associated with rescue of the Rpgr mutant phenotype in mice that completely lacked theRPGR protein as measured by ERG responses of retinas from Rpgr mutant mice and assessments of RPGR protein. Restored levels, localization andfunction of RPGR protein in mutant mice lacking the Rpgr gene were observed, along with the restoration and localization of photosensitive proteinsin both rod and cone photoreceptors, and reduction of long-term retinal degeneration. In addition to these experiments in Rpgr mutant mice, we haveobserved that our RPGR-ORF 15 construct also rescued levels, localization and function of RPGR protein in human photoreceptors, in HMRs derivedfrom patients with RPGR mutations. We believe that AAV-RPGR may have the potential to positively impact human photoreceptor function andsurvival in XLRP-RPGR patients, slowing or halting the degeneration that leads to blindness in these patients.Figure 7 and Figure 8 show data from these preclinical studies regarding the effect of subretinal delivery of AAV-RPGR containing ourstabilized RPGR-ORF 15 in mice lacking the Rpgr gene. In these studies, different color stains were used to investigate the restoration of (i) RPGRprotein expression, (ii) correct localization of RPGR protein and (iii) RPGR function. Figure 7 and Figure 8 show a retina from a normal mouse (7A and8A), a retina from a mutant mouse strain lacking the gene for Rpgr (7B and 8B) and a retina from the same mutant mouse strain that lacks the gene forRpgr, but treated with AAV-RPGR containing our stabilized RPGR-ORF 15 (7C and 8C).The red stain in Figure 7 marks a photoreceptor protein, rootletin, within the cilium, or middle section, of every photoreceptor, while thegreen stain reveals the localization of RPGR protein. The green stain in Figure 7A shows the localization of RPGR protein within the photoreceptor atthe end of the cilium in a normal mouse retina. Figure 7B shows a retina from a mouse lacking the Rpgr gene without any green staining, indicating theabsence of the RPGR protein. Figure 7C shows a retina from a mouse lacking the Rpgr gene that was treated with AAV-RPGR containing our stabilizedRPGR-ORF 15. The green staining at the end of the photoreceptor cilium is similar to the normal mouse retina. Treatment with AAV-RPGR containingour stabilized RPGR-ORF 15 was associated with the restoration of RPGR protein expression and localization within the individual photoreceptor cellsconsistent with normal RPGR expression. Figure 7. In these preclinical mouse models, the red staining marks a normal protein in the photoreceptor cilia, rootletin, while the greenstain reveals localization of RPGR protein within the photoreceptor.Figure 7A. The retina from a normal mouse. 23Table of ContentsFigure 7B. The retina from a mouse completely lacking the Rpgr gene.Figure 7C. The retina from a mouse completely lacking the Rpgr gene and treated with AAV-RPGR containing our stabilized RPGR-ORF15. The green stain indicates that RPGR protein expression was restored and was similarly localized within the individual photoreceptor consistentwith normal RPGR expression.Figure 8 shows a different analysis of retinas from the same mutant mouse strain that completely lacks the gene for RPGR expression. Thisanalysis uses a yellow tag to reveal the location of photoreceptor light pigment, or opsins. The top row shows the rod opsin (8A; 8B; 8C), rhodopsin,and the bottom row shows cone opsin (8A1; 8B1; 8C1). Fully functional RPGR is critical for the correct localization of opsins and Figures 8B and 8B1show that in a Rpgr mutant mouse retina the opsins are incorrectly localized. Figures 8C and 8C1 show that treatment of these mutant animals withsubretinal injection of AAV-RPGR containing our stabilized RPGR-ORF 15 enabled the correct location of the opsins within the photoreceptor as seenin the similarity to the normal mouse, indicating that RPGR is functionally active in supporting correct localization of key photoreceptor proteins. Figure 8. In these preclinical mouse experiments, a yellow tag reveals the location of the opsins.Figures 8A and 8A1. Normal mouse retina showing the localization of rhodopsin and opsin.Figures 8B and 8B1. Rpgr mutant mouse retina showing the localization of rhodopsin and opsin.Figures 8C and 8C1. Rpgr mutant mouse retina that was treated with AAV-RPGR containing our stabilized RPGR-ORF 15 shows thelocalization of rhodopsin and opsin. The similar localization between the normal mouse retina and the Rpgr mutant mouse retina that was treated withAAV-RPGR containing our stabilized RPGR-ORF 15 indicates that the rhodopsin and opsin are correctly localized and can perform their key functionin the visual cycle.We are further advancing the HMR system to evaluate the potential for our product candidates to functionally restore mutantphotoreceptor cells in a human retina.One of the many tasks that RPGR performs in the photoreceptor is to enable a process called glutamylation of a key cilia protein calledtubulin, which is required for photoreceptor function. We conducted an in vitro study at UCL IO in 2016 using HMRs derived from stem cells fromXLRP-RPGR patients to determine the effect of AAV-RPGR containing our stabilized RPGR-ORF 15 on human photoreceptors. The data from this 24Table of Contentsstudy has not been published. An HMR grown from hPSC from a normal individual is shown in Figure 9A, in which glutamylation of tubulin in normalhuman photoreceptors is stained in green. Figure 9B shows a HMR derived from a XLRP-RPGR patient and cultured over several months to form manyof the anatomic layers of the retina and consisting of all photoreceptor types, while Figure 9C shows the impact of AAV-RPGR containing ourstabilized RPGR-ORF 15 treatment on a similarly cultured HMR derived from a XLRP-RPGR patient.The HMR derived from a XLRP-RPGR patient lacks RPGR protein, making the photoreceptors dysfunctional with no glutamylation oftubulin present (Figure 9B). When HMRs from a XLRP-RPGR patient were treated with AAV-RPGR containing our stabilized RPGR-ORF 15 weobserved that glutamylation of tubulin returned (Figure 9C). We also stained HMRs derived from an XLRP-RPGR patient with a pink marker of RPGRprotein. In Figure 9B no pink staining was observed, confirming the lack of RPGR protein. In Figure 9C, when the HMR from a XLRP-RPGR patientwas treated with AAV-RPGR containing our stabilized RPGR-ORF 15, pink staining can be seen at the correct localization in the base of the cilium ofthe photoreceptors that are now stained with green glutamylation of tubulin marker. This restoration of RPGR protein expression and localization, andrestoration of markers of photoreceptor function in human photoreceptors in HMRs derived from XLRP-RPGR patients, indicates potential clinicalutility of AAV-RPGR containing our stabilized RPGR-ORF 15. Figure 9. A HMR derived from hPSCs from a normal individual (9A) or a XLRP-RPGR patient (9B and 9C) cultured over several months toform many of the anatomic layers of the retina and consisting of all photoreceptor types with the glutamylation of tubulin having a green fluorescentstain.Figure 9A. A HMR derived from normal hPSCs.Figure 9B. A HMR derived from a XLRP-RPGR patient.Figure 9C. A HMR derived from a XLRP-RPGR patient that was treated with AAV-RPGR containing our stabilized RPGR-ORF 15. Theappearance of pink RPGR protein at the base of the photoreceptor cilium and glutamylation of tubulin returns, demonstrating the restoration of RPGRprotein expression, localization and function.To support the advancement of AAV-RPGR into clinical development, we conducted three single-dose toxicology studies fromJanuary 2016 to August 2017 at UCL IO. We performed a six-month toxicology study in normal mice delivering doses of saline in both eyes for thecontrol (n=5), and doses of AAV-RPGR in both eyes of 2E9 vg/eye (n=5) and 4E9 vg/eye (n=5), with a toxicology assessment at one, three and sixmonths post-administration. Two further toxicology studies were performed. An eight week mouse study in which groups of mice were dosed withsaline in both eyes as a control (n=15), AAV-RPGR in both eyes at 1E9 vg/eye (n=15) or AAV-RPGR in both eyes at 4E9 vg/eye (n=15), providing fivemice from each group for studying local and 25Table of Contentssystemic effects at each time-point of one, four and eight weeks after treatment. An eight-week rabbit study was also conducted, in which nine righteyes were dosed per group, with doses of saline as a control, and AAV-RPGR doses of 0.8E11 vg/eye and 2.4E11 vg/eye, providing three rabbits forstudying local and systemic effects at each time-point of one, four and eight weeks after treatment. Biodistribution was examined in the eight-weekmouse and rabbit studies. No harmful effects on the retina or systemically were observed. We used these data to support our CTA and IND for treatmentof XLRP-RPGR patients with AAV-RPGR.Clinical Development of AAV-RPGRWe have an ongoing natural history study in XLRP-RPGR including approximately 100 patients, which allows us to collect structural andfunctional data for up to five years on prospectively defined endpoints, including functional tests (visual acuity and contrast sensitivity), retinalimaging (color fundus photography, fundus autofluoresence imaging, spectral domain optical coherence tomography, adaptive optics and visual fieldtesting) and electrophysiological assessments. The study centers are the Moorfields Eye Hospital in London, the Kellogg Eye Center at the Universityof Michigan, the Medical College of Wisconsin & Froedtert Hospital and Massachusetts Eye and Ear. We believe access to this large population ofXLRP-RPGR patients has enabled us to efficiently enroll appropriate patients into our XLRP-RPGR Phase 1/2 clinical trial. In addition to giving usaccess to patients and potentially accelerated enrollment in our treatment studies, we believe the prospective natural history data on each treatedpatient will potentially allow us to enroll a more heterogenous population into our treatment studies and may allow us to gather robust data from ourPhase 1/2 clinical trial in a condensed timeframe.RP initially presents as nighttime blindness during childhood or early adulthood, progressing to peripheral visual field loss and “tunnelvision,” central visual impairment, reduced visual acuity and, ultimately, complete blindness. XLRP-RPGR is a progressive disease in which the retinagradually degenerates starting in the outer, or peripheral, regions of the retina and initially causing “tunnel vision” with final degeneration of thecentral retina resulting in the complete loss of visual acuity and blindness that generally occurs by the time patients are 30 to 40 years old. We believethat to prevent this ultimate degeneration to blindness and to retain visual acuity, the central region of the retina, including the macula and fovea, mustbe preserved. To this end, we aim to deliver AAV-RPGR to this central region of the retina. In our Phase 1/2 clinical trial, AAV-RPGR viral vector isdelivered via subretinal injection of up to 1mL with the potential for the surgeon to use multiple retinotomies targeting the region of the central retina,including the macula and fovea. One eye is treated in each patient, which is the eye assessed to be the worst eye at baseline.We are conducting a Phase 1⁄2 clinical trial of AAV-RPGR in both adult and pediatric XLRP-RPGR patients. This trial is open in the UnitedKingdom under our CTA and in the United States under our IND.We have treated ten young adult patients (aged 18 to 30 years old) in the Phase 1/2 XLRP-RPGR clinical trial. We have treated onepediatric patient in the pediatric extension arm of this trial. We expect to treat up to 40 additional patients in the extension cohorts of this study in2019. In all patients, subretinal injection covered the central part of the retina, including the macula and fovea.We expect to report preliminary safety and efficacy data from the dose escalation cohorts in the second half of 2019.The FDA and EMA have granted orphan status to AAV-RPGR for the treatment of retinitis pigmentosa. The FDA has also granted FastTrack designation to AAV-RPGR for the treatment of XLRP caused by defects in RPGR. 26Table of ContentsRPE65-DeficiencyDisease Background and Market OpportunityRPE65-deficiency causes rod photoreceptor dysfunction and impaired vision from birth. The RPE65 gene encodes a protein that isproduced in the retinal pigment epithelium, or RPE, a thin layer of cells at the back of the eye that support photoreceptors and nourish the retina. TheRPE65 protein is essential for rod function because it recycles the light sensing machinery in rod photoreceptors. Absence of RPE65 results in severedysfunction of rods and causes impaired vision in dim lighting conditions. Although cone photoreceptors are generally preserved during childhood inRPE65-deficient patients, the lack of function and degeneration of the rods eventually results in the loss of cones and degeneration of the whole retinaover time. Consequently, most RPE65-deficient patients experience central vision loss progressing to complete blindness by early adulthood.RPE65-deficiency is often characterized as a specific subtype of Leber congenital amaurosis or RP that is caused by mutations in theRPE65 gene, called Leber congenital amaurosis 2 and RP20, respectively. However, we believe a more precise approach to genetic diseases is to focuson the underlying genetic defect, rather than the phenotype, and have therefore identified RPE65-deficiency as the target of our product candidateAAV-RPE65.Based on an estimated prevalence of approximately one in 500,000 people in the United States suffering from Leber congenital amaurosis,or LCA, related to mutations in the RPE65 gene, and approximately one in 70,000 people in the United States having RP due to mutations in theRPE65 gene, RPE65-deficiency occurs in approximately one in 125,000 people in the United States. There are estimated to be approximately 6,000RPE65-deficiency patients in the United States, Japan and EU5, with almost 30% of those patients being under the age of 30 and approximately 50new cases being diagnosed annually. We believe the availability of a therapeutic option may increase patient identification and estimated prevalenceof RPE65-deficiency.Our Gene Therapy ProgramOur gene therapy candidate for the treatment of RPE65-deficiency, AAV-RPE65, is an AAV2/5 viral vector, in which a codon optimizedRPE65 gene is driven by a novel synthetic RPE cell specific promoter.RPE65-deficiency is a well validated condition for gene replacement therapy. In multiple clinical trials, including one conducted by ourcollaborators at UCL, replacing the mutant RPE65 gene with a normal copy of the gene resulted in improved nighttime vision in affected children andyoung adults, suggesting the potential impact of gene therapy on rod function in this disease. The FDA recently approved the first gene treatment forRPE65-deficiency, Luxturna. While RPE65-deficiency primarily causes a loss of rod function initially leading to impaired vision in dim light, thesepatients ultimately experience complete blindness because of degeneration of the cone rich fovea. To prevent blindness, therefore, we believe it iscritical to treat the central retina in order to maintain structural integrity in this region and save central vision. We aim to treat as extensive an area ofthe central retina as possible, including the cone rich fovea. Thus, in addition to improving rod function, we aim to provide sufficient RPE65 protein tothe cells in the central retina to prevent the degeneration of both rods and cones in this region, and thereby prevent the progression to completeblindness.We focus on detailed structural and functional data in our treatment studies with the aim of demonstrating an impact on the degenerationof the central retina.Our novel AAV2/5 vector, AAV-RPE65, has been optimized for both transduction of RPE cells and RPE65 protein production and oursurgical approach targets the central area of retina whose preservation is most critical for long term maintenance of visual function. Building on thework of Professor Ali and in collaboration with the team at UCL, we have developed AAV-RPE65. AAV-RPE65 is a second generation viral vector thathas compared favorably to our first generation AAV2/2 vector in a number of ways, including being two to three logs, or 100 to 1,000 times, morepotent on a particle for particle basis than our original AAV2/2 vector in a head 27Table of Contentsto head Rpe65 null animal model rescue experiment. On a logarithmic scale, each “log” represents a 10-fold change. We believe this increased potencywill improve transgene expression and RPE65 protein production in the back of the eye. The table below summarizes elements of the optimization ofAAV-RPE65 compared to our original AAV2/2 vector. Vector optimization studies were performed at UCL IO and were carried out from 2010 to 2015.The data summarized here was referred to in Gene Therapy in 2016. We used these data to support our IND and CTA for treatment of RPE65-deficientpatients with AAV-RPE65. Optimization of AAV2/5 compared to AAV2/2 Transfection Efficiency Changing the capsid from AAV2/2 to AAV2/5 was associated with an improvement of thetransfection efficiency of RPE by four times. Protein Expression (Promoter) AAV2/5’s optimized promoter was associated with a 20 times increase in protein expression. Protein Expression (Codon) AAV2/5’s optimized codon usage was associated with a seven times increase in proteinexpression in human cells. mRNA Stability Utilizing SV40 intron increases mRNA stability and was associated with a two and one half timesincrease in protein production. Overall Efficacy The optimized construct was associated with a 300 to 1,000 times increase in efficacy in mousefunctional rescue and is expected to be two to three logs more potent on a particle for particlebasis in humans.We conducted four single-dose toxicology and biodistribution studies of AAV-RPE65 from January 2015 to December 2016. Weperformed a long-term toxicology study in normal mice in which four eyes were dosed with saline as a control and nine eyes were dosed withAAV-RPE65 at 4E9 vg/eye, with a toxicology assessment at one, three, six and nine months post-administration. We also conducted an eight-weekmouse study, an eight-week rabbit study and an eight-week minipig study. In the mouse study, 15 mice were dosed in both eyes with saline as acontrol, 15 mice were dosed in both eyes with AAV-RPE65 at 1.2E9 vg/eye and 15 mice were dosed in both eyes with AAV-RPE65 at 3.7E9 vg/eye,providing five mice from each group for studying local and systemic effects at each time-point of one, four and eight weeks after treatment. In therabbit study, nine rabbit’s right eyes were dosed for each group with a saline control group, a 0.6E11 vg/eye group and a 1.9E11 vg/eye group,providing three animals from each group for studying local and systemic effects at each time-point of one, four and eight weeks after treatment. Theminipig study was a two-week single dose injection study in minipigs (n=2, each dosed in one eye) at a dose of 1.84E1011 vg/eye. The minipig studywas performed by Charles River Laboratories, while the other three studies were performed at UCL IO. No harmful effects on the retina were observedduring these studies. We used these data in support of our CTA and IND for treatment of RPE65-deficient patients with AAV-RPE65.Clinical development of AAV-RPE65We have an ongoing natural history study in patients with RPE65-deficiency with approximately 30 patients enrolled that allows us tocollect structural and functional data on prospectively defined endpoints, including functional tests (mobility maze, color vision, visual acuity andquality of life measures), retinal imaging (color fundus photography, fundus autofluoresence imaging, visual field testing, spectral domain opticalcoherence tomography and adaptive optics) and electrophysiological assessments. The study is global, with study centers including at the MoorfieldsEye Hospital in London, University of Michigan Kellogg Eye Center and the Medical College of Wisconsin & Froedtert Hospital. We plan to presentthis natural history data to regulatory agencies in our meetings following completion of the Phase 1/2 clinical trial for discussion of our pivotal trialdesign and path to regulatory approval.This clinical trial enrolled patients in the United Kingdom under our CTA, as well as the United States under our IND. Dosing in the Phase1/2 clinical trial was completed June of 2018. The primary endpoint of this 28Table of Contentsopen-label, dose-escalation clinical trial is the safety of delivering AAV-RPE65 through subretinal injection in patients with RPE65-deficiency.Secondary endpoints include the outcomes of a range of functional tests, detailed structural analysis of the retina and quality of life measures. A totalof 15 patients were treated in this clinical trial, including nine adult patients in three dose escalating cohorts and six pediatric patients in the pediatricextension arm of the trial. Surgeries in this trial have been carried out in the United States at the University of Michigan Kellogg Eye Center and in theUnited Kingdom at the Moorfields Eye Hospital by three different surgeons. No differences in outcomes have been observed between the differentsurgeons.In cohort one, three adults (aged 19, 20 and 20 years old) were administered up to 1mL of 1E11 vg/mL of AAV-RPE65 vector. In eachpatient, the central region of the retina was targeted. No serious adverse events or dose limiting events have been reported in this cohort as of the18-month time point following vector administration.In each patient treated in cohort one, we observed improvement or stability in central visual function following treatment compared tobaseline and compared to the untreated fellow eye. Retinal sensitivity was measured with threshold static perimetry using Octopus 900 perimetry andvisual field modelling and analysis, or VFMA. Observations at 18 months following vector administration in the first cohort have demonstrated apotential increase in retinal sensitivity by up to 40% from baseline. In contrast, we observed that the untreated eyes of patients in this cohort suffered areduction in retinal sensitivity of up to 40%, which is in consistent with data from our parallel prospective natural history study of RPE65-deficientpatients. In addition to the perimetry and VFMA data, improvements in time and error rate in mobility maze navigation have been observed in thesepatients at six and 12 months following treatment. However, this is preliminary data and needs to be supported by data collected at later time-pointsand in larger numbers of patients.In addition to the functional data discussed above, we are collecting detailed structural data on all patients in our Phase 1/2 clinical trial.This includes data to visualize the mosaic of individual cones in the central part of the retina over time in each patient. As of May 4, 2018, we hadsequential cone map data for more than 18 months following subretinal injections that covered the macula and fovea in the patients in cohort one andwe had not observed retinal thinning or loss of cones over time following treatment. We are continuing to monitor the detailed structure of both treatedand untreated fellow eye in the five-year follow on study to assess if AAV-RPE65 treatment has an impact on degeneration in the treated area of the eyecompared to the untreated fellow eye.In cohort two, three adults (aged 16, 20 and 22) were administered vector peripherally via subretinal injection into the outer region of theretina at a higher dose of up to 1mL of 3E11 vg/mL. Following completion of the four-week steroid taper, in two patients inflammatory responses werenoted by the investigator as being probably related to vector administration. Both responded to a further course of steroids. Improvements in visualmobility in study eye over non-study eye were observed at 12 months at the four lux lighting level in this cohort. Similar increases in retinal sensitivityto cohort one have not been observed at this dose level. We believe data from cohort two is suggestive that peripheral administration of the vector intomore degenerating areas of the retina may result in an increased chance of an immune response with a decreased potential benefit compared centraladministration of the vector. Following the review of the safety data for each adult cohort treated in this study, the IDMC recommended escalation tothe higher dose in the next cohort.Based on the safety and activity data from the patients treated at the 1E11 vg/mL dose level (cohort one), the decision was made to treatpediatric patients at this dose prior to receiving the full safety data from adult cohort three, and to target the central part of the retina in all pediatricpatients. Treatment of pediatric patients in the United Kingdom and United States has been completed and six pediatric patients have been treated.We carry out detailed assessments of retinal sensitivity across the entire retina, with the majority of tested retinal locations covering thecentral retina, at baseline and at prospectively defined time-points following 29Table of Contentstreatment using the Octopus 900 perimeter. The Octopus 900 perimeter, unlike other standard clinical devices, enables full-field static perimetry usingtesting algorithms specifically designed for retinal conditions, affords the use of custom-developed testing grids, and allows the full dataset to beextracted and modelled for more accurate measurement of change over time.Discreet small spots of light are shone onto multiple predetermined points (the test grid) covering the central retinal field and peripheralretina, with the patient responding each time they perceive a spot of light. Each point on the retina is tested multiple times with different intensities oflight, thereby allowing the determination of both the reliability and reproducibility of the patients’ response to each light level at each spot and anaccurate measurement of the exact retinal sensitivity at topographically determined points. The data is read out as a ‘heat map’ of sensitivity across theretinal field. The reliability of the patient and the data produced is also calculated and provided by the machine. In addition to the heat map of retinalsensitivity and conventional mean sensitivity metric produced by Octopus 900 assessment, we also conduct advanced VFMA. This produces avolumetric measure of retinal sensitivity presented both numerically as well as in a topographical retinal sensitivity map, which better represents theentire dataset across the visual field. We compare the VFMA data from the retinal map at each timepoint following treatment to the baseline data tomeasure the extent and position of changes in retinal sensitivity over time in treated and untreated eyes.We completed dosing of the final pediatric patients in June of 2018, and we closed out the study six months after the last pediatric patientwas treated. Patients will then move into a long term follow up study in which patients will be followed for safety and an indication of benefit for anadditional four and a half years.We expect to report topline six-month follow up safety and efficacy data in the first half of 2019, with full data expected to be presented ina scientific forum in the second half of 2019.The FDA and EMA each granted orphan status to AAV-RPE65 for the treatment of LCA caused by mutations in RPE65. The FDA alsogranted AAV-RPE65 rare pediatric disease designation for the treatment of inherited retinal dystrophy due to biallelic RPE65 mutations.LCA4Disease BackgroundLCA4 is an IRD that causes complete blindness before age five. AIPL1 is a central protein for the maintenance of photoreceptor structureand function. Deletion of the AIPL1 gene causes the most severe form of early retinal dystrophy, LCA4, in which the retinal structure is destroyed withcomplete vision loss. LCA4 is rare, representing approximately 8% of all LCA cases.There are currently no approved treatments for LCA4, and we believe an effective intervention will require introducing a normal functionalcopy of the AIPL1 gene into rod and cone photoreceptors early in a patient’s life while some retinal structure remains in order to activate function andsurvival of the photoreceptors that are still present. We believe gene therapy has the potential to be the only effective way to address the disease’s rootcause.Our Gene Therapy ProgramLCA4’s extremely rapid progression, rarity and early age of onset make the standard process of seeking regulatory approval throughclinical development challenging because adult safety trials would not yield meaningful data given the early onset of the disease. We believe we arewell placed to initiate the first clinical intervention in this indication through our relationships with UCL and Moorfields Eye Hospital, whoseexpertise and large IRD patient population enables such an aggressive and uncommon IRD to be treated.To address LCA4, we developed a viral vector to replace the AIPL1 gene in all photoreceptors by using the AIPL1 cDNA driven by therhodopsin kinase promoter, which is active in both rods and cones. 30Table of ContentsMuch of the preclinical work to support the proof of concept for gene therapy in LCA4 was conducted by our collaborators at UCL IO, ledby Professor Robin Ali, and was published in Gene Therapy in 2010.Figure 10 and Figure 11 show histological sections revealing the structure of retinas from Aipl1 mutant mice treated with AAV-AIPL1,compared to untreated retinas from Aipl1 mutant mice, at six months and 23 months after treatment.It was observed at both time-points that Aipl1 mutant mice treated with AAV-AIPL1 suffered less photoreceptor loss than untreated Aipl1mutant mice, with the retinas of untreated mutant mice almost completely degenerated by 24 months of age. Aipl1 mutant mice were treated with.5uLof AAV-AIPL1 at 1E12 at four weeks post-natal and eyes were sectioned at six months and 23 months after treatment. These treated retinas werecompared to retinas from untreated control Aipl1 mice (n=17 eyes), and retinas from normal mice without the Aipl1 mutation sectioned at the samepost-natal ages as the treated Aipl mutant mice. The data indicates that subretinal administration of AAV-AIPL1 is associated with the protection of theretina from degradation that occurs in Aipl1 mutant mice, and this effect was durable out to two years, the oldest age studied in these mice. Figure 10. Histological sections of retinas of Aipl1 mutant mice treated with AAV-AIPL1 six months after treatment with AAV-AIPL1,compared to untreated retina. The top image is the retina of an untreated Aipl1 mutant mouse at seven months post-natal. The lower image is the retinaof a Aipl1 mutant mouse at seven months post-natal, and six months following treatment with AAV-AIPL1. Figure 11. Histological sections of retinas of Aipl1 mutant mice treated with AAV-AIPL1 23 months after treatment with AAV-AIPL1,compared to untreated retina and retina from a normal mouse. The left image 31Table of Contentsis the retina of an normal mouse at 24 months post-natal. The middle image is the retina of an Aipl1 mutant mouse at 24 months post-natal, and 23months following treatment with AAV-AIPL1. The right image is the retina of an untreated Aipl1 mutant mouse at 24 months post-natal.Clinical material was manufactured to cGMP standards under a specials license. Some of this clinical material was used in studies in 2017at UCL to confirm potency in an AIPL1 deficient mouse and to perform toxicology testing in normal mice and rabbits. A potency study was conductedat a AAV-AIPL1 dose of 3E9 vg/eye with a group size of three eyes. Assessments were performed at three weeks and supported the potency of the cGMPclinical material through analysis of the ERG response.A toxicology study was performed with normal mice and rabbits. In the mouse portion, five animals per group were evaluated for local andsystemic effects at four weeks. In the rabbit portion, three animals per group were evaluated for local and systemic effects at four and eight weeks.Safety evaluation included functional assessments using ERG, fundoscopy, and structural assessments.Our product candidate, AAV-AIPL1, was manufactured and released for compassionate use under a specials license in the UK to treat LCA4patients at the Moorfields Eye Hospital. This allows physicians at the Moorfields Eye Hospital to prescribe a treatment of AAV-AIPL1 for LCA4patients they deem appropriate. We play no role in the physician’s treatment decision. We intend to use the data produced by the compassionate usetreatment to inform any potential clinical development plan as well as any interactions with the regulatory agencies that would enable us to make thisintervention more widely available to the LCA4 patient population. Although patients have been reviewed at the Moorfields Eye Hospital, so far nonehave been treated with AAV-AIPL1. As the manufacturer of AAV-AIPL1 under a specials license, we have a record retention requirement and acontinuing obligation to inform the MHRA of any suspected adverse reaction to our medicinal product which is a serious adverse reaction.The UK’s Human Medicines Regulations 2012 allow for the manufacture and supply of medicinal products not authorized for marketing topatients with special needs at the request of the healthcare professional responsible for the patient’s care (these products are referred to as “specials”). Aspecial may only be supplied in: (i) response to an unsolicited order from a healthcare professional responsible for the care of the patient, (ii) if theproduct is manufactured and assembled in accordance with the specifications of that healthcare professional to fulfil the special needs of the individualpatient that cannot be met by products already authorized for marketing and (iii) if the product is manufactured under a specials license granted by theUK’s Medicines and Healthcare Products Regulatory Agency, or MHRA.Manufacturing a special also imposes a five year record retention requirements subject to review by the MHRA, including details of anysuspected adverse reaction to the product so sold or supplied of which the person is aware or subsequently becomes aware, as well as a continuingobligation to notify the MHRA of any suspected adverse reaction to the medicinal product which is a serious adverse reaction.The FDA and EMA granted orphan designation to our product candidate, AAV-AIPL1, for treatment of inherited retina dystrophy due todefects in AIPL1 gene.Age-Related Macular Degeneration (AMD)Disease Background and Market OpportunityAMD is a chronic eye condition that causes gradual decline of central vision. It is the leading cause of vision loss in individuals more thanfifty years old in the United States and it affects 6.2 million people globally. Although AMD does not usually lead to complete blindness becauseperipheral vision is generally maintained, losing central vision has a critical impact on visual acuity, which is achieved by the closely packed cones ofthe central retina. As a result, loss of central retinal function has a significant impact on a person’s independence and quality of life. 32Table of ContentsAMD is a complex disease attributed to several different causes. A number of genetic and environmental risk factors have been associatedwith the development of AMD. Two late forms of AMD have been characterized: geographic atrophy, or dry AMD, and neovascular AMD, or wetAMD. Dry AMD is characterized by the progressive, irreversible loss of many of the cell types in the retina, including the RPE, photoreceptors, andunderlying capillaries of the macula, and causes a decline in central visual function. Wet AMD is defined by the growth and invasion of immatureblood vessels from the underlying choroid into the retina. Leakage from these fragile blood vessels causes build-up of blood and fluid under the retina,leading to detachment of the RPE or retina and long-term scarring.Current therapeutic options for AMD are limited. There is no approved therapy that impacts the disease progression of dry AMD. The bestavailable treatment for patients after they lose central vision and acuity is support and rehabilitation services to help them better utilize the remainingperipheral part of their retina.Effective treatments for wet AMD are focused on blocking blood vessel growth, or angiogeneisis, that underlies the pathology of thedisease. These wet AMD treatments specifically block the activity of vascular endothelial growth factor, or VEGF, one of the key drivers ofangiogenesis. There are two approved anti-VEGF treatments on the market, ranibizumab, or Lucentis, and aflibercept, or Eyelea. These may be effectivein some wet AMD patients, but require challenging dosing regimens that typically include intra-ocular injections every one to three months.Our Gene Therapy ProgramsWet AMDWe aim to use a gene-based strategy to deliver an anti-angiogenic molecule to the retina and create a “local factory” that will deliver acontinual supply of antiangiogenic molecules to the key parts of the eye. This would require a one-time injection procedure to deliver the gene therapyto the affected eye. We focus our blockade on the receptor involved in driving aberrant blood vessel growth and leakage, VEGF Receptor 2, orVEGFR2. Rather than depleting the VEGF ligand from the system we aim to block the receptor for that ligand preventing VEGF binding to the receptorand thereby preventing downstream pathology of wet AMD that is normally triggered by VEGF binding to VEGFR2. We use an anti-VEGFR2antibody. In some rodent models the inhibition of the VEGFR2 with this antibody appears to have reduced long term toxicity as compared to thedepletion of the VEGF ligand. Initial studies have identified a lead candidate that we have now moved from the research stage to formal preclinicaldevelopment. We are also working to integrate our gene regulation technology with our wet AMD program. Our ultimate aim is to activate the anti-VEGFR2 antibody gene that we have installed in the eye with a small molecule applied topically with an eye drop. This could potentially allow forintermittent dosing of the antibody using an eye drop rather than an injection.Dry AMD—Rod to Cone ProgramTo treat dry AMD, and specifically to treat patients with geographic atrophy in which the central part of the retina is irreversibly damaged,we are advancing a novel strategy to alter the very character of rod photoreceptors.In advanced forms of dry AMD, central visual function is lost after the fovea and the larger central retina area has degenerated, and anyresidual vision is generally via the outer retina’s rods. A critical characteristic of cones that allows for response to high light levels is that they recoverrapidly after they are stimulated by light. In contrast to cones, rods work in low light levels and require longer exposure to trigger an impulse and arethen much slower to recover to a state when they can respond again. Therefore, seeing via rods is slow and imprecise, whereas cones mediate acutehigh-resolution vision. By genetically engineering rods with molecules that will improve their speed of recovery and response to light, we aim toeffectively transform a patch of rod photoreceptors to behave more like cones. This rod-to-cone transformation would potentially be achieved 33Table of Contentsby using localized gene therapy vector placement in the rod-rich peripheral retina to create a small patch or “pseudo fovea,” where cone like behaviorof rods would enable the patient’s brain to fixate on a functional part of the peripheral retina and recover a more cone like response to higher lightlevels.The proof of concept has already been achieved in animal models, with first generation molecules designed to speed up the visual cyclerecovery times in rods. We will continue to develop and optimize these molecules using our screening systems to allow selection for sensitivity tonatural high light intensities and for the high refresh rate required.XerostomiaDisease Background and Market Opportunity—RIXRadiation induced xerostomia, or RIX, is a severe and debilitating long-term side effect of radiation treatment for head and neck cancer.There is not currently any FDA approved treatment for RIX. Worldwide, there are approximately 500,000 new cases of head and neck cancer diagnosedeach year, with approximately 50,000 cases in the United States alone, making it the fifth most common malignancy. Approximately 85% of patientswho receive radiation treatment for head and neck cancer experience reduced saliva production during treatment, and approximately 40% of thosepatients who remain cancer free for two or more years after treatment continue to suffer from grade 2 or 3 RIX. There are approximately 170,000 suchpatients in the United States, with approximately 10,000 new cases each year. Severity of RIX is measured from grade 1 (mildest) to 4 (most severe). Asthese patients tend to be under the care of a physician and dentist and have access to some form of health insurance, we believe this is an accessibleindication with much larger commercial potential than some genetic diseases that are currently targets of our gene therapy programs.RIX is a frequent side effect of head and neck cancer treatment because the standard treatment of locally advanced head and neck cancerinvolves radiotherapy, which can cause irreversible damage to non-diseased tissues located near oral tumors, such as the salivary glands. The fluidsecreting, or acinar cells, of the salivary glands are uniquely sensitive to radiation, are destroyed acutely on IR exposure and to a large extent do notregenerate, resulting in chronically reduced salivary output. Because saliva plays such a critical role in the physiology and protection of uppergastrointestinal, or GI, tract tissues, patients with chronic RIX suffer severe long-term complications of head and neck irradiation that has a significantimpact on the patient’s daily living. Chronic RIX results in severe side effects, including difficulty swallowing, or dysphagia, oral discomfort,malnutrition, oral mucositis, changes in taste, increased oral infections and dental cavities.Our Gene Therapy Program—RIXSalivary glands are an attractive target organ for gene therapy treatments because they are self-contained, partially immune protected andeasily accessible, allowing for non-invasive delivery of small vector doses.We are developing AAV-AQP1 to treat RIX by increasing water conduction in the chronically damaged salivary glands by introducing awater conducting channel into the remaining epithelial cells of these damaged glands. Adequate water secretion by surviving epithelial cells has thepotential to deliver the protective exocrine proteins produced by remaining gland cells into the mouth.The key to our approach is that, unlike the water conducting acinar cells, the water impermeable duct cells of the glands appear to beresilient to IR exposure. As a consequence of this relative resilience to radiation treatment, salivary glands damaged by radiation treatment tend tocontain mostly water impermeable ductal epithelial cells. To make these duct cells permeable to water, AAV-AQP1 introduces the gene for the humanaquaporin water channel, or hAQP1. We have demonstrated that this has the potential to convey water permeability and causes ductal cells to generatean osmotic gradient, which causes them to secrete fluid into the lumen of the duct. 34Table of ContentsThe proof of concept for this mechanism and its ability to increase the volume of saliva secreted by damaged salivary glands was observedin a Phase 1/2 clinical trial conducted by the NIH in patients with chronic grade 2 or 3 RIX. The trial was designed as a short-term dose escalation trialof doses of AAV-AQP1 between 4.8E7 and 5.8E9 pu/gland using adenovirus as the vector to deliver the hAQP1 to the remaining epithelial cells in theparotid gland of 11 patients suffering from chronic RIX. There were no reported severe adverse events among the patients treated, two out of threepatients in each of the first three cohorts in this clinical trial were observed to have objective increases in saliva volume produced by the treatedparotid gland, with increases in parotid flow ranging from 60% to 540%, and all but one of these patients showed a decrease in symptoms of dry mouthas measured by subjective visual analog scales, validated in other forms of xerostomia. The results of this study were published in Proceedings of theNational Academy of Sciences in 2012.We are currently conducting a Phase 1 dose escalation clinical trial in patients with grade 2 or 3 RIX who remain cancer free for at least fiveyears after receiving radiation treatment. In this trial we are using AAV2 to deliver the hAQP1 gene, as we believe AAV2 efficiently transfects thesalivary gland cells and does not spread beyond the target cells. Up to 18 adult patients may be administered doses of AAV-AQP1 between 3E9 and6E11 viral particles per gland in dose escalation cohorts of three patients each. The aim of the trial is to determine the safety of inserting hAQP1 locallyinto the salivary glands of RIX patients, as well as to measure changes in salivary flow resulting from the introduction of this channel. We havecompleted dosing in the first cohort and second cohort, having treated six patients. This clinical trial is being conducted in conjunction with theNational Institute of Dental and Craniofacial Research at the NIH Dental Clinic.We expect to initiate an additional clinical trial at Memorial Sloan Kettering Cancer Center and up to five additional clinical sites in theUnited States in 2019.The FDA granted orphan drug designation for AAV-AQP1 to treat symptoms of grade 2 and grade 3 late xerostomia from parotid glandhypofunction caused by radiotherapy for cancer of the oral cavity.Sjogren’s SyndromeThe destruction of salivary tissue resulting in chronic xerostomia may also be caused by chronic autoimmune disease. Sjogren’s syndromeis an autoimmune disease in which a patient’s immune system may target the salivary glands. Chronic inflammation of the salivary glands results inlong term damage and chronic xerostomia in many Sjogren’s patients. Data from preclinical studies in animal models of Sjogren’s syndrome and datafrom explants of minor salivary glands of Sjogren’s patients suggest that Sjogren’s syndrome may also be treatable with our AAV-AQP1 vector.Supported by data from our preclinical studies and our ongoing RIX clinical trials, we are currently conducting IND-enabling studies of AAV-AQP1 forxerostomia caused by Sjogren’s syndrome.Neurodegenerative DiseasesWe also have clinical stage, research and preclinical programs targeting neurodegenerative diseases, with a clinical program in Parkinson’sdisease and a research focus in ALS and Alzheimer’s disease. In ALS and Alzheimer’s disease, we aim to target what we believe is a central factor in theunderlying cell biology of the diseases - RNA metabolism in ALS and endosomal trafficking dysfunction in Alzheimer’s disease. Relying on ourexpertise in viral vector design, delivery, production and manufacturing, we are aiming to develop and optimize vectors to effectively treat bothgenetic and sporadic forms of these diseases.Parkinson’s DiseaseDisease Background and Market OpportunityAffecting nearly one million Americans and 10 million worldwide, Parkinson’s disease is the second-most common neurodegenerativedisease after Alzheimer’s disease and is the 14th-leading cause of death in the 35Table of ContentsUnited States. It is associated with a progressive loss of motor control (e.g., shaking or tremor at rest and lack of facial expression), as well as non-motorsymptoms (e.g., depression and anxiety). There is no cure for Parkinson’s disease and 60,000 new cases are diagnosed each year in the United Statesalone.Our Gene Therapy ApproachAAV-GAD is an investigational gene therapy product candidate designed to deliver the glutamic acid decarboxylase, or GAD, gene to thesubthalamic nucleus in order to increase production of GABA, the primary inhibitory neurotransmitter in the human brain. GAD is the rate-limitingenzyme in the synthesis of GABA, therefore it is believed that increasing subthalamic nucleus GAD expression through gene therapy has the potentialto result in normalization of motor circuits and improve symptoms in Parkinson’s disease patients without affecting other brain regions, which can beresponsible for complications of existing therapies.AAV-GAD Clinical ResultsIn a blinded Phase 2 clinical trial of AAV-GAD in patients with medically refractory Parkinson’s disease, 45 patients were randomized 1:1to receive either AAV-GAD gene therapy delivered by injection into the subthalamic nucleus on both sides of the brain or bilateral sham surgery.Subjects were followed for one year and all results remained blinded until the final treated patient reached the 6-month primary endpoint. The trial metthe primary endpoint, with a significant improvement in the off-medication motor section of the Unified Parkinson’s Disease Rating Scale, or UPDRS,part 3 observed in treated patients compared to baseline. There was also a significant difference in the degree of improvement observed compared withpatients in the sham arm. The primary outcome measure was the six-month change from baseline in double-blind assessment of off-medication UPDRSmotor scores. At the six-month endpoint, UPDRS score for the AAV-GAD group decreased by 8.1 points (SD 1.7, 23.1%; p<0.0001) and by 4.7 pointsin the sham group (1.5, 12.7%; p=0.003). The AAV-GAD group showed a significantly greater improvement from baseline in UPDRS scores comparedwith the sham group over the six-month course of the study (RMANOVA, p=0.04). Significant difference in the response rate between groups wereobserved, with responders being defined as patients achieving a 9-point or more improvement in UPDRS, which may be deemed clinically meaningful.At six months, 50% of AAV-GAD treated patients were responders compared with only 14% of patients in the sham arm. At 12 months, response rateswere 63% and 24%, in AAV-GAD and sham arms, respectively. An improvement in complications of medical therapy as measured by the UPDRS part 4was observed in the AAV-GAD group at both six and 12 months. A significant decline in duration of disabling dyskinesia was observed only in theAAV-GAD treated patients.AAV-GAD was reported to be well-tolerated, with no significant adverse events related to the therapy and no speech or cognitivecomplications observed. The most commonly reported adverse events were transient mild or moderate headache (seven in treated arm vs. two in shamarm), nausea (six in treated arm vs. two in sham arm) and worsening of Parkinson’s disease (zero in treated arm vs. four in sham arm). The results of thetrial were published in the March 2011 issue of The Lancet Neurology, the August 2014 issue of the Journal of Clinical Investigation and theApril 2017 issue of JCI Insight, building upon publications of the Phase 1 trial data in The Lancet and the Proceedings of the National Academy ofSciences. In addition, in research published in the November 28, 2018 issue of Science Translational Medicine, fifteen patients treated with AAV-GADgene therapy were observed to have expressed a treatment-related reorganization of functional brain connectivity that was related to disease symptomimprovement. This flurodeoxyglucose positron emission tomography analyses provided objective biological evidence of improvements in abnormalbrain networks associated with Parkinson’s disease following AAV-GAD gene therapy.These results were observed in patients treated in both Phase 1 and Phase 2 studies. Blinded analyses showed significant improvements inabnormal thalamic metabolism, a key node in the movement circuitry, in the AAV-GAD treated patients. This pattern of brain network activity was notseen in untreated hemispheres or patients in the sham arm. Furthermore, a specific pattern of brain network activity was identified in those 36Table of Contentssubjects with clinical improvements in the sham arm, which was different from the pattern observed in AAV-GAD responders.ALSDisease Background and Market OpportunityALS is a devastating, progressive, neurodegenerative disease leading to the loss of motor neurons, which are the neurons that control theability to move, speak, swallow and ultimately to breathe. The gradual paralysis in ALS invariably leads to death. While 10% of ALS cases are causedby inherited genetic mutations, most ALS occurs sporadically, with no known genetic cause. Mutations in over 20 genes have been identified thatcause the inherited ALS cases. Characterization of these disease-causing genes have implicated several cellular pathways in the disease, with aprominent role emerging for genes involved in the cellular control of RNA. Many new regulatory roles are being discovered for RNA, particularly inneurons.One gene linked to ALS is the transactive response DNA-binding protein 43, or TDP-43. The TDP-43 protein is one of a number of proteinsthat binds to cellular RNA and regulates its function and stability. When TDP-43 does not function normally, RNA regulation in the neurons loses itstight control and the motor neurons die. While mutations in TDP-43 account for only about 0.5% of ALS cases, aggregates of TDP-43 protein areobserved in motor neurons of more than 95% of all ALS patients, including both inherited and sporadic, and TDP-43 aggregation is a central feature ofdiseased motor neurons in ALS patients. TDP-43, therefore, may be a critical therapeutic target for the treatment of both sporadic and inherited forms ofALS. TDP-43 aggregates are also seen in approximately 50% of patients with frontotemporal dementia, or FTD, suggesting a potentially similarunderlying mechanism of this neurodegenerative disease.Our Gene Therapy ApproachGenetic approaches using yeast as the primary model system have been used to identify ways of protecting motor neurons from death inALS. A genetic screen was conducted to look for activated genes that rescue the toxicity of TDP-43 aggregation. A group of fives genes that reduceTDP-43 toxicity, was identified including members of the nonsense mediated decay, or NMD, machinery, and two genes in particular: up frameshiftprotein 1, or UPF1, and up frameshift protein 2, or UPF2.NMD is the quality control system regulating RNA processing and activity in all cells, and has additional and broader functionsspecifically in regulating RNA in neurons.Over the past decade, increasing evidence suggests that motor neurons may be particularly vulnerable to defects in RNA regulation, whichmay be caused by a number of defects, including TDP-43 aggregation. We believe that increasing the master quality control machinery for RNAprocessing, NMD, in motor neurons may improve the ability of motor neurons to survive in ALS.We have designed a viral vector product candidate, AAV-UPF1, with the aim of increasing UPF1 expression in the motor neurons of ALSpatients. In preclinical studies, we observed that administration of AAV-UPF1 reduced motor neuron death thought to be driven by the toxic effects ofseveral different genetic causes of ALS including, TDP-43, FUS and C9orf72. Improvements in ALS-like symptoms related to limb strength andmobility in rodent models of ALS have also been observed following administration of AAV-UPF1.We believe that gene therapy using AAV-UPF1 may increase UPF1 levels in cells affected by ALS, and we intend to deliver our viralvector product candidate to the central nervous system via intrathecal injection, or injection into the spinal canal. We are currently conducting INDenabling studies in animal models and have observed the preservation of neurons and a positive impact on functional endpoints in Fus and TDP-43neuronal cell cultures. Based on data from our non-human primate studies conducted in 2018, we have identified a 37Table of Contentsproprietary capsid for use in our AAV-UPF1 vector. We anticipate completing pre-IND activity in 2019 and aim to file an IND in the second half of2019. We expect that AAV-UPF1 clinical material for the Phase 1/2 clinical trial will be manufactured at our cGMP manufacturing facility.Alzheimer’s DiseaseDisease Background and Market OpportunityWith the world population aging, Alzheimer’s disease has emerged as an extremely common and costly disease. While some treatmentsthat have temporary effects on Alzheimer’s disease symptoms are available, there is currently no approved treatment that halts the progression of thedisease. Two biological pathways have been identified that are considered causes of Alzheimer’s disease. They are identified as causative becausemutations in genes that affect these pathways alone cause patients to develop the disease. The first causative pathway, identified in rare forms of earlyonset Alzheimer’s disease, is the misprocessing of amyloid precursor protein, or APP, caused by genetic defects in APP itself and the APP processingproteins presenilin 1 and 2. The second causative pathway is related to the correct movement, or trafficking, of cellular protein which is controlled by acell component called the endosome. Loss of function mutations in the endosomal protein sortilin-related receptor 1, or SORL1, have recently beenshown to cause Alzheimer’s disease.The endosome is an organelle within the cell that is responsible for correctly directing protein trafficking to different sites within the cell aswell as to the cell surface. In neurons, there is significant protein trafficking, particularly at dendrites or neuronal connections where the endosomaltrafficking system regulates the movement of the cell signaling machinery that allows neurons to communicate with one another.The molecular machine that drives endosomal protein trafficking is made up of a complex of proteins together called the retromer. Theprotein encoded by the Alzheimer’s disease causing gene SORL1 is a retromer component. SORL1 and other components of the retromer complex arecommonly deficient in Alzheimer’s disease patients’ brains. Defects in the endosomal trafficking pathway have also been implicated in otherneurodegenerative diseases, including Parkinson’s disease, ALS and frontotemporal dementia.Our Gene Therapy ApproachOur Alzheimer’s disease program focuses on the endosomal trafficking pathway. In preclinical studies, we observed that increasing levelsof key retromer proteins may reverse endosomal trafficking defects. We are identifying suitable retromer targets for gene augmentation inpre-symptomatic Alzheimer’s patients.There are several reasons why gene therapy is, in principle, well suited for Alzheimer’s disease and other neurodegenerative diseases. Thefirst relates to the pathophysiology, time course, and anatomical spread of these disorders. Neurodegenerative diseases generally begin locally inselectively vulnerable regions with “cell sickness” years before rampant cell death and wide-spread anatomical distribution. To be most effective, webelieve interventions should be administrated early and will benefit from local delivery. Even then, however, an intervention must maintain its efficacyfor years because, unlike other cells in the body, neurons do not typically divide over the course of their life. We believe AAV-delivered gene therapyproducts may have a durable effect. In the best case scenario, one delivery successfully taken up by targeted neurons would be sufficient for years ofefficacy.An important component of our approach is the development and validation of surrogate markers of endosomal dysfunction and predictivemarkers of Alzheimer’s disease. In particular, several well studied biomarkers linked to Alzheimer’s disease, such as amyloid-beta and tau, have alsobeen shown to be biomarkers of endosomal trafficking dysfunction in neurons. Such biomarkers could potentially be used to identify patients withAlzheimer’s disease, as well as demonstrate potential product efficacy in the absence of Alzheimer’s disease symptoms. By targeting endosomaltrafficking dysregulation we aim to address the underlying cause of Alzheimer’s disease as well as other neurodegenerative diseases, such as certainforms of Parkinson’s disease. 38Table of ContentsOur Gene Regulation PlatformWe are developing a potentially transformative technology designed to enable us to use small molecules to turn gene therapy productcandidates on and off. The aim of this gene regulation platform is to transform gene therapy into a generalizable mechanism for the delivery of biologicdrugs. The idea is that the gene encoding a particular biologic drug, for example epoetin alfa, or a therapeutic antibody, would be delivered to targetcells in the body, but these genes would only be activated in the presence of a specific small molecule. The therapeutic protein would be manufacturedby the body only in the presence of the small molecule so that intermittent production of the therapeutic protein would be achieved by dosing with thesmall molecule drug.This temporal regulation of gene therapy products by exogenous small molecules has long been a goal of gene therapy researchers. Theability to regulate transgenes by introducing temporal control has the potential to transform the gene therapy landscape and the biologics industry as awhole. Our approach focuses on riboswitches to regulate gene expression rather than on the modulation of transcription factor activity, and this is thebasis of our gene regulation platform.Riboswitches are pieces of RNA that fold into alternative shapes depending on the binding of a specific small molecule to that RNAsequence. One RNA shape allows the gene containing the riboswitch to be active, while the alternative shape inactivates the gene. Riboswitches areused extensively by bacteria, but none have been identified in mammalian cells to date.We designed de-novo mammalian riboswitches that we have observed respond to small molecules to switch genes on and off in mammaliancells and in vivo in mice. Our riboswitch contains a stretch of RNA sequence, called an aptamer, that binds to a specific small molecule. The riboswitchis inserted into the therapeutic transgene cDNA. In the absence of the specific small molecule, the unbound riboswitch folds into the shape that drivesthe destruction of the RNA message and no therapeutic protein is produced in the absence of the small molecule. However, when the small molecule ispresent and binds to the riboswitch it adopts the alternative RNA shape, causing stable messages to be formed and the therapeutic protein to beproduced.One of the features of our mammalian riboswitch is its range of regulation. Using a small molecule we were able to switch the riboswitchcontaining gene on to levels greater than 1,000x higher than in the absence of the small molecule. We believe this technology is viable for atherapeutic product and is also the first instance of a proprietary system for screening randomized aptamers and small molecules within mammaliancells for functional interactions.Our Manufacturing CapabilitiesWe own and operate a cGMP manufacturing facility situated in London, United Kingdom. Supporting our global approach to clinicaldevelopment and market supply, we designed the 29,000 square foot facility to meet multiple regulatory standards, including the Medicines andHealth Products Regulatory Agency, or MHRA, in the UK, EMA and FDA standards. In the first quarter of 2018, we had our final MHRA certificationinspection. In May 2018, we were granted a license to manufacture gene therapy product candidates in our cGMP compliant manufacturing facility bythe UK Medicines and Healthcare products Regulatory Agency.We believe our facility can supply all of our current clinical and preclinical programs through regulatory approval and, should they beapproved, provide sufficient capacity, for commercial production. Strategically, we believe our facility will minimize our dependence on third-partyCMOs, which we believe provides a significant strategic, clinical and commercial advantage.Our facility is flexible and scalable, with eleven independent air handling units, two cell culture suites and three separate viral vectorproduction suites, which allows us to produce multiple product candidates in parallel, as well as sequentially at different scales. This allows us toaccommodate up to three independent parallel manufacturing streams of viral vector products that are isolated within dedicated production areas. 39Table of ContentsOur manufacturing facility includes an integrated analytical department and in-house analytical tool kit that allows for in-house release ofclinical and commercial manufactured products. Equipped with dedicated areas for microbiology, molecular biology, and cell-based analytics. Ouranalytical department can perform product related assays, allowing us to retain and gain expertise that is normally lost to third parties. The closeintegration allows for rapid turnaround and flexibility in scheduling of key assays, reducing lead times for product candidate releases. Further, ourdedicated product fill and finish suite allows us to manufacture a full range of clinical and commercial products under one roof and in our control.We have more than 40 highly trained multidisciplinary staff on our manufacturing team with backgrounds in manufacturing, managingand delivering gene therapy products.We have identified and licensed a proprietary HEK293 cell line that is well characterized and that we have banked in 400 vials. Thespecific cell line, size of the bank, culture media, and cryopreservation agents have been selected to facilitate bridging between process developmentplatforms and targets. Our HEK-293 cells lack the T antigen component and are suitable for both the current adherent culture platform and thebioreactor process. We believe the ability to use the same cell line throughout the product and process development lifecycle will allow us to use abracketed approach to process validation and comparability, which we believe may reduce the time and costs related to their implementation.CompetitionThe biotechnology and pharmaceutical industries are characterized by rapidly changing technologies, significant competition and a strongemphasis on intellectual property. This is true in the field of gene therapy generally, and in the treatments for our key disease areas. While we believethat the strength of our team, gene therapy expertise, scientific knowledge and intellectual property provide us with competitive advantages, we facecompetition from several sources, including large and small biopharmaceutical companies, academic research institutions, government agencies andpublic and private research institutions. Not only must we compete with other companies that are focused on gene therapy, but any product candidatesthat we successfully develop and commercialize will compete with existing therapies and new therapies that may become available in the future.Many of our competitors have significantly greater financial resources and expertise in research and development, manufacturing,preclinical testing, clinical trials, regulatory approvals and product marketing than we do. These competitors also compete with us in recruiting andretaining qualified scientific and management personnel, establishing clinical trial sites and patient registration for clinical trials and acquiringtechnologies complementary to, or necessary for, clinical programs. Mergers and acquisitions in the pharmaceutical and biotechnology industries mayresult in even more resources being concentrated among a smaller number of our competitors. Smaller or early stage companies may also prove to besignificant competitors, particularly through collaborative arrangements with large and established companies.There are other organizations working to improve existing therapies or to develop new therapies for our initially selected diseaseindications. Depending on how successful these efforts are, it is possible they may increase the barriers to adoption and success for our productcandidates, if approved. These efforts include two product candidates Applied Genetic Technologies Corporation, or AGTC, have in Phase 1/2 clinicaltrials to treat ACHM related to CNGB3 and CNGA3, respectively, a product candidate in Phase 1/2 clinical trials by Nightstar Therapeutics plc and aprogram AGTC is running to treat XLRP, as well as Luxturna, which is marketed by Spark Therapeutics, Inc. and has been approved to treat RPE65-deficiency. We are not aware of any other gene therapy product candidates in clinical development targeting xerostomia. We are aware of other ALSgene therapies utilizing different treatment mechanisms to treat different genetically defined subsets of ALS patients, as well as gene therapy productcandidates being developed for the treatment of Parkinson’s disease, including those being developed by Voyager Therapeutics, Inc. and AxovantSciences Ltd.We anticipate that we will face intense and increasing competition as new drugs enter the market and advanced technologies becomeavailable. We expect any treatments that we develop and commercialize to 40Table of Contentscompete on the basis of, among other things, efficacy, safety, convenience of administration and delivery, price, the level of generic competition andthe availability of reimbursement from government and other third-party payors.Intellectual PropertyOur success depends in large part upon our ability to secure and maintain proprietary protection for our technologies and products and tooperate without infringing the proprietary rights of others. Our policy is to protect our proprietary position by, among other methods, filing orcollaborating with our licensors to file U.S. and foreign patent applications related to our proprietary technology, inventions and improvements thatare important to the development and implementation of our business. We also use other forms of protection, such as confidential information andtrademark protection, particularly where we do not believe patent protection is appropriate or obtainable. Our patent portfolio consists of acombination of issued patents and pending patent applications that are owned or licensed from third parties.As of December 31, 2018, we own, have an exclusive license or co-exclusive license under, or an exclusive option to license 38 UnitedStates and foreign issued or allowed patents and 176 patent applications, pending in the United States and internationally. For any individual patent,the term depends on the applicable law in the country in which the patent is granted. In most countries where we have filed patent applications orin-licensed patents and patent applications, patents have a term of 20 years from the application filing date or earliest claimed non-provisional prioritydate. In the United States, the patent term is 20 years but may be shortened if a patent is terminally disclaimed over another patent that expires earlier.The term of a U.S. patent may also be lengthened by a patent term adjustment, in order to address administrative delays by the United States Patent andTrademark Office in granting a patent. In the United States, the term of a patent that covers an FDA-approved drug or biologic may be eligible forpatent term extension in order to restore the period of a patent term lost during the premarket FDA regulatory review process. The Drug PriceCompetition and Patent Term Restoration Act of 1984, or the Hatch-Waxman Act, permits a patent term extension of up to five years beyond thenatural expiration of the patent. The patent term restoration period is generally equal to the regulatory review period for the approved product whichperiod occurs after the date the patent issued, subject to certain exceptions. Only one patent may be extended for a regulatory review period for anyproduct, and the application for the extension must be submitted prior to the expiration of the patent. In the future, we may decide to apply forrestoration of patent term for one of our currently owned or licensed patents to extend its current expiration date, depending on the expected length ofthe clinical trials and other factors involved in the filing of the relevant BLA.Company-Owned Intellectual PropertyWe own six patent families relating to gene regulation platform technologies developed by us. The first patent family includes 22 pendingpatent applications with claims directed to compositions of matter and methods of use in the United States, Europe, Australia, Canada, China, HongKong, India, Japan, Brazil, Egypt, Indonesia, Israel, Republic of Korea, Malaysia, Mexico, New Zealand, Vietnam, African Regional IPO, Philippines,Singapore, South Africa and Eurasia. Patents issued from this family are expected to expire February 2, 2036, not including any patent termadjustments that may extend the patent term in certain jurisdictions.The second patent family includes 20 pending patent applications with claims directed to compositions of matter and methods of use inthe United States, Europe, Australia, China, India, Japan, Brazil, Egypt, Indonesia, Israel, Republic of Korea, Malaysia, Mexico, New Zealand, Vietnam,African Regional IPO, Philippines, Singapore, South Africa and Eurasia. Additional filings in Hong Kong and Canada will be made in due course.Patents issued from this family are expected to expire February 2, 2037, not including any patent term adjustments that may extend the patent term incertain jurisdictions. 41Table of ContentsThe third patent family includes 20 pending patent applications with claims directed to compositions of matter and methods of use in theUnited States, Europe, Australia, China, India, Japan, Brazil, Egypt, Indonesia, Israel, Republic of Korea, Malaysia, Mexico, New Zealand, Vietnam,African Regional IPO, Philippines, Singapore, South Africa and Eurasia. Additional filings in Hong Kong and Canada will be made in due course.Patents issued from this family are expected to expire February 2, 2037, not including any patent term adjustments that may extend the patent term incertain jurisdictions.The fourth patent family includes 19 pending patent applications with claims directed to compositions of matter and methods of use in theUnited States, Europe, Australia, India, Japan, Brazil, Egypt, Indonesia, Israel, Republic of Korea, Malaysia, Mexico, New Zealand, Vietnam, AfricanRegional IPO, Philippines, Singapore, South Africa, and Eurasia. Additional filings in China, Hong Kong, and Canada will be made in due course.Patents issued from this family are expected to expire August 3, 2037, not including any patent term adjustments that may extend the patent term incertain jurisdictions.The other two families include two international applications relating to different gene regulations platform technologies with claimsdirected to compositions of matter and methods of use. We expect to convert each of these international applications to U.S. and international patentfilings in due course. Patents issued from these two patent families are expected to expire in 2037 and 2038, not including any patent term adjustmentsthat may extend the patent term in certain jurisdictions.We also own one patent relating to a vector technology developed by Vector Neurosciences Inc, acquired on October 5, 2018, with claimsdirected to compositions of matter. This patent is expected to expire October 21, 2025, not including any patent term adjustments that may extend thepatent term in certain jurisdictions.Licensed Intellectual PropertyCertain of our issued patents and pending patent applications are exclusively licensed to us from UCL Business, Plc (“UCLB”), BrandeisUniversity (“Brandeis”) and the National Institute of Dental and Craniofacial Research (“NIDCR”).UCLBThe UCLB portfolio includes three licensed patent families relating to our RPE65, CNGA3, and RPGR gene therapy programs and oneoptioned patent family relating to our dry AMD gene therapy program with a combined 49 pending patent applications.The first patent family, with claims directed to compositions of matter and methods of use relating to our RPE65 program, and theAAV-RPE65 product candidate includes 18 pending patent applications in the United States, Europe, Australia, Canada, China, Hong Kong, India,Japan, Brazil, Egypt, Israel, Malaysia, Mexico, New Zealand, Nigeria, Philippines, Singapore, and Thailand. Patents issued from this family areexpected to expire February 8, 2036, not including any patent term extensions or adjustments that may extend the patent term in certain jurisdictions.The second patent family, with claims directed to compositions of matter and methods of use relating to our achromatopsia program andthe AAV-CNGA3 product candidate, includes one pending patent application, which we expect to convert to an international application andsubsequent U.S. and international patent filings in due course. Patents issued from this family are expected to expire in January 2039, not includingany patent term extensions or adjustments that may extend the patent term in certain jurisdictions.The third patent family, with claims directed to compositions of matter and methods of use relating to our retinitis pigmentosa program andthe AAV-RPGR product candidate, includes one allowed patent application 42Table of Contentsin the United States and four pending applications in Europe, Canada, China, and Japan. Patents issued from this family are expected to expire inJuly 2035, not including any patent term extensions or adjustments that may extend the patent term in certain jurisdictions.The fourth patent family which we have optioned, with claims directed to compositions of matter and methods of use relating to our dryAMD gene therapy program, includes 25 pending applications in the United States, Europe, Australia, Canada, China, Hong Kong (two applications),India, Japan, Brazil, Egypt, Indonesia, Israel, Republic of Korea, Malaysia, Mexico, New Zealand, Nigeria, Vietnam, African Regional IPO, Philippines,Singapore, South Africa, Thailand and Eurasia. Patents issued from this family are expected to expire February 19, 2036, not including any patent termextensions or adjustments that may extend the patent term in certain jurisdictions.BrandeisThe licensed Brandeis portfolio includes one patent family with claims directed to compositions of matter and methods of use relating toour ALS gene therapy program and the AAV-UPF1 product candidate.This patent family includes an issued patent in Australia and pending patent applications in the United States, Europe, Canada and HongKong. Patents issued from this family are expected to expire October 8, 2033, not including any patent term extensions or adjustments that may extendthe patent term in certain jurisdictions.National Institute of Dental and Craniofacial ResearchThe exclusively licensed NIDCR portfolio includes one patent family with claims directed to compositions of matter and methods of userelating to our Sjogren’s Syndrome gene therapy program. This patent family includes four issued or allowed patents in the United States, Europe,Canada and Australia, and one pending patent application in the United States. Patents issued from this family are expected to expire August 30, 2033,not including any patent term extensions or adjustments that may extend the patent term in certain jurisdictions.License AgreementsLicense Agreements with UCLBWe previously entered into several license agreements with UCLB, covering the following inherited retinal disease programs: (a) ACHMcaused by mutations in CNGB3; (b) ACHM caused by mutations in CNGA3; (c) XLRP; and (d) RPE65-mediated IRD (together, the “Licensed GeneTherapy Programs”). The terms of these license agreements were set forth in (i) the license agreement, dated February 4, 2015, as amended, betweenAthena Vision Ltd. and UCLB (the “First UCLB License Agreement”); (ii) the license agreements, dated July 29, 2017, as amended, between MeiraGTxUKII Limited and UCL Business, Plc (the “Second UCLB License Agreement”); and (iii) the license agreement, dated March 15, 2018, amongMeiraGTx Limited, MeiraGTx UKII Limited and UCL Business Plc (the “Third UCLB License Agreement” and, collectively, the “prior UCLB licenseagreements”). In January and February 2019, we amended and restated the prior UCLB license agreements to establish new standalone licenseagreement (each, a “StandAlone UCLB Agreement”) for each of the Licensed Gene Therapy Programs. We have removed from each of the Stand-aloneAgreements our obligation to pay UCLB a share of certain sublicensing revenues as was provided under the First UCLB License Agreement and havealigned the material terms of the Stand-Alone Agreements to track those under the Third UCLB License Agreement as previously disclosed and asummary of which is set forth below as is now reflected in each of the Stand-Alone Agreements. 43Table of ContentsUnder the terms of the Third UCLB License Agreement, we paid an initial upfront payment of £6,994, and issued to UCLB £100,000 of ourordinary shares. Under the Stand-Alone Agreement related to CNGB3, we paid UCLB an upfront payment of £1.5 million and issued £1.5 million ofour ordinary shares.Under each of the Stand-Alone UCLB Agreements, UCLB granted us an exclusive, worldwide, and sublicensable license under certainintellectual property rights controlled by UCLB relating to one of the Licensed Gene Therapy Programs to develop and commercialize licensedproducts in a relevant field of gene therapy. We must use diligent efforts to develop and commercialize the licensed products.Under the terms of each Stand-Alone UCLB Agreement, we are required to pay UCLB sales milestone payments of up to a total of£39.75 million in the aggregate and an annual management fee of £50,000 until certain royalty payments have been paid. Additionally, pursuant tothe Stand-Alone UCLB Agreement related to CNGB3, we agreed to pay UCLB an upfront payment of £1.5 million and issue £1.5 million of theCompany’s ordinary shares.Commencing on the first commercial sale of licensed products under each Stand-Alone UCLB Agreement, we must make low single-digitpercentage royalty payments to UCLB on net sales of such products. Our royalty obligations under each agreement continue on a licensedproduct-by-licensed product and country-by-country basis until the latest to occur of the expiration of the last valid claim of a patent claiming suchlicensed product in such country, the expiration of any regulatory exclusivity for all licensed products in such country, or the tenth anniversary of firstcommercial sale of such licensed product in such country.Each Stand-Alone UCLB Agreement will remain in effect on a country-by-country basis until the expiration of the last payment obligationin such country. Each Stand-Alone UCLB Agreement may be terminated: (a) by either party in the event of the other party’s material breach thatremains uncured for 30 days (or for 14 days in the case of breaches related to payment obligations), (b) by either party for the other party’s insolvency,(c) immediately by UCLB if we are in persistent breach of the agreement and the parties fail to agree upon a mechanism to remedy such persistentbreach (or we do not comply with such agreed upon mechanism), or (d) immediately by UCLB if we undergo certain change of control events or if weenter into a sublicense with certain prohibited persons, which may adversely affect UCL’s and/or UCLB’s reputation. Each Stand-Alone UCLBAgreement may also be terminated or converted to a non-exclusive license by UCLB upon three months’ notice if we, based on an independent expertdetermination, fail to use diligent efforts to develop and commercially exploit licensed products and do not cure such failure within a certain cureperiod.License Agreement between Bri-Alzan Inc. and BrandeisIn May 2013, BRI-Alzan Inc., or BRI-Alzan, entered into a license agreement with Brandeis, or the Brandeis Agreement. On December 31,2015, we entered into an Agreement and Plan of Merger, or the BRI-Alzan Merger Agreement, with BRI-Alzan, and the Brandeis Agreement wasassigned to us as a result of such merger. Pursuant to the terms of the BRI-Alzan Merger Agreement, we agreed to make cash payments to BRI-Alzanupon the achievement of certain milestones, subject to an aggregate cap of $4,500,000. In addition, we agreed to make low single-digit percentageroyalty payments to BRI-Alzan on net sales of any product for the therapeutic or prophylactic treatment of ALS that is covered by a valid claim of thepatent rights licensed under the Brandeis Agreement. The BRI-Alzan Merger Agreement includes customary confidentiality, indemnification,non-competition and non-solicitation provisions. 44Table of ContentsPursuant to the Brandeis Agreement, Brandeis granted us an exclusive, worldwide license under certain patent rights with claims directedto compositions of matter and methods of use relating to our ALS gene therapy program and the AAV-UPF1 product candidate to develop andcommercialize licensed products.We must use commercially reasonable efforts to develop and commercialize licensed products. We also acquired non-exclusive, worldwidelicenses to certain know-how controlled by Brandeis’ to exploit licensed products. We are required to pay Brandeis developmental and regulatorymilestone payments of up to a total of $1.0 million in the aggregate. We are also required to pay Brandeis annual license maintenance fees rangingfrom $15,000 to $100,000 depending on the development stage of the licensed product. Commencing on the first commercial sale of licensedproducts, we must make low single-digit percentage royalty payments to Brandeis on net sales of such products. In addition, we must pay Brandeismid-teen percentages of sublicensing revenues.The Brandeis Agreement will remain in effect on a country-by-country basis until the earlier of: (a) 1 year after the date that we, ouraffiliates or sublicensees last sell any licensed product in such country or (b) until the expiration of the last–to-expire of the licensed patent rights insuch country. The Brandeis Agreement may be terminated by Brandeis for our insolvency or for our material breach that remains uncured for 60 days(or for 30 days in the case of breaches related to payment obligations). Such material breach may be cured only once in any 12-month period. Brandeismay also terminate any license granted under the Brandeis Agreement if we fail to timely achieve certain regulatory milestone events.Trade SecretsWe also rely on trade secrets, technical know-how and continuing innovation to develop and maintain our competitive advantage. Ourpolicy requires inventors who are identified on any company-owned patent applications to assign rights to us. We also rely on confidentialityagreements with our employees, consultants and other advisors to protect our proprietary information. Our policy is to require third parties that receivematerial confidential information to enter into confidentiality agreements with us.TrademarksOur trademark MeiraGTx has been registered in the European Union and United States.Government Regulation and Product ApprovalGovernmental authorities in the U.S., at the federal, state and local level, and other countries extensively regulate, among other things, theresearch, development, testing, manufacture, labeling, packaging, promotion, storage, advertising, distribution, marketing, post-approval monitoringand reporting and export and import of products such as those we are developing. The processes for obtaining regulatory approvals in the United Statesand in foreign countries and jurisdictions, along with subsequent compliance with applicable statutes and regulations and other regulatory authorities,are extensive and require the expenditure of substantial time and financial resources.FDA Approval ProcessWe expect our future product candidates to be regulated as biologics. Biological products, including gene therapy products, are subject toextensive regulation by the FDA under the Federal Food, Drug, and Cosmetic Act, or FDCA, and the Public Health Service Act, or PHSA, and otherfederal, state, local and foreign statutes and regulations. Both the FDCA and the PHSA and their corresponding regulations govern, among other things,the research, development, safety, testing, packaging, manufacture, storage, recordkeeping, approval, labeling, promotion and marketing, distribution,post-approval monitoring and reporting, sampling, and import and export of biological products. Before clinical testing of biological products in theUnited States may begin, we must submit an investigational new drug application, or IND, to the FDA, which reviews the clinical protocol, and the INDmust become effective before clinical trials may begin. 45Table of ContentsGene therapy products must be approved by the FDA before they may be legally marketed in the United States and by the appropriateforeign regulatory agencies before they may be legally marketed in foreign countries. The process of obtaining regulatory approvals and thesubsequent compliance with appropriate federal, state, local and foreign statutes and regulations require the expenditure of substantial time andfinancial resources and we may not be able to obtain the required regulatory approvals.Within the FDA, the Center for Biologics Evaluation and Research, or CBER, regulates gene therapy products. The FDA has publishedguidance documents with respect to the development and submission of gene therapy protocols. The FDA also has published guidance documentsrelated to, among other things, gene therapy products in general, their preclinical assessment, observing subjects involved in gene therapy clinicaltrials for delayed adverse events, potency testing, and chemistry, manufacturing and control information in gene therapy INDs.To date, the FDA has approved three human gene therapy products for sale, including Kite Pharma’s Yescarta, Novartis’ Kymriah andSpark’s Luxturna, and has provided general guidance regarding the development of gene therapy products. For example, the FDA has established theOffice of Tissue and Advanced Therapies within CBER, to consolidate the review of gene therapy and related products, and the Cellular, Tissue andGene Therapies Advisory Committee, to advise CBER on its reviews. In addition, the FDA has issued a growing body of clinical guidelines, chemical,manufacturing and control, or CMC, guidelines and other guidelines, all of which are intended to facilitate industry’s development of gene therapyproducts.Ethical, social and legal concerns about gene-editing technology, gene therapy, genetic testing and genetic research could result inadditional regulations restricting or prohibiting the processes we may use. Federal and state agencies, congressional committees and foreigngovernments have expressed interest in further regulating biotechnology. More restrictive regulations or claims that our products are unsafe or pose ahazard could prevent us from commercializing any product candidates. New government requirements may be established that could delay or preventregulatory approval of our product candidates under development. It is impossible to predict whether legislative changes will be enacted, regulations,policies or guidance changed, or interpretations by agencies or courts changed, or what the impact of such changes, if any, may be.U.S. Biological Products Development ProcessThe FDA determined that more than minimally manipulated products must be approved by the FDA through the Biologics LicenseApplication, or BLA, process before they may be legally marketed in the United States. The process required by the FDA before a biologic may bemarketed in the United States generally involves the following: • completion of extensive nonclinical, sometimes referred to as preclinical laboratory tests, and preclinical studies and applicablerequirements for the humane use of laboratory animals and formulation studies in accordance with applicable regulations,including good laboratory practices, or GLPs; • submission to the FDA of an IND application, which must become effective before clinical trials may begin; • approval by an independent Institutional Review Board, or IRB, or ethics committee at each clinical site before the trial iscommenced; • performance of adequate and well controlled human clinical trials according to the FDA’s regulations commonly referred to as goodclinical practices, or GCPs, and any additional requirements for the protection of human research subjects and their healthinformation, to establish the safety and efficacy of the proposed biological product for its intended use; • submission to the FDA of a BLA for marketing approval that includes substantive evidence of safety, purity, potency and efficacyfrom results of nonclinical testing and clinical trials; 46Table of Contents • satisfactory completion of an FDA inspection of the manufacturing facility or facilities where the biological product is produced toassess compliance with cGMP to assure that the facilities, methods and controls are adequate to preserve the biological product’sidentity, strength, quality and purity; • potential FDA audit of the nonclinical and clinical study sites that generated the data in support of the BLA; and • FDA review and approval, or licensure, of the BLA.Before testing any biological product candidate, including a gene therapy product, in humans, the product candidate enters the preclinicaltesting stage. Preclinical tests, also referred to as nonclinical studies, include laboratory evaluations of product chemistry, toxicity and formulation, aswell as animal studies to assess the potential safety and activity of the product candidate. The conduct of the preclinical tests must comply with federalregulations and requirements, including GLPs. The clinical trial sponsor must submit the results of the preclinical tests, together with manufacturingand controls, information about product chemistry, analytical data, any available clinical data or literature and a proposed clinical protocol, to the FDAas part of the IND. Some preclinical testing, such as reproductive toxicity tests and carcinogenicity in animals, may continue even after the IND issubmitted. The IND automatically becomes effective 30 days after receipt by the FDA, after which human clinical trials may begin unless the FDAplaces the clinical trial on a clinical hold within that 30-day time period. In such a case, the IND sponsor and the FDA must resolve any outstandingconcerns before the clinical trial can begin. The FDA may also impose clinical holds on a biological product candidate at any time before or duringclinical trials due to safety concerns or non-compliance. If the FDA imposes a clinical hold, trials may not recommence without FDA authorization andthen only under terms authorized by the FDA. Accordingly, we cannot be sure that submission of an IND will result in the FDA allowing clinical trialsto begin, or that, once begun, issues will not arise that suspend or terminate such trials. In addition to the IND submission process, sponsors of certainhuman clinical trials of cells containing recombinant or synthetic nucleic acid molecules, including human gene transfer studies, had historically beensubject to review by the Recombinant DNA Advisory Committee, or RAC, pursuant to the National Institutes of Health’s Guidelines for ResearchInvolving Recombinant or Synthetic Nucleic Acid Molecules, or NIH Guidelines. On August 17, 2018, the NIH issued a notice in the Federal Registerand issued a public statement proposing changes to the oversight framework for gene therapy trials, including changes to the applicable NIHGuidelines to modify the roles and responsibilities of the RAC with respect to human clinical trials of gene therapy products, and requesting publiccomment on its proposed modifications. During the public comment period, which closed October 16, 2018, the NIH has announced that it will nolonger accept new human gene transfer protocols for review as a part of the protocol registration process or convene the RAC to review individualclinical protocols. These trials will remain subject to the FDA’s oversight and other clinical trial regulations, and oversight at the local level willcontinue as set forth in the NIH Guidelines. Specifically, under the NIH Guidelines, supervision of human gene transfer trials includes evaluation andassessment by an IBC, a local institutional committee that reviews and oversees research utilizing recombinant or synthetic nucleic acid molecules atthat institution. The IBC assesses the safety of the research and identifies any potential risk to the public health or the environment, and such reviewmay result in some delay before initiation of a clinical trial. While the NIH Guidelines are not mandatory unless the research in question is beingconducted at or sponsored by institutions receiving NIH funding of recombinant or synthetic nucleic acid molecule research, many companies andother institutions not otherwise subject to the NIH Guidelines voluntarily follow them.Clinical trials involve the administration of the biological product candidate to healthy volunteers or patients under the supervision ofqualified investigators, generally physicians not employed by or under the study sponsor’s control. Clinical trials are conducted under protocolsdetailing, among other things, the objectives of the clinical trial, dosing procedures, subject selection and exclusion criteria, the efficacy measurementsto be evaluated and the parameters to be used to monitor subject safety, including stopping rules that assure a clinical trial will be stopped if certainadverse events should occur. Each protocol and any amendments to the protocol must be submitted to the FDA as part of the IND. Clinical trials mustbe conducted and monitored in accordance 47Table of Contentswith the FDA’s regulations comprising the GCP requirements, including the requirement that all research subjects provide informed consent. Further,each clinical trial must be reviewed and approved by an independent institutional review board, or IRB, at or servicing each institution at which theclinical trial will be conducted. An IRB is charged with protecting the welfare and rights of study participants and considers such items as whether therisks to individuals participating in the clinical trials are minimized and are reasonable in relation to anticipated benefits. The IRB also approves theform and content of the informed consent that must be signed by each clinical trial subject or his or her legal representative and must monitor theclinical trial until completed. Clinical trials also must be reviewed by an IBC, a local institutional committee that reviews and oversees basic andclinical research conducted at that institution. The IBC assesses the safety of the research and identifies any potential risk to public health or theenvironment.Human clinical trials are typically conducted in three sequential phases that may overlap or be combined: • Phase 1. The biological product candidate is initially introduced into healthy human subjects and tested for safety. In the case ofsome products for severe or life-threatening diseases, especially when the product may be too inherently toxic to ethicallyadminister to healthy volunteers, the initial human testing is often conducted in patients. • Phase 2. The biological product candidate is evaluated in a limited patient population to identify possible adverse effects andsafety risks, to preliminarily evaluate the efficacy of the product for specific targeted diseases and to determine dosage tolerance,optimal dosage and dosing schedule. • Phase 3. Clinical trials are undertaken to further evaluate dosage, clinical efficacy, potency, and safety in an expanded patientpopulation at geographically dispersed clinical trial sites. These clinical trials are intended to establish the overall risk/benefit ratioof the product and provide an adequate basis for product labeling.In most cases, the FDA requires two adequate and well controlled Phase 3 clinical trials to demonstrate the safety and efficacy of abiological product. In rare instances, a single Phase 3 trial, together with other confirmatory evidence may be sufficient to support a BLA submission.Post-approval clinical trials, sometimes referred to as Phase 4 clinical trials, may be conducted after initial marketing approval. These clinical trials areused to gain additional experience from the treatment of patients in the intended therapeutic indication, particularly for long-term safety follow-up.The FDA recommends that sponsors observe subjects for potential gene therapy-related delayed adverse events for a 15-year period, including aminimum of five years of annual examinations followed by ten years of annual queries, either in person or by questionnaire.During all phases of clinical development, regulatory agencies require extensive monitoring and auditing of all clinical activities, clinicaldata, and clinical trial investigators. Annual progress reports detailing the results of the clinical trials must be submitted to the FDA. Written IND safetyreports must be promptly submitted to the FDA, the NIH and the investigators for serious and unexpected adverse events, any findings from other trials,tests in laboratory animals or in vitro testing that suggest a significant risk for human subjects, or any clinically important increase in the rate of aserious suspected adverse reaction over that listed in the protocol or investigator brochure. The sponsor must submit an IND safety report within 15calendar days after the sponsor determines that the information qualifies for reporting. The sponsor also must notify the FDA of any unexpected fatal orlife-threatening suspected adverse reaction within seven calendar days after the sponsor’s initial receipt of the information. Phase 1, Phase 2 and Phase3 clinical trials may not be completed successfully within any specified period, if at all. The FDA or the sponsor or its data safety monitoring boardmay suspend or permanently discontinue a clinical trial at any time on various grounds, including a finding that the research subjects or patients arebeing exposed to an unacceptable health risk or the clinical trial is not being conducted in accordance with FDA regulations. Similarly, an IRB cansuspend or terminate approval of a clinical study at its institution if the clinical trial is not being conducted in accordance with the IRB’s requirementsor if the biological product candidate has been associated with unexpected serious harm to patients. The FDA and the IRB may also halt, terminate orimpose other conditions if either believes the patients are subject to unacceptable risk. 48Table of ContentsThere are also requirements governing the reporting of ongoing clinical trials and completed clinical trial results to public registries.Sponsors of clinical trials of FDA-regulated products, including biologics, are required to register and disclose certain clinical trial information, whichis publicly available at www.clinicaltrials.gov. Information related to the product, patient population, phase of investigation, study sites andinvestigators, and other aspects of the clinical trial is then made public as part of the registration. Sponsors are also obligated to discuss the results oftheir clinical trials after completion. Disclosure of the results of these trials can be delayed until the new product or new indication being studied hasbeen approved.Human gene therapy products based on gene-editing technology are a new category of therapeutics. Because this is a relatively new andexpanding area of novel therapeutic interventions, there can be no assurance as to the length of the study period, the number of patients the FDA willrequire to be enrolled in the trials in order to establish the safety, efficacy, purity and potency of human gene therapy products, or that the datagenerated in these trials will be acceptable to the FDA to support marketing approval. The NIH and the FDA have a publicly accessible database, theGenetic Modification Clinical Research Information System, which includes information on gene transfer trials and serves as an electronic tool tofacilitate the reporting and analysis of adverse events in these trials.Concurrent with clinical trials, companies usually complete additional animal trials and must also develop additional information aboutthe physical characteristics of the biological product candidate as well as finalize a process for manufacturing the product in commercial quantities inaccordance with cGMP requirements. To help reduce the risk of the introduction of adventitious agents with use of biological products, the PHS Actemphasizes the importance of manufacturing control for products whose attributes cannot be precisely defined. The manufacturing process must becapable of consistently producing quality batches of the product candidate and, among other things, the sponsor must develop methods for testing theidentity, strength, quality, potency and purity of the final biological product. Additionally, appropriate packaging must be selected and tested andstability studies must be conducted to demonstrate that the biological product candidate does not undergo unacceptable deterioration over its shelflife.U.S. Review and Approval ProcessesAfter the completion of clinical trials of a biological product candidate, FDA approval of a BLA must be obtained before commercialmarketing and distribution of the biological product. The BLA must include results of product development, laboratory and animal trials, human trials,information on the manufacture, pharmacology, chemistry and controls of the product, proposed labeling and other relevant information. In addition,under the Pediatric Research Equity Act, or PREA, a BLA or supplement to a BLA must contain data to assess the safety and effectiveness of thebiological product candidate for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for eachpediatric subpopulation for which the product is safe and effective.The Food and Drug Administration Safety and Innovation Act, or FDASIA, requires that a sponsor who is planning to submit a marketingapplication for a drug or biological product that includes a new active ingredient, new indication, new dosage form, new dosing regimen or new routeof administration submit an initial Pediatric Study Plan, or PSP, within sixty days after an end-of-Phase 2 meeting or as may be agreed between thesponsor and FDA. The initial PSP must include, among other things, an outline of the pediatric study or studies that the sponsor plans to conduct,including to the extent practicable study objectives and design, age groups, relevant endpoints and statistical approach, or a justification for notincluding such detailed information, and any request for a deferral of pediatric assessments or a full or partial waiver of the requirement to provide datafrom pediatric studies along with supporting information, along with any other information specified in FDA regulations. The FDA and the sponsormust reach agreement on the PSP. A sponsor can submit amendments to an agreed-upon initial PSP at any time if changes to the pediatric plan need tobe considered based on data collected from nonclinical studies, early phase clinical trials, and/or other clinical development programs. The FDA maygrant deferrals for submission of data or full or partial waivers. Unless otherwise required by 49Table of Contentsregulation, PREA does not apply to any biological product for an indication for which orphan designation has been granted. The testing and approvalprocesses require substantial time and effort and there can be no assurance that the FDA will accept the BLA for filing and, even if filed, that anyapproval will be granted on a timely basis, if at all.Under the Prescription Drug User Fee Act, or PDUFA, as amended, each BLA must be accompanied by a user fee. The FDA adjusts thePDUFA user fees on an annual basis. PDUFA also imposes an annual program fee for products. Fee waivers or reductions are available in certaincircumstances, including a waiver of the application fee for the first human drug application filed by a small business. Additionally, no user fees areassessed on BLAs for products designated as orphan drugs, unless the product also includes a non-orphan indication.Within 60 days following submission of the application, the FDA reviews a BLA submitted to determine if it is substantially completebefore the agency accepts it for filing. The FDA may refuse to file any BLA that it deems incomplete or not properly reviewable at the time ofsubmission and may request additional information. In this event, the BLA must be resubmitted with the additional information. The resubmittedapplication also is subject to review before the FDA accepts it for filing. Once the submission is accepted for filing, the FDA begins an in-depthsubstantive review of the BLA. Under PDUFA, the FDA has agreed to certain performance goals to complete the review of BLAs. The FDA may give apriority review designation to biological products that offer major advances in treatment, or provide a treatment where no adequate therapy exists. Apriority review means that the goal for the FDA to review an application is six months, rather than the standard review of ten months under currentPDUFA guidelines. Under the current PDUFA agreement, these six and ten month review periods are measured from the “filing” date rather than thereceipt date for original BLAs, which typically adds approximately two months to the timeline for review and decision from the date of submission.The FDA reviews the BLA to determine, among other things, whether the proposed product is safe and potent, or effective, for its intendeduse, and has an acceptable purity profile, and whether the product is being manufactured in accordance with cGMP requirements to assure and preservethe product’s identity, safety, strength, quality, potency and purity. The FDA may refer applications for novel biological products or biologicalproducts that present difficult questions of safety or efficacy to an advisory committee, typically a panel that includes clinicians and other experts, forreview, evaluation and a recommendation as to whether the application should be approved and under what conditions. The FDA is not bound by therecommendations of an advisory committee, but it considers such recommendations carefully when making decisions. During the biological productapproval process, the FDA also will determine whether a Risk Evaluation and Mitigation Strategy, or REMS, is necessary to assure the safe use of thebiological product candidate. If the FDA concludes a REMS is needed, the sponsor of the BLA must submit a proposed REMS; the FDA will notapprove the BLA without a REMS, if required.Before approving a BLA, the FDA will inspect the facilities at which the product is manufactured. The FDA will not approve the productunless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to assure consistentproduction of the product within required specifications. Additionally, before approving a BLA, the FDA will typically inspect one or more clinicalsites to assure that the clinical trials were conducted in compliance with IND study requirements and GCP requirements. To assure cGMP and GCPcompliance, an applicant must incur significant expenditure of time, money and effort in the areas of training, record keeping, production, and qualitycontrol.Notwithstanding the submission of relevant data and information, the FDA may ultimately decide that the BLA does not satisfy itsregulatory criteria for approval and deny approval. Data obtained from clinical trials are not always conclusive and the FDA may interpret datadifferently than we interpret the same data. If the agency decides not to approve the BLA in its present form, the FDA will issue a complete responseletter that usually describes all of the specific deficiencies in the BLA identified by the FDA. The deficiencies identified 50Table of Contentsmay be minor, for example, requiring labeling changes, or major, for example, requiring additional clinical trials. Additionally, the complete responseletter may include recommended actions that the applicant might take to place the application in a condition for approval. If a complete response letteris issued, the applicant may either resubmit the BLA, addressing all of the deficiencies identified in the letter, or withdraw the application. If, or when,those deficiencies have been addressed to the FDA’s satisfaction in a resubmission of the BLA, the FDA will issue an approval letter. Under the currentPDUFA guidelines, the FDA has committed to reviewing such resubmissions in two or six months of receipt depending on the type of informationincluded.If regulatory approval of a product is granted, such approval will be granted for particular indications and may entail limitations on theindicated uses for which such product may be marketed. For example, the FDA may approve the BLA with REMS, to ensure the benefits of the productoutweigh its potential risks. A REMS is a safety strategy to manage a known or potential serious risk associated with a medicine and to enable patientsto have continued access to such medicines by managing their safe use, and could include medication guides, physician communication plans, orelements to assure safe use, such as restricted distribution methods, patient registries and other risk minimization tools. The FDA also may conditionapproval on, among other things, changes to proposed labeling or the development of adequate controls and specifications. The requirement for aREMS can materially affect the potential market and profitability of the product.Once approved, the FDA may withdraw the product approval if compliance with pre- and post-marketing requirements is not maintained orif problems occur after the product reaches the marketplace. Changes to some of the conditions established in an approved BLA, including changes inindications, product labeling, manufacturing processes or facilities, require submission and FDA approval of a new BLA or BLA supplement before thechange can be implemented. A BLA supplement for a new indication typically requires clinical data similar to that in the original application, and theFDA uses the same procedures and actions in reviewing BLA supplements as it does in reviewing BLAs. The FDA may require one or more Phase 4post-market studies or surveillance to further assess and monitor the product’s safety and effectiveness after commercialization, and may limit furthermarketing of the product based on the results of these post-marketing studies.Additionally, new government requirements, including those resulting from new legislation, may be established, or the FDA’s policies maychange, which could impact the timeline for regulatory approval or otherwise impact ongoing development programs. For example, in December 2016,the 21st Century Cures Act was signed into law. This act is intended, among other things, to modernize the regulation of drugs and biologics and tospur innovation, and contains provisions specific to the development of cell therapies.One of the performance goals agreed to by the FDA under the PDUFA is to review 90% of standard BLAs in ten months from the filing dateand 90% of priority BLAs in six months from the filing date, whereupon a review decision is to be made. The FDA does not always meet its PDUFAgoal dates for standard and priority BLAs and its review goals are subject to change from time to time. The review process and the PDUFA goal datemay be extended by three months if the FDA requests or the BLA sponsor otherwise provides additional information or clarification regardinginformation already provided in the submission within the last three months before the PDUFA goal date.Orphan Drug DesignationThe FDA may grant Orphan drug designation to drugs or biologics intended to treat a rare disease or condition that affects fewer than200,000 individuals in the United States, or if it affects more than 200,000 individuals in the United States, there is no reasonable expectation that thecost of developing and marketing the drug or biologic for this type of disease or condition will be recovered from its sales in the United States. Orphandrug designation must be requested before submitting a BLA. After the FDA grants orphan product designation, the identity of the therapeutic agentand its potential orphan use are disclosed publicly by the FDA. Orphan drug designation does not convey any advantage in or shorten the duration ofthe regulatory review and approval process. 51Table of ContentsIn the United States, Orphan drug designation entitles a party to financial incentives such as opportunities for grant funding towardsclinical trial costs, tax advantages and BLA user-fee waivers. In addition, if a product receives the first FDA approval for the indication for which it hasorphan designation, the product is entitled to orphan drug exclusivity, which means the FDA may not approve any other application, including a fullBLA, to market the same drug or biologic for the same indication for a period of seven years, except in limited circumstances, such as a showing ofclinical superiority over the product with orphan exclusivity or where the manufacturer with orphan exclusivity is unable to assure sufficient quantitiesof the approved orphan-designated product. Competitors, however, may receive approval of different products for the indication for which the orphanproduct has exclusivity or obtain approval for the same product but for a different indication for which the orphan product has exclusivity. Orphanproduct exclusivity also could block the approval of one of our products for seven years if a competitor obtains approval of the same biologicalproduct as defined by the FDA or if our product candidate is determined to be contained within the competitor’s product for the same indication ordisease. If a drug or biological product designated as an orphan product receives marketing approval for an indication broader than what is designated,it may not be entitled to orphan product exclusivity. In addition, exclusive marketing rights in the United States may be lost if the FDA laterdetermines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantities of the product tomeet the needs of patients with the rare disease or condition.Expedited Development and Review ProgramsThe FDA has a Fast Track program that is intended to expedite or facilitate the process for reviewing new biological products that meetcertain criteria. Specifically, new biological products are eligible for Fast Track designation if they are intended to treat a serious or life-threateningdisease or condition and demonstrate the potential to address unmet medical needs for the disease or condition. Fast Track designation applies to thecombination of the product and the specific indication for which it is being studied. The sponsor of a new biologic may request that the FDA designatethe biologic as a Fast Track product at any time during the clinical development of the product. The FDA must determine if the biologic productcandidate qualifies for Fast Track designation within 60 days of receipt of the sponsor’s request. Unique to a Fast Track product, the FDA may considerfor review sections of the marketing application on a rolling basis before the complete application is submitted, if the sponsor provides a schedule forthe submission of the sections of the application, the FDA agrees to accept sections of the application and determines that the schedule is acceptable,and the sponsor pays any required user fees upon submission of the first section of the application.Any product submitted to the FDA for marketing, including under a Fast Track program, may be eligible for other types of FDA programsintended to expedite development and review, such as priority review and accelerated approval. Any product is eligible for priority review if it has thepotential to provide safe and effective therapy where no satisfactory alternative therapy exists or a significant improvement in the treatment, diagnosisor prevention of a disease compared to marketed products. The FDA will attempt to direct additional resources to the evaluation of an application for anew biological product designated for priority review in an effort to facilitate the review. Additionally, a product may be eligible for acceleratedapproval. Biological products studied for their safety and effectiveness in treating serious or life-threatening illnesses and that provide meaningfultherapeutic benefit over existing treatments may be eligible for accelerated approval, which means that they may be approved on the basis of adequateand well controlled clinical trials establishing that the product has an effect on a surrogate endpoint that is reasonably likely to predict a clinicalbenefit, or on the basis of an effect on a clinical endpoint other than survival or irreversible morbidity or mortality or other clinical benefit, taking intoaccount the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments. As a condition of approval, the FDAmay require that a sponsor of a biological product subject to accelerated approval perform adequate and well-controlled post-marketing Phase 4clinical trials. Failure to conduct required post-approval trials, or to confirm a clinical benefit during post-marketing trials, will allow the FDA towithdraw the approved biologic product from the market on an expedited basis. In addition, the FDA currently requires as a condition for acceleratedapproval pre-approval of promotional materials, which could adversely impact the timing of the commercial launch of the product. Fast Trackdesignation, priority review and 52Table of Contentsaccelerated approval do not change the standards for approval but may expedite the development or approval process.In addition, under the provisions of FDASIA, enacted in 2012, the FDA established a Breakthrough Therapy Designation which is intendedto expedite the development and review of products that treat serious or life-threatening diseases or conditions. A breakthrough therapy is defined as adrug that is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, and preliminaryclinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significantendpoints, such as substantial treatment effects observed early in clinical development. The designation includes all of the features of Fast Trackdesignation, as well as more intensive FDA interaction and guidance. The Breakthrough Therapy Designation is a distinct status from both acceleratedapproval and priority review, but these can also be granted to the same product candidate if the relevant criteria are met. The FDA must take certainactions, such as holding timely meetings and providing advice, intended to expedite the development and review of an application for approval of abreakthrough therapy. All requests for breakthrough therapy designation will be reviewed within 60 days of receipt, and FDA will either grant or denythe request.Furthermore, as part of its implementation of the 21st Century Cures Act, the FDA established the Regenerative Medicine AdvancedTherapy, or RMAT, designation, to facilitate an efficient development program for, and expedite review of, certain drugs and biological products. Abiological product is eligible for RMAT designation if it qualifies as a RMAT, which is defined as a cell therapy, therapeutic tissue engineeringproduct, human cell and tissue product, or any combination product using such therapies or products, with limited exceptions, and is intended to treat,modify, reverse, or cure a serious or life-threatening disease or condition and for which preliminary clinical evidence indicates that the biologicalproduct has the potential to address unmet medical needs for such a disease or condition. Like breakthrough therapy designation, RMAT designationprovides potential benefits that include more frequent meetings with FDA to discuss the development plan for the product candidate, and eligibility forrolling review and priority review. Products granted RMAT designation may also be eligible for accelerated approval on the basis of a surrogate orintermediate endpoint reasonably likely to predict long-term clinical benefit, or reliance upon data obtained from a meaningful number of sites,including through expansion to additional sites. RMAT-designated products that receive accelerated approval may, as appropriate, fulfill their post-approval requirements through the submission of clinical evidence, clinical trials, patient registries, or other sources of real world evidence (such aselectronic health records); through the collection of larger confirmatory data sets; or via post-approval monitoring of all patients treated with suchtherapy prior to approval of the therapy.Fast Track designation, priority review, accelerated approval, breakthrough therapy designation and RMAT designation do not change thestandards for approval but may expedite the development or approval process. Even if we receive one of these designations for our product candidates,the FDA may later decide that our product candidates no longer meets the conditions for qualification. In addition, receiving these designations maynot provide us with a material commercial advantage.Post-Approval RequirementsMaintaining substantial compliance with applicable federal, state, and local statutes and regulations requires the expenditure of substantialtime and financial resources. Rigorous and extensive FDA regulation of biological products continues after approval, particularly with respect tocGMP requirements. We will rely, and expect to continue to rely, on third parties for the production of clinical and commercial quantities of anyproducts that we may commercialize. Manufacturers of our products are required to comply with applicable requirements in the cGMP regulations,including quality control and quality assurance and maintenance of records and documentation. Other post-approval requirements applicable tobiological products, include reporting of cGMP deviations that may affect the identity, potency, purity and overall safety of a distributed product,record-keeping requirements, reporting of adverse effects, reporting updated safety and efficacy information, and complying with electronic record andsignature requirements. 53Table of ContentsAfter a BLA is approved, the product also may be subject to official lot release. As part of the manufacturing process, the manufacturer isrequired to perform certain tests on each lot of the product before it is released for distribution. If the product is subject to official release by the FDA,the manufacturer submits samples of each lot of product to the FDA together with a release protocol showing a summary of the history of manufactureof the lot and the results of all of the manufacturer’s tests performed on the lot. The FDA also may perform certain confirmatory tests on lots of someproducts, such as viral vaccines, before releasing the lots for distribution by the manufacturer. In addition, the FDA conducts laboratory research relatedto the regulatory standards on the safety, purity, potency, and effectiveness of biological products.To help reduce the increased risk of the introduction of adventitious agents, the PHSA Act emphasizes the importance of manufacturingcontrols for products whose attributes cannot be precisely defined. The PHSA Act also provides authority to the FDA to immediately suspend biologicslicenses in situations where there exists a danger to public health, to prepare or procure products in the event of shortages and critical public healthneeds, and to authorize the creation and enforcement of regulations to prevent the introduction or spread of communicable diseases within the UnitedStates.The FDA may require one or more Phase 4 post-market trials or surveillance to further assess and monitor the product’s safety andeffectiveness after commercialization, and may limit further marketing of the product based on the results of these post-marketing studies. We also mustcomply with the FDA’s advertising and promotion requirements, such as those related to direct-to-consumer advertising, the prohibition on promotingproducts for uses or in patient populations that are not described in the product’s approved labeling (known as “off-label use”), industry-sponsoredscientific and educational activities, and promotional activities involving the Internet. Biologics may be marketed only for the approved indicationsand in accordance with the provisions of the approved labeling.Discovery of previously unknown problems or the failure to comply with the applicable regulatory requirements may result in restrictionson the marketing of a product or withdrawal of the product from the market as well as possible civil or criminal sanctions. Failure to comply with theapplicable U.S. requirements at any time during the product development process, approval process or after approval, may subject an applicant ormanufacturer to administrative or judicial civil or criminal sanctions and adverse publicity. FDA sanctions could include refusal to approve pendingapplications, withdrawal of an approval, clinical hold, warning or untitled letters, product recalls, product seizures, total or partial suspension ofproduction or distribution, injunctions, fines, refusals of government contracts, mandated corrective advertising or communications with doctors,debarment, restitution, disgorgement of profits, or civil or criminal penalties. Any agency or judicial enforcement action could have a material adverseeffect on us.Biological product manufacturers and other entities involved in the manufacture and distribution of approved biological products arerequired to register their establishments with the FDA and certain state agencies, and are subject to periodic unannounced inspections by the FDA andcertain state agencies for compliance with cGMP requirements and other laws. Accordingly, manufacturers must continue to expend time, money, andeffort in the area of production and quality control to maintain cGMP compliance. Discovery of problems with a product after approval may result inrestrictions on a product, manufacturer, or holder of an approved BLA, including withdrawal of the product from the market. In addition, changes to themanufacturing process or facility generally require prior FDA approval before being implemented and other types of changes to the approved product,such as adding new indications and additional labeling claims, are also subject to further FDA review and approval.U.S. Patent Term Restoration and Marketing ExclusivityDepending upon the timing, duration and specifics of the FDA approval of the use of our product candidates, some of our U.S. patents maybe eligible for limited patent term extension under Hatch-Waxman Act. The Hatch-Waxman Act permit a patent restoration term of up to five years ascompensation for patent term lost 54Table of Contentsduring product development and the FDA regulatory review process. However, patent term restoration cannot extend the remaining term of a patentbeyond a total of 14 years from the product’s approval date. The patent term restoration period is generally equal to the regulatory review period for theapproved product which period occurs after the date the patent issued, subject to certain exceptions. Only one patent may be extended for a regulatoryreview period for any product, and the application for the extension must be submitted prior to the expiration of the patent. The USPTO, inconsultation with the FDA, reviews and approves the application for any patent term extension or restoration. In the future, we may intend to apply forrestoration of patent term for one of our currently owned or licensed patents to extend its current expiration date, depending on the expected length ofthe clinical trials and other factors involved in the filing of the relevant BLA.For patents that might expire during the BLA review phase, the patent owner may request an interim patent term extension. If eligible, aninterim patent term extension may be granted for a period of not more than one year. The patent owner may apply for not more than four subsequentinterim extensions. Any interim extension granted will not be longer than the maximum period of extension allowed post-approval.Biosimilars and ExclusivityThe Biologics Price Competition and Innovation Act of 2009, or BPCIA, created an abbreviated approval pathway for biological productsthat are biosimilar to or interchangeable with an FDA-licensed reference biological product. To date, only a handful of biosimilars have been licensedunder the BPCIA, and numerous biosimilars have been approved in Europe. The FDA has issued several guidance documents outlining an approach toreview and approval of biosimilars.Biosimilarity, which requires that there be no clinically meaningful differences between the biological product and the reference productin terms of safety, purity, and potency, can be shown through analytical studies, animal studies, and a clinical trial or trials. Interchangeability requiresthat a product is biosimilar to the reference product and the product must demonstrate that it can be expected to produce the same clinical results as thereference product in any given patient and, for products that are administered multiple times to an individual, the biologic and the reference biologicmay be alternated or switched after one has been previously administered without increasing safety risks or risks of diminished efficacy relative toexclusive use of the reference biologic. However, complexities associated with the larger, and often more complex, structures of biological products, aswell as the processes by which such products are manufactured, pose significant hurdles to implementation of the abbreviated approval pathway thatare still being worked out by the FDA.Under the BPCIA, an application for a biosimilar product may not be submitted to the FDA until four years following the date that thereference product was first licensed by the FDA. In addition, the approval of a biosimilar product may not be made effective by the FDA until 12 yearsfrom the date on which the reference product was first licensed. During this 12-year period of exclusivity, another company may still market acompeting version of the reference product if the FDA approves a full BLA for the competing product containing the sponsor’s own preclinical dataand data from adequate and well-controlled clinical trials to demonstrate the safety, purity and potency of their product. The BPCIA also createdcertain exclusivity periods for biosimilars approved as interchangeable products. At this juncture, it is unclear whether products deemed“interchangeable” by the FDA will, in fact, be readily substituted by pharmacies, which are governed by state pharmacy law.A biological product can also obtain pediatric market exclusivity in the United States. Pediatric exclusivity, if granted, adds six months toexisting exclusivity periods and patent terms. This six-month exclusivity, which runs from the end of other exclusivity protection or patent term, maybe granted based on the voluntary completion of a pediatric study in accordance with an FDA-issued “Written Request” for such a study.The BPCIA is complex and continues to be interpreted and implemented by the FDA. In addition, recent government proposals havesought to reduce the 12-year reference product exclusivity period. Other 55Table of Contentsaspects of the BPCIA, some of which may impact the BPCIA exclusivity provisions, have also been the subject of recent litigation. As a result, theultimate impact, implementation, and meaning of the BPCIA remains subject to significant uncertainty.Other Healthcare Laws and Compliance RequirementsPharmaceutical companies are subject to additional healthcare regulation and enforcement by the federal government and by authorities inthe states and foreign jurisdictions in which they conduct their business. Such laws include, without limitation: the federal Anti-Kickback Statute,which prohibits, among other things, persons and entities from knowingly and willfully soliciting, receiving, offering or paying remuneration, toinduce, or in return for, either the referral of an individual, or the purchase or recommendation of an item or service for which payment may be madeunder any federal healthcare program; federal civil and criminal false claims laws and civil monetary penalty laws, which prohibit, among other things,individuals or entities from knowingly presenting, or causing to be presented, claims for payment to the federal government, including federalhealthcare programs, that are false or fraudulent; the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which createdadditional federal criminal statutes which prohibit, among other things, executing a scheme to defraud any healthcare benefit program and makingfalse statements relating to healthcare matters; HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of2009, or HITECH, and their implementing regulations, which imposes certain requirements on certain types of individuals and entities relating to theprivacy, security and transmission of individually identifiable health information; the federal Physician Payments Sunshine Act, which requires certainmanufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s HealthInsurance Program, with specific exceptions, to annually report to the federal government, information related to payments or other transfers of valuemade to physicians and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members; andstate and foreign law equivalents of each of the above federal laws, which, in some cases, differ from each other in significant ways, and may not havethe same effect, thus complicating compliance efforts. If their operations are found to be in violation of any of such laws or any other governmentalregulations that apply, they may be subject to penalties, including, without limitation, civil and criminal penalties, damages, fines, the curtailment orrestructuring of operations, exclusion from participation in federal and state healthcare programs, integrity oversight and reporting obligations toresolve allegations of non-compliance and individual imprisonment.Coverage and ReimbursementSignificant uncertainty exists as to the coverage and reimbursement status of any pharmaceutical or biological product for which we obtainregulatory approval. Sales of any product depend, in part, on the extent to which such product will be covered by third-party payors, such as federal,state, and foreign government healthcare programs, commercial insurance and managed healthcare organizations, and the level of reimbursement forsuch product by third-party payors. Decisions regarding the extent of coverage and amount of reimbursement to be provided are made on aplan-by-plan basis. For products administered under the supervision of a physician, obtaining coverage and adequate reimbursement may beparticularly difficult because of the higher prices often associated with such drugs. Additionally, separate reimbursement for the product itself or thetreatment or procedure in which the product is used may not be available, which may impact physician utilization.In addition, the U.S. government, state legislatures and foreign governments have continued implementing cost-containment programs,including price controls, restrictions on coverage and reimbursement and requirements for substitution of generic products. Third party payors areincreasingly challenging the prices charged for medical products and services, examining the medical necessity and reviewing the cost effectiveness ofpharmaceutical or biological products, medical devices and medical services, in addition to questioning safety and efficacy. Adoption of price controlsand cost-containment measures, and adoption of more restrictive policies 56Table of Contentsin jurisdictions with existing controls and measures, could further limit sales of any product. Decreases in third-party reimbursement for any product ora decision by a third-party payor not to cover a product could reduce physician usage and patient demand for the product.Healthcare ReformThe United States and some foreign jurisdictions are considering or have enacted a number of reform proposals to change the healthcaresystem. There is significant interest in promoting changes in healthcare systems with the stated goals of containing healthcare costs, improving qualityor expanding access. In the United States, the pharmaceutical industry has been a particular focus of these efforts and has been significantly affected byfederal and state legislative initiatives, including those designed to limit the pricing, coverage, and reimbursement of pharmaceutical andbiopharmaceutical products, especially under government-funded health care programs, and increased governmental control of drug pricing.In March 2010, the ACA was signed into law, which substantially changed the way healthcare is financed by both governmental andprivate insurers in the United States, and significantly affected the pharmaceutical industry. The ACA contains a number of provisions of particularimport to the pharmaceutical and biotechnology industries, including, but not limited to, those governing enrollment in federal healthcare programs, anew methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused,instilled, implanted or injected, and annual fees based on pharmaceutical companies’ share of sales to federal health care programs. Since its enactment,there have been judicial, Congressional and executive branch challenges to certain aspects of the ACA, and we expect there will be additionalchallenges and amendments to the ACA in the future. For example, the Tax Cuts and Jobs Act of 2017 was enacted, which, among other things,removes penalties for not complying with ACA’s individual mandate to carry health insurance. On December 14, 2018, a Texas U.S. District CourtJudge ruled that the ACA is unconstitutional in its entirety because the “individual mandate” was repealed by Congress as part of the Tax Cuts andJobs Act of 2017. While the Texas U.S. District Court Judge, as well as the Trump administration and CMS, have stated that the ruling will have noimmediate effect pending appeal of the decision, it is unclear how this decision, subsequent appeals, and other efforts to repeal and replace the ACAwill impact the law.There is still uncertainty with respect to the impact President Trump’s administration and the U.S. Congress may have, if any, andany changes will likely take time to unfold, and could have an impact on coverage and reimbursement for healthcare items and services covered byplans that were authorized by the ACA.Other legislative changes have been proposed and adopted since the ACA was enacted, including aggregate reductions of Medicarepayments to providers of 2% per fiscal year and reduced payments to several types of Medicare providers. Moreover, there has recently beenheightened governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which has resulted in severalCongressional inquiries and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to productpricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies fordrug products. At the federal level,CMS announced that it will allow Medicare Advantage Plans the option to use step therapy for Part B drugsbeginning January 1, 2019, and in October 2018, CMS proposed a new rule that would require direct-to-consumer television advertisements ofprescription drugs and biological products, for which payment is available through or under Medicare or Medicaid, to include in the advertisement theWholesale Acquisition Cost, or list price, of that drug or biological product. On January 31, 2019, the HHS Office of Inspector General proposedmodifications to U.S. federal Anti-Kickback Statute safe harbors which, among other things, will affect rebates paid by manufacturers to Medicare PartD plans, the purpose of which is to further reduce the cost of drug products to consumers. Although some of these, and other proposals will requireauthorization through additional legislation to become effective, Congress and the Trump administration have each indicated that it will continue toseek new legislative and/or administrative measures to control drug costs At the state level, legislatures have increasingly passed legislation andimplemented regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, discounts,restrictions on certain product access and 57Table of Contentsmarketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.Additionally, on May 30, 2018, the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, orthe Right to Try Act, was signed into law. The law, among other things, provides a federal framework for certain patients to access certaininvestigational new drug products that have completed a Phase 1 clinical trial and that are undergoing investigation for FDA approval. Under certaincircumstances, eligible patients can seek treatment without enrolling in clinical trials and without obtaining FDA permission under the FDA expandedaccess program. There is no obligation for a pharmaceutical manufacturer to make its drug products available to eligible patients as a result of theRight to Try Act.U.S. Foreign Corrupt Practices ActThe U.S. Foreign Corrupt Practices Act of 1977, or FCPA, prohibits U.S. corporations and individuals from engaging in certain activities toobtain or retain business or secure any improper advantage, or to influence a person working in an official capacity. It is illegal to pay, offer to pay orauthorize the payment of anything of value to any employee or official of a foreign government or public international organization, or political party,political party official, or political candidate in an attempt to obtain or retain business or to otherwise influence a person working in an officialcapacity. The scope of the FCPA also includes employees and officials of state-owned or controlled enterprises, which may include healthcareprofessionals in many countries. Equivalent laws have been adopted in other foreign countries that impose similar obligations.Government Regulation Outside of the United StatesIn addition to regulations in the United States, we will be subject to a variety of regulations in other jurisdictions governing, among otherthings, clinical trials and any commercial sales and distribution of our products. Because biologically sourced raw materials are subject to uniquecontamination risks, their use may be restricted in some countries.Whether or not we obtain FDA approval of a product, we must obtain the requisite approvals from regulatory authorities in foreigncountries prior to the commencement of clinical trials or marketing of the product in those countries. Certain countries outside of the United Stateshave a similar process that requires the submission of a clinical trial application, or CTA, much like the IND prior to the commencement of humanclinical trials. In the European Union, for example, a CTA must be submitted to each country’s national health authority and an independent ethicscommittee, much like the FDA and the IRB, respectively. Once the CTA is approved in accordance with a country’s requirements, clinical trialdevelopment may proceed.The requirements and process governing the conduct of clinical trials, product licensing, pricing and reimbursement vary from country tocountry. In all cases, the clinical trials are conducted in accordance with GCP and the applicable regulatory requirements and the ethical principles thathave their origin in the Declaration of Helsinki. If we fail to comply with applicable foreign regulatory requirements, we may be subject to, amongother things, fines, suspension or withdrawal of regulatory approvals, product recalls, seizure of products, operating restrictions and criminalprosecution.Clinical TrialsClinical trials of medicinal products in the European Union must be conducted in accordance with European Union and nationalregulations and the International Conference on Harmonization, or ICH, guidelines on GCPs. Additional GCP guidelines from the EuropeanCommission, focusing in particular on traceability, apply to clinical trials of advanced therapy medicinal products. If the sponsor of the clinical trial isnot established within the European Union, it must appoint an entity within the European Union to act as its legal representative. The sponsor musttake out a clinical trial insurance policy, and in most EU countries, the sponsor is liable to provide ‘no fault’ compensation to any study subject injuredin the clinical trial. 58Table of ContentsPrior to commencing a clinical trial, the sponsor must obtain a clinical trial authorization from the competent authority, and a positiveopinion from an independent ethics committee. The application for a clinical trial authorization must include, among other things, a copy of the trialprotocol and an investigational medicinal product dossier containing information about the manufacture and quality of the medicinal product underinvestigation. Currently, clinical trial authorization applications must be submitted to the competent authority in each EU Member State in which thetrial will be conducted. Under the new Regulation on Clinical Trials, which is currently expected to take effect in 2019, there will be a centralizedapplication procedure where one national authority takes the lead in reviewing the application and the other national authorities have only a limitedinvolvement. Any substantial changes to the trial protocol or other information submitted with the clinical trial applications must be notified to orapproved by the relevant competent authorities and ethics committees. Medicines used in clinical trials must be manufactured in accordance withcGMP. Other national and European Union-wide regulatory requirements also apply.During the development of a medicinal product, the EMA and national medicines regulators within the European Union provide theopportunity for dialogue and guidance on the development program. At the EMA level, this is usually done in the form of scientific advice, which isgiven by the Scientific Advice Working Party of the Committee for Medicinal Products for Human Use, or CHMP. A fee is incurred with each scientificadvice procedure. Advice from the EMA is typically provided based on questions concerning, for example, quality (chemistry, manufacturing andcontrols testing), nonclinical testing and clinical trials, and pharmacovigilance plans and risk-management programs. Advice is not legally bindingwith regard to any future marketing authorization application of the product concerned.Marketing AuthorizationsTo obtain regulatory approval of an investigational biological product under EU regulatory systems, we must submit a marketingauthorization application. The application used to file the BLA in the United States is similar to that required in the European Union, with theexception of, among other things, country-specific document requirements. The process for doing this depends, among other things, on the nature ofthe medicinal product.The centralized procedure results in a single marketing authorization, or MA, granted by the European Commission that is valid across theEEA (i.e., the European Union as well as Iceland, Liechtenstein and Norway). The centralized procedure is compulsory for human drugs that are:(i) derived from biotechnology processes, such as genetic engineering, (ii) contain a new active substance indicated for the treatment of certaindiseases, such as HIV/AIDS, cancer, diabetes, neurodegenerative diseases, autoimmune and other immune dysfunctions and viral diseases,(iii) officially designated orphan medicines and (iv) advanced-therapy medicines, such as gene therapy, somatic cell therapy or tissue-engineeredmedicines. The centralized procedure may at the request of the applicant also be used in certain other cases. Therefore, the centralized procedure wouldbe mandatory for the products we are developing.The Committee for Advanced Therapies, or CAT, is responsible in conjunction with the CHMP for the evaluation of ATMPs. The CAT isprimarily responsible for the scientific evaluation of ATMPs and prepares a draft opinion on the quality, safety and efficacy of each ATMP for which amarketing authorization application is submitted. The CAT’s opinion is then taken into account by the CHMP when giving its final recommendationregarding the authorization of a product in view of the balance of benefits and risks identified. Although the CAT’s draft opinion is submitted to theCHMP for final approval, the CHMP may depart from the draft opinion, if it provides detailed scientific justification. The CHMP and CAT are alsoresponsible for providing guidelines on ATMPs and have published numerous guidelines, including specific guidelines on gene therapies and celltherapies. These guidelines provide additional guidance on the factors that the EMA will consider in relation to the development and evaluation ofATMPs and include, among other things, the preclinical studies required to characterize ATMPs; the manufacturing and control information thatshould be submitted in a marketing authorization application; and post-approval measures required to monitor patients and evaluate the long term 59Table of Contentsefficacy and potential adverse reactions of ATMPs. Although these guidelines are not legally binding, we believe that our compliance with them islikely necessary to gain and maintain approval for any of our product candidates.Under the centralized procedure in the European Union, the maximum timeframe for the evaluation of an MAA by the EMA is 210 days.This excludes so-called clock stops, during which additional written or oral information is to be provided by the applicant in response to questionsasked by the CHMP. At the end of the review period, the CHMP provides an opinion to the European Commission. If this is opinion favorable, theCommission may then adopt a decision to grant an MA. In exceptional cases, the CHMP might perform an accelerated review of an MAA in no morethan 150 days (not including clock stops). This is usually when the product is of major interest from the point of view of public health and, inparticular, from the viewpoint of therapeutic innovation.The European Commission may grant a so-called “conditional marketing authorization” prior to obtaining the comprehensive clinical datarequired for an application for a full marketing authorization. Such conditional marketing authorizations may be granted for product candidates(including medicines designated as orphan medicinal products), if (i) the risk-benefit balance of the product candidate is positive, (ii) it is likely thatthe applicant will be in a position to provide the required comprehensive clinical trial data, (iii) the product fulfills an unmet medical need and (iv) thebenefit to public health of the immediate availability on the market of the medicinal product concerned outweighs the risk inherent in the fact thatadditional data are still required. A conditional marketing authorization may contain specific obligations to be fulfilled by the marketing authorizationholder, including obligations with respect to the completion of ongoing or new studies, and with respect to the collection of pharmacovigilance data.Conditional marketing authorizations are valid for one year, and may be renewed annually, if the risk-benefit balance remains positive, and after anassessment of the need for additional or modified conditions and/or specific obligations. The timelines for the centralized procedure described abovealso apply with respect to the review by the CHMP of applications for a conditional marketing authorization.The European Commission may also grant a so-called “marketing authorization under exceptional circumstances”. Such authorization isintended for products for which the applicant can demonstrate that it is unable to provide comprehensive data on the efficacy and safety under normalconditions of use, because the indications for which the product in question is intended are encountered so rarely that the applicant cannot reasonablybe expected to provide comprehensive evidence, or in the present state of scientific knowledge, comprehensive information cannot be provided, or itwould be contrary to generally accepted principles of medical ethics to collect such information. Consequently, marketing authorization underexceptional circumstances may be granted subject to certain specific obligations, which may include the following: • the applicant must complete an identified program of studies within a time period specified by the competent authority, the resultsof which form the basis of a reassessment of the benefit/risk profile; • the medicinal product in question may be supplied on medical prescription only and may in certain cases be administered onlyunder strict medical supervision, possibly in a hospital and in the case of a radio-pharmaceutical, by an authorized person; andThe package leaflet and any medical information must draw the attention of the medical practitioner to the fact that the particularsavailable concerning the medicinal product in question are as yet inadequate in certain specified respects.A marketing authorization under exceptional circumstances is subject to annual review to reassess the risk-benefit balance in an annualreassessment procedure. Continuation of the authorization is linked to the annual reassessment and a negative assessment could potentially result inthe marketing authorization being suspended or revoked. The renewal of a marketing authorization of a medicinal product under exceptional 60Table of Contentscircumstances, however, follows the same rules as a “normal” marketing authorization. Thus, a marketing authorization under exceptionalcircumstances is granted for an initial five years, after which the authorization will become valid indefinitely, unless the EMA decides that safetygrounds merit one additional five-year renewal. A marketing authorization under exceptional circumstances should not be granted when a conditionalmarketing authorization is more appropriate.The European Union medicines rules expressly permit the EU Member States to adopt national legislation prohibiting or restricting thesale, supply or use of any medicinal product containing, consisting of or derived from a specific type of human or animal cell, such as embryonic stemcells. While the products we have in development do not make use of embryonic stem cells, it is possible that the national laws in certain EU MemberStates may prohibit or restrict us from commercializing our products, even if they have been granted an MA.Data and Marketing ExclusivityThe European Union also provides opportunities for market exclusivity. Marketing authorization applications for generic medicinalproducts do not need to include the results of preclinical and clinical trials, but instead can refer to the data included in the marketing authorization ofa reference product for which regulatory data exclusivity has expired. In the European Union, upon receiving marketing authorization, new chemicalentities generally receive eight years of data exclusivity and an additional two years of market exclusivity. The two-year period may be extended tothree years if during the first eight years a new therapeutic indication with significant clinical benefit over existing therapies is approved. If granted,data exclusivity prevents regulatory authorities in the European Union from referencing the innovator’s data to assess a generic application. During theadditional two-year period of market exclusivity, a generic marketing authorization can be submitted, and the innovator’s data may be referenced, butno generic product can be marketed until the expiration of the market exclusivity. However, there is no guarantee that a product will be considered bythe EU regulatory authorities to be a new chemical entity, and products may not qualify for data exclusivity.There is a special regime for biosimilars, or biological medicinal products that are similar to a reference medicinal product but that do notmeet the definition of a generic medicinal product, for example, because of differences in raw materials or manufacturing processes. For such products,the results of appropriate preclinical or clinical trials must be provided, and guidelines from the EMA detail the type of quantity of supplementary datato be provided for different types of biological product. There are no such guidelines for complex biological products, such as gene or cell therapymedicinal products, and so it is unlikely that biosimilars of those products will currently be approved in the European Union. However, guidance fromthe EMA states that they will be considered in the future in light of the scientific knowledge and regulatory experience gained at the time.Orphan Medicinal ProductsProducts receiving orphan designation in the European Union can receive ten years of market exclusivity. During the ten-year marketexclusivity period, the EMA cannot accept another application for a marketing authorization, or grant a marketing authorization or accept anapplication to extend an existing marketing authorization, for the same therapeutic indication, in respect of a similar medicinal product. An orphanproduct can also obtain an additional two years of market exclusivity in the European Union for pediatric studies. No extension to any supplementaryprotection certificate can be granted on the basis of pediatric studies for orphan indications.The criteria for designating an “orphan medicinal product” in the European Union are similar in principle to those in the United States.Under Article 3 of Regulation (EC) 141/2000, a medicinal product may be designated as orphan if (1) it is intended for the diagnosis, prevention ortreatment of a life-threatening or chronically debilitating condition; (2) either (a) such condition affects no more than five in 10,000 persons in the 61Table of ContentsEuropean Union when the application is made, or (b) the product, without the benefits derived from orphan status, would not generate sufficient returnin the European Union to justify investment; and (3) there exists no satisfactory method of diagnosis, prevention or treatment of such conditionauthorized for marketing in the European Union, or if such a method exists, the product will be of significant benefit to those affected by the condition,as defined in Regulation (EC) 847/2000. Orphan medicinal products are eligible for financial incentives such as reduction of fees or fee waivers. Theapplication for orphan drug designation must be submitted before the MAA. The applicant will receive a fee reduction for the MAA if the orphan drugdesignation has been granted, but not if the designation is still pending at the time the marketing authorization is submitted. Orphan drug designationdoes not convey any advantage in, or shorten the duration of, the regulatory review and approval process.The ten-year market exclusivity may be reduced to six years if, at the end of the fifth year, it is established that the product no longer meetsthe criteria for orphan designation, for example, if the product is sufficiently profitable not to justify maintenance of market exclusivity. Additionally,an MA may be granted to a similar product for the same indication at any time if: • the second applicant can establish that its product, although similar, is safer, more effective or otherwise clinically superior; • the applicant consents to a second orphan medicinal product application; or • the applicant cannot supply enough orphan medicinal product.Pediatric Investigation PlanIn the EMA, MAAs for new medicinal products not authorized have to include the results of trials conducted in the pediatric population, incompliance with a pediatric investigation plan, or PIP, agreed with the EMA’s Pediatric Committee, or PDCO. The PIP sets out the timing and measuresproposed to generate data to support a pediatric indication of the drug for which an MA is being sought. The PDCO can grant a deferral of theobligation to implement some or all of the measures of the PIP until there are sufficient data to demonstrate the efficacy and safety of the product inadults. Further, the obligation to provide pediatric clinical trial data can be waived by the PDCO when these data are not needed or appropriate becausethe product is likely to be ineffective or unsafe in children, the disease or condition for which the product is intended occurs only in adult populations,or when the product does not represent a significant therapeutic benefit over existing treatments for pediatric patients. Once the MA is obtained in allEU Member States and trial results are included in the product information, even when negative, the product is eligible for a six-months supplementaryprotection certificate extension.Post-Approval ControlsThe holder of an MA must establish and maintain a pharmacovigilance system and appoint an individual qualified person forpharmacovigilance who is responsible for oversight of that system. Key obligations include expedited reporting of suspected serious adverse reactionsand submission of periodic safety update reports, or PSURs.All new MAAs must include a risk management plan, or RMP, describing the risk management system that the company will put in placeand documenting measures to prevent or minimize the risks associated with the product. The regulatory authorities may also impose specificobligations as a condition of the marketing authorization. Such risk-minimization measures or post-authorization obligations may include additionalsafety monitoring, more frequent submission of PSURs, or the conduct of additional clinical trials or post-authorization safety studies. RMPs andPSURs are routinely available to third parties requesting access, subject to limited redactions.All advertising and promotional activities for the product must be consistent with the approved summary of product characteristics, andtherefore all off-label promotion is prohibited. Direct-to-consumer 62Table of Contentsadvertising of prescription medicines is also prohibited in the European Union. Although general requirements for advertising and promotion ofmedicinal products are established under EU directives, the details are governed by regulations in each EU Member State and can differ from onecountry to another.Pricing and ReimbursementGovernments influence the price of medicinal products in the European Union through their pricing and reimbursement rules and controlof national healthcare systems that fund a large part of the cost of those products to consumers. Some jurisdictions operate positive and negative listsystems under which products may only be marketed once a reimbursement price has been agreed. To obtain reimbursement or pricing approval, someof these countries may require the completion of clinical trials that compare the cost-effectiveness of a particular product candidate to currentlyavailable therapies. Other EU Member States allow companies to fix their own prices for medicines, but monitor and control company profits. Thedownward pressure on healthcare costs in general, particularly prescription medicines, has become very intense. As a result, increasingly high barriersare being erected to the entry of new products.PRIME SchemeIn July 2016 the EMA launched PRIME scheme. PRIME is a voluntary scheme aimed at enhancing the EMA’s support for the developmentof medicines that target unmet medical needs. It is based on increased interaction and early dialogue with companies developing promising medicines,to optimize their product development plans and speed up their evaluation to help them reach patients earlier. Product developers that benefit fromPRIME designation can expect to be eligible for accelerated assessment but this is however not guaranteed. The benefits of a PRIME designationincludes the appointment of a rapporteur from the CHMP before submission of an MAA, early dialogue and scientific advice at key developmentmilestones, and the potential to qualify products for accelerated review earlier in the application process.UK Specials RegulationThe UK’s Human Medicines Regulations 2012 allow for the manufacture and supply of medicinal products not authorized for marketing topatients with special needs at the request of the healthcare professional responsible for the patient’s care (these products are referred to as “specials”). Aspecial may only be supplied in: (i) response to an unsolicited order from a healthcare professional responsible for the care of the patient, (ii) theproduct is manufactured and assembled in accordance with the specifications of that healthcare professional to fulfil the special needs of the individualpatient which cannot be met by products already authorized for marketing, and (iii) the product is manufactured under a specials license granted by theUK’s MHRA.Manufacturing a special also imposes a five year record retention requirements subject to review by the MHRA, including details of anysuspected adverse reaction to the product so sold or supplied of which the person is aware or subsequently becomes aware, as well as a continuingobligation to notify the MHRA of any suspected adverse reaction to the medicinal product which is a serious adverse reaction.Privacy and Data Protection LawsWe are also subject to laws and regulations in non-U.S. countries covering data privacy and the protection of health-related and otherpersonal information. EU member states and other jurisdictions have adopted data protection laws and regulations, which impose significantcompliance obligations. Laws and regulations in these jurisdictions apply broadly to the collection, use, storage, disclosure, processing and security ofpersonal information that identifies or may be used to identify an individual, such as names, contact information, and sensitive personal data such ashealth data. These laws and regulations are subject to frequent revisions and differing interpretations, and have generally become more stringent overtime. 63Table of ContentsThe General Data Protection Regulation, or GDPR, is a European framework law which imposes many requirements for controllers andprocessors of personal data, including, for example, higher standards for obtaining consent from individuals, if this is required, to process their personaldata, more robust disclosures to individuals and a strengthened individual data rights regime, shortened timelines for data breach notifications,limitations on retention and secondary use of personal data, increased requirements pertaining to health data and pseudonymised (i.e., key-coded) dataand additional obligations when we contract third-party processors in connection with the processing of t personal data. The GDPR allows EU memberstates to make additional laws and regulations further limiting the processing of genetic, biometric or health data. Failure to comply with therequirements of GDPR and the applicable national data protection laws of the EU member states may result in fines of up to €20,000,000 or up to 4% ofthe total worldwide annual turnover of the preceding financial year, whichever is higher, and other administrative penalties.The United Kingdom is expected to leave the European Union on March 29, 2019 further to the “Brexit” referendum. The UK’s DataProtection Act 2018, or DPA2018, governs the UK’s privacy regime and will continue to do so after the United Kingdom exits the EU. The DPA2018incorporates the GDPR’s text in full, with only minor amendments and further derogations including those pertaining to the processing of health data.Accordingly, the terms of the GDPR, and its significant penalties, will continue to apply after the UK exits the EU. At time of writing, it is still unclearwhether the UK will be granted adequacy by the European Commission. In any event, for, at least, an interim period, export mechanisms, such asModel Clauses, will likely need to be put in place to govern the transfer of personal data from the EU to the UK.EmployeesAs of December 31, 2018, we had 68 employees, all of which are full-time employees and 12 of which have M.D. or Ph.D. degrees. Of thesefull-time employees, 54 are engaged in research and development activities. None of our employees is subject to a collective bargaining agreement orrepresented by a trade or labor union. We consider our relationship with our employees to be good.Corporate InformationMeiraGTx Holdings plc was formed on May 1, 2018 under the laws of the Cayman Islands. Our predecessor, MeiraGTx Limited, a limitedcompany under the laws of England and Wales, was formed on March 20, 2015. In connection with our IPO, we reorganized whereby MeiraGTxLimited became a wholly owned subsidiary of MeiraGTx Holdings plc.Available InformationWe file annual, quarterly and current reports, proxy statements and other information with the U.S. Securities and Exchange Commission(“SEC”). Our SEC filings are available to the public over the Internet at the SEC’s website at http://www.sec.gov. Our SEC filings are also availableunder the Investors and Media section of our website at www.meiragtx.com. Our website and the information contained on or connected to that site arenot incorporated into this Form 10-K. 64Table of ContentsITEM 1A.RISK FACTORSYou should carefully consider the risks and uncertainties described below and the other information in this Form 10-K before making aninvestment in our ordinary shares. Our business, financial condition, results of operations or prospects could be adversely affected if any of theserisks occurs, and as a result, the market price of our ordinary shares could decline and you could lose all or part of your investment. This Form 10-Kalso contains forward-looking statements that involve risks and uncertainties. See “Special Note Regarding Forward-Looking Statements.” Ouractual results could differ materially from those anticipated in these forward-looking statements as a result of certain factors, including those setforth below.Risks Related to Our Financial Position and Need for Additional CapitalWe have incurred significant losses since inception and anticipate that we will incur continued losses for the foreseeable future, and may neverachieve or maintain profitability.We are a clinical stage company with limited operating history. We were formed and began operations in 2015. We have never beenprofitable and do not expect to be profitable in the foreseeable future. We have incurred net losses since inception, including net losses ofapproximately $82.9 million and $31.0 million for the years ended December 31, 2018 and December 31, 2017, respectively. As of December 31,2018, we had an accumulated deficit of approximately $148.3 million. Since our inception, we have devoted substantially all of our resources todeveloping our technology platform, establishing our viral vector manufacturing facility and developing manufacturing processes, advancing theproduct candidates in our ophthalmology, salivary gland and neurodegenerative disease programs, building our intellectual property portfolio,organizing and staffing our company, business planning, raising capital, and providing general and administrative support for these operations. Wehave not yet demonstrated an ability to successfully complete a clinical program, including large-scale, pivotal clinical trials, obtain marketingapproval, manufacture product at a commercial scale, or arrange for a third party to do so on our behalf, or conduct sales and marketing activitiesnecessary for successful product commercialization. Typically, it takes about six to ten years to develop a new drug from the time it enters Phase 1clinical trials to when it is approved for treating patients, but in many cases it may take longer. Consequently, predictions about our future success orviability may not be as accurate as they could be if we had a longer operating history or a history of successfully developing and commercializinggenetic medicine products.We expect to continue to incur significant expenses and additional operating losses for the foreseeable future as we seek to advanceproduct candidates through preclinical and clinical development, expand our research and development activities, develop new product candidates,complete clinical trials, seek regulatory approval and, if we receive regulatory approval, commercialize our products. Furthermore, the costs ofadvancing product candidates into each succeeding clinical phase tend to increase substantially over time. The total costs to advance any of ourproduct candidates to marketing approval in even a single jurisdiction would be substantial. Because of the numerous risks and uncertaintiesassociated with gene therapy product development, we are unable to accurately predict the timing or amount of increased expenses or when, or if, wewill be able to begin generating revenue from the commercialization of products or achieve or maintain profitability. Our expenses will also increasesubstantially as we operate as a public company and add clinical, scientific, operational, financial and management information systems and personnel,including personnel to support our product development and planned future commercialization efforts, as well as to support our transition to a publicreporting company.Before we generate any revenue from product sales, each of our programs and product candidates will require additional preclinical and/orclinical development, potential regulatory approval in multiple jurisdictions, manufacturing, building of a commercial organization, substantialinvestment and significant marketing efforts. Our expenses could increase beyond expectations if we are required by the U.S. Food and DrugAdministration, or FDA, European Medicines Agency, or EMA, or other regulatory authorities to perform preclinical studies and clinical trials inaddition to those that we currently anticipate. These risks are further described under “—Risks Related to Discovery, Development, Clinical Testing,Manufacturing and Regulatory Approval” and “—Risks 65Table of ContentsRelated to Commercialization.” As a result, we expect to continue to incur net losses and negative cash flows for the foreseeable future. These netlosses and negative cash flows have had, and will continue to have, an adverse effect on our shareholders’ equity and working capital.As we continue to build our business, we expect our financial condition and operating results may fluctuate significantly from quarter toquarter and year to year due to a variety of factors, many of which are beyond our control. Accordingly, you should not rely upon the results of anyparticular quarterly or annual period as indications of future operating performance. If we are unable to develop and commercialize one or more of ourproduct candidates either alone or with collaborators, or if revenues from any product candidate that receives marketing approval are insufficient, wewill not achieve profitability. Even if we do achieve profitability, we may not be able to sustain or increase profitability. If we are unable to achieveand then maintain profitability, the value of our equity securities will be adversely affected.We will require additional capital to fund our operations, which may not be available on acceptable terms, if at all.We expect to spend substantial amounts to complete the development of, seek regulatory approvals for and commercialize our productcandidates. We will require additional capital, which we may raise through equity offerings, debt financings, marketing and distribution arrangementsand other collaborations, strategic alliances and licensing arrangements or other sources to enable us to complete the development and potentialcommercialization of our product candidates. Furthermore, we expect to continue to incur costs associated with operating as a public company.Adequate additional financing may not be available to us on acceptable terms, or at all. Our failure to raise capital as and when needed would have anegative effect on our financial condition and our ability to pursue our business strategy. In addition, attempting to secure additional financing maydivert the time and attention of our management from day-to-day activities and harm our product candidate development efforts. If we are unable toraise capital when needed or on acceptable terms, we would be forced to delay, reduce or eliminate certain of our research and development programs.Our operations have consumed significant amounts of cash since inception. As of December 31, 2018, our cash and cash equivalents were$68.1 million. Additionally, during the period from January 1, 2019 through the date of this filing, we have received additional funds in the aggregateamount of approximately $77.4 million and expect to receive an additional $100 million pursuant to the terms of the Collaboration Agreement.Therefore, based on our cash and cash equivalents after receiving the additional $177.4 million, we estimate that such funds will be sufficient to enableus to fund our operating expenses and capital expenditure requirements into 2022. This estimate is based on assumptions that may prove to be wrong,and we could use our available capital resources sooner than we currently expect. Changing circumstances could cause us to consume capitalsignificantly faster than we currently anticipate, and we may need to spend more than currently expected because of circumstances beyond our control.Because the length of time and activities associated with successful development of our product candidates is highly uncertain, we are unable toestimate the actual funds we will require for development and any approved marketing and commercialization activities. Our future fundingrequirements, both near and long-term, will depend on many factors, including, but not limited to: • the progress, timing, costs and results of our ongoing clinical development for our CNGB3 gene therapy product candidate,AAV-CNGB3, for our RPE65-deficiency product candidate, AAV-RPE65, for our X-linked retinitis pigmentosa product candidate,AAV-RPGR, for our radiation induced xerostomia and Sjogren’s syndrome-associated xerostomia or xerophthalmia productcandidate, AAV-AQP1, and continue to conduct our ongoing natural history studies for inherited retinal diseases, or IRDs; • the initiation of Phase 1/2 clinical trials for our CNGA3 gene therapy product candidate, AAV-CNGA3, and for our productcandidate for the treatment of xerostomia associated with Sjogren’s syndrome, AAV-AQP1; 66Table of Contents • future discussions with regulatory agencies and potential subsequent initiation of future clinical trials for our product candidate forthe treatment of Parkinson’s disease, AAV-GAD; • continuing our current research programs, our preclinical development of product candidates from our current research programsand further developing our gene regulation technology; • seeking to identify, assess, acquire and/or develop additional research programs and additional product candidates; • the preclinical testing and clinical trials for any product candidates we identify and develop; • establishing a sales, marketing and distribution infrastructure to commercialize any product candidates for which we may obtainmarketing approval; • the outcome, timing and cost of meeting regulatory requirements established by the FDA, EMA and other regulatory authorities; • the cost of expanding and protecting our intellectual property portfolio, including filing, prosecuting, defending and enforcing ourpatent claims and other intellectual property rights; • the cost of defending potential intellectual property disputes, including patent infringement actions brought by third partiesagainst us or any of our product candidates; • the effect of competing technological and market developments; • the cost of further developing and scaling our manufacturing facility and processes; • the cost and timing of completion of commercial-scale manufacturing activities; • the cost of making royalty, milestone or other payments under current and any future in-license agreements; • the extent to which we in-license or acquire other products and technologies; • the cost of establishing sales, marketing and distribution capabilities for our product candidates in regions where we choose tocommercialize our products; and • the initiation, progress, timing and results of our commercialization of our product candidates, if approved for commercial sale.We cannot be certain that additional funding will be available on acceptable terms, or at all. If we are unable to raise additional capital insufficient amounts or on terms acceptable to us, we may have to significantly delay, scale back or discontinue the development or commercializationof our product candidates or potentially discontinue operations.To the extent that we raise additional capital through the sale of equity or convertible debt securities, your ownership interest will bediluted, and the terms of these securities may include liquidation or other preferences that adversely affect your rights as a common shareholder. Debtfinancing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specificactions, such as incurring additional debt, making capital expenditures or declaring dividends. If we raise additional funds through collaborations,strategic alliances or marketing, distribution or licensing arrangements with third parties, we may be required to relinquish valuable rights to ourtechnologies, future revenue streams or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raiseadditional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development orfuture commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and marketourselves. 67Table of ContentsWe are heavily dependent on the success of our Most Advanced Product Candidates, which are still in development, and if none of them receiveregulatory approval or are successfully commercialized, our business may be harmed.To date, we have invested a significant portion of our efforts and financial resources in the development of AAV-CNGB3, AAV-CNGA3,AAV-RPGR, AAV-RPE65 and AAV-AQP1. Our future success and ability to generate product revenue is substantially dependent on our ability tosuccessfully develop, obtain regulatory approval for and successfully commercialize these product candidates. We currently have no products that areapproved for commercial sale and may never be able to develop marketable products. We expect to invest a meaningful portion of our efforts andexpenditures over the next few years in AAV-GAD, AAV-CNGB3, AAV-CNGA3, AAV-RPGR, AAV-RPE65 and AAV-AQP1 (the “Most AdvancedProduct Candidates”), which will require additional clinical development, management of clinical and manufacturing activities, regulatory approval inmultiple jurisdictions, manufacturing sufficient supply, building of a commercial organization, substantial investment and significant marketingefforts before we can generate any revenues from any commercial sales. Accordingly, our business currently depends heavily on the successfuldevelopment, regulatory approval and commercialization of our Most Advanced Product Candidates, which may never occur. We cannot be certainthat our Most Advanced Product Candidates will be successful in clinical trials, receive regulatory approval or be successfully commercialized even ifwe receive regulatory approval. Even if we receive approval to market our Most Advanced Product Candidates from the FDA, EMA or other regulatorybodies, we cannot be certain that our product candidate will be successfully commercialized, widely accepted in the marketplace or more effective thanother commercially available alternatives. Additionally, the research, testing, manufacturing, labeling, approval, sale, marketing and distribution ofgene therapy products are and will remain subject to extensive and evolving regulation by the FDA, EMA and other regulatory authorities. We are notpermitted to market our Most Advanced Product Candidates in the United States until they receive approval of a biologics license application, or BLA,from the FDA, we cannot market them in the European Union until we receive approval for a Marketing Authorization Application, or MAA, from theEMA, and we cannot market them in other countries until we receive any other required regulatory approval in those countries.AAV-GAD, AAV-CNGB3, AAV-CNGA3, AAV-RPGR, AAV-RPE65 and AAV-AQP1 are our most advanced product candidates, andbecause some of our other product candidates are based on similar technology, if our Most Advanced Product Candidates show unexpected adverseevents or a lack of efficacy in the indications we intend to treat, or if we experience other regulatory or developmental issues, our development plansand business could be significantly harmed. Further, competitors may be developing products with similar technology and may experience problemswith their products that could identify problems that would potentially harm our business.To date, we have not had any formal meetings with regulatory agencies nor determined the regulatory pathway and any potential relateddevelopment costs for our recently acquired AAV-GAD gene therapy program for Parkinson’s disease.We may not be successful in our efforts to identify additional product candidates.Part of our strategy involves identifying novel product candidates. The process by which we identify product candidates may fail to yieldproduct candidates for clinical development for a number of reasons, including those discussed in these risk factors and also: • we may not be able to assemble sufficient resources to acquire or discover additional product candidates; • competitors may develop alternatives that render our potential product candidates obsolete or less attractive; • potential product candidates we develop may nevertheless be covered by third parties’ patents or other exclusive rights; 68Table of Contents • potential product candidates may, on further study, be shown to have harmful side effects, toxicities or other characteristics thatindicate that they are unlikely to be products that will receive marketing approval and achieve market acceptance; • potential product candidates may not be effective in treating their targeted diseases; • the market for a potential product candidate may change so that the continued development of that product candidate is no longerreasonable; • a potential product candidate may not be capable of being produced in commercial quantities at an acceptable cost, or at all; or • the regulatory pathway for a potential product candidate may be too complex and difficult to navigate successfully oreconomically.In addition, we may choose to focus our efforts and resources on a potential product candidate that ultimately proves to be unsuccessful. Asa result, we may fail to capitalize on viable commercial products or profitable market opportunities, be required to forego or delay pursuit ofopportunities with other product candidates or other diseases that may later prove to have greater commercial potential, or relinquish valuable rights tosuch product candidates through collaboration, licensing or other royalty arrangements in cases in which it would have been advantageous for us toretain sole development and commercialization rights. If we are unable to identify additional suitable product candidates for clinical development, thiswould adversely impact our business strategy and our financial position and share price and could potentially cause us to cease operations.Risks Related to Discovery, Development, Clinical Testing, Manufacturing and Regulatory ApprovalWe intend to identify and develop product candidates based on our novel gene therapy platform, which makes it difficult to predict the time and costof product candidate development. Very few products that utilize transduction technology have been approved in the United States or in Europe, andthere have only been a limited number of clinical trials involving a gene therapy product candidate.We have concentrated a portion of our research and development efforts on our gene therapy platform, which uses both transduction andgene regulation technology. Our future success depends on the successful development of these novel therapeutic approaches. To date, very fewproducts that utilize gene transfer have been approved in the United States or Europe. There have been a limited number of clinical trials of genetransduction technologies, with only one product candidate ever approved by the FDA.Our gene therapy platform is based on a suite of viral vectors which we can deploy with gene therapy constructs, which relies on the abilityof AAV to efficiently transmit a therapeutic gene to certain kinds of cells. The mechanism of action by which these vectors target particular tissues isstill not completely understood. Therefore, it is difficult for us to determine that our vectors will be able to properly deliver gene transfer constructs toenough tissue cells to reach therapeutic levels. We cannot be certain that our viral vectors will be able to meet safety and efficacy levels needed to betherapeutic in humans or that they will not cause significant adverse events or toxicities. Furthermore, recent work conducted by a third party innon-human primates suggests that intravenous, or IV, delivery of certain AAV vectors at very high doses may result in severe toxicity. The indicationsthat we target do not use IV administration for viral vector delivery and do not use doses as high as those tested in these publications, and to date wehave not observed the severe toxicities described in these publications with the naturally occurring AAV vectors that we use. However, we cannot becertain that we will be able to avoid triggering toxicities in our future preclinical studies or clinical trials. Any such results could impact our ability todevelop a product candidate. As a result of these factors, it is more difficult for us to predict the time and cost of product candidate development, andwe cannot predict whether the application of our gene therapy platform, or any similar or competitive gene therapy platforms, will result in theidentification, development, and regulatory approval of any product candidates, or that other gene therapy technologies will not 69Table of Contentsbe considered better or more attractive. There can be no assurance that any development problems we experience in the future related to our genetherapy platform or any of our research programs will not cause significant delays or unanticipated costs, or that such development problems can besolved. We may also experience delays and challenges in utilizing our manufacturing facility and achieving sustainable, reproducible, and scalableproduction. Any of these factors may prevent us from completing our preclinical studies or clinical trials or commercializing any product candidateswe may develop on a timely or profitable basis, if at all.In addition, because our gene regulation technology is still in the research stage, we have not yet been able to assess safety in humans, andthere may be long-term effects from treatment that we cannot predict at this time. Also, animal models may not exist for some of the diseases we expectto pursue.Because gene therapy is novel and the regulatory landscape that governs any product candidates we may develop is uncertain and may change, wecannot predict the time and cost of obtaining regulatory approval, if we receive it at all, for any product candidates we may develop.The regulatory requirements that will govern any novel gene therapy product candidates we develop are not entirely clear and may change.Within the broader genetic medicine field, very few therapeutic products have received marketing authorization from the EMA and FDA. Even withrespect to more established products that fit into the categories of gene therapies or cell therapies, the regulatory landscape is still developing.Regulatory requirements governing gene therapy products and cell therapy products have changed frequently and will likely continue to change in thefuture. Moreover, there is substantial, and sometimes uncoordinated, overlap in those responsible for regulation of existing gene therapy products andcell therapy products. For example, in the United States, the FDA has established the Office of Tissues and Advanced Therapies within its Center forBiologics Evaluation and Research, or CBER, to consolidate the review of gene therapy and related products, and the Cellular, Tissue and GeneTherapies Advisory Committee to advise CBER on its review. Gene therapy clinical trials are also subject to review and oversight by an institutionalbiosafety committee, or IBC, a local institutional committee that reviews and oversees basic and clinical research conducted at the institutionparticipating in the clinical trial. Gene therapy clinical trials conducted at institutions that receive funding for recombinant DNA research from theUnited States National Institutes of Health, or the NIH, had historically been subject to review by the NIH Office of Biotechnology Activities’Recombinant DNA Advisory Committee, or the RAC, pursuant to the NIH Guidelines for Research Involving Recombinant DNA Molecules, or NIHGuidelines. Following an initial review, RAC members would make a recommendation as to whether the protocol raised important scientific, safety,medical, ethical or social issues that warranted in-depth discussion at the RAC’s quarterly meetings. Although the FDA decides whether individualgene therapy protocols may proceed under an IND, the RAC’s recommendations were shared with the FDA and the RAC public review process, ifundertaken, could have impeded or delayed the initiation of a clinical trial, even if the FDA had reviewed the trial and approved its initiation or hadnotified the sponsor that the study may begin. Conversely, the FDA can put an IND on clinical hold even if the RAC provided a favorable review or hasrecommended against an in-depth, public review.On August 17, 2018, the NIH issued a notice in the Federal Register and issued a public statement proposing changes to the oversightframework for gene therapy trials, including changes to the applicable NIH Guidelines to modify the roles and responsibilities of the RAC with respectto human clinical trials of gene therapy products, and requesting public comment on its proposed modifications. During the public comment period,which closed on October 16, 2018, the NIH has announced that it will no longer accept new human gene transfer protocols for review as part of theprotocol registration process under the existing NIH Guidelines or convene the RAC to review individual clinical protocols. These trials will remainsubject to the FDA’s oversight and other clinical trial regulations, and oversight at the local level will continue as otherwise set forth in the NIHGuidelines. Specifically, under the NIH Guidelines, supervision of human gene transfer trials includes evaluation and assessment by an IBC. The IBCassesses the safety of the research and identifies any potential risk to the public health or the environment, and such review may result in some delaybefore initiation of a clinical trial. While the NIH Guidelines are not mandatory unless the research in question is being conducted at or sponsored 70Table of Contentsby institutions receiving NIH funding of recombinant or synthetic nucleic acid molecule research, many companies and other institutions nototherwise subject to the NIH Guidelines voluntarily follow them. Even though we may not be required to submit a protocol for our gene therapyproduct candidates through the NIH for RAC review, we will still be subject to significant regulatory oversight by the FDA, and in addition to thegovernment regulators, the applicable IBC and institutional review board, or IRB, of each institution at which we or our collaborators conduct clinicaltrials of our product candidates, or a central IRB if appropriate, would need to review and approve the proposed clinical trial.In Europe, the EMA’s Committee for Advanced Therapies, or CAT, is responsible for assessing the quality, safety, and efficacy ofadvanced-therapy medicinal products. Advanced-therapy medicinal products include gene therapy medicines, somatic-cell therapy medicines andtissue-engineered medicines. The role of the CAT is to prepare a draft opinion on an application for marketing authorization for a gene therapymedicinal candidate that is submitted to the EMA. In the European Union, the development and evaluation of a gene therapy product must beconsidered in the context of the relevant EU guidelines. The EMA may issue new guidelines concerning the development and marketing authorizationfor gene therapy products and require that we comply with these new guidelines. As a result, the procedures and standards applied to gene therapyproducts and cell therapy products may be applied to any gene therapy product candidate we may develop, but that remains uncertain at this point.Adverse developments in preclinical studies or clinical trials conducted by others in the field of gene therapy and gene regulation productsmay cause the FDA, the EMA, and other regulatory bodies to revise the requirements for approval of any product candidates we may develop or limitthe use of products utilizing gene regulation technologies, either of which could harm our business. In addition, the clinical trial requirements of theFDA, the EMA, and other regulatory authorities and the criteria these regulators use to determine the safety and efficacy of a product candidate varysubstantially according to the type, complexity, novelty, and intended use and market of the potential products. The regulatory approval process forproduct candidates such as ours can be more expensive and take longer than for other, better known, or more extensively studied pharmaceutical orother product candidates. Further, as we are developing novel treatments for diseases in which there is little clinical experience with new endpoints andmethodologies, there is heightened risk that the FDA, the EMA or other regulatory bodies may not consider the clinical trial endpoints to provideclinically meaningful results, and the resulting clinical data and results may be more difficult to analyze. The prospectively designed natural historystudies with the same endpoints as our corresponding clinical trials may not be accepted by the FDA, EMA or other regulatory authorities. Regulatoryagencies administering existing or future regulations or legislation may not allow production and marketing of products utilizing gene regulationtechnology in a timely manner or under technically or commercially feasible conditions. In addition, regulatory action or private litigation could resultin expenses, delays, or other impediments to our research programs or the commercialization of resulting products.The regulatory review committees and advisory groups described above and the new guidelines they promulgate may lengthen theregulatory review process, require us to perform additional preclinical studies or clinical trials, increase our development costs, lead to changes inregulatory positions and interpretations, delay or prevent approval and commercialization of these treatment candidates, or lead to significant post-approval limitations or restrictions. As we advance our research programs and develop future product candidates, we will be required to consult withthese regulatory and advisory groups and to comply with applicable guidelines. If we fail to do so, we may be required to delay or discontinuedevelopment of any product candidates we identify and develop.Clinical trials are expensive, time-consuming, difficult to design and implement, and involve an uncertain outcome. Further, we may encountersubstantial delays in our clinical trials.The clinical trials and manufacturing of our product candidates are, and the manufacturing and marketing of our products, if approved, willbe, subject to extensive and rigorous review and regulation by numerous government authorities in the United States and in other countries where weintend to test and market 71Table of Contentsour product candidates. Before obtaining regulatory approvals for the commercial sale of any of our product candidates, we must demonstrate throughlengthy, complex and expensive preclinical testing and clinical trials that our product candidates are both safe and effective for use in each targetindication. In particular, because our product candidates are subject to regulation as biological drug products, we will need to demonstrate that they aresafe, pure, and potent for use in their target indications. Each product candidate must demonstrate an adequate risk versus benefit profile in its intendedpatient population and for its intended use.Clinical testing is expensive and can take many years to complete, and is subject to uncertainty. We cannot guarantee that any clinicaltrials will be conducted as planned or completed on schedule, if at all. Failure can occur at any time during the clinical trial process. Even if our futureclinical trials are completed as planned, we cannot be certain that their results will support the safety and effectiveness of our product candidates fortheir targeted indications. Our future clinical trial results may not be successful.In addition, even if such trials are successfully completed, we cannot guarantee that the FDA or foreign regulatory authorities will interpretthe results as we do, and more trials could be required before we submit our product candidates for approval. To the extent that the results of the trialsare not satisfactory to the FDA or foreign regulatory authorities for support of a marketing application, we may be required to expend significantresources, which may not be available to us, to conduct additional trials in support of potential approval of our product candidates.To date, we have not completed any clinical trials required for the approval of our product candidates. Although we have already begunPhase 1/2 clinical trials, we may experience delays in conducting any clinical trials and we do not know whether our clinical trials will begin on time,need to be redesigned, recruit and enroll patients on time or be completed on schedule, or at all. Events that may prevent successful or timelycompletion of clinical development include: • inability to generate sufficient preclinical, toxicology, or other in vivo or in vitro data to support the initiation of clinical trials; • delays in sufficiently developing, characterizing or controlling a manufacturing process suitable for advanced clinical trials; • delays in developing suitable assays for screening patients for eligibility for trials with respect to certain product candidates; • delays in reaching agreement with the FDA, EMA or other regulatory authorities as to the design or implementation of our clinicaltrials; • obtaining regulatory approval to commence a clinical trial; • reaching an agreement on acceptable terms with clinical trial sites or prospective CROs, the terms of which can be subject toextensive negotiation and may vary significantly among different clinical trial sites; • obtaining institutional review board, or IRB, approval at each site; • recruiting suitable patients to participate in a clinical trial; • developing and validating the companion diagnostic to be used in a clinical trial, if applicable; • having patients complete a clinical trial or return for post-treatment follow-up; • clinical sites, contract research organizations, or other third parties deviating from trial protocol or dropping out of a trial; • failure to perform in accordance with the FDA’s good clinical practice, or GCP, requirements, or applicable regulatory guidelines inother countries; 72Table of Contents • addressing patient safety concerns that arise during the course of a trial, including occurrence of adverse events associated with theproduct candidate that are viewed to outweigh its potential benefits; • adding a sufficient number of clinical trial sites; or • manufacturing sufficient quantities of product candidate for use in clinical trials.We may experience numerous unforeseen events during, or as a result of, clinical trials that could delay or prevent our ability to receivemarketing approval or commercialize our product candidates or significantly increase the cost of such trials, including: • we may experience changes in regulatory requirements or guidance, or receive feedback from regulatory authorities that requires usto modify the design of our clinical trials; • clinical trials of our product candidates may produce negative or inconclusive results, and we may decide, or regulators may requireus, to conduct additional clinical trials or abandon development programs; • the number of patients required for clinical trials of our product candidates may be larger than we anticipate, enrollment in theseclinical trials may be slower than we anticipate, or participants may drop out of these clinical trials at a higher rate than weanticipate; • our third-party contractors may fail to comply with regulatory requirements or meet their contractual obligations to us in a timelymanner, or at all; • we or our investigators might have to suspend or terminate clinical trials of our product candidates for various reasons, includingnon-compliance with regulatory requirements, a finding that our product candidates have undesirable side effects or otherunexpected characteristics, or a finding that the participants are being exposed to unacceptable health risks; • the cost of clinical trials of our product candidates may be greater than we anticipate and we may not have funds to cover the costs; • the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates maybe insufficient or inadequate; • regulators may revise the requirements for approving our product candidates, or such requirements may not be as we anticipate; and • any future collaborators that conduct clinical trials may face any of the above issues, and may conduct clinical trials in ways theyview as advantageous to them but that are suboptimal for us.If we are required to conduct additional clinical trials or other testing of our product candidates beyond those that we currentlycontemplate, if we are unable to successfully complete clinical trials of our product candidates or other testing, if the results of these trials or tests arenot positive or are only modestly positive or if there are safety concerns, we may: • incur unplanned costs; • be delayed in obtaining marketing approval for our product candidates or not obtain marketing approval at all; • obtain marketing approval in some countries and not in others; • obtain marketing approval for indications or patient populations that are not as broad as intended or desired; • obtain marketing approval with labeling that includes significant use or distribution restrictions or safety warnings, includingboxed warnings; 73Table of Contents • be subject to additional post-marketing testing requirements; or • have the product removed from the market after obtaining marketing approval.We could encounter delays if a clinical trial is suspended or terminated by us, by the IRBs of the institutions in which such trials are beingconducted, by the Data Safety Monitoring Board, or DSMB, for such trial or by the FDA, EMA or other regulatory authorities. Such authorities mayimpose such a suspension or termination due to a number of factors, including failure to conduct the clinical trial in accordance with regulatoryrequirements or our clinical protocols, inspection of the clinical trial operations or trial site by the FDA, EMA or other regulatory authorities resultingin the imposition of a clinical hold, unforeseen safety issues or adverse side effects, failure to demonstrate a benefit from using a drug, changes ingovernmental regulations or administrative actions or lack of adequate funding to continue the clinical trial. For example, our IND for AAV-RPE65was filed in July 2017. On August 16, 2017, we received notification from the FDA supporting the use of the described batches of product candidate inthe Phase 1/2 clinical trial. However, we received a recommendation from the FDA on a certain aspect of the manufacturing process for future clinicaltrials, thus putting our IND for AAV-RPE65 on partial clinical hold. We responded to the FDA on October 2, 2017 and, based on this response, thepartial clinical hold was lifted on October 17, 2017. As another example, our IND for AAV-CNGB3 was filed on October 31, 2017. We received aquestion from the FDA around our injection device compatibility assay, thus putting our AAV-CNGB3 IND on clinical hold. In the devicecompatibility assay, the FDA noted a disparity between the target titer for the intended low dose dilution and the actual titer obtained on polymerasechain reaction, or PCR, analysis. The FDA requested clarification on whether this was an imprecise dilution scheme for the low dose or a PCR assayissue. We submitted our second response to the FDA on May 2, 2018 providing data that identified the issue as a PCR assay artifact and also showingdata that we believe supports that this has now been addressed. On May 31, 2018, the FDA released the clinical hold on our IND for AAV-CNGB3.Our most advanced product candidates, AAV-GAD, AAV-CNGB3, AAV-CNGA3, AAV-RPGR, AAV-RPE65 and AAV-AQP1, will requireextensive clinical testing before we are prepared to submit a BLA or MAA for regulatory approval. We cannot predict with any certainty if or when wemight complete the clinical development for our product candidates and submit a BLA or MAA for regulatory approval of any of our productcandidates or whether any such BLA or MAA will be approved. We may also seek feedback from the FDA, EMA or other regulatory authorities on ourclinical development program, and the FDA, EMA or such regulatory authorities may not provide such feedback on a timely basis, or such feedbackmay not be favorable, which could further delay our development programs.If we experience delays in the commencement or completion of our clinical trials, or if we terminate a clinical trial prior to completion, thecommercial prospects of our product candidates could be harmed, and our ability to generate revenues from our product candidates may be delayed. Inaddition, any delays in our clinical trials could increase our costs, slow down the development and approval process and jeopardize our ability tocommence product sales and generate revenues. Any of these occurrences may harm our business, financial condition and results of operations. Inaddition, many of the factors that cause, or lead to, a delay in the commencement or completion of clinical trials may also ultimately lead to the denialof regulatory approval of our product candidates.The affected populations for our other product candidates may be smaller than we or third parties currently project, which may affect theaddressable markets for our product candidates.Our projections of the number of people who have the diseases we are seeking to treat, as well as the subset of people with these diseaseswho have the potential to benefit from treatment with our product candidates, are estimates based on our knowledge and understanding of thesediseases. The total addressable market opportunity for our product candidates will ultimately depend upon a number of factors including the diagnosisand treatment criteria included in the final label, if approved for sale in specified indications, 74Table of Contentsacceptance by the medical community, patient access and product pricing and reimbursement. Incidence and prevalence estimates are frequently basedon information and assumptions that are not exact and may not be appropriate, and the methodology is forward-looking and speculative. The processwe have used in developing an estimated incidence and prevalence range for the indications we are targeting has involved collating limited data frommultiple sources. Accordingly, the incidence and prevalence estimates included, or supporting the information, in this Form 10-K should be viewedwith caution. Further, the data and statistical information included, or supporting the information, in this Form 10-K, including estimates derived fromthem, may differ from information and estimates made by our competitors or from current or future studies conducted by independent sources.The use of such data involves risks and uncertainties and is subject to change based on various factors. Our estimates may prove to beincorrect and new studies may change the estimated incidence or prevalence of the diseases we seek to address. The number of patients with thediseases we are targeting in the United States, the European Union and elsewhere may turn out to be lower than expected or may not be otherwiseamenable to treatment with our products, or new patients may become increasingly difficult to identify or access, all of which would harm our results ofoperations and our business.Negative public opinion of gene therapy and increased regulatory scrutiny of gene therapy and genetic research may adversely impact publicperception of our current and future product candidates.Our potential therapeutic products involve introducing genetic material into patients’ cells. The clinical and commercial success of ourpotential products will depend in part on public acceptance of the use of gene therapy and gene regulation for the prevention or treatment of humandiseases. Public attitudes may be influenced by claims that gene therapy and gene regulation are unsafe, unethical, or immoral, and, consequently, ourproducts may not gain the acceptance of the public or the medical community. Adverse public attitudes may adversely impact our ability to enrollclinical trials. Moreover, our success will depend upon physicians prescribing, and their patients being willing to receive, treatments that involve theuse of product candidates we may develop in lieu of, or in addition to, existing treatments with which they are already familiar and for which greaterclinical data may be available.More restrictive government regulations or negative public opinion would have a negative effect on our business or financial conditionand may delay or impair the development and commercialization of our product candidates or demand for any products once approved. For example,in 2003, trials using early versions of murine gamma-retroviral vectors, which integrate with, and thereby alter, the host cell’s DNA, have led to severalwell-publicized adverse events, including reported cases of leukemia. Although none of our current product candidates utilize murine gamma-retroviral vectors, our product candidates use a viral delivery system. Adverse events in our clinical trials, even if not ultimately attributable to ourproduct candidates, and the resulting publicity could result in increased governmental regulation, unfavorable public perception, potential regulatorydelays in the testing or approval of our product candidates, stricter labeling requirements for those product candidates that are approved and a decreasein demand for any such product candidates. The risk of cancer remains a concern for gene therapy and we cannot assure that it will not occur in any ofour planned or future clinical trials. In addition, there is the potential risk of delayed adverse events following exposure to gene therapy products dueto persistent biological activity of the genetic material or other components of products used to carry the genetic material. If any such adverse eventsoccur, commercialization of our product candidates or further advancement of our clinical trials could be halted or delayed, which would have anegative impact on our business and operations. 75Table of ContentsEven though we have been granted access to the PRIME scheme by the EMA for AAV-CNGB3 and the FDA granted Fast Track designation toAAV-RPGR and AAV-CNGB3, in the future we may seek and fail to obtain access to the PRIME scheme by the EMA or Fast Track designation bythe FDA for other of our current or potential future product candidates. We may also seek and fail to obtain breakthrough therapy designation fromthe FDA for our current or any future product candidates. Such designations or access may also not lead to faster development or regulatory reviewor approval, and it does not increase the likelihood that our product candidates will receive marketing approval.A sponsor may seek approval of its product candidate under programs designed to accelerate FDA’s review and approval of new drugs andbiological products that meet certain criteria. For example, the FDA has a Fast Track program that is intended to expedite or facilitate the process forreviewing new products that meet certain criteria. Specifically, new drugs and biological products are eligible for Fast Track designation if they areintended to treat a serious or life-threatening disease or condition and demonstrate the potential to address unmet medical needs, or if the drug has beendesignated as a qualified infectious disease product. Fast Track designation applies to the combination of the product and the specific indication forwhich it is being studied. Under Fast Track, the FDA may consider for review sections of the BLA on a rolling basis before the complete application issubmitted if relevant criteria are satisfied, including an agreement with FDA on the proposed schedule for the submission of portions of the BLA, andthe payment of applicable user fees before FDA may initiate a review. Even if Fast Track designation is granted, it may be rescinded if the product nolonger meets the qualifying criteria. In April 2018, AAV-RPGR was designated a Fast Track program by the FDA for the treatment of X-linked retinitispigmentosa owing to defects in RPGR. In August 2018, AAV-CNGB3 was designated a Fast Track program by the FDA for the treatment ofachromatopsia caused by CNGB3 mutations to improve visual function.In 2012, the Food and Drug Administration Safety and Innovation Act, or FDASIA, established the breakthrough therapy designation. Asponsor may seek FDA designation of its product candidate as a breakthrough therapy if the product candidate is intended, alone or in combinationwith one or more other drugs or biologics, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that theproduct candidate may demonstrate substantial improvement over existing therapies on one or more clinically-significant endpoints, such assubstantial treatment effects observed early in clinical development. Sponsors may request that FDA designate a product candidate as a breakthroughtherapy at the time of or any time after the submission of an IND, but ideally before an end-of-phase 2 meeting with FDA. If the FDA grantsbreakthrough therapy designation, it may take actions appropriate to expedite the development and review of the product candidate, which mayinclude but are not limited to holding meetings with the sponsor and the review team throughout the development of the therapy; providing timelyadvice to, and interactive communication with, the sponsor regarding the development of the product candidate to ensure collection of appropriatedata needed to support approval; more frequent written correspondence from the FDA about such things as the design of the proposed clinical trialsand use of biomarkers; intensive guidance on an efficient drug development program, beginning as early as Phase 1; organizational commitmentinvolving senior managers; and eligibility for rolling review and priority review of a BLA. Breakthrough therapy designation comes with all of thebenefits of Fast Track designation, which means that the sponsor may file sections of the BLA for review on a rolling basis if certain conditions aresatisfied, including an agreement with FDA on the proposed schedule for submission of portions of the application and the payment of applicable userfees before the FDA may initiate a review. Fast Track designation, priority review and breakthrough therapy designation do not change the standardsfor approval but may expedite the development or approval process.Similarly, the EMA has established the PRIME scheme to expedite the development and review of product candidates that show apotential to address to a significant extent an unmet medical need, based on early clinical data. In February 2018, AAV-CNGB3 in the treatment ofachromatopsia associated with defects in CNGB3 was admitted to the PRIME scheme of the EMA.Fast Track designation and designation as a breakthrough therapy are within the discretion of the FDA. Accordingly, even if we believeone of our other product candidates meets the criteria for Fast Track designation 76Table of Contentsor designation as a breakthrough therapy and we seek such designation, the FDA may disagree and instead determine not to make such designation forsuch product candidate. We cannot be sure that our evaluation of our product candidates as qualifying for Fast Track designation or breakthroughtherapy designation will meet the FDA’s expectations. In any event, the receipt of a Fast Track designation or breakthrough therapy designation for aproduct candidate may not result in a faster development process, review, or approval compared to product candidates considered for approval underconventional FDA procedures and does not assure ultimate approval by the FDA. In addition, even if additional product candidates are granted FastTrack designation or one or more of our product candidates qualify as breakthrough therapies, the FDA may later decide that such product candidatesno longer meet the conditions for qualification or decide that the time period for FDA review or approval will not be shortened. Similarly, access to thePRIME scheme is at the discretion of the EMA, and we cannot be sure that any additional current or future product candidates will be granted access tothe scheme; that participation in the scheme will result in expedited regulatory review or approval of our product candidates; or that access to thescheme, once granted, will not be revoked.We have received orphan drug designation from the FDA and EMA for AAV-CNGB3, AAV-CNGA3, AAV-RPE65, AAV-RPGR, AAV-AIPL1 andFDA for AAV-AQP1 and may seek orphan drug designation for additional product candidates in the future, but any orphan drug designations wehave received or may receive in the future may not confer marketing exclusivity or other expected benefits.Under the Orphan Drug Act, the FDA may designate a product as an orphan drug if it is intended to treat a rare disease or condition, definedas one occurring in a patient population of fewer than 200,000 in the United States, or a patient population greater than 200,000 in the United Stateswhere there is no reasonable expectation that the cost of developing the drug will be recovered from sales in the United States. In the European Union,the EMA’s Committee for Orphan Medicinal Products grants orphan drug designation to promote the development of products that are intended for thediagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than five in 10,000 persons in theEuropean Union. Additionally, designation is granted for products intended for the diagnosis, prevention, or treatment of a life-threatening, seriouslydebilitating, or serious and chronic condition when, without incentives, it is unlikely that sales of the drug in the European Union would be sufficientto justify the necessary investment in developing the drug or biological product or where there is no satisfactory method of diagnosis, prevention, ortreatment, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.In the United States, orphan drug designation entitles a party to financial incentives such as opportunities for grant funding towardsclinical trial costs, tax credits for qualified clinical testing, and user-fee waivers. In addition, if a product receives the first FDA approval of that drug forthe indication for which it has orphan designation, the product is entitled to orphan drug exclusivity, which means the FDA may not approve any otherapplication to market the same drug for the same indication for a period of seven years, except in limited circumstances, such as a showing of clinicalsuperiority over the product with orphan exclusivity or where the manufacturer is unable to assure the availability of sufficient quantities of the orphandrug to meet the needs of patients with the rare disease or condition. Under the FDA’s regulations, the FDA will deny orphan drug exclusivity to adesignated drug upon approval if the FDA has already approved another drug with the same active ingredient for the same indication, unless the drugis demonstrated to be clinically superior to the previously approved drug. In the European Union, orphan drug designation entitles a party to financialincentives such as reduction of fees or fee waivers and ten years of market exclusivity following approval for the approved therapeutic indication. Thisperiod may be reduced to six years if, at the end of the fifth year, the orphan drug designation criteria are no longer met, including where it is shownthat the drug is sufficiently profitable not to justify maintenance of market exclusivity. In the European Union, a marketing authorization for an orphandesignated product will not be granted if a similar drug has been approved in the European Union for the same therapeutic indication, unless theapplicant can establish that its product is safer, more effective or otherwise clinically superior. A similar drug is a product containing a similar activesubstance or substances as those contained in an already authorized product. Similar active substance is defined as an identical active substance, 77Table of Contentsor an active substance with the same principal molecular structural features (but not necessarily all of the same molecular features) and which acts viathe same mechanism.We have obtained orphan drug designation from the FDA and European Commission for AAV-CNGB3 for the treatment of achromatopsiacaused by mutations in the CNGB3 gene, AAV-RPE65 for the treatment of Leber congenital amaurosis, AAV-RPGR for the treatment of retinitispigmentosa and AAV-AIPL1 for the treatment of inherited retina dystrophy due to defects in AIPL1 gene, and we obtained orphan drug designationfrom the FDA for AAV-CNGA3 for the treatment of achromatopsia due to autosomal-recessive CNGA3 gene mutations and for AAV-AQP1 for thetreatment of grade 2 and grade 3 late xerostomia from parotid gland hypofunction caused by radiotherapy, and we obtained orphan medicinal productdesignation from the EMA for AAV-CNGA3 for the treatment of achromatopsia. We plan to seek orphan drug designation for other current and futureproduct candidates. Even with orphan drug designation, we may not be the first to obtain marketing approval for any particular orphan indication dueto the uncertainties associated with developing pharmaceutical products, which could prevent us from marketing our product candidates if anothercompany is able to obtain orphan drug exclusivity before we do. In addition, exclusive marketing rights in the United States may be unavailable if weseek approval for an indication broader than the orphan-designated indication or may be lost in the United States if the FDA later determines that therequest for designation was materially defective or if we are unable to assure sufficient quantities of the drug to meet the needs of patients with the raredisease or condition following approval. Further, even if we obtain orphan drug exclusivity, that exclusivity may not effectively protect our productcandidates from competition because different drugs with different active moieties can be approved for the same condition. In addition, the FDA andthe EMA can subsequently approve products with the same active moiety for the same condition if the FDA or the EMA concludes that the later drug issafer, more effective, or makes a major contribution to patient care. Orphan drug designation neither shortens the development time or regulatoryreview time of a drug nor gives the drug any advantage in the regulatory review or approval process. In addition, while we intend to seek orphan drugdesignation for other existing and future product candidates, we may never receive such designations. There have been legal challenges to aspects ofthe FDA’s regulations and policies concerning the exclusivity provisions of the Orphan Drug Act, and future challenges could lead to changes thataffect the protections afforded our product candidates in ways that are difficult to predict. In 2014, a U.S. district court invalidated the FDA’s denial oforphan exclusivity to an orphan designated drug, which the FDA had based on its determination that the drug was not proven to be clinically superiorto a previously approved “same drug.” In response to the decision, the FDA released a policy statement stating that the court’s decision is limited to thefacts of that particular case and that the FDA will continue to deny orphan drug exclusivity to a designated drug upon approval if the drug is the“same” as a previously approved drug, unless the drug is demonstrated to be clinically superior to that previously approved drug. Since then, similarlegal challenges have been initiated against the FDA for its denial of orphan drug exclusivity to other designated drugs, and in 2017, Congressamended the Orphan Drug Act to require a demonstration of clinical superiority upon approval as a condition of receiving orphan drug exclusivitywhen another “same drug” has already been approved for the same indication. In the future, there is the potential for additional legal challenges to theFDA’s orphan drug regulations and policies, and it is uncertain how ongoing and future challenges might affect our business.We and our contract manufacturer for plasmid are subject to significant regulation with respect to manufacturing our products. Our manufacturingfacilities and the third-party manufacturing facility which we rely on may not continue to meet regulatory requirements and have limited capacity.We currently have relationships with a limited number of suppliers for the manufacturing of plasmid, a component of our viral vectors andproduct candidates. We completed the fit-out of our cGMP manufacturing facility in early 2018. However, if we experience slowdowns or problemswith our facility and are unable to establish or scale our internal manufacturing capabilities, we will need to continue to contract with manufacturersthat can produce the preclinical, clinical and commercial supply of our products. Each supplier may require licenses to manufacture such componentsif such processes are not owned by the supplier or in the public domain 78Table of Contentsand we may be unable to transfer or sublicense the intellectual property rights we may have with respect to such activities.All entities involved in the preparation of therapeutics for clinical trials or commercial sale, including our existing contract manufacturersfor components our product candidates, are subject to extensive regulation. Components of a finished therapeutic product approved for commercialsale or used in late-stage clinical trials in the European Union must be manufactured in accordance with cGMP. These regulations governmanufacturing processes and procedures (including record keeping) and the implementation and operation of quality systems to control and assure thequality of investigational products and products approved for sale. Poor control of production processes can lead to the introduction of adventitiousagents or other contaminants, or to inadvertent changes in the properties or stability of our product candidates that may not be detectable in finalproduct testing. We or our contract manufacturers must supply all necessary documentation in support of a BLA or MAA on a timely basis. Ourfacilities and quality systems and the facilities and quality systems of some or all of our third-party contractors must pass a pre-approval inspection forcompliance with the applicable regulations as a condition of regulatory approval of our product candidates or any of our other potential products. Inaddition, the regulatory authorities may, at any time, audit or inspect a manufacturing facility involved with the preparation of our product candidatesor our other potential products or the associated quality systems for compliance with the regulations applicable to the activities being conducted. Ifthese facilities do not pass a pre-approval plant inspection, FDA approval of the products will not be granted.The regulatory authorities also may, at any time following approval of a product for sale, audit our manufacturing facilities or those of ourthird-party contractors. If any such inspection or audit identifies a failure to comply with applicable regulations or if a violation of our productspecifications or applicable regulations occurs independent of such an inspection or audit, we or the relevant regulatory authority may require remedialmeasures that may be costly and/or time-consuming for us or a third party to implement and that may include the temporary or permanent suspensionof a clinical trial or commercial sales or the temporary or permanent closure of a facility. Any such remedial measures imposed upon us or third partieswith whom we contract could harm our business. If we or any of our third-party manufacturers fail to maintain regulatory compliance, the FDA canimpose regulatory sanctions including, among other things, refusal to approve a pending application for a new drug product or biologic product, orrevocation of a pre-existing approval. As a result, our business, financial condition and results of operations may be harmed. Additionally, if supplyfrom one approved manufacturer is interrupted, there could be a significant disruption in commercial supply. An alternative manufacturer would needto be qualified through a BLA and/or MAA supplement which could result in further delay. The regulatory agencies may also require additionalstudies if a new manufacturer is relied upon for commercial production. Switching manufacturers may involve substantial costs and is likely to result ina delay in our desired clinical and commercial timelines.These factors could cause the delay of clinical trials, regulatory submissions, required approvals or commercialization of our productcandidates, cause us to incur higher costs and prevent us from commercializing our products successfully. Furthermore, if our suppliers fail to meetcontractual requirements, and we are unable to secure one or more replacement suppliers capable of production at a substantially equivalent cost, ourclinical trials may be delayed or we could lose potential revenue.Any contamination in our manufacturing process, shortages of raw materials or failure of our plasmid supplier to deliver necessary componentscould result in delays in our clinical development or marketing schedules.Given the nature of biologics manufacturing, there is a risk of contamination. Any contamination could adversely affect our ability toproduce product candidates on schedule and could, therefore, harm our results of operations and cause reputational damage. Some of the raw materialsrequired in our manufacturing process are derived from biologic sources. Such raw materials are difficult to procure and may be subject tocontamination or recall. A material shortage, contamination, recall or restriction on the use of biologically derived substances in 79Table of Contentsthe manufacture of our product candidates could adversely impact or disrupt the commercial manufacturing or the production of clinical material,which could adversely affect our development timelines and our business, financial condition, results of operations and prospects.If we encounter difficulties enrolling patients in our clinical trials, our clinical development activities could be delayed or otherwise adverselyaffected.The timely completion of clinical trials in accordance with their protocols depends, among other things, on our ability to enroll a sufficientnumber of patients who remain in the study until its conclusion. The natural history studies may fail to provide us with patients for our clinical trialsbecause patients enrolled in the natural history studies may not be good candidates for our clinical trials, or may choose to not enroll in our clinicaltrials. We may encounter delays in enrolling, or be unable to enroll, a sufficient number of patients to complete any of our clinical trials, and even onceenrolled we may be unable to retain a sufficient number of patients to complete any of our trials. The enrollment of patients depends on many factors,including: • the size and nature of the patient population; • the patient eligibility criteria defined in the protocol; • the size of the patient population required for analysis of the trial’s primary endpoints; • the proximity of patients to study sites; • the design of the trial; • our ability to recruit clinical trial investigators with the appropriate competencies and experience; • clinicians’ and patients’ perceptions as to the potential advantages of the product candidate being studied in relation to otheravailable therapies, including any new products that may be approved for the indications we are investigating; • our ability to obtain and maintain patient consents; and • the risk that patients enrolled in clinical trials will drop out of the trials before completion.In addition, our clinical trials may compete with other clinical trials for product candidates that are in the same therapeutic areas as ourproduct candidates or approved products for the same clinical indications, and this competition may reduce the number and types of patients availableto us, because some patients who might have opted to enroll in our trials may instead opt to enroll in a trial being conducted by one of our competitors,or chose to be treated using Luxturna, a commercially available product by Spark Therapeutics, Inc. Since the number of qualified clinicalinvestigators is limited, we expect to conduct some of our clinical trials at the same clinical trial sites that some of our competitors use, which mayreduce the number of patients who are available for our clinical trials in such clinical trial site.Delays or failures in planned patient enrollment or retention may result in increased costs, program delays or both, which could have aharmful effect on our ability to develop our product candidates, or could render further development impossible.Our product candidates may cause serious adverse events or undesirable side effects or have other properties which may delay or prevent theirregulatory approval, limit the commercial profile of an approved label, or, result in significant negative consequences following marketingapproval, if any.Serious adverse events or undesirable side effects caused by our product candidates could cause us or regulatory authorities to interrupt,delay or halt clinical trials and could result in a more restrictive label or the delay or denial of regulatory approval by the FDA, EMA or otherauthorities. Results of our clinical trials could reveal a high and unacceptable severity and prevalence of side effects, toxicities or unexpectedcharacteristics, including death. A risk in any gene therapy product based on viral vectors is the risk of insertional oncogenesis. 80Table of ContentsIf unacceptable side effects or deaths arise in the development of our product candidates, we, the FDA, the IRBs at the institutions in whichour studies are conducted, DSMB, EMA or CAT could suspend or terminate our clinical trials or the FDA, EMA or other regulatory authorities couldorder us to cease clinical trials or deny approval of our product candidates for any or all targeted indications. Undesirable side effects or deaths inclinical trials with our product candidates may cause the FDA or comparable foreign regulatory authorities to place a clinical hold on the associatedclinical trials, to require additional studies, or otherwise to delay or deny approval of our product candidates for any or all targeted indications.Treatment-related side effects could also affect patient recruitment or the ability of enrolled patients to complete the trial or result in potential productliability claims. In addition, these side effects may not be appropriately recognized or managed by the treating medical staff. We expect to have to trainmedical personnel using our product candidates to understand the side effect profiles for our clinical trials and upon any commercialization of any ofour product candidates. Inadequate training in recognizing or managing the potential side effects of our product candidates could result in patientinjury or death. Any of these occurrences may harm our business, financial condition and prospects significantly.If any of our product candidates receives marketing approval, and we or others later identify undesirable side effects caused by any suchproduct, including during any long-term follow-up observation period recommended or required for patients who receive treatment using our products,a number of potentially significant negative consequences could result, including: • regulatory authorities may withdraw approvals of such product; • we may be required to recall a product or change the way such product is administered to patients; • additional restrictions may be imposed on the marketing of the particular product or the manufacturing processes for the product; • regulatory authorities may require additional warnings on the label, such as a “black box” warning or contraindication; • we may be required to implement a Risk Evaluation and Mitigation Strategy, or REMS, or create a medication guide outlining therisks of such side effects for distribution to patients; • the product could become less competitive; • we could be sued and held liable for harm caused to patients; and • our reputation may suffer.Any of these events could prevent us from achieving or maintaining market acceptance of the particular product candidate, if approved,and could significantly harm our business, results of operations and prospects.Success in preclinical studies or clinical trials may not be indicative of results in future clinical trials.Results from previous preclinical studies or clinical trials are not necessarily predictive of future clinical trial results, and interim results ofa clinical trial are not necessarily indicative of final results. Our product candidates may fail to show the desired safety and efficacy in clinicaldevelopment despite positive results in preclinical studies or having successfully advanced through initial clinical trials.Success in preclinical testing and early clinical trials does not ensure that later clinical trials will generate the same results or otherwiseprovide adequate data to demonstrate the efficacy and safety of a product candidate. Frequently, product candidates that have shown promising resultsin early clinical trials have subsequently suffered significant setbacks in later clinical trials. In addition, the design of a clinical trial can determinewhether its results will support approval of a product and flaws in the design of a clinical trial may not become apparent until the clinical trial is welladvanced. We have limited experience designing clinical trials and may be unable to design and execute a clinical trial to support regulatory approval.There is a high failure rate for drugs and biologic products proceeding through clinical trials. Many companies in the pharmaceutical and 81Table of Contentsbiotechnology industries have suffered significant setbacks in late-stage clinical trials even after achieving promising results in preclinical testing andearlier-stage clinical trials. Data obtained from preclinical and clinical activities are subject to varying interpretations, which may delay, limit orprevent regulatory approval. In addition, we may experience regulatory delays or rejections as a result of many factors, including due to changes inregulatory policy during the period of our product candidate development. Any such delays could negatively impact our business, financial condition,results of operations and prospects.The regulatory approval processes of the FDA, EMA and other regulatory authorities are lengthy, time consuming and inherently unpredictable,and if we are ultimately unable to obtain regulatory approval for our product candidates, our business will be substantially harmed.The time required to obtain approval by the FDA, EMA and other regulatory authorities is unpredictable but typically takes many yearsfollowing the commencement of clinical trials and depends upon numerous factors, including the substantial discretion of the regulatory authorities. Inaddition, approval policies, regulations, or the type and amount of clinical data necessary to gain approval may change during the course of a productcandidate’s clinical development and may vary among jurisdictions. We have not obtained regulatory approval for any product candidate and it ispossible that none of our product candidates in clinical programs or any other product candidates we may seek to develop in the future will ever obtainregulatory approval. Neither we nor any future collaborator is permitted to market any of our product candidates in the United States or the EuropeanUnion until we receive regulatory approval of a BLA from the FDA or a MAA from the EMA, respectively. It is possible that the FDA may refuse toaccept for substantive review any biologic license applications, or BLAs, or the EMA any of our MAAs, that we submit for our product candidates ormay conclude after review of our data that our application is insufficient to obtain marketing approval of our product candidates.Prior to obtaining approval to commercialize a product candidate in the United States, the European Union or elsewhere, we or ourcollaborators must demonstrate with substantial evidence from well-controlled clinical trials, and to the satisfaction of the FDA, EMA or foreignregulatory agencies, that such product candidates are safe and effective for their intended uses. Results from nonclinical studies and clinical trials canbe interpreted in different ways. Even if we believe the nonclinical or clinical data for our product candidates are promising, such data may not besufficient to support approval by the FDA, EMA or other regulatory authorities. The FDA or EMA may also require us to conduct additional preclinicalstudies or clinical trials for our product candidates either prior to or post-approval, or it may object to elements of our clinical development program.Depending on the extent of these or any other FDA or EMA required studies, approval of any regulatory approval applications that we submit may bedelayed by several years, or may require us to expend significantly more resources than we have available.Of the large number of potential products in development, only a small percentage successfully complete the FDA, EMA or other foreignregulatory approval processes and are commercialized. The lengthy approval process as well as the unpredictability of future clinical trial results mayresult in our failing to obtain regulatory approval to market our product candidates, which would significantly harm our business, results of operationsand prospects.Even if we and / or our Collaboration Agreement partner obtain FDA or EMA approval for AAV-GAD, AAV-CNGB3, AAV-CNGA3, AAV-RPGR,AAV-RPE65 or AAV-AQP1 in the United States or European Union, we may never obtain approval for or commercialize it in any other jurisdiction,which would limit our ability to realize their full market potential.In order to market any products in any particular jurisdiction, we must establish and comply with numerous and varying regulatoryrequirements on a country-by-country basis regarding safety and efficacy. Approval by the FDA in the United States or the EMA in the EuropeanUnion does not ensure approval by regulatory authorities in other countries or jurisdictions. However, the failure to obtain approval in one 82Table of Contentsjurisdiction may negatively impact our ability to obtain approval elsewhere. In addition, clinical trials conducted in one country may not be acceptedby regulatory authorities in other countries, and regulatory approval in one country does not guarantee regulatory approval in any other country.Approval processes vary among countries and can involve additional product testing and validation and additional administrative reviewperiods. Seeking foreign regulatory approval could result in difficulties and increased costs for us and require additional preclinical studies or clinicaltrials which could be costly and time consuming. Regulatory requirements can vary widely from country to country and could delay or prevent theintroduction of our products in those countries. We do not have any product candidates approved for sale in any jurisdiction, including ininternational markets, and we do not have experience in obtaining regulatory approval in international markets. If we fail to comply with regulatoryrequirements in international markets or to obtain and maintain required approvals, or if regulatory approvals in international markets are delayed, ourtarget market will be reduced and our ability to realize the full market potential of any product we develop will be unrealized.Even if we receive regulatory approval of our product candidates, we will be subject to ongoing regulatory obligations and continued regulatoryreview, which may result in significant additional expense, and we may be subject to penalties if we fail to comply with regulatory requirements orexperience unanticipated problems with our product candidates.Any product candidate for which we obtain marketing approval, along with the manufacturing processes, post-approval clinical data,labeling, packaging, distribution, adverse event reporting, storage, recordkeeping, export, import, advertising and promotional activities for suchproduct, among other things, will be subject to extensive and ongoing requirements of and review by the FDA, EMA and other regulatory authorities.These requirements include submissions of safety and other post-marketing information and reports, establishment registration and drug listingrequirements, continued compliance with cGMP requirements relating to manufacturing, quality control, quality assurance and correspondingmaintenance of records and documents, requirements regarding the distribution of samples to physicians and recordkeeping and GCP requirements forany clinical trials that we conduct post-approval.The FDA and EMA closely regulate the post-approval marketing and promotion of genetic therapy medicines to ensure they are marketedonly for the approved indications and in accordance with the provisions of the approved labeling. The FDA and EMA imposes stringent restrictions onmanufacturers’ communications regarding off-label use and if we market our products for uses beyond their approved indications, we may be subject toenforcement action for off-label marketing. Violations of the U.S. federal Food, Drug, and Cosmetic Act, or FDCA, relating to the promotion ofprescription drugs may lead to FDA enforcement actions and investigations alleging violations of federal and state health care fraud and abuse laws, aswell as state consumer protection laws.In addition, later discovery of previously unknown adverse events or other problems with our products, manufacturers or manufacturingprocesses, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failureto comply with regulatory requirements, may yield various results, including: • restrictions on manufacturing such products; • restrictions on the labeling or marketing of a product; • restrictions on product distribution or use; • requirements to conduct post-marketing studies or clinical trials; • warning letters or holds on clinical trials; • withdrawal of the products from the market; 83Table of Contents • refusal to approve pending applications or supplements to approved applications that we submit; • recall of products; • fines, restitution or disgorgement of profits or revenues; • suspension or withdrawal of marketing approvals; • refusal to permit the import or export of our products; • product seizure or detention; or • injunctions or the imposition of civil or criminal penalties.The FDA’s policies may change and additional government regulations may be enacted that could prevent, limit or delay regulatoryapproval of our product candidates. For example, in December 2016, the 21st Century Cures Act, or Cures Act, was signed into law. The Cures Act,among other things, is intended to modernize the regulation of drugs and biologics and spur innovation and contains provisions applicable to thedevelopment of gene therapies, but its ultimate implementation is unclear. If we are slow or unable to adapt to changes in existing requirements or theadoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may lose any marketing approval that we mayhave obtained which would adversely affect our business, prospects and ability to achieve or sustain profitability.We also cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative orexecutive action, either in the United States or abroad. For example, certain policies of the Trump administration may impact our business andindustry. Namely, the Trump administration has taken several executive actions, including the issuance of a number of Executive Orders, that couldimpose significant burdens on, or otherwise materially delay, FDA’s ability to engage in routine regulatory and oversight activities such asimplementing statutes through rulemaking, issuance of guidance, and review and approval of marketing applications. It is difficult to predict how theserequirements will be implemented, and the extent to which they will impact the FDA’s ability to exercise its regulatory authority. If these executiveactions impose constraints on FDA’s ability to engage in oversight and implementation activities in the normal course, our business may be negativelyimpacted.Interim “top-line” and preliminary data from our clinical trials that we announce or publish from time to time may change as more patient databecome available and are subject to audit and verification procedures that could result in material changes in the final data.From time to time, we may publish interim “top-line” or preliminary data from our clinical trials. Interim data from clinical trials that wemay complete are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and more patientdata become available. Preliminary or “top-line” data also remain subject to audit and verification procedures that may result in the final data beingmaterially different from the preliminary data we previously published. As a result, interim and preliminary data should be viewed with caution untilthe final data are available. Adverse differences between preliminary or interim data and final data could significantly harm our business prospects.We may expend our limited resources to pursue a particular product candidate or indication and fail to capitalize on product candidates orindications that may be more profitable or for which there is a greater likelihood of success.Because we have limited financial and managerial resources, we focus on research programs and product candidates that we identify forspecific indications. As a result, we may forego or delay pursuit of opportunities with other product candidates or for other indications that later proveto have greater commercial potential. Our resource allocation decisions may cause us to fail to timely capitalize on viable commercial products orprofitable market opportunities. Our spending on current and future research and development 84Table of Contentsprograms and product candidates for specific indications may not yield any commercially viable products. If we do not accurately evaluate thecommercial potential or target market for a particular product candidate, we may relinquish valuable rights to that product candidate throughcollaboration, licensing or other royalty arrangements in cases in which it would have been more advantageous for us to retain sole development andcommercialization rights to such product candidate.Risks Related to Healthcare Laws and Other Legal Compliance MattersEnacted and future healthcare legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our productcandidates and may affect the prices we may set.In the United States, the European Union and other jurisdictions, there have been, and we expect there will continue to be, a number oflegislative and regulatory changes and proposed changes to the healthcare system that could affect our future results of operations. In particular, therehave been and continue to be a number of initiatives at the U.S. federal and state levels that seek to reduce healthcare costs and improve the quality ofhealthcare. For example, in March 2010, the Patient Protection and Affordable Care Act, as amended by the Health Care and Education ReconciliationAct, or collectively the ACA, was enacted, which substantially changed the way healthcare is financed by both governmental and private insurers.Among the provisions of the ACA, those of greatest importance to the pharmaceutical and biotechnology industries include the following: • an annual, non-deductible fee payable by any entity that manufactures or imports certain branded prescription drugs and biologicagents (other than those designated as orphan drugs), which is apportioned among these entities according to their market share incertain government healthcare programs; • a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs thatare inhaled, infused, instilled, implanted or injected; • expansion of eligibility criteria for Medicaid programs by, among other things, allowing states to offer Medicaid coverage tocertain individuals with income at or below 133% of the federal poverty level, thereby potentially increasing a manufacturer’sMedicaid rebate liability; • a licensure framework for follow on biologic products; • a new Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinicaleffectiveness research, along with funding for such research; and • establishment of a Center for Medicare & Medicaid Innovation at the Centers for Medicare & Medicaid Services, or CMS, to testinnovative payment and service delivery models to lower Medicare and Medicaid spending, potentially including prescriptiondrug spending.Since its enactment, there have been judicial, Congressional and executive branch challenges to certain aspects of the ACA, and we expectthere will be additional challenges and amendments to the ACA in the future. For example, the Tax Cuts and Jobs Act of 2017 was enacted, whichincludes a provision repealing, effective January 1, 2019, the tax-based shared responsibility payment imposed by the ACA on certain individuals whofail to maintain qualifying health coverage for all or part of a year that is commonly referred to as the “individual mandate”. Additionally, onDecember 14, 2018, a U.S. District Court Judge in Texas ruled that the ACA is unconstitutional in its entirety because the “individual mandate” wasrepealed by Congress as part of the legislation enacted in 2017, informally titled the Tax Cuts and Jobs Act, or the Tax Act. While such U.S. DistrictCourt Judge, as well as the Trump administration and CMS, have stated that the ruling will have no immediate effect pending appeal of the decision, itis unclear how this decision, subsequent appeals, and other efforts to repeal and replace the ACA will impact the law. The current Trump administrationand Congress will likely continue to seek to modify, repeal, or otherwise invalidate all, or certain provisions of, the ACA. It is uncertain the extent towhich any such changes may impact our business or financial condition.In addition, other legislative changes have been proposed and adopted in the United States since the ACA was enacted. In August 2011,the Budget Control Act of 2011, among other things, led to aggregate 85Table of Contentsreductions of Medicare payments to providers of 2% per fiscal year. These reductions went into effect in April 2013 and, due to subsequent legislativeamendments to the statute will remain in effect through 2027 unless additional action is taken by Congress. In January 2013, the American TaxpayerRelief Act of 2012 was signed into law, which, among other things, further reduced Medicare payments to several types of providers, includinghospitals, imaging centers and cancer treatment centers, and increased the statute of limitations period for the government to recover overpayments toproviders from three to five years. These new laws or any other similar laws introduced in the future may result in additional reductions in Medicareand other health care funding, which could negatively affect our customers and accordingly, our financial operations.Moreover, payment methodologies may be subject to changes in healthcare legislation and regulatory initiatives. For example, CMS maydevelop new payment and delivery models, such as bundled payment models. In addition, recently there has been heightened governmental scrutinyover the manner in which manufacturers set prices for their marketed products, which has resulted in several U.S. Congressional inquiries and proposedand enacted federal legislation designed to, among other things, bring more transparency to drug pricing, reduce the cost of prescription drugs underMedicare, and review the relationship between pricing and manufacturer patient programs. The Trump administration’s budget proposal for fiscal year2019 contains further drug price control measures that could be enacted during the 2019 budget process or in other future legislation, including, forexample, measures to permit Medicare Part D plans to negotiate the price of certain drugs under Medicare Part B, to allow some states to negotiate drugprices under Medicaid, and to eliminate cost sharing for generic drugs for low-income patients. On January 31, 2019, the HHS Office of InspectorGeneral proposed modifications to U.S. federal Anti-Kickback Statute safe harbors which, among other things, will affect rebates paid by manufacturersto Medicare Part D plan sponsors, Medicaid managed care organizations, and those entities’ pharmacy benefit managers (“PBMs”), the purpose ofwhich is to further reduce the cost of drug products to consumers. Although some of these, and other, proposals will require authorization throughadditional legislation to become effective, Congress and the Trump administration have each indicated that it will continue to seek new legislativeand/or administrative measures to control drug costs. We expect that additional U.S. federal healthcare reform measures will be adopted in the future,any of which could limit the amounts that the U.S. federal government will pay for healthcare products and services, which could result in reduceddemand for our product candidates or additional pricing pressures.Individual states in the United States have also increasingly passed legislation and implemented regulations designed to controlpharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product accessand marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulkpurchasing. Legally mandated price controls on payment amounts by third-party payors or other restrictions could harm our business, results ofoperations, financial condition and prospects. In addition, regional healthcare authorities and individual hospitals are increasingly using biddingprocedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug and other healthcare programs.This could reduce the ultimate demand for our product candidates or put pressure on our product pricing.Additionally, on May 30, 2018, the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, orthe Right to Try Act, was signed into law. The law, among other things, provides a federal framework for certain patients to access certaininvestigational new drug products that have completed a Phase 1 clinical trial and that are undergoing investigation for FDA approval. Under certaincircumstances, eligible patients can seek treatment without enrolling in clinical trials and without obtaining FDA permission under the FDA expandedaccess program. There is no obligation for a pharmaceutical manufacturer to make its drug products available to eligible patients as a result of theRight to Try Act.In the European Union, similar political, economic and regulatory developments may affect our ability to profitably commercialize ourproduct candidates, if approved. In addition to continuing pressure on prices and cost containment measures, legislative developments at the EU ormember state level may result in significant additional requirements or obstacles that may increase our operating costs. The delivery of healthcare inthe 86Table of ContentsEuropean Union, including the establishment and operation of health services and the pricing and reimbursement of medicines, is almost exclusively amatter for national, rather than European Union, law and policy. National governments and health service providers have different priorities andapproaches to the delivery of health care and the pricing and reimbursement of products in that context. In general, however, the healthcare budgetaryconstraints in most EU member states have resulted in restrictions on the pricing and reimbursement of medicines by relevant health service providers.Coupled with ever-increasing European Union and national regulatory burdens on those wishing to develop and market products, this could prevent ordelay marketing approval of our product candidates, restrict or regulate post-approval activities and affect our ability to commercialize our productcandidates, if approved.In markets outside of the United States and the European Union, reimbursement and healthcare payment systems vary significantly bycountry, and many countries have instituted price ceilings on specific products and therapies.We cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative actionin the United States, the European Union or any other jurisdiction. If we or any third parties we may engage are slow or unable to adapt to changes inexisting requirements or the adoption of new requirements or policies, or if we or such third parties are not able to maintain regulatory compliance, ourproduct candidates may lose any regulatory approval that may have been obtained and we may not achieve or sustain profitability.Our business operations and current and future relationships with investigators, healthcare professionals, consultants, third-party payors, patientorganizations and customers will be subject to applicable healthcare regulatory laws, which could expose us to penalties.Our business operations and current and future arrangements with investigators, healthcare professionals, consultants, third-party payors,patient organizations and customers, may expose us to broadly applicable fraud and abuse and other healthcare laws and regulations. These laws mayconstrain the business or financial arrangements and relationships through which we conduct our operations, including how we research, market, selland distribute our product candidates, if approved. Such laws include: • the U.S. federal Anti-Kickback Statute, which prohibits, among other things, persons or entities from knowingly and willfullysoliciting, offering, receiving or providing any remuneration (including any kickback, bribe, or certain rebate), directly orindirectly, overtly or covertly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or thepurchase, lease, order or recommendation of, any good, facility, item or service, for which payment may be made, in whole or inpart, under U.S. federal and state healthcare programs such as Medicare and Medicaid. A person or entity does not need to haveactual knowledge of the statute or specific intent to violate it in order to have committed a violation; • the U.S. federal civil and criminal false claims and civil monetary penalties laws, including the civil False Claims Act, whichprohibit, among other things,, including through civil whistleblower or qui tam actions, individuals or entities from knowinglypresenting, or causing to be presented, to the U.S. federal government, claims for payment or approval that are false or fraudulent,knowingly making, using or causing to be made or used, a false record or statement material to a false or fraudulent claim, or fromknowingly making a false statement to avoid, decrease or conceal an obligation to pay money to the U.S. federal government. Inaddition, the government may assert that a claim including items and services resulting from a violation of the U.S. federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the False Claims Act; • the U.S. federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which created additional federal criminalstatutes which prohibit, among other things, knowingly and willfully executing, or attempting to execute, a scheme to defraud anyhealthcare benefit program, 87Table of Contents or knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false statement, inconnection with the delivery of, or payment for, healthcare benefits, items or services. Similar to the U.S. federal Anti-KickbackStatute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to havecommitted a violation; • HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 and its implementingregulations, which imposes certain obligations, including mandatory contractual terms, with respect to safeguarding the privacy,security and transmission of individually identifiable health information without appropriate authorization by covered entitiessubject to the rule, such as certain health plans, healthcare clearinghouses and healthcare providers as well as their businessassociates, independent contractors of a covered entity that perform certain services involving the use or disclosure of individuallyidentifiable health information; • the FDCA, which prohibits, among other things, the adulteration or misbranding of drugs, biologics and medical devices; • the U.S. Public Health Service Act, which prohibits, among other things, the introduction into interstate commerce of a biologicalproduct unless a biologics license is in effect for that product; • federal consumer protection and unfair competition laws, which broadly regulate marketplace activities and activities thatpotentially harm consumers; • the U.S. Physician Payments Sunshine Act and its implementing regulations, which requires certain manufacturers of drugs, devices,biologics and medical supplies that are reimbursable under Medicare, Medicaid, or the Children’s Health Insurance Program, withspecific exceptions, to report annually to the government information related to certain payments and other transfers of value tophysicians and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate familymembers; • analogous U.S. state laws and regulations, including: state anti-kickback and false claims laws, which may apply to our businesspractices, including but not limited to, research, distribution, sales and marketing arrangements and claims involving healthcareitems or services reimbursed by any third-party payor, including private insurers; state laws that require pharmaceutical companiesto comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgatedby the U.S. federal government, or otherwise restrict payments that may be made to healthcare providers and other potential referralsources; state laws and regulations that require drug manufacturers to file reports relating to pricing and marketing information,which requires tracking gifts and other remuneration and items of value provided to healthcare professionals and entities; state andlocal laws that require the registration of pharmaceutical sales representatives; and state laws governing the privacy and security ofhealth information in certain circumstances, many of which differ from each other in significant ways and often are not preemptedby HIPAA, thus complicating compliance efforts; and • similar healthcare laws and regulations in the European Union and other jurisdictions, including reporting requirements detailinginteractions with and payments to healthcare providers and requirements regarding the collection, distribution, use, security, andstorage of personally identifiable information and other data relating to individuals (including the EU General Data ProtectionRegulation 2016/679, or GDPR.As of May 25, 2018, the GDPR replaced the Data Protection Directive with respect to the processing of personal data in the EuropeanUnion. The GDPR imposes many requirements for controllers and processors of personal data, including, for example, higher standards for obtainingconsent from individuals to process their personal data, more robust disclosures to individuals and a strengthened individual data rights regime,shortened timelines for data breach notifications, limitations on retention and secondary use of information, increased 88Table of Contentsrequirements pertaining to health data and pseudonymised (i.e., key-coded) data and additional obligations when we contract third-party processors inconnection with the processing of the personal data. The GDPR allows EU member states to make additional laws and regulations further limiting theprocessing of genetic, biometric or health data. Failure to comply with the requirements of GDPR and the applicable national data protection laws ofthe EU member states may result in fines of up to €20,000,000 or up to 4% of the total worldwide annual turnover of the preceding financial year,whichever is higher, and other administrative penalties.Ensuring that our internal operations and future business arrangements with third parties comply with applicable healthcare laws andregulations will involve substantial costs. It is possible that governmental authorities will conclude that our business practices do not comply withcurrent or future statutes, regulations, agency guidance or case law involving applicable fraud and abuse or other healthcare laws and regulations. If ouroperations are found to be in violation of any of the laws described above or any other governmental laws and regulations that may apply to us, wemay be subject to significant penalties, including civil, criminal and administrative penalties, damages, fines, exclusion from government-fundedhealthcare programs, such as Medicare and Medicaid or similar programs in other countries or jurisdictions, integrity oversight and reportingobligations to resolve allegations of non-compliance, disgorgement, individual imprisonment, contractual damages, reputational harm, diminishedprofits and the curtailment or restructuring of our operations. If any of the physicians or other providers or entities with whom we expect to do businessare found to not be in compliance with applicable laws, they may be subject to criminal, civil or administrative sanctions, including exclusions fromgovernment funded healthcare programs and imprisonment, which could affect our ability to operate our business. Further, defending against any suchactions can be costly, time-consuming and may require significant personnel resources. Therefore, even if we are successful in defending against anysuch actions that may be brought against us, our business may be impaired.We are subject to environmental, health and safety laws and regulations, and we may become exposed to liability and substantial expenses inconnection with environmental compliance or remediation activities.Our operations, including our development, testing and manufacturing activities, are subject to numerous environmental, health and safetylaws and regulations. These laws and regulations govern, among other things, the controlled use, handling, release and disposal of and the maintenanceof a registry for, hazardous materials and biological materials, such as chemical solvents, human cells, carcinogenic compounds, mutagenic compoundsand compounds that have a toxic effect on reproduction, laboratory procedures and exposure to blood-borne pathogens. If we fail to comply with suchlaws and regulations, we could be subject to fines or other sanctions.As with other companies engaged in activities similar to ours, we face a risk of environmental liability inherent in our current and historicalactivities, including liability relating to releases of or exposure to hazardous or biological materials. Environmental, health and safety laws andregulations are becoming more stringent. We may be required to incur substantial expenses in connection with future environmental compliance orremediation activities, in which case, the production efforts of our third-party manufacturers or our development efforts may be interrupted or delayed.Due to our international operations, we are subject to anti-corruption laws, as well as export control laws, customs laws, sanctions laws and otherlaws governing our operations. If we fail to comply with these laws, we could be subject to civil or criminal penalties, other remedial measures andlegal expenses.Our operations are subject to anti-corruption laws, including the U.K. Bribery Act 2010, or Bribery Act; the U.S. Foreign Corrupt PracticesAct, or FCPA; and other anti-corruption laws that apply in countries where we do business and may do business in the future. The Bribery Act, FCPA,and these other laws generally prohibit us, our officers and our employees and intermediaries from bribing, being bribed by, or providing prohibitedpayments or anything else of value to government officials or other persons to obtain or retain business or gain some other business advantage. Wemay in the future operate in jurisdictions that pose a high 89Table of Contentsrisk of potential Bribery Act or FCPA violations, and we may participate in collaborations and relationships with third parties whose actions couldpotentially subject us to liability under the Bribery Act, FCPA, or local anti-corruption laws. In addition, we cannot predict the nature, scope, or effectof future regulatory requirements to which any of our international operations might be subject or the manner in which existing laws might beadministered or interpreted.We also are subject to other laws and regulations governing any international operations, including regulations administered by thegovernments of the United Kingdom and the United States, and authorities in the EU, including applicable export control regulations, economicsanctions on countries and persons, customs requirements and currency exchange regulations, or, collectively, the Trade Control laws.There is no assurance that we will be completely effective in ensuring our compliance with all applicable anti-corruption laws, includingthe Bribery Act, the FCPA, or other legal requirements, including Trade Control laws. If we are not in compliance with the Bribery Act, the FCPA, andother anti-corruption laws or Trade Control laws, we may be subject to criminal and civil penalties, disgorgement, and other sanctions and remedialmeasures and legal expenses. Any investigation of any potential violations of the Bribery Act, the FCPA, other anti-corruption laws, or Trade Controllaws by U.K., U.S., or other authorities, even if it is ultimately determined that we did not violate such laws, could be costly and time-consuming,require significant personnel resources, and harm our reputation.We will seek to build and continuously improve our systems of internal controls and to remedy any weaknesses identified. There can be noassurance, however, that the policies and procedures will be followed at all times or effectively detect and prevent violations of the applicable laws byone or more of our employees, consultants, agents, or collaborators and, as a result, we could be subject to fines, penalties, or prosecution.Risks Related to CommercializationWe face significant competition in an environment of rapid technological change, and there is a possibility that our competitors may achieveregulatory approval before us or develop therapies that are safer or more advanced or effective than ours, which may harm our financial conditionand our ability to successfully market or commercialize any product candidates we may develop.The development and commercialization of new gene therapy products is highly competitive. Moreover, the gene regulation andmanufacturing fields are characterized by rapidly changing technologies, significant competition, and a strong emphasis on intellectual property. Wewill face competition with respect to any product candidates that we may seek to develop or commercialize in the future from major pharmaceuticalcompanies, specialty pharmaceutical companies, and biotechnology companies worldwide. Potential competitors also include academic institutions,government agencies, and other public and private research organizations that conduct research, seek patent protection, and establish collaborativearrangements for research, development, manufacturing, and commercialization.There are a number of large pharmaceutical and biotechnology companies that currently market and sell products or are pursuing thedevelopment of products for the treatment of the disease indications for which we have research programs, including inherited retinal diseases andneurodegenerative diseases. Some of these competitive products and therapies are based on scientific approaches that are similar to our approach, andothers are based on entirely different approaches.Our platform and products focus on the development of gene therapies and gene regulation technology. There are a number of companiesdeveloping ocular gene therapy products, including Applied Genetic Technologies Corporation, Nightstar Therapeutics plc and Spark Therapeutics,Inc. There are a number of companies developing gene therapy products for neurodegenerative diseases, including Voyager Therapeutics, Inc. andAxovant Sciences Ltd. In addition to competition from other gene therapies, any products we may develop may also face competition from other typesof therapies, such as small molecule, antibody, protein or other therapies. 90Table of ContentsMany of our current or potential competitors, either alone or with their collaboration partners, have greater financial resources andexpertise in research and development, manufacturing, preclinical testing, conducting clinical trials, obtaining regulatory approvals, and marketingapproved products than we do. Mergers and acquisitions in the pharmaceutical, biotechnology, and gene therapy industries may result in even moreresources being concentrated among a smaller number of our competitors. Smaller or early-stage companies may also prove to be significantcompetitors, particularly through collaborative arrangements with large and established companies. These competitors also compete with us inrecruiting and retaining qualified scientific and management personnel and establishing clinical trial sites and patient registration for clinical trials, aswell as in acquiring technologies complementary to, or necessary for, our programs. Our commercial opportunity could be reduced or eliminated if ourcompetitors develop and commercialize products that are safer, more effective, have fewer or less severe side effects, are more convenient, or are lessexpensive than any products that we may develop or that would render any products that we may develop obsolete or non-competitive. Ourcompetitors also may obtain FDA, EMA or other regulatory approval for their products more rapidly than we may obtain approval for ours, which couldresult in our competitors establishing a strong market position before we are able to enter the market. Additionally, technologies developed by ourcompetitors may render our potential product candidates uneconomic or obsolete, and we may not be successful in marketing any product candidateswe may develop against competitors.In addition, as a result of the expiration or successful challenge of our patent rights, we could face more litigation with respect to thevalidity and/or scope of patents relating to our competitors’ products. The availability of our competitors’ products could limit the demand, and theprice we are able to charge, for any products that we may develop and commercialize.The successful commercialization of our product candidates will depend in part on the extent to which governmental authorities and health insurersestablish coverage, adequate reimbursement levels and pricing policies. Failure to obtain or maintain coverage and adequate reimbursement forour product candidates, if approved, could limit our ability to market those products and decrease our ability to generate revenue.The availability of coverage and adequacy of reimbursement by governmental healthcare programs such as Medicare and Medicaid,private health insurers and other third-party payors are essential for most patients to be able to afford medical services and pharmaceutical productssuch as our product candidates, assuming FDA approval. Our ability to achieve acceptable levels of coverage and reimbursement for our products orprocedures using our products by governmental authorities, private health insurers and other organizations will have an effect on our ability tosuccessfully commercialize our product candidates. Obtaining coverage and adequate reimbursement for our products may be particularly difficultbecause of the higher prices often associated with drugs administered under the supervision of a physician. Separate reimbursement for the productitself or the treatment or procedure in which our product is used may not be available. A decision by a third-party payor not to cover or separatelyreimburse for our products or procedures using our products, could reduce physician utilization of our products once approved. Assuming there iscoverage for our product candidates or procedures using our product candidates by a third-party payor, the resulting reimbursement payment rates maynot be adequate or may require co-payments that patients find unacceptably high. We cannot be sure that coverage and reimbursement in the UnitedStates, the European Union or elsewhere will be available for our product candidates or any product that we may develop, and any reimbursement thatmay become available may not be adequate or may be decreased or eliminated in the future.Third-party payors increasingly are challenging prices charged for pharmaceutical products and services, and many third-party payors mayrefuse to provide coverage and reimbursement for particular drugs or biologics when an equivalent generic drug, biosimilar or a less expensive therapyis available. It is possible that a third-party payor may consider our product candidates as substitutable and only offer to reimburse patients for the lessexpensive product. Even if we show improved efficacy or improved convenience of administration with our product candidates, pricing of existingthird-party therapeutics may limit the amount we will be able to charge for our product candidates. These payors may deny or revoke thereimbursement status of a given product 91Table of Contentsor establish prices for new or existing marketed products at levels that are too low to enable us to realize an appropriate return on our investment in ourproduct candidates. If reimbursement is not available or is available only at limited levels, we may not be able to successfully commercialize ourproduct candidates, and may not be able to obtain a satisfactory financial return on our product candidates.There is significant uncertainty related to the insurance coverage and reimbursement of newly-approved products. In the United States,third-party payors, including private and governmental payors, such as the Medicare and Medicaid programs, play an important role in determining theextent to which new drugs and biologics will be covered. The Medicare and Medicaid programs increasingly are used as models in the United Statesfor how private payors and other governmental payors develop their coverage and reimbursement policies for drugs and biologics. Some third-partypayors may require pre-approval of coverage for new or innovative devices or drug therapies before they will reimburse healthcare providers who usesuch therapies. We cannot predict at this time what third-party payors will decide with respect to the coverage and reimbursement for our productcandidates.No uniform policy for coverage and reimbursement for products exists among third-party payors in the United States. Therefore, coverageand reimbursement for products can differ significantly from payor to payor. As a result, the coverage determination process is often a time-consumingand costly process that will require us to provide scientific and clinical support for the use of our product candidates to each payor separately, with noassurance that coverage and adequate reimbursement will be applied consistently or obtained in the first instance. Furthermore, rules and regulationsregarding reimbursement change frequently, in some cases on short notice, and we believe that changes in these rules and regulations are likely.Outside the United States, international operations are generally subject to extensive governmental price controls and other marketregulations, and we believe the increasing emphasis on cost-containment initiatives in Europe and other countries have and will continue to putpressure on the pricing and usage of our product candidates. In many countries, the prices of medical products are subject to varying price controlmechanisms as part of national health systems. Other countries allow companies to fix their own prices for medical products, but monitor and controlcompany profits. Additional foreign price controls or other changes in pricing regulation could restrict the amount that we are able to charge for ourproduct candidates. Accordingly, in markets outside the United States, the reimbursement for our product candidates may be reduced compared withthe United States and may be insufficient to generate commercially-reasonable revenue and profits.Moreover, increasing efforts by governmental and third-party payors in the United States and abroad to cap or reduce healthcare costs maycause such organizations to limit both coverage and the level of reimbursement for newly approved products and, as a result, they may not cover orprovide adequate payment for our product candidates. We expect to experience pricing pressures in connection with the sale of our product candidatesdue to the trend toward managed health care, the increasing influence of health maintenance organizations and additional legislative changes. Thedownward pressure on healthcare costs in general, particularly prescription drugs and biologics and surgical procedures and other treatments, hasbecome intense. As a result, increasingly high barriers are being erected to the entry of new products.Even if our product candidates receive marketing approval, they may fail to achieve market acceptance by physicians, patients, third-party payors orothers in the medical community necessary for commercial success.If our product candidates receive marketing approval, they may nonetheless fail to gain sufficient market acceptance by physicians,patients, third-party payors and others in the medical community. If they do not achieve an adequate level of acceptance, we may not generatesignificant product revenues or become profitable. The degree of market acceptance of our product candidates, if approved for commercial sale, willdepend on a number of factors, including but not limited to: • the efficacy and potential advantages compared to alternative treatments; 92Table of Contents • effectiveness of sales and marketing efforts; • the cost of treatment in relation to alternative treatments, including any similar generic treatments; • our ability to offer our products for sale at competitive prices; • the convenience and ease of administration compared to alternative treatments; • the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies; • the strength of marketing and distribution support; • the timing of market introduction of competitive products; • the availability of third-party coverage and adequate reimbursement; • product labeling or product insert requirements of the FDA, EMA or other regulatory authorities, including any limitations orwarnings contained in a product’s approved labeling; • the prevalence and severity of any side effects; and • any restrictions on the use of our product together with other medications.Because we expect sales of our product candidates, if approved, to generate substantially all of our product revenues for a substantialperiod, the failure of this product to find market acceptance would harm our business and could require us to seek additional financing.If we are unable to establish sales, marketing and distribution capabilities either on our own or in collaboration with third parties, we may not besuccessful in commercializing our product candidates or realizing the synergies in the target indications of our programs, even if they are approved.We do not have any infrastructure for the sales, marketing or distribution of our products, and the cost of establishing and maintaining suchan organization may exceed the cost-effectiveness of doing so. We expect to build a focused sales, distribution and marketing infrastructure to marketour product candidates in the United States and European Union, if approved. There are significant expenses and risks involved with establishing ourown sales, marketing and distribution capabilities, including our ability to hire, retain and appropriately incentivize qualified individuals, generatesufficient sales leads, provide adequate training to sales and marketing personnel, and effectively manage a geographically dispersed sales andmarketing team. Any failure or delay in the development of our internal sales, marketing and distribution capabilities could delay any product launch,which would adversely impact the commercialization of our product candidates. Additionally, if the commercial launch of our product candidates forwhich we recruit a sales force and establish marketing capabilities is delayed or does not occur for any reason, we would have prematurely orunnecessarily incurred these commercialization expenses. This may be costly, and our investment would be lost if we cannot retain or reposition oursales and marketing personnel.We may not have the resources in the foreseeable future to allocate to the sales and marketing of our product candidates in certaininternational markets. Therefore, our future sales in these markets will largely depend on our ability to enter into and maintain collaborativerelationships for such capabilities, the collaborator’s strategic interest in the product and such collaborator’s ability to successfully market and sell theproduct. We may pursue collaborative arrangements regarding the sale and marketing of AAV-GAD, , AAV-RPE65, AAV-AQP1 or other future genetherapy programs, if approved, for the United States and/or certain markets overseas; however, we cannot assure that we will be able to establish ormaintain such collaborative arrangements, or if able to do so, that they will have effective sales forces.If we are unable to build our own sales force or negotiate a collaborative relationship for the commercialization of AAV-GAD, AAV-RPE65or AAV-AQP1, we may be forced to delay the potential 93Table of Contentscommercialization of AAV-GAD, AAV-RPE65 or AAV-AQP1 or reduce the scope of our sales or marketing activities for AAV-GAD, AAV-RPE65 orAAV-AQP1. If we elect to increase our expenditures to fund commercialization activities internationally, we will need to obtain additional capital,which may not be available to us on acceptable terms, or at all. We could enter into arrangements with collaborative partners at an earlier stage thanotherwise would be ideal and we may be required to relinquish rights to AAV-GAD, AAV-RPE65 or AAV-AQP1 or otherwise agree to termsunfavorable to us, any of which may have an adverse effect on our business, operating results and prospects.Some indications targeted by our ophthalmology programs are rare, but we anticipate realizing synergies in commercializing of our IRDproduct candidates, should they be approved. Failure to realize synergies in our sales, marketing and distribution efforts may harm ourcommercialization efforts.If we are unable to establish adequate sales, marketing and distribution capabilities, either on our own or in collaboration with third parties,we will not be successful in commercializing AAV-GAD, AAV-RPE65 or AAV-AQP1 and may not become profitable and may incur significantadditional losses. We will be competing with many companies that currently have extensive and well-funded marketing and sales operations. Withoutan internal team or the support of a third party to perform marketing and sales functions, we may be unable to compete successfully against these moreestablished companies.If we obtain approval to commercialize any products outside of the United States or the European Union, a variety of risks associated withinternational operations could adversely affect our business.If AAV-GAD, AAV-RPE65 or AAV-AQP1 are approved for commercialization, we intend to enter into agreements with third parties tomarket them in certain jurisdictions outside the United States and the European Union. We expect that we will be subject to additional risks related tointernational pharmaceutical operations, including: • different regulatory requirements for drug and biologic approvals and rules governing drug and biologic commercialization inforeign countries; • reduced protection for intellectual property rights; • foreign reimbursement, pricing and insurance regimes; • unexpected changes in tariffs, trade barriers and regulatory requirements; • economic weakness, including inflation, or political instability in particular foreign economies and markets; • foreign currency fluctuations, which could result in increased operating expenses and reduced revenues, and other obligationsincident to doing business in another country; • business interruptions resulting from geopolitical actions, including war and terrorism or natural disasters including earthquakes,typhoons, floods and fires, or from economic or political instability; • greater difficulty with enforcing our contracts; • potential noncompliance with the U.S. Foreign Corrupt Practices Act, the U.K. Bribery Act 2010 and similar anti-bribery andanticorruption laws in other jurisdictions; and • production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad.We have no prior experience in these areas. In addition, there are complex regulatory, tax, labor and other legal requirements imposed byindividual countries in Europe with which we will need to comply. If we are unable to successfully manage the challenges of international expansionand operations, our business and operating results could be harmed. 94Table of ContentsAny product candidates for which we intend to seek approval as biologic products may face competition sooner than anticipated.The ACA includes a subtitle called the Biologics Price Competition and Innovation Act of 2009, or BPCIA, which created an abbreviatedapproval pathway for biological products that are biosimilar to or interchangeable with an FDA-licensed reference biological product. Under theBPCIA, an application for a biosimilar product may not be submitted to the FDA until four years following the date that the reference product was firstlicensed by the FDA. In addition, the approval of a biosimilar product may not be made effective by the FDA until 12 years from the date on which thereference product was first licensed. During this 12-year period of exclusivity, another company may still market a competing version of the referenceproduct if the FDA approves a full BLA for the competing product containing the sponsor’s own pre-clinical data and data from adequate and well-controlled clinical trials to demonstrate the safety, purity and potency of their product. The law is complex and is still being interpreted andimplemented by the FDA. As a result, its ultimate impact, implementation, and meaning are subject to uncertainty. While it is uncertain when suchprocesses intended to implement BPCIA may be fully adopted by the FDA, any such processes could have an adverse effect on the future commercialprospects for our biological products.There is a risk that any of our product candidates approved as a biological product under a BLA would not qualify for the 12-year period ofexclusivity or that this exclusivity could be shortened due to congressional action or otherwise, or that the FDA will not consider our productcandidates to be reference products for competing products, potentially creating the opportunity for generic competition sooner than anticipated. Otheraspects of the BPCIA, some of which may impact the BPCIA exclusivity provisions, have also been the subject of recent litigation. Moreover, theextent to which a biosimilar, once approved, will be substituted for any one of our reference products in a way that is similar to traditional genericsubstitution for non-biological products is not yet clear, and will depend on a number of marketplace and regulatory factors that are still developing.Risks Related to Our Dependence on Third PartiesIf our cGMP manufacturing facility is unable to supply our product candidates for all of our current preclinical, clinical and potential commercialneeds, we will be forced to seek out third-party manufacturers. We currently contract with third parties for the manufacture of plasmid used inproducing our product candidates. Relying on third parties increases the risk that we will not have sufficient quantities of such materials, productcandidates, or any medicines that we may develop and commercialize, or that such supply will not be available to us at an acceptable cost, whichcould delay, prevent, or impair our development or commercialization efforts.We produce our product candidates in our facility. However, if our facility is damaged, suffers any form of delay or regulatory challenges,or we are unable to scale our internal manufacturing capabilities to meet demand for our product candidates, we will need to contract with third-partymanufacturers to produce our product candidates.We currently rely on third-party manufacturers for the manufacture of plasmid used in the production of our product candidates. We do nothave a long-term supply agreement with any of the third-party manufacturers, and we purchase our required supply on a purchase order basis.We may be unable to establish any agreements with third-party manufacturers or to do so on acceptable terms. Even if we are able toestablish agreements with third-party manufacturers, reliance on third-party manufacturers entails additional risks, including: • the possible breach of the manufacturing agreement by the third party; • the possible termination or nonrenewal of the agreement by the third party at a time that is costly or inconvenient for us; and • reliance on the third party for regulatory compliance, quality assurance, safety, and pharmacovigilance and related reporting. 95Table of ContentsThird-party manufacturers may not be able to comply with cGMP regulations or similar regulatory requirements that might be required bythe FDA or EMA. Our failure, or the failure of our third-party manufacturers, to comply with applicable regulations could result in sanctions beingimposed on us, including fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocations, seizures or recalls ofproduct candidates or medicines, operating restrictions, and criminal prosecutions, any of which could adversely affect supplies of our candidates andharm our business, financial condition, results of operations, and prospects.Any therapies that we may develop may compete with other product candidates and products for access to manufacturing facilities. Thereare a limited number of manufacturers that operate under cGMP regulations and that might be capable of manufacturing for us. Any performance failureon the part of our existing or future manufacturers could delay clinical development or marketing approval.Our current and anticipated future dependence upon others for the manufacture of any product candidates we may develop or anycomponents required for the manufacture of our product candidates may adversely affect our future profit margins and our ability to commercialize anyproduct candidates that receive marketing approval on a timely and competitive basis.We may collaborate with third parties for the development and commercialization of our product candidates. We may not succeed in establishingand maintaining collaborative relationships, which may significantly limit our ability to develop and commercialize our product candidatessuccessfully, if at all.We may seek collaborative relationships for the development and commercialization of our product candidates. Failure to obtain acollaborative relationship for our product candidates may significantly impair their commercial potential. We also may need to enter into collaborativerelationships to provide funding to support our other research and development programs. The process of establishing and maintaining collaborativerelationships is difficult, time-consuming and involves significant uncertainty, such as: • a collaboration partner may shift its priorities and resources away from our product candidates due to a change in businessstrategies, or a merger, acquisition, sale or downsizing; • a collaboration partner may seek to renegotiate or terminate their relationships with us due to unsatisfactory clinical results,manufacturing issues, a change in business strategy, a change of control or other reasons; • a collaboration partner may cease development in therapeutic areas which are the subject of our strategic collaboration; • a collaboration partner may not devote sufficient capital or resources towards our product candidates; • a collaboration partner may change the success criteria for a product candidate thereby delaying or ceasing development of suchcandidate; • a significant delay in initiation of certain development activities by a collaboration partner will also delay payment of milestonestied to such activities, thereby impacting our ability to fund our own activities; • a collaboration partner could develop a product that competes, either directly or indirectly, with our product candidate; • a collaboration partner with commercialization obligations may not commit sufficient financial or human resources to themarketing, distribution or sale of a product; • a collaboration partner with manufacturing responsibilities may encounter regulatory, resource or quality issues and be unable tomeet demand requirements; 96Table of Contents • a collaboration partner may terminate a strategic alliance; • a dispute may arise between us and a partner concerning the research, development or commercialization of a product candidateresulting in a delay in milestones, royalty payments or termination of an alliance and possibly resulting in costly litigation orarbitration which may divert management attention and resources; and • a partner may use our products or technology in such a way as to make us subject to litigation with a third party.If any collaborator fails to fulfill its responsibilities in a timely manner, or at all, our research, clinical development, manufacturing orcommercialization efforts related to that collaboration could be delayed or terminated, or it may be necessary for us to assume responsibility forexpenses or activities that would otherwise have been the responsibility of our collaborator. If we are unable to establish and maintain collaborativerelationships on acceptable terms or to successfully transition terminated collaborative agreements, we may have to delay or discontinue furtherdevelopment of one or more of our product candidates, undertake development and commercialization activities at our own expense or find alternativesources of capital. Moreover, any collaborative partners we enter into agreements with in the future may shift their priorities and resources away fromour product candidates or seek to renegotiate or terminate their relationships with us.Risks Related to Intellectual PropertyWe depend on proprietary technology licensed from others. If we lose our existing licenses or are unable to acquire or license additional proprietaryrights from third parties, we may not be able to continue developing our product candidates.We currently in-license certain intellectual property from UCL Business, Plc, or UCLB, and Brandeis University, or Brandeis, and theNational Institute of Dental and Craniofacial Research, or NIDCR, a division of the NIH. We are a party to agreements with UCLB for certaintechnology and AAV vector-related patents and with Brandeis for certain preclinical technology for the treatment of ALS. Further, we are party to anagreement with NIDCR for technology relating to the treatment of Sjogren’s syndrome. We may enter into additional agreements, including licenseagreements, with other parties in the future that impose diligence, development and commercialization timelines, milestone payments, royalties,insurance and other obligations on us. For example, in exchange for the rights granted to us by UCLB, we are obligated to pay an annual managementfee, milestone payments for certain commercial sales thresholds, and a certain percentages of proceeds on sublicensing revenues. If we fail to complywith our obligations to UCLB, Brandeis, NIDCR, or any of our other collaborators, our counterparties may have the right to terminate these agreements,in which event we might not be able to develop, manufacture or market any product candidate that is covered by these agreements, which couldadversely affect the value of the product candidate being developed under any such agreement. Termination of these agreements or reduction orelimination of our rights under these agreements may result in our having to negotiate new or reinstated agreements with less favorable terms, or causeus to lose our rights under these agreements, including our rights to important intellectual property or technology.We may rely on other third parties from whom we license proprietary technology to file and prosecute patent applications and maintainpatents and otherwise protect the intellectual property we license from them. We may have limited control over these activities or any other intellectualproperty that may be related to our in-licensed intellectual property. For example, we cannot be certain that such activities by these licensors will beconducted in compliance with applicable laws and regulations or will result in valid and enforceable patents and other intellectual property rights. Wemay have limited control over the manner in which our licensors initiate an infringement proceeding against a third-party infringer of the intellectualproperty rights, or defend certain of the intellectual property that may be licensed to us. It is possible that the licensors’ infringement proceeding ordefense activities may be less vigorous than if we conduct them ourselves. The licensing and acquisition of third-party intellectual property rights is acompetitive practice, and companies that may be more established, or have 97Table of Contentsgreater resources than we do, may also be pursuing strategies to license or acquire third-party intellectual property rights that we may considernecessary or attractive in order to commercialize our product candidates. More established companies may have a competitive advantage over us dueto their larger size and cash resources or greater clinical development and commercialization capabilities. There can be no assurance that we will beable to successfully complete such negotiations and ultimately acquire the rights to the intellectual property surrounding the additional productcandidates that we may seek to acquire. If we are unable to obtain and maintain patent protection for our technology and product candidates or if thescope of the patent protection obtained is not sufficiently broad, we may not be able to compete effectively in our markets.We rely upon a combination of patents, trade secret protection and confidentiality agreements to protect the intellectual property related toour proprietary technologies, product candidate development programs and product candidates. Our success depends in large part on our ability tosecure and maintain patent protection in the United States and other countries with respect to our current product candidates and any future productcandidates we may develop. We seek to protect our proprietary position by filing or collaborating with our licensors to file patent applications in theUnited States and abroad related to our proprietary technologies, development programs and product candidates. The patent prosecution process isexpensive and time-consuming, and we may not be able to file and prosecute all necessary or desirable patent applications at a reasonable cost or in atimely manner. Moreover, the issuance, scope, validity, enforceability and commercial value of our patent rights are highly uncertain.It is also possible that we will fail to identify patentable aspects of our research and development output before it is too late to obtainpatent protection. We may not have the right to control the preparation, filing, and prosecution of patent applications, or to maintain the rights topatents licensed to third parties. Therefore, these patents and patent applications may not be prosecuted and enforced in a manner consistent with thebest interests of our business. The patent applications that we own or in-license may fail to result in issued patents with claims that cover ourproprietary products and technology, including current product candidates, any future product candidates we may develop, and our gene regulationtechnology in the United States or in other foreign countries, in whole or in part. Alternately, our existing patents and any future patents we obtain maynot be sufficiently broad to prevent others from using our technology or from developing competing products and technologies. There is no assurancethat all potentially relevant prior art relating to our patents and patent applications has been found, which can prevent a patent from issuing from apending patent application or later invalidate or narrow the scope of an issued patent. For example, publications of discoveries in the scientificliterature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until18 months after filing or, in some cases, not at all. Therefore, we cannot know with certainty whether we were the first to make the inventions claimedin our patents or pending patent applications, or that we were the first to file for patent protection of such inventions. Even if patents do successfullyissue and even if such patents cover our current product candidates, any future product candidates we may develop and our gene regulationtechnology, third parties may challenge their validity, enforceability or scope thereof, which may result in such patents being narrowed, invalidated, orheld unenforceable. Any successful challenge to these patents or any other patents owned by or licensed to us could deprive us of rights necessary forthe successful commercialization of any of our product candidates or gene regulation technology. Our competitors may be able to circumvent ourpatents by developing similar or alternative product candidates in a non-infringing manner. Further, if we encounter delays in regulatory approvals, theperiod of time during which we could market a product candidate and our gene regulation technology under patent protection could be reduced.If the patent applications we hold or have in-licensed with respect to our development programs and product candidates fail to issue, iftheir validity, breadth or strength of protection is threatened, or if they fail to provide meaningful exclusivity for any of our current or future productcandidates or technology, it could dissuade companies from collaborating with us to develop product candidates, encourage competitors to developcompeting products or technologies and threaten our ability to commercialize future product candidates. Any such outcome could harm our business. 98Table of ContentsThe patent position of biotechnology and pharmaceutical companies is highly uncertain, involves complex legal and factual questions,and is characterized by the existence of large numbers of patents and frequent litigation based on allegations of patent or other intellectual propertyinfringement or violation. In addition, the laws of jurisdictions outside the United States may not protect our rights to the same extent as the laws of theUnited States. For example, European patent law restricts the patentability of methods of treatment of the human body more than United States lawdoes. Changes in either the patent laws or interpretation of the patent laws in the United States and other countries may diminish the value of ourpatents or narrow the scope of our patent protection. Since patent applications in the United States and other jurisdictions are confidential for a periodof time after filing, we cannot be certain that we were the first to file for patents covering our inventions. As a result, the issuance, scope, validity,enforceability and commercial value of our patent rights are highly uncertain. Our pending and future patent applications may not result in theissuance of patents, or may result in the issuance of patents which fail to protect our technology or products, in whole or in part, or which fail toeffectively prevent others from commercializing competitive technologies and products.The issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, and our owned and licensed patents maybe challenged in the courts or patent offices in the United States and abroad. Such challenges may result in loss of exclusivity or freedom to operate orin patent claims being narrowed, invalidated or held unenforceable, in whole or in part, which could limit our ability to stop others from using orcommercializing similar or identical technology and products, or limit the duration of the patent protection of our technology and products. Thus,even if our patent applications issue as patents, they may not issue in a form that will provide us with meaningful protection, prevent competitors fromcompeting with us or otherwise provide us with any competitive advantage. Moreover, patents have a limited lifespan. In the United States, the naturalexpiration of a patent is generally 20 years after it is filed. Various extensions may be available; however, the life of a patent, and the protection itaffords, is limited. Without patent protection for our current or future product candidates, we may be open to competition from generic versions of suchproducts. Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting suchcandidates might expire before or shortly after such candidates are commercialized. As a result, our owned and licensed patent portfolio may notprovide us with sufficient rights to exclude others from commercializing products similar or identical to ours.Third parties may assert claims against us alleging infringement of their patents and proprietary rights, or we may need to become involved inlawsuits to defend or enforce our patents, either of which could result in substantial costs or loss of productivity, delay or prevent the developmentand commercialization of our product candidates, prohibit our use of proprietary technology or sale of products or put our patents and otherproprietary rights at risk.Our commercial success depends, in part, upon our ability to develop, manufacture, market and sell our product candidates without allegedor actual infringement, misappropriation or other violation of the patents and proprietary rights of third parties. However, our research, developmentand commercialization activities may be subject to claims that we infringe or otherwise violate patents or other intellectual property rights owned orcontrolled by third parties. Litigation relating to infringement or misappropriation of patent and other intellectual property rights in thepharmaceutical and biotechnology industries is common, including patent infringement lawsuits, interferences, oppositions and inter partes reviews,and reexamination proceedings before the U.S. Patent and Trademark Office, or USPTO, and corresponding foreign patent offices. The various marketsin which we plan to operate are subject to frequent and extensive litigation regarding patents and other intellectual property rights. In addition, manycompanies in intellectual property-dependent industries, including the biotechnology and pharmaceutical industries, have employed intellectualproperty litigation as a means to gain an advantage over their competitors. Numerous U.S., EU and foreign issued patents and pending patentapplications, which are owned by third parties, exist in the fields in which we are developing product candidates, and as the biotechnology andpharmaceutical industries expand and more patents are issued, the risk increases that our product candidates may be subject to claims of infringementof the intellectual property rights of third parties. Some claimants may have substantially greater resources than we do and may be able to sustain thecosts of 99Table of Contentscomplex intellectual property litigation to a greater degree and for longer periods of time than we could. In addition, patent holding companies thatfocus solely on extracting royalties and settlements by enforcing patent rights may target us.We may be subject to third-party claims including infringement, interference or derivation proceedings, post-grant review and inter partesreview before the USPTO or similar adversarial proceedings or litigation in other jurisdictions. Even if such claims are without merit, a court ofcompetent jurisdiction could hold that these third-party patents are valid, enforceable and infringed, and the holders of any such patents may be able toblock our ability to commercialize the applicable product candidate unless we obtained a license under the applicable patents, or until such patentsexpire or are finally determined to be invalid or unenforceable. There may be third-party patents or patent applications with claims to compositions,formulations, or methods of treatment, prevention use, or manufacture of our product candidates or technologies. Because patent applications can takemany years to issue, there may be currently pending patent applications which may later result in issued patents that our product candidates mayinfringe. In addition, third parties may obtain patents in the future and claim that use of our technologies infringes upon these patents. If any third-party patents were held by a court of competent jurisdiction to cover aspects of our compositions, formulations, or methods of treatment, prevention oruse, the holders of any such patents may be able to prohibit our use of those compositions, formulations, methods of treatment, prevention or use orother technologies, effectively blocking our ability to develop and commercialize the applicable product candidate until such patent expires or isfinally determined to be invalid or unenforceable or unless we obtained a license.In addition, defending such claims would cause us to incur substantial expenses and, if successful, could cause us to pay substantialdamages if we are found to be infringing a third party’s patent rights. These damages potentially include increased damages (possibly treble damages)and attorneys’ fees if we are found to have infringed such rights willfully. Further, if a patent infringement suit is brought against us or our third-partyservice providers, our development, manufacturing or sales activities relating to the product or product candidate that is the subject of the suit may bedelayed or terminated. As a result of patent infringement claims, or in order to avoid potential infringement claims, we may choose to seek, or berequired to seek, a license from the third party, which may require payment of substantial royalties or fees, or require us to grant a cross-license underour intellectual property rights. These licenses may not be available on reasonable terms or at all. Even if a license can be obtained on reasonableterms, the rights may be nonexclusive, which would give our competitors access to the same intellectual property rights. If we are unable to enter into alicense on acceptable terms, we could be prevented from commercializing one or more of our product candidates, or forced to modify such productcandidates, or to cease some aspect of our business operations, which could harm our business significantly. We might also be forced to redesign ormodify our product candidates so that we no longer infringe the third-party intellectual property rights, which may result in significant cost or delay tous, or which redesign or modification could be impossible or technically infeasible. Even if we were ultimately to prevail, any of these events couldrequire us to divert substantial financial and management resources that we would otherwise be able to devote to our business. In addition, if thebreadth or strength of protection provided the patents and patent applications we own or in-license is threatened, it could dissuade companies fromcollaborating with us to license, develop or commercialize current or future product candidates.Competitors may infringe our patents or other intellectual property. If we or one of our licensors were to initiate legal proceedings against athird party to enforce a patent covering one of our product candidates, the defendant could counterclaim that our patent is invalid or unenforceable. Inpatent litigation in the United States and in Europe, defendant counterclaims alleging invalidity or unenforceability are commonplace. Grounds for avalidity challenge could be an alleged failure to meet any of several statutory requirements, for example, lack of novelty, obviousness lack of writtendescription, or non-enablement. Third parties might allege unenforceability of our patents because during prosecution of the patent an individualconnected with such prosecution withheld relevant information, or made a misleading statement. Interference or derivation proceedings provoked bythird parties or brought by us or declared by the USPTO may be necessary to determine the priority of inventions with respect to our patents or patentapplications. The outcome of proceedings involving assertions of invalidity and 100Table of Contentsunenforceability during patent litigation is unpredictable. With respect to the validity of patents, for example, we cannot be certain that there is noinvalidating prior art of which we and the patent examiner were unaware during prosecution, but that an adverse third party may identify and submit insupport of such assertions of invalidity. If a defendant were to prevail on a legal assertion of invalidity or unenforceability, we would lose at least part,and perhaps all, of the patent protection on our product candidates. Our patents and other intellectual property rights also will not protect ourtechnology if competitors design around our protected technology without infringing our patents or other intellectual property rights.Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significantexpenses and could distract our technical and management personnel from their normal responsibilities. In addition, because of the substantial amountof discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could becompromised by disclosure during this type of litigation. There could also be public announcements of the results of hearings, motions or other interimproceedings or developments, and if securities analysts or investors view these announcements in a negative light, the price of our ordinary sharescould be adversely affected. Such litigation or proceedings could substantially increase our operating losses and reduce our resources available fordevelopment activities. We may not have sufficient financial or other resources to adequately conduct such litigation or proceedings. Some of ourcompetitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of their substantially greaterfinancial resources. Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could have an adverse effecton our ability to compete in the marketplace.We may not identify relevant third-party patents or may incorrectly interpret the relevance, scope or expiration of a third-party patent, which mightadversely affect our ability to develop, manufacture and market our product candidates.We cannot guarantee that any of our or our licensors’ patent searches or analyses, including but not limited to the identification of relevantpatents, analysis of the scope of relevant patent claims or determination of the expiration of relevant patents, are complete or thorough, nor can we becertain that we have identified each and every third-party patent and pending application in the United States, Europe and elsewhere that is relevant toor necessary for the commercialization of our product candidates in any jurisdiction. For example, in the United States, applications filed beforeNovember 29, 2000 and certain applications filed after that date that will not be filed outside the United States remain confidential until patents issue.Patent applications in the United States, the European Union and elsewhere are published approximately 18 months after the earliest filing for whichpriority is claimed, with such earliest filing date being commonly referred to as the priority date. Therefore, patent applications covering our productcandidates could be filed by others without our knowledge. Additionally, pending patent applications that have been published can, subject to certainlimitations, be later amended in a manner that could cover our product candidates or the use of our product candidates. After issuance, the scope ofpatent claims remains subject to construction as determined by an interpretation of the law, the written disclosure in a patent and the patent’sprosecution history. Our interpretation of the relevance or the scope of a patent or a pending application may be incorrect, which may negativelyimpact our ability to market our product candidates. We may incorrectly determine that our product candidates are not covered by a third-party patentor may incorrectly predict whether a third party’s pending application will issue with claims of relevant scope. Our determination of the expiration dateof any patent in the United States, the European Union or elsewhere that we consider relevant may be incorrect, which may negatively impact ourability to develop and market our product candidates. Our failure to identify and correctly interpret relevant patents may negatively impact our abilityto develop and market our product candidates.If we fail to correctly identify or interpret relevant patents, we may be subject to infringement claims. We cannot guarantee that we will beable to successfully settle or otherwise resolve such infringement claims. If we fail in any such dispute, in addition to being forced to pay monetarydamages, we may be temporarily or permanently prohibited from commercializing our product candidates. We might, if possible, also be forced to 101Table of Contentsredesign our product candidates in a manner that no longer infringes third-party intellectual property rights. Any of these events, even if we wereultimately to prevail, could require us to divert substantial financial and management resources that we would otherwise be able to devote to ourbusiness.Changes in patent laws or patent jurisprudence could diminish the value of patents in general, thereby impairing our ability to protect our productcandidates.As is the case with other biotechnology companies, our success is heavily dependent on intellectual property, particularly patents.Obtaining and enforcing patents in the biotechnology and genetic medicine industries involve both technological complexity and legal complexity.Therefore, obtaining and enforcing biotechnology and genetic medicine patents is costly, time-consuming and inherently uncertain. In addition, theLeahy-Smith America Invents Act, or the AIA, which was passed in September 2011, resulted in significant changes to the U.S. patent system.An important change introduced by the AIA is that, as of March 16, 2013, the United States transitioned from a “first-to-invent” to a“first-to-file” system for deciding which party should be granted a patent when two or more patent applications are filed by different parties claimingthe same invention. Under a “first-to-file” system, assuming the other requirements for patentability are met, the first inventor to file a patentapplication generally will be entitled to a patent on the invention regardless of whether another inventor had made the invention earlier. A third partythat files a patent application in the USPTO after that date but before us could therefore be awarded a patent covering an invention of ours even if wemade the invention before it was made by the third party. This will require us to be cognizant going forward of the time from invention to filing of apatent application and diligent in filing patent applications, but circumstances could prevent us from promptly filing patent applications on ourinventions.Among some of the other changes introduced by the AIA are changes that limit where a patentee may file a patent infringement suit andproviding opportunities for third parties to challenge any issued patent in the USPTO. This applies to all of our U.S. patents, even those issued beforeMarch 16, 2013. Because of a lower evidentiary standard in USPTO proceedings compared to the evidentiary standard in U.S. federal courts necessaryto invalidate a patent claim, a third party could potentially provide evidence in a USPTO proceeding sufficient for the USPTO to hold a claim invalideven though the same evidence would be insufficient to invalidate the claim if first presented in a district court action.Accordingly, a third party may attempt to use the USPTO procedures to invalidate our patent claims that would not have been invalidatedif first challenged by the third party as a defendant in a district court action. It is not clear what, if any, impact the AIA will have on the operation of ourbusiness. However, the AIA and its implementation could increase the uncertainties and costs surrounding the prosecution of our or our licensors’patent applications and the enforcement or defense of our or our licensors’ issued patents.We may become involved in opposition, interference, derivation, inter partes review or other proceedings challenging our or our licensors’patent rights, and the outcome of any proceedings are highly uncertain. An adverse determination in any such proceeding could reduce the scope of, orinvalidate, our owned or in-licensed patent rights, allow third parties to commercialize our technology or products and compete directly with us,without payment to us, or result in our inability to manufacture or commercialize products without infringing third-party patent rights.Additionally, the U.S. Supreme Court has ruled on several patent cases in recent years either narrowing the scope of patent protectionavailable in certain circumstances or weakening the rights of patent owners in certain situations, and there are other open questions under patent lawthat courts have yet to decisively address. In addition to increasing uncertainty with regard to our ability to obtain patents in the future, thiscombination of events has created uncertainty with respect to the value of patents, once obtained. Depending on decisions by Congress, the federalcourts and the USPTO, the laws and regulations governing patents could change in 102Table of Contentsunpredictable ways and could weaken our ability to obtain new patents or to enforce our existing patents and patents that we might obtain in thefuture. In addition, the European patent system is relatively stringent in the type of amendments that are allowed during prosecution, but, thecomplexity and uncertainty of European patent laws has also increased in recent years. Complying with these laws and regulations could limit ourability to obtain new patents in the future that may be important for our business.Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment and otherrequirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with theserequirements.The USPTO, European and other patent agencies require compliance with a number of procedural, documentary, fee payment and othersimilar provisions during the patent application process. In addition, periodic maintenance and annuity fees on any issued patent are due to be paid tothe USPTO, European and other patent agencies over the lifetime of a patent. While an inadvertent failure to make payment of such fees or to complywith such provisions can in many cases be cured by additional payment of a late fee or by other means in accordance with the applicable rules, thereare situations in which non-compliance with such provisions will result in the abandonment or lapse of the patent or patent application, and the partialor complete loss of patent rights in the relevant jurisdiction. Non-compliance events that could result in abandonment or lapse of a patent or patentapplication include failure to respond to official actions within prescribed time limits, non-payment of fees and failure to properly legalize and submitformal documents within prescribed time limits. If we or our licensors fail to maintain the patents and patent applications covering our productcandidates or if we or our licensors otherwise allow our patents or patent applications to be abandoned or lapse, it can create opportunities forcompetitors to enter the market, which would hurt our competitive position and could impair our ability to successfully commercialize our productcandidates in any indication for which they are approved.We enjoy only limited geographical protection with respect to certain patents and we may not be able to protect our intellectual property rightsthroughout the world.Filing, prosecuting and defending patents covering our product candidates in all countries throughout the world would be prohibitivelyexpensive, and our intellectual property rights in some countries outside the United States can be less extensive than those in the United States.In-licensing patents covering our product candidates in all countries throughout the world may similarly be prohibitively expensive, if suchopportunities are available at all. And in-licensing or filing, prosecuting and defending patents even in only those jurisdictions in which we develop orcommercialize our product candidates may be prohibitively expensive or impractical. Competitors may use our and our licensors’ technologies injurisdictions where we have not obtained patent protection or licensed patents to develop their own products and, further, may export otherwiseinfringing products to territories where we and our licensors have patent protection, but enforcement is not as strong as that in the United States or theEuropean Union. These products may compete with our product candidates, and our or our licensors’ patents or other intellectual property rights maynot be effective or sufficient to prevent them from competing.In addition, we may decide to abandon national and regional patent applications while they are still pending. The grant proceeding of eachnational or regional patent is an independent proceeding which may lead to situations in which applications may be rejected by the relevant patentoffice, while substantively similar applications are granted by others. For example, relative to other countries, China has a heightened requirement forpatentability and specifically requires a detailed description of medical uses of a claimed drug. Furthermore, generic drug manufacturers or othercompetitors may challenge the scope, validity or enforceability of our or our licensors’ patents, requiring us or our licensors to engage in complex,lengthy and costly litigation or other proceedings. Generic drug manufacturers may develop, seek approval for and launch generic versions of ourproducts. It is also quite common that depending on the country, the scope of patent protection may vary for the same product candidate ortechnology. 103Table of ContentsThe laws of some jurisdictions do not protect intellectual property rights to the same extent as the laws or regulations in the United Statesand the European Union, and many companies have encountered significant difficulties in protecting and defending proprietary rights in suchjurisdictions. Moreover, the legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents, tradesecrets or other forms of intellectual property, which could make it difficult for us to prevent competitors in some jurisdictions from marketingcompeting products in violation of our proprietary rights generally. Proceedings to enforce our patent rights in foreign jurisdictions, whether or notsuccessful, are likely to result in substantial costs and divert our efforts and attention from other aspects of our business, and additionally could put atrisk our or our licensors’ patents of being invalidated or interpreted narrowly, could increase the risk of our or our licensors’ patent applications notissuing, or could provoke third parties to assert claims against us. We may not prevail in any lawsuits that we initiate, while damages or other remediesmay be awarded to the adverse party, which may be commercially significant. If we prevail, damages or other remedies awarded to us, if any, may notbe commercially meaningful. Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain asignificant commercial advantage from the intellectual property that we develop or license. Furthermore, while we intend to protect our intellectualproperty rights in our expected significant markets, we cannot ensure that we will be able to initiate or maintain similar efforts in all jurisdictions inwhich we may wish to market our product candidates. Accordingly, our efforts to protect our intellectual property rights in such countries may beinadequate, which may have an adverse effect on our ability to successfully commercialize our product candidates in all of our expected significantforeign markets. If we or our licensors encounter difficulties in protecting, or are otherwise precluded from effectively protecting, the intellectualproperty rights important for our business in such jurisdictions, the value of these rights may be diminished and we may face additional competition inthose jurisdictions.In some jurisdictions, compulsory licensing laws compel patent owners to grant licenses to third parties. In addition, some countries limitthe enforceability of patents against government agencies or government contractors. In these countries, the patent owner may have limited remedies,which could materially diminish the value of such patent. If we or any of our licensors are forced to grant a license to third parties under patentsrelevant to our business, or if we or our licensors are prevented from enforcing patent rights against third parties, our competitive position may besubstantially impaired in such jurisdictions.Patent terms may be inadequate to protect our competitive position on our product candidates for an adequate amount of time.The term of any individual patent depends on applicable law in the country where the patent is granted. In the United States, provided allmaintenance fees are timely paid, a patent generally has a term of 20 years from its application filing date or earliest claimed non-provisional filingdate. Extensions may be available under certain circumstances, but the life of a patent and, correspondingly, the protection it affords is limited. Even ifwe or our licensors obtain patents covering our product candidates, when the terms of all patents covering a product expire, our business may becomesubject to competition from competitive medications, including generic medications. Given the amount of time required for the development, testingand regulatory review and approval of new product candidates, patents protecting such candidates may expire before or shortly after such candidatesare commercialized. As a result, our owned and licensed patent portfolio may not provide us with sufficient rights to exclude others fromcommercializing products similar or identical to ours.If we do not obtain patent term extension in the United States under the Hatch-Waxman Act and in foreign countries under similar legislation,thereby potentially extending the term of marketing exclusivity for our product candidates, our business may be harmed.In the United States, a patent that covers an FDA-approved drug or biologic may be eligible for a term extension designed to restore theperiod of the patent term that is lost during the premarket regulatory review process conducted by the FDA. Depending upon the timing, duration andconditions of FDA marketing approval of our product candidates, one or more of our U.S. patents may be eligible for limited patent term extension 104Table of Contentsunder the Drug Price Competition and Patent Term Restoration Act of 1984, or the Hatch- Waxman Act, which permits a patent term extension of up tofive years for a patent covering an approved product as compensation for effective patent term lost during product development and the FDAregulatory review process. In the European Union, our product candidates may be eligible for term extensions based on similar legislation. In eitherjurisdiction, however, we may not receive an extension if we fail to apply within applicable deadlines, fail to apply prior to expiration of relevantpatents or otherwise fail to satisfy applicable requirements. Even if we are granted such extension, the duration of such extension may be less than ourrequest. If we are unable to obtain a patent term extension, or if the term of any such extension is less than our request, the period during which we canenforce our patent rights for that product will be in effect shortened and our competitors may obtain approval to market competing products sooner.The resulting reduction of years of revenue from applicable products could be substantial.Our proprietary rights may not adequately protect our technologies and product candidates, and do not necessarily address all potential threats toour competitive advantage.The degree of future protection afforded by our intellectual property rights is uncertain because intellectual property rights havelimitations, and may not adequately protect our business, or permit us to maintain our competitive advantage. The following examples are illustrative: • others may be able to make products that are the same as or similar to our product candidates but that are not covered by the claimsof the patents that we own or have exclusively licensed; • others, including inventors or developers of our owned or in-licensed patented technologies who may become involved withcompetitors, may independently develop similar technologies that function as alternatives or replacements for any of ourtechnologies without infringing our intellectual property rights; • we or our licensors or our other collaboration partners might not have been the first to conceive and reduce to practice theinventions covered by the patents or patent applications that we own, license or will own or license; • we or our licensors or our other collaboration partners might not have been the first to file patent applications covering certain ofthe patents or patent applications that we or they own or have obtained a license, or will own or will have obtained a license; • we or our licensors may fail to meet obligations to the U.S. government with respect to in-licensed patents and patent applicationsfunded by U.S. government grants, leading to the loss of patent rights; • it is possible that our pending patent applications will not result in issued patents; • it is possible that there are prior public disclosures that could invalidate our or our licensors’ patents; • issued patents that we own or exclusively license may not provide us with any competitive advantage, or may be held invalid orunenforceable, as a result of legal challenges by our competitors; • our competitors might conduct research and development activities in countries where we do not have patent rights, or in countrieswhere research and development safe harbor laws exist, and then use the information learned from such activities to developcompetitive products for sale in our major commercial markets; • ownership, validity or enforceability of our or our licensors’ patents or patent applications may be challenged by third parties; and • the patents of third parties or pending or future applications of third parties, if issued, may have an adverse effect on our business. 105Table of ContentsOur reliance on third parties may require us to share our trade secrets, which increases the possibility that our trade secrets will be misappropriatedor disclosed, and confidentiality agreements with employees and third parties may not adequately prevent disclosure of trade secrets and protectother proprietary information.We consider proprietary trade secrets, confidential know-how and unpatented know-how to be important to our business. We may rely ontrade secrets and confidential know-how to protect our technology, especially where patent protection is believed by us to be of limited value.However, trade secrets and confidential know-how are difficult to protect, and we have limited control over the protection of trade secrets andconfidential know-how used by our licensors, collaborators and suppliers. Because we have relied in the past on third parties to manufacture ourproduct candidates, because we may continue to do so in the future, and because we expect to collaborate with third parties on the development of ourcurrent product candidates and any future product candidates we develop, we may, at times, share trade secrets with them. We also conduct jointresearch and development programs that may require us to share trade secrets under the terms of our research and development partnerships or similaragreements. Under such circumstances, trade secrets and confidential know-how can be difficult to maintain as confidential.To protect this type of information against disclosure or appropriation by competitors, our policy is to require our employees, consultants,contractors and advisors to enter into confidentiality agreements and, if applicable, material transfer agreements, consulting agreements or other similaragreements with us prior to beginning research or disclosing proprietary information. These agreements typically limit the rights of the third parties touse or disclose our confidential information, including our trade secrets. However, current or former employees, consultants, contractors and advisersmay unintentionally or willfully disclose our confidential information to competitors, and confidentiality agreements may not provide an adequateremedy in the event of unauthorized disclosure of confidential information. The need to share trade secrets and other confidential information increasesthe risk that such trade secrets become known by our competitors, are inadvertently incorporated into the technology of others, or are disclosed or usedin violation of these agreements. Given that our competitive position is based, in part, on our know-how and trade secrets, a competitor’s discovery ofour trade secrets or other unauthorized use or disclosure would impair our competitive position and may have an adverse effect on our business andresults of operations. Enforcing a claim that a third party obtained illegally and is using trade secrets and/or confidential know-how is expensive, timeconsuming and unpredictable, and the enforceability of confidentiality agreements may vary from jurisdiction to jurisdiction. Courts outside theUnited States are sometimes less willing to protect proprietary information, technology and know-how.In addition, these agreements typically restrict the ability of our advisors, employees, third-party contractors and consultants to publishdata potentially relating to our trade secrets, although our agreements may contain certain limited publication rights. Despite our efforts to protect ourtrade secrets, our competitors may discover our trade secrets, either through breach of our agreements with third parties, independent development orpublication of information by any of our third-party collaborators. A competitor’s discovery of our trade secrets would impair our competitive positionand have an adverse impact on our business.If our trademarks and trade names are not adequately protected, then we may not be able to build name recognition in our markets of interest andour business may be adversely affected.If our trademarks and trade names are not adequately protected, then we may not be able to build name recognition in our markets ofinterest and our business may be adversely affected. Our trademark MeiraGTx has been registered in the EU and United States. We may not be able toprotect our rights to these trademarks and trade names, which we need to build name recognition among potential partners or customers in our marketsof interest. At times, competitors may adopt trade names or trademarks similar to ours, thereby impeding our ability to build brand identity andpossibly leading to market confusion. In addition, there could be potential trade name or trademark infringement claims brought by owners of otherregistered trademarks or trademarks that incorporate variations of our unregistered trademarks or trade names. Over the long term, if we are unable tosuccessfully register our trademarks and trade names and establish name recognition based on our trademarks 106Table of Contentsand trade names, then we may not be able to compete effectively and our business may be adversely affected. Our efforts to enforce or protect ourproprietary rights related to trademarks, trade secrets, domain names, copyrights or other intellectual property may be ineffective and could result insubstantial costs and diversion of resources and could adversely impact our financial condition or results of operations.We may need to license additional intellectual property from third parties, and such licenses may not be available or may not be available oncommercially reasonable terms.The growth of our business may depend in part on our ability to acquire or in-license additional proprietary rights. For example, ourprograms may involve product candidates that may require the use of additional proprietary rights held by third parties. Our product candidates mayalso require specific formulations to work effectively and efficiently. These formulations may be covered by intellectual property rights held by others.We may develop products containing our compositions and pre-existing pharmaceutical compositions. These pharmaceutical products may be coveredby intellectual property rights held by others. We may be required by the FDA, EMA or other foreign regulatory authorities to provide a companiondiagnostic test or tests with our product candidates. These diagnostic test or tests may be covered by intellectual property rights held by others. Wemay be unable to acquire or in-license any relevant third-party intellectual property rights that we identify as necessary or important to our businessoperations. We may fail to obtain any of these licenses at a reasonable cost or on reasonable terms, if at all, which would harm our business. We mayneed to cease use of the compositions or methods covered by such third-party intellectual property rights, and may need to seek to develop alternativeapproaches that do not infringe on such intellectual property rights which may entail additional costs and development delays, even if we were able todevelop such alternatives, which may not be feasible. Even if we are able to obtain a license under such intellectual property rights, any such licensemay be non-exclusive, which may allow our competitors access to the same technologies licensed to us.We may be subject to claims that our employees, consultants or independent contractors have wrongfully used or disclosed confidential informationof their former employers or other third parties.We employ individuals who were previously employed at other biotechnology or pharmaceutical companies. Although we seek to protectour ownership of intellectual property rights by ensuring that our agreements with our employees, collaborators and other third parties with whom wedo business include provisions requiring such parties to assign rights in inventions to us, we may be subject to claims that we or our employees,consultants or independent contractors have inadvertently or otherwise used or disclosed confidential information of our employees’ former employersor other third parties. We may also be subject to claims that former employers or other third parties have an ownership interest in our patents. Litigationmay be necessary to defend against these claims. There is no guarantee of success in defending these claims, and if we fail in defending any suchclaims, in addition to paying monetary damages, we may lose valuable intellectual property rights, such as exclusive ownership of, or right to use,valuable intellectual property. Even if we are successful, litigation could result in substantial cost and reputational loss and be a distraction to ourmanagement and other employees.Risks Related to Employee Matters and Managing GrowthWe will need to expand our organization, and we may experience difficulties in managing this growth, which could disrupt our operations.As of December 31, 2018, we had 68 full-time employees. We will need to significantly expand our organization, and we may havedifficulty identifying, hiring and integrating new personnel. Future growth would impose significant additional responsibilities on our management,including the need to identify, recruit, maintain, motivate and integrate additional employees, consultants and contractors. Also, our management mayneed to divert a disproportionate amount of its attention away from our day-to-day activities and devote a substantial amount of time to managingthese growth activities. We may not be able to effectively manage the 107Table of Contentsexpansion of our operations, which may result in weaknesses in our infrastructure, give rise to operational mistakes, loss of business opportunities, lossof employees and reduced productivity among remaining employees. Our expected growth could require significant capital expenditures and maydivert financial resources from other projects, such as the development of product candidates. If our management is unable to effectively manage ourgrowth, our expenses may increase more than expected, our ability to generate and/or grow revenues could be reduced, and we may not be able toimplement our business strategy. Our future financial performance and our ability to commercialize our product candidates and compete effectivelywill depend, in part, on our ability to effectively manage any future growth. Our expected growth could require significant capital expenditures andmay divert financial resources from other projects, such as the development of additional product candidates. If our management is unable toeffectively manage our expected growth, our expenses may increase more than expected, our potential ability to generate revenue could be reducedand we may not be able to implement our business strategy. Many of the biotechnology companies that we compete against for qualified personnel andconsultants have greater financial and other resources, different risk profiles and a longer history in the industry than we do. If we are unable tocontinue to attract and retain high-quality personnel and consultants, the rate and success at which we can discover and develop product candidatesand operate our business will be limited.Our future success depends on our ability to retain our key personnel and to attract, retain and motivate qualified personnel.Our industry has experienced a high rate of turnover of management personnel in recent years. We are highly dependent on thedevelopment, regulatory, commercialization and business development expertise of Alexandria Forbes, Ph.D., our President and Chief ExecutiveOfficer, Rich Giroux, our Chief Operating Officer and Stuart Naylor, Ph.D., our Chief Development Officer, as well as the other principal members of ourmanagement, scientific and clinical teams. Although we have formal employment agreements with our executive officers, these agreements do notprevent them from terminating their employment with us at any time and, for certain of our executive officers, entitle them to receive severancepayments in connection with their voluntary resignation of employment.If we lose one or more of our executive officers or key employees, our ability to implement our business strategy successfully could beseriously harmed. Furthermore, replacing executive officers and key employees may be difficult and may take an extended period of time because ofthe limited number of individuals in our industry with the breadth of skills and experience required to develop, gain regulatory approval of andcommercialize product candidates successfully. Competition to hire from this limited pool is intense, and we may be unable to hire, train, retain ormotivate these additional key personnel on acceptable terms given the competition among numerous pharmaceutical and biotechnology companiesfor similar personnel. We also experience competition for the hiring of scientific and clinical personnel from universities and research institutions. Inaddition, we rely on consultants and advisors, including scientific and clinical advisors, to assist us in formulating our research and development andcommercialization strategy. Our consultants and advisors may be engaged by entities other than us and may have commitments under consulting oradvisory contracts with other entities that may limit their availability to us. If we are unable to continue to attract and retain high quality personnel, ourability to develop and commercialize product candidates will be limited.Potential product liability lawsuits against us could cause us to incur substantial liabilities and limit commercialization of any products that we maydevelop.The use of our product candidates in clinical trials and the sale of any products for which we obtain marketing approval exposes us to therisk of product liability claims. Product liability claims might be brought against us by consumers, health care providers, pharmaceutical companies orothers selling or otherwise coming into contact with our products. On occasion, large judgments have been awarded in class action lawsuits based onproducts that had unanticipated adverse effects. If we cannot successfully defend against product liability claims, 108Table of Contentswe could incur substantial liability and costs. In addition, regardless of merit or eventual outcome, product liability claims may result in: • impairment of our business reputation and significant negative media attention; • withdrawal of participants from our clinical trials; • significant costs to defend the related litigation and related litigation; • distraction of management’s attention from our primary business; • substantial monetary awards to patients or other claimants; • inability to commercialize our product candidates; • product recalls, withdrawals or labeling, marketing or promotional restrictions; • decreased demand for our product candidates, if approved for commercial sale; and • loss of revenue.Our insurance policies are expensive and protect us only from some business risks, which leaves us exposed to significant uninsured liabilities.We do not carry insurance for all categories of risk that our business may encounter. Some of the policies we currently maintain includegeneral liability, clinical trial liability, employment practices liability, property, auto, workers’ compensation, umbrella, cyber and directors’ andofficers’ insurance.Any additional product liability insurance coverage we acquire in the future, may not be sufficient to reimburse us for any expenses orlosses we may suffer. Moreover, insurance coverage is becoming increasingly expensive and in the future we may not be able to maintain insurancecoverage at a reasonable cost or in sufficient amounts to protect us against losses due to liability. If we obtain marketing approval for our productcandidates, we intend to acquire insurance coverage to include the sale of commercial products; however, we may be unable to obtain product liabilityinsurance on commercially reasonable terms or in adequate amounts. A successful product liability claim or series of claims brought against us couldcause our share price to decline and, if judgments exceed our insurance coverage, could adversely affect our results of operations and business,including preventing or limiting the commercialization of any product candidates we develop. We do not carry specific biological or hazardous wasteinsurance coverage, and our property, casualty and general liability insurance policies specifically exclude coverage for damages and fines arisingfrom biological or hazardous waste exposure or contamination. Accordingly, in the event of contamination or injury, we could be held liable fordamages or be penalized with fines in an amount exceeding our resources, and our clinical trials or regulatory approvals could be suspended.We also expect that continuing to operate as a public company will make it more difficult and more expensive for us to obtain director andofficer liability insurance, and we may be required to accept reduced policy limits and coverage or incur substantially higher costs to obtain the sameor similar coverage. As a result, it may be more difficult for us to attract and retain qualified people to serve on our board of directors, our boardcommittees or as executive officers. We do not know, however, if we will be able to maintain existing insurance with adequate levels of coverage. Anysignificant uninsured liability may require us to pay substantial amounts, which would adversely affect our cash position and results of operations.Our employees and independent contractors, including consultants, vendors, and any third parties we may engage in connection with developmentand commercialization may engage in misconduct or other improper activities, including noncompliance with regulatory standards andrequirements, which could harm our business.Misconduct by our employees and independent contractors, including consultants, vendors, and any third parties we may engage inconnection with development and commercialization, could include intentional, 109Table of Contentsreckless or negligent conduct or unauthorized activities that violate: (i) the laws and regulations of the FDA, EMA and other similar regulatoryauthorities, including those laws that require the reporting of true, complete and accurate information to such authorities; (ii) manufacturing standards;(iii) data privacy, security, fraud and abuse and other healthcare laws and regulations; or (iv) laws that require the reporting of true, complete andaccurate financial information and data. Specifically, sales, marketing and business arrangements in the healthcare industry are subject to extensivelaws and regulations intended to prevent fraud, misconduct, kickbacks, self-dealing and other abusive practices. These laws and regulations mayrestrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other businessarrangements. Activities subject to these laws could also involve the improper use or misrepresentation of information obtained in the course ofclinical trials, creation of fraudulent data in preclinical studies or clinical trials or illegal misappropriation of drug product, which could result inregulatory sanctions and cause serious harm to our reputation. It is not always possible to identify and deter misconduct by employees and other thirdparties, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or inprotecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with such laws or regulations.Additionally, we are subject to the risk that a person or government could allege such fraud or other misconduct, even if none occurred. If any suchactions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impacton our business and results of operations, including the imposition of significant civil, criminal and administrative penalties, damages, monetary fines,disgorgements, possible exclusion from participation in Medicare, Medicaid, other U.S. federal healthcare programs or healthcare programs in otherjurisdictions, integrity oversight and reporting obligations to resolve allegations of non-compliance, individual imprisonment, other sanctions,contractual damages, reputational harm, diminished profits and future earnings, and curtailment of our operations.Our business and operations would suffer in the event of system failures.Our computer systems, as well as those of our contractors and consultants, are vulnerable to damage from computer viruses, unauthorizedaccess, natural disasters (including hurricanes), terrorism, war and telecommunication and electrical failures. If such an event were to occur and causeinterruptions in our operations, it could result in a material disruption of our product candidate development programs. For example, the loss ofpreclinical study or clinical trial data from completed, ongoing or planned trials could result in delays in our regulatory approval efforts andsignificantly increase our costs to recover or reproduce the data. To the extent that any disruption or security breach were to result in a loss of ordamage to our data or applications, or inappropriate disclosure of personal, confidential or proprietary information, we could incur liability and thefurther development of our product candidates could be delayed.In the ordinary course of our business, we collect and store sensitive data, including intellectual property, clinical trial data, proprietarybusiness information, personal data and personally identifiable information of our clinical trial subjects and employees, in our data centers and on ournetworks. The secure processing, maintenance and transmission of this information is critical to our operations. Despite our security measures, ourinformation technology and infrastructure may be vulnerable to attacks by hackers or internal bad actors, or breached due to employee error, atechnical vulnerability, malfeasance or other disruptions. Although, to our knowledge, we have not experienced any such material security breach todate, any such breach could compromise our networks and the information stored there could be accessed, publicly disclosed, lost or stolen. Any suchaccess, disclosure or other loss of information could result in legal claims or proceedings, liability under laws that protect the privacy of personalinformation, significant regulatory penalties, and such an event could disrupt our operations, damage our reputation, and cause a loss of confidence inus and our ability to conduct clinical trials, which could adversely affect our reputation and delay clinical development of our product candidates. 110Table of ContentsWe may engage in acquisitions that could disrupt our business, cause dilution to our shareholders or reduce our financial resources.In the future, we may enter into transactions to acquire other businesses, products or technologies. If we do identify suitable candidates, wemay not be able to make such acquisitions on favorable terms, or at all. Any acquisitions we make may not strengthen our competitive position, andthese transactions may be viewed negatively by customers or investors. We may decide to incur debt in connection with an acquisition or issue ourordinary shares or other equity securities to the shareholders of the acquired company, which would reduce the percentage ownership of our existingshareholders. We could incur losses resulting from undiscovered liabilities of the acquired business that are not covered by the indemnification we mayobtain from the seller. In addition, we may not be able to successfully integrate the acquired personnel, technologies and operations into our existingbusiness in an effective, timely and nondisruptive manner. Acquisitions may also divert management attention from day-to-day responsibilities,increase our expenses and reduce our cash available for operations and other uses. We cannot predict the number, timing or size of future acquisitionsor the effect that any such transactions might have on our operating results.The United Kingdom’s withdrawal from the European Union may have a negative effect on global economic conditions, financial markets and ourbusiness, which could reduce the price of our shares.Following the vote of a majority of the eligible members of the electorate in the United Kingdom to withdraw from the European Union ina national referendum held on June 23, 2016, the U.K. government served notice under Article 50 of the Treaty of the European Union on March 29,2017 to formally initiate a withdrawal process. The withdrawal agreement and political declaration that were endorsed at a special meeting of theEuropean Council on November 25, 2018 did not receive the approval of the UK Parliament in January and March 2019. Further discussions areongoing, although the European Commission has stated that the EU will not reopen the withdrawal agreement. The United Kingdom and the EuropeanUnion have a two-year period under Article 50 to negotiate the terms for withdrawal. Any extension of the negotiation period for withdrawal willrequire the consent of all of the remaining 27 EU member states.The referendum and anticipation of withdrawal have created significant uncertainty about the future relationship between the UnitedKingdom and the European Union. Lack of clarity about future U.K. laws and regulations as the United Kingdom determines which EU-derived lawsand regulations to replace or replicate as part of a withdrawal, including healthcare and pharmaceutical regulations; financial laws and regulations; taxand free trade agreements; intellectual property rights; supply chain logistics; environmental, health, and safety laws and regulations; immigrationlaws; and employment laws, could decrease foreign direct investment in the United Kingdom, increase costs, depress economic activity, and restrict ouraccess to capital. If the United Kingdom and the European Union are unable to negotiate mutually acceptable withdrawal terms or if other EU memberstates pursue withdrawal, barrier-free access between the U.K. and other EU member states or among the European economic area overall could bediminished or eliminated. These developments, or the perception that any of them could occur, have had and may continue to have a significantadverse effect on global economic conditions and the stability of global financial markets, and could significantly reduce global market liquidity andrestrict the ability of key market participants to operate in certain financial markets. Asset valuations, currency exchange rates, and credit ratings maybe especially subject to increased market volatility. In addition, changes to U.K. border and immigration policy could occur as a result of the UnitedKingdom’s withdrawal from the European Union, affecting our ability to recruit and retain employees from outside the United Kingdom. Any of thesefactors could have an adverse effect on our business, financial condition, results of operations, and prospects.Further, the vote for the United Kingdom’s withdrawal from the European Union has resulted in a decision to move the EMA from theUnited Kingdom to the Netherlands, with operations currently scheduled to begin in the Netherlands by end of March 2019. This transition has caused,and may continue to cause, disruption in the administrative and medical scientific links between the EMA and the UK Medicines and Healthcareproducts Regulatory Agency, or the MHRA, including delays in granting clinical trial authorization or marketing 111Table of Contentsauthorization, disruption of importation and export of active substance and other components of new drug formulations, and disruption of the supplychain for clinical trial product and final authorized formulations. The cumulative effects of the disruption to the regulatory framework may addconsiderably to the development lead time to marketing authorization and commercialization of products in the European Union and/or the UnitedKingdom.Exchange rate fluctuations may adversely affect our results of operations and financial condition.Owing to the international scope of our operations, fluctuations in exchange rates, particularly between the pound sterling and the U.S.dollar, may adversely affect us. Although some of our operations are based in the United Kingdom, we source research and development,manufacturing, consulting and other services from the United States and the European Union. Further, potential future revenue may be derived fromabroad, particularly from the United States. As a result, our business and the market price of our securities may be affected by fluctuations in foreignexchange rates not only between the pound sterling and the U.S. dollar, but also the euro, which may have a significant impact on our results ofoperations and cash flows from period to period. Currently, we do not have any exchange rate hedging arrangements in place.Risks Related to Our Ordinary SharesThe market price of our ordinary shares may be volatile and fluctuate substantially, which could result in substantial losses for purchasers of ourordinary shares.Our share price is likely to be volatile. The stock market in general and the market for smaller biopharmaceutical companies in particularhave experienced extreme volatility that has often been unrelated to the operating performance of particular companies. As a result of this volatility,you may not be able to sell your ordinary shares at or above your purchase price. The market price for our ordinary shares may be influenced by manyfactors, including: • the success of competitive products or technologies; • actual or expected changes in our growth rate relative to our competitors; • results of clinical trials of our product candidates or those of our competitors; • developments related to our existing or any future collaborations; • regulatory or legal developments in the United States and other countries; • development of new product candidates that may address our markets and make our product candidates less attractive; • changes in physician, hospital or healthcare provider practices that may make our product candidates less useful; • announcements by us, our partners or our competitors of significant acquisitions, strategic partnerships, joint ventures,collaborations or capital commitments; • developments or disputes concerning patent applications, issued patents or other proprietary rights; • the recruitment or departure of key personnel; • the level of expenses related to any of our product candidates or clinical development programs; • failure to meet or exceed financial estimates and projections of the investment community or that we provide to the public; • the results of our efforts to discover, develop, acquire or in-license additional product candidates or products; 112Table of Contents • actual or expected changes in estimates as to financial results, development timelines or recommendations by securities analysts; • variations in our financial results or those of companies that are perceived to be similar to us; • changes in the structure of healthcare payment systems; • market conditions in the pharmaceutical and biotechnology sectors; • general economic, industry and market conditions; • changes in accounting principles; and • the other factors described in this “Item 1A. Risk Factors” section and elsewhere in this Form 10-K.In addition, the stock market in general, and Nasdaq and biopharmaceutical companies in particular, have experienced extreme price andvolume fluctuations that have often been unrelated or disproportionate to the operating performance of these companies. In the past, when the marketprice of a security has been volatile, holders of that security have sometimes instituted securities class action litigation against the issuer. If any of theholders of our ordinary shares were to bring such a lawsuit against us, we could incur substantial costs defending the lawsuit and the attention of oursenior management would be diverted from the operation of our business. Any adverse determination in litigation could also subject us to significantliabilities. Broad market and industry factors may negatively affect the market price of our ordinary shares, regardless of our actual operatingperformance. Further, a decline in the financial markets and related factors beyond our control may cause the price of our ordinary shares to declinerapidly and unexpectedly. If the market price of our ordinary shares does not exceed the your purchase price, you may not realize any return on yourinvestment in us and may lose some or all of your investment.Our executive officers, directors and principal shareholders, if they choose to act together, have the ability to control or significantly influence allmatters submitted to shareholders for approval.As of December 31, 2018, our executive officers, directors and shareholders who owned more than 5% of our outstanding ordinary sharesand their respective affiliates, in the aggregate, hold ordinary shares representing approximately60.2% of our outstanding ordinary shares.As a result, if these shareholders choose to act together, they would be able to control or significantly influence all matters submitted to ourshareholders for approval, as well as our management and affairs. For example, these persons, if they choose to act together, would control orsignificantly influence the election of directors, the composition of our management and approval of any merger, consolidation, sale of all orsubstantially all of our assets or other business combination that other shareholders may desire. Any of these actions could adversely affect the marketprice of our ordinary shares.A significant portion of our total outstanding shares are eligible to be sold into the market, which could cause the market price of our ordinaryshares to drop significantly, even if our business is doing well.Sales of a substantial number of our ordinary shares in the public market, or the perception in the market that the holders of a large numberof shares intend to sell shares, could reduce the market price of our ordinary shares.All lock-up agreements entered into in connection with our initial public offering expired on December 5, 2018. Subject to any applicablelockup agreement described below, our outstanding ordinary shares may be freely sold in the public market at any time to the extent permitted byRules 144 and 701 under the Securities Act, or to the extent that such shares have already been registered under the Securities Act and are held bynon-affiliates of ours. Moreover, certain holders of ordinary shares have rights, subject to specified 113Table of Contentsconditions, to require us to file registration statements covering their shares or to include their ordinary shares in registration statements that we mayfile for ourselves or other shareholders, until such shares can otherwise be sold without restriction under Rule 144 or until the rights terminate pursuantto the terms of the shareholders agreement between us and such holders. We also have registered all ordinary shares that we may issue under our equitycompensation plans or that are issuable upon exercise of outstanding options. Upon issuance, these ordinary shares can be freely sold in the publicmarket, subject to volume limitations applicable to affiliates and any applicable lock-up agreements. Furthermore, we and our executive officers,directors and certain of our shareholders have agreed with the underwriters that, subject to certain exceptions, we and they will not directly orindirectly sell or otherwise transfer their ordinary shares for a period of 90 days after the completion of the offering.Any sales of securities by these shareholders could have a negative impact on the trading price of our ordinary shares.We are an “emerging growth company” and a “smaller reporting company,” and the reduced disclosure requirements applicable to emerginggrowth companies and smaller reporting companies may make our ordinary shares less attractive to investors.We are an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act of 2012 (“JOBS Act”), and may remain anemerging growth company until the last day of the fiscal year following the fifth anniversary of our IPO. However, if certain events occur prior to theend of such five-year period, including if we become a “large accelerated filer,” our annual gross revenues exceed $1.07 billion or we issue more than$1.0 billion of non-convertible debt in any three-year period, we will cease to be an emerging growth company prior to the end of such five-yearperiod. For so long as we remain an emerging growth company, we are permitted and intend to rely on exemptions from certain disclosure requirementsthat are applicable to other public companies that are not emerging growth companies. These exemptions include: • being permitted to provide only two years of audited financial statements, in addition to any required unaudited interim financialstatements, with correspondingly reduced “Management’s Discussion and Analysis of Financial Condition and Results ofOperations” disclosure in this Form 10-K; • not being required to comply with the auditor attestation requirements in the assessment of our internal control over financialreporting; • not being required to comply with any requirement that may be adopted by the Public Company Accounting Oversight Boardregarding mandatory audit firm rotation or a supplement to the auditor’s report providing additional information about the auditand the financial statements; • reduced disclosure obligations regarding executive compensation; and • exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and shareholder approval ofany golden parachute payments not previously approved.In addition, the JOBS Act provides that an emerging growth company can take advantage of an extended transition period for complyingwith new or revised accounting standards. This allows an emerging growth company to delay the adoption of these accounting standards until theywould otherwise apply to private companies. We have elected to take advantage of this extended transition period.We are also a smaller reporting company, and we will remain a smaller reporting company until the fiscal year following the determinationthat our voting and non-voting common shares held by non-affiliates is more than $250 million measured on the last business day of our second fiscalquarter, or our annual revenues are more than $100 million during the most recently completed fiscal year and our voting and non-voting common 114Table of Contentsshares held by non-affiliates is more than $700 million measured on the last business day of our second fiscal quarter. Similar to emerging growthcompanies, smaller reporting companies are able to provide simplified executive compensation disclosure, are exempt from the auditor attestationrequirements of Section 404, and have certain other reduced disclosure obligations, including, among other things, being required to provide only twoyears of audited financial statements and not being required to provide selected financial data, supplemental financial information or risk factors.We may choose to take advantage of some, but not all, of the available exemptions for emerging growth companies and smaller reportingcompanies. We cannot predict whether investors will find our ordinary shares less attractive if we rely on these exemptions. If some investors find ourordinary shares less attractive as a result, there may be a less active trading market for our ordinary shares and our shares price may be more volatile.We will continue to incur increased costs as a result of operating as a public company, and our management will be required to devote substantialtime to new compliance initiatives and corporate governance practices.As a public company, and particularly after we are no longer an emerging growth company and smaller reporting company, we willcontinue to incur significant legal, accounting and other expenses that we did not incur as a private company. The Sarbanes-Oxley Act of 2002, theDodd-Frank Wall Street Reform and Consumer Protection Act, The Nasdaq Global Select listing requirements and other applicable securities rules andregulations impose various requirements on public companies, including establishment and maintenance of effective disclosure and financial controlsand corporate governance practices. Our management and other personnel need to devote a substantial amount of time to these compliance initiatives.Moreover, these rules and regulations increase our legal and financial compliance costs and make some activities more time consuming and costly. Forexample, we expect that these rules and regulations may make it more difficult and more expensive for us to obtain director and officer liabilityinsurance, which in turn could make it more difficult for us to attract and retain qualified members of our board of directors.We are evaluating these rules and regulations, and cannot predict or estimate the amount of additional costs we may incur or the timing ofsuch costs. These rules and regulations are often subject to varying interpretations, in many cases due to their lack of specificity, and, as a result, theirapplication in practice may evolve over time as new guidance is provided by regulatory and governing bodies. This could result in continuinguncertainty regarding compliance matters and higher costs necessitated by ongoing revisions to disclosure and governance practices.Pursuant to Section 404 of the Sarbanes-Oxley Act of 2002, or Section 404, we are required to furnish a report by our management on ourinternal control over financial reporting. However, while we remain an emerging growth company, we will not be required to include an attestationreport on internal control over financial reporting issued by our independent registered public accounting firm. To achieve compliance withSection 404 within the prescribed period, we will be engaged in a process to document and evaluate our internal control over financial reporting,which is both costly and challenging. In this regard, we will need to continue to dedicate internal resources, potentially engage outside consultants,adopt a detailed work plan to assess and document the adequacy of internal control over financial reporting, continue steps to improve controlprocesses as appropriate, validate through testing whether such controls are functioning as documented, and implement a continuous reporting andimprovement process for internal control over financial reporting. Despite our efforts, there is a risk that we will not be able to conclude, within theprescribed timeframe or at all, that our internal control over financial reporting is effective as required by Section 404. If we identify one or morematerial weaknesses, it could result in an adverse reaction in the financial markets due to a loss of confidence in the reliability of our financialstatements. 115Table of ContentsIf securities or industry analysts cease to publish research or reports about our business, or if they issue an adverse or misleading opinion regardingour ordinary shares, our share price and trading volume could decline.The trading market for our ordinary shares relies in part on the research and reports that industry or securities analysts publish about us orour business. We do not control these analysts. Furthermore, if any of the analysts who cover us issue an adverse or misleading opinion regarding us,our business model, our intellectual property or our share performance, or if any of our preclinical studies or clinical trials and operating results fail tomeet the expectations of analysts, our share price would likely decline. If one or more of these analysts ceases coverage of us or fails to publish reportson us regularly, we could lose visibility in the financial markets, which in turn could cause our share price or trading volume to decline.Anti-takeover provisions in our organizational documents and Cayman Islands law may discourage or prevent a change of control, even if anacquisition would be beneficial to our shareholders, which could depress the price of our ordinary shares and prevent attempts by our shareholdersto replace or remove our current management.Our memorandum and articles of association contain provisions that may discourage unsolicited takeover proposals that shareholders mayconsider to be in their best interests. Our board of directors is divided into three classes with staggered, three-year terms. Our board of directors has theability to designate the terms of and issue preferred shares without shareholder approval. We are also subject to certain provisions under CaymanIslands law that could delay or prevent a change of control. Together these provisions may make more difficult the removal of management and maydiscourage transactions that otherwise could involve payment of a premium over prevailing market prices for our ordinary shares.There may be difficulties in enforcing foreign judgments against our management or us.Certain of our directors and management reside outside the United States. A significant portion of our assets and such persons’ assets arelocated outside the United States. As a result, it may be difficult or impossible for investors to effect service of process upon us within the United Statesor other jurisdictions, including judgments predicated upon the civil liability provisions of the federal securities laws of the United States.In particular, investors should be aware that there is uncertainty as to whether the courts of the Cayman Islands or any other applicablejurisdictions would recognize and enforce judgments of U.S. courts obtained against us or our directors or management as well as against the sellingshareholders predicated upon the civil liability provisions of the securities laws of the United States or any state in the United States or entertainoriginal actions brought in the Cayman Islands or any other applicable jurisdictions courts against us or our directors or officers as well as against theselling shareholders predicated upon the securities laws of the United States or any state in the United States.The rights of our shareholders differ from the rights typically offered to shareholders of a U.S. corporation.Our corporate affairs and the rights of holders of ordinary shares are governed by Cayman Islands law, including the provisions of theCayman Islands Companies Law (2018 Revision), or the Companies Law, the common law of the Cayman Islands and by our memorandum and articlesof association. We are also subject to the federal securities laws of the United States. The rights of shareholders to take action against the directors,actions by minority shareholders and the fiduciary responsibilities of our directors to us under Cayman Islands law are to a large extent governed bythe common law of the Cayman Islands. The common law of the Cayman Islands is derived in part from comparatively limited judicial precedent in theCayman Islands as well as from English common law, the decisions of whose courts are of persuasive authority, but are not binding on a court in theCayman Islands. The rights of our shareholders and the fiduciary responsibilities of our directors under Cayman Islands law are different from what theywould be under statutes or judicial precedent in some 116Table of Contentsjurisdictions in the United States. In particular, the Cayman Islands has a different body of securities laws as compared to the United States, and certainstates, such as Delaware, may have more fully developed and judicially interpreted bodies of corporate law. In addition, Cayman Islands companiesmay not have standing to initiate a shareholders derivative action in a Federal court of the United States.As a result of all of the above, public shareholders may have more difficulty in protecting their interests in the face of actions taken bymanagement, members of the board of directors or controlling shareholders than they would as public shareholders of a United States company.If we fail to maintain an effective system of internal control over financial reporting, we may not be able to accurately report our financial results orprevent fraud. As a result, shareholders could lose confidence in our financial and other public reporting, which would harm our business and thetrading price of our ordinary shares.Section 404(a) of the Sarbanes-Oxley Act, or Section 404(a), requires that beginning with our second annual report following our IPO onJune 7, 2018, management assess and report annually on the effectiveness of our internal control over financial reporting and identify any materialweaknesses in our internal control over financial reporting. Although Section 404(b) of the Sarbanes-Oxley Act, or Section 404(b), requires ourindependent registered public accounting firm to issue an annual report that addresses the effectiveness of our internal control over financial reporting,we have opted to rely on the exemptions provided in the JOBS Act, and consequently will not be required to comply with SEC rules that implementSection 404(b) until such time as we are no longer an “emerging growth company.”We expect our first Section 404(a) assessment will take place for our annual report for the fiscal year ending December 31, 2019. Effectiveinternal controls over financial reporting are necessary for us to provide reliable financial reports and, together with adequate disclosure controls andprocedures, are designed to prevent fraud. Any failure to implement required new or improved controls, or difficulties encountered in theirimplementation could cause us to fail to meet our reporting obligations. In addition, any testing by us conducted in connection with Section 404, orany subsequent testing by our independent registered public accounting firm, may reveal deficiencies in our internal controls over financial reportingthat are deemed to be material weaknesses or that may require prospective or retroactive changes to our financial statements or identify other areas forfurther attention or improvement. Inadequate internal controls could also cause investors to lose confidence in our reported financial information,which could have a negative effect on the trading price of our ordinary shares.Because we do not anticipate paying any cash dividends on our ordinary shares in the foreseeable future, capital appreciation, if any, would be yoursole source of gain.Under Cayman Islands law, we may only make distributions by way of dividend out of profits, or out of our share premium account(provided that immediately following the date that the dividend is proposed to be paid we are able to pay our debts as they fall due in the ordinarycourse of business). We have never declared or paid any cash dividends on our ordinary shares. We currently anticipate that we will retain futureearnings for the development, operation and expansion of our business and do not anticipate declaring or paying any cash dividends for theforeseeable future. As a result, capital appreciation, if any, of our ordinary shares would be your sole source of gain on an investment in our ordinaryshares for the foreseeable future. See the “Dividend Policy” section of this Form 10-K for additional information.We could be subject to securities class action litigation.In the past, securities class action litigation has often been brought against a company following a decline in the market price of itssecurities. This risk is especially relevant for us because biopharmaceutical companies have experienced significant stock price volatility in recentyears. If we face such litigation, it could result in substantial costs and a diversion of management’s attention and resources, which could harm ourbusiness. 117Table of ContentsWe expect to be treated as resident in the United Kingdom for tax purposes, but may be treated as a dual resident company for United Kingdom taxpurposes.Our board of directors conducts our affairs so that the central management and control of the company is exercised in the United Kingdom.As a result, we expect to be treated as resident in the United Kingdom for UK tax purposes. Accordingly, we expect to be subject to UK taxation on ourincome and gains, except where an exemption applies.However, we may be treated as a dual resident company for UK tax purposes. As a result, our right to claim certain reliefs from UK tax maybe restricted, and changes in law or practice in the United Kingdom could result in the imposition of further restrictions on our right to claim UK taxreliefs.We may be classified as a passive foreign investment company for U.S. federal income tax purposes, which could result in adverse U.S. federalincome tax consequences to U.S. investors in our ordinary shares.Based on the current and anticipated value of our assets, including goodwill, and the composition of our income, assets and operations, wedo not believe we were a “passive foreign investment company,” or PFIC, for the taxable year ending on December 31, 2018, and do not expect to be aPFIC for the current taxable year. However, the application of the PFIC rules is subject to uncertainty in several respects, and we cannot assure you thatthe U.S. Internal Revenue Service, or the IRS, will not take a contrary position. Furthermore, a separate determination must be made after the close ofeach taxable year as to whether we are a PFIC for that year. Accordingly, we cannot assure you that we were not a PFIC for our taxable year ending onDecember 31, 2018 and that we will not be a PFIC for our current taxable year or any future taxable year. A non-U.S. company will be considered aPFIC for any taxable year if (i) at least 75% of its gross income is passive income (including interest income), or (ii) at least 50% of the value of itsassets (based on an average of the quarterly values of the assets during a taxable year) is attributable to assets that produce or are held for theproduction of passive income. If we were to be classified as a PFIC for any taxable year during which a U.S. Holder (as defined below under “MaterialU.S. Federal Income Tax Consequences”) holds our ordinary shares, certain adverse U.S. federal income tax consequences could apply to such U.S.Holder, including (i) the treatment of all or a portion of any gain on disposition of our ordinary shares as ordinary income, (ii) the application of adeferred interest charge on such gain and the receipt of certain dividends and (iii) the obligation to comply with certain reporting requirements.If a United States person is treated as owning at least 10% of our ordinary shares, such holder may be subject to adverse U.S. federal income taxconsequences.If a U.S. Holder is treated as owning (directly, indirectly or constructively) at least 10% of the value or voting power of our ordinary shares,such U.S. Holder may be treated as a “United States shareholder” with respect to each “controlled foreign corporation” in our group (if any). If ourgroup includes one or more U.S. subsidiaries, certain of our non-U.S. subsidiaries could be treated as controlled foreign corporations (regardless ofwhether we are treated as a controlled foreign corporation). A United States shareholder of a controlled foreign corporation may be required to reportannually and include in its U.S. taxable income its pro rata share of “Subpart F income,” “global intangible low-taxed income” and investments in U.S.property by controlled foreign corporations, regardless of whether we make any distributions. An individual that is a United States shareholder withrespect to a controlled foreign corporation generally would not be allowed certain tax deductions or foreign tax credits that would be allowed to aUnited States shareholder that is a U.S. corporation. Failure to comply with these reporting obligations may subject you to significant monetarypenalties and may prevent the statute of limitations from starting with respect to your U.S. federal income tax return for the year for which reporting wasdue. We cannot provide any assurances that we will assist investors in determining whether any of our non-U.S. subsidiaries is treated as a controlledforeign corporation or whether such investor is treated as a United States shareholder with respect to any of such controlled foreign corporations.Further, we cannot provide any assurances that we will furnish to any United States shareholders information that may be necessary 118Table of Contentsto comply with the aforementioned reporting and tax payment obligations. U.S. Holders should consult their tax advisors regarding the potentialapplication of these rules to their investment in our ordinary shares.Comprehensive tax reform legislation could adversely affect our business and financial condition.The U.S. government has enacted comprehensive tax legislation that includes significant changes to the taxation of business entities,referenced herein as the Tax Reform Act. These changes include, among others, a permanent reduction to the corporate income tax rate, limitinginterest deductions and the use of net operating losses, adopting elements of a territorial tax system and introducing certain anti-base erosionprovisions. We continue to examine the impact this tax reform legislation may have on our business. The effect of the Tax Reform Act on our business,whether adverse or favorable, is uncertain, and may not become evident for some period of time. U.S. Holders should consult their legal and taxadvisors regarding any such legislation and the potential tax consequences of investing in our ordinary shares.Changes in tax laws or challenges to our tax position could adversely affect our results of operations and financial condition.We are subject to complex tax laws. Changes in tax laws, regulations and treaties, or the interpretation thereof, tax policy initiatives andreforms under consideration and the practices of tax authorities in jurisdictions in which we operate could adversely affect our tax position, includingour effective tax rate or tax payments.In October 2015, the Organization for Economic Co-Operation and Development released a final package of measures to be implementedby member nations in response to a 2013 action plan calling for a coordinated multi-jurisdictional approach to “base erosion and profit shifting” bymultinational companies. Multiple member jurisdictions, including the countries in which we operate, have begun implementing recommendedchanges such as country-by-country reporting requirements and changes to double tax treaties. Additional multilateral changes are anticipated inupcoming years. We often rely on generally available interpretations of applicable tax laws, treaties and regulations. There cannot be certainty that therelevant tax authorities are in agreement with our interpretation of these laws, regulations or treaties, or with tax positions that we have taken. If ourinterpretation or tax position is challenged by the relevant tax authorities, we could be required to pay taxes that we currently do not collect or pay,may be subject to interest and penalties and there could be an increase to the costs of our services to track and collect such taxes, which could increaseour costs of operations or our effective tax rate. Similarly, a tax authority could assert that we are subject to tax in a jurisdiction where we believe wehave not established a taxable connection, often referred to as a “permanent establishment” under international tax treaties, and such an assertion, ifsuccessful, could increase our expected tax liability in one or more jurisdictions. The occurrence of any of the foregoing tax risks could have a materialadverse effect on our business, financial condition and results of operations.We are unable to predict what national or international tax reform may be proposed or enacted in the future or what effect such changeswould have on our business, but such changes, to the extent they are brought into tax legislation, regulations, policies or practices, could impact thetax treatment of our earnings, adversely affect our profitability and increase the complexity, burden and cost of tax compliance.We have significant net operating losses, or NOLs, and U.K. carryforward tax losses which we may not be able to realize or which may be restrictedfollowing the Reorganization Transactions or any future change of control. We also benefit from certain tax incentive regimes, such as research anddevelopment tax credits, in the jurisdictions in which we operate and any adverse change to these regimes, the application thereof or challenges tothe tax position we have adopted under these regimes could adversely affect our results of operations and financial condition.As of December 31, 2018, we had federal and state NOL carryforwards in the United States of $14.2 million and $14.2 million, respectively,and cumulative carryforward tax losses in the United Kingdom of 119Table of Contents$94.1 million, which we expect to be available to reduce future taxable income subject to any relevant restrictions (including those in the UK that limitthe percentage of profits that can be reduced by carried forward losses). The U.S. federal and state NOL carryforwards incurred prior to January 1, 2018in the amount of approximately $6.8 million and $6.7 million will begin to expire in 2036. The U.K. carryforward tax losses will continue indefinitely,subject to relevant restrictions, under current UK legislation. Under the Tax Cuts and Jobs Act of 2017, U.S. federal NOL carryforwards generated afterDecember 31, 2017 are not subject to expiration but such NOLs may only offset 80% of taxable income. As of December 31, 2018, we also had orphandrug and research and development credits in the U.S. in the amount of $1.1 million.The NOL carryforwards and U.K. carryforward tax losses are subject to review and possible adjustment by the U.S., U.K. and state taxauthorities. NOL carryforwards and U.K. carryforward tax losses may become subject to limitations in the event of certain cumulative changes in theownership interest of significant shareholders, as defined under Sections 382 Internal Revenue Code, as well as the Corporation Tax Act 2010 Part 14under the UK tax rules. This could limit the amount of NOLs or carryforward tax losses that we can utilize annually to offset future taxable income ortax liabilities. We have conducted a review of changes in the ownership interest of significant shareholders and determined that as of December 31,2018, there were no limitations in the U.K. However, for U.S. purposes, we have determined that a change of ownership occurred in April 2016. We arestill in the process of determining the annual limitation on losses that occurred prior to April 2016. Subsequent ownership changes and changes to theUK (or US) tax rules in respect of the utilization of losses carried forward may further affect the limitation in future years.Additionally, we have not undertaken a study on the completeness of the U.S. research and development and orphan drug credits. As such,the U.S. research and development and orphan drug credits may change and may be subject to review and adjustment by the tax authorities. ITEM 1B.UNRESOLVED STAFF COMMENTSNot applicable. ITEM 2.PROPERTIESOur principal office is located at 430 East 29th Street, 10th Floor, New York, New York 10016, USA, where we lease 5,887 square feet ofoffice space. We lease this office space under a lease that terminates on December 31, 2021.We also own a long leasehold interest in the ground rights where our 29,000 square foot manufacturing facility is located, at 92 BritanniaWalk, London N1 7NQ, United Kingdom. The long leasehold interest is for 125 years, expiring in 2126, and there is no rent payable thereunder. ITEM 3.LEGAL PROCEEDINGSWe are not subject to any material legal proceedings. ITEM 4.MINE SAFETY DISCLOSURESNot applicable. 120Table of ContentsPART IIITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OFSECURITIESMarket InformationOn June 8, 2018, our ordinary shares began trading on the Nasdaq Global Market under the symbol “MGTX.” Prior to that time, there wasno public market for our stock.Holders of RecordAs of March 15, 2019, there were 75 holders of record. The actual number of shareholders of our ordinary shares is greater than this numberof record holders and includes shareholders who are beneficial owners but whose ordinary shares are held in street name by brokers and other nominees.This number of holders of record also does not include shareholders whose ordinary shares may be held in trust by other entities.Dividend PolicyWe have never declared or paid any cash dividends on our ordinary shares. We intend to retain future earnings, if any, to finance theoperation and expansion of our business and do not anticipate paying any cash dividends in the foreseeable future. However, if we do pay a cashdividend on our ordinary shares in the future, we will only pay such dividend out of our profits or share premium (subject to solvency requirements) aspermitted under Cayman Islands law.Recent Sales of Unregistered SecuritiesOn March 1, 2019, we consummated a private placement with various investors, including one of our existing shareholders, Perceptive LifeSciences Master Fund Ltd., pursuant to which we issued and sold an aggregate of 5,797,102 of our ordinary shares at a purchase price of $13.80 pershare. Evercore Group L.L.C. and Chardan Capital Markets LLC served as co-lead placement agents for the private placement and received anaggregate underwriting commission of approximately $2.4 million. The financing was led by Johnson & Johnson Innovation – JJDC, Inc. (“JJDC”), theinvestment arm of Johnson & Johnson, which made a $40.0 million equity investment in the Company and received 2,898,550 ordinary shares. Each ofthe foregoing securities issuances were in reliance on the exemption contained in Section 4(a)(2) of the Securities Act, as transactions by issuers notinvolving a public offering.In connection with our amendment and restatement of our license agreements with UCLB whereby we agreed to issue UCLB £1.5 millionof the Company’s ordinary shares, on March 21, 2019, we issued 158,832 of the Company’s ordinary shares to UCLB. This securities issuance was inreliance on the exemption contained in Section 4(a)(2) of the Securities Act, as a transaction by issuers not involving a public offering.Use of ProceedsOn June 12, 2018, we completed our IPO and issued and sold 5,000,000 ordinary shares at a price to the public of $15.00 per sharereceiving $65.2 million in proceeds after deducting underwriting discounts and commissions and offering expenses payable by us. There has been nomaterial change in the expected use of the net proceeds from our IPO as described in our prospectus. As of December 31, 2018, we had used$34.1 million of such net proceeds and had total unrestricted cash and cash equivalents of $68.1 million. ITEM 6.SELECTED FINANCIAL DATANot applicable. 121Table of ContentsITEM 7. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONSYou should read the following discussion and analysis of financial condition and operating results our financial statements and therelated notes appearing at the end of this Form 10-K. This discussion contains forward-looking statements that involve risks and uncertainties. As aresult of many factors, such as those set forth in the section of this Form 10-K captioned “Item 1A. Risk Factors” and elsewhere in this Form 10-K, ouractual results may differ materially from those anticipated in these forward-looking statements. For convenience of presentation some of the numbershave been rounded in the text below.OverviewWe are a vertically integrated, clinical stage gene therapy company with five programs in clinical development and a broad pipeline ofpreclinical and research programs. We have core capabilities in viral vector design and optimization, gene therapy manufacturing as well as apotentially transformative gene regulation technology. Led by an experienced management team, we have taken a portfolio approach by licensing,acquiring and developing technologies that give us depth across both product candidates and indications. Though initially focusing on the eye,salivary gland and central nervous system, we intend to expand our focus in the future to develop additional gene therapy treatments for patientssuffering from a range of serious diseases.We are an exempted company incorporated under the laws of the Cayman Islands in 2018, and prior to that, we commenced operations asMeiraGTx Limited, a private limited company incorporated under the laws of England and Wales in 2015. Our discussion of our financial conditionand results of operations is based upon our financial statements, which have been prepared in accordance with generally accepted accountingprinciples in the United States. Since our formation, we have devoted substantially all of our resources to developing our technology platform,establishing our viral vector manufacturing facility and developing manufacturing processes, advancing the product candidates in our ophthalmology,salivary gland and neurodegenerative disease programs, building our intellectual property portfolio, organizing and staffing our company, businessplanning, raising capital, and providing general and administrative support for these operations. In 2016, we completed the acquisition of assets heldby BRI-Alzan, Inc., a Delaware corporation, including a worldwide license agreement to develop certain preclinical technology for the treatment ofALS. In October 2018, we acquired Vector Neurosciences, Inc., a Delaware corporation. In connection with that acquisition, we acquired its AAV-GADgene therapy program which had completed a randomized, sham-controlled Phase 2 study for treatment of Parkinson’s disease. To date, we havefinanced our operations primarily with cash on hand and proceeds from the sales of our Series A ordinary shares, Convertible Preferred C Shares andordinary shares. Through December 31, 2018, we received gross proceeds of approximately $203.7 million from sales of our ordinary shares, Series Aordinary shares and Convertible Preferred C Shares. As of December 31, 2018, we had cash and cash equivalents of $68.1million.We are a clinical stage company and have not generated any product revenues to date. We have five clinical programs and a pipeline ofpreclinical programs. Since inception, we have incurred significant operating losses. Our net losses for the years ended December 31, 2018 and 2017were $82.9 million and $31.1 million, respectively. As of December 31, 2018, we had an accumulated deficit of $148.3 million. While we do notexpect to generate revenue from sales of any products for several years, if at all, in January 2019, we entered into the Collaboration Agreement, whichprovides for Janssen to pay us an $100 million upfront payment and provide us with research funding, and we are eligible to receive potentialmilestone payments and royalties.Our total operating expenses were $78.1 million and $31.7 million for the years ended December 31, 2018 and 2017, respectively. Whilewe expect our operating expenses to increase substantially in connection with our ongoing development activities related to our product candidates,we believe that these increases may be partially offset by the research funding in connection with a collaboration, option and license agreement weentered into in January 2019. We anticipate that our expenses will increase due to costs associated with our 122Table of Contentsclinical development program targeting in achromatopsia due to mutations in the CNGB3 or CNGA3 gene, inherited retinal dystrophy caused bymutations in RPE65, or RPE65-deficiency, and X-Linked retinitis pigmentosa, or XLRP. In addition, we expect to continue incurring increasing costsassociated with our clinical activities for hAQP1 for the treatment of radiation-induced xerostomia and xerostomia associated with Sjogren’s syndrome.We are currently evaluating potential next steps for clinical development of AAV-GAD, which remains pending future discussions with regulatoryagencies. We also expect to incur expenses related to research activities in additional therapeutic areas to expand our pipeline, hiring additionalpersonnel in manufacturing, research, clinical trials, quality and other functional areas, and associated cash and share-based compensation expense, aswell as the further development of internal manufacturing capabilities and capacity and other associated costs including the management of ourintellectual property portfolio.As a result of these anticipated expenditures, we will require additional capital, which we may raise through equity offerings, debtfinancings, marketing and distribution arrangements and other collaborations, strategic alliances and licensing arrangements or other sources to enableus to complete the development and potential commercialization of our product candidates. Furthermore, we expect to continue incurring costsassociated with being a public company. Adequate additional financing may not be available to us on acceptable terms, or at all. Our failure to raisecapital as and when needed would have a negative effect on our financial condition and our ability to pursue our business strategy. In addition,attempting to secure additional financing may divert the time and attention of our management from day-to-day activities and harm our productcandidate development efforts. If we are unable to raise capital when needed or on acceptable terms, we would be forced to delay, reduce or eliminatecertain of our research and development programs.Based on our cash and cash equivalents at December 31, 2018 and after receiving an additional $77.4 million from a private placement ofordinary shares during the first quarter of 2019 and our receipt of the $100 million payment under the terms of the Collaboration Agreement, weestimate that such funds will be sufficient to enable us to fund our operating expenses and capital expenditure requirements into 2022. We have basedthese estimates on assumptions that may prove to be wrong, and we may use our available capital resources sooner than we currently expect. See “—Liquidity and Capital Resources.” Because of the numerous risks and uncertainties associated with the development of our product candidates, anyfuture product candidates, our platform and technology and because the extent to which we may enter into collaborations with third parties fordevelopment of any of our product candidates is unknown, we are unable to estimate the amounts of increased capital outlays and operating expensesassociated with completing the research and development of our product candidates. Our future capital requirements will depend on many factors,including: • the initiation, progress, timing, costs and results of our planned clinical trials for our product candidates; • the outcome, timing and cost of meeting regulatory requirements established by the FDA, EMA and other regulatory authorities; • the cost of filing, prosecuting, defending and enforcing our patent claims and other intellectual property rights; • the cost of defending potential intellectual property disputes, including patent infringement actions brought by third partiesagainst us or any of our product candidates; • the effect of competing technological and market developments; • the costs and timing of further developing our manufacturing facilities in the United Kingdom; • the costs of operating as a public company. • the extent to which we in-license or acquire other products and technologies; 123Table of Contents • the cost of establishing sales, marketing and distribution capabilities for our product candidates in regions where we choose tocommercialize our products; and • the initiation, progress, timing and results of our commercialization of our product candidates, if approved for commercial sale.Adequate additional funds may not be available to us on acceptable terms, or at all. To the extent that we raise additional capital throughthe sale of equity or convertible securities, your ownership interest will be diluted, and the terms of these securities may include liquidation or otherpreferences that adversely affect your rights as a shareholder. Any future debt financing or preferred equity or other financing, if available, may involveagreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capitalexpenditures or declaring dividends and may require the issuance of warrants, which could potentially dilute your ownership interests.If we raise additional funds through collaborations, strategic alliances, or licensing arrangements with third parties, we may have torelinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or grant licenses on terms that may notbe favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce,or terminate our product development programs or any future commercialization efforts or grant rights to develop and market product candidates thatwe would otherwise prefer to develop and market ourselves.Because of the numerous risks and uncertainties associated with drug development, we are unable to predict the timing or amount ofincreased expenses or when or if we will be able to achieve or maintain profitability. Even if we are able to generate revenue from product sales, wemay not become profitable. If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable tocontinue our operations at planned levels and be forced to reduce or terminate our operations.Highlights and Recent DevelopmentOn October 5, 2018, we acquired Vector Neurosciences Inc. (“Vector”) and acquired Vector’s rights to the clinical stage gene therapyproduct candidate adeno-associated virus encoding glutamic acid decarboxylase (“AAV-GAD”), an investigational gene therapy medicine ready forcontinued Phase 2 clinical development for Parkinson’s disease. We issued an aggregate of 225,000 of our ordinary shares as initial mergerconsideration, consisting of 202,500 shares which were issued at the closing of the merger and an additional 22,500 shares to be issued 18 monthsfollowing the closing.On December 14, 2018, we acquired from Moorfields Eye Hospital NHS Foundation Trust of Moorfields Eye Hospital a long leaseholdinterest (the “Head Lease”) in the site of our manufacturing facility, 92 Britannia Walk, London N1 7LU, for a purchase price of approximately$6,615,000. As a result of this transaction, we are now the tenant under the Head Lease, which has a remaining term of 108 years, with no facility rentdue.In January and February, 2019, we amended and restated the following agreements: (i) the License Agreement, dated February 4, 2015, asamended, between Athena Vision Ltd. and UCLB; (ii) the License Agreement, dated July 28, 2017, as amended, between MeiraGTx UK II Limited andUCLB; and (iii) the License Agreement, dated March 15, 2018, among MeiraGTx Limited, MeiraGTx UK II Limited and UCLB to establish new stand-alone license agreements for our inherited retinal disease (“IRD”) programs. In connection with the stand-alone agreement related to CNGB3, weagreed to make an upfront payment to UCLB of £1,500,000 and issue £1,500,000 of our ordinary shares.On January 30, 2019, we entered into a strategic collaboration with Janssen to develop and commercialize gene therapies for the treatmentof inherited retinal diseases (IRDs). This Collaboration 124Table of ContentsAgreement provides for Janssen to pay us an $100 million upfront payment and provide us with research funding, and we are eligible to receivepotential milestone payments and royalties. We will collaborate with Janssen to develop our current clinical programs in Retinitis Pigmentosa and twogenetic forms of Achromatopsia and Janssen has the exclusive right to commercialize these products globally. We will manufacture these products forcommercial supply. Janssen will pay 100% of the clinical and commercialization costs of the products and we are eligible to receive untiered20 percent royalties on net sales of products and additional development and commercialization milestones of up to $340 million. In addition, we willenter a research collaboration with Janssen in the area of IRDs, with Janssen paying for the majority of the research costs. Janssen has the right toexclusively license any product coming out of the collaboration at the time of an IND. Janssen will then pay 100% of the clinical andcommercialization costs for these products and we will receive an untiered royalty in the high teens on net sales as well as development milestones. Inaddition, we have entered into a manufacturing research collaboration with Janssen to further develop processes for manufacturing AAV viral vectorsin which the costs of the research will be shared.On March 1, 2019, we consummated a private placement with various investors, including one of our existing shareholders Perceptive LifeSciences Master Fund Ltd., pursuant to which we issued and sold an aggregate of 5,797,102 ordinary shares for gross proceeds of approximately$80.0 million. The financing was led by JJDC, the investment arm of Johnson & Johnson, which made a $40.0 million equity investment in theCompany and received 2,898,550 ordinary shares.Components of Our Results of OperationsOperating ExpensesOur operating expenses since inception have consisted primarily of general and administrative costs and research and development costs.General and Administrative ExpensesGeneral and administrative expenses consist primarily of salaries and other related costs, including share-based compensation, forpersonnel in our executive, finance, business development and administrative functions. General and administrative expenses also include legal feesrelating to intellectual property and corporate matters; professional fees for accounting, auditing, tax and consulting services; insurance costs; travelexpenses; and office facility-related expenses, which include direct depreciation costs.We expect that our general and administrative expenses will increase in the future as we increase our personnel headcount to supportincreased research and development activities. We have also incurred and expect to continue to incur increased expenses associated with being apublic company, including costs of accounting, audit, legal, regulatory and tax-related services associated with maintaining compliance with Nasdaqand SEC requirements; director and officer insurance costs; and investor and public relations costs.Research and Development ExpensesResearch and development expenses consist primarily of costs incurred for our research activities, including our discovery efforts, and thedevelopment of our product candidates, and include: • employee-related expenses, including salaries, benefits and travel of our research and development personnel; • expenses incurred in connection with third-party vendors that conduct clinical and preclinical studies and manufacture the drugproduct for the clinical trials and preclinical activities; • acquisition of in-process research and development; 125Table of Contents • costs associated with clinical and preclinical activities including costs related to facilities, supplies, rent, insurance, certain legalfees, share-based compensation, and depreciation; and • expenses incurred with the development and operation of our manufacturing facility.We expense research and development costs as incurred.We typically use our employee and infrastructure resources across our development programs. We track outsourced development costs byproduct candidate or development program, but we do not allocate personnel costs, license payments made under our licensing arrangements or otherinternal costs to specific development programs or product candidates. These costs are included in other research and development expenses in thetable below.The following table summarizes our research and development expenses: Year EndedDecember 31, 2018 2017 Change Ophthalmology programs $7,069,280 $4,133,015 $2,936,265 Salivary gland programs 1,136,355 913,706 222,649 Neurodegenerative diseases programs 5,164,761 2,220,843 2,943,918 Manufacturing 5,224,272 3,213,861 2,010,411 Other research and development costs 15,025,555 11,878,287 3,147,268 Total research and development expenses $33,620,223 $22,359,712 $11,260,511 Research and development activities are central to our business model. We expect that our research and development expenses willcontinue to increase substantially for the foreseeable future as we initiate additional preclinical and clinical trials of our existing product candidatesand continue to discover and develop additional product candidates. This increase in research and development costs may be partially offset by theresearch funding provided in connection with the Collaboration Agreement we entered into in January 2019We cannot determine with certainty the duration and costs of future clinical trials of our product candidates or any other product candidatewe may develop or if, when, or to what extent we will generate revenue from the commercialization and sale of any product candidate for which weobtain marketing approval. We may never succeed in obtaining marketing approval for any product candidate. The duration, costs and timing ofclinical trials and development of our existing product candidates or any other product candidate we may develop will depend on a variety of factors,including: • the scope, rate of progress, expense and results of clinical trials of our existing product candidates, as well as of any future clinicaltrials of other product candidates and other research and development activities that we may conduct; • uncertainties in clinical trial design and patient enrollment rates; • the actual probability of success for our product candidates, including the safety and efficacy, early clinical data, competition,manufacturing capability and commercial viability; • significant and changing government regulation and regulatory guidance; • the timing and receipt of any marketing approvals; and • the expense of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights.A change in the outcome of any of these variables with respect to the development of a product candidate could mean a significant changein the costs and timing associated with the development of that 126Table of Contentsproduct candidate. For example, if the FDA or another U.S. or foreign regulatory authority were to require us to conduct clinical trials beyond thosethat we anticipate will be required for the completion of clinical development of a product candidate, or if we experience significant delays in ourclinical trials due to patient enrollment or other reasons, we would be required to expend significant additional financial resources and time on thecompletion of clinical development.Other non-operating income (expense)Other non-operating income (expense) includes the following:Foreign currency (loss)gainOur consolidated financial statements are presented in U.S. dollars, which is our reporting currency. The financial position and results ofoperations of our subsidiaries MeiraGTx UK II and MeiraGTx B.V. are measured using the foreign subsidiaries’ local currency as the functionalcurrency. MeiraGTx UK II cash accounts holding U.S. dollars are remeasured based upon the exchange rate at the date of remeasurement with theresulting gain or loss included in the consolidated statement of operations and comprehensive loss. Expenses of such subsidiaries have been translatedinto U.S. dollars at average exchange rates prevailing during the period. Assets and liabilities have been translated at the rates of exchange on theconsolidated balance sheet date. The resulting translation gain and loss adjustments are recorded directly as a separate component of shareholders’equity and as other comprehensive loss on the consolidated statement of operations and comprehensive loss.Convertible note inducement expenseConvertible note inducement expense consists of the issuance of a warrant to a convertible note holder as an inducement to convert thenote payable into Series C preferred shares.Change in fair value of warrant liabilityWe have determined that our warrants are classified as liabilities on our balance sheet because the Preferred Shares underlying the warrantshave a redemption feature in the event of a change of control of the Company. The fair values of the warrants are estimated using the Black-Scholesvaluation model with certain assumptions regarding risk free interest rate, expected volatility, expected divided yield and expected life. The Black-Scholes value of the warrants was recorded as a warrant liability and is remeasured quarterly. Any changes in the quarterly valuation of the warrants ischarged to operations.Income TaxesThe 2018 income tax provision consisted of current tax expense of $0 and a deferred tax benefit of $474,391. The 2018 deferred taxbenefit includes a $474,391 benefit recorded due to the required intraperiod tax allocation resulting from the loss from continuing operations and othercomprehensive income.Critical Accounting Policies and Use of EstimatesOur management’s discussion and analysis of financial condition and results of operations is based on our financial statements, which havebeen prepared in accordance with generally accepted accounting principles in the United States. The preparation of our financial statements andrelated disclosures requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities, costs and expenses and thedisclosure of contingent assets and liabilities in our financial statements. We base our estimates on historical experience, known trends and events andvarious other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about thecarrying values of assets and liabilities that are not readily apparent from other sources. We evaluate our estimates and assumptions on an ongoingbasis. Our actual results may differ from these estimates under different assumptions or conditions. 127Table of ContentsWhile our significant accounting policies are described in more detail in the notes to our financial statements appearing in this Form 10-K,we believe that the following accounting policies are those most critical to the judgments and estimates used in the preparation of our financialstatements.ConsolidationThe accompanying consolidated financial statements include the accounts of Meira Holdings and its wholly owned subsidiaries:MeiraGTx Limited, a limited company under the laws of England and Wales (“Meira Limited);MeiraGTx, LLC, a Delaware corporation (“Meira LLC”);BRI-Alzan, Inc., a Delaware corporation (“BRI-Alzan”);MeiraGTx B.V., a Netherlands corporation (“Meira BV”);MeiraGTx Neurosciences, Inc. a Delaware corporation (“Meira Neuro”);MeiraGTx UK II Limited, (“Meira UK II”), a limited company under the laws of England and Wales; andMeiraGTx UK Limited (“Meira UK”), a limited company under the laws of England and Wales.All intercompany balances and transactions between the consolidated companies have been eliminated in consolidation.Foreign CurrenciesOur consolidated financial statements are presented in U.S. dollars, our reporting currency. The financial position and results of operationsof MeiraGTx UK II and MeiraGTx UK II cash accounts holding U.S. dollars are remeasuring based upon the exchange rate at the date of remeasurementwith the resulting gain or loss included in the consolidated statement of operations and comprehensive loss. Expenses of such subsidiaries have beentranslated into U.S. dollars at average exchange rates prevailing during the period. Assets and liabilities have been translated at the rates of exchangeon the consolidated balance sheet dates. The resulting translation gain and loss adjustments are recording directly as a separate component ofshareholders’ equity and as other comprehensive loss on the consolidated statements of operations and comprehensive loss.Income TaxesSince we have recurring losses and a valuation allowance against deferred tax assets, there was no tax expense (benefit) throughDecember 31, 2017. For the year ended December 31, 2018, we recognized a tax benefit of $(474,391). As of December 31, 2018, we had federal andstate net operating loss (“NOL”) carryforwards in the United States of approximately $14.2 million and $14.2 million respectively, and in the U.K. ofapproximately $94.1 million, which are available to reduce future taxable income. The U.S. federal and state NOL carryforwards incurred prior toJanuary 1, 2018 in the amount of approximately $6.8 million and $6.7 million, respectively, will begin to expire in 2036. The U.S. NOL incurred afterDecember 31, 2017 and the U.K. NOL will be indefinitely carried forward. As of December 31, 2018, we also had orphan drug and research anddevelopment credits in the U.S. in the amount of $1.1 million, which will begin to expire 2036.Research and DevelopmentResearch and development costs are charged to expense as incurred. These costs include, but are not limited to, employee-related expenses,including salaries, benefits and travel of our research and development personnel; expenses incurred under agreements with contract researchorganizations and investigative sites that 128Table of Contentsconduct clinical and preclinical studies and manufacture the drug product for the clinical studies and preclinical activities; acquisition of in-processresearch and development; facilities; supplies; rent, insurance, certain legal fees, stock-based compensation, depreciation and other costs associatedwith clinical and preclinical activities and regulatory operations. Refundable research and development tax credits received are recorded as an offset tothese costs.Costs for certain development activities, such as outside research programs funded by us, are recognized based on an evaluation of theprogress to completion of specific tasks with respect to their actual costs incurred. Payments for these activities are based on the terms of the individualarrangements, which may differ from the pattern of costs incurred, and are reflected in the financial statements as prepaid or accrued research anddevelopment expense, as the case may be.Share-Based CompensationOptionsWe grant share options to employees, non-employee members of our board of directors and non-employee consultants as compensation forservices performed. Employee and non-employee members of the board of directors’ awards of share-based compensation are accounted for inaccordance with ASC 718, Compensation—Stock Compensation, or ASC 718. ASC 718 requires all share-based payments to employees andnon-employee directors, including grants of share options, to be recognized in the statement of operations and comprehensive loss based on their grantdate fair values. The grant date fair value of share options is estimated using the Black-Scholes option valuation model.Using this model, fair value is calculated based on assumptions with respect to (i) the fair value our ordinary shares on the grant date;(ii) expected volatility of our ordinary share price, (iii) the periods of time over which employees and members of our board of directors are expected tohold their options prior to exercise (expected term), (iv) expected dividend yield on our ordinary shares, and (v) risk-free interest rates.Our ordinary shares were not traded on a public exchange prior to our IPO in June 2018. Therefore, we believe that our future volatility willdiffer materially during the expected term from the volatility that would be calculated from our historical share prices to date. Consequently, expectedvolatility is based on an analysis of guideline companies in accordance with ASC 718. The expected dividend yield is zero as we have never paiddividends and do not currently anticipate paying any in the foreseeable future. Risk-free interest rates are based on quoted U.S. Treasury rates forsecurities with maturities approximating the option’s expected term.As of January 1, 2016, we early adopted ASU 2016-09, Improvements to Employee Share-Based Payment Accounting, and accounts forforfeitures as they occur from that date. Additionally, excess tax benefits and deficiencies will be recognized as income tax expense or benefit in theincome statement. There was no cumulative effect adjustment as we did not issue any options prior to January 1, 2016.We had accounted for options granted to non-employee consultants under ASC 505-50, Equity-Based Payments to Non-Employees. Assuch, we estimate the fair value of each such option using the Black-Scholes model, with the expected term of share options granted to non-employeesinitially equal to the options’ maximum contractual life of ten years, at issuance. On each subsequent reporting date until performance is complete, werevalue all outstanding options granted to non-employee consultants during the vesting period of each tranche. Under ASC 505-50, uponre-measurement of each award, income or expense is recognized during its vesting term. Compensation cost relating to awards with service-basedgraded vesting schedules is recognized as general and administrative and research and development expenses in the consolidated statement ofoperations and comprehensive loss using the straight-line method. On July 1, 2018, we early adopted ASU 2018-07, Improvements to NonemployeeShare-Based Payment Accounting, which simplifies the accounting for share-based payments granted to nonemployees for goods and services. Theadoption did not have a material effect on the consolidated financial statements. 129Table of ContentsRestricted SharesIn connection with certain service agreements and research agreements, we have granted restricted ordinary shares as compensation. Theshares are recognized in the statement of operations and comprehensive loss based on their grant date fair values. Compensation cost relating to sharegrants with service-based graded vesting schedules is recognized based on the vesting schedule.Results of OperationsThe following table summarizes our results of operations for the years ended December 31, 2018 and 2017, respectively 2018 2017 Change Operating expenses: General and administrative $44,483,938 $9,325,017 $35,158,921 Research and development 33,620,223 22,359,712 11,260,511 Total operating expenses 78,104,161 31,684,729 46,419,432 Loss from operations (78,104,161) (31,684,729) (46,419,432) Other non-operating income (expense) Other income 83,075 — 83,075 Foreign currency (loss) gain (3,824,383) 1,676,117 (5,500,500) Convertible note inducement expense — (553,500) 553,500 Change in fair value of warrant liability (1,514,775) (465,633) (1,049,142) Interest income 53,408 26,073 27,335 Interest expense (33,429) (42,863) 9,434 Loss before income taxes (83,340,265) (31,044,535) (52,295,730) Benefit for income taxes 474,391 — 474,391 Net loss $(82,865,874) $(31,044,535) $(51,821,339) General and Administrative ExpensesGeneral and administrative expenses were $44.5 million for the year ended December 31, 2018, compared to $9.3 million for the yearended December 31, 2017. The increase of $35.2 million was primarily due to increases of $19.7 million in payroll, $14.0 million in share-basedcompensation, $1.1 million in legal, $0.7 million in insurance $0.5 million in accounting, $0.4 million in travel, and $0.3 million in investor relations,which was partially offset by a decrease of $1.5 million in rent.Research and Development ExpensesResearch and development expenses for the year ended December 31, 2018 were $33.6 million, compared to $22.4 million for the yearended December 31, 2017. The increase of $11.2 million was primarily due to an increase in costs of $3.0 million for clinical trial costs related to ourophthalmology programs, $3.0 million for acquired neurology research and development, $2.3 million related costs of payroll and consultants,$1.6 million of depreciation, $1.3 million related to the preparation for production of our manufacturing facility, $1.0 million in share-basedcompensation, and $0.7 million in rent, which was partially offset an increase of $1.7 million in the research and development credit in the UnitedKingdom in 2018. 130Table of ContentsForeign Currency (Loss) GainForeign currency loss was $3.8 million for the year ended December 31, 2018 compared to a gain of $1.7 million for the year endedDecember 31, 2017. The change of $5.5 million was primarily due to a strengthening of the U.S. dollar against the pound sterling in 2018.Convertible Note Inducement ExpenseWe recorded a $0.5 million convertible note inducement expense for the year ended December 31, 2017 primarily due to the issuance of awarrant to purchase 231,898 Convertible Preferred C Shares in 2017 to a convertible noteholder as an inducement to convert the note into ConvertiblePreferred C Shares.Change in Fair Market Value of Warrant LiabilityWe recorded $1.5 million change in fair value of a warrant liability for the year ended December 31, 2018, compared to $0.5 million for theyear ended December 31, 2017. The increase of $1.0 million was primarily due to the revaluation of certain warrants, which were issued to certaininvestors in September and November 2017, using the Black-Scholes valuation model at June 7, 2018, when the warrants were exercised andDecember 31, 2017.Income TaxesThe 2018 income tax provision consisted of current tax expense of $0 and a deferred tax benefit of $474,391. The 2018 deferred taxbenefit includes a $474,391 benefit recorded due to the required intraperiod tax allocation resulting from the loss from continuing operations and othercomprehensive income.Liquidity and Capital ResourcesSince our inception, we have incurred significant operating losses. We have not generated positive cash flows from operations, and thereare no assurances that we will be successful in obtaining an adequate level of financing for the development and commercialization of our productcandidates. We expect to incur significant expenses and operating losses for the foreseeable future as we advance the preclinical and clinicaldevelopment of our product candidates. We expect that our research and development and general and administrative costs will increase in connectionwith conducting preclinical studies and clinical trials for our product candidates, building out internal capacity to have product manufactured tosupport preclinical studies and clinical trials, expanding our intellectual property portfolio, and providing general and administrative support for ouroperations. As a result, we will need additional capital to fund our operations, which we may obtain from additional equity or debt financings,collaborations, licensing arrangements, or other sources.We are required to maintain a stand-by letter of credit as a security deposit under a certain lease with ARE, an entity that is under commoncontrol with an entity that is a minority shareholder of the Company and whose executive chairman and founder is on our board of directors. Our bankrequires us to maintain restricted cash balances to serve as collateral for the letter of credit issued to the landlord by the bank. As of December 31, 2018,the restricted cash balances for the ARE lease was invested in a commercial money market account. The restricted cash balance for the ARE leaseremains at $123,376 through the end of the lease term in December 2021, plus three months. We had $123,376 of restricted cash included in long-termassets as of December 31, 2018. We do not currently have any approved products and have never generated any revenue from product sales. We havehistorically financed our operations primarily through the sale of our ordinary shares and Convertible Preferred C Shares. In January 2019, we enteredinto the Collaboration Agreement, which provides for Janssen to pay us an $100 million upfront payment and provide us with research funding, and weare eligible to receive potential milestone payments and royalties. 131Table of ContentsCash FlowsWe had $68.1 million and $8.5 million of cash and cash equivalents as of December 31, 2018 and 2017, respectively.The following table summarizes our sources and uses of cash for the period presented: For the year ended December 31, 2018 2017 Net cash used in operating activities $(58,887,870) $(18,055,386) Net cash used in investing activities (11,258,479) (10,535,717) Net cash provided by financing activities 130,040,415 19,340,215 Increase (decrease) in cash $59,894,066 $(9,250,888) Operating ActivitiesDuring the year ended December 31, 2018, our cash used in operating activities of $58.9 million was primarily due to our net loss of$82.9 million as we incurred expenses associated with research activities on our clinical programs and research activities for our other productcandidates and incurred general and administrative expenses. The loss included non-cash charges of $27.9 million, which consisted of $18.1 million ofshare-based compensation, issuance of shares for acquired research and development in the amount of $3.0 million, change in fair value of warrantliability in the amount of $1.5 million, depreciation of $2.1 million, and a foreign currency loss of $3.9 million, which was partially offset by anincome tax benefit of $0.5 million. Additionally, current assets, consisting of prepaid expenses, other current assets and security deposits increased by$3.9 million. Current liabilities, consisting of accounts payable, accrued expenses, deferred rent, due to affiliate and other liabilities, decreased by$0.1 millionDuring the year ended December 31, 2017, our cash used in operating activities of $18.1 million was primarily due to our net loss of$31.0 million as we incurred expenses associated with research activities on our clinical programs and research activities for our other productcandidates and incurred general and administrative expenses. The loss included non-cash charges of $3.0 million, which consisted of $3.0 million ofshare-based compensation, change in fair value of warrant liability in the amount of $0.5 million, convertible note inducement expense of $0.5 millionand depreciation of $0.7 million, which was partially offset by a foreign currency gain of $1.7 million. Additionally, current liabilities, consisting ofaccounts payable, accrued expenses deferred rent, and due to affiliate, increased by $11.1 million. Current assets, consisting of prepaid expenses andother current assets, decreased by $1.2 million.Investing ActivitiesNet cash used in investing activities for the years ended December 31, 2018 and December 31, 2017 of $11.3 million and $10.5 million,respectively, consisted of purchase of property plant and equipment, primarily for our manufacturing facility.Financing ActivitiesNet cash provided by financing activities was $130.0 million for the year ended December 31, 2018, represented proceeds of $65.6 millionfrom the issuance of Ordinary Shares in connection with our initial public offering, $56.1 million from the issuance of Convertible Preferred C Sharesand $9.7 million from the exercise of warrants, which was partially offset by the repayment of a note payable in the amount of $1.4 million.Net cash provided by financing activities was $19.3 million for the year ended December 31, 2017, represented proceeds of $16.8 millionfrom the issuance of Convertible Preferred C Shares and $2.5 million from the issuance of a note payable 132Table of ContentsFunding RequirementsOur operating expenses increased substantially in 2018 and 2017 and are expected to increase substantially in the future in connectionwith our ongoing activities, particularly as we advance our clinical activities including scale-up of manufacturing processes and additional clinicaltrials. In addition, we expect to continue to incur additional costs associated with operating as a public company.Specifically, our expenses will increase as we: • pursue the preclinical and clinical development of our product candidates; • scale up our manufacturing processes and capabilities to support our preclinical studies and clinical trials of our productcandidates; • in-license or acquire the rights to other products, product candidates or technologies; • maintain, expand and protect our intellectual property portfolio; • hire additional personnel in research, manufacturing and regulatory and clinical development as well as management personnel;and • continue to expand our operational, financial and management systems and increase personnel, including personnel to support ouroperations as a public company.Based on our planned use of the net proceeds of the IPO, our current cash and cash equivalents, the proceeds from a private placement ofordinary shares in March 2019 and the proceeds we are to receive pursuant to the terms of the Collaboration Agreement, we estimate that such fundswill be sufficient to enable us to fund our operating expenses and capital expenditure requirements. We have based these estimates on assumptions thatmay prove to be wrong, and we could utilize our available capital resources sooner than we expect.Because of the numerous risks and uncertainties associated with research, development and commercialization of gene therapies, it isdifficult to estimate with certainty the amount of our working capital requirements. Our future funding requirements will depend on many factors,including: • the progress, costs and results of our preclinical development and initial clinical trials for our gene therapy programs; • the progress, costs and results of our additional clinical, research and preclinical development programs in gene therapy; • the costs and timing of process development and manufacturing scale-up activities associated with our clinical programs; • our ability to establish and maintain strategic collaborations, licensing or other agreements and the financial terms of suchagreements; • the scope, progress, results and costs of any product candidates that we may derive from our platform technology or any otherproduct candidates that we may develop; • the extent to which we in-license or acquire rights to other products, product candidates or technologies; and • the costs and timing of preparing, filing and prosecuting patent applications, maintaining and protecting our intellectual propertyrights and defending against any intellectual property-related claims.Until such time, if ever, that we can generate product revenue sufficient to achieve profitability, we expect to finance our cash needsthrough a combination of equity offerings, debt financings, collaboration agreements, other third-party funding, strategic alliances, licensingarrangements and marketing and distribution arrangements. 133Table of ContentsTo the extent that we raise additional capital through the sale of equity or convertible debt securities, your ownership interest will bediluted, and the terms of these securities may include liquidation or other preferences that adversely affect your rights as a holder of ordinary shares.Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to takespecific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we raise additional funds through other third-party funding, collaboration agreements, strategic alliances, licensing arrangements or marketing and distribution arrangements, we may have torelinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or grant licenses on terms that may notbe favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduceor terminate our product development or future commercialization efforts or grant rights to develop and market products or product candidates that wewould otherwise prefer to develop and market ourselves.Contractual Obligations and CommitmentsThe following is a summary of our significant contractual obligations as of December 31, 2018. Payments by Period Total Less Than 1Year More Than1 Year andLess Than 3 More Than3 Years andLess Than 5 More Than5 Years Operating leases 4,615,676 889,465 1,828,838 700,569 1,196,805 Capitalized leases 35,894 35,894 — — — 4,651,570 925,359 1,828,838 700,569 1,196,805 (1)Represents the leases for office, laboratory, and manufacturing space in London, United Kingdom and New York, New York undernon-cancelable operating leases that expire between December 2021 and May 2027 and December 2021.(2)Represents future payments under capitalized leases for office equipment.The contractual obligations table does not include any potential future payments we may be required to make under (1) our licenseagreements with UCLB, Brandeis and other entities or (2) our sponsored research agreements with universities and commercial research organizations.These agreements may be terminated upon 30-90 days written notice and, therefore, the amounts to be paid by us are not fixed or determinable at thistime.Off-Balance Sheet ArrangementsWe have not entered into any off-balance sheet arrangements under applicable SEC rules and do not have any holdings in variable interestentities. ITEM 7A.QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK.We are exposed to market risks in the ordinary course of our business. These risks primarily include foreign currency exchange ratesensitivities. However, relative to foreign currency exposures as of December 31, 2018, a 10% unfavorable movement in foreign currency exchangerates would not expose us to a significant increase in net loss. We had cash and cash equivalents of $68.1 million as of December 31, 2018, whichconsist of non-interest-bearing bank deposits. Other than accounts payable and accrued expenses incurred in the ordinary course of business, we had noother debt outstanding as of December 31, 2018. We had cash and cash equivalents of $8.5 million as of December 31, 2017, which consist of bankdeposits and money market funds. Such interest-earning instruments carry a degree of interest rate risk; however, historical fluctuations in interestincome have not been significant for us. Other than accounts payable and accrued expenses incurred in the ordinary course of business, we had a notepayable of $1.4 million as of December 31, 2017. 134Table of ContentsITEM 8.FINANCIAL STATEMENTS AND SUPPLEMENTARY DATAINDEX TO CONSOLIDATED FINANCIAL STATEMENTSMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESFOR THE YEARS ENDED DECEMBER 31, 2018 AND 2017 Report of Independent Registered Public Accounting Firm 136 Consolidated: Balance Sheets 137 Statements of Operations and Comprehensive Loss 138 Statements of Convertible Preferred C Shares and Shareholders’ Equity (Deficit) 139 Statements of Cash Flows 140 Notes to Consolidated Financial Statements 141 135Table of ContentsReport of Independent Registered Public Accounting FirmTo the Shareholders and the Board of Directors of MeiraGTX Holdings plc and SubsidiariesOpinion on the Financial StatementsWe have audited the accompanying consolidated balance sheets of MeiraGTX Holdings plc and Subsidiaries(the Company) as of December 31, 2018 and 2017, the related consolidated statements of operations and comprehensive loss, shareholders’ deficit andcash flows for each of the two years in the period ended December 31, 2018, and the related notes (collectively referred to as the “consolidatedfinancial statements”). In our opinion, the consolidated financial statements present fairly, in all material respects, the financial position of theCompany at December 31, 2018 and 2017, and the results of its operations and its cash flows for each of the two years in the period endedDecember 31, 2018, in conformity with U.S. generally accepted accounting principles.Basis for OpinionThese financial statements are the responsibility of the Company’s management. Our responsibility is to express an opinion on the Company’sfinancial statements based on our audits. We are a public accounting firm registered with the Public Company Accounting Oversight Board (UnitedStates) (PCAOB) and are required to be independent with respect to the Company in accordance with the U.S. federal securities laws and the applicablerules and regulations of the Securities and Exchange Commission and the PCAOB.We conducted our audits in accordance with the standards of the PCAOB. Those standards require that we plan and perform the audit to obtainreasonable assurance about whether the financial statements are free of material misstatement, whether due to error or fraud. The Company is notrequired to have, nor were we engaged to perform, an audit of its internal control over financial reporting. As part of our audits we are required to obtainan understanding of internal control over financial reporting but not for the purpose of expressing an opinion on the effectiveness of the Company’sinternal control over financial reporting. Accordingly, we express no such opinion.Our audits included performing procedures to assess the risks of material misstatement of the financial statements, whether due to error or fraud, andperforming procedures that respond to those risks. Such procedures included examining, on a test basis, evidence regarding the amounts anddisclosures in the financial statements. Our audits also included evaluating the accounting principles used and significant estimates made bymanagement, as well as evaluating the overall presentation of the financial statements. We believe that our audits provide a reasonable basis for ouropinion./s/ Ernst & Young LLPWe have served as the Company’s auditor since 2016.Stamford, ConnecticutMarch 26, 2019 136Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESCONSOLIDATED BALANCE SHEETS December 31,2018 December 31,2017 ASSETS CURRENT ASSETS: Cash and cash equivalents $68,080,175 $8,548,638 Prepaid expenses 1,937,785 1,961,243 Other current assets 4,634,105 965,233 Total Current Assets 74,652,065 11,475,114 Property, plant and equipment, net 22,014,237 14,255,729 Security deposits 105,085 — Restricted cash 123,376 123,376 TOTAL ASSETS $96,894,763 $25,854,219 LIABILITIES, CONVERTIBLE PREFERRED C SHARES AND SHAREHOLDERS’ EQUITY (DEFICIT) CURRENT LIABILITIES: Accounts payable $3,042,861 $7,055,380 Accrued expenses 11,991,697 9,332,944 Note payable — 1,442,009 Warrant liability — 2,679,633 Capitalized lease obligation—current portion 27,199 30,850 Due to Kadmon — 861,030 Other current liabilities 437,053 — Total Current Liabilities 15,498,810 21,401,846 Capitalized lease obligation 7,097 34,298 Deferred rent 201,264 266,290 Asset retirement obligation 128,119 178,419 TOTAL LIABILITIES 15,835,290 21,880,853 COMMITMENTS CONVERTIBLE PREFERRED C SHARES Convertible Preferred C Shares0 and 5,005,935 outstanding at December 31, 2018 and December 31, 2017, respectively (liquidationpreference of $52,455,700 at December 31, 2017) — 51,338,631 SHAREHOLDERS’ EQUITY (DEFICIT): Ordinary Shares, $0.00003881 nominal value, 1,288,327,750 authorized 27,386,632 issued and outstanding atDecember 31, 2018 8,826,190 issued and 8,714,563 issued and outstanding at December 31, 2017 1,064 342 Capital in excess of nominal value 229,054,460 20,080,713 Accumulated other comprehensive income (loss) 293,666 (2,022,477) Accumulated deficit (148,289,717) (65,423,843) Total Shareholders’ Equity (Deficit) 81,059,473 (47,365,265) TOTAL LIABILITIES, CONVERTIBLE PREFERRED C SHARES AND SHAREHOLDERS’ EQUITY (DEFICIT) $96,894,763 $25,854,219 See Notes to Consolidated Financial Statements 137Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESCONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS For the Year Ended December 31, 2018 2017 Operating expenses: General and administrative $44,483,938 $9,325,017 Research and development 33,620,223 22,359,712 Total operating expenses 78,104,161 31,684,729 Loss from operations (78,104,161) (31,684,729) Other non-operating income (expense): Other income 83,075 — Foreign currency (loss) gain (3,824,383) 1,676,117 Convertible note inducement expense — (553,500) Change in fair value of warrant liability (1,514,775) (465,633) Interest income 53,408 26,073 Interest expense (33,429) (42,863) Loss before income taxes (83,340,265) (31,044,535) Benefit for income taxes 474,391 — Net loss (82,865,874) (31,044,535) Other comprehensive income (loss): Foreign currency translation, net of tax of $474,391 and $0 in 2018 and 2017, respectively 2,316,143 (1,361,365) Total comprehensive loss $(80,549,731) $(32,405,900) Net loss $(82,865,874) $(31,044,535) Accretion on convertible preferred C shares and warrants (1,806,512) (806,963) Adjusted net loss $(84,672,386) $(31,851,498) Basic and diluted net loss per ordinary share $(4.47) $(3.72) Weighted-average number of ordinary shares outstanding 18,948,520 8,572,315 See Notes to Consolidated Financial Statements 138Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESCONSOLIDATED STATEMENTS OF CONVERTIBLE PREFERRED C SHARES AND SHAREHOLDERS’ EQUITY (DEFICIT)AS OF DECEMBER 31, 2018 Convertible PreferredC Shares Shareholders’ Equity (Deficit) Shares Amount OrdinaryShares Amount Capital inExcess ofNominalValue AccumulatedOtherComprehensiveIncome (Loss) AccumulatedDeficit TotalShareholders’Equity(Deficit) Balance at December 31, 2016 1,574,739 $32,833,660 8,818,461 $342 $17,900,995 $(661,112) $(34,379,308) $(17,139,083) Exercised stock options — — 1,288 — 9,950 — — 9,950 Issuance of A ordinary shares in connectionwith a license agreement — — 6,441 — 17,000 — — 17,000 Extinguishment of convertible preferred Cshares, net of unaccreted issuance costs (1,584,469) (33,115,157) — — 33,115,157 — — 33,115,157 Issuance of convertible preferred C shares inconnection with extinguishment 3,168,929 33,206,360 — — (33,206,360) — — (33,206,360) Conversion of note payable into convertiblepreferred C shares 238,579 2,500,000 — — — — — — Issuance of convertible preferred C shares,net of warrants and issuance costs 1,608,157 15,198,008 — — — — — — Accretion of issuance costs on convertiblepreferred C shares — 100,760 — — (100,760) — — (100,760) Accretion of warrants issued in connectionwith convertible preferred C shares — 615,000 — — (615,000) — — (615,000) Share-based compensation — — — — 2,959,731 — — 2,959,731 Foreign currency translation — — — — — (1,361,365) — (1,361,365) Net loss for the year ended December 31,2017 — — — — — — (31,044,535) (31,044,535) Balance at December 31, 2017 5,005,935 $51,338,631 8,826,190 $342 $20,080,713 $(2,022,477) $(65,423,843) $(47,365,265) Issuance of convertible preferred C shares inconnection with payables 129,419 1,356,129 — — — — — — Issuance of convertible preferred C shares inconnection with a license agreement 13,360 140,000 — — — — — — Sale of convertible preferred C shares, net ofissuance costs 5,425,124 56,159,119 — — — — — — Accretion of issuance costs on convertiblepreferred C shares — 761,012 — — (761,012) — — (761,012) Accretion of warrants issued in connectionwith convertible preferred C shares — 1,045,500 — — (1,045,500) — — (1,045,500) Exercise of warrants 927,594 9,720,000 — — 4,194,408 — — 4,194,408 Conversion of convertible preferred C sharesinto A ordinary shares (11,501,432) (120,520,391) 11,501,432 446 120,519,945 — — 120,520,391 Sale of ordinary shares in initial publicoffering, net of issuance costs of$9,807,622 — — 5,000,000 194 65,192,184 — — 65,192,378 Issuance of ordinary shares in connectionwith Vector Neurosciences acquisition 202,500 9 2,990,241 — — 2,990,250 Share-based compensation — — 1,856,510 73 17,883,481 — — 17,883,554 Foreign currency translation, net of incometaxes — — — — — 2,316,143 — 2,316,143 Net loss for the year ended December 31,2018 — — — — — — (82,865,874) (82,865,874) Balance at December 31, 2018 — $— 27,386,632 $1,064 $229,054,460 $293,666 $(148,289,717) $81,059,473 See Notes to Consolidated Financial Statements 139Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESCONSOLIDATED STATEMENTS OF CASH FLOWS For the Year Ended December 31, 2018 2017 Cash flows from operating activities: Net loss $(82,865,874) $(31,044,535) Adjustments to reconcile net loss to net cash used in operating activities: Preferred C shares issued in connection with a license agreement 140,000 — Issuance of shares for services — 17,000 Share-based compensation expense 17,883,554 2,959,731 Foreign currency loss (gain) 3,824,383 (1,676,117) Depreciation 2,053,220 679,177 Amortization of interest on asset retirement obligation (38,301) 19,313 Change in fair value of warrant liability 1,514,775 465,633 Convertible note inducement expense — 553,500 Issuance of shares for acquired research and development expense 2,990,250 — Issuance of note payable in connection with lease termination — 1,442,009 Benefit for income taxes (474,391) — (Increase) in operating assets: Prepaid expenses (35,465) (669,756) Other current assets (3,684,465) (493,424) Security deposits (115,573) — Increase (decrease) in operating liabilities: Accounts payable (2,119,493) 4,728,491 Accrued expenses 2,529,568 4,969,619 Due to Kadmon (861,030) 317,992 Other liabilities 436,161 — Deferred rent (65,189) (324,019) Net cash used in operating activities (58,887,870) (18,055,386) Cash flows from investing activities: Purchase of property, plant and equipment (11,258,479) (10,535,717) Net cash used in investing activities (11,258,479) (10,535,717) Cash flows from financing activities: Payments on capitalized lease obligation (30,852) (24,388) Exercise of warrants 9,720,000 — Proceeds from the sale of ordinary shares 69,750,000 — Issuance costs in connection with ordinary shares (4,115,843) — Issuance costs in connection with convertible preferred C shares (690,475) — Proceeds from the sale of convertible preferred C shares 56,849,594 16,854,653 (Payment) issuance of note payable (1,442,009) 2,500,000 Proceeds from excercised stock options — 9,950 Net cash provided by financing activities 130,040,415 19,340,215 Net increase (decrease) in cash, cash equivalents and restricted cash 59,894,066 (9,250,888) Effect of exchange rate changes on cash (362,529) 1,417 Cash, cash equivalents and restricted cash at beginning of year 8,672,014 17,921,485 Cash, cash equivalents and restricted cash at end of year $68,203,551 $8,672,014 Supplemental disclosure of non-cash transactions: Fixed asset acquisition included in accounts payable and accrued expenses at end of year $293,051 $415,650 Issuance of convertible preferred C shares in connection with payables $1,356,129 $— Conversion of convertible preferred C shares into ordinary shares $120,520,391 $— Reclassification of warrant liability upon exercise of warrants $4,194,408 $— Capitalized lease obligation for equipment purchase $— $78,063 Issuance costs in connection with sale of ordinary shares in accounts payable and accrued expenses at end of period $441,779 $— Conversion of note payable into convertible preferred C shares $— $2,500,000 Asset retirement obligation in connection with a lease $(29,804) $(75,011) Supplemental disclosure of cash flow information: Cash paid for interest $34,546 $20,894 See Notes to Consolidated Financial Statements 140Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS 1.Principal Business Activity:The CompanyMeiraGTx Holdings plc (the “Company” or “Meira Holdings”), a limited company under the laws of the Cayman Islands, is a clinical-stagebiotech company developing novel gene therapy treatments for a wide range of inherited and acquired disorders for which there are no effectivetreatments available. The Company is focused on developing therapies for ocular diseases, including rare inherited blindness as well asXerostomia following radiation treatment for head and neck cancers and neurodegenerative diseases such as amyothrophic lateral sclerosis(“ALS”) and Parkinson’s disease (“PD”).Reorganization and Initial Public OfferingWe commenced operations as MeiraGTx Limited, a private limited company incorporated under the laws of England and Wales in 2015. OnMay 28, 2018, the Board of Directors of MeiraGTx Limited approved the Reorganization Transactions, effective June 7, 2018, pursuant to whichthe Board of Directors approved the transfer of the shares held by each of the MeiraGTx Limited’s shareholders for the equivalent class andnumber of shares issued by Meira Holdings. On June 7, 2018, the Company completed its initial public offering (“IPO”), selling 5,000,000ordinary shares (“Ordinary Shares”) at a public offering price of $15.00 per share, and receiving $65.2 million in net proceeds, after deductingunderwriting discounts and commissions and offering expenses payable by us.Reverse Share SplitOn June 7, 2018 MeiraGTx Limited’s Board of Directors and shareholders approved a 1:3.881 reverse share split. All share information presentedin these financial statements and accompanying footnotes have been retroactively adjusted to reflect the decreased number of shares resultingfrom this action.Basis of PresentationThe accompanying consolidated financial statements have been prepared in conformity with accounting principles generally accepted in theUnited States of America (“GAAP”). Any reference in these notes to applicable guidance is meant to refer to the authoritative United Statesgenerally accepted accounting principles as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update(“ASU”) of the Financial Accounting Standards Board (“FASB”).LiquidityThe Company has not yet achieved profitable operations. There is no assurance that profitable operations, if ever achieved, could be sustained ona continuing basis. In addition, development activities, clinical and preclinical testing, and commercialization of the Company’s productcandidates will require significant additional financing. The Company’s accumulated deficit at December 31, 2018 totaled $(148,289,717), andmanagement expects to incur substantial losses in future periods. The success of the Company is subject to certain risks and uncertainties,including among others, uncertainty of product development; competition in the Company’s field of use; uncertainty of capital availability;uncertainty in the Company’s ability to enter into agreements with collaborative partners; dependence on third parties; and dependence on keypersonnel. The Company has not generated positive cash flows from operations, and there are no assurances that the Company will be successfulin obtaining an adequate level of financing for the development and commercialization of its product candidates. 141Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS As of December 31, 2018, the Company had cash and cash equivalents in the amount of $68,080,175, which consisted of depository accounts.On January 30, 2019, the Company entered into a collaboration, option and license agreement with Janssen Pharmaceuticals, Inc., one of theJanssen Pharmaceuticals Companies of Johnson & Johnson (the “Collaboration Agreement”). Under the terms of the Collaboration Agreement,the Company will receive an upfront payment of $100 million. The Company will also receive research funding for certain research,manufacturing, clinical development and commercialization costs, potential additional milestone payments upon the achievement of suchmilestones and royalties on future net sales of products. On February 27, 2019, the Company issued 5,797,102 ordinary shares in an $80 millionprivate placement led by JJDC, Inc., the investment arm of Johnson & Johnson, (the “Private Placement”) for net proceeds of $77.4 million. TheCompany estimates that its cash and cash equivalents on hand at December 31, 2018 as well as proceeds from the Collaboration Agreement andthe Private Placement will be sufficient to cover its expenses for at least the next twelve months from the date of issuance of these financialstatements.Risks and UncertaintiesThe Company operates in an industry that is subject to intense competition, government regulation and rapid technological change. TheCompany’s operations are subject to significant risk and uncertainties including financial, operational, technological, regulatory and other risks,including the potential risk of business failure.The Company’s limited capital resources and operations to date have been funded primarily with the proceeds from private equity offerings andthe IPO. In the future, the Company may seek to raise additional capital through equity offerings, debt financings, marketing and distributionarrangements and other collaborations, strategic alliances and licensing arrangements or other sources to enable it to complete the developmentand potential commercialization of its product candidates. 2.Summary of Significant Accounting Policies:ConsolidationThe accompanying consolidated financial statements include the accounts of Meira Holdings and its wholly owned subsidiaries:MeiraGTx Limited, a limited company under the laws of England and Wales (“Meira Limited);MeiraGTx, LLC, a Delaware corporation (“Meira LLC”);BRI-Alzan, Inc., a Delaware corporation (“BRI-Alzan”);MeiraGTx B.V., a Netherlands corporation (“Meira BV”);MeiraGTx Neurosciences, Inc. a Delaware corporation (“Meira Neuro”);MeiraGTx UK II Limited, (“Meira UK II”), a limited company under the laws of England and Wales; andMeiraGTx UK Limited (“Meira UK”), a limited company under the laws of England and Wales.All intercompany balances and transactions between the consolidated companies have been eliminated in consolidation.Use of EstimatesManagement considers many factors in selecting appropriate financial accounting policies and controls, and in developing the estimates andassumptions that are used in the preparation of these consolidated financial statements. Management must apply significant judgment in thisprocess. In addition, other factors may affect estimates, including expected business and operational changes, sensitivity and volatilityassociated with the assumptions used in developing estimates, and whether historical trends are expected to be representative of future trends.The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes and management must selectan amount that falls within that range 142Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS of reasonable estimates. This process may result in actual results differing materially from those estimated amounts used in the preparation of thefinancial statements if these results differ from historical experience, or other assumptions do not turn out to be substantially accurate, even ifsuch assumptions are reasonable when made. In preparing these consolidated financial statements, management used significant estimates in thefollowing areas, among others: valuation of A ordinary shares (“A Ordinary Shares”) issued prior to the Company’s initial public offering, theaccounting for research and development costs, warrants, share-based compensation and accrued expenses.Subsequent EventsThe Company considers events or transactions that occur after the balance sheet date but before the financial statements are issued to provideadditional evidence relative to certain estimates or to identify matters that require additional disclosure. The Company evaluated all events andtransactions through March 26, 2019, the date these consolidated financial statements were issued. See Note 18 for additional information.Cash and Cash EquivalentsThe Company considers all highly liquid instruments with an original maturity of 90 days or less at the time of purchase to be cash equivalents.Cash and cash equivalents consist of deposits that are readily convertible into cash.Warrant LiabilityDuring 2017, the Company issued warrants to purchase Convertible Preferred C Shares (the “Preferred Shares”) to certain investors. Due to thepotential redemption feature of the underlying Preferred Shares, the warrants had been classified as a liability. Liability accounting requires thatthe fair value of warrants be remeasured each reporting period with changes recorded in the statements of operations and comprehensive loss.These Preferred Shares warrants remained outstanding until their exercise in June 2018, at which time the warrant liability was remeasured to fairvalue and reclassified to additional paid-in capital.Financial InstrumentsThe carrying value of prepaid expenses, other current assets, accounts payable, accrued expenses, notes payable and amounts due to an affiliatereported in the consolidated balance sheets equal or approximate fair value due to their short maturities.Fair Value MeasurementsFair value is defined as the price that would be received upon sale of an asset or paid upon transfer of a liability in an orderly transaction betweenmarket participants at the measurement date and in the principal or most advantageous market for that asset or liability. The fair value should becalculated based on assumptions that market participants would use in pricing the asset or liability, not on assumptions specific to the entity. Inaddition, the fair value of liabilities should include consideration of non-performance risk including our own credit risk.The Company follows ASC Topic 820, Fair Value Measurements and Disclosures, or ASC 820, for application to financial assets and liabilities.In addition to defining fair value, the standard expands the disclosure requirements around fair value and establishes a fair value hierarchy forvaluation inputs. The hierarchy prioritizes the inputs into three levels based on the extent to which inputs used in measuring fair 143Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS value are observable in the market. Each fair value measurement is reported in one of the three levels which are determined by the lowest levelinput that is significant to the fair value measurement in its entirety. These levels are: • Level 1: Observable inputs such as quoted prices in active markets for identical assets the reporting entity has the ability to accessas of the measurement date; • Level 2: Inputs, other than the quoted prices in active markets, that are observable either directly or indirectly; and • Level 3: Unobservable inputs in which there is little or no market data, which require the reporting entity to develop its ownassumptions.The table below represents the values of the Company’s financial assets and liabilities that are required to be measured at fair value on a recurringbasis: Fair Value Measurement Using: Description December 31,2018 SignificantObservable Inputs(Level 1) Significant OtherObservable Inputs(Level 2) SignificantUnobservable(Level 3) Restricted cash $123,376 $123,376 $— $— Fair Value Measurement Using: Description December 31,2017 SignificantObservable Inputs(Level 1) Significant OtherObservable Inputs(Level 2) SignificantUnobservable(Level 3) Restricted cash $123,376 $123,376 $— $— Warrants $2,679,633 $— $— $2,679,633 The table below represents a rollforward of the assets and liabilities that are required to be measured at fair value on a recurring basis fromDecember 31, 2016 to December 31, 2018: SignificantObservable Inputs(Level 1) Significant OtherObservable Inputs(Level 2) SignificantUnobservable Inputs(Level 3) Balance as of December 31, 2016 $444,844 $— $— Cash released from restriction (321,468) — — Fair value of warrants issued — — 2,214,000 Change in fair value of warrants — — 465,633 Balance as of December 31, 2017 123,376 — 2,679,633 Change in fair value of warrants 1,514,775 Exercise of warrants — — (4,194,408) Balance as of December 31, 2018 $123,376 $— $— The warrants were classified as liabilities because the underlying Preferred Shares had a redemption feature in the event of a change of control ofthe Company. On June 5, 2018, the warrants were exercised at which time the warrant liability was determined to be $4,194,408, whichrepresented the difference in the market value of the Preferred Shares and the exercise price of the warrants. This resulted in an increase of thewarrant liability in the amount of $1,514,775 for the year ended December 31, 2018. The related warrant liability of $4,194,408 was reclassifiedas Capital in Excess of Nominal Value at such time. 144Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS The fair values of the warrants were estimated using the Black-Scholes valuation model with the following assumptions: June 4, 2018 December 31, 2017 September 21, 2017 Risk-free interest rate 1.77% 1.72% 1.38% Expected volatility 80% 80% 80% Expected dividend yield 0 0 0 Expected life 1 day 9 months 18 months For the unobservable inputs for the warrants, the expected volatility was determined at each measurement date by taking an average of thevolatility of other publicly-traded peer biotechnology companies. The expected life was determined at each measurement date based upon theCompany’s estimate of the time until the Company has a conversion event, as described in Note 12.The fair value of the Preferred Shares were based upon recent issuances of the Company’s Preferred Shares on or about those dates.The estimated fair values of the Company’s warrants were not necessarily indicative of the amounts that would have been realized in a currentmarket exchange. The determination of the fair value of the warrants were sensitive to changes in the assumptions used and a change in thoseinputs could result in a significantly higher or lower fair value measurement. If the volatility were to increase or the expected life were toincrease, the fair value of the warrant would increase. Conversely, if the volatility were to decrease or the expected life were to decrease, the fairvalue of the warrant would decrease.Convertible Preferred C SharesThe Preferred Shares were not redeemable, except in the event of a change of control which was outside the control of the Company and requiredshareholder approval. The redemption value of the Preferred Shares upon a change in control is equal to its liquidation value described below.The Company is accounting for its Preferred Shares under the requirements of ASC 480, Distinguishing Liabilities from Equity, which establishesstandards for how an issuer classifies and measures certain financial instruments with characteristics of both liabilities and equity. The carryingvalue of the Preferred Shares is presented as temporary equity and is increased by periodic accretions so that the carrying amount will equal theredemption amount at the estimated date that the Preferred Shares will be converted into Ordinary Shares. These increases are affected throughcharges against additional paid-in capital, to the extent it is available, or accumulated deficit. For all Preferred Shares issuance, the differencebetween the amount invested by the holders of the Preferred Shares, net of issuance costs and the initial fair value of warrants issued inconnection with the Preferred Shares (if applicable), and the liquidation value of the Preferred Shares is recorded as accretion over the estimatedlife of the Preferred Shares. The accretion is added to net loss to arrive at the net loss available to Ordinary Shareholders in the calculation of lossper Ordinary Share.Concentrations of Credit RiskThe Company maintains its cash and cash equivalents primarily in depository and money market accounts within two large financial institutionsin the United States and one large financial institution in the United Kingdom. Cash balances deposited at these major financial bankinginstitutions exceed the insured limit. The Company has not experienced any losses on its bank deposits and believes these deposits do notexpose the Company to any significant credit risk. 145Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS Property, Plant and Equipment, NetProperty, plant and equipment are stated at cost, net of accumulated depreciation. Depreciation is calculated using the straight-line method overthe estimated useful lives of the respective assets. Leasehold improvements are depreciated over the lesser of their useful lives or the life of thelease (see Note 5).The estimated useful lives of the asset categories are set forth in the table below: Asset Category Useful Lives Computer and office equipment 3 years Laboratory equipment 5 years Manufacturing equipment 7 years Furniture and fixtures 5 years Capitalized leasehold interest 25 years Leasehold improvements lesser of usefullife or remainingterm of lease Expenditures for leasehold improvements are capitalized, and expenditures for maintenance and repairs are expensed to operations as incurred.ASC Topic 360, Property, Plant and Equipment, addresses the financial accounting and reporting for impairment or disposal of long-lived assets.The Company reviews the recorded values of long-lived assets for impairment whenever events or changes in business circumstances indicatethat the carrying amount of an asset or group of assets may not be fully recoverable. The Company recorded no impairment charges in 2018 or2017.Net Loss per Ordinary ShareBasic net loss per Ordinary Share is computed by dividing net loss by the weighted average number of shares of the Company’s Ordinary Sharesassumed to be outstanding during the period of computation. Diluted net loss per ordinary share is computed similar to basic net loss per shareexcept that the denominator is increased to include the number of additional Ordinary Shares that would have been outstanding if the potentialordinary shares had been issued at the beginning of the year and if the additional Ordinary Shares were dilutive (treasury stock method) or thetwo-class method, whichever is more dilutive. For all periods presented, basic and diluted net loss per Ordinary Share are the same, as anyadditional Ordinary Share equivalents would be anti-dilutive (see Note 13).Income TaxesIncome taxes are recorded in accordance with ASC Topic 740, Income Taxes, or ASC 740, which provides for deferred taxes using an asset andliability approach. The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of events that have beenincluded in the financial statements or tax returns. Deferred tax assets and liabilities are determined based on the difference between the financialstatement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse.Realization of net deferred tax assets is dependent on future taxable income. Valuation allowances are provided, if based upon the weight ofavailable evidence, it is more likely than not that some, or all, of the deferred tax assets will not be realized. Realization of net deferred tax assetsis dependent on future taxable income (see Note 14).The Company accounts for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, theCompany recognizes the tax benefit of tax positions to the extent that the 146Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS benefit will more likely than not be realized. The determination as to whether the tax benefit will more likely than not be realized is based uponthe technical merits of the tax position as well as consideration of the available facts and circumstances. As of December 31, 2018 and 2017, theCompany does not have any significant uncertain tax positions.The Company is required to estimate income taxes in each of the jurisdictions in which it operates.The Company’s reserves related to taxes are based on a determination of whether and how much of a tax benefit taken by the Company in its taxfilings or positions is more likely than not to be realized. As of December 31, 2018, the Company had no unrecognized tax benefits or relatedinterest and penalties accrued.In the United States, on December 22, 2017, the “Tax Cuts and Jobs Act” (the “Act”) was signed into law. Substantially all of the provisions ofthe Act are effective for taxable years beginning after December 31, 2017. The Act includes significant changes to the Internal Revenue Code of1986 (as amended, the “Code”), including amendments which significantly change the taxation of individuals, and business entities. The Actcontains numerous provisions impacting the Company, the most significant of which reduces the Federal corporate statutory tax rate from 34% to21%.The staff of the U.S. Securities and Exchange Commission (“SEC”) has recognized the complexity of reflecting the impacts of the Act, and onDecember 22, 2017 issued guidance in Staff Accounting Bulletin 118 (“SAB 118”), which clarifies accounting for income taxes under ASC 740if information is not yet available or complete and provides for up to a one year period in which to complete the required analyses andaccounting (the measurement period). SAB 118 describes three scenarios (or “buckets”) associated with a company’s status of accounting forincome tax reform: (1) a company is complete with its accounting for certain effects of tax reform, (2) a company is able to determine a reasonableestimate for certain effects of tax reform and records that estimate as a provisional amount, or (3) a company is not able to determine a reasonableestimate and therefore continues to apply ASC 740, based on the provisions of the tax laws that were in effect immediately prior to the Act beingenacted.The various provisions under the Act deemed most relevant to the Company have been considered in preparation of its financial statements as ofDecember 31, 2017. The Company had made a reasonable estimate for certain effects of tax reform and had recorded provisional amounts as partof its income tax provision. To the extent that clarifications or interpretations materialized in the future that would impact upon the effects of theAct incorporated into the December 31, 2017 financial statements, those effects would have been reflected in the future as or if theymaterialize. As of December 31, 2018, the Company has completed its accounting for all of the tax effects of the Act. Based on the additionalanalysis performed, no adjustments to the provisional amounts were made in the reporting period which had an impact to the tax provision orconsolidated financial statements.Research and DevelopmentResearch and development costs are charged to expense as incurred. These costs include, but are not limited to, employee-related expenses,including salaries, benefits and travel of the Company’s research and development personnel; expenses incurred under agreements with contractresearch organizations and investigative sites that conduct clinical and preclinical studies and manufacture the drug product for the clinicalstudies and preclinical activities; facilities; supplies; rent, insurance, certain legal fees, share-based compensation, depreciation, other costsassociated with clinical and preclinical activities and regulatory operations and acquisition of in-process research and development write-offs.Refundable research and development credits / tax credits received are recorded as an offset to these costs.Costs for certain development activities, such as Company funded outside research programs, are recognized based on an evaluation of theprogress to completion of specific tasks with respect to their actual 147Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS costs incurred. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of costsincurred, and are reflected in the consolidated financial statements as prepaid or accrued research and development expense, as the case may be.Share-Based Compensation ExpenseOptionsThe Company grants share options to employees, non-employee members of the Company’s board of directors and non-employee consultants ascompensation for services performed. Employee and non-employee members of the board of directors’ awards of share-based compensation areaccounted for in accordance with ASC 718, Compensation - Stock Compensation, or ASC 718. ASC 718 requires all share-based payments toemployees and non-employee directors, including grants of share options, to be recognized in the Statement of Operations and ComprehensiveLoss based on their grant date fair values. The grant date fair value of share options is estimated using the Black-Scholes option valuation model.Using this model, fair value is calculated based on assumptions with respect to (i) the fair value of the Company’s Ordinary Shares on the grantdate; (ii) expected volatility of the Company’s Ordinary Share price, (iii) the periods of time over which employees and members of theCompany’s board of directors are expected to hold their options prior to exercise (expected term), (iv) expected dividend yield on the Company’sOrdinary Shares, and (v) risk-free interest rates.As there had been no public market for the Company’s Ordinary Shares until the Company’s IPO on June 7, 2018, the estimated fair value of theOrdinary Shares until that time had been determined by the Company’s board of directors as of the date of each option grant, with input frommanagement, considering the most recently available third-party valuations of Ordinary Shares and the board of directors’ assessment ofadditional objective and subjective factors that it believed were relevant and which may have changed from the date of the most recent valuationthrough the date of the grant. The fair value of Ordinary Shares after the Company’s IPO was determined based upon the closing share price onthe date of grant.The third party estimated the fair value of the equity using a special case of the market approach known as the backsolve method. The backsolvemethod was used to solve for the implied total equity value based on the Company’s recent Series C financing round. Consideration was given tothe rights and preferences of each of the Company’s classes of equity and the expected time to a liquidity event. An option pricing allocationmethod, or OPM, was selected to allocate the total equity value. The OPM treats ordinary shares and preferred shares as call options on the totalequity value of a company, with exercise prices based on the value thresholds at which the allocation among the various holders of a company’ssecurities changes. Under this method, the Ordinary Shares have value only if the funds available for distribution to shareholders exceeded thevalue of the Preferred Shares’ liquidation preference at the time of the liquidity event, such as a strategic sale or a merger. These third-partyvaluations resulted in a valuation of the Company’s Ordinary Shares of $7.57, $2.64 and $5.63 per share as of December 31, 2016, September 15,2017 and December 31, 2017, respectively.The assumptions underlying these valuations represented management’s best estimate, which involved inherent uncertainties and the applicationof management’s judgment. As a result, if the Company had used different assumptions or estimates, the fair value of its Ordinary Shares and itsshare-based compensation expense could have been materially different.Since the Company’s Ordinary Shares had not been traded on a public exchange prior to the Company’s IPO and have only been traded on apublic exchange for a short period of time since the Company’s IPO, the Company believes that it does not have sufficient company-specificinformation available to determine the expected term based on its historical data. As a result, the expected term of share options granted to 148Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS employees and members of the Company’s board of directors is determined using the average of the vesting period and contractual life of theoption, an accepted method for the Company’s option grants under the SEC’s Staff Accounting Bulletin No. 107 and No. 110, Share-BasedPayment.Similarly, the Company believes that its future volatility will differ materially during the expected term from the volatility that would becalculated from its historical share prices to date. Consequently, expected volatility is based on an analysis of guideline companies inaccordance with ASC 718. The expected dividend yield is zero as the Company has never paid dividends and does not currently anticipatepaying any in the foreseeable future. Risk-free interest rates are based on quoted U.S. Treasury rates for securities with maturities approximatingthe option’s expected term.The Company accounted for options granted to non-employee consultants under ASC 505-50, Equity-Based Payments to Non-Employees, orASC 505-50. As such, the Company estimates the fair value of each such option using the Black-Scholes model, with the expected term of shareoptions granted to non-employees initially equal to the options’ maximum contractual life of ten years, at issuance. On each subsequentreporting date until performance is complete, the Company revalued all outstanding options granted to non-employee consultants during thevesting period of each tranche. Under ASC 505-50, upon re-measurement of each award, income or expense is recognized during its vesting term.Compensation cost relating to awards with service-based graded vesting schedules is recognized as general and administrative and research anddevelopment expenses in the consolidated statements of operations and comprehensive loss using the straight-line method.On July 1, 2018, the Company early adopted ASU 2018-07, Compensation—Stock Compensation (Topic 718): Improvements to NonemployeeShare-Based Payment Accounting (“ASU No. 2018-07”) which simplifies the accounting for share-based payments granted to nonemployees forgoods and services. The ASU supersedes ASC 505-50 and expands the scope of ASC 718 to include all share-based payment arrangementsrelated to the acquisition of goods and services from both nonemployees and employees. The adoption of ASU No. 2018-17 did not have amaterial effect on the consolidated financial statements.Restricted SharesIn connection with certain service agreements and research agreements, the Company has granted restricted Ordinary Shares as compensation.The Ordinary Shares are recognized in the consolidated statements of operations and comprehensive loss based on their grant date fair values.Compensation cost relating to share grants with service-based graded vesting schedules is recognized based on the vesting schedule.LeasesThe Company recognizes rent expense for operating leases on a straight-line basis over the term of the lease, beginning on the date the Companytakes possession of the property. Rent expense includes the base amounts stated in the lease agreement as well as the effect of reduced or free rentand rent escalations. At lease inception, the Company determines the lease term by assuming the exercise of those renewal options that arereasonably assured because of the significant economic penalty that exists for not exercising those options. The expected lease term is one of thefactors used to determine whether a lease is classified as an operating or capital lease and is used to calculate the straight-line rent expense. Thedifference between the cash paid to the landlord and the amount recognized as rent expense on a straight-line basis is included in deferred rentand classified within long-term liabilities. Lease incentives made by landlords to or on behalf of the Company for leasehold improvements arerecorded as deferred rent and classified as long-term liabilities. 149Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS Asset Retirement ObligationAccounting for Asset Retirement Obligations requires legal obligations associated with the retirement of long-lived assets to be recognized at fairvalue when incurred and capitalized as part of the related long-lived asset. In the absence of quoted market prices, we estimate the fair value ofour asset retirement obligations using Level 3 present value techniques, in which estimates of future cash flows associated with retirementactivities are discounted using a credit-adjusted risk-free rate. Asset retirement obligations currently reported on our Consolidated BalanceSheets were measured during a period of historically low interest rates. The impact on measurements of new asset retirement obligations usingdifferent rates in the future may be significant.The Company uses estimates to determine the amount of the asset retirement obligations at the end of the lease term and discounts such assetretirement obligations using an estimated discount rate. Interest on the discounted asset retirement obligation is amortized over the term of thelease using the effective interest method and is recorded as interest expense in the consolidated statements of operations and comprehensive loss.The change in asset retirement obligations is as folows: For the year endedDecember 31, 2018 2017 Balance at beginning of year $178,419 $221,254 Inception of asset retirement obligation 69,286 — Amortization of interest (38,301) 19,313 Change in estimate (99,090) (75,011) Effects of exchange rate 17,805 12,863 Balance at end of year $128,119 $178,419 Other Comprehensive Income (Loss)Other comprehensive income (loss) is defined as the change in equity of a business enterprise during a period from transactions and other eventsand circumstances from non-owner sources. The only component of other comprehensive income (loss) impacting the Company is foreigncurrency translation.Foreign CurrenciesThe Company’s consolidated financial statements are presented in U.S. dollars, the reporting currency of the Company. The financial positionand results of operations of Meira UK II and Meira B.V. are measured using the foreign subsidiaries’ local currency as the functional currency.Meira UK II’s cash accounts holding U.S. dollars are remeasured based upon the exchange rate at the date of remeasurement with the resultinggain or loss included in the consolidated statements of operations and comprehensive loss. Expenses of such subsidiaries have been translatedinto U.S. dollars at average exchange rates prevailing during the period. Assets and liabilities have been translated at the rates of exchange on theconsolidated balance sheets dates. The resulting translation gain and loss adjustments are recorded directly as a separate component ofshareholders’ equity and as other comprehensive loss on the consolidated statements of operations and comprehensive loss.Segment InformationManagement has concluded it has a single reporting segment for purposes of reporting financial condition and results of operations. 150Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS The following table summarizes non-current assets by geographical area: December 31,2018 December 31,2017 United States $454,568 $436,463 United Kingdom 21,788,130 13,942,642 $22,242,698 $14,379,105 Accounting Pronouncements Recently AdoptedIn June 2018, the FASB issued ASU 2018-07, Compensation—Stock Compensation (Topic 718): Improvements to Nonemployee Share-BasedPayment Accounting, which simplifies the accounting for share-based payments granted to nonemployees for goods and services. The ASUsupersedes ASC 505-50 and expands the scope of ASC 718 to include all share-based payment arrangements related to the acquisition of goodsand services from both nonemployees and employees. As a result, most of the guidance in ASC 718 associated with employee share-basedpayments, including most of its requirements related to classification and measurement, applies to nonemployee share-based paymentarrangements. ASU 2018-07 generally requires an entity to use a modified retrospective transition approach, with a cumulative-effect adjustmentto retained earnings as of the beginning of the period of adoption, for all (1) liability-classified nonemployee awards that have not been settled asof the adoption date and (2) equity-classified nonemployee awards for which a measurement date has not been established. The guidance isapplicable to public business entities for fiscal years beginning after December 15, 2019 and interim periods within those years. Early adoption ispermitted, and the Company adopted ASU No. 2018-07 on July 1, 2018, which did not have a material effect on the consolidated financialstatements.In January 2017, the FASB issued ASU No. 2017-01, Business Combinations (Topic 805), Clarifying the Definition of a Business, or ASU 2017-01, that clarifies the definition of a business to assist entities with evaluating whether transactions should be accounted for as acquisitions (ordisposals) of assets or businesses. ASU 2017-01 requires an entity to evaluate if substantially all of the fair value of the gross assets acquired ordisposed of is concentrated in a single identifiable asset or a group of similar identifiable assets; if so, the set of transferred assets and activities isnot a business. ASU 2017-01 also requires a business to include at least an input and one substantive process that together significantlycontribute to the ability to create output and removes the evaluation of whether a market participant could replace missing elements. ASU 2017-01 should be applied prospectively and is effective for annual periods beginning after December 15, 2017 and interim periods within thoseannual periods. The adoption of ASU 2017-01 on January 1, 2018 did not have a material effect on the Company’s financial position, results ofoperations or cash flows.In December 2016, the FASB issued ASU No. 2016-20, Technical Corrections and Improvements to Topic 606, Revenue from Contracts withCustomers, or ASU 2016-20, that allows entities not to disclose variable consideration allocated to performance obligations related to either:(1) sales-or usage-based royalties on licenses of intellectual property or (2) variable consideration allocated entirely to a wholly unsatisfiedperformance obligation or to a wholly unsatisfied promise to transfer a distinct good or service that forms part of a single performance obligationwhen certain criteria are met. ASU 2016-20 also requires entities that use any of the new or previously existing optional exemptions to expandtheir qualitative disclosures. It also makes 12 additional technical corrections and improvements to the new revenue standard, ASU 2014-09. Theamendments have the same effective date and transition requirements as ASU 2014-09. The adoption of ASU 2016-20 did not have a materialeffect on its financial position, results of operations or cash flows. 151Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS In November 2016, the Financial Accounting Standards Board, or FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230),Restricted Cash (a consensus of the Emerging Issues Task Force), or ASU 2016-18, which changes the presentation of the cash flow statement toinclude amounts generally described as restricted cash or restricted cash equivalents, together with cash and cash equivalents, when reconcilingthe beginning-of-period and end-of-period amounts shown on the statement of cash flows. ASU 2016-18 also requires additional disclosuresconcerning the nature of the restrictions on cash and cash equivalents and a reconciliation between amounts of cash, cash equivalents andrestricted cash on the balance sheet and statement of cash flows for each period presented. ASU 2016-18 will be applied retrospectively to allperiods presented and is effective for public business entities for fiscal years beginning after December 15, 2017, and interim periods within thosefiscal years. Early adoption is permitted, including adoption in an interim period. Prior to adoption, the Company presented changes in restrictedcash as an operating activity in the statement of cash flows. Upon adoption of ASU 2016-18 on January 1, 2018, such changes are now reflectedin the beginning and ending balances of cash, cash equivalents and restricted cash for all periods presented.In May 2016, the FASB issued ASU No. 2016-12, Revenue from Contracts with Customers (Topic 606), Narrow-Scope Improvements andPractical Expedients, or ASU 2016-12, which amends guidance in the new revenue standard, ASU No. 2014-09 Revenue from Contracts withCustomers (Topic 606), or ASU 2014-09, on collectability, noncash consideration, presentation of sales tax and transition. The amendments inASU 2016-12 are effective for annual reporting periods beginning after December 15, 2017 (i.e., January 1, 2018), including interim periodswithin those reporting periods, which is the same as for ASU 2014-09, as amended by ASU No. 2015-14, Revenue from Contracts with Customers(Topic 606): Deferral of the Effective Date, or ASU 2015-14. In April 2016, the FASB issued ASU No. 2016-10, Revenue from Contracts withCustomers (Topic 606), Identifying Performance Obligations and Licensing, or ASU 2016-10, which clarifies the principle for determiningwhether a good or service is “separately identifiable” from other promises in the contract and, therefore, should be accounted for as a separateperformance obligation. In that regard, ASU 2016-10 requires that an entity determine whether its promise is to transfer individual goods orservices to the customer, or a combined item (or items) to which the individual goods and services are inputs. In addition, ASU 2016-10 categorizes intellectual property, or IP, into two categories: “functional” and “symbolic.” Functional IP has significant standalonefunctionality. All other IP is considered symbolic IP. Revenue from licenses of functional IP is generally recognized at a point in time, whilerevenue from licenses of symbolic IP is recognized over time. ASU 2016-10 has the same effective date and transition requirements as ASU 2014-09, as amended by ASU 2015-14. In March 2016, the FASB issued ASU No. 2016-08, Revenue from Contracts with Customers (Topic 606),Principal versus Agent Considerations (Reporting Revenue Gross versus Net), or ASU 2016-08, which clarifies the implementation guidance onprincipal versus agent considerations contained in ASU 2014-09 by specifying that the determination as to whether an entity that is involved inproviding a good or a service to a customer is a principal or an agent is based upon whether the entity controls the good or the service before it istransferred to the customer. ASU 2016-08 has the same effective date and transition requirements as ASU 2014-09, as amended by ASU 2015-14. The adoption of these ASU’s on January 1, 2018 did not have a material effect on the Company’s financial position, results of operations orcash flows.In January 2016, the FASB issued ASU No. 2016-01, Financial Instruments—Overall (Subtopic 825-10): Recognition and Measurement ofFinancial Assets and Financial Liabilities. The new guidance is intended to improve the recognition and measurement of financial instrumentsby requiring separate presentation of financial assets and financial liabilities by measurement category and form of financial asset (i.e., securitiesor loans and receivables) within the balance sheet or the accompanying notes to the financial statements, eliminating the requirement for publicbusiness entities to disclose the method(s) and significant assumptions used to estimate the fair value that is required to be disclosed for financialinstruments measured at amortized cost within the balance sheet, requiring public business entities to use the exit price 152Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS notion when measuring the fair value of financial instruments for disclosure purposes, requiring equity investments (except those accounted forunder the equity method of accounting, or those that result in consolidation of the investee) to be measured at fair value with changes in fairvalue recognized in net income, and requiring a reporting organization to present separately in other comprehensive income the portion of thetotal change in the fair value of a liability resulting from a change in the instrument-specific credit risk (also referred to as “own credit”) when theorganization has elected to measure the liability at fair value in accordance with the fair value option for financial instruments, among others. InFebruary 2018, the FASB issued ASU No. 2018-03, Technical Corrections and Improvements to Financial Instruments—Overall (Subtopic 825-10): Recognition and Measurement of Financial Assets and Financial Liabilities, which is intended to clarify certain aspects of the guidanceissued in ASU 2016-01. The Company adopted these standards effective January 1, 2018, which had no impact on the Company’s consolidatedfinancial statements.Recent Accounting Pronouncements Not Yet AdoptedIn November 2018, the FASB issued Accounting Standards Update No. 2018-18, Collaborative Arrangements (Topic 808): Clarifying theInteraction between Topic 808 and Topic 606 (“ASU 2018-18”). The standard amends Accounting Standards Codification 808, CollaborativeAgreements and Accounting Standards Codification 606, Revenue from Contracts with Customers, to clarify the interaction betweencollaborative arrangement participants that should be accounted for as revenue under ASC 606. In transactions when the collaborativearrangement participant is a customer in the context of a unit of account, revenue should be accounted for using the guidance in Topic 606. Theamendments in Update No. 2018-18 are effective for fiscal years beginning after December 15, 2019, and interim periods within those fiscalyears. The Company is currently evaluating the new guidance included in ASU 2018-18, but does not expect it to have a material impact on itsconsolidated financial statements.In October 2016, the FASB issued ASU 2016-16, Income Taxes (Topic 740): Intra-Entity Transfers of Assets Other than Inventory, or ASU 2016-16 which requires that an entity recognize the income tax consequences of an intra-entity transfer of assets other than inventory when the transferoccurs. The guidance must be applied using the modified retrospective basis. The Company does not expect the provisions of ASU 2016-16 tohave a material impact on its current financial statements. This update will be effective for the Company at the beginning of fiscal 2019.In February 2016, the FASB issued ASU No. 2016-02, “Leases” (ASC 842). The amended guidance requires lessees to recognize lease liabilitiesand right-of-use assets on the balance sheet for all leases with terms longer than 12 months and provides enhanced disclosures on keyinformation of leasing arrangements. In July 2018, further amendments were issued to clarify how to apply certain aspects of the amended leaseguidance and to address certain implementation issues. The amended guidance is effective for us commencing in the first quarter of 2019. Earlyadoption is permitted. We plan to adopt the amended guidance on the effective date and expect to elect the package of practical expedients. Weexpect the adoption of the amended guidance will materially affect our consolidated balance sheet and that the primary impact will berecognition of minimum commitments at present value of our noncancelable operating leases as lease liabilities and corresponding right-of-useassets. In July 2018, the FASB issued ASU No. 2018-10, which provides narrow amendments to ASU No. 2016-02 to clarify how to apply the rateimplicit in the lease, impairment of the net investment in the lease, lessee reassessment of lease classification, variable payments that depend onan index or rate and certain transition adjustments. In July 2018, the FASB also issued ASU No. 2018-11, which provides targeted improvementsto ASU No. 2016-02 to provide entities the transition option to not apply the standard in the comparative periods presented in the year ofadoption. The Company will adopt the new standard effective January 1, 2019 using the modified retrospective transition method using thepractical expedients model and a discount rate in the range of 8% to 10%, and 153Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS elect to not apply the standard in the comparative periods presented in the year of adoption. We estimate that upon implementation, we willrecord a right-of-use asset between $3.3 million and $3.6 million and a corresponding liability between $3.6 million and $3.8 million. We arecontinuing to evaluate the impact that the amended lease guidance will have on our consolidated financial statements, systems, processes andinternal controls. 3.Asset Acquisition:On October 5, 2018, the Company entered into an agreement to acquire Vector Neurosciences Inc. (“Vector”) pursuant to an Agreement and Planof Merger (the “Merger Agreement”) by and among the Company, Vector, VN Acquisition, Inc., a wholly-owned subsidiary of the Company(“Merger Sub 1”), VN Acquisition 2, Inc., a wholly-owned subsidiary of the Company (“Merger Sub 2”), the Vector stockholders named thereinand the Vector stockholder representative, pursuant to which Merger Sub 1 was merged with and into Vector, with Vector being the survivingcorporation (“Merger 1”) and, immediately following Merger 1, Vector was merged with and into Merger Sub 2, with Merger Sub 2 being thesurviving corporation (together with Merger 1, the “Merger”). As a result of the Merger, Vector is a wholly-owned subsidiary of the Company.The Company’s board of directors, Vector’s board of directors and Vector’s stockholders have, in each case, unanimously approved the Merger,the Merger Agreement and the transactions contemplated by the Merger Agreement. The merger consideration to Vector’s stockholders consistsof 225,000 shares of the Company’s Ordinary Shares as initial merger consideration, consisting of 202,500 shares which were issued at theclosing of the Merger and an additional 22,500 shares to be issued 18 months following the closing, subject to any indemnification claims underthe Merger Agreement (See Note 12).In addition, pursuant to the terms of the Merger Agreement, the Company will issue to Vector’s stockholders additional Ordinary Shares equal toa maximum value of $21,000,000 if specified regulatory milestones are met and will make royalty payments to Vector’s stockholders in anamount equal to a percentage of the value of sales of certain products developed based on the Vector assets, which royalty payments are alsopayable in Ordinary Shares. The number of Ordinary Shares to be issued in connection with such milestones and royalties will be based on thethree-day average closing price of the Company’s Ordinary Shares immediately prior to the date of determination of the value of the payment.The Company determined this transaction represented an asset acquisition as substantially all of the value was in the intellectual property asdefined by ASC 805, Business Combinations (“ASC 805”). The asset acquisition of in process research and development was recorded at a fairvalue of $2,990,250 as of October 5, 2018. The acquired in process research and development was immediately charged to research anddevelopment expense in the consolidated statement of operations and comprehensive loss as of the acquisition date since the Companydetermined that there was no additional alternative use of these assets. Additionally, under ASC 805, the Company determined that as of theacquisition date and as of December 31, 2018, the contingent milestone payments in the aggregate amount of $21,000,000, and royaltypayments have not been resolved and therefore have not been recorded as liability. 154Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS 4.Prepaid Expenses:Prepaid expenses at December 31, 2018 and 2017 consist of the following: December 31,2018 December 31,2017 Insurance $623,314 $163,284 Clinical Trial Costs 373,723 839,644 Research and Development 352,658 624,348 Other 330,233 160,595 Dues and License Fees 169,073 145,594 Rent 88,784 27,778 $1,937,785 $1,961,243 5.Property, Plant and Equipment, net:Property, plant and equipment, net at December 31, 2018 and 2017 consist of the following: December 31,2018 December 31,2017 Leasehold Improvements $11,538,377 $10,873,895 Capitalized Leasehold Interest 7,150,611 — Manufacturing Equipment 3,779,950 2,477,637 Laboratory Equipment 1,485,544 993,409 Computer and Office Equipment 334,525 276,100 Asset Retirement Obligation 113,678 153,133 Furniture & Fixtures 88,660 93,786 24,491,345 14,867,960 Less: Accumulated depreciation (2,477,108) (612,231) $22,014,237 $14,255,729 In February 2016, the Company sublet a manufacturing facility for a term of 5 years, that included an additional 5-year option. This sub-leasewas accounted for as an operating lease. On December 14, 2018, the Company executed a new sub-lease for this manufacturing facility wherebythe sub landlords remaining term of 108 years was assigned to the Company. Under the new sub-lease, the Company paid a one-time fee of£5,250,000 (approximately $6,615,000 assuming a rate of $1.26 per GBP on the date of the acquisition) for the assignment and will no longerpay any base rent for the remaining 108 years. The one-time fee and related transaction costs, in the aggregate amount of £5,613,165(approximately $7,150,611 using a rate of $1.2739 per GBP at December 31, 2018), have been accounted for as a capital lease and recorded asproperty, plant and equipment. The Company determined that the cost of the new sub-lease would be amortized on a straight-line basis over a 25-year estimated useful life.In connection with the above-mentioned lease, the Company estimated that it had an asset retirement obligation at the end of the initial five-yearlease term in the amount of $306,400. The Company discounted the asset retirement obligation using an 8% discount rate and recorded an assetretirement obligation in the amount of $205,659 as of December 31, 2016, which is included in leasehold improvements and was beingdepreciated over the five-year term of the lease (see Note 15). As of December 31, 2017, the Company determined that it was probable that itwould exercise the additional five-year option provided for in the sub-lease. Therefore, the company remeasured the asset retirement obligationusing the remaining eight-year new sub-lease term and recorded a reduction in the asset retirement obligation of $75,011 recorded in 155Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS leasehold improvements. On December 14, 2018, upon execution of the new sub-lease, the Company remeasured the asset retirement obligationusing the remaining 25-year estimated useful life and recorded a reduction in the asset retirement obligation of $99,090 recorded in leaseholdimprovements.In connection with two operating leases entered into in July 2018, the Company estimated that it had asset retirement obligations at the end ofthe eight-year terms in the amount of $140,129. The Company discount the asset retirement obligation using an 8% discount rate and recordedan asset retirement obligation in the aggregate amount of $69,286, which is included in leasehold improvements and is being depreciated overthe eight-year term of the lease.Capitalized leases in the amount of $95,880 are included in computer and office equipment at December 31, 2018 and 2017, and accumulateddepreciation of $62,912 and 34,552 at December 31, 2018 and 2017, respectively.A capitalized lease in the amount of $7,150,611 is included in capitalized leasehold interest at December 31, 2018 and accumulated depreciationof $6,928 at December 31, 2018.Depreciation expense was $2,052,948 and $679,177 for the years ended December 31, 2018 and 2017 respectively. 6.Restricted Cash:The Company is required to maintain a stand-by letter of credit as a security deposit under the ARE-East River Science Park LLC (“ARE”) lease(see Note 15) through the end of the lease term in December 2021, plus three months. The fair value of the letter of credit approximates itscontract value. The Company’s bank requires the Company to maintain a restricted cash balance to serve as collateral for the letter of creditissued to the landlord by the bank. As of December 31, 2018 and 2017, the restricted cash balance for the ARE lease was invested in acommercial money market account.The Company had $123,376 of restricted cash included in long-term assets as of December 31, 2018 and 2017 and is measured using level 1inputs. 7.Accrued Expenses:Accrued expenses at December 31, 2018 and 2017 were comprised of the following: December 31,2018 December 31,2017 Compensation and Benefits $5,731,438 $2,386,903 Clinical Trial Costs 4,013,094 4,859,410 Professional Fees 914,540 231,923 Consulting 821,009 1,220,477 Research and Development 236,271 73,379 Rent 122,770 387,267 Interest 40,800 33,437 Other 111,775 140,148 $11,991,697 $9,332,944 8.Capitalized Leases:In 2015, the Company acquired certain office equipment in the amount of $17,817 under a 3-year lease arrangement. The Company determinedthat the lease should be capitalized since it contained a bargain 156Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS purchase option for the equipment at the end of the lease term. Total payments under the capital lease amounted to $20,502 and had an interestrate of 9.35%.In 2017, the Company acquired additional office equipment in the amount of $78,063 under a 3-year lease arrangement. The Companydetermined that the lease should be capitalized since it contained a bargain purchase option for the equipment at the end of the lease term. Totalpayments under the capital lease will amount to $86,145 and has an interest rate of 6.90%.In December 2018, the Company entered into a sub-lease for the remaining term of 108 years (see Note 5). The Company determined that thelease should be capitalized since the lease term exceeded 75% of the estimated 25-year useful life of the leasehold interest. The Company made aone-time upfront payment for the assignment of the lease and will not have any obligation to make future rent payments. The Companycapitalized as property, plant and equipment, the initial one-time upfront payment and related transaction costs in the aggregate amount of$7,150,611.The following is a schedule, by year, of future minimum lease payments under the capital leases together with the present value of the netminimum lease payments as of December 31, 2018: 2019 28,715 2020 7,179 Total minimum lease payments 35,894 Less: amount representing interest (1,598) Present value of net minimum lease payments 34,296 Less: current portion (27,199) Obligations under capital lease, less current portion $7,097 9.Notes PayableOn October 26, 2017, in connection with an amendment and termination of a lease, the Company issued a promissory note in the amount of$1,442,009 to ARE, the landlord and also a related party (see Note 15). The note bears interest at the rate of 5% per annum and was due onDecember 31, 2018. However, if the Company had sufficient liquidity, as defined in the note, then the note, including accrued interest, wouldbecome due and payable at that time. In accordance with the sufficient liquidity provision, the Company repaid the note, plus accrued interest, inthe amount of $1,472,433 during the three-month period ended March 31, 2018. The Company recorded interest expense in the consolidatedstatements of operations and comprehensive loss in connection with the note in the amount of $17,386 and $13,037 for the years endedDecember 31, 2018 and 2017, respectively. 10.Collaboration AgreementOn October 16, 2018, the Company entered into a research collaboration agreement with Janssen Pharmaceuticals, Inc., (“Janssen”), to developregulatable gene therapy treatment using the Company’s proprietary riboswitch technology. As part of the agreement, the Company will use itsproprietary riboswitch technology to engineer regulatable gene therapy constructs encoding proprietary gene sequences from Janssen.Upon execution of the agreement, Janssen paid the stage 1 fee in the amount of $658,667 and such payment was recorded as deferred researchfunding. The stage 1 fee is being amortized over the estimated research term of eight months. During the year ended December 31, 2018, theCompany amortized $224,576 of the deferred research funding, which was recorded as an offset to research and development expenses. Deferredresearch funding in the amount of $434,091 is included as other current liabilities on the consolidated balance sheet at December 31, 2018. 157Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS 11.Share-Based Compensation2018 and 2016 Equity Incentive PlansThe Company’s 2018 Incentive Award Plan and 2016 Equity Incentive Plan (the “Plans”), were adopted by the Company’s board of directors andshareholders. Under the Plans, the Company has granted share options to selected officers, employees and non-employee consultants. TheCompany’s board of directors administer the Plans. Options granted under the Plans have a maximum contractual term of ten years. Optionsgranted generally vest 25% on the first anniversary date of grant and the balance ratably over the next 36 months. Options granted to directorsgenerally vest on the first anniversary date of grant. Upon the adoption of the 2018 Incentive Award Plan, the Company ceased issuing awardsunder the 2016 Equity Incentive Plan.A summary of the Company’s share option activity related to employees, non-employee members of the board of directors and non-employeeconsultants as of and for the years ended December 31, 2018 and 2017 is as follows:STOCK OPTION TABLE Number ofOptions Weighted-AverageExercisePrice AggregateInstrinsicValue Outstanding at December 31, 2016 333,660 $7.72 Granted 611,933 3.73 Exercised (1,288) (7.57) Expired — — Forfeited (5,668) (7.72) Outstanding at December 31, 2017 938,637 $5.12 Granted 2,334,285 8.63 Exercised — — Expired — — Forfeited (10,557) (5.51) Outstanding at December 31, 2018 3,262,365 $7.64 $6,903,313 Weighted average remaining contractual life of options outstanding as ofDecember 31, 2017 (yrs) 9.09 Weighted average remaining contractual life of options outstanding as ofDecember 31, 2018 (yrs) 9.24 Options exercisable at December 31, 2017 186,394 $7.72 Options exercisable at December 31, 2018 535,241 $5.79 Weighted average remaining contractual life of options exercisable as of December 31,2017 (yrs) 8.21 Weighted average remaining contractual life of options exercisable as of December 31,2018 (yrs) 7.88 The total fair value of options vested during the years ended December 31, 2018 and 2017 was $1,387,607 and $898,699, respectively. 158Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS During the years ended December 31, 2018 and 2017, the Company granted 2,334,285 and 611,933 share options, respectively. The grant datefair values of the stock options granted were estimated using the Black-Scholes option valuation model with the following ranges of assumptions(see Note 2): 2018 2017Risk-free interest rate 2.32% - 2.98% 2.28% - 2.51%Expected volatility 90% 80%Expected dividend yield 0% 0%Expected life (in years) 5.5 - 10.0 5.5 - 10.0As of December 31, 2018 and 2017, the total compensation expense relating to unvested options granted that had not yet been recognized was$17,415,098 and $987,413, respectively which is expected to be realized over a period of 4.0 and 3.42 years, respectively. The Company willissue shares upon exercise of options from Ordinary Shares reserved.The weighted average grant date fair value of options granted during the years ended December 31, 2018 and 2017 was $6.53 and $3.10,respectively.Restricted Ordinary SharesIn 2015, in connection with certain service and consulting agreements, certain employees and a consultant were awarded an aggregate of867,935 restricted Ordinary Shares of the Company. Such shares were subject to forfeiture over a three-year service period. The shares granted tothe consultant and employees were valued at $7.72 and $7.76 per share, respectively, and were included in loss from operations over the requisiteservice period. As of December 31, 2018, all such shares are no longer subject to forfeiture as the three-year service period has been completed.On June 7, 2018, 1,306,348 restricted Ordinary Shares, which represented 5% of the fully-diluted outstanding shares of the Company as of suchdate, were issued to certain members of senior management in accordance with their employment agreements. One-third of such shares vestedimmediately, with the balance vesting quarterly over the next eight quarters beginning three months after the effectiveness of the Company’sregistration statement on Form S-1 filed with the SEC on June 7, 2018 (the “Registration Statement”). The shares were valued at $15.00 per shareand the related share-based compensation expense, which is recognized over the requisite service period, is included in general andadministrative expenses in the consolidated statements of operations and comprehensive loss. Additionally, under the terms of the employmentagreements, the Company was required to pay the income taxes incurred by the grantees in connection with the grant of those restricted shares.Total compensation expense in connection with the issuance of those restricted Ordinary Shares, in the amount of $20,141,876, of which$10,156,868 was share-based, was recorded as general and administrative expense during the year ended December 31, 2018 (See Note 16).A summary of the restricted Ordinary Shares is as follows: Ordinary Shares $ Value Non-vested at December 31, 2016 386,608 $3,020,191 Vesting during 2017 (280,695) (2,154,330) Non-vested at December 31, 2017 105,913 865,861 Issued during 2018 1,306,348 19,595,220 Vesting during 2018 (759,087) (10,663,471) Non-vested at December 31, 2018 653,174 $9,797,610 159Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS Ordinary SharesOn March 1, 2018, a funding milestone was met under the employment agreements for certain members of senior management. Accordingly, theemployees were issued an aggregate of 550,162 fully vested Ordinary Shares, which represented 3% of the fully-diluted outstanding shares of theCompany as of such date. The shares were recorded as share-based compensation in the amount of $3,096,104. Additionally, under the terms ofthe employment agreements, the Company was required to pay the income taxes incurred by the grantees in connection with the grant of thoseshares. Total compensation expense in connection with the issuance of those Ordinary Shares, in the amount of $6,456,215, of which $3,096,104was share-based, was recorded as general and administrative expense during the year ended December 31, 2018.During the years ended December 31, 2018 and 2017 the Company recognized total share-based compensation expense in the accompanyingconsolidated statements of operations and comprehensive loss as follows: 2018 2017 Research and development $3,372,054 $2,374,899 General and administrative 14,511,500 584,832 Total share based compensation $17,883,554 $2,959,731 The Company does not expect to realize any tax benefits from its share option activity or the recognition of share-based compensation expensebecause the Company currently has net operating losses and has a full valuation allowance against its deferred tax assets. Accordingly, noamounts related to excess tax benefits have been reported in cash flows from operations or cash flows from financing activities for the yearsended December 31, 2018 and 2017. 12.Ordinary Shares, Convertible Preferred C Shares and Shareholders’ Deficit:Ordinary Share Issuances2018As discussed in Note 11, on March 1, 2018, a funding milestone was met under the employment agreements for certain members of seniormanagement. Accordingly, the employees were issued an aggregate of 550,162 fully vested Ordinary Shares.In connection with the Company’s initial public offering, on June 7, 2018, the Company issued 5,000,000 Ordinary Shares at an offering price of$15.00 per share for gross proceeds of $75,000,000, excluding offering costs of $9,807,622.Also, as discussed in Note 11, on June 7, 2018, upon the effectiveness of the Company’s Registration Statement, 1,306,348 restricted OrdinaryShares, which represented 5% of the fully-diluted outstanding shares of the Company as of such date, were issued to certain members of seniormanagement in accordance with their employment agreements. One-third of such shares vested immediately, with the balance vesting quarterlyover the next eight quarters.On October 5, 2018, in connection with an acquisition, the Company issued 202,500 Ordinary Shares with an additional 22,500 shares to beissued 18 months following the closing, subject to any indemnification claims under the merger agreement.2017On July 31, 2017, the Company issued 1,288 Ordinary Shares in connection with the exercise of an option.On August 16, 2017, the Company issued 6,441 Ordinary Shares in connection with a research agreement. 160Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS Convertible Preferred C SharesIssuances2018During the year ended December 31, 2018, the Company issued 5,425,124 Preferred Shares at an offering price of approximately $10.48 per sharefor gross proceeds of $56,849,611, excluding offering costs of $690,473.Also, during the year ended December 31, 2018, the Company issued 129,419 Preferred Shares in lieu of payment of accounts payable in theaggregate amount $1,355,097 to certain vendors.On March 15, 2018, the Company issued 13,360 Preferred Shares in connection with a license agreement.On June 7, 2018, upon effectiveness of the Company’s Registration Statement on Form S-1, all of the 11,501,432 outstanding Preferred Shareswere automatically converted into 11,501,432 Ordinary Shares. In connection with the conversion of the Preferred Shares, $664,718 ofunaccredited financing costs were fully accreted.2017During the year ended December 31, 2017, the Company issued 9,739 Preferred Shares at an offering price of $20.96 per share and 1,598,418Preferred Shares at an offering price of $10.48 per share for gross proceeds of $16,854,656, excluding offering costs of $98,804. The net proceedsof the offering were used for working capital, research and development and general corporate purposes.On November 2, 2017, a note payable to a related party in the amount of $2,500,000 was converted at the rate of $10.48 per share, into 238,579Preferred Shares (see Note 15).WarrantsIn connection with the issuance of 715,737 Preferred Shares on September 21, 2017, at an offering price of $10.48 per share, the Company issuedwarrants to purchase 695,696 Preferred Shares at an exercise price of $10.48 per share. The warrants expired on the first of the following to occur: (i)an Asset Sale; (ii)a Qualified IPO; (iii)a Share Sale; (iv)the winding up of the Company; or (v)On the third anniversary of the date of issuanceThe Black-Sholes value of the warrants in the amount of $1,660,500 was accounted for as a warrant liability and a discount to the PreferredShares at the time of issuance and were being accreted over the expected term of the Preferred Shares (see Note 2).The Black-Scholes value of the warrants in the amount of $553,500 was recorded as a warrant liability and charged to convertible noteinducement expense within the statement operations and comprehensive loss at the time of issuance.Both of the warrants were revalued under the Black-Scholes valuation model at December 31, 2017, which resulted in an increase of the warrantliability in the amount of $465,633, which was charged to change in fair value of warrant liability within the statement of operations andcomprehensive loss. 161Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS The warrant liability at December 31, 2017 was $2,679,633.On June 5, 2018, all of the outstanding warrants to purchase 927,594 Preferred Shares at an exercise price of approximately $10.48 per share wereexercised for aggregate cash proceeds of $9,720,000. 13.Net Loss per ShareThe Company computes net loss per share in accordance with ASC 260-10, Earnings per Share (see Note 2).Basic and diluted net loss per share is computed as follows: 2018 2017 Net loss—basic and diluted $(82,865,874) $(31,044,535) Accretion of Preferred Shares financing costs (1,806,512) (191,963) Accretion of warrant — (615,000) Adjusted net loss—basic and diluted $(84,672,386) $(31,851,498) Weighted-average ordinary shares outstanding: Basic and Diluted 18,948,520 8,572,315 Net loss per share: Basic and Diluted $(4.47) $(3.72) The following securities are considered to be Ordinary Share equivalents, but were not included in the computation of diluted net loss perOrdinary Share because to do so would have been anti-dilutive: December 31,2018 December 31,2017 Preferred Shares — 5,005,934 Restricted Ordinary Shares subject to forfeiture 653,174 105,913 Stock options 3,262,365 938,637 Warrants — 927,594 3,915,539 6,978,078 14.Income Taxes:Since the Company has recurring losses and a valuation allowance against deferred tax assets, there is no tax expense (benefit) for the year ended2017. For the year ended December 31, 2018, the Company recognized a tax benefit of $(474,391).The subsidiaries each file separate tax returns in their respective tax jurisdictions.As of December 31, 2018, the Company had federal and state net operating loss (“NOL”) carryforwards in the United States of approximately$14,210,000 and $14,155,000, respectively, and in the United Kingdom of approximately $94,100,000, which are available to reduce futuretaxable income. The U.S. federal and state NOL carry forwards incurred prior to January 1, 2018 in the amount of approximately $6.8 million and$6.7 million, respectively, will begin to expire in 2036. The U.S. NOL incurred after December 31, 2018 and the U.K. NOL will be indefinitelycarried forward. Also, as of December 31, 2018, the Company had orphan drug and research and development credits in the U.S. in the amount of$1,134,000 which will begin to expire in 2036. The NOL carry forwards are subject to review and possible adjustment by the U.S., U.K. and statetax authorities. NOL carry forwards may become subject to an annual limitation in the event of certain cumulative changes in the ownershipinterest of significant shareholders, as defined under 162Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS Sections 382 Internal Revenue Code, as well as CTA 2010 Part 14 under the UK tax rules. This could limit the amount of NOLs that theCompany can utilize annually to offset future taxable income or tax liabilities. As of December 31, 2018, the Company has performed such ananalysis and determined that there were no limitations in the U.K. However, for U.S. purposes the Company determined that a change ofownership occurred in April 2016. The Company is still in the process of determining the annual limitation on losses that occurred prior to April2016. Subsequent ownership changes and proposed future changes to the UK (or US) tax rules in respect of the utilization of losses carriedforward may further affect the limitation in future years, if any. Additionally, the Company has not undertaken a study on the completeness of theU.S. orphan drug and research and development credit.The Company’s pre tax earnings from the United Kingdom and United States locations are as follows: December 31,2018 December 31,2017 United Kingdom $(73,359,977) $(26,458,625) United States (9,980,287) (4,585,910) $(83,340,264) $(31,044,535) The Company is subject to the corporate tax rate in the U.K. as a Limited U.K. corporation.The following table summarizes a reconciliation of income tax benefit compared with the amounts at the U.K. statutory income tax rate: December 31,2018 December 31,2017 Statutory rate (15,834,650) 19.00% (5,976,073) 19.25% Permanent differences—other 1,438,934 -1.73% 654,648 -2.11% RTP and other adjustments 387,509 -0.46% (152,948) 0.49% U.K. tax credit 1,707,489 -2.05% 539,136 -1.74% U.S. tax credit (436,250) 0.52% (363,665) 1.17% Foreign tax rate differential (171,693) 0.21% (673,619) 2.17% State and local rate, net of federal tax (1,159,522) 1.39% (446,683) 1.44% UK Rate Change (17% at expected DTA turn) 1,104,863 -1.33% 482,351 -1.55% U.S. state rate change (6,496) 0.01% 993,998 -3.20% Change in valuation allowance 12,495,426 -14.99% 4,942,855 -15.92% Actual income tax benefit effective tax rate (474,391) 0.57% — 0.00% 163Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS The Expense/(Benefit) for income taxes from continuing operations consists of the following: December 31,2018 December 31,2017 Current Tax Expense/(Benefit) United Kingdom — — United States — — Total Current — — Deferred Tax Expense/(Benefit) United Kingdom $(8,888,096) $(3,759,109) United States (3,606,275) (1,183,746) Total Deferred (12,494,371) (4,942,855) Change in Valuation Allowance 12,019,880 4,942,855 Total Income Tax Expense/(Benefit) $(474,391) $— Income tax (benefit) expense for each year is allocated to continuing operations, discontinued operations, extraordinary items, othercomprehensive income, the cumulative effects of accounting changes, and other charges or credits recorded directly to shareholders’ equity.ASC 740-20-45 Income Taxes, Intraperiod Tax Allocation, Other Presentation Matters includes an exception to the general principle ofintraperiod tax allocations. The codification source states that the tax effect of pretax income or loss from continuing operations generally shouldbe determined by a computation that considers only the tax effects of items that are included in continuing operations. The exception to thatincremental approach is that all items (i.e. other comprehensive income, discontinued operations, etc.) be considered in determining the amountof tax benefit that results from a loss from continuing operations and that benefit should be allocated to continuing operations. That is, when acompany has a current period loss from continuing operations, management must consider income recorded in other categories in determiningthe tax benefit that is allocated to continuing operations. This includes situations in which a company has recorded a full valuation allowance atthe beginning and end of the period, and the overall tax provision for the year is zero. The intraperiod tax allocation is performed once theoverall tax provision has been computed and allocates that provision to various income statement (continuing operations, discontinuedoperations), other comprehensive income and balance sheet captions. While the intraperiod tax allocation does not change the overall taxprovision, it results in a gross-up of the individual components. The level of application has been applied on the group level.As the Company experienced a net loss from operations for the year ended December 31, 2018 and other comprehensive income from foreigncurrency translation adjustments, the Company has allocated income tax expense against the components of other comprehensive income in2018 using a 17% effective tax rate. Income tax benefit for the year ended December 31, 2018 includes a benefit of $(474,391) due to therequired intraperiod tax allocation. Conversely, other comprehensive income for the year ended December 31, 2018 includes income tax expenseof $474,391. 164Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS Deferred Tax Assets/(Liabilities) December 31, 2018 Total UK US Deferred Tax Assets: Net operating loss carryforwards $20,646,185 $16,000,329 $4,645,856 Other 1,414,690 (46,604) 1,461,294 Tax Credit 1,133,656 — 1,133,656 Deferred tax assets 23,194,531 15,953,725 7,240,806 Less: valuation allowance (23,194,531) (15,953,725) (7,240,806) Net deferred tax asset $— $— $— December 31, 2017 Total UK US Deferred Tax Assets: Net operating loss carryforwards $9,462,690 $6,909,754 $2,552,937 Other 539,008 152,554 386,454 Tax Credit 697,406 — 697,406 Deferred tax assets 10,699,105 7,062,308 3,636,797 Less: valuation allowance (10,699,105) (7,062,308) (3,636,797) Net deferred tax asset $— $— $— ASC 740 requires a valuation allowance to reduce the deferred tax assets reported if, based on the weight of available evidence, it is more likelythan not that some portion or all of the deferred tax assets will not be realized. After consideration of all the evidence, both positive and negative,the Company has recorded a full valuation allowance against its deferred tax assets at December 31, 2018 and 2017 because the Company’smanagement has determined that is it more likely than not that these assets will not be fully realized.Changes to the U.K. and U.S. corporation tax rates have been announced which will impact future accounting periods. In his budget of July 8,2015, the Chancellor of the Exchequer announced a reduction in the U.K. corporation tax rate to 19% for the financial year beginning April 1,2017 and a further reduction to 18% for the financial year beginning April 1, 2020. These changes received Royal Assent on November 18, 2015.The U.K. Finance Act 2016 provides for a further reduction in the corporation tax rate to 17% for the Financial Year beginning April 1,2020. This change was enacted on September 15, 2016. As the Company does not expect to be able to utilize its NOL’s in the U.K. prior to itsfinancial year beginning on January 1, 2021, if at all, the deferred tax has been calculated using a tax rate of 17%.In the United States, the corporation tax rate was reduced to 21% for the financial year beginning January 1, 2018. As these changes were enactedprior to the December 31, 2017 balance sheet date, deferred tax has been calculated accordingly in these consolidated financial statements,which represented a decrease in the prior years deferred tax assets of approximately $994,000.The Company will recognize interest and penalties related to uncertain tax positions in income tax expense. As of December 31, 2018 and 2017,the Company had no accrued interest or penalties related to uncertain tax positions and no amounts have been recognized in the Company’sstatements of operations and comprehensive loss.The Company files income tax returns in the United States, United Kingdom and the Netherlands, and various state jurisdictions. For the US, thestatute of examination is open for tax years 2015, 2016, 2017 and 2018. For the UK and the Netherlands, the statute of examination is open fortax years 2017 and 2018. 165Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS MeiraGTx Holdings plc is a UK tax resident with no earnings in its foreign subsidiaries and the Company does not expect any temporary basisdifference in its investment in these subsidiaries to reverse in the foreseeable future. Therefore, the Company has not recorded deferred taxes onthe outside basis difference in its foreign subsidiaries. It is not probable to compute the amounts, if any. 15.Related Party Transactions:Transition Services AgreementEffective April 24, 2015, the Company entered into a transition services agreement (the “TSA”) with Kadmon, which owned 12.9% of theCompany at December 31, 2018, whereby Kadmon would provide office and laboratory facilities as well as certain other personnel supportactivities to the Company. Under the agreement, the Company is charged for (i) rent based upon the square footage of the office and laboratoryfacilities used by the Company (ii) other personnel support activities based upon the hours of the personnel providing the support activities, and(iii) and other direct costs incurred by Kadmon on behalf of the Company, plus a 7% administrative fee. The TSA terminated on April 24, 2018and the Company is currently leasing office space on a month to month basis from Kadmon.During the years ended December 31, 2018 and 2017, the Company incurred the following charges in connection with the TSA which areincluded in loss from operations: 2018 2017 Rent $557,698 $548,229 Personnel 6,493 39,721 Other 6,334 5,983 Total charges incurred $570,525 $593,933 During the year ended December 31, 2018 and 2017, the Company made cash payments totaling $1,431,555 and $275,941, respectively.The amount due to Kadmon at December 31, 2018 and 2017 is $0 and $861,030, respectively, and is disclosed as Due to Kadmon on theCompany’s consolidated balance sheets.Research AgreementEffective October 23, 2016, the Company entered into a four-year master services agreement with UCL Consultants Limited, an entity affiliatedwith University College of London (“UCL”), which is a shareholder of the Company. Pursuant to the agreement, UCL Consultants Limitedprovides pre-clinical research and development under the direction of the Company. In connection with the agreement, the Company issuedseveral work orders during the years ended December 31, 2016 and 2017 in the aggregate amounts of £1,402,202, or approximately $1,885,000,based upon the average exchange rates during the years ended December 31, 2016 and 2017, respectively. Either party may terminate theagreement by giving 30 days written notice. Total research and development expenses under this agreement for the years ended December 31,2018 and 2017 was approximately $636,000 and $538,000, respectively. Future obligations under the agreement equal £612,382, orapproximately $779,685, through October 2020.The amount due to UCL under the master services agreement at December 31, 2018 and 2017 is $389,101 and $775,315, respectively and isincluded in accounts payable and accrued expenses on the Company’s consolidated balance sheets.Effective September 1, 2016, the Company entered into a manufacturing and drug supply agreement with UCL. Pursuant to the agreement, UCLmanufactured materials for the Company’s clinical trials under the 166Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS direction of the Company. The agreement was terminated in January 2018. Total research and development expenses under this agreement for theyears ended December 31, 2018 and 2017 was approximately $0 and $1,904,352, respectively.The amount due to UCL under the manufacturing and drug supply agreement at December 31, 2018 and 2017 is $0 and $2,466,142, respectivelyand is included in accrued expenses on the balance sheet.LeasesJuly 2016 LeaseEffective July 1, 2016, the Company entered into a non-cancellable operating lease for laboratory and related office facilities in New York withARE, an entity that is under common control by an entity that is a minority shareholder of the Company and whose executive chairman andfounder is a director of the Company. The lease provides for monthly base rent and property management fees, including rent escalations and rentholidays, plus operating expenses during the lease term, which expires on December 31, 2021. The Company records monthly rent expense on astraight-line basis from July 1, 2016 through December 31, 2021. As of December 31, 2018 and 2017, the balance of deferred rent, representingthe difference between cash rent paid and straight-line rent expense, was $201,264 and $231,276, respectively.Total rent expense under this operating lease was $487,555 and $487,559 for the years ended December 31, 2018 and 2017, respectively. As ofDecember 31, 2018, the aggregate future minimum rental payments under this lease are $1,663,952.In connection with the signing of this lease, the Company entered into a standby letter of credit agreement for $122,866, which serves as asecurity deposit for the premises. The standby letter of credit expired on July 7, 2017 and was automatically renewed annually through July 7,2021. This standby letter of credit is secured with restricted cash in a money market account (see Note 6).December 2016 LeaseEffective December 15, 2016, the Company entered into another non-cancellable operating lease with ARE, expiring on October 31, 2032, forlaboratory and office facilities in New York. The lease provided for monthly base rent, including rent escalations, property management fees andrent holidays, plus operating expenses during the lease term. The Company recorded monthly rent expense on a straight-line basis fromDecember 15, 2016 through October 31, 2032. On October 26, 2017, the lease was amended, whereby the lease would terminate on March 31,2018 and only base rent and management fees in the aggregate amount of $563,507 would be due from November 1, 2017 through March 31,2018. Under the amendment, the Company issued a note to ARE in the amount of $1,442,009 (see Note 9), removed the balance of the deferredrent and accrued the future rent payments, all of which were recorded as rent expense at the time of the amendment, in accordance with ASC 420,Exit and Disposal Activities, as the Company had a cease use date as of the date of the amendment.Total rent expense under this operating lease was $0 and $1,660,806 for the years ended December 31, 2018 and 2017, respectively.Convertible Note PayableOn May 1, 2017, the Company issued a convertible note in the amount of $2,500,000 to ARE. The note had an interest rate of 10% per annumand was convertible into Preferred Shares at any time at the option of the holder or would automatically convert into Preferred Shares in the eventof an equity investment by a 167Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS mutually agreed upon institutional investor at a price per share equal to the lowest price paid per share by a purchaser of the Company’s PreferredShares. On November 2, 2017 the note was converted to 238,579 Preferred Shares at $2.70 per share. In accordance with the terms of theconvertible note, the accrued interest in the amount of $145,833 was cancelled.As an inducement to convert the convertible note, the Company issued a warrant to purchase 900,000 Preferred Shares, at an exercise price of$2.70 per share, to the holder of the convertible note, which was expenses in accordance with ASC 470 (see Note 12). 16.Commitments:Operating LeasesIn February 2016, the Company entered into a non-cancellable operating lease, expiring in February 2021, for manufacturing and office facilitiesin London, UK. The lease provides for an additional five-year term at the Company’s option. The lease provides for monthly base rent, plusoperating expenses and real estate taxes during the lease term.Total rent expense under this operating lease was $273,430 and $279,303 for the years ended December 31, 2018 and 2017, respectively.On October 1, 2017, the Company entered into a one-year non-cancellable operating lease, expiring in September 2018, for office and laboratoryfacilities in Leiden, Netherlands. The lease provides for monthly base rent plus operating expenses during the lease term. The lease provides forsuccessive one-year extensions up to a maximum of four extensions. Total rent expense under this operating lease was $9,313 and $5,273 for theyears ended December 31, 2018 and 2017, respectively.In June 2017, the Company entered into two non-cancellable operating leases, expiring in July 2018, for office and laboratory facilities inLondon, UK. The lease provides for monthly base rent, rent holidays plus operating expenses and real estate taxes during the lease term. TheCompany records monthly rent expense on a straight-line basis from June 1, 2017 through July 23, 2018. As of December 31, 2018, the balanceof deferred lease obligation, representing the difference between cash rent paid and straight-line rent expense, was $0. Total rent expense undertheses operating leases was $73,846 and $85,222 for the years ended December 31, 2018 and 2017, respectively.On July 27, 2018 the two leases for office and laboratory facilities in London, UK expired. Effective July 27, 2018, the Company entered intotwo new non-cancellable operating leases for the same office and laboratory facilities in London. The leases provide for annual base rent in theaggregate amount of approximately $363,000, plus operating expenses, through May 31, 2022, at which time the annual base rent will berevalued based on market rates at that time. The leases expire on May 24, 2027. Total rent expense under these operating leases was $148,561and $0 for the years ended December 31, 2018 and 2017, respectively.The aggregate future minimum rental payments under these operating leases are as follows: 2019 $353,782 2020 $350,284 2021 $350,284 2022 $350,284 2023 $350,284 Thereafter $1,196,805 Total future rent payments $2,951,723 168Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS The aggregate future minimum rental payments of all leases, including those discussed in Note 15 are as follows: 2019 $889,465 2020 $904,716 2021 $924,121 2022 $350,284 2023 $350,284 Thereafter $1,196,805 Total future rent payments $4,615,675 Service AgreementsOn April 27, 2015, the Company entered into service agreements with a senior officer and a greater than 5% shareholder of the Company. Underthe terms of the agreements, the employees will receive aggregate compensation of £300,000, which has been increased to a maximum aggregateamount of £410,000 per annum, or approximately $522,000 using exchange rates as of December 31, 2018. The agreements also provide forcontributions to a defined contribution pension plan to be set up by the Company and a discretionary bonus. The agreements may be terminatedat any time by either party by giving twelve-months’ notice, or the Company may terminate the officer’s employment effective immediatelyupon notice, and within 28 days making payment in lieu of notice consisting of a sum equivalent to the officer’s annual salary for the relevantperiod. For the years ended December 31, 2018 and 2017, the Company recorded £1,001,000 and £724,000 or approximately $1,334,000 and$933,000, respectively, using the average exchange rates during the years ended December 31, 2018 and 2017, respectively, in research anddevelopment costs under these agreements. Future obligations to be paid under these agreements equal £150,333, or approximately $192,000,using exchange rates as of December 31, 2018.In connection with the service agreements, on April 24, 2015, the employees were awarded, under a share award agreement (the “Share AwardAgreement”), an aggregate of 2,704,800 restricted Ordinary Shares and 750 B ordinary shares, which B ordinary shares have been converted intoOrdinary Shares, of the Company. Under the Share Award Agreement, such shares are subject to forfeiture ratably over a period of three years ifthe employee’s do not remain an employee or consultant to the Company. The shares were valued at $7.76 per share and, in accordance with ASC718, were charged to operations as share-based compensation ratably over the forfeiture period.Employment AgreementsIn February 2016, the Company entered into three-year employment agreements with certain senior officers of the Company. Under the terms ofthe agreements, which automatically renew for successive one-year terms, the employees will receive annual compensation in the aggregateamount of $710,000, which has been increased to a maximum aggregate amount of $1,075,000. The employment agreements also provide for anannual guaranteed cash bonus targeted at 100% of annual compensation. The agreements also provide for discretionary annual performancebonuses targeted to be not less than 50-60% of the employee’s base salary and grants of restricted stock. In January 2018 the Company’scompensation committee approved a discretionary bonus in the aggregate amount of $1,196,000. This discretionary bonus and the guaranteedbonus for 2017, in the amount of $850,000, were subject to compensation committee approval and meeting certain future funding conditions. OnFebruary 28, 2018, the funding conditions were met. The senior officers were granted their guaranteed and discretionary bonuses for the yearended December 31, 2018, in the aggregate amount of $3,415,000, which was paid in January 2019. 169Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS Additionally, the agreements provided for equity incentives of up to an aggregate maximum of 8.0% of the Company’s fully diluted outstandingshares upon the attainment of certain milestones. On March 1, 2018, a funding milestone was met. Accordingly, the employees were issued anaggregate of 3% of the fully-diluted outstanding shares of the Company as of such date. On June 7, 2018, an additional milestone was met.Accordingly, the employees were issued an aggregate of 5% of the fully-diluted outstanding shares of the Company as of such date (see Note 11).The employees are also entitled to participate in all incentive and deferred compensation and employee benefit programs available to employeesand executive officers of the Company. Future obligations to be paid under these agreements equal $1,258,750, as of December 31, 2018.Consulting and other AgreementsEffective September 28, 2015, the Company entered into a three-year consulting agreement with a consultant to provide ongoing strategicadvice and to serve on the Company’s board of directors. In connection with the agreement, the Company issued 662,910 restricted OrdinaryShares. Under the consulting agreement, such shares were subject to forfeiture ratably over a period of three years if the consultant does notremain a consultant to the Company. The shares were valued at $7.72 per share and were charged to general and administrative expenses uponthe expiration of each forfeiture period. For the years ended December 31, 2018 and 2017, the Company recorded $263,970 and $351,960,respectively, in general and administrative expense under this agreement. There are no future obligations to be paid under the agreement.Research AgreementsOn April 24, 2015, the Company entered into a cooperative research and development agreement (CRADA) with the U.S. Department of Health &Human Services, as represented by the National Institute of Dental and Craniofacial Research (NIDCR) and Institute or Center of the NationalInstitutes of Health (NIH). The CRADA provided for quarterly payments of $21,250 for three years through April 30, 2017 and a cost per patientfor each patient enrolled in the Company’s xerostomia clinical trial. The CRADA was amended on March 25, 2016 to extend the term throughMarch 25, 2021 and to extend the annual payments throughout the revised term. Research and development expenses under the CRADA for theyears ended December 31, 2018 and 2017 were $111,938 and $115,374, respectively. Future obligations to be paid under the CRADA, asamended, through March 25, 2021 equal $191,250.On March 22, 2016, the Company entered into another CRADA with the NIDCR and NIH for the treatment of Sjögren’s syndrome associatedsalivary hypofunction. The CRADA provides for quarterly payments of $104,500 for the first three years of the agreement plus a cost per patientfor each patient enrolled in a clinical trial. The costs associated with years four and five of the Sjögren’s syndrome CRADA will be determined ata later date. Total research and development expenses under this agreement for each of the years ended December 31, 2018 and 2017 were$418,000. There are no future obligations to be paid under the agreement.Effective December 5, 2016, the Company entered into a three-year research collaboration agreement with Cornell University. Pursuant to theagreement, Cornell University provides research and development under the direction of the Company. In connection with the agreement, in July2017, the Company issued 25,000 Ordinary Shares to Cornell University, which were recorded as research and development expenses in theamount of $17,000. The Company amended this agreement, effective June 12, 2017, to add a second three-year research collaboration projectthrough September 2019. The Company further amended this agreement, effective October 18, 2018 to include additional costs related to theresearch. Total research and 170Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS development expenses under this agreement, as amended, for the years ended December 31, 2018 and 2017 were $1,625,152 and $1,029,904,respectively. Future obligations to be paid under the agreement through December 5, 2019 equal $2,002,228.On February 14, 2017, the Company entered into a one-year research collaboration agreement with Cornell University in the amount of$679,473. On August 24, 2017, the agreement was amended to add an additional study in the amount of $182,520. Total research anddevelopment expenses under this agreement for the years ended December 31, 2018 and 2017 were $143,073 and $698,307, respectively.License AgreementsEffective February 4, 2015, the Company entered into an exclusive worldwide license agreement with UCL Business, PLC (“UCL Business”) todevelop up to eight programs using certain ocular gene therapy technology. Under the terms of the agreement, as amended, the Company willpay UCL Business certain sales milestone payments, if achieved, in the aggregate amount of £39.8 million, or approximately $50.7 million usingthe exchange rate at December 31, 2018, and royalties on net sales, as defined upon commercialization. Additionally, the Company isresponsible for all patent prosecution and maintenance costs incurred and will also pay UCL Business an annual maintenance fee of £50,000, orapproximately $64,000, until the first commercial sale of a product. The agreement will terminate upon the later of (i) the last valid claim in arelevant product, (ii) the expiration of regulatory exclusivity to all licensed products, or (iii) the 10th anniversary of the first commercial sale of aproduct. Total research and development expenses under the agreement for the years ended December 31, 2018 and 2017 were $75,073 and$73,250, respectively.Effective July 28, 2017, the Company entered into another exclusive worldwide license agreement with UCL Business for an additional programusing certain ocular gene therapy technology. The Company will pay UCL Business certain milestone payments, royalties and annualmaintenance fees under the same terms and conditions as the license dated February 4, 2015. Total research and development expenses under theagreement for the years ended December 31, 2018 and 2017 were $66,630 and $82,260, respectively.Effective March 15, 2018, the Company entered into another exclusive worldwide license agreement with UCL Business for an additionalprogram using certain ocular gene therapy technology. The Company will pay UCL Business certain milestone payments, royalties and annualmaintenance fees under the same terms and conditions as the license dated February 4, 2015. Total research and development expenses under theagreement for the years ended December 31, 2018 and 2017 were $133,728 and $0, respectively.On September 7, 2018, the Company entered into an exclusive licensing agreement with the National Institutes of Health for worldwide rights toexpanded indications for use of AAV-AQP1 for treatment of xerostomia (dry mouth) and xerophthalmia (dry eye) associated with Sjögren’ssyndrome. This agreement expands the Company’s original exclusive licensing agreement with the NIH for exclusive worldwide rights to AAV-AQP1 that was executed as of November 9, 2017. AAV-AQP1 is currently in Phase 1/2 development for treatment of grade 2 or 3 radiation-induced xerostomia. Total research and development expenses under the agreement for the years ended December 31, 2018 and 2017 were$50,000 and $0, respectively. 171Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS Effective January 1, 2016, the Company entered into an Agreement (“Agreement”) and Plan of Merger to acquire all of the outstanding shares ofBRI-Alzan from the shareholders of BRI-Alzan. In connection with the Agreement, the Company will pay certain development milestonepayments if achieved, in the aggregate amount of $4.5 million, and annual royalty payments on annual net sales following the first commercialsale of any product containing the technology acquired. Total research and development expenses under the agreement for the years endedDecember 31, 2018 and 2017 were $15,000 and $30,000, respectively. 17.Employee Benefit PlansUnited StatesOn January 1, 2017, Meira LLC adopted a defined contribution retirement plan that complies with Section 401(k) of the Internal Revenue Code.All Meira LLC employees over the age of 21 are eligible to participate in the plan after three consecutive months of service. Employees are ableto defer a portion of their pay into the plan on the first day of the month or after the day all age and service requirements have been met. The planprovides for a Company matching contribution. All eligible employees receive an employer matching contribution equal to the lesser of theamount the employee contributes to the plan or 6% of their salary up to the annual IRS limit.United KingdomOn August 1, 2016, Meira UK II adopted a defined contribution group personal pension plan that complies with HM Revenue and Customs(HMRC) for tax relief. All Meira UK II employees are eligible to participate in the plan upon joining the company and providing the requiredservices. All eligible employees, if they elect to join the pension scheme, receive an employer pension contribution equal to 7.5% to 10.0% oftheir pensionable earnings. Currently, employees are not required to contribute, but may make optional contributions up to the annual allowanceHMRC limits.Under the HMRC requirements, current required 6+minimum employer contributions are 5-6% but will rise to 8-9% after April 2019.During the years ended December 31, 2018 and 2017, employer contributions to all plans were $440,368 and $252,700, respectively. 18.Subsequent Events:Management has evaluated subsequent events through the date these financial statements were issued. Based on our evaluation, the followingdisclosures have been made:License AgreementOn January 29, 2019, the Company amended and restated the following agreements: (i) the License Agreement, dated February 4, 2015, asamended, between the Company and UCL Business, Plc (“UCLB”); (ii) the License Agreement, dated July 28, 2017, as amended, between theCompany and UCLB; and (iii) the License Agreement, dated March 15, 2018, between the Company and UCLB to establish new stand-alonelicense agreements for the following inherited retinal disease programs: (a) achromatopsia (“ACHM”) caused by mutations in CNGB3; (b) ACHMcaused by mutations in CNGA3; (c) X-linked retinitis pigmentosa (“XLRP”); and (d) RPE65-mediated IRD.The Company’s obligation to pay UCLB a share of certain sublicensing revenues, as was provided under the February 4, 2015 agreement, hasbeen removed from each of the stand-alone agreements. Each of the stand-alone agreements now reflects terms substantially similar to those ofthe March 15, 2018 agreement. 172Table of ContentsMEIRAGTX HOLDINGS PLC AND SUBSIDIARIESNOTES TO CONSOLIDATED FINANCIAL STATEMENTS Additionally, the new stand-alone agreement related to CNGB3 provides for the Company to pay UCLB an upfront payment of £1,500,000, orapproximately $1,976,000, and issue £1,500,000 of the Company’s ordinary shares.Collaboration AgreementOn January 30, 2019, (the “Agreement Date”), the Company entered into a Collaboration Agreement with Janssen Pharmaceuticals, Inc.(“Janssen”) for the research, development and commercialization of gene therapies for the treatment of inherited retinal diseases (“IRDs”).Under the terms of the agreement, the Company will receive a $100 million cash upfront payment. Janssen and the Company will collaborate todevelop the Company’s current clinical programs in Retinitis Pigmentosa and two genetic forms of Achromatopsia and Janssen has the exclusiveright to commercialize these products globally. The Company will manufacture these products for commercial supply. Janssen will pay 100% ofthe clinical and commercialization costs of the products and the Company is eligible to receive untiered 20 percent royalties on net sales ofproducts and additional development and commercialization milestones of up to $340 million. In addition, the Company and Janssen haveentered into a research collaboration in the area of IRDs, with Janssen paying for the majority of the research costs. Janssen has the right toexclusively license any product coming out of the collaboration at the time of IND. Janssen will then pay 100% of the clinical andcommercialization costs for these products and the Company will receive an untiered royalty on net sales in the high teens as well asdevelopment milestones. In addition, Janssen and the Company have entered into a manufacturing research collaboration to further developprocesses for manufacturing AAV viral vectors in which the costs of the research will be shared.Private PlacementOn February 27, 2018, the Company issued 5,797,102 Ordinary Shares in a private placement for gross proceeds of $80 million, excludingoffering costs of approximately $2.6 million. Johnson & Johnson Innovation – JJDC, Inc., the investment arm of Johnson and Johnson, led theoffering and purchased 2,898,550 of the Ordinary shares issued on the same terms and conditions as the other investors in the offering.In connection with the offering, the Company also entered into a registration rights agreement whereby, promptly following the date on whichthe Company becomes eligible to use a registration statement on Form S-3, but in no event later than July 31, 2019, the Company shall prepareand file a registration statement covering the resale of all of the Registrable Securities, as defined in the agreement. The Company shall usecommercially reasonable efforts to have the registration statement declared effective as soon as practicable. If the registration statement is notdeclared effective prior to the 120th day after July 31, 2019 (or the 150th day if the Securities and Exchange Commission reviews suchregistration statement), then the Company will make pro rata payments in cash to each investor then holding Registrable Securities, as liquidateddamages, in an amount equal to 1% of the aggregate amount invested by such investor for each thirty (30)-day period or pro rata for any portionthereof following the date by which such registration statement should have been effective. 173Table of ContentsITEM 9.CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURENot Applicable. ITEM 9A.CONTROLS AND PROCEDURESLimitations on Effectiveness of Controls and ProceduresIn designing and evaluating our disclosure controls and procedures, management recognizes that any controls and procedures, no matterhow well designed and operated, can provide only reasonable assurance of achieving the desired control objectives. In addition, the design ofdisclosure controls and procedures must reflect the fact that there are resource constraints and that management is required to apply judgment inevaluating the benefits of possible controls and procedures relative to their costs.Evaluation of Disclosure Controls and ProceduresOur management, with the participation of our Chief Executive Officer (principal executive officer) and Chief Financial Officer (principalfinancial officer), evaluated, as of the end of the period covered by this Form 10-K, the effectiveness of our disclosure controls and procedures (asdefined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the “Exchange Act”)). Based on that evaluation, ourChief Executive Officer (principal executive officer) and Chief Financial Officer (principal financial officer) concluded that our disclosure controls andprocedures were effective at the reasonable assurance level as of December 31, 2018.Exemption from Management’s Report on Internal Control Over Financial ReportingThis Form 10-K does not include a report of management’s assessment regarding internal control over financial reporting or an attestationreport of our independent registered public accounting firm due to a transition period established by rules of the SEC for newly public companies.In addition, for so long as we qualify as an “emerging growth company” as defined under the JOBS Act, our independent registeredaccounting firm is not required to issue an attestation report on our internal control over financial reporting.Changes in Internal Control Over Financial ReportingThere were no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the ExchangeAct) during the three months ended December 31, 2018 that have materially affected, or are reasonably likely to materially affect, our internal controlover financial reporting. ITEM 9B.OTHER INFORMATIONNot applicable. 174Table of ContentsPART III ITEM 10.DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCEThe information required by this Item is incorporated by reference to our definitive proxy statement for our 2019 annual shareholdermeeting to be filed with the SEC within 120 days of the fiscal year ended December 31, 2018. ITEM 11.EXECUTIVE COMPENSATIONThe information required by this Item is incorporated by reference to our definitive proxy statement for our 2019 annual shareholdermeeting to be filed with the SEC within 120 days of the fiscal year ended December 31, 2018. ITEM 12.SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDERMATTERSSecurities Authorized for Issuance Under Equity Compensation Plans (as of December 31, 2018)The following table provides information as of December 31, 2018, regarding our ordinary shares that may be issued under the MeiraGTx Holdings plc2016 Equity Incentive Plan, as amended (the “2016 Plan”), the MeiraGTx Holdings plc 2018 Incentive Award Plan (the “2018 Plan”) and theMeiraGTx Holdings plc 2018 Employee Stock Purchase Plan (the “2018 ESPP”). Plan category: Number of Securitiesto be Issued UponExercise ofOutstanding Options,Warrants, andRights(a) Weighted-Average ExercisePrice ofOutstandingOptions,Warrants, andRights(b) Number of SecuritiesAvailable for FutureIssuance Under EquityCompensation Plans(excludes securitiesreflected in column (a))(c) Equity compensation plans approved by shareholders 2016 Plan(1) 1,603,765 $5.34 — 2018 Plan (2) 1,658,600 $9.87 1,396,396 2018 ESPP (3) — — 509,166 Equity compensation plans not approved by shareholders — — — Total 3,262,365 $7.64 1,905,562 (1)In connection with our IPO, we assumed the 2016 Plan. As the 2016 Plan was previously approved by our shareholders and, as we will not makefuture grants or awards under these plans, it is listed as “approved by shareholders.” As such, the securities remaining available under the 2016Plan have been excluded from the table above. (2)Pursuant to the terms of the 2018 Plan, the number of ordinary shares available for issuance under the 2018 Plan automatically increases on eachJanuary 1, until and including January 1, 2028, by an amount equal to the lesser of: (a) 4% of the aggregate number of ordinary sharesoutstanding on the final day of the immediately preceding calendar year and (b) such smaller number of ordinary shares as is determined by ourboard of directors. (3)Pursuant to the terms of the 2018 ESPP, the number of ordinary shares available for issuance under the 2018 ESPP automatically increases oneach January 1, until and including January 1, 2028, by an amount equal to the lesser of: (a) 1% of the aggregate number of ordinary sharesoutstanding on the final day of the immediately preceding calendar year and (b) such smaller number of ordinary shares as is determined by ourboard of directors, subject to the limit set forth in the 2018 ESPP. 175Table of ContentsOtherThe remaining information required by this Item is incorporated by reference to our definitive proxy statement for our 2019 annualshareholder meeting to be filed with the SEC within 120 days of the fiscal year ended December 31, 2018. ITEM 13.CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTOR INDEPENDENCEThe information required by this Item is incorporated by reference to our definitive proxy statement for our 2019 annual shareholdermeeting to be filed with the SEC within 120 days of the fiscal year ended December 31, 2018. ITEM 14.PRINCIPAL ACCOUNTANT FEES AND SERVICESThe information required by this Item is incorporated by reference to our definitive proxy statement for our 2019 annual shareholdermeeting to be filed with the SEC within 120 days of the fiscal year ended December 31, 2018. 176Table of ContentsPART IV ITEM 15.EXHIBITS(a) List of documents filed as part of this Form 10-K:(1) Financial StatementsThe financial statements included in Part II, Item 8 of this document are filed as part of this Form 10-K.(2) Financial Statement SchedulesAll schedules have been omitted because they are not required or because the required information is given in the consolidated financialstatements or notes thereto.(3) ExhibitsThe following documents are filed as exhibits to this Form 10-K. Exhibit Number Exhibit Description Incorporated by Reference Form File No. Exhibit FilingDate Filed/FurnishedHerewith 2.1† Agreement and Plan of Merger, dated October 5, 2018, by andamong MeiraGTx Holdings plc, Vector Neurosciences Inc., VNAcquisition, Inc., VN Acquisition 2, Inc., the Vectorstockholders named therein and the Vector stockholderrepresentative, Stephen Kaplitt. * 3.1 Restated Articles of Association of the Registrant. S-1 333-224914 3.1 5/29/18 4.1 Specimen Share Certificate evidencing the ordinary shares ofthe Registrant. S-1 333-224914 4.1 5/29/18 4.2 Shareholder Agreement 333-224914 4.2 6/4/18 10.1# 2016 Equity Incentive Plan, as amended, and form of optionagreements thereunder. S-1/A 333-224914 10.1 5/29/18 10.2# 2018 Incentive Award Plan and forms of award agreementsthereunder. S-1/A 333-224914 10.2 5/29/18 10.3# Non-Employee Director Compensation Program. S-1/A 333-224914 10.3 5/29/18 10.4# Form of Indemnification Agreement for Directors and Officers. S-1/A 333-224914 10.4 5/29/18 10.5 Lease Agreement, dated June 29, 2016, as amended, betweenMeiraGTx Limited and ARE-East River Science Park LLC. S-1 333-224914 10.5 5/14/18 177Table of ContentsExhibit Number Exhibit Description Incorporated by Reference Form File No. Exhibit FilingDate Filed/FurnishedHerewith 10.6 Lease Agreement, effective February 2, 2016, amongMeiraGTx Limited, Moorfields Eye Hospital NHS,Foundation Trust and Kadmon Corporation LLC. S-1 333-224914 10.6 5/14/18 10.7# Employment Agreement, dated February 15, 2016, betweenMeiraGTx Limited and Alexandria Forbes, Ph.D., asamended. S-1/A 333-224914 10.7 5/29/18 10.8# Employment Agreement, dated February 15, 2016 betweenMeiraGTx Limited and Richard Giroux, as amended. S-1/A 333-224914 10.8 5/29/18 10.9# Employment Agreement, dated April 27, 2015, betweenMeiraGTx Limited and Stuart Naylor, Ph.D., as amended S-1/A 333-224914 10.9 5/29/18 10.10† Agreement and Plan of Merger, dated December 31, 2015,among MeiraGTx Acquisition Corporation, BRI-Alzan, Inc.,F-Prime Inc., Gregory Petsko, Dagmar Ringe, BrandeisUniversity and MeiraGTx Limited. S-1/A 333-224914 10.14 5/29/18 10.11# 2018 Employee Share Purchase Plan. S-1/A 333-224914 10.15 5/29/18 10.12# UK Sub-Plan Under the 2018 Incentive Award Plan. * 10.13# Form of Option Grant Notice and Option Agreement Underthe UK Sub-Plan to the 2018 Incentive Award Plan. * 10.14# Employment Offer Letter, dated October 2, 2018, betweenMeiraGTx Holdings plc and Katherine Breedis 8-K 001-38520 10.1 10/9/18 10.15 Lease agreement by and between Moorfields Eye HospitalNHS Foundation Trust and MeiraGTx UK II Limited, datedJuly 30, 2018 10-Q 001-38520 10.4 8/08/18 10.16 Lease agreement by and between Moorfields Eye HospitalNHS Foundation Trust and MeiraGTx UK II Limited, datedJuly 30, 2018. 10-Q 001-38520 10.5 8/08/18 10.17 Transfer of Title, dated December 14, 2018, and Lease,dated October 12, 2001, relating to the PharmacyManufacturing Unit, Britannia Walk, London, England 8-K 001-38520 10.1 12/14/18 178Table of ContentsExhibit Number Exhibit Description Incorporated by Reference Form File No. Exhibit FilingDate Filed/FurnishedHerewith 10.18 Overage Deed, dated December 14, 2018, between MoorfieldsEye Hospital NHS Foundation Trust and MeiraGTx UK IILimited relating to the Pharmacy Manufacturing Unit, BritanniaWalk, London, England 8-K 001-38520 10.2 12/14/18 10.19† Consulting Agreement, dated October 5, 2018, betweenMeiraGTx Holdings plc, Vector Consulting LLC, Michael G.Kaplitt, Matthew During, and Stephen B. Kaplitt. * 10.20† License Agreement (RPE65), dated January 29, 2019, asamended and restated by and among UCL Business PLC,MeiraGTx UK II Limited and MeiraGTx Limited. * 10.21† License Agreement (CNGB3), dated January 29, 2019, asamended and restated by and among UCL Business PLC,MeiraGTx Holdings plc, MeiraGTx UK II Limited andMeiraGTx Limited. * 10.22† License Agreement (CNGA3), dated January 29, 2019, asamended and restated by and among UCL Business PLC,MeiraGTx UK II Limited and MeiraGTx Limited. * 10.23† License Agreement (RPGR), dated February 5, 2019, asamended and restated by and among UCL Business PLC,MeiraGTx UK II Limited and MeiraGTx Limited. * 10.24† Amendment No. 4 to Exclusive License Agreement, datedJanuary 29, 2019, between UCLB and MeiraGTx Limited. * 10.25† Collaboration, Option and License Agreement, datedJanuary 30, 2019, by and among Janssen Pharmaceuticals, Inc.,MeiraGTx UK II Limited and MeiraGTx Holdings plc. * 10.26† Registration Rights Agreement, dated February 26, 2019, byand among MeiraGTx Holdings plc and the investors namedtherein. 8-K 001-38520 10.2 2/26/19 21 List of Subsidiaries * 23.1 Consent of Ernst & Young LLP * 179Table of ContentsExhibit Number Exhibit Description Incorporated by Reference Form FileNo. Exhibit FilingDate Filed/FurnishedHerewith 31.1 Certification of Chief Executive Officer pursuant to Rules13a-14(a)/15d-14(a) under the Securities Exchange Act of 1934,as amended. * 31.2 Certification of Chief Financial Officer pursuant to Rules13a-14(a)/15d-14(a) under the Securities Exchange Act of 1934,as amended. * 32.1 Certification of Chief Executive Officer pursuant to 18 U.S.C.Section 1350, as adopted pursuant to Section 906 of theSarbanes-Oxley Act of 2002. ** 32.2 Certification of Chief Financial Officer pursuant to 18 U.S.C.Section 1350, as adopted pursuant to Section 906 of theSarbanes-Oxley Act of 2002. ** 101.INS XBRL Instance Document. * 101.SCH XBRL Taxonomy Extension Schema Document. * 101.CAL XBRL Taxonomy Extension Calculation Linkbase Document. * 101.DEF XBRL Taxonomy Definition Linkbase Document. * 101.LAB XBRL Taxonomy Label Linkbase Document. * 101.PRE XBRL Taxonomy Extension Presentation Linkbase Document. * *Filed herewith**Furnished herewith#Management contract or compensation plan or arrangement†Portions of this exhibit (indicated by asterisks) have been omitted pursuant to a request for confidential treatment pursuant to Rule 406 under theSecurities Act of 1933, as amendedCertain agreements filed as exhibits to this Form 10-K contain representations and warranties that the parties thereto made to each other.These representations and warranties have been made solely for the benefit of the other parties to such agreements and may have been qualified bycertain information that has been disclosed to the other parties to such agreements and that may not be reflected in such agreements. In addition, theserepresentations and warranties may be intended as a way of allocating risks among parties if the statements contained therein prove to be incorrect,rather than as actual statements of fact. Accordingly, there can be no reliance on any such representations and warranties as characterizations of theactual state of facts. Moreover, information concerning the subject matter of any such representations and warranties may have changed since the dateof such agreements. ITEM 16.FORM 10-K SUMMARYNone. 180Table of ContentsSIGNATURESPursuant to the requirements of Section 13 or 15(d) the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on itsbehalf by the undersigned thereunto duly authorized. MeiraGTx Holdings plc (Registrant)Date: March 26, 2019 By: /s/ Alexandria Forbes Alexandria Forbes President and Chief Executive Officer and Director (PrincipalExecutive Officer) 181Table of ContentsSIGNATURESPursuant to the requirements of the Securities Exchange Act of 1934, this report has been signed below by the following persons on behalfof Registrant and in the capacities and on the dates indicated. Signature Title Date/s/ Alexandria Forbes, Ph.D President and Chief Executive Officer and Director March 26, 2019Alexandria Forbes, Ph.D (Principal Executive Officer) /s/ Katherine Breedis Chief Financial Officer March 26, 2019Katherine Breedis (Principal Financial and Accounting Officer) /s/ Keith R. Harris, Ph.D. Chairman of the Board and Director March 26, 2019Keith R. Harris, Ph.D. /s/ Stuart Naylor, Ph.D. Director March 26, 2019Stuart Naylor, Ph.D. /s/ Ellen Hukkelhoven Director March 26, 2019Ellen Hukkelhoven /s/ Martin Indyk Director March 26, 2019Martin Indyk /s/ Arnold J. Levine, Ph.D. Director March 26, 2019Arnold J. Levine, Ph.D. /s/ Joel S. Marcus Director March 26, 2019Joel S. Marcus /s/ Neil Mendoza Director March 26, 2019Neil Mendoza /s/ Gregory S. Moss Director March 26, 2019Gregory S. Moss /s/ Thomas E. Shenk, Ph.D. Director March 26, 2019Thomas E. Shenk, Ph.D. 182Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Exhibit 2.1Execution VersionAGREEMENT AND PLAN OF MERGERThis Agreement and Plan of Merger (this “Agreement”) is made and entered into as of October 5, 2018, by and among MeiraGTx Holdings plc,a company incorporated in the Cayman Islands (the “Buyer”), VN Acquisition, Inc., a Delaware corporation and a wholly-owned subsidiary of theBuyer (“Merger Sub 1”), VN Acquisition 2, Inc., a Delaware corporation and a wholly-owned subsidiary of the Buyer (“Merger Sub 2”), VectorNeurosciences Inc., a Delaware corporation, the Company Stockholders named on the signature pages hereto and Stephen B. Kaplitt, as therepresentative of the Company Stockholders (in such capacity, the “Company Stockholder Representative” and, collectively with the Buyer, MergerSub 1, Merger Sub 2, the Company and the Company Stockholders, the “Parties”). “Company Stockholders” means each stockholder of the Companyas of immediately prior to the Merger 1 Effective Time.RECITALSWHEREAS, the respective Boards of Directors of the Buyer, Merger Sub 1, Merger Sub 2 and the Company have each determined that it isadvisable and in the best interests of the Buyer, Merger Sub 1, Merger Sub 2 and the Company, respectively, and their respective shareholders orstockholders that the Company be acquired indirectly by the Buyer;WHEREAS, the acquisition of the Company by the Buyer shall be effected through the following transactions: (1) a merger of Merger Sub 1 withand into the Company, with the Company surviving as a wholly owned subsidiary of the Buyer (“Merger 1”) and (2) a merger of the Company withand into Merger Sub 2, with Merger Sub 2 surviving as a wholly owned subsidiary of the Buyer (“Merger 2” and, together with Merger 1, the“Mergers”); andWHEREAS, for United States federal income tax purposes, the Parties intend that the Mergers qualify as a reorganization within the meaning ofSection 368(a) of the Internal Revenue Code, and the Parties intend, by executing this Agreement, to adopt a plan of reorganization within themeaning of Treasury Regulations Sections 1.368-2(g) and 1.368-3(a).NOW THEREFORE, in consideration of the premises, and the mutual representations, warranties, covenants and agreements contained herein,and for other good and valuable consideration, the receipt and sufficiency of which are hereby acknowledged by each of the Parties, and intending tobe legally bound, the Parties hereby agree as follows: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 ARTICLE ITHE MERGERS1.1 The Mergers. Upon the terms and subject to the conditions set forth in this Agreement, Merger Sub 1 shall merge with and into theCompany at the Merger 1 Effective Time. From and after the Merger 1 Effective Time, the separate corporate existence of Merger Sub 1 shall cease andthe Company shall continue as the surviving entity of Merger 1 (“Intermediate Surviving Corporation”). Promptly following the consummation ofMerger 1, and upon the terms and subject to the conditions set forth in this Agreement, the Company shall merge with and into Merger Sub 2 at theMerger 2 Effective Time. From and after the Merger 2 Effective Time, the separate corporate existence of the Company shall cease and Merger Sub 2shall continue as the surviving entity of Merger 2 (“Surviving Corporation”). The Mergers shall have the effects set forth in Section 259 of theDelaware General Corporation Law.1.2 The Closing. The Closing shall take place at the offices of Latham & Watkins LLP, 885 Third Avenue, New York, NY 10022, commencingat 10:00 a.m., local time, on the Closing Date, or such other place as the Parties may agree prior to the Closing.1.3 Actions at the Closing. At the Closing:(a) the Company shall deliver to the Buyer, Merger Sub 1 and Merger Sub 2 the various certificates, instruments and documents referredto in Section 6.1;(b) the Buyer, Merger Sub 1 and Merger Sub 2 shall deliver to the Company the various certificates, instruments and documents referredto in Section 6.2;(c) each Company Stockholder shall deliver to the Buyer for cancellation a certificate or certificates or an affidavit of book entrypositions representing all of such Company Stockholder’s Company Shares;(d) the Buyer shall cause the Certificate of Merger 1 to be filed with the Secretary of State of the State of Delaware;(e) the Buyer shall cause the Certificate of Merger 2 to be filed with the Secretary of State of the State of Delaware;(f) the Buyer shall issue to each Company Stockholder the portion of the Closing Shares (as defined below) allocable to such CompanyStockholder, as set forth on the Allocation Schedule and deliver a certificate or evidence of a book-entry position with respect to such Closing Sharesto such Company Stockholders; and(g) the Buyer shall deliver a copy of a resolution approved by the board of directors of the Buyer, authorizing the issuance to eachCompany Stockholder of the Closing Shares and Holdback Shares, the securities to be issued pursuant to the Consulting Agreement and the securitiesissuable pursuant to the Milestone Payments. Upon determination of the number of Holdback Shares which such Company Stockholder may ultimatelybe entitled to Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 2 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 receive pursuant to Section 1.5(a)(ii), the Buyer shall issue to each Company Stockholder the portion of the Holdback Shares allocable to suchCompany Stockholder. “Holdback Shares” shall mean 22,500 Buyer Capital Shares. The Holdback Shares shall be a source for effecting thesatisfaction and discharge of any indemnification obligation owed by any Company Stockholder to the Buyer under this Agreement, and the Buyershall have the right to recover any Losses for which it is entitled to indemnification (1) under Section 7.1(a) of this Agreement, via the forfeiturepursuant to Section 7.4(a) of the right to have issued the Holdback Shares, and (2) under Section 7.1(b) of this Agreement, via the forfeiture pursuant toSection 7.4(a) of the right to have issued Holdback Shares allocable to the applicable Company Stockholder as set forth on the Allocation Schedule.1.4 Additional Actions. The Surviving Corporation may, at any time after the either the Merger 1 Effective Time or the Merger 2 EffectiveTime, take any action, including executing and delivering any document or instrument, in the name and on behalf of the Company, Merger Sub 1 orMerger Sub 2, in order to consummate the transactions contemplated by this Agreement.1.5 Conversion of Company Shares; Conversion of Merger Sub 1 Shares.(a) Conversion of Company Shares.(i) At the Merger 1 Effective Time, by virtue of Merger 1 and without any action on the part of any Party or the holder of anyCompany Shares, all Company Shares issued and outstanding as of immediately prior to the Merger 1 Effective Time and held by anyCompany Stockholder shall be converted into and represent the right to receive (A) the number of Closing Shares allocable to suchCompany Stockholder, as set forth on the Allocation Schedule, (B) the number of Holdback Shares allocable to such CompanyStockholder, less any Holdback Shares forfeited by such Company Stockholder in accordance with this Agreement, as set forth on theAllocation Schedule, (C) any Milestone Payment due pursuant to Section 1.11; and (D) any Royalty payments due pursuant toSection 1.12, in each case in accordance with and subject to the terms and conditions of this Agreement (including anyindemnification obligations hereunder) and the Certificate of Incorporation. “Closing Shares” shall mean 202,500 Buyer CapitalShares.(ii) On the date that is 18 months after the Closing Date the Buyer shall issue to each Company Stockholder from the HoldbackShares the number of the Releasable Shares (rounded down to the nearest whole share) allocable to such Company Stockholder, as setforth on the Allocation Schedule and deliver a certificate or evidence of a book-entry position with respect to such Releasable Sharesto such Company Stockholder; provided that if on or prior to such date, the Buyer has delivered any Claim Notice or Expected ClaimNotice to the Company Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 3 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Stockholder Representative containing a claim which has not been resolved in accordance with Section 7.2, the Buyer shall not berequired to issue a number of Holdback Shares (rounded down to the nearest whole share) equal to (A) the amount of the contestedportion of such unresolved claim or claims divided by (B) the Buyer Share Value, and shall instead issue to each CompanyStockholder the number of Releasable Shares (rounded down to the nearest whole share) that are allocable to such CompanyStockholder; provided, further, that upon the resolution of all such unresolved claims, Buyer shall promptly issue to each CompanyStockholder the Holdback Shares (rounded down to the nearest whole share) remaining, if any, allocable to such CompanyStockholder, less the amount of such Holdback Shares forfeited by such Company Stockholder in accordance with this Agreement,if any. “Releasable Shares” shall mean a number of Buyer Shares (rounded down to the nearest whole share) equal to the number ofHoldback Shares, minus the number of Buyer Shares equal to (1) the amount of all Losses, if any, for which Buyer has theretoforebecome entitled to indemnification pursuant to Section 7.1 and that has resulted in the forfeiture of the right to be issued theHoldback Shares pursuant to Section 7.4(a) and (2) the amount of any unresolved claim reflected in a Claim Notice or ExpectedClaim Notice, divided by (y) the Buyer Share Value.(b) Cancellation of Treasury Stock. Any Company Shares held in the Company’s treasury immediately prior to the Merger 1 EffectiveTime and any Company Shares owned by the Buyer, Merger Sub 1 or Merger Sub 2 shall be cancelled and retired without payment of anyconsideration therefor.(c) Capital Stock of Merger Sub 1. Each share of common stock, $0.001 par value per share, of Merger Sub 1 issued and outstandingimmediately prior to the Merger 1 Effective Time shall be converted into and thereafter evidence one share of common stock, $0.001 par value pershare, of the Intermediate Surviving Corporation.(d) Capital Stock of the Intermediate Surviving Corporation. Each share of common stock, $0.001 par value per share, of the IntermediateSurviving Corporation issued and outstanding immediately prior to the Merger 2 Effective Time shall be converted into and thereafter evidence oneshare of common stock, $0.001 par value per share, of the Surviving Corporation.1.6 Certificate of Incorporation and Bylaws; Directors and Officers.(a) The Certificate of Incorporation of the Company in effect as of immediately prior to the Merger 1 Effective Time will be amended andrestated in its entirety in the form of the certificate of incorporation of Merger Sub 1 in effect as of immediately prior to the Merger 1 Effective Time,and as so amended shall be the certificate of incorporation of the Intermediate Surviving Corporation until amended or repealed in accordance with theprovisions Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 4 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 thereof, and applicable Law. The Certificate of Incorporation of the Intermediate Surviving Corporation in effect as of immediately prior to the Merger2 Effective Time will be amended and restated in its entirety in the form of the certificate of incorporation of Merger Sub 2 in effect as of immediatelyprior to the Merger 2 Effective Time, and as so amended shall be the certificate of incorporation of the Surviving Corporation until amended orrepealed in accordance with the provisions thereof, and applicable Law.(b) The bylaws of Merger Sub 1 in effect as of immediately prior to the Merger 1 Effective Time shall be the bylaws of the IntermediateSurviving Corporation until amended or repealed in accordance with the provisions thereof, the certificate of incorporation of the IntermediateSurviving Corporation, and applicable Law. The bylaws of the Intermediate Surviving Corporation in effect as of immediately prior to the Merger 2Effective Time will be amended and restated in their entirety in the form of the bylaws of Merger Sub 2 in effect as of immediately prior to the Merger 2Effective Time, and as so amended shall be the bylaws of the Surviving Corporation until amended or repealed in accordance with the provisionsthereof, the certificate of incorporation of the Surviving Corporation, and applicable Law.(c) The directors of Merger Sub 1 immediately prior to the Merger 1 Effective Time shall be the initial directors of the IntermediateSurviving Corporation, and the officers of Merger Sub 1 immediately prior to the Merger 1 Effective Time shall be the initial officers of theIntermediate Surviving Corporation, each to hold office in accordance with the Certificate of Incorporation and bylaws of the Intermediate SurvivingCorporation. The directors of Merger Sub 2 immediately prior to the Merger 2 Effective Time shall be the initial directors of the Surviving Corporation,and the officers of Merger Sub 2 immediately prior to the Merger 2 Effective Time shall be the initial officers of the Surviving Corporation, each tohold office in accordance with the Certificate of Incorporation and bylaws of the Surviving Corporation.1.7 No Further Rights. From and after the Merger 1 Effective Time, no Company Shares shall be deemed to be outstanding, and any holders ofcertificates formerly representing Company Shares shall cease to have any rights with respect thereto, except as provided herein or by Law.1.8 Closing of Transfer Books. At the Merger 1 Effective Time, the stock transfer books of the Company shall be closed and no transfer ofCompany Shares shall thereafter be made. If, after the Merger 1 Effective Time, valid and authentic certificates formerly representing Company Sharesare presented to the Buyer or the Surviving Corporation, they shall be cancelled and exchanged for the applicable consideration payable, if any, inrespect of such Company Shares pursuant to Section 1.5, subject to the provisions of Article VII.1.9 Company Stockholder Representative.(a) To facilitate the administration of the transactions contemplated by this Agreement, including the resolution of any disputes relatingto claims for indemnification pursuant to Article VII and any other actions required or permitted to be taken by the Company StockholderRepresentative under this Agreement, the Company Stockholders, by their execution Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 5 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 and delivery of this Agreement and/or the Written Consent, hereby (i) designate the Company Stockholder Representative as their representative,attorney-in-fact and agent and (ii) authorize the Company Stockholder Representative to give and receive all notices required to be given under thisAgreement, to amend this Agreement, and to take any and all additional action as is contemplated to be taken by or on their behalf or by the CompanyStockholder Representative by the terms of this Agreement. All such actions shall be deemed to be facts ascertainable outside this Agreement and shallbe binding on the Company Stockholders.(b) In the event that the Company Stockholder Representative named in the Preamble above, or any successor thereto, becomes unable toperform his responsibilities hereunder or resigns from such position, a majority in interest of the Company Stockholders, voting together as a singleclass based on the relative size of their respective ownership interests in the Company as of immediately prior to the Merger 1 Effective Time (asreflected on the Allocation Schedule) (the “Stockholder Majority”) shall select another representative to fill the vacancy of the Company StockholderRepresentative named in this Agreement, and such substituted representative shall be deemed to be the Company Stockholder Representative for allpurposes of this Agreement and the documents delivered pursuant hereto; provided, however, that such substitution shall not be effective prior to thetime that the Company Stockholders provide written notice to the Buyer of such substitution. The Stockholder Majority may, from time to time, uponwritten notice to the Company Stockholder Representative and the Buyer, remove the Company Stockholder Representative and appoint a newCompany Stockholder Representative in the place thereof.(c) All decisions and actions by the Company Stockholder Representative in connection with the transactions contemplated by thisAgreement, including the resolution and disposition of any claims for indemnification pursuant to Article VII and any other actions required orpermitted to be taken by the Company Stockholder Representative under this Agreement, shall be binding upon each Company Stockholder, and noCompany Stockholder shall have the right to object, dissent, protest or otherwise contest the same.(d) Any decision, act, consent, waiver or instruction of the Company Stockholder Representative in connection with this Agreement shallconstitute a decision of all the Company Stockholders and shall be final, binding and conclusive upon each Company Stockholder, and the Buyer andthe Surviving Corporation shall be entitled to rely conclusively on the decisions, acts, consents, waivers and instructions of the Company StockholderRepresentative as to any determination relating to the transactions contemplated by this Agreement as being the decision, act, consent, waiver orinstruction of every Company Stockholder, including the resolution and disposition of any claims for indemnification pursuant to Article VII and anyother actions required or permitted to be taken by the Company Stockholder Representative under this Agreement, all of which shall be binding uponeach Company Stockholder; no Person shall have any cause of action against the Buyer, the Surviving Corporation, or any of their respective directors,officers, employees, agents or Affiliates for any action taken by the Buyer in reliance upon any decision, act, consent, waiver or instruction of theCompany Stockholder Representative; and the Buyer and the Surviving Corporation are each hereby relieved from any liability to any Person for anyacts done by it in accordance with such decision, act, consent, waiver or instruction of the Company Stockholder Representative. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 6 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (e) All actions, decisions and instructions of the Company Stockholder Representative shall be conclusive and binding upon eachCompany Stockholder and none of the Buyer, the Surviving Corporation or any Company Stockholder shall have any cause of action against theCompany Stockholder Representative for any action taken, decision made or instruction given by the Company Stockholder Representative under thisAgreement, except for fraud or willful breach of this Agreement by the Company Stockholder Representative.(f) The provisions of this Section 1.9 are independent and severable, are irrevocable and coupled with an interest, and shall beenforceable notwithstanding any rights or remedies that the Buyer or any Company Stockholder may have in connection with the transactionscontemplated by this Agreement.(g) The Company Stockholders shall severally (based on the relative size of their respective ownership interests in the Company as ofimmediately prior to the Merger 1 Effective Time (as reflected on the Allocation Schedule)) and not jointly indemnify the Company StockholderRepresentative for any reasonable expense incurred without gross negligence or bad faith on the part of the Company Stockholder Representative andarising out of or in connection with the acceptance or administration of its duties hereunder (“Representative Reimbursable Expenses”). Withoutlimiting the foregoing, the Company Stockholder Representative shall have the right to engage legal counsel and other professional advisers to assistit in the administration of the Company Stockholder Representative’s duties hereunder, and any and all reasonable and documented fees and expensesof such counsel and advisers shall be deemed Representative Reimbursable Expenses, and the Company Stockholder Representative shall not be liableto any Company Stockholder for any action taken or omitted to be taken under this Agreement in good faith in accordance with the advice of suchcounsel or other professional advisors.1.10 Withholding Rights. Notwithstanding anything to the contrary, each of the Buyer, the Intermediate Surviving Corporation and theSurviving Corporation will be entitled to deduct and withhold from any consideration otherwise payable or deliverable pursuant to this Agreement toany Person, such amounts as may be required to be deducted and withheld with respect to the making of such payment under the Internal RevenueCode or any other Law. To the extent that amounts are so deducted or withheld by the Buyer, the Intermediate Surviving Corporation or the SurvivingCorporation, such deducted or withheld amounts will be treated for all purposes of this Agreement as having been paid on behalf of such Person inrespect of which such deduction or withholding was made. Buyer, the Intermediate Surviving Corporation or the Surviving Corporation will promptlyfurnish in writing notice to such Person of the amounts deducted or withheld, and the identity and address of the Governmental Entity to which suchamounts have been or will be transmitted.1.11 Milestone Payments. At any time after the Merger 2 Effective Time, upon the achievement of any of the milestones set forth in the chartbelow under the heading “Milestone” Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 7 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (each, a “Milestone”), the payment set forth opposite such Milestone in the chart below (each, a “Milestone Payment”) shall become due and payableby the Buyer to the Company Stockholders. Each Milestone Payment will be paid a maximum of one time (and one time only) for each Milestoneachieved. Milestone Milestone PaymentRegulatory Approval by the FDA of any GAD Product (the “U.S. RegulatoryMilestone”). A number of Buyer Capital Shares equal to $[***] divided by theBuyer Share Value on the date of achievement of the U.S. RegulatoryMilestoneRegulatory Approval by the EMA of any GAD Product (the “EMARegulatory Milestone”). A number of Buyer Capital Shares equal to $[***] divided by theBuyer Share Value on the date of achievement of the EMA RegulatoryMilestoneRegulatory Approval by the FDA or EMA of any IP-related Covered Productin the United States or Europe (as applicable based on the regulatoryauthority granting Regulatory Approval) as of the date that such RegulatoryApproval is obtained (the “IP Assets Related Milestone”) A number of Buyer Capital Shares equal to $[***] divided by theBuyer Share Value on the date of achievement of the IP Assets RelatedMilestone(a) The Buyer shall pay or cause to be paid to the Company Stockholder Representative for distribution to the Company Stockholders (inaccordance with the proportions set forth in the Allocation Schedule) within thirty (30) days following the achievement of the U.S. RegulatoryMilestone, the EMA Regulatory Milestone or the IP Assets Related Milestone, the corresponding Milestone Payments. For clarity, the MilestonePayment related to the IP Assets Related Milestone shall only be paid once based on the first Regulatory Approval for a given IP-related CoveredProduct, and no additional Milestone Payments shall be due or payable in respect of any further Regulatory Approvals of any IP-related CoveredProducts.(b) The right of the Company Stockholders to receive any Milestone Payment or Royalty Payment: (i) does not give the CompanyStockholder Representative or the Company Stockholders dividend rights, voting rights, liquidation rights, preemptive rights or other rights of holdersof capital stock of the Buyer; (ii) shall not be evidenced by a certificate or other instrument; (iii) shall not be assignable or otherwise transferable by theCompany Stockholder Representative or Company Stockholders except to and among the Company Stockholders and subsequently shall not beassignable except by will, upon death or by operation of the laws of descent and distribution, or to a trust formed by a Company Stockholder for thesole benefit of immediate family members; (iv) shall not accrue or pay interest on any portion thereof; and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 8 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (v) does not represent any right other than the right to receive the consideration set forth in this Section 1.11. Any attempted transfer of the right to anyMilestone Payment by any holder thereof (other than as specifically permitted by the immediately preceding sentence) shall be null and void.(c) The Company Stockholders shall not be subject to any holding or standstill restrictions, or any other restrictions on resale, in respectof any Buyer Capital Shares received as Milestone Payments, except as provided in Sections 3.6 and 3.7 or for such restrictions as may be imposed byLaw.1.12 Net Sales Royalty Payments.(a) Subject to Section 1.12(c), after the Merger 2 Effective Time and for the duration of the Royalty Term, the Buyer shall make Royaltypayments to the Company Stockholders (in accordance with the proportions set forth in the Allocation Schedule) on a fiscal quarterly basis in anamount equal to [***] of aggregate, worldwide Net Sales of such GAD Products during the Royalty Term payable in Buyer Capital Shares based on theBuyer Share Value on the last day of each such fiscal quarter.(b) Within thirty (30) days after the end of the first calendar quarter during which the First Commercial Sale of a GAD Product occurs, andwithin thirty (30) days after the end of each fiscal quarter thereafter during the Royalty Term, the Buyer shall deliver to the Company Stockholders,together with the applicable Royalty payment due (payable in Buyer Capital Shares in accordance with the proportions set forth in the AllocationSchedule), a written report of the aggregate, worldwide Net Sales of GAD Products for such fiscal quarter. In addition, and for clarification purposesonly, the Buyer shall make Royalty payments on all Net Sales received during the Royalty Term, including those for which the Buyer receivespayment after the Royalty Term has expired.(c) If the Buyer has licensed rights that are necessary or reasonably useful to develop, manufacture or commercialize the GAD Productsfrom one or more third party(ies) then the Buyer may deduct from the Royalties it would otherwise owe to the Company Stockholders pursuant toSection 1.12(a) for the GAD Products an amount equal to [***] of all amounts aggregate payable to such third party(ies) in consideration for suchlicensed rights, up to a maximum aggregate deduction of [***] of the Royalties otherwise due to the Company Stockholders on a fiscal quarterly basispursuant to Section 1.12(a) for such GAD Products (the “Capped Percentage”). Any amounts in excess of the Capped Percentage in a given fiscalquarter will accrue and be deductible against Royalties otherwise due to the Company Stockholders pursuant to Section 1.12(a) for such GAD Productsin subsequent quarters, until the accrued amounts are fully deducted (but in all events subject to the Capped Percentage). For clarity, the quarterlyaccruals of any amounts in excess of the Capped Percentage may be carried over from year to year, until the accrued amounts are fully deducted (but inall events subject to the Capped Percentage). The Buyer may, in its sole discretion, obtain any licenses or other rights from third parties as the Buyerdeems reasonably necessary to make, use, sell, offer for sale, import and otherwise commercialize any GAD Products. Notwithstanding the foregoing, if Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 9 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 the Buyer has licensed rights from Medtronic that are necessary or reasonably useful to develop, manufacture or commercialize the GAD Products thenthe Buyer may deduct from the Royalties it would otherwise owe to the Company Stockholders pursuant to Section 1.12(a) for the GAD Products anamount equal to [***] of all amounts aggregate payable to Medtronic in consideration for such license rights, up to an additional [***] such that theCapped Percentage may be up to [***].1.13 Tax Consequences. It is intended by the Parties hereto that, for United States federal income tax purposes the Mergers shall constitute areorganization within the meaning of Section 368(a) of the Internal Revenue Code. The Parties hereto adopt this Agreement as a plan of reorganizationwithin the meaning of Treasury Regulations Sections 1.368-2(g) and 1.368-3(a).ARTICLE IIREPRESENTATIONS AND WARRANTIES OF THE COMPANYThe Company represents and warrants to the Buyer, Merger Sub 1 and Merger Sub 2 that, except as set forth in the disclosure schedule deliveredto the Buyer on the date of this Agreement (the “Disclosure Schedule”), the statements contained in this Article II are true and correct. The DisclosureSchedule shall be arranged in Sections and paragraphs corresponding to the numbered and lettered Sections and paragraphs contained in this Article II.The disclosures in any Section or paragraph of the Disclosure Schedule shall qualify only (a) the corresponding Section or paragraph in this Article IIor Article III and (b) other Sections or paragraphs in this Article II or Article III to the extent that it is reasonably apparent from a reading of thedisclosure that such disclosure also qualifies or applies to such other Section or paragraph.2.1 Organization, Qualification and Corporate Power. The Company is a corporation duly organized, validly existing and in corporate and taxgood standing under the Laws of the State of Delaware. The Company is duly qualified to conduct business and is in corporate and tax good standingunder the Laws of each jurisdiction listed in Section 2.1 of the Disclosure Schedule, which jurisdictions constitute the only jurisdictions in which thenature of the Company’s businesses or the ownership or leasing of its properties requires such qualification, except for such failures to be so qualifiedor in good standing that have not had, and would not reasonably be expected to have in the future, a Company Material Adverse Effect. The Companyhas all requisite corporate power and authority to carry on the businesses in which it is engaged and to own and use the properties owned and used byit. The Company has furnished to the Buyer complete and accurate copies of the Certificate of Incorporation. The Company is not in default under or inviolation of any provision of the Certificate of Incorporation. The Company has not adopted bylaws.2.2 Capitalization.(a) The authorized capital stock of the Company consists of 5,000 Company Shares, par value $0.0000100000 per share, of which 100shares are issued and outstanding. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 10 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (b) Section 2.2(b) of the Disclosure Schedule sets forth a complete and accurate list of the holders of capital stock of the Company,showing the number of shares of capital stock, and the class or series of such shares, held by each stockholder. Section 2.2(b) of the DisclosureSchedule also indicates all outstanding Company Shares, if any, that constitute restricted stock or that are otherwise subject to a repurchase orredemption right, indicating the name of the applicable stockholder, the vesting schedule (including any acceleration provisions with respect thereto),and the repurchase price payable by the Company. All of the issued and outstanding shares of capital stock of the Company have been duly authorizedand validly issued and are fully paid and nonassessable. All of the issued and outstanding shares of capital stock of the Company have been offered,issued and sold by the Company in compliance with all applicable federal and state securities Laws.(c) No subscription, warrant, option, convertible security or other right (contingent or otherwise) to purchase or acquire any shares ofcapital stock of the Company is authorized or outstanding. The Company has no obligation (contingent or otherwise) to issue any subscription,warrant, option, convertible security or other such right, or to issue or distribute to holders of any shares of its capital stock any evidences ofIndebtedness or assets of the Company. The Company has no obligation (contingent or otherwise) to purchase, redeem or otherwise acquire any sharesof its capital stock or any interest therein or to pay any dividend or to make any other distribution in respect thereof. There are no outstanding orauthorized stock appreciation, phantom stock or similar rights with respect to the Company.(d) Section 2.2(d)of the Disclosure Schedule lists each agreement, written or oral, between the Company and any holder of its securities,or among any holders of its securities, relating to the sale or transfer (including agreements relating to rights of first refusal, co-sale rights or“drag-along” rights), registration under the Securities Act, or voting, of the capital stock of the Company.(e) There is no claim pending, and to the knowledge of the Company, no claim threatened, against the Company by any Person that seeksto assert: (i) ownership or rights to ownership of any shares of Company capital stock; (ii) any rights of a stockholder, including any option, preemptiverights or rights to notice or to vote; (iii) any rights under the Certificate of Incorporation of the Company, as amended or restated; or (iv) any claim thathis, her or its shares have been wrongfully repurchased by the Company.2.3 Authorization of Transaction. The Company has all requisite corporate power and authority to execute and deliver this Agreement and toperform its obligations hereunder. The execution and delivery by the Company of this Agreement and, subject to obtaining the unanimous approval ofthe stockholders of the Company (the “Requisite Stockholder Approval”) in the form of the Written Consent, the consummation by the Company ofthe transactions contemplated hereby have each been duly and validly authorized by all necessary corporate action on the part of the Company.Without limiting the generality of the foregoing, the Board of Directors of the Company, by the unanimous vote of all directors (i) determined that theMergers are advisable, fair and in the best interests of the Company and its stockholders, (ii) adopted this Agreement in accordance with the provisionsof the Delaware General Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 11 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Corporation Law, and (iii) directed that this Agreement and the Mergers be submitted to the stockholders of the Company for their adoption andapproval and resolved to recommend that the stockholders of the Company vote in favor of the adoption of this Agreement and the approval of theMergers. This Agreement has been duly and validly executed and delivered by the Company and, assuming due authorization, execution and deliveryby the other Parties, constitutes a valid and binding obligation of the Company, enforceable against the Company in accordance with its terms, subjectto bankruptcy, insolvency, fraudulent transfer, reorganization, moratorium and similar Laws of general applicability relating to or affecting creditors’rights and to general equity principles (the “Bankruptcy and Equity Exception”).2.4 Noncontravention. Subject to the filing of the Certificate of Merger 1 and the Certificate of Merger 2 as required by the Delaware GeneralCorporation Law, none of the execution and delivery by the Company of this Agreement, the performance by the Company of any of its obligationshereunder or the consummation by the Company of the transactions contemplated hereby, does or will (a) conflict with or violate any provision of theCertificate of Incorporation of the Company, (b) require on the part of the Company any notice to or filing with, or any permit, authorization, consentor approval of, any Governmental Entity, (c) conflict with, result in a breach of, constitute (with or without due notice or lapse of time or both) a defaultunder, result in the acceleration of obligations or loss of any right or benefit under, create in any party the right to terminate, modify or cancel, orrequire any notice, consent or waiver under, any material contract or other instrument to which the Company is a party or by which the Company isbound or to which any of the Company’s assets is subject, (d) result in the imposition of any Security Interest upon any assets of the Company or(e) violate any order, writ, injunction, decree, statute, rule or regulation applicable to the Company or any of its properties or assets.2.5 Subsidiaries. The Company does not have, nor has it ever had, any Subsidiaries. The Company does not control, nor has it ever controlled,directly or indirectly, nor does the Company have, nor has it ever had, any direct or indirect equity participation or similar interest in any Person.2.6 Absence of Certain Changes. Except as set forth on Section 2.6 of the Disclosure Schedule, since December 31, 2015, the Company hasconducted its business in the Ordinary Course of Business and there has occurred no event or development which, individually or in the aggregate, hashad, or would reasonably be expected to have in the future, a Company Material Adverse Effect.2.7 Liabilities. Section 2.7 of the Disclosure Schedule sets forth, as of December 31, 2017 and as of the date hereof, all liabilities andobligations of the Company required by GAAP to be set forth on a balance sheet of the Company (the “Liability Statement”). Except for the liabilities(a) set forth on the Liability Statement or (b) expressly disclosed in the Disclosure Schedule, (i) the Company has no liabilities or obligations of anykind greater than or equal to $10,000, individually or in the aggregate and (ii) there is no outstanding Company Debt.2.8 Tax Matters. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 12 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (a) Except as set forth in Section 2.8(a) of the Disclosure Schedule, the Company has properly filed on a timely basis all income and othermaterial Tax Returns that it was required to file, and all such Tax Returns are true, correct and complete in all material respects and were prepared inmaterial compliance with all applicable Laws. Except as set forth in Section 2.8(a) of the Disclosure Schedule, the Company has paid on a timely basisall material Taxes, whether or not shown on any Tax Return, that were due and payable. As of the date of the latest Liability Statement, the unpaidTaxes of the Company did not exceed the reserve for Tax liability (excluding any reserve for deferred Taxes established to reflect timing differencesbetween book and Tax income) set forth or included on the Liability Statement, and since the date of the latest Liability Statement, the Company hasnot incurred any liability for Taxes outside the ordinary course of business or inconsistent with past practice.(b) All material Taxes that the Company is or was required by Law to withhold or collect have been duly withheld or collected and, to theextent required, have been properly paid to the appropriate Governmental Entity, and the Company has complied with all information reporting andbackup withholding requirements, including the maintenance of required records with respect thereto, in connection with amounts paid to anyemployee, independent contractor, creditor, or other third party.(c) The Company is not, and has never been, a member of an affiliated group with which it has filed (or been required to file)consolidated, combined, unitary or similar Tax Returns. The Company (i) does not have any liability under Treasury Regulation Section 1.1502-6 (orany comparable or similar provision of federal, state, local or foreign Law), as a transferee or successor, pursuant to any contractual obligation, orotherwise for any Taxes of any Person other than the Company, and (ii) is not a party to or bound by any Tax indemnity, Tax sharing, Tax allocation orsimilar agreement.(d) The Company has delivered to the Buyer (i) complete and correct copies of all Tax Returns filed by the Company relating to Taxes forall taxable periods for which the applicable statute of limitations has not yet expired, (ii) complete and correct copies of all private letter rulings,revenue agent reports, information document requests, notices of proposed deficiencies, deficiency notices, protests, petitions, closing agreements,settlement agreements, pending ruling requests and any similar documents submitted by, received by, or agreed to by or on behalf of the Companyrelating to Taxes for all taxable periods for which the statute of limitations has not yet expired, if any, and (iii) complete and correct copies of allagreements, rulings, settlements or other Tax documents with or from any Governmental Entity relating to Tax incentives of the Company, if any.(e) No examination or audit or other action of or relating to any Tax Return of the Company by any Governmental Entity is currently inprogress or has been threatened in writing or, to the knowledge of the Company, in any other manner. No deficiencies for Taxes of the Company havebeen claimed, proposed or assessed, in each case in writing or, to the knowledge of the Company, in any other manner, by any Governmental Entity forany taxable period for which the period of assessment remains open. The Company has not been informed in writing or, to the knowledge of theCompany, in any other manner, by any jurisdiction in which Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 13 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 the Company did not file a Tax Return that the jurisdiction believes that the Company was required to file any Tax Return that was not filed or issubject to Tax in such jurisdiction. The Company has not (i) waived any statute of limitations with respect to Taxes or agreed to extend the period forassessment or collection of any Taxes, which waiver or extension is still in effect, (ii) requested any extension of time within which to file any TaxReturn, which Tax Return has not yet been filed, or (iii) executed or filed any power of attorney with any taxing authority, which is still in effect.(f) The Company will not be required to include any material item of income in, or exclude any material item of deduction from, taxableincome for any period (or any portion thereof) ending after the Closing Date as a result of (i) any adjustments under Section 481 of the InternalRevenue Code (or any similar adjustments under any provision of the Internal Revenue Code or the corresponding foreign, state or local Tax Law), (ii)installment sale or open transaction disposition made on or prior to the Closing Date, (iii) prepaid amount received on or prior to the Closing Date, or(iv) any election made pursuant to Section 108(i) of the Internal Revenue Code on or prior to the Closing Date.(g) The Company has not been a United States real property holding corporation within the meaning of Section 897(c)(2) of the InternalRevenue Code during the applicable period specified in Section 897(c)(l)(A)(ii) of the Internal Revenue Code.(h) The Company has not distributed to its shareholders or security holders stock or securities of a controlled corporation, nor has stock orsecurities of the Company been distributed, in a transaction to which Section 355 of the Internal Revenue Code applies.(i) There are no liens or other encumbrances with respect to Taxes upon any of the assets or properties of the Company, other than withrespect to Taxes not yet due and payable.(j) The Company has not engaged in a “reportable transaction” as set forth in Treasury Regulation Section 1.6011-4(b) or a “listedtransaction” as set forth in Treasury Regulation Section 301.6111-2(b)(2) or any analogous provision of state or local Law. The Company has disclosedon its federal income Tax Returns all positions taken therein that could give rise to a substantial understatement of federal income Tax within themeaning of Section 6662 of the Internal Revenue Code.(k) The Company is and has been since its formation properly treated as a corporation for U.S. federal income tax purposes.(l) The Company currently conducts a business. Such business is the Company’s “historic business” within the meaning of TreasuryRegulations Section 1.368-1(d), and no assets of the Company have been sold, transferred, or otherwise disposed of that would prevent the SurvivingCorporation from continuing the “historic business” of the Company or from using a “significant portion” of the Company’s “historic business assets”in a business following the Mergers, as such terms are used in Treasury Regulations Section 1.368-1(d). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 14 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (m) The Company is not an investment company within the meaning of Section 368(a)(2)(F)(iii) and (iv) of the Internal Revenue Code.(n) The Company is not under the jurisdiction of a court in a Title 11 or similar case within the meaning of Section 368(a)(3)(A) of theInternal Revenue Code.(o) The Company has not redeemed, purchased or otherwise acquired, or made any distributions with respect to, any of the Company’scapital stock prior to and in contemplation of the Mergers, or otherwise as part of a plan of which the Mergers are a part.(p) Neither the Company nor, to the knowledge of the Company, any Affiliate of the Company, has taken or agreed to take any actionthat would prevent the Mergers, from qualifying as a reorganization within the meaning of Section 368(a)(1)(A) of the Internal Revenue Code, and tothe knowledge of the Company, there is no agreement, plan or other circumstance to which the Company is a party or of which the Company hasknowledge that is not expressly contemplated by this Agreement and that would prevent the Mergers from qualifying as a reorganization within themeaning of Section 368(a)(1)(A) of the Internal Revenue Code.2.9 Assets and Properties. Other than the Neurologix Assets (addressed below and in Section 2.12), the Company is the true and lawful owner,and has good title to, all of the tangible assets owned or purported to be owned by the Company, free and clear of all Security Interests. The Companyis the true and lawful owner of, and has good title to, all of the Neurologix Assets. To the knowledge of the Company, no Person has asserted anySecurity Interest in the Neurologix Assets, other than Security Interests that may have been asserted prior or pursuant to the Neurologix Chapter 7bankruptcy. Each such tangible asset is free from defects, has been maintained in accordance with normal industry practice, is in good operatingcondition and repair (subject to normal wear and tear) and is suitable for the purposes for which it presently is used. The Company does not own, andhas never owned, any Real Property and does not lease, and has never leased, any Real Property. The Company has not sold, assigned, conveyed,transferred, pledged or otherwise encumbered any of its assets, including the Neurologix Assets.2.10 Intellectual Property. Company Registrations. (a) Section 2.10(a) of the Disclosure Schedule lists all Company Registrations, in eachcase enumerating specifically the applicable filing or registration number, title, jurisdiction in which filing was made or from which registration issued,date of filing or issuance and names of all current applicant(s) and registered owners(s), as applicable, but excluding any Company Registrations inrespect of any Neurologix Assets. All assignments of Company Registrations to the Company are valid and enforceable and have been properlyexecuted and recorded. To the knowledge of the Company, all Company Registrations contain patentable subject matter and, except with respect tothe Neurologix Assets, all issuance, renewal, maintenance and other payments that are or have become due with respect Company Registrations havebeen timely paid by or on behalf of the Company. Section 2.10(a) of the Disclosure Schedule lists all patents and patent applications owned orco-owned by Neurologix to the Company’s knowledge, pursuant to (i) a basic online search of the U.S. Patent & Trademark Office website(www.uspto.gov), and (ii) the Neurologix Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 15 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Bankruptcy Schedules. The Company has not made any payments with respect to the Neurologix Assets, and the Company has not received anynotices or billing statements in respect of renewal, maintenance or other payments that are or may have become due with respect to the NeurologixAssets. Except as disclosed in Section 2.10(a) of the Disclosure Schedule, the Company has no knowledge of any renewal, maintenance or otherpayments that are or may have become due with respect to the Neurologix Assets.(b) Prosecution Matters. Except with respect to the Neurologix Assets, there are no inventorship challenges, inter partes review,opposition or nullity proceedings or interferences declared, commenced or provoked, or to the knowledge of the Company threatened, with respect toany Company Registrations. The Company has complied with its duty of candor and disclosure to the United States Patent and Trademark Office andany relevant foreign patent office with respect to all Intellectual Property applications filed by or on behalf of the Company and has made no materialmisrepresentation in such applications. The Company has no knowledge of any information that would preclude the Company from having clear titleto the Company Registrations. The Company has no knowledge of any inventorship challenges, inter partes review, opposition or nullity proceedingsor interferences declared, commenced or provoked, or to the knowledge of the Company threatened, with respect to any Neurologix Assets.(c) Ownership; Sufficiency. Except for the Neurologix Assets, each item of Company Intellectual Property will be owned or available foruse by the Surviving Corporation immediately following the Closing without any impairment and on the same terms and conditions as it was availableto the Company immediately prior to the Closing. To the knowledge of the Company, each item of Company Intellectual Property that is or was part ofthe Neurologix Assets will be owned or available for use by the Surviving Corporation immediately following the Closing without any impairment andon the same terms and conditions as it was available to the Company immediately prior to the Closing. Except for the Neurologix Assets, the Companyis the sole and exclusive owner of all Company Owned Intellectual Property, free and clear of any Security Interests, except for any joint owners of theCompany Owned Intellectual Property, all of whom are listed in Section 2.10(c) of the Disclosure Schedule. Except for the Neurologix Assets, theCompany has a valid license to use the Company Licensed Intellectual Property to conduct the activities of the Company in the manner currentlyconducted, subject only to the terms of the agreements listed or required to be listed in Section 2.10(h) of the Disclosure Schedule. Except for theNeurologix Assets, the Company Intellectual Property constitutes all Intellectual Property necessary for the Company to conduct the activities of theCompany in the manner currently conducted by the Company.(d) Protection Measures. The Company has taken reasonable measures to protect the proprietary nature of each item of Company OwnedIntellectual Property which is material to the Company, and to maintain in confidence all trade secrets and confidential information comprising a partof the Company Owned Intellectual Property. No confidential information or trade secrets have been disclosed by the Company to any Person exceptpursuant to valid and appropriate non-disclosure and/or license agreements that have not been breached, or disclosures otherwise protected byattorney-client privilege and a legal duty of confidentiality Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 16 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 owed by the recipient of such confidential information or trade secrets to the Company. The Company has complied in all material respects with allapplicable contractual and legal requirements pertaining to information privacy and security. No complaint relating to an improper use or disclosureof, or a breach in the security of, any such information has been made in writing to the Company or, to the knowledge of the Company, threatenedagainst the Company. To the knowledge of the Company, there has been no unauthorized disclosure of any third party proprietary or confidentialinformation in the possession, custody or control of the Company.(e) Infringement by Company. Except as listed on Section 2.10(e) of the Disclosure Schedule, no past or current activity of the Company(excluding activities of Neurologix) infringes or violates (or has infringed or violated), or constitutes (or constituted) a misappropriation of, anyIntellectual Property rights of any third party. To the knowledge of the Company, no past or current activity of Neurologix infringes or violates (or hasinfringed or violated), or constitutes (or constituted) a misappropriation of, any Intellectual Property rights of any third party. For clarification purposesonly, the foregoing representations and warranties in this subsection (e) expressly exclude any activities by the Surviving Corporation at or after theMerger 1 Effective Time. Section 2.10(e) of the Disclosure Schedule lists any written complaint, claim or notice, or threat of any of the foregoing(including any notification that a license under any patent is or may be required), received by the Company alleging any such infringement, violationor misappropriation by the Company and any request or demand for indemnification or defense received by the Company from any third party; and theCompany has provided to the Buyer copies of all such complaints, claims, notices, requests, demands or threats in its possession relating to any allegedor potential infringement, violation or misappropriation.(f) Infringement of Company Rights. To the knowledge of the Company, no Person is infringing, violating or misappropriating (or hasinfringed, violated or misappropriated) any of the Company Owned Intellectual Property or any Company Licensed Intellectual Property exclusivelylicensed to the Company by any Person. The Company has provided to the Buyer copies of all correspondence in its possession concerning theinfringement, violation or misappropriation of any Company Intellectual Property, except for the Neurologix Assets. The Company has not receivedany correspondence concerning the infringement, violation or misappropriation of any Neurologix Assets, and the Company has no knowledge of anysuch correspondence.(g) Outbound IP Agreements. Section 2.10(g) of the Disclosure Schedule identifies each license, covenant or other agreement (includingany options to license) pursuant to which the Company has assigned, transferred, licensed, distributed or otherwise granted any right or access to anyPerson, or covenanted not to assert any right, with respect to any past, existing or future Company Intellectual Property. Except as disclosed inSection 2.10(g) of the Disclosure Schedule, the Company has no knowledge of the existence, status or enforceability of any license, covenant or otheragreement that may have been entered into by Neurologix (including any options to license) pursuant to which Neurologix may have assigned,transferred, licensed, distributed or otherwise granted any right or access to any Person, or covenanted not to assert any right, with respect to any past,existing or future Neurologix Assets. The Company Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 17 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 has not agreed to indemnify any Person against any infringement, violation or misappropriation of any Intellectual Property rights, except as set forthin the Contracts listed in Section 2.11(a) of the Disclosure Schedule. The Company has no knowledge of the status or enforceability of anyindemnification agreement that may have been entered into by Neurologix. The Company is not a member of or party to any patent pool, industrystandards body, trade association or other organization pursuant to the rules of which it is obligated to license any existing or future IntellectualProperty to any Person.(h) Inbound IP Agreements. Section 2.10(h) of the Disclosure Schedule identifies (i) each item of Company Licensed Intellectual Property(but specifically excluding generally commercially available off the shelf software that has not been modified by or for the Company and is licensed tothe Company for a one-time or annual fee of $10,000 or less) and the license or agreement pursuant to which the Company licenses it or has obtainedan option to license it and (ii) each agreement, contract, assignment or other instrument pursuant to which the Company has obtained any joint or soleownership interest in or to each item of Company Owned Intellectual Property; but in all cases except for the Neurologix Assets. Except as disclosed inSection 2.10(g) of the Disclosure Schedule, the Company has no knowledge of in respect of, the existence, status or enforceability of any license oragreement that may have been entered into by Neurologix.(i) IP Assignments. Except as set forth in Section 2.10(i) of the Disclosure Schedule, each Person who has contributed to the developmentof any Company Owned Intellectual Property (but excluding the Neurologix Assets) has executed a valid and binding written agreement expresslyassigning to the Company all right, title and interest in any inventions and works of authorship, whether or not patentable, invented, created,developed, conceived and/or reduced to practice during the term of such Person’s engagement with the Company, and all Intellectual Property rightstherein.(j) Support and Funding. Except as set forth in Section 2.10(j) of the Disclosure Schedule, the Company has not received any support,funding, resources or assistance from any Governmental Entity, university, military, educational institution or research center and no such Person hasany claim of right to, ownership of or other Security Interest on any Company Owned Intellectual Property.(k) Effect on Buyer. Consummation of the transactions contemplated by this Agreement will not result in (i) the creation of any SecurityInterest on or license to any Intellectual Property that is owned by or licensed to the Buyer or any of its Affiliates prior to the Closing, (ii) Buyer or anyof its Affiliates being bound by or subject to any non-compete or licensing obligation, covenant not to sue, or other restriction on the operation orscope of its business, which Buyer or its Affiliates were not bound by or subject to prior to the Closing, or (iii) Buyer or any of its Affiliates, or theSurviving Corporation, being obligated to pay any royalties, honoraria, fees or other similar payments to any Person (other than Taxes payablepursuant to Section 5.5) in excess of those payable by the Company prior to the Closing.2.11 Contracts. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 18 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (a) Section 2.11(a) of the Disclosure Schedule lists all agreements (whether written or oral) currently in effect (either in whole or in part,including agreements with ongoing post-termination “tails” and ongoing post-termination obligations) to which the Company is a party, butexcluding the Neurologix Assets and any agreements to which Neurologix is or was a party. The Company makes no representations or warranties as towhether or not any agreements related to the Neurologix Assets or to which Neurologix is a party are currently in force and effect. Except as describedin Section 2.11(a) of the Disclosure Schedule, the Company is not a party to:(i) any agreement (or group of related agreements) for the lease of personal property from or to third parties;(ii) any agreement (or group of related agreements) in which the Company has granted manufacturing rights, “most favorednation” pricing provisions or marketing or distribution rights relating to any products or territory or has agreed to purchase aminimum quantity of goods or services or has agreed to purchase goods or services exclusively from a certain party;(iii) any agreement concerning the establishment or operation of a partnership, joint venture or limited liability company;(iv) any agreement (or group of related agreements) under which it has created, incurred, assumed or guaranteed (or may create,incur, assume or guarantee) Indebtedness;(v) any agreement for the disposition of any significant portion of the assets of the Company or any agreement for the acquisitionof the assets or business of any other entity;(vi) any employment, independent contractor or consulting agreement;(vii) any severance, retention, change in control or other similar agreement;(viii) any agreement with a third party concerning the research, development or commercialization of Intellectual Property,confidentiality, noncompetition and/or nonsolicitation;(ix) any agreement with any professional employer organization;(x) any material agreement involving any current or former officer, director or stockholder of the Company or an Affiliate thereof;(xi) any agreement which contains any provisions requiring the Company to indemnify any other party; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 19 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (xii) any agreement that purports to bind or otherwise could bind the Buyer or any Affiliate of the Buyer (other than theCompany) in any way;(xiii) any agreement under which the Company is restricted or prohibited from selling, licensing or otherwise distributing any ofits technology or products, or providing services to, customers or potential customers, or otherwise engaging in any aspect of itsbusiness, in any geographic area, during any period of time or with any Person, or any segment of the market or line of business;(xiv) any Government Contract; or(xv) any other agreement (or group of related agreements) either involving more than $10,000 or not entered into in the OrdinaryCourse of Business.(b) The Company has delivered to the Buyer a complete and accurate copy of each agreement (if any) listed or required to be listed inSection 2.10 of the Disclosure Schedule or Section 2.11(a) of the Disclosure Schedule. With respect to each agreement so listed or required to be listed:(i) the agreement is legal, valid, binding and enforceable against the Company, and to the knowledge of the Company, against each other party thereto,and is in full force and effect, subject to the Bankruptcy and Equity Exception; and (ii) neither the Company nor, to the knowledge of the Company,any other party, is in breach or violation of, or default under, any such agreement, and no event has occurred, is pending or, to the knowledge of theCompany, is threatened, which, after the giving of notice, with lapse of time, or otherwise, would constitute a material breach or default by theCompany or, to the knowledge of the Company, any other party under such agreement.2.12 Neurologix, Inc.(a) The Transfer Statements and the Neurologix Bankruptcy Schedules attached at Annex 2.12 of the Disclosure Schedulelist all Neurologix Assets acquired by the Company pursuant to the Foreclosure Action. To the knowledge of the Company: (i) the assets listed onAnnex 2.12 of the Disclosure Schedule represent all assets that the Neurologix bankruptcy trustee offered for sale by auction in connection with theNeurologix Chapter 7 bankruptcy; and (ii) prior to the sale of assets in connection with its bankruptcy, Neurologix was the true and lawful owner, andhad good title to, all the tangible assets owned and acquired or purported to be owned and acquired by the Company pursuant to the ForeclosureAction, free and clear of all Security Interests except such Security Interests existing at the time of filing of the Neurologix Chapter 7 bankruptcypetition.(b) Attached at Annex 2.12 of the Disclosure Schedule are true and correct copies of: (i) the Transfer Statement, dated April 14, 2016, of theCompany in respect of a portion of the Neurologix Assets; (ii) the Transfer Statement, dated July 13, 2016, of the Company in respect of a portion ofthe Neurologix Assets; and (iii) the Neurologix Bankruptcy Schedules. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 20 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 2.13 Powers of Attorney. There are no outstanding powers of attorney executed on behalf of the Company.2.14 Litigation. There is no Legal Proceeding which is pending or has been threatened in writing against the Company or any of its current orformer officers, directors, employees or consultants (in their respective capacities as such). There are no judgments, orders or decrees outstandingagainst the Company or any of its current or former officers, directors, employees or consultants (in their respective capacities as such). There is noLegal Proceeding initiated by the Company pending, or which the Company has commenced preparations to initiate, against any other Person.2.15 Employees.(a) Other than the Company Stockholders, there are no current or former officers and directors of the Company. Other than the Company’sattorneys and accountants, no person has been paid any compensation by the Company and no person is entitled to any compensation of any kindfrom the Company. The Company has no, and has never had any, employees or any obligations or liabilities to, or in respect of, any employees. TheCompany has never been obligated to pay any payroll Taxes. The Company is not liable for any payment to any trust or other fund or to anyGovernmental Entity, with respect to unemployment compensation benefits, social security or other benefits or obligations for employees (other thanroutine payments to be made in the normal course of business and consistently with past practice). The Company is, and at all times has been, incompliance with all applicable Laws and regulations respecting labor, employment, hiring and termination, fair employment practices, terms andconditions of employment, worker classification (including the proper classification of workers as independent contractors and consultants), workers’compensation, occupational health and safety and wage and hour Laws, and has complied with all employment agreements respecting current and pastemployees of the Company and has complied with all independent contractor and consulting agreements respecting current and former independentcontractors and consultants of the Company. The Company is, and at all times has been, in compliance with the requirements of the ImmigrationReform and Control Act of 1986, respecting current and past employees of the Company. The Company is not a party to any labor agreement with anylabor organization, union, group or association and there are no Company employee unions (nor any other similar labor or employee organizations)under local statutes, custom or practice. There are no controversies pending or, to the knowledge of the Company, threatened, between the Companyand any other person related to employment or consulting matters.(b) The Company is not a party to, and has no commitments or obligations in respect of, any change in control, severance, stay bonuses orother bonuses or similar benefits with respect to the transactions contemplated.(c) All persons who have performed services for the Company while classified as independent contractors have satisfied all Laws to be soclassified, and the Company has fully and accurately reported their compensation on IRS Forms 1099 or other applicable Tax forms for independentcontractors when required to do so. The Company has Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 21 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 provided to the Buyer a true, correct and complete list of all of its current consultants, advisory board members and independent contractors; copies ofall consultant and independent contractor agreements have previously been delivered to the Buyer.2.16 Employee Benefits.(a) The Company does not sponsor, maintain, contribute to, or have any actual or contingent liability with respect to any EmployeeBenefit Plan and has not previously sponsored, maintained, contributed to or had any such actual or contingent liability. The Company neither has norhas had any ERISA Affiliates. No act or omission has occurred and no condition exists with respect to any Employee Benefit Plan that would subjectthe Company to (i) any fine, penalty, tax or liability of any kind imposed under ERISA or the Internal Revenue Code or (ii) any contractualindemnification or contribution obligation protecting any fiduciary, insurer or service provider with respect to any Employee Benefit Plan.(b) Section 2.16(b) of the Disclosure Schedule discloses each: (i) agreement between the Company and any Person (A) the benefits ofwhich are contingent, or the terms of which are altered, upon the occurrence of a transaction involving the Company of the nature of any of thetransactions contemplated by this Agreement, (B) providing any term of employment or engagement or compensation guarantee or (C) providingseverance benefits or other benefits after the termination of the applicable employment or engagement; (ii) agreement, plan or arrangement underwhich any person may receive payments from the Company that may be subject to the tax imposed by Section 4999 of the Internal Revenue Code orincluded in the determination of such person’s “parachute payment” under Section 280G of the Internal Revenue Code; and (iii) agreement or planbinding the Company, including any stock option plan, stock appreciation right plan, restricted stock plan, stock purchase plan, severance benefit,retention or incentive plan or other Employee Benefit Plan, any of the benefits of which will be increased, funded, “grossed-up”, or the vesting of thebenefits of which will be accelerated, or under which any obligation will be forgiven by the occurrence of any of the transactions contemplated by thisAgreement (or in combination with any other event such as employment termination) or the value of any of the benefits of which will be calculated onthe basis of any of the transactions contemplated by this Agreement.(c) There is no corporate-owned life insurance (COLI), split-dollar life insurance policy or any other life insurance policy on the life ofany director, officer, employee or consultant of the Company.(d) No Person is entitled to any accrued vacation, accrued sick time or earned time off from the Company.2.17 Environmental Matters. The Company has complied with all applicable Environmental Laws. There is no pending or, to the knowledge ofthe Company, threatened civil or criminal litigation, written notice of violation, formal administrative proceeding, or, to the knowledge of theCompany, investigation, inquiry or information request, relating to any Environmental Law involving the Company. The Company has no liabilitiesor obligations Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 22 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 arising from the release or threatened release of any Materials of Environmental Concern into the environment. The Company is not a party to or boundby any court order, administrative order, consent order or other agreement entered into in connection with any legal obligation or liability arisingunder any Environmental Law. The Company is not aware of any environmental liability of any solid or hazardous waste transporter or treatment,storage or disposal facility that has been used by the Company.2.18 Legal Compliance. The Company is currently conducting, and has at all times conducted, its activities in compliance with each applicableLaw in all material respects. The Company has not received any written notice or, to the knowledge of the Company, other notice allegingnoncompliance with any applicable Law. The Company has no material liability for failure to comply with any Law and, to the knowledge of theCompany, there is no act, omission, event or circumstance that would reasonably be expected to give rise to any such liability. The Company is notconducting and has not conducted any internal investigation with respect to any actual, potential or alleged violation of any Law.2.19 Permits. Section 2.19 of the Disclosure Schedule sets forth a list of all Permits issued to or held by the Company, if any. Such listed Permitsare the only Permits that are required for the Company to conduct its activities as currently conducted, except where the failure to hold any Permitwould not be material to the Company. Each such Permit is in full force and effect; the Company is in material compliance with the terms of each suchPermit; and, to the knowledge of the Company, no suspension or cancellation of any such Permit is threatened and there is no reasonable basis forbelieving that any such Permit will not be renewable upon expiration.2.20 Unlawful Payments. The Company is and has been in compliance with the Foreign Corrupt Practices Act, 15 U.S.C. §§ 78dd-1, et seq., theOrganization for Economic Cooperation and Development Convention Against Bribery of Foreign Public Officials in International BusinessTransactions and legislation implementing such convention, all other international anti-bribery conventions and all applicable anti-corruption orbribery Laws in any jurisdiction in which the Company has conducted its business (collectively, “Anti-Bribery Laws”). The Company has notreceived any written notice or, to the knowledge of the Company, any other notice that alleges that the Company, or any current or formerrepresentatives of the Company in their capacities as such, is in violation of, or has any liability under, any Anti-Bribery Laws, and no such potentialviolation of Anti-Bribery Laws has been discovered by the Company. The Company has not made and does not anticipate making any disclosures toany Governmental Entity for actual or potential violations of Anti-Bribery Laws. None of the Company’s current or former directors, officers,employees or consultants is currently an officer, agent or employee of a Governmental Entity. The Company has not, nor have its current or formerrepresentatives in their capacities as such, directly or indirectly, offered, given, reimbursed, paid or promised to pay, or authorized the payment of, anymoney or other thing of value (including any fee, gift, sample, travel expense or entertainment) or any commission payment payable to (a) any official,officer, agent, employee or representative of any Governmental Entity or of any existing or prospective customer of the Company (whether or notowned by a Governmental Entity), (b) any political party or official thereof, (c) any Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 23 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 candidate for political or political party office or (d) any other Person affiliated with any such customer, political party or official or political office, ineach case while knowing or having reason to believe that all or any portion of such money or thing of value would be offered, given, reimbursed, paidor promised, directly or indirectly, for unlawful purposes under the Anti-Bribery Laws.2.21 No Customers; Suppliers. The Company has never sold or otherwise supplied any goods or services to any third party. Section 2.21 of theDisclosure Schedule sets forth a list of each supplier that is the sole supplier of any significant product or service to the Company. No such supplier hasindicated within the past year that it will stop, or decrease the rate of, supplying products or services, as applicable, to the Company. The Company hasno purchase order with an unpaid balance.2.22 Certain Business Relationships With Affiliates. Except as set forth in Section 2.22 of the Disclosure Schedule, no Affiliate of the Company(a) owns any property or right, tangible or intangible, which is used in the current activities of the Company, or (b) has any claim or cause of actionagainst the Company, or (c) owes any money to, or is owed any money by, the Company. There have not been any commercial transactions orrelationships between the Company and any Affiliate thereof which occurred or have existed since the Company’s inception.2.23 Brokers’ Fees. The Company has no liability or obligation to pay any fees or commissions to any broker, finder or agent with respect to thetransactions contemplated by this Agreement.2.24 Books and Records. The minute books and/or other similar records of the Company contain complete and accurate records of all actionstaken at any meetings of the Company’s stockholders, Board of Directors or any committee thereof and of all written consents executed in lieu of theholding of any such meeting. The Company has maintained books and/or other similar records with respect to, and which accurately reflect, in allmaterial respects, the assets, business and operations of the Company. Section 2.24 of the Disclosure Schedule contains a list of all bank accounts andsafe deposit boxes of the Company and the names of persons having signature authority with respect thereto or access thereto.2.25 Disclosure. No representation or warranty by the Company contained in this Agreement, and no statement contained in the DisclosureSchedule or any certificate delivered or to be delivered by or on behalf of the Company pursuant to this Agreement, contains or will contain any untruestatement of a material fact or omits or will omit to state any material fact necessary, in light of the circumstances under which it was or will be made, inorder to make the statements herein or therein not misleading. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 24 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 ARTICLE IIIREPRESENTATIONS AND WARRANTIES OF THE COMPANY STOCKHOLDERSEach Company Stockholder represents and warrants to the Buyer, Merger Sub 1 and Merger Sub 2, individually (and not jointly), that thestatements contained in this Article III are true and correct.3.1 Organization and Power. If such Company Stockholder is not a natural Person, such Company Stockholder is a corporation or other entityduly organized, validly existing and in good standing under the laws of the jurisdiction of its formation and such Company Stockholder has allrequisite power and authority to carry on the businesses in which it is engaged and to own and use the properties owned and used by it.3.2 Authorization. Such Company Stockholder has all requisite power and authority to execute and deliver this Agreement and to perform itsobligations hereunder. The execution and delivery by such Company Stockholder of this Agreement and the consummation by such CompanyStockholder of the transactions contemplated hereby have each been duly and validly authorized by all necessary action on the part of such CompanyStockholder. This Agreement has been duly and validly executed and delivered by such Company Stockholder and, assuming due authorization,execution and delivery by the other Parties, constitutes a valid and binding obligation of such Company Stockholder, enforceable against suchCompany Stockholder in accordance with its terms, subject to the Bankruptcy and Equity Exception.3.3 Noncontravention. Subject to the filing of the Certificate of Merger 1 and the Certificate of Merger 2 as required by the Delaware GeneralCorporation Law, none of the execution and delivery by such Company Stockholder of this Agreement, the performance by such CompanyStockholder of any of its obligations hereunder or the consummation by such Company Stockholder of the transactions contemplated hereby, does orwill (a) if such Company Stockholder is not a natural person, conflict with or violate any provision of the organizational documents of such CompanyStockholder, (b) require on the part of such Company Stockholder any notice to or filing with, or any permit, authorization, consent or approval of, anyGovernmental Entity, (c) conflict with, result in a material breach of, constitute (with or without due notice or lapse of time or both) a material defaultunder, result in the acceleration of obligations or loss of any right or benefit under, create in any party the right to terminate, modify or cancel, orrequire any notice, consent or waiver under, any material contract or other instrument to which such Company Stockholder is a party or by which suchCompany Stockholder is bound or to which any of such Company Stockholder’s assets is subject, (d) result in the imposition of any Security Interestupon any of the Company Shares held by such Company Stockholder or (e) violate any order, writ, injunction, decree, statute, rule or regulationapplicable to such Company Stockholder or any of such Company Stockholder’s properties or assets.3.4 Title to Company Shares. Such Company Stockholder holds beneficially and of record all of such Company Stockholder’s Company Sharesas set forth in Section 2.2(b) of the Disclosure Schedule, free and clear of any Security Interests (other than restrictions on transfer Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 25 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 arising under the Securities Act and state or foreign securities Laws). Such Company Stockholder is not a party to any voting trust, proxy, or otheragreement or understanding with respect to the voting or transfer of any Company Shares.3.5 Litigation. There is no Legal Proceeding which is pending or has been threatened in writing against such Company Stockholder thatquestions the validity of this Agreement or any action taken or to be taken by such Company Stockholder in connection herewith or that couldreasonably be expected to adversely affect such Company Stockholder’s ability to consummate the transactions contemplated by this Agreement.3.6 Absence of Intent to Distribute. Such Company Stockholder is acquiring the Buyer Shares for its own account and not with a present viewto, or for sale in connection with, any distribution thereof in violation of the Securities Act; and such Company Stockholder has no present orcontemplated agreement, undertaking, arrangement, obligation, Indebtedness or commitment providing for the disposition thereof. Such CompanyStockholder consents to the placement of the following legend on each certificate for the Buyer Shares until such time as the Buyer Shares are eligiblefor sale pursuant to Rule 144 under the Securities Act:“THE SECURITIES EVIDENCED BY THIS CERTIFICATE HAVE NOT BEEN REGISTERED UNDER THE SECURITIES ACT OF 1933AND MAY NOT BE TRANSFERRED OR SOLD UNLESS (i) A REGISTRATION STATEMENT UNDER SUCH ACT IS THEN IN EFFECTWITH RESPECT THERETO, (ii) A WRITTEN OPINION FROM COUNSEL FOR THE ISSUER OR COUNSEL FOR THE HOLDERREASONABLY ACCEPTABLE TO THE ISSUER HAS BEEN OBTAINED TO THE EFFECT THAT NO SUCH REGISTRATION ISREQUIRED OR (iii) A ‘NO ACTION’ LETTER OR ITS THEN EQUIVALENT HAS BEEN ISSUED BY THE STAFF OF THE SECURITIESAND EXCHANGE COMMISSION WITH RESPECT TO SUCH TRANSFER OR SALE.”The Buyer may also place stop-transfer instructions in respect of the Buyer Shares with respect to such legend.3.7 Restricted Securities.(a) Such Company Stockholder is an “accredited investor” as such term is defined for purposes of Regulation D under the Securities Act.Such Company Stockholder understands that the Buyer Shares will not be registered under the Securities Act for the reason that the issuance of BuyerShares provided for in this Agreement is exempt pursuant to Section 4 of the Securities Act and that the reliance of the Buyer on such exemption ispredicated in part on such Company Stockholder’s representations set forth herein. Such Company Stockholder represents that it is experienced inevaluating companies such as the Buyer, has such knowledge and experience in financial and business matters as to be capable of evaluating the meritsand risks of its investment, and has the financial wherewithal to suffer the total loss of its investment in the Buyer Shares. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 26 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (b) Such Company Stockholder understands that the Buyer Shares may not be sold, transferred or otherwise disposed of withoutregistration under the Securities Act or an exemption therefrom and that in the absence of an effective registration statement covering the Buyer Sharesor an available exemption from registration under the Securities Act, the Buyer Shares must be held indefinitely.3.8 Certain Information. Such Company Stockholder has carefully reviewed this Agreement, including the representations concerning theBuyer contained herein, and understands the information set forth herein. Such Company Stockholder has made detailed inquiry concerning the Buyer,its business and its personnel, and the officers and other representatives of the Buyer have made available to such Company Stockholder any and allwritten information which such Company Stockholder has requested and have answered to such Company Stockholder’s satisfaction all inquiries madeby such Company Stockholder. Such Company Stockholder understands that the Buyer Shares are being or will be issued without any particularoffering or disclosure document. Such Company Stockholder has been represented by such financial, legal and tax counsel and others selected by suchCompany Stockholder as such Company Stockholder has found necessary or appropriate to consult concerning this transaction and to review andevaluate the financial, legal, tax, and other ramifications of an investment in the Buyer, including the tax consequences to such Company Stockholderof an investment in the Buyer Shares. Except as expressly set forth in this Agreement, no representation or warranty of any kind has been made by theBuyer, or any other Person, with respect to the risks or consequences of an investment in the Buyer. Without limiting the generality of the foregoing,except as expressly set forth in this Agreement, neither the Buyer nor any officer, director or other representative of the Buyer has made anyrepresentation or warranty with respect to any business plan, presentation, projection, estimate or budget delivered to or made available to suchCompany Stockholder with respect to future results of operations, future financial condition or the future business and operations of the Buyer.3.9 Not a Bad Actor. Such Company Stockholder has not taken any of the actions set forth in, and is not otherwise subject to, thedisqualification provisions of Rule 506(d)(1) under the Securities Act.3.10 Brokers’ Fees. Such Company Stockholder has no liability or obligation to pay any fees or commissions to any broker, finder or agent withrespect to the transactions contemplated by this Agreement.3.11 Section 368(a). To such Company Stockholder’s knowledge after consultation with its tax counsel, such Company Stockholder has nottaken or agreed to take any action that would prevent the Mergers from qualifying as a reorganization within the meaning of Section 368(a) of theInternal Revenue Code. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 27 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 ARTICLE IVREPRESENTATIONS AND WARRANTIES OF THE BUYER, MERGER SUB 1 AND MERGER SUB 2The Buyer, Merger Sub 1 and Merger Sub 2 represent and warrant to the Company Stockholders (jointly and severally) that the statementscontained in this Article IV are true and correct.4.1 Organization and Corporate Power. The Buyer is a company incorporated under the laws of the Cayman Islands. Merger Sub 1 and MergerSub 2 are each a corporation duly organized, validly existing and in good standing under the Laws of the State of Delaware. The Buyer has all requisitecorporate power and authority to carry on the businesses in which it is engaged and to own and use the properties owned and used by it. The Buyer hasfurnished to the Company complete and accurate copies of the Articles of Association and the Shareholder Agreement, as amended or restated,respectively. The Company is not in default under or in violation of any provision of the Articles of Association. The Company is not in default underor in violation of any provision of the Shareholder Agreement.4.2 Capitalization.(a) The authorized capital stock of the Buyer consists of $50,000 divided into 1,288,327,750 Buyer Capital Shares, of which 27,184,132were issued and outstanding as of July 31, 2018.(b) The table set forth under the heading “Principal Shareholders” set forth in the Company’s prospectus supplement, dated June 7, 2018is accurate in all material respects as of such date. All of the issued and outstanding shares of capital stock of the Buyer have been duly authorized andvalidly issued and are fully paid and nonassessable. All of the issued and outstanding shares of capital stock of the Buyer have been offered, issued andsold by the Buyer in compliance with all applicable Laws.(c) Other than as set forth in Section 4.2(c) of the Disclosure Schedule, no subscription, warrant, option, convertible security or other right(contingent or otherwise) to purchase or acquire any shares of capital stock of the Buyer is authorized or outstanding. The Buyer has no obligation(contingent or otherwise) to issue any subscription, warrant, option, convertible security or other such right, or to issue or distribute to holders of anyshares of its capital stock any evidences of Indebtedness or assets of the Buyer. The Buyer has no obligation (contingent or otherwise) to purchase,redeem or otherwise acquire any shares of its capital stock or any interest therein or to pay any dividend or to make any other distribution in respectthereof. There are no outstanding or authorized stock appreciation, phantom stock or similar rights with respect to the Buyer.(d) Section 4.2(d) of the Disclosure Schedule lists each agreement, written or oral, between the Buyer and any holder of its securities, oramong any holders of its securities, relating to the sale or transfer (including agreements relating to rights of first refusal, co-sale rights or “drag-along”rights), registration under the Securities Act, or voting, of the capital stock of the Buyer. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 28 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (e) There is no claim pending, and to the knowledge of the Buyer, no claim threatened, against the Buyer by any Person that seeks toassert: (i) ownership or rights to ownership of any shares of Buyer capital stock; (ii) any rights of a stockholder, including any option, preemptive rightsor rights to notice or to vote; (iii) any rights under the Articles of Association or the Shareholder Agreement; or (iv) any claim that his, her or its shareshave been wrongfully repurchased by the Buyer.4.3 Operations of Merger Sub 1 and Merger Sub 2. Merger Sub 1 and Merger Sub 2 are each a corporation newly formed by the Buyer for thesole purpose of effecting the Mergers, and has not engaged in any activity other than as contemplated in this Agreement.4.4 Authorization of Transaction. Each of the Buyer, Merger Sub 1 and Merger Sub 2 has all requisite corporate power and authority to executeand deliver this Agreement and to perform its obligations hereunder. The execution and delivery by the Buyer, Merger Sub 1 and Merger Sub 2 of thisAgreement and, subject to obtaining the approval of the Buyer as the sole stockholder of Merger Sub 1 and the approval of Merger Sub 1 as the solestockholder of Merger Sub 2, the consummation by the Buyer, Merger Sub 1 and Merger Sub 2 of the transactions contemplated hereby have been dulyand validly authorized by all necessary corporate action on the part of the Buyer, Merger Sub 1 and Merger Sub 2, respectively. This Agreement hasbeen duly and validly executed and delivered by the Buyer, Merger Sub 1 and Merger Sub 2 and, assuming due authorization, execution and deliveryby the other Parties, constitutes a valid and binding obligation of the Buyer, Merger Sub 1 and Merger Sub 2, enforceable against them in accordancewith its terms, subject to the Bankruptcy and Equity Exception.4.5 Noncontravention. Subject to the filing of the Certificate of Merger 1 and the Certificate of Merger 2 as required by the Delaware GeneralCorporation Law, neither the execution and delivery by the Buyer, Merger Sub 1 or Merger Sub 2 of this Agreement, nor the consummation by theBuyer, Merger Sub 1 or Merger Sub 2 of the transactions contemplated hereby, does or will (a) conflict with or violate any provision of the charter orbylaws of the Buyer, Merger Sub 1 or Merger Sub 2, (b) require on the part of the Buyer, Merger Sub 1 or Merger Sub 2 any filing with, or permit,authorization, consent or approval of, any Governmental Entity, (c) conflict with, result in a material breach of, constitute (with or without due noticeor lapse of time or both) a material default under, result in the acceleration of obligations under, create in any party any right to terminate, modify orcancel, or require any notice, consent or waiver under, any material contract or other instrument to which the Buyer, Merger Sub 1 or Merger Sub 2 is aparty or by which either is bound or to which any of their assets are subject, except for (i) any conflict, breach, default, acceleration, termination,modification or cancellation which would not adversely affect the consummation of the transactions contemplated hereby or (ii) any notice, consent orwaiver the absence of which would not adversely affect the consummation of the transactions contemplated hereby or (d) violate any order, writ,injunction, decree, statute, rule or regulation applicable to the Buyer, Merger Sub 1 or Merger Sub 2 or any of their properties or assets. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 29 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 4.6 Issuance of Buyer Shares. The issuance and delivery of Buyer Shares in accordance with this Agreement has been duly authorized by allnecessary corporate action on the part of the Buyer and, when issued as contemplated hereby, such Buyer Shares shall be duly authorized, duly andvalidly issued, fully paid and nonassessable.ARTICLE VCOVENANTS5.1 Closing Efforts. Each of the Parties shall use its Reasonable Best Efforts to take all actions and to do all things necessary, proper oradvisable to consummate the transactions contemplated by this Agreement, including using its commercially reasonable efforts to ensure that (i) itsrepresentations and warranties remain true and correct in all material respects through, on and as of the Closing Date and (ii) the conditions to theobligations of the other Parties to consummate the Mergers are satisfied.5.2 Governmental and Third-Party Notices and Consents; Bylaws.(a) Each Party shall use its Reasonable Best Efforts to obtain, at its expense, all waivers, permits, consents, approvals or otherauthorizations from Governmental Entities, and to effect all registrations, filings and notices with or to Governmental Entities, as may be required forsuch Party to consummate the transactions contemplated by this Agreement and to otherwise comply with all applicable Laws and regulations inconnection with the consummation of the transactions contemplated by this Agreement.(b) The Company shall use its Reasonable Best Efforts to obtain, at its expense, all such waivers, consents or approvals from third parties,and to give all such notices to third parties, as are listed or required to be listed in Section 6.1 of the Disclosure Schedule.(c) The Company shall not adopt bylaws.5.3 Stockholder Approval. As expeditiously as possible following the execution of this Agreement, the Company shall secure and cause to befiled with the Company written consents in the form of Exhibit A (“Written Consents”) duly executed by all Company Stockholders. The Company,acting through its Board of Directors, shall include in the Written Consent (a) the unanimous recommendation of its Board of Directors that thestockholders of the Company vote in favor of the adoption of this Agreement and the approval of the Mergers and (b) a statement that appraisal rightsare available for the Company Shares pursuant to the Dissenters’ Rights Statute and a copy thereof, or in the alternative, a written waiver of suchappraisal rights duly executed by the applicable Company Stockholder.5.4 Operation of Business. Except as contemplated by this Agreement, during the period from the date of this Agreement to the earlier of thetermination of this Agreement in accordance with its terms or the Merger 1 Effective Time, the Company shall conduct its operations in the OrdinaryCourse of Business and in compliance with all applicable Laws and regulations, and for the avoidance of doubt, during such period shall not incur anyobligations or liabilities in respect of any employees or Employee Benefit Plans. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 30 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 5.5 Tax Matters. The Parties intend that the Mergers be treated as a “reorganization” within the meaning of Section 368(a) of the InternalRevenue Code. However, neither Buyer, Merger Sub 1 nor Merger Sub 2 makes any representation or warranty to the other or to the Company or anyCompany Stockholder regarding the Tax treatment of the Mergers or whether the Mergers will qualify as a “reorganization” under the Internal RevenueCode and any representation or warranty expressed to the contrary under any other provision of this Agreement shall be interpreted consistently withthe provisions of this Section 5.5. Each of the Buyer, Merger Sub 1, Merger Sub 2, the Company and the Company Stockholders acknowledges that itis relying on its own advisors in connection with the Tax treatment of the Mergers and the other transactions contemplated by this Agreement. Each ofthe Buyer, Merger Sub 1, Merger Sub 2, the Company and the Company Stockholders each agree to use their respective commercially reasonableefforts to cause the Mergers to qualify, and will not take any actions not expressly contemplated by this Agreement which could reasonably beexpected to prevent the Mergers from qualifying, as a “reorganization” under Section 368(a)(1)(A) of the Internal Revenue Code. The Parties agree toreport the Mergers for U.S. federal income tax purposes as a “reorganization” within the meaning of Section 368(a) of the Internal Revenue Code(including by attaching the statement described in Treasury Regulations Section 1.368-3(a) on or with its return for the taxable year of the Mergers)unless (i) otherwise required by law or a taxing authority or (ii) the accounting firm preparing the applicable Tax Returns determines that there is noreasonable basis for taking such a position; and in the event of either (i) or (ii), the Parties shall promptly notify the other and shall consult promptlyand in good faith in respect thereof.5.6 Section 280G Matters. The Company is a “small business corporation” as defined in Section 1361(b) of the Internal Revenue Code. To theextent that (i) any “disqualified individual” (as such term is defined for purposes of Section 280G of the Internal Revenue Code) of the Companywould be entitled to any payment or benefit as a result of the transactions contemplated by this Agreement and (ii) such payment or benefit would orcould potentially constitute a “parachute payment” under Section 280G of the Internal Revenue Code or could reasonably be expected to result in theimposition of any excise Tax imposed under Section 4999 of the Internal Revenue Code, prior to the Closing:(a) The Company shall obtain stockholder approval in accordance with the requirements of Section 280G(b)(5)(B) of the InternalRevenue Code and Regulations § 1.280G-1 thereunder (the “280G Shareholder Approval Requirements”) in respect of the portion of such parachutepayment that exceeds three times less one dollar such individual’s “base amount” within the meaning of Section 280G(b)(3) of the Internal RevenueCode with respect to all such “disqualified individuals”;(b) The Company shall provide all required disclosure to all Persons entitled to vote under Section 280G(b)(5)(B)(ii) of the InternalRevenue Code prior to such vote and shall hold a vote of stockholders in the manner intended to satisfy the 280G Shareholder ApprovalRequirements; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 31 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (c) The Company shall obtain any required waivers or consents from the disqualified individual(s) prior to the vote, which vote shallestablish whether the disqualified individual is entitled to receive or retain the waived payments or other waived compensation; and(d) The Buyer and its counsel shall be given the right to review and comment on all documents required to be delivered to the CompanyStockholders in connection with such vote and any required disqualified individual waivers or consents, and the Company shall reflect all reasonablecomments of the Buyer thereon. The Buyer and its counsel shall be provided copies of all documents executed by the Company Stockholders anddisqualified individuals in connection with the vote.5.7 No Claim Against the Company. Effective as of the Closing, each Company Stockholder, by its execution and delivery of this Agreementand/or the Written Consent, hereby (a) waives any and all rights of indemnification, contribution and other similar rights against the Company(whether arising pursuant to any charter document of the Company, any contract, applicable Law or otherwise) arising out of the representations,warranties, covenants and agreements contained in this Agreement and/or out of the negotiation, execution or performance of this Agreement, andagrees that any claim of the Buyer, whether for indemnity or otherwise, may be asserted directly against the Company Stockholders or any CompanyStockholder (solely to the extent, and subject to the limitations, provided in this Agreement), without any need for any claim against, or joinder of, theCompany and (b) forever waives, releases and discharges (and hereby agrees to cause each of its representatives to forever waive, release and discharge)with prejudice the Company from any and all claims, rights (including rights of indemnification, contribution and other similar rights, from whateversource, whether under contract, law or otherwise), causes of action, protests, suits, disputes, orders, obligations, debts, demands, proceedings, contracts,agreements, promises, liabilities, controversies, costs, expenses, fees (including attorneys’ fees), or damages of any kind, arising by any means(including subrogation, assignment, reimbursement, operation of law or otherwise), whether known or unknown, suspected or unsuspected, accrued ornot accrued, foreseen or unforeseen, or mature or unmature related or with respect to, in connection with, or arising out of, directly or indirectly, anyevent, fact, condition, circumstance, occurrence, act or omission that was in existence (or that occurred or failed to occur) at or prior to the Closing;provided, however, this clause (b) shall not be construed as releasing any Party from its obligations otherwise expressly set forth in this Agreement orany agreement delivered pursuant to this Agreement (including, without limitation, the Buyer indemnification obligations set forth in Section 7.1(d)).ARTICLE VICONDITIONS TO CONSUMMATION OF THE MERGER6.1 Conditions to Obligations of the Buyer, Merger Sub 1 and Merger Sub 2. The obligation of each of the Buyer, Merger Sub 1 and MergerSub 2 to consummate the Mergers is subject to the satisfaction (or waiver by the Buyer) of the following conditions:(a) the Written Consent evidencing the Requisite Stockholder Approval shall have been duly executed and delivered to the Buyer; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 32 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (b) the Company shall have obtained at its own expense (and shall have provided copies thereof to the Buyer of) all of the waivers,permits, consents, approvals or other authorizations, and effected all of the registrations, filings, notices and contract terminations as are listed inSection 6.1 of the Disclosure Schedule or are otherwise required on the part of the Company to effect the Mergers, if any;(c) the representations and warranties of the Company and the Company Stockholders set forth in this Agreement shall be true andcorrect, in each case as of the date of this Agreement and as of the Closing as though made as of the Closing, except to the extent such representationsand warranties are specifically made as of a particular date (in which case such representations and warranties shall be true and correct as of such date);(d) each of the Company and the Company Stockholders shall have performed or complied with, in all material respects, its agreementsand covenants required to be performed or complied with under this Agreement as of or prior to the Closing;(e) no Legal Proceeding shall be pending or threatened in writing wherein an unfavorable judgment, order, decree, stipulation orinjunction could reasonably be expected to (i) prevent consummation of the transactions contemplated by this Agreement, (ii) cause the transactionscontemplated by this Agreement to be rescinded following consummation or (iii) have, individually or in the aggregate, a Company Material AdverseEffect, and no such judgment, order, decree, stipulation or injunction shall be in effect;(f) the Company shall have delivered to the Buyer, Merger Sub 1 and Merger Sub 2 the Company Closing Certificate;(g) the Company shall have delivered to the Buyer a certificate executed by the Secretary of the Company certifying that attached theretoare (i) a true, complete and correct copy of the Certificate of Incorporation, as in effect on the Closing Date, and, in the case of the Certificate ofIncorporation, certified by the Secretary of State of the State of Delaware, (ii) true, complete and correct copies of resolutions unanimously adopted bythe Company’s Board of Directors and stockholders, authorizing the execution and delivery of this Agreement, the transactions contemplated herebyand the performance by the Company of its obligations hereunder, which resolutions have not been modified, rescinded or revoked, and (iii) aspecimen signature of the duly authorized officer of the Company signing this Agreement on behalf of the Company;(h) the Company shall have delivered to the Buyer a certificate, issued by the Secretary of State of the State of Delaware and each otherjurisdiction in which the Company is qualified to do business, if any, certifying as of a date no more than five (5) business days prior to the ClosingDate that the Company is in good standing under the Laws of such jurisdiction;(i) the Buyer shall have received copies of the resignations, effective as of the Closing, of each director and officer of the Company fromsuch positions as a director or officer, as applicable; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 33 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (j) the Company shall have delivered to the Buyer evidence reasonably satisfactory to the Buyer that all contracts and other arrangementslisted on Schedule 6.1(j) have been terminated on terms reasonably satisfactory to the Buyer;(k) the Company shall have delivered to the Buyer a certification in form and substance reasonably satisfactory to the Buyer that theCompany Shares are not United States real property interests as defined in Section 897(c) of the Internal Revenue Code, together with a notice to theInternal Revenue Service, in accordance with the Treasury Regulations under Sections 897 and 1445 of the Internal Revenue Code;(l) the Company Stockholders and, if requested by the Company Stockholders, a limited liability company or other entity wholly ownedby the Company Stockholders shall have entered into a consulting agreement, in substantially the form attached hereto as Exhibit B (the “ConsultingAgreement”), with the Buyer (or a wholly-owned subsidiary of the Buyer).6.2 Conditions to Obligations of the Company. The obligation of the Company and the Company Stockholders to consummate the Mergers issubject to the satisfaction of the following conditions:(a) the representations and warranties of the Buyer, Merger Sub 1 and Merger Sub 2 set forth in this Agreement shall be true and correct,in each case as of the date of this Agreement and as of the Closing as though made as of the Closing, except to the extent such representations andwarranties are specifically made as of a particular date (in which case such representations and warranties shall be true and correct as of such date);(b) each of the Buyer, Merger Sub 1 and Merger Sub 2 shall have performed or complied with, in all material respects, its agreements andcovenants required to be performed or complied with under this Agreement as of or prior to the Closing;(c) no Legal Proceeding shall be pending or threatened in writing wherein an unfavorable judgment, order, decree, stipulation orinjunction would (i) prevent consummation of the transactions contemplated by this Agreement, (ii) cause the transactions contemplated by thisAgreement to be rescinded following consummation or (iii) have, individually or in the aggregate, a material adverse effect on the ability of the Buyer,Merger Sub 1 and Merger Sub 2 to perform their respective obligations under this Agreement or consummate the transactions contemplated by thisAgreement, and no such judgment, order, decree, stipulation or injunction shall be in effect;(d) the Buyer, Merger Sub 1 and Merger Sub 2 shall have delivered to the Company the Buyer Closing Certificate;(e) the Buyer shall have delivered to the Company a certificate executed by the Secretary of each of the Buyer, Merger Sub 1 and MergerSub 2 certifying that attached thereto are (i) a true, complete and correct copy of the Articles of Association of the Buyer, the Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 34 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Certificate of Incorporation of Merger Sub 1 and the Certificate of Incorporation of Merger Sub 2, as in effect on the Closing Date, and, in the case ofthe Certificate of Incorporation, certified by the Secretary of State of the State of Delaware, (ii) true, complete and correct copies of resolutions duly andvalidly adopted by the Buyer’s, Merger Sub 1’s and Merger Sub 2’s Boards of Directors, authorizing the execution and delivery of this Agreement, thetransactions contemplated hereby and the performance by the Buyer, Merger Sub 1 and Merger Sub 2, respectively, of their obligations hereunder,which resolutions have not been modified, rescinded or revoked, and (iii) a specimen signature of the duly authorized officer of the Buyer, Merger Sub1 and Merger Sub 2 signing this Agreement on behalf of the Buyer, Merger Sub 1 and Merger Sub 2, respectively;(f) the Buyer shall have delivered to the Company a certificate, issued by the Secretary of State of the State of Delaware, certifying as of adate no more than five (5) business days prior to the Closing Date that Merger Sub 1 and Merger Sub 2 are in good standing under the Laws of suchjurisdiction; and(g) the Buyer (or a wholly-owned subsidiary of the Buyer) shall have entered into the Consulting Agreement with the CompanyStockholders and, if requested by the Company Stockholders, a limited liability company or other entity wholly owned by the Company Stockholders.ARTICLE VIIINDEMNIFICATION7.1 Indemnification by the Company Stockholders and the Buyer.(a) Expressly subject to the conditions and limitations set forth in this Article VII, the Company Stockholders shall severally and jointlydefend and indemnify the Buyer in respect of, and hold it harmless against and will compensate and reimburse the Buyer for, any and all Lossesincurred or suffered by the Surviving Corporation or the Buyer or any Affiliate thereof (regardless of whether such Losses relate to any Third-PartyAction) resulting from, relating to or constituting:(i) any inaccuracy in any representation or breach of any warranty of the Company contained in Article II or in the CompanyClosing Certificate, whether as of the date of this Agreement or as of the Closing Date;(ii) any breach or nonperformance of (or noncompliance with) any covenant or agreement of the Company or any CompanyStockholder contained in this Agreement to the extent required to be performed at or prior to the Closing;(iii) any inaccuracy in the Allocation Schedule; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 35 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (iv) any claim by a stockholder or former stockholder of the Company, or any other Person, seeking to assert, or based upon:(A) ownership or rights to ownership of any shares of stock of the Company; (B) any rights of a stockholder (other than the right toreceive the amounts payable to such holder pursuant to Article I), including any option, preemptive rights or rights to notice or tovote; (C) any rights under the Certificate of Incorporation of the Company; (D) any claim based on or arising out of any breach offiduciary duty by the Company’s Board of Directors or stockholders; or (E) any claim that his, her or its shares were wrongfullyrepurchased by the Company; in each case, which claim arises from acts or omissions of the Company or the CompanyStockholders prior to the Closing Date;(v) the following Taxes: (A) any Taxes for any taxable period (or portion thereof) ending on or before the Closing Date due andpayable by the Company, which Taxes shall be apportioned to the pre-closing portion of such taxable period, in the case of ataxable period that includes but does not end on the Closing Date, (1) in the case of property or similar ad valorem Taxes, based onthe number of days in the portion of such taxable period ending on the Closing Date as compared to the number of days in suchtaxable period, and (2) in the case of all other Taxes, on a closing of the books basis as of the Closing Date; (B) any Taxes for whichthe Company has any liability under Treasury Regulation Section 1.1502-6 or under any comparable or similar provision of state,local or foreign Laws as a result of being a member of an affiliated, consolidated, combined, unitary or similar group on or prior tothe Closing Date; (C) any Taxes for which the Company has any liability as a transferee or successor, pursuant to any contractualobligation or otherwise, which Tax is attributable to the operations of the Company on or prior to the Closing Date or an event ortransaction occurring before the Closing; and (D) any transfer, sales, use, stamp, conveyance, value added, recording, registration,documentary, filing and other non-income Taxes and administrative fees (including notary fees) imposed on or assessed against theCompany prior to the Closing; provided, however, that the Company Stockholders shall have no liability, and shall not indemnifythe Buyer, for any Taxes resulting from any actions or elections with respect to any tax period (or portion thereof) ending on orprior to the Closing Date made by Buyer, the Intermediate Surviving Corporation, Merger Sub 1, Merger Sub 2 or the SurvivingCorporation as of or after the Merger 1 Effective Time;(vi) to the extent not taken into account in determining the number of Closing Shares, any amounts that become payable (inwhole or in part) by the Company to any Person as a result of the execution and delivery of this Agreement or the consummation ofthe transactions contemplated hereby, whether taken alone or in combination with any other event; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 36 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (vii) any fraud, intentional or knowing misrepresentation or willful breach on the part of the Company in connection with thetransactions contemplated by this Agreement, but excluding any breach or alleged breach under the Consulting Agreement, whichshall be resolved pursuant to the terms of the Consulting Agreement;(viii) any claim arising from any agreement listed on Section 7.1(a)(viii) of the Disclosure Schedule; or(ix) any Indebtedness or trade payables of the Company as of immediately prior to the Closing in excess of $10,000.(b) Each Company Stockholder shall individually (and not jointly) defend and indemnify the Buyer in respect of, and hold it harmlessagainst and will compensate and reimburse the Buyer for, any and all Losses incurred or suffered by the Surviving Corporation or the Buyer or anyAffiliate thereof (regardless of whether such Losses relate to any Third-Party Action) resulting from, relating to or constituting: (i) any inaccuracy inany representation or breach of any warranty of such Company Stockholder contained in Article III or in the Company Closing Certificate, whether asof the date of this Agreement or as of the Closing Date; (ii) any breach or nonperformance of (or noncompliance with) any covenant or agreement ofsuch Company Stockholder contained in this Agreement; or (iii) any fraud, intentional or knowing misrepresentation or willful breach on the part ofsuch Company Stockholder in connection with the transactions contemplated by this Agreement, but excluding any breach or alleged breach under theConsulting Agreement, which shall be resolved pursuant to the terms of the Consulting Agreement.(c) [intentionally omitted].(d) The right to indemnification, payment, reimbursement, or other remedy based upon any representation, warranty, covenant orobligation pursuant to this Section 7.1 will not be affected by any investigation conducted or any knowledge acquired at any time, whether before orafter the execution and delivery of this Agreement or the Closing Date, with respect to the accuracy or inaccuracy of, or compliance with, suchrepresentation, warranty, covenant, or obligation.(e) Expressly subject to the conditions and limitations set forth in this Article VII, the Buyer shall defend and indemnify each of theCompany Stockholders in respect of, and hold each of them harmless against and will compensate and reimburse each of the Company Stockholdersfor, any and all Losses incurred or suffered by any of them (regardless of whether such Losses relate to any Third-Party Action) resulting from, relatingto or constituting:(i) any inaccuracy in any representation or breach of any warranty of the Buyer contained in Article II or in the Buyer ClosingCertificate, whether as of the date of this Agreement or as of the Closing Date; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 37 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (ii) any breach or nonperformance of (or noncompliance with) any covenant or agreement of the Buyer or the SurvivingCorporation contained in this Agreement to the extent required to be performed at or prior to the Closing;(iii) any fraud, intentional or knowing misrepresentation or willful breach on the part of the Buyer or the Surviving Corporation inconnection with the transactions contemplated by this Agreement but excluding any breach or alleged breach under the ConsultingAgreement, which shall be resolved pursuant to the terms of the Consulting Agreement; or(iv) any Third-Party Action against the Surviving Corporation in respect of a claim for which the Company Stockholders have noindemnification obligations to the Buyer pursuant to Section 7.1(a) or (b) above; provided, however, that for purposes of thisSection 7.1(e)(iv) only, the representations and warranties of the Company and the Company Stockholders shall be deemed tosurvive indefinitely.7.2 Indemnification Claims.(a) A Person entitled to indemnification under this Article VII (an “Indemnified Party”) shall give reasonably prompt written notificationto any other Person obligated to indemnify the Indemnified Party (an “Indemnifying Party”) of the commencement, or threatened commencement, ofany Third-Party Action that the Indemnified Party reasonably expects may result in a claim for indemnification pursuant to this Article VII. Forpurposes of notices and communications with respect to the Company Stockholders as potential indemnitees or indemnitors pursuant to this ArticleVII, “Indemnified Party” and “Indemnifying Party” shall mean the Company Stockholder Representative on behalf of the Company Stockholders. Suchnotification shall be given reasonably promptly after receipt by the Indemnified Party of notice of such Third-Party Action or threatened Action, andshall describe in reasonable detail (to the extent known by the Indemnified Party) the facts constituting the basis for such Third-Party Action and theamount of the claimed damages; provided, however, that no delay or failure on the part of the Indemnified Party in so notifying the Indemnifying Partyshall relieve the Indemnifying Party of any liability or obligation hereunder except to the extent of any damage or liability caused by or arising out ofsuch failure.(b) The Indemnifying Party shall be entitled, at its own and sole expense, to participate in any defense of such Third-Party Action. TheIndemnified Party shall have the right in its sole and absolute discretion to control the defense of any Third-Party Action against the Indemnified Partyand the Indemnified Party shall thereafter from time to time promptly provide to the Indemnifying Party copies of all pleadings filed and all ordersissued in such Third-Party Action, and shall consult in good faith with the Indemnifying Party at reasonable periodic intervals on matters regarding thedefense and potential settlement of such Third-Party Action. The Indemnified Party shall have the right to settle, adjust or compromise any such Third-Party Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 38 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Action, subject to Indemnifying Party’s right to dispute its obligation to indemnify; provided, however, that except with the prior written consent ofthe Indemnifying Party (which consent shall not be unreasonably withheld, delayed or conditioned), no settlement of any such Third-Party Action withthird-party claimants shall be determinative of any Losses relating to such matter.(c) In order to seek indemnification under this Article VII, the Indemnified Party shall, as promptly as reasonably practicable, deliver aClaim Notice to the Indemnifying Party.(d) Within twenty (20) business days after delivery of a Claim Notice, the Indemnifying Party shall deliver to the Indemnified Party aresponse (a “Response”), in which the Indemnifying Party shall either: (i) agree that the Indemnified Party is entitled to receive the Claimed Amount orthe Agreed Amount or (ii) dispute that the Indemnified Party is entitled to receive any of the Claimed Amount.(e) During the twenty (20)-business-day period following the delivery of a Response that reflects a Dispute, the Indemnifying Party andthe Indemnified Party shall use good faith efforts to resolve the Dispute. If the Dispute is not resolved within such twenty (20)-business-day period,either Party may submit the Dispute to a court of competent jurisdiction in accordance with the terms of this Agreement. In the event of a violation ofSection 7.1(a)(vii) or Section 7.1(b)(iii) by the Company or the Company Stockholders, which violation has been determined by a court or othercompetent judicial authority, in a final non-appealable order or decision, then the Indemnifying Party shall deliver to the Indemnified Party, promptlyfollowing the resolution of the Dispute (whether by mutual agreement, judicial decision or otherwise), payment of any amount required to be paid tothe Indemnified Party consistent with the terms of the resolution of the Dispute, to the extent not satisfied via the forfeiture of the right to be issuedHoldback Shares and, if applicable, the forfeiture and cancellation of Buyer Shares pursuant to Section 7.4.(f) The Company Stockholder Representative shall have full power and authority on behalf of each Company Stockholder to take anyand all actions on behalf of, execute any and all instruments on behalf of, and execute or waive any and all rights of, the Company Stockholders underthis Article VII. The Company Stockholder Representative shall have no liability to any Company Stockholder for any action taken or omitted onbehalf of the Company Stockholders pursuant to this Article VII.7.3 Survival of Representations and Warranties. Each Party’s representations and warranties in this Agreement shall survive the Closing andshall expire at 11:59 p.m., Eastern Time, on the date that is 18 months after the Closing Date; provided, however, that the representations andwarranties contained in Sections, 2.5, 2.14, 2.15, 2.22, 3.6, 3.7, 3.8 and 3.9, shall survive until the date that is 24 months after the Closing Date;provided, further, that the representations and warranties contained in Sections 2.1, 2.2, 2.3, 2.4(a), 2.8, 2.10, 3.1, 3.2, 3.3, 3.10, 4.1, 4.2, 4.3, 4.4 and4.5(a) shall survive until the date that is six (6) years after the Closing Date. If an Indemnified Party delivers to the Indemnifying Party, beforeexpiration of a Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 39 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 representation or warranty, either a Claim Notice based upon a breach of such representation or warranty, or an Expected Claim Notice based upon abreach of such representation or warranty, then the applicable representation or warranty shall survive until, but only for purposes of, the resolution ofany claims arising from or related to the matter covered by such notice. Except as provided above with respect to indemnification obligations underSection 7.1(a)(v), nothing in this Section 7.3 shall be construed to limit the survival of covenants, agreements and obligations that by their terms are tobe performed or observed after the Merger 1 Effective Time or for which another time period is specified in this Agreement.7.4 Limitations and Related Matters.(a) (a) The exclusive source for the recovery of any Losses for which the Buyer is entitled toindemnification under Section 7.1 (other than Section 7.1(a)(v), Section 7.1(a)(vii), Section 7.1(a)(viii), and Section 7.1(b)(iii)) shall be expresslylimited to the forfeiture and cancellation of any Holdback Shares issuable to the Company Stockholders pursuant to this Agreement (and recovery ofan amount equal to any proceeds received by any Company Stockholder in respect of the sale, transfer or other disposition thereof (assuming for allpurposes that the proceeds per share are equal to the Buyer Share Value)).(b) For purposes of determining the number of Buyer Shares that the Buyer may be entitled to recover pursuant to this Article VII, eachBuyer Share shall be deemed to have a value equal to Buyer Share Value, and the number of shares recoverable shall be rounded down to the nearestwhole share.(c) [intentionally omitted].(d) Other than for claims involving fraud, intentional or knowing misrepresentation or willful breach of this Agreement, but excludingany breach or alleged breach under the Consulting Agreement, which shall be resolved pursuant to the terms of the Consulting Agreement, and subjectto the provisions of Section 10.13, from and after the Closing, the remedies set forth in this Article VII shall be the sole and exclusive remedies of theParties with respect to any breach of the respective representations, warranties, covenants and agreements pursuant to this Agreement or otherwisearising in connection with the transactions contemplated by this Agreement, including the enforcement, interpretation or negotiation of thisAgreement. For clarification purposes only, no Company Stockholder shall have any personal or individual liability to Buyer or any other Personunder this Agreement, except for the Company Stockholder indemnification obligations to Buyer under this Article VII, as expressly limited by thisSection 7.4.(e) Nothing in this Section 7.4 shall be construed to limit the Parties’ rights under Section 10.13. No Company Stockholder shall haveany right of contribution against the Company or the Surviving Corporation with respect to any breach by the Company of any of its representations,warranties, covenants or agreements.7.5 Tax Treatment of Indemnification Payments. All amounts paid under this Article VII shall be treated as adjustments to the purchase price forall Tax purposes unless otherwise required by Law. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 40 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 ARTICLE VIIITERMINATION8.1 Termination of Agreement. The Parties may terminate this Agreement prior to the Closing (whether before or after the Requisite StockholderApproval), as provided below:(a) the Parties may terminate this Agreement by mutual written consent;(b) the Buyer may terminate this Agreement by giving written notice to the Company in the event the Company is in breach of anyrepresentation, warranty or covenant contained in this Agreement, and such breach (i) individually or in combination with any other such breach,would cause the conditions set forth in Section 6.1(c) or Section 6.1(d) not to be satisfied and (ii) is not cured within 10 days following delivery by theBuyer to the Company of written notice of such breach;(c) the Company may terminate this Agreement by giving written notice to the Buyer in the event the Buyer, Merger Sub 1 or Merger Sub2 is in breach of any representation, warranty or covenant contained in this Agreement, and such breach (i) individually or in combination with anyother such breach, would cause the conditions set forth in Section 6.2(a) or Section 6.2(b) not to be satisfied and (ii) is not cured within 10 daysfollowing delivery by the Company to the Buyer of written notice of such breach;(d) the Buyer may terminate this Agreement by giving written notice to the Company if the Closing shall not have occurred on or beforethe date that is forty-five (45) days after the date of this Agreement, by reason of the non-satisfaction of any condition precedent under Section 6.1(unless the non-occurrence of the Closing results primarily from a breach or nonperformance by the Buyer, Merger Sub 1 or Merger Sub 2 of anyrepresentation, warranty, covenant or agreement contained in this Agreement);(e) the Buyer may terminate this Agreement if the Company shall have failed to deliver evidence of the satisfaction of the condition setforth in Section 6.1(a) within four (4) hours after the execution and delivery of this Agreement; or(f) the Company may terminate this Agreement by giving written notice to the Buyer if the Closing shall not have occurred on or beforethe date that is forty-five (45) days after the date of this Agreement, by reason of the non-satisfaction of any condition precedent under Section 6.2(unless the non-occurrence of the Closing results primarily from a breach by the Company of any representation, warranty, covenant or agreementcontained in this Agreement).8.2 Effect of Termination. If any Party terminates this Agreement pursuant to Article IX, all obligations of the Parties hereunder shall terminatewithout any liability of any Party to any other Party (except for any liability of any Party for intentional breaches of this Agreement prior to suchtermination). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 41 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 ARTICLE IXCERTAIN DEFINITIONSCapitalized terms used and not otherwise defined in this Agreement shall have the respective meanings set forth below:“Affiliate” shall mean any affiliate, as defined in Rule 12b-2 under the Exchange Act.“Agreed Amount” shall mean part, but not all, of the Claimed Amount.“Agreement” shall have the meaning set forth in the first paragraph of this Agreement.“Allocation Schedule” shall mean the schedule identified as Schedule A in the Disclosure Schedules, which sets forth (i) the name and address ofeach Company Stockholder, (ii) the number of Company Shares held by such Company Stockholder, (iii) the number of Closing Shares allocable tosuch Company Stockholder, (iv) the number of Holdback Shares allocable to such Company Stockholder, (v) the proportion of each MilestonePayment allocable to such Company Stockholder, and (vi) the proportion of each Royalty Payment allocable to such Company Stockholder.“Arbitrator” shall have the meaning set forth in Section 10.12.“Articles of Association” shall mean the articles of association of the Buyer, as amended and restated.“Buyer Capital Shares” shall mean the ordinary shares of the Buyer with a nominal value of $0.00003881 per ordinary share; provided that ifthe Buyer has experienced a Change of Control, then Buyer Capital Shares shall mean the shares of capital stock, membership interests or similarequity securities in the entity that is the successor-in-interest of the Buyer following such Change of Control.“Buyer Closing Certificate” shall mean a certificate to the effect that each of the conditions specified in Section 6.2(a) through Section 6.2(c) issatisfied in all respects.“Buyer Covered Person” means, with respect to the Buyer as an “issuer” for purposes of Rule 506 promulgated under the Securities Act, anyPerson listed in the first paragraph of Rule 506(d)(1).“Buyer Material Adverse Effect” shall mean any material adverse change, event, circumstance, occurrence, state of facts or development that,individually or in the aggregate with all other changes, events, circumstances, occurrences, states of facts or developments occurring prior to thedetermination of a Buyer Material Adverse Effect, has a material adverse effect on, (i) the business, assets, prospects, Intellectual Property, liabilities,capitalization, condition (financial or other), or results of operations of the Buyer and its Subsidiaries, taken as a whole, or (ii) the ability of the Buyer,Merger Sub 1 or Merger Sub 2 to consummate the transactions contemplated hereby; provided, however, that any adverse effects attributable to any Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 42 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 of the following as they relate to the Buyer shall not be deemed to constitute, and the following shall not be taken into account in determining whetherthere has been or will be, a Buyer Material Adverse Effect: (a) changes after the date of this Agreement in conditions affecting the industries in whichthe Buyer or any of its Subsidiaries participates or the U.S. economy or financial markets as a whole (other than those that disproportionately affect theBuyer and its Subsidiaries, taken as a whole); (b) compliance by the Buyer with the terms of, or the taking of any action required by, this Agreement, orotherwise taken by the Buyer at the Company’s or any Company Stockholder’s express written direction; (c) any change in GAAP or applicable Laws(or interpretation thereof); and (d) any acts of God, calamities, acts of war or terrorism, or national or international political or social conditions.“Buyer Shares” shall mean the Buyer Capital Shares issuable to the Company Stockholders pursuant to this Agreement.“Buyer Share Value” shall mean: (i) if the Buyer Shares are not publicly traded on a national securities exchange, the fair market value asdetermined in good faith by the Company’s Board of Directors and (ii) if the Buyer Shares are publicly traded on a national securities exchange, theaverage three (3) day closing price per Buyer Share for the three (3) days immediately prior to the date of determination of value as reported by suchnational securities exchange, in all cases, as adjusted for any stock split, stock dividend, combination, or other recapitalization or reclassificationeffected after the date hereof.“CERCLA” shall mean the federal Comprehensive Environmental Response, Compensation and Liability Act of 1980, as amended.“Certificate of Incorporation” shall mean the certificate of incorporation of the Company, Merger Sub 1 or Merger Sub 2, as the contextrequires, and as amended or restated.“Certificate of Merger 1” shall mean the certificate of merger or other appropriate documents prepared and executed in accordance withSection 251(c) of the Delaware General Corporation Law to effect Merger 1.“Certificate of Merger 2” shall mean the certificate of merger or other appropriate documents prepared and executed in accordance withSection 251(c) of the Delaware General Corporation Law to effect Merger 2.“Change of Control” shall mean any transaction or series of related transactions involving: (i) the sale, lease, exclusive license, or otherdisposition of all or substantially all of the assets of the Buyer, (ii) any merger or consolidation of the Buyer into or with another person or entity (otherthan a merger or consolidation effected with an Affiliate of the Buyer or to change the Buyer’s domicile), or any other corporate reorganization, inwhich the shareholders of the Buyer in their capacity as such immediately prior to such merger, consolidation or reorganization, own less than amajority of the Buyer’s (or the surviving or successor entity’s) outstanding voting power on a fully-diluted basis immediately after such merger,consolidation or reorganization; or (iii) any sale or other transfer by the shareholders in which the shareholders Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 43 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 of the Buyer in their capacity as such immediately prior to such sale or transfer own less than a majority of the Buyer’s (or the surviving or successorentity’s) outstanding voting power on a fully-diluted basis immediately after such sales or transfer.“Claim Notice” shall mean a written notification which contains (i) a description of the Losses incurred or reasonably expected to be incurred bythe Indemnified Party and the Claimed Amount of such Losses, to the extent then known, (ii) a statement that the Indemnified Party is entitled toindemnification under Article VII for such Losses and a reasonable explanation of the basis therefor, and (iii) a demand for payment in the amount ofsuch Losses.“Claimed Amount” shall mean the amount of any Losses incurred or reasonably expected to be incurred by the Indemnified Party.“Closing” shall mean the closing of the transactions contemplated by this Agreement.“Closing Date” shall mean the first date on which all of the conditions to the obligations of the Parties to consummate the transactionscontemplated hereby (excluding the delivery at the Closing of any of the documents set forth in Article VI) are satisfied or waived, or such other date asmay be mutually agreeable to the Parties.“Company” shall mean Vector Neurosciences Inc., a/k/a and f/k/a “Vector Neurosciences LLC”, a Delaware corporation, provided that noreferences to the “Company” shall be deemed or interpreted to be a reference to Neurologix, except as otherwise expressly stated.“Company Closing Certificate” shall mean a certificate to the effect that each of the conditions specified in Section 6.1(a) throughSection 6.1(e) is satisfied in all respects.“Company Debt” shall mean the aggregate amount of (i) any Indebtedness of the Company, (ii) any guaranties or arrangements having theeconomic effect of a guaranty by the Company of any Indebtedness of any other Person, and (iii) any accrued interest or penalties on any of theforegoing.“Company Intellectual Property” shall mean the Company Owned Intellectual Property and the Company Licensed Intellectual Property.“Company Licensed Intellectual Property” shall mean all Intellectual Property that is licensed to the Company by any third party or that theCompany is otherwise permitted by any third party to use.“Company Material Adverse Effect” shall mean any material adverse change, event, circumstance, occurrence, state of facts or developmentthat, individually or in the aggregate with all other changes, events, circumstances, occurrences, states of facts or developments occurring prior to thedetermination of a Company Material Adverse Effect, has a material adverse effect on, (i) the business, assets, prospects, Intellectual Property,liabilities, capitalization, condition (financial or other), or results of operations of the Company, taken as a Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 44 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 whole, or (ii) the ability of the Company to consummate the transactions contemplated hereby; provided, however, that any adverse effects attributableto any of the following as they relate to the Company shall not be deemed to constitute, and the following shall not be taken into account indetermining whether there has been or will be, a Company Material Adverse Effect: (a) changes after the date of this Agreement in conditions affectingthe industries in which the Company participates or the U.S. economy or financial markets as a whole (other than those that disproportionately affectthe Company); (b) compliance by the Company with the terms of, or the taking of any action required by, this Agreement, or otherwise taken by theCompany at the Buyer’s express written direction; (c) any change in GAAP or applicable Laws (or interpretation thereof); and (d) any acts of God,calamities, acts of war or terrorism, or national or international political or social conditions.“Company Owned Intellectual Property” shall mean all Intellectual Property owned or purported to be owned by the Company, in whole or inpart.“Company Registrations” shall mean, collectively, all Company Owned Intellectual Property that is Registered.“Company Shares” shall mean the shares of common stock, $0000100000 par value per share, of the Company.“Company Stockholders” shall mean the holders of record of the Company Shares outstanding immediately prior to the Merger 1 EffectiveTime.“Consulting Agreement” shall have the meaning set forth in Section 6.1(l).“Contract Dispute” shall have the meaning set forth in Section 10.11.“Contract Dispute Notice” shall have the meaning set forth in Section 10.12.“Covered Products” means the GAD Products and the IP-related Covered Products.“Dispute” shall mean the dispute resulting if the Indemnifying Party in a Response disputes the Indemnifying Party’s liability for all or part ofthe Claimed Amount.“Dissenters’ Rights Statute” shall mean Section 262 of the Delaware General Corporation Law.“Employee Benefit Plan” shall mean (a) all employee benefit plans as defined in Section 3(3) of ERISA; and (b) all other pension, retirement,group insurance, severance pay, stay or retention bonus, deferred compensation, excess or supplemental benefit, vacation, stock, stock option, equity-based compensation, phantom stock, plant closing benefits, patent award programs, salary continuation or insurance for disability, consulting, or othercompensation arrangements, workers’ compensation, fringe benefit and incentive plans, programs, or arrangements, life insurance, tuitionreimbursement or scholarship programs, employee discount programs, meals, travel, or vehicle allowances, any plans subject to Section 125 of theInternal Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 45 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Revenue Code and any plans providing benefits or payments in the event of a change of control, change in ownership or effective control, or sale of asubstantial portion (including all or substantially all) of the assets of any business or portion thereof which pertain to any current or former employee,director, officer or independent contractor of the Company and as to which the Company is a sponsor, a party or by which it is bound or with respect towhich the Company has or may have any liability.“Environmental Law” shall mean any Law relating to the environment, occupational health and safety, or exposure of Persons or property toMaterials of Environmental Concern, including any statute, regulation, administrative decision or order pertaining to: (i) the presence of or thetreatment, storage, disposal, generation, transportation, handling, distribution, manufacture, processing, use, import, export, labeling, recycling,registration, investigation or remediation of Materials of Environmental Concern or documentation related to the foregoing; (ii) air, water or noisepollution; (iii) surface water, groundwater or soil contamination; (iv) the release, threatened release, or accidental release of Materials of EnvironmentalConcern, including emissions, discharges, injections, spills, escapes or dumping of Materials of Environmental Concern; (v) transfer of interests in orcontrol of real property which may be contaminated; (vi) community or worker right-to-know disclosures with respect to Materials of EnvironmentalConcern; (vii) the protection of wild life, marine life and wetlands, and endangered and threatened species; and (viii) storage tanks, vessels, containers,abandoned or discarded barrels and other closed receptacles. As used above, the term “release” shall have the meaning set forth in CERCLA.“ERISA” shall mean the Employee Retirement Income Security Act of 1974, as amended.“ERISA Affiliate” shall mean any entity that is, or at any applicable time was, a member of (i) a controlled group of corporations (as defined inSection 414(b) of the Internal Revenue Code), (ii) a group of trades or businesses under common control (as defined in Section 414(c) of the InternalRevenue Code), or (iii) an affiliated service group (as defined under Section 414(m) of the Internal Revenue Code or the regulations underSection 414(o) of the Internal Revenue Code), any of which includes or included the Company.“Exchange Act” shall mean the Securities Exchange Act of 1934, as amended.“Expected Claim Notice” shall mean a notice that, as a result of a Legal Proceeding instituted by or written claim made by a third party, theIndemnified Party reasonably expects to incur Losses for which it is entitled to indemnification under Article VII.“FDA” means the United States Food and Drug Administration and any successor thereto.“First Commercial Sale” means, with respect to any country, the first sale of a GAD Product to a third party purchaser after Regulatory Approvalis granted with respect to such country for such GAD Product. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 46 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 “Foreclosure Action” means, collectively, (i) the auction of certain assets formerly owned by Neurologix on April 14, 2016, (ii) the secondauction of certain intellectual property assets formerly owned by Neurologix on July 13, 2016, and (iii) the acquisition of such assets by the Companythrough the exercise by the Company of its right, as a secured creditor, to credit bid at each foregoing auction.“EMA” means the European Medicines Agency and any successor thereto.“GAD Products” means any products produced by the Buyer or its Affiliates (directly or through any third party contract manufacturers) thatuses the adeno-associated virus gene therapy encoding glutamic acid decarboxylase (formerly known as NLX-P101) for the treatment of Parkinson’sdisease that was previously under development by Neurologix.“GAAP” shall mean United States generally accepted accounting principles, consistently applied.“Government Contract” shall mean any prime contract or subcontract between the Company and any Governmental Entity, any primecontractor to a Governmental Entity or any subcontractor to any such Governmental Entity or prime contractor.“Governmental Entity” shall mean any domestic or foreign court, arbitrational tribunal, administrative agency or commission or othergovernmental or regulatory authority or agency.“Hill Records” means the documents and records, and any equipment and other tangible personal property, formerly owned by Neurologix andheld in storage at Hill Archive in West Berlin, New Jersey.“Indebtedness” shall mean, without duplication, with respect to any Person (i) all obligations for borrowed money or extensions of credit(including bank overdrafts and advances), (ii) all obligations evidenced by bonds, debentures, notes or other similar instruments, (iii) all obligations topay the deferred purchase price of property or services, except trade accounts payable arising in the Ordinary Course of Business, (iv) all obligations aslessee capitalized in accordance with GAAP, (v) all obligations of others secured by a Security Interest on any asset, whether or not such obligations areassumed, (vi) all obligations, contingent or otherwise, directly or indirectly guaranteeing any obligations of any other Person, all obligations toreimburse the issuer in respect of letters of credit or under performance or surety bonds, or other similar obligations, (vii) all obligations in respect ofbankers’ acceptances and under reverse repurchase agreements, and (viii) all obligations in respect of futures contracts, swaps, other financial contractsand other similar obligations (determined on a net basis as if such contract or obligation was being terminated early on such date).“Intellectual Property” shall mean all of the following throughout the world and all rights therein and thereto: (i) Patent Rights; (ii) Trademarksand all goodwill in the Trademarks; (iii) copyrights, designs, data and database rights and registrations and applications for registration thereof,including moral rights of authors; (iv) inventions, invention disclosures, Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 47 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 statutory invention registrations, trade secrets and confidential business information, know-how, manufacturing and product processes and techniques,research and development information, financial, marketing and business data, pricing and cost information, business and marketing plans andcustomer and supplier lists and information, whether patentable or nonpatentable, whether copyrightable or noncopyrightable and whether or notreduced to practice; (v) computer software and (vi) all other intellectual property and proprietary rights recognized in any country or jurisdictions inthe world, including proprietary rights relating to any of the foregoing (including remedies against infringement thereof and rights of protection ofinterest therein under the Laws of all jurisdictions).“Internal Revenue Code” shall mean the Internal Revenue Code of 1986, as amended.“Intermediate Surviving Corporation” shall mean the Company, as the surviving corporation in Merger 1.“IP-related Covered Products” means any products (other than the GAD Products) produced by the Buyer or its Affiliates (directly or throughany third party contract manufacturers) that (a) uses or incorporates adeno-associated virus gene therapy encoding glutamic acid decarboxylase to treata disease other than Parkinson’s disease; or (b) (i) uses any of the Intellectual Property listed on Exhibit C, and (ii) is covered by a Valid Claim.“JAMS” shall have the meaning set forth in Section 10.12.“knowledge” with respect to the Company and the Company Stockholders, and in the context of phrases such as “to the knowledge of theCompany,”, “to the knowledge of the Company Stockholders”, “the Company is not aware”, “the Company has no knowledge” or any phrase ofsimilar import, shall be deemed to refer to the actual knowledge of each of Michael G. Kaplitt, Matthew During and Stephen B. Kaplitt, after(a) performing a reasonable review of the books, records, files, email and other correspondence and other documents and information of the Companyto which such Person has access or such other documents and information expressly specified in this Agreement and (b) consultation with theCompany’s outside advisors; provided, however, that with respect to clause (a), the Parties acknowledge that (i) no inspection of the Hill Records hasbeen undertaken by Michael G. Kaplitt, Matthew During or Stephen B. Kaplitt, (ii) no such inspection by the Company Stockholders is required by theBuyer for purposes of this Agreement, and (iii) except for any actual knowledge held by Michael G. Kaplitt, Matthew During and Stephen B. Kaplitt,neither the Company nor the Company Stockholders shall be deemed to have “knowledge” of any matter solely because such information is containedin the Hill Records.“knowledge” with respect to the Buyer, Merger Sub 1 and Merger Sub 2, and in the context of phrases such as “to the knowledge of Buyer,”, “theBuyer is not aware”, “the Buyer has no knowledge” or any phrase of similar import, shall be deemed to refer to the actual knowledge of AlexandriaForbes and Rich Giroux after (a) performing a reasonable review of the books, records, files, email and other correspondence and other documents andinformation of the Buyer, Merger Sub 1 and Merger Sub 2 to which such Person has access or such other documents and information expresslyspecified in this Agreement and (b) consultation with the Buyer’s, Merger Sub 1’s and Merger Sub 2’s outside advisors. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 48 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 “Law” shall mean any applicable domestic or foreign law, order, judgment, rule, code, statute, regulation, requirement, variance, decree, writ,injunction, award, ruling, Permit or ordinance of any Governmental Entity, including the common law and any applicable stock exchange rule orrequirement.“Legal Proceeding” shall mean any action, audit, suit, proceeding, claim, arbitration or investigation before any Governmental Entity or beforeany arbitrator.“Losses” shall mean any and all debts, obligations and other liabilities (whether absolute, accrued, contingent, fixed or otherwise, known orunknown, or due or to become due, or otherwise), judgments, monetary damages, Taxes, fines, fees, penalties, interest obligations, deficiencies,expenses (including amounts paid in settlement, interest, court costs, reasonable costs of investigators, reasonable fees and expenses of attorneys,accountants, financial advisors and other experts, and other reasonable expenses of litigation, arbitration or other dispute resolution proceedingsrelating to a Third-Party Action or an indemnification claim under Article VII), but excluding any punitive, incidental, consequential, special or,indirect damages or damages relating to diminution in value or lost profits, unless paid or payable to a third party.“Materials of Environmental Concern” shall mean any: pollutants or contaminants (as such terms are defined under the Clean Water Act, 33U.S.C. Section 401 et seq.) or hazardous substances (as such terms are defined under CERCLA), pesticides (as such term is defined under the FederalInsecticide, Fungicide and Rodenticide Act), solid wastes and hazardous wastes (as such terms are defined under the Resource Conservation andRecovery Act), chemicals, other hazardous, radioactive or toxic materials, oil, petroleum and petroleum products (and fractions thereof), listed orsubject to regulation under any Law, statute, rule, regulation, order, Permit, or directive due to its potential, directly or indirectly, to harm theenvironment or the health of humans or other living beings.“Merger 1 Effective Time” shall mean the time at which Merger Sub 1 files the Certificate of Merger 1 with the Secretary of State of the State ofDelaware.“Merger 2 Effective Time” shall mean the time at which Merger Sub 2 files the Certificate of Merger 2 with the Secretary of State of the State ofDelaware.“Net Sales” means the gross amount received or receivable by Buyer (or any Affiliate, assignee or licensee of Buyer) for sales of a GAD Productto a third party purchaser, less the following to the extent included in such sale or otherwise actually allowed or incurred with respect to such sales:(a) discounts, including cash, trade and quantity discounts, price reduction programs, retroactive price adjustments with respect to sales of a GADProduct, charge-back payments and rebates granted to managed health care organizations or to federal, state and local governments (or their respectiveagencies, purchasers and reimbursers) or to trade customers, including but not limited to, wholesalers and chain and pharmacy buying groups, providedthat Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 49 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 all of the foregoing shall be (i) negotiated and offered on an arm’s length basis in accordance with the Buyer’s customary business practices, (ii) offeredon terms that are customary or usual for the relevant industry or product market, and (iii) otherwise permitted by applicable law; (b) credits orallowances for rejections or returns of GAD Products, including for recalls or damaged goods; (c) freight, postage, shipping and insurance charges fordelivery of GAD Products, to the extent billed and paid; (d) customs duties, surcharges and other governmental charges incurred in connection with theexportation or importation of a GAD Product; (e) costs due to the factoring of receivables; (f) any write-downs or bad debts for uncollected receivables;and (g) taxes, duties or other governmental charges levied on, absorbed or otherwise imposed on sale of GAD Products, including value-added taxes, orother governmental charges otherwise measured by the billing amount, when included in billing, as adjusted for rebates and refunds, but specificallyexcluding taxes based on net income of the seller; in each case, only if the foregoing deductions are customary and reasonable, and are calculated inaccordance with GAAP, consistently applied.For sake of clarity and avoidance of doubt, sales by the Buyer of a GAD Product to a third party distributor of such GAD Product in a givencountry will be considered a sale to a third party purchaser. Any GAD Products that are: used (but not sold for consideration) for promotional oradvertising purposes (including free samples); used for clinical or other research purposes; or supplied as part of a compassionate use program (or otherprogram for providing a GAD Product before it has received Regulatory Approval in a country), will in each case not be considered in determining NetSales hereunder.If a GAD Product is sold as a combined product or service, Net Sales, for purposes of determining royalty payments on such GAD Product,will be calculated by multiplying the Net Sales of the combined product or service by the fraction A/A+B, in which A is the gross selling price (in theapplicable country) of the GAD Product portion of the combined product or service when such GAD Product is sold separately during the applicableaccounting period in which the sales of the combined product were made, and B is the gross selling price (in the applicable country) of the otherproducts or services, as the case may be, of the combined product and/or service sold separately during the accounting period in question. All grossselling prices of the components of the combined product or service will be calculated as the average gross selling price of the components during theapplicable accounting period for which the Net Sales are being calculated.In any country, if no separate sale of either such above-designated GAD Product or such above designated elements of the end-user productand/or service are made during the accounting period in which the sale was made, or if gross retail selling price for an active functional element,component or service, as the case may be, cannot be determined for an accounting period, Net Sales allocable to the GAD Product in each such countrywill be determined by Buyer in good faith based on an equitable method of determining same that takes into account, on a country-by-country basis,variations in potency, the relative contribution of each active agent, component or service, as the case may be, in the combination, and relative valueto the end user of each active agent, component or service, as the case may be. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 50 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 “Neurologix” means Neurologix, Inc., a Delaware corporation.“Neurologix Assets” means all assets (including all Intellectual Property and the Hill Records) formerly owned by Neurologix that were acquiredby the Company pursuant to the Foreclosure Action.“Neurologix Bankruptcy Schedules” means the schedules listing assets, liabilities and creditors in the Neurologix bankruptcy case filed inDelaware (Case No.12-10936 (CSS)) and attached at Annex 2.12 of the Disclosure Schedule.“Ordinary Course of Business” shall mean the ordinary course of business consistent with past practice (including with respect to generaltiming, frequency and amount).“Patent Rights” shall mean all patents, patent applications (including provisional patent applications), utility models, design registrations andcertificates of invention and other governmental grants for the protection of inventions or industrial designs (including all related continuations,continuations-in-part, divisionals, reissues and reexaminations).“Permits” shall mean all permits, licenses, registrations, certificates, orders, approvals, franchises, variances, waivers and similar rights issued byor obtained from any Governmental Entity (including those issued or required under Environmental Laws).“Person” shall mean a natural person, partnership (general or limited), corporation, limited liability company, business trust, joint stockcompany, trust, unincorporated association, joint venture, Governmental Entity or other entity or organization.“Reasonable Best Efforts” shall mean best efforts, to the extent commercially reasonable.“Registered” shall mean issued by, registered with, renewed by or the subject of a pending application before any Governmental Entity orInternet domain name registrar.“Regulatory Approval” means, with respect to a Covered Product, receipt or issuance of all approvals, licenses, registrations, clearances andauthorizations by the FDA or EMA necessary to lawfully market and sell such GAD Product in the United States or Europe, as applicable.“Royalty” and “Royalties,” shall mean periodic payments to the Company Stockholders in the nature of royalties, calculated as a percentage ofNet Sales of GAD Products, provided that such payments shall be payable to the Company Stockholders only to the extent the Buyer actually receivespayments or proceeds on such Net Sales.“Royalty Term” means, as to the GAD Products, the period beginning on the First Commercial Sale of such GAD Product in any country in theworld and ending upon the 10th anniversary of the Buyer’s receipt of Net Sales in respect of such First Commercial Sale of such GAD Product in suchcountry. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 51 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 “Securities Act” shall mean the Securities Act of 1933, as amended.“Security Interest” shall mean any mortgage, pledge, security interest, encumbrance, charge or other lien (whether arising by contract or byoperation of law), except that, for purposes of this Agreement (other than Article III), the following shall not constitute “Security Interests”: (i) allstatutory or other liens for current Taxes or assessments which are not yet due and payable; (ii) all landlords’, workmen’s, repairmen’s, warehousemen’sand carriers’ liens and other similar liens imposed by Law, incurred in the Ordinary Course of Business; (iii) all Laws and judgments, orders, decrees,stipulations or injunctions; and (iv) all pledges or deposits in connection with workers compensation, unemployment insurance and other socialsecurity legislation.“Shareholder Agreement” means the Shareholder Agreement, dated as of June 7, 2018, between Buyer, and the individual shareholders partythereto.“Subsidiary” shall mean any corporation, partnership, trust, limited liability company or other non-corporate business enterprise in which theCompany (or another Subsidiary) holds stock or other ownership interests representing (a) more than 50% of the voting power of all outstanding stockor ownership interests of such entity or (b) the right to receive more than 50% of the net assets of such entity available for distribution to the holders ofoutstanding stock or ownership interests upon a liquidation or dissolution of such entity.“Surviving Corporation” shall mean Merger Sub 2, as the surviving corporation in Merger 2.“Taxes” shall mean any and all taxes, charges, fees, duties, contributions, levies or other similar assessments or liabilities in the nature of a tax,including income, gross receipts, corporation, ad valorem, premium, value-added, net worth, capital stock, capital gains, documentary, recapture,alternative or add-on minimum, disability, registration, recording, excise, real property, personal property, sales, use, license, lease, service, service use,transfer, withholding, employment, unemployment, insurance, social security, national insurance, business license, business organization,environmental, workers compensation, payroll, profits, severance, stamp, occupation, escheat, windfall profits, customs duties, franchise, estimated andother taxes of any kind whatsoever imposed by the United States of America or any state, local or foreign government, or any agency or politicalsubdivision thereof, and any interest, fines, penalties, assessments or additions to tax imposed with respect to such items or any contest or disputethereof.“Tax Returns” shall mean any and all reports, returns (including information returns), declarations, or statements relating to Taxes, includingany schedule or attachment thereto and any related or supporting workpapers or information with respect to any of the foregoing, including anyamendment thereof filed with or submitted to any Governmental Entity in connection with the determination, assessment, collection or payment ofTaxes or in connection with the administration, implementation or enforcement of or compliance with any legal Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 52 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 requirement relating to any Tax, and including, for the avoidance of doubt, U.S. Department of the Treasury Form TD F 90-22.1.“Third-Party Action” shall mean any lawsuit or Legal Proceeding by a Person other than a Party for which indemnification may be sought by aParty under Article VII.“Trademarks” shall mean all trademarks and service marks, logos, Internet domain names, corporate names, doing business designations andother indicia of commercial source or origin (whether registered, common law, statutory or otherwise), together with all goodwill associated therewith,and all registrations and applications for registration of the foregoing.“Valid Claim” means a claim in an issued patent listed on Exhibit C that has not: (a) expired or been canceled; (b) been declared invalid by anunreversed and unappealable or unappealed decision of a court or other appropriate body of competent jurisdiction; (c) been admitted to be invalid orunenforceable through reissue, disclaimer or otherwise; or (d) been abandoned.ARTICLE XMISCELLANEOUS10.1 Press Releases and Announcements. Neither the Company (prior to the Closing) nor any Company Stockholder shall issue any pressrelease or public announcement relating to the subject matter of this Agreement without the prior written approval of the Buyer. The Buyer shall givethe Company Stockholder Representative a reasonable opportunity to review the press release initially announcing the Closing prior to the issuancethereof, and the Buyer shall give reasonable consideration to any comments of the Company Stockholder Representative relating to such press releaseif such comments are received by the Buyer within 24 hours of providing a draft of such press release to the Company Stockholder Representative.10.2 Further Assurances; Post-Closing Cooperation. At any time or from time to time after the Closing, at the request of any Party, the otherParties shall execute and deliver to the requesting Party such other documents and instruments, provide such materials and information and take suchother actions as the requesting Party may reasonably request to consummate the transactions contemplated by this Agreement and otherwise to causethe other Party or Parties to fulfill its or their respective obligations under this Agreement and the transactions contemplated hereby.10.3 Third-Party Beneficiaries. The terms and provisions of this Agreement are intended solely for the benefit of the Parties and their respectivesuccessors or permitted assigns, and it is not the intention of the Parties to confer third-party beneficiary rights, and this Agreement does not confer anysuch rights, upon any other Person.10.4 Entire Agreement. This Agreement (including the documents referred to herein) constitutes the entire agreement among the Parties andsupersedes any prior understandings, Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 53 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 agreements or representations by or among the Parties, written or oral, with respect to the subject matter hereof; provided, however, that the Sectionsentitled “Exclusivity” and “Confidentiality” pursuant to that certain term sheet, dated June 28, 2016, between the Company and the Buyer shallsurvive until the Merger 1 Effective Time, at which time it shall terminate.10.5 Succession and Assignment. This Agreement shall be binding upon and inure to the benefit of the Parties named herein and theirrespective successors and permitted assigns. No Party may assign any of its rights or delegate any of its obligations hereunder without the prior writtenapproval of the other Parties; provided that Merger Sub 1 and Merger Sub 2 may assign its rights, interests and obligations hereunder to an Affiliate ofthe Buyer without the consent of any Party. For the avoidance of doubt, the Buyer or the Surviving Corporation may, without the consent of any Party,(a) assign all or any part of its rights or obligations hereunder to any Person (whether or not an Affiliate of the Buyer) in connection with a merger orconsolidation of the Buyer or the sale, lease, exclusive license or other disposition of all or substantially all of the assets of the Buyer and (b) grantlicenses of the Covered Product to Buyer’s Affiliates or any Third Parties. Any purported assignment of rights or delegation of performance obligationsin violation of this Section 10.5 is void.10.6 Counterparts and Facsimile Signature. This Agreement may be executed in two or more counterparts, each of which shall be deemed anoriginal but all of which together shall constitute one and the same instrument. This Agreement may be executed by facsimile signature.10.7 Headings. The Section headings contained in this Agreement are inserted for convenience only and shall not affect in any way themeaning or interpretation of this Agreement.10.8 Notices. All notices, requests, demands, claims, and other communications hereunder shall be in writing. Any notice, request, demand,claim or other communication hereunder shall be deemed duly delivered four (4) business days after it is sent by registered or certified mail, returnreceipt requested, postage prepaid, or one business day after it is sent for next business day delivery via a reputable nationwide overnight courierservice, or on the same business day (if sent before 2 p.m. local time in the time zone of the recipient’s physical address (as specified below) andotherwise on the next business day) if sent by fax with electronic or telephonic confirmation of receipt, in each case to the intended recipient as setforth below:If to the Company (prior to the Closing)or to the Company StockholderRepresentative or any CompanyStockholder:Stephen B. Kaplitt[contact information] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 54 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 If to the Buyer, Merger Sub 1 or Merger Sub 2 or(after the Closing) the Company: MeiraGTx Holdings plc430 East 29th Street, 10th FloorNew York, NY 10016Attn: Richard GirouxTel: (646) 490-2971Email: rich@meiragtx.com With a copy (which shall not constitute notice) to: Latham & Watkins LLPJohn Hancock Tower200 Clarendon StreetBoston, MA 02116Attention: Peter N. Handrinos, Esq.Tel: (617) 948-6060Fax: (617) 948-6001Email: Peter.Handrinos@lw.comAny Party may give any notice, request, demand, claim or other communication hereunder using any other means (including personal delivery,expedited courier, messenger service, or ordinary mail), but no such notice, request, demand, claim or other communication that is given by such othermeans shall be deemed to have been duly given unless and until it actually is received by the party for whom it is intended. In addition, any notice,request, demand, claim or other communication hereunder delivered by email and acknowledged by reply email from the recipient shall be deemed tobe effective delivery hereunder. Any Party may change the address to which notices, requests, demands, claims, and other communications hereunderare to be delivered by giving the other Parties notice in the manner herein set forth.10.9 Amendments and Waivers. The Parties may mutually amend any provision of this Agreement at any time prior to the Closing; provided,however, that any amendment effected subsequent to the Requisite Stockholder Approval shall be subject to any restrictions contained in the DelawareGeneral Corporation Law. No amendment of any provision of this Agreement shall be valid unless the same shall be in writing and signed by all of theParties. No waiver of any right or remedy hereunder shall be valid unless the same shall be in writing and signed by the Party giving such waiver. Nowaiver by any Party with respect to any default, misrepresentation or breach of warranty or covenant hereunder shall be deemed to extend to any prioror subsequent default, misrepresentation or breach of warranty or covenant hereunder or affect in any way any rights arising by virtue of any prior orsubsequent such occurrence.10.10 Severability; Invalid Provisions. If any provision of this Agreement is finally judicially determined to be illegal, invalid or unenforceableunder any present or future Law, and if the rights or obligations of any Party under this Agreement will not be materially and adversely affectedthereby, (a) such provision shall be fully severable, (b) this Agreement shall be construed and enforced as if such illegal, invalid or unenforceableprovision had never comprised a part hereof, (c) the remaining provisions of this Agreement shall remain in full force and effect and shall not beaffected by the illegal, invalid or unenforceable provision or by its severance herefrom, and (d) in lieu of such illegal, invalid or unenforceableprovision, there shall be added automatically as a part of this Agreement a legal, valid and enforceable provision as similar in terms to such illegal,invalid or unenforceable provision as may be possible. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 55 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 10.11 Governing Law. This Agreement (and all claims, grievances, demands, controversies, causes of action or disputes of any naturewhatsoever (including, but not limited to, tort and contract claims, and claims upon any law, statute, order, or regulation (“Contract Disputes”)), shallbe governed in all respects, including validity, interpretation, and effect, by and construed in accordance with the internal Laws of the State ofDelaware without giving effect to any choice or conflict of Law provision or rule (whether of the State of Delaware or any other jurisdiction) that wouldcause the application of Laws of any jurisdictions other than those of the State of Delaware.10.12 Arbitration. It is understood and agreed between the parties hereto that any and all Contract Disputes, arising out of, in connection with,or in relation to (a) this Agreement, or (b) questions of arbitrability under this Agreement, shall be resolved by final, binding, nonjudicial arbitration inaccordance with the Federal Arbitration Act, 9 U.S.C. Section 1, et seq. pursuant to the following procedures:(a) Any party may send another party or parties written notice identifying the matter in dispute and invoking the procedures of thisSection (the “Contract Dispute Notice”). Within 14 days from delivery of the Dispute Notice, each party involved in the dispute shall meet at amutually agreed location in New York, New York, for the purpose of determining whether they can resolve the dispute themselves by writtenagreement, and, if not, whether they can agree upon an impartial third-party arbitrator (the “Arbitrator”) to whom to submit the matter in dispute forfinal and binding arbitration.(b) If such parties fail to resolve the dispute by written agreement or agree on the Arbitrator within the later of fourteen (14) days from anysuch initial meeting or within thirty (30) days from the delivery of the Dispute Notice, any such party may make written application to the JudicialArbitration and Mediation Services (“JAMS”), in New York, New York for the appointment of a single Arbitrator to resolve the dispute by arbitration.At the request of JAMS the parties involved in the dispute shall meet with JAMS at its offices within ten (10) days of such request to discuss the disputeand the qualifications and experience which each party respectively believes the Arbitrator should have; provided, however, that the selection of theArbitrator shall be the exclusive decision of JAMS and shall be made within thirty (30) days of the written application to JAMS. The Arbitrator shall bea disinterested party.(c) Within thirty (30) days of the selection of the Arbitrator, the parties involved in the dispute shall meet in New York, New York withsuch Arbitrator at a place and time designated by such Arbitrator after consultation with such parties and present their respective positions on thedispute. Each party shall have no longer than one day to present its position, the entire proceedings before the Arbitrator shall be no more than threeconsecutive days, and the decision of the Arbitrator shall be made in writing no more than thirty (30) days following the end of the proceeding. Suchan award shall be a final and binding determination of the dispute and shall be fully enforceable as an arbitration decision in any court havingjurisdiction and venue over such parties. The parties shall bear their own legal fees and expenses, and shall share equally fees and expenses of theArbitrator and JAMS, regardless of the outcome. The non-prevailing party or parties (as determined by the Arbitrator) shall pay the Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 56 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Arbitrator’s fees. Except as may be required by applicable law (or as required to enter and enforce the judgment in court), no party (or its representative,witnesses or arbitrators) may disclose the existence, content or result of any arbitration under this Agreement without the prior written consent of theother party (or parties) involved in such arbitration.10.13 Specific Performance. The Parties hereto agree that irreparable damage would occur to the Parties in the event that any of the provisionsof this Agreement were not performed in accordance with its specific terms or were otherwise breached. It is agreed that the Parties shall be entitled toan injunction or injunctions to prevent breaches of this Agreement and to enforce specifically the terms and provisions hereof in any court of theUnited States or any state having jurisdiction without the requirement of posing a bond or other security, this being in addition to any other remedy towhich the Parties are entitled at law or in equity.10.14 Construction.(a) The language used in this Agreement shall be deemed to be the language chosen by the Parties to express their mutual intent, and norule of strict construction shall be applied against any Party.(b) Any reference to any federal, state, local or foreign statute or Law shall be deemed also to refer to all rules and regulationspromulgated thereunder, unless the context requires otherwise.(c) Any reference to any Article, Section or paragraph shall be deemed to refer to an Article, Section or paragraph of this Agreement,unless the context clearly indicates otherwise.(d) The Parties hereto agree that this Agreement is the product of negotiation between sophisticated parties and individuals, all of whomwere represented by counsel, and each of whom had an opportunity to participate in and did participate in the drafting of each provision hereof.Accordingly, ambiguities in this Agreement, if any, shall not be construed strictly or in favor of or against any Party but rather shall be given a fair andreasonable construction without regard to the rule of contra proferentem.(e) Unless the context of this Agreement otherwise requires, (i) words of either gender or the neuter include the other gender and theneuter, (ii) words using the singular number also include the plural number and words using the plural number also include the singular number,(iii) the terms “hereof,” “herein,” “hereby” and derivative or similar words refer to this entire Agreement as a whole and not to any particular Article,Section or other subdivision, (iv) the terms “Article” or “Section” or other subdivision refer to the specified Article, Section or other subdivision of thebody of this Agreement, (v) the words “include,” “includes,” “including” and other similar words shall be deemed to be followed by the phrase “butnot limited to,” (vi) when a reference is made in this Agreement to Exhibits, such reference shall be to an Exhibit to this Agreement unless otherwiseindicated, (vii) for any document or other item to have been “made available” heretofore or prior to the execution or date of this Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 57 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Agreement such document or other item must be deposited at least twenty-four (24) hours prior to the date hereof (or if deposited more recently,provided notice of such deposit and a copy of thereof was given to the Buyer) into the data room heretofore established by the Company with writtennotice of such deposit and a copy of such deposit was made to the Buyer and (viii) all references to “dollars” or “$” shall mean United States dollars.All accounting terms used herein and not expressly defined herein shall have the meanings given to them under GAAP, unless otherwise expresslystated. When used herein, the terms “Party” or “Parties” refer to the Buyer, Merger Sub 1 and Merger Sub 2, on the one hand, and the Company, on theother hand, and the terms “third party,” “third-party” or “third parties” refers to Persons other than the Buyer, Merger Sub 1 and Merger Sub 2, on theone hand, and the Company, on the other hand.(f) The drafting and negotiation of the representations, warranties, covenants and conditions to the obligations of the Company, theBuyer, Merger Sub 1 and Merger Sub 2 herein reflect compromises, and certain provisions may overlap with other provisions or may address the sameor similar subject matters in different ways or for different purposes. It is the intention of the Parties that, to the extent possible, unless provisions are bytheir terms mutually exclusive and effect cannot be given to both or all such provisions, (i) the representations, warranties, covenants and closingconditions in this Agreement shall be construed to be cumulative, (ii) each representation, warranty, covenant and closing condition in this Agreementshall be given full separate and independent effect, and (iii) no limitation in or exception to any representation, warranty, covenant or closingcondition shall be construed to limit or apply to any other representation, warranty, covenant or closing condition unless such limitation or exceptionis expressly made applicable to such other representation, warranty, covenant or closing condition. Subject to Section 7.4, all remedies, either underthis Agreement or by law or otherwise afforded, shall be cumulative and not alternative.10.15 Expenses. The Company Stockholders, jointly and severally, shall bear all costs and expenses (including investment banking, legal andaccounting fees and expenses) incurred by the Company or any Company Stockholder in connection with this Agreement and the transactionscontemplated hereby. The Buyer shall bear all costs and expenses (including investment banking, legal and accounting fees and expenses) incurred bythe Buyer in connection with this Agreement and the transactions contemplated hereby. For clarification purposes only, except as otherwise expresslyprovided herein (including, without limitation, the parties’ respective indemnification obligations), the Company and the Company Stockholders shallbe responsible for all costs and expenses of the Company incurred prior to the Closing, and the Buyer, Merger Sub 1, Merger Sub 2 and the SurvivingCorporation shall be responsible for all costs and expenses of the Company incurred on or after the Closing.[Remainder of Page Intentionally Left Blank] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. - 58 - Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 IN WITNESS WHEREOF, the Parties have executed this Agreement under seal as of the date first above written. MEIRAGTX HOLDINGS PLCBy: /s/ Richard Giroux Name: Richard GirouxTitle: Chief Operating OfficerVN ACQUISITION, INC.By: /s/ Richard Giroux Name: Richard GirouxTitle: Secretary and TreasurerVN ACQUISITION 2, INC.By: /s/ Richard Giroux Name: Richard GirouxTitle: Secretary and TreasurerVECTOR NEUROSCIENCES INC.By: /s/ Matthew During Name: Matthew DuringTitle: PresidentCOMPANY STOCKHOLDER REPRESENTATIVE /s/ Stephen B. Kaplitt Name: Stephen B. Kaplitt SIGNATURE PAGE TO AGREEMENT AND PLAN OF MERGERSConfidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 COMPANY STOCKHOLDER /s/ Michael G. KaplittName: Michael G. KaplittCOMPANY STOCKHOLDER /s/ Matthew DuringName: Matthew DuringCOMPANY STOCKHOLDER /s/ Stephen B. KaplittName: Stephen B. Kaplitt SIGNATURE PAGE TO AGREEMENT AND PLAN OF MERGERSConfidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Exhibit 10.12MEIRAGTX HOLDINGS PLC2018 INCENTIVE AWARD PLANSUB-PLAN FOR UK EMPLOYEES 1.PurposePursuant to the powers granted by the Administrator in Section 10.5 of the MeiraGTx Holdings plc 2018 Incentive Award Plan (as it may beamended or restated from time to time, the “Plan”), the Administrator has adopted this Sub-Plan (the “Sub-Plan”). The purpose of the Sub-Plan is topromote the success and enhance the value of MeiraGTx Holdings plc (the “Company”), by linking the individual interests of Employees, to those ofCompany shareholders and by providing such individuals with an incentive for outstanding performance to generate superior returns to Companyshareholders. The Sub-Plan is further intended to provide flexibility to the Company in its ability to motivate, attract, and retain the services ofEmployees upon whose judgment, interest, and special effort the successful conduct of the Company’s operation is largely dependent. Only Employeesmay receive Awards under the Sub-Plan. 2.Definitions, Construction and Eligibility(a) Capitalized terms used in the Sub-Plan which are not defined herein shall have the meaning given in the Plan, and where the context requiresany references to the “Plan” in those definitions shall be a reference to the Sub-Plan. The singular pronoun shall include the plural where the context soindicates.(b) In the event of a conflict between the terms of the Sub-Plan and the Plan with respect to Awards granted to Employees based in the UnitedKingdom under the Sub-Plan, the terms of the Sub-Plan will control. 2.1DefinitionsWherever the following terms are: (i) used in the Sub-Plan; or (ii) used in the Plan but apply to Awards made under the Sub-Plan, they shall havethe meanings specified below, unless the context clearly indicates otherwise:(c) “Award” means, individually, or collectively, a grant under the Sub-Plan of an Option, a Share Appreciation Right, a Restricted Share award, aRestricted Share Unit award or an Other Share or Cash Based Award;(d) “Service Provider” shall mean any person who is an Employee. 2.2EligibilityThe Sub-Plan forms the rules of the employee share scheme applicable to Awards made under the Sub-Plan to Employees of the Company andany Subsidiaries based in the United Kingdom or in any other jurisdiction at the discretion of the Administrator. Other Service Providers who are notEmployees (such as Consultants and non-employee Directors) are not eligible to receive Awards and become Participants under this Sub-Plan.References to the phrase “Service Provider” shall be interpreted as referring only to Employees when that phrase in the Plan is used in the context ofthe Sub-Plan and Awards granted to Employees under this Sub-Plan.3.Administration and DelegationThe provisions of Article 3 of the Plan shall apply to this Sub-Plan as if references to the Plan are references to the Sub-Plan. 4.Shares Available for Awards(a) The provisions of Article 4 of the Plan shall apply to this Sub-Plan as if references to the Plan are references to the Sub-Plan.(b) The aggregate number of Shares which may be issued or transferred pursuant to Awards under the Sub-Plan, when taken together with thenumber of Shares which may be issued or transferred pursuant to Awards under the Plan or any other sub-plan, shall not exceed the limits specified byArticle 4 of the Plan, as amended from time to time. 5.Options and Share Appreciation Rights(a) Except as set out below, the provisions of Article 5 of the Plan shall apply to this Sub-Plan as if references to the Plan are references to theSub-Plan.(b) Unless otherwise determined appropriate by the Administrator, any Option granted under this Sub-Plan shall be a Non-Qualified StockOption. 6.Restricted Shares; Restricted Share UnitsThe provisions of Article 6 of the Plan shall apply to this Sub-Plan as if references to the Plan are references to the Sub-Plan. On request by theCompany, Participants tax resident in the United Kingdom will be required to make an election under Section 431 of Chapter 2 Income Tax (Earningsand Pensions) Act 2003 (“ITEPA”) pursuant to which, for the relevant tax purposes, the market value of the Shares acquired will be calculated as if theShares were not restricted. Participants tax resident in other jurisdictions may be required to make equivalent elections appropriate to theirjurisdictions. 7.Other Share or Cash Based AwardsThe provisions of Article 7 of the Plan shall apply to this Sub-Plan as if references to the Plan are references to the Sub-Plan. 8.Adjustments For Changes In Ordinary Shares And Certain Other EventsThe provisions of Article 8 of the Plan shall apply to this Sub-Plan as if references to the Plan are references to the Sub-Plan. 29.General Provisions Applicable To Awards(a) Except as set out below, the provisions of Article 9 of the Plan shall apply to this Sub-Plan as if references to the Plan are references to theSub-Plan.(b) Section 9.5 of the Plan shall be amended so that the terms “taxes required by law to be withheld” and any similar phrases relating to taxobligations or tax liability when used in Section 9.5 shall include income tax, employee’s National Insurance contributions and (at the discretion ofthe Company) employer’s National Insurance contributions or other similar taxes arising in any jurisdiction (any a “Tax Liability”). The Participantwill indemnify and keep indemnified the Company and his/her employing company, if different, from and against any liability for or obligation to payany Tax Liability arising in consequence of any Award. 10.Miscellaneous(a) Except as set out below, the provisions of Article 10 of the Plan shall apply to this Sub-Plan as if references to the Plan are references to theSub-Plan.(b) The following language set out below is in addition to the terms of Article 10:“Neither the Sub-Plan nor any Award made under the Sub-Plan shall give the Participant any rights to compensation or damages including forany loss or potential loss that the Participant may suffer by reason of being unable to exercise any Option or forfeiting any Award or Shares as aresult of the termination of the Sub-Plan, the lapsing or termination of an Award or the Participant’s Termination of Service including where anyTermination of Service is subsequently held to be wrongful or unfair.”(c) The following language set out below shall replace Section 10.9:“The Company and all its Subsidiaries may transfer, collect, use, process or disclose, in electronic or other form, such information to third parties,including where they are situated outside the European Economic Area in countries where the level of data protection may not be as high as inthe Participant’s country of residence, in the event that such disclosure is in their view required for the performance of their obligations under thePlan. The Company and all Group Companies shall ensure that such collection, use, processing and transfers are made in accordance with the EUGeneral Data Protection Regulation and other applicable data protection laws in any other jurisdiction.” 11.Definitions(a) Except as set out below, the provisions of Article 11 of the Plan shall apply to this Sub-Plan as if references to the Plan are references to theSub-Plan.(b) Section 11.11 (“Consultant”) shall not apply to this Sub-Plan.(c) Section 11.38, (“Service Provider”) shall mean an Employee. 3Exhibit 10.13MEIRAGTX HOLDINGS PLC2018 INCENTIVE AWARD PLAN UK SUB-PLANOPTION GRANT NOTICE FOR UK PARTICIPANTSCapitalized terms not specifically defined in this Option Grant Notice for UK Participants (the “Grant Notice”) have the meanings given to themin the 2018 Incentive Award Plan UK Sub-Plan (the “UK Sub-Plan”) of MeiraGTx Holdings plc (the “Company”), which incorporates terms from theCompany’s 2018 Incentive Award Plan (the “Plan”).The Company has granted to the participant listed below (“Participant”) the option described in this Grant Notice (the “Option”), subject to theterms and conditions of the UK Sub-Plan and the Option Agreement attached as Exhibit A (the “UK Option Agreement”), both of which areincorporated into this Grant Notice by reference. Participant: Grant Date: Exercise Price per Share: Shares Subject to the Option: Final Expiration Date: Vesting Schedule: Subject to the terms of the Agreement, the Option will become vestedand exercisable as to 25% of the Shares subject to the Option on thefirst anniversary of the grant date specified above and as to theremaining 75% of the Shares subject to the Option in 36 equalmonthly installments occurring upon Participant’s completion of eachsuccessive month of service as a Service Provider following the firstanniversary of the grant date specified above.Type of Option Incentive Stock OptionBy Participant’s signature below, Participant agrees to be bound by the terms of this Grant Notice, the UK Sub-Plan and the UK OptionAgreement. Participant has reviewed the UK Sub-Plan, this Grant Notice and the UK Option Agreement in their entirety, has had an opportunity toobtain the advice of counsel prior to executing this Grant Notice and fully understands all provisions of the UK Sub-Plan, this Grant Notice and the UKOption Agreement. Participant hereby agrees to accept as binding, conclusive and final all decisions or interpretations of the Administrator upon anyquestions arising under the UK Sub-Plan, this Grant Notice or the UK Option Agreement.MEIRAGTX HOLDINGS PLC PARTICIPANTBy: Name: [Participant Name]Title: 2Exhibit AOPTION AGREEMENT FOR UK PARTICIPANTSCapitalized terms not specifically defined in this UK Option Agreement have the meanings specified in the Grant Notice or, if not defined in theGrant Notice, in the UK Sub-Plan.ARTICLE I.GENERAL1.1 Grant of Option. The Company has granted to Participant the Option effective as of the grant date set forth in the Grant Notice (the “GrantDate”).1.2 Incorporation of Terms of UK Sub-Plan. The Option is subject to the terms and conditions set forth in this UK Option Agreement and the UKSub-Plan, which is incorporated herein by reference. In the event of any inconsistency between the UK Sub-Plan and this UK Option Agreement, theterms of the UK Option Agreement will control.ARTICLE II.PERIOD OF EXERCISABILITY2.1 Commencement of Exercisability. The Option will vest and become exercisable according to the vesting schedule in the Grant Notice (the“Vesting Schedule”) except that any fraction of a Share as to which the Option would be vested or exercisable will be accumulated and will vest andbecome exercisable only when a whole Share has accumulated. Notwithstanding anything in the Grant Notice, the UK Sub-Plan or this UK OptionAgreement to the contrary, unless the Administrator otherwise determines, the Option will immediately expire and be forfeited as to any portion that isnot vested and exercisable as of the Participant’s Termination of Service for any reason.2.2 Duration of Exercisability. The Vesting Schedule is cumulative. Any portion of the Option which vests and becomes exercisable will remainvested and exercisable until the Option expires. The Option will be forfeited immediately upon its expiration.2.3 Expiration of Option. The Option may not be exercised to any extent by anyone after, and will expire on, the first of the following to occur:(a) The final expiration date in the Grant Notice;(b) Except as the Administrator may otherwise approve, the expiration of three (3) months from the date of Participant’s Termination ofService, unless Participant’s Termination of Service is for Cause or by reason of Participant’s death or Disability;(c) Except as the Administrator may otherwise approve, the expiration of one (1) year from the date of Participant’s Termination of Serviceby reason of Participant’s death or Disability; and(d) Except as the Administrator may otherwise approve, Participant’s Termination of Service for Cause.ARTICLE III.EXERCISE OF OPTION3.1 Person Eligible to Exercise. During Participant’s lifetime, only Participant may exercise the Option. After Participant’s death, any exercisableportion of the Option may, prior to the time the Option expires, be exercised by Participant’s Designated Beneficiary as provided in the UK Sub-Plan.3.2 Partial Exercise. Any exercisable portion of the Option or the entire Option, if then wholly exercisable, may be exercised, in whole or in part,according to the procedures in the UK Sub-Plan at any time prior to the time the Option or portion thereof expires, except that the Option may only beexercised for whole Shares.3.3 UK Tax Obligations.(a) Tax Indemnity. Participant agrees to indemnify and keep indemnified the Company and his/her employing company (“Employer”), ifdifferent, from and against any liability for or obligation to pay any Tax Liability (a “Tax Liability” being any liability for income tax, employee’sNational Insurance contributions and (at the discretion of the Company) employer’s National Insurance Contributions (or other similar obligations topay tax and social security wherever in the world arising)) that is attributable to: (1) the grant or exercise of, or any benefit derived by Participant from,the Option or the Shares which are the subject of the Option; (2) the transfer or issue of Shares to Participant on satisfaction of the Option or any otherbenefit on exercise of the Option; (3) any restrictions applicable to the Shares held by the Participant ceasing to apply to those shares; or (4) thedisposal of any Shares.(b) Tax Liability. The Option cannot be exercised until Participant has made such arrangements as the Company may require for thesatisfaction of any Tax Liability that may arise in connection with the exercise of the Option and/or the acquisition of the Shares by the Participant.The Company shall not be required to issue, allot or transfer Shares until Participant has satisfied this obligation.(c) Election. Participant undertakes that, upon request by the Company, he/she will (on or within 14 days of acquiring the Shares) join withhis/her Employer in electing, pursuant to Section 431(1) of the Income Tax (Earnings and Pensions) Act 2003 (“ITEPA”) that, for relevant taxpurposes, the market value of the Shares acquired on exercise of the Option on any occasion will be calculated as if the Shares were not restricted andSections 425 to 430 (inclusive) of ITEPA are not to apply to such Shares.(d) The Company has the right and option, but not the obligation, to treat Participant’s failure to provide timely payment in accordancewith the UK Sub-Plan of any withholding tax arising in connection with the Option as Participant’s election to satisfy all or any portion of thewithholding tax by requesting the Company retain Shares otherwise issuable under the Option.(e) Participant acknowledges that Participant is ultimately liable and responsible for all taxes owed in connection with the Option,regardless of any action the Company or any Subsidiary takes with respect to any tax withholding obligations that arise in connection with the Option.Neither the Company nor any Subsidiary makes any representation or undertaking regarding the treatment of any tax withholding in connection withthe awarding, vesting or exercise of the Option or the subsequent sale of Shares. The Company and the Subsidiaries do not commit and are under noobligation to structure the Option to reduce or eliminate Participant’s Tax Liability. A-2ARTICLE IV.OTHER PROVISIONS4.1 Adjustments. Participant acknowledges that the Option is subject to adjustment, modification and termination in certain events as providedin this UK Option Agreement and the UK Sub-Plan.4.2 Notices. Any notice to be given under the terms of this UK Option Agreement to the Company must be in writing and addressed to theCompany in care of the Company’s Secretary at the Company’s principal office or the Secretary’s then-current email address or facsimile number. Anynotice to be given under the terms of this UK Option Agreement to Participant must be in writing and addressed to Participant (or, if Participant is thendeceased, to the person entitled to exercise the Option) at Participant’s last known mailing address, email address or facsimile number in theCompany’s personnel files. By a notice given pursuant to this Section, either party may designate a different address for notices to be given to thatparty. Any notice will be deemed duly given when actually received, when sent by email, when sent by certified mail (return receipt requested) anddeposited with postage prepaid in a post office or branch post office regularly maintained by the United States Postal Service, when delivered by anationally recognized express shipping company or upon receipt of a facsimile transmission confirmation.4.3 Titles. Titles are provided herein for convenience only and are not to serve as a basis for interpretation or construction of this UK OptionAgreement.4.4 Conformity to Securities Laws. Participant acknowledges that the UK Sub-Plan, the Grant Notice and this UK Option Agreement are intendedto conform to the extent necessary with all Applicable Laws and, to the extent Applicable Laws permit, will be deemed amended as necessary toconform to Applicable Laws.4.5 Successors and Assigns. The Company may assign any of its rights under this UK Option Agreement to single or multiple assignees, and thisUK Option Agreement will inure to the benefit of the successors and assigns of the Company. Subject to the restrictions on transfer set forth in the UKSub-Plan, this UK Option Agreement will be binding upon and inure to the benefit of the heirs, legatees, legal representatives, successors and assigns ofthe parties hereto.4.6 Limitations Applicable to Section 16 Persons. Notwithstanding any other provision of the UK Sub-Plan or this UK Option Agreement, ifParticipant is subject to Section 16 of the Exchange Act, the UK Sub-Plan, the Grant Notice, this UK Option Agreement and the Option will be subjectto any additional limitations set forth in any applicable exemptive rule under Section 16 of the Exchange Act (including any amendment toRule 16b-3) that are requirements for the application of such exemptive rule. To the extent Applicable Laws permit, this UK Option Agreement will bedeemed amended as necessary to conform to such applicable exemptive rule.4.7 Entire Agreement. The UK Sub-Plan, the Grant Notice and this UK Option Agreement (including any exhibit hereto) constitute the entireagreement of the parties and supersede in their entirety all prior undertakings and agreements of the Company and Participant with respect to thesubject matter hereof.4.8 Agreement Severable. In the event that any provision of the Grant Notice or this UK Option Agreement is held illegal or invalid, theprovision will be severable from, and the illegality or invalidity of the provision will not be construed to have any effect on, the remaining provisionsof the Grant Notice or this UK Option Agreement. A-34.9 Limitation on Participant’s Rights. Participation in the UK Sub-Plan confers no rights or interests other than as herein provided. This UKOption Agreement creates only a contractual obligation on the part of the Company as to amounts payable and may not be construed as creating atrust. Neither the UK Sub-Plan nor any underlying program, in and of itself, has any assets. Participant will have only the rights of a general unsecuredcreditor of the Company with respect to amounts credited and benefits payable, if any, with respect to the Option, and rights no greater than the right toreceive the Shares as a general unsecured creditor with respect to the Option, as and when exercised pursuant to the terms hereof.4.10 Not a Contract of Employment. Nothing in the UK Sub-Plan, the Grant Notice or this UK Option Agreement confers upon Participant anyright to continue in the employ of the Company or any Subsidiary or interferes with or restricts in any way the rights of the Company and itsSubsidiaries, which rights are hereby expressly reserved, to discharge or terminate the services of Participant at any time for any reason whatsoever,with or without Cause, except to the extent expressly provided otherwise in a written agreement between the Company or a Subsidiary and Participant.4.11 Counterparts. The Grant Notice may be executed in one or more counterparts, including by way of any electronic signature, subject toApplicable Law, each of which will be deemed an original and all of which together will constitute one instrument.4.12 Incentive Stock Options. If the Option is designated as an Incentive Stock Option:(a) Participant acknowledges that to the extent the aggregate fair market value of shares (determined as of the time the option with respectto the shares is granted) with respect to which options intended to qualify as “incentive stock options” under Section 422 of the Code, including theOption, are exercisable for the first time by Participant during any calendar year exceeds $100,000 or if for any other reason such options do notqualify or cease to qualify for treatment as “incentive stock options” under Section 422 of the Code, such options (including the Option) will betreated as non-qualified options. Participant further acknowledges that the rule set forth in the preceding sentence will be applied by taking the Optionand other options into account in the order in which they were granted, as determined under Section 422(d) of the Code. Participant acknowledges thatamendments or modifications made to the Option pursuant to the UK Sub-Plan that would cause the Option to become a Non-Qualified Stock Optionwill not materially or adversely affect Participant’s rights under the Option, and that any such amendment or modification shall not requireParticipant’s consent. Participant also acknowledges that if the Option is exercised more than three (3) months after Participant’s Termination ofService as an Employee, other than by reason of death or disability, the Option will be taxed as a Non-Qualified Stock Option.(b) Participant will give prompt written notice to the Company of any disposition or other transfer of any Shares acquired under this UKOption Agreement if such disposition or other transfer is made (a) within two (2) years from the Grant Date or (b) within one (1) year after the transfer ofsuch Shares to Participant. Such notice will specify the date of such disposition or other transfer and the amount realized, in cash, other property,assumption of indebtedness or other consideration, by Participant in such disposition or other transfer.4.13 Data Protection. The Company and all its Subsidiaries may transfer, collect, use, process or disclose, in electronic or other form, suchinformation to third parties, including where they are situated outside the European Economic Area in countries where the level of data protection maynot be as high as in the Participant’s country of residence, in the event that such disclosure is in their view required for the performance of theirobligations under the Plan. The Company and all Group Companies shall ensure that such collection, use, processing and transfers are made inaccordance with the EU General Data Protection Regulation and other applicable data protection laws in any other jurisdiction. A-44.14 Acknowledgement. Participant acknowledges that neither this UK Option Agreement nor the UK Sub-Plan has been issued, nor has it beenapproved by, an authorised person within the meaning of the Financial Services and Markets Act 2000 of the United Kingdom and is being directed atthe Participant because the offer to which this UK Option Agreement and the UK Sub-Plan relate has been determined as having regard to theParticipant’s circumstances as an employee of the Company. This UK Option Agreement is strictly confidential and is not for distribution to, and maynot be acted upon by, any other person other than the person to whom it has been specifically addressed.* * * * * A-5Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Exhibit 10.19Execution VersionCONSULTING AGREEMENTTHIS CONSULTING AGREEMENT (this “Agreement”) is made and entered into as of October 5, 2018, by and between MeiraGTx Holdingsplc, a company incorporated in the Cayman Islands (the “Company”) with offices at 450 East 29th Street, 15th Floor, New York, NY 10016, VectorConsulting LLC, a limited liability company organized in New York with a mailing address at 62 N. Livingston Avenue, Livingston, NJ 07039 (the“Consulting Entity”), Michael G. Kaplitt, an individual residing at 1113 York Avenue, Apt. 26E, New York, NY 10065 , Matthew During, anindividual residing at 8 Nearwater Road, Rowayton, CT 06853, and Stephen B. Kaplitt, an individual residing at 62 N. Livingston Avenue, Livingston,NJ 07039 (each such individual, a “Consultant” and collectively, the “Consultants”). The Company, the Consulting Entity and the Consultants arecollectively referred to as the “Parties” and each a “Party”.INTRODUCTIONOn October 5, 2018, the Company acquired Vector Neurosciences Inc., a Delaware corporation (“Vector”), pursuant to that certain Agreementand Plan of Merger, dated October 5, 2018 (the “Merger Agreement”), pursuant to which VN Acquisition, Inc., a wholly-owned subsidiary of theCompany merged with and into Vector with Vector surviving as a wholly owned subsidiary of the Company (“Merger 1”) followed by Vector mergingwith and into VN Acquisition 2, Inc. with VN Acquisition 2, Inc. surviving as the surviving corporation (together with Merger 1, the “Mergers”).Capitalized terms used herein but not otherwise defined herein shall have the meaning ascribed to such terms in the Merger Agreement.The Consultants were the only stockholders of Vector prior to Merger 1.The Consultants are the only equity holders and members of the Consulting Entity.The Company, the Consulting Entity and the Consultants desire to establish the terms and conditions under which the Consulting Entity willprovide services to the Company. In consideration of the mutual covenants and promises contained herein and other good and valuable consideration,the receipt and sufficiency of which are hereby acknowledged by the parties hereto, the parties agree as follows:1. Services; Competitive Activities.1.1 The Consulting Entity agrees to make available the services of each Consultant to perform the Services, to and for the Company inconnection with the Project as may be reasonably requested from time to time by the Company as set forth on Schedule A to this Agreement. TheCompany acknowledges and agrees that: (a) the Services are not to be provided on a full-time basis; (b) each Consultant has work and professionalcommitments and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 activities unrelated to the Services; and (c) the Services reasonably requested by the Company hereunder shall not unduly or unreasonably interferewith any unrelated commitments and activities of the Consultants, provided that, subject to the foregoing reasonableness requirement, Consultantsshall perform the Services with commercially reasonably diligence and commercially reasonable best efforts.1.2 Subject to Section 3.2, the Company shall pay for all costs and expenses necessary for, or incurred in relation to, the Services and theProject, and the Consulting Entity and the Consultants shall not be responsible for any such costs or expenses.1.3 During the Consultation Period (as defined below) and for a period of twelve (12) months thereafter, the Consulting Entity and eachConsultant shall not engage in any Competitive Activity.2. Term. This Agreement shall commence on the date hereof and shall continue until terminated in accordance with the provisions of Section 4(the term of effectiveness of this Agreement being referred to herein as the “Consultation Period”).3. Compensation.3.1 Equity Grants. The Company shall issue to the Consultants the Options set forth on Schedule A to this Agreement.3.2 Reimbursement of Expenses. The Company shall reimburse the Consulting Entity or the Consultants individually (as applicable) forall reasonable and necessary documented out of pocket expenses incurred or paid by the Consulting Entity or a Consultant in connection with, orrelated to, the performance of services under this Agreement, provided that no single expenses greater than $1,000 shall be incurred without the priorwritten approval of the Company (which may be delivered by email). To the extent the Consulting Entity or a Consultant has expenses forreimbursement, the Consulting Entity or such Consultant shall submit to the Company itemized statements on a monthly basis for each month inwhich such expenses are incurred, in a form reasonably satisfactory to the Company, of such expenses incurred in the previous monthly period. Unlessdisputed in good faith, the Company shall pay to the Consulting Entity or the Consultant amounts shown on each such statement within 30 days afterreceipt thereof. The Parties shall promptly and diligently cooperate in good faith to address any good faith disputes related to expenses.4. Termination.4.1 Termination for Convenience. The Company may, subject in all events to the terms and provisions of this Agreement and withoutprejudice to any right or remedy it may have due to any failure of the Consulting Entity or any Consultant to perform the Consulting Entity’s or theConsultants’ obligations under this Agreement, terminate the Consultation Period for no reason or any reason other than those provided inSection 4.2(a) or 4.3, effective upon ten (10) Business Days’ prior written notice to the Consulting Entity. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 2 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 4.2 Termination for Breach.(a) The Company may terminate this Agreement upon a material breach of this Agreement by the Consulting Entity or anyConsultant by providing thirty (30) days’ prior written notice to the Consulting Entity and the Consultants describing with reasonable specificity thealleged breach or breaches. The termination shall become effective at the end of the notice period unless the breaching Consulting Entity orConsultant cures such breach or breaches during such notice period. Notwithstanding the foregoing, if the breach, by its nature, is incurable, theCompany may terminate this Agreement immediately upon written notice to the Consulting Entity and the Consultants.(b) The Consulting Entity and the Consultants may terminate this Agreement for Cause or upon a material breach of thisAgreement by the Company by providing thirty (30) days’ prior written notice to the Company describing with reasonable specificity the event(s)constituting “Cause” or the alleged breach or breaches. The termination shall become effective at the end of the notice period unless the Companycures such event(s) constituting “Cause” or breach or breaches during such notice period. Notwithstanding the foregoing, if the breach, by its nature, isincurable, the Consulting Entity and the Consultants may terminate this Agreement immediately upon written notice to the Company.4.3 Termination for Changes in Consulting Entity. The Company may terminate this Agreement effective upon five (5) Business Days’prior written notice to the Consulting Entity in the event that: (a) the Consulting Entity amends its operating agreement or similar organizationaldocuments, which amendments have or could reasonably be expected to have a material adverse effect on the ability of the Consulting Entity or theConsultants to provide the Services; (b) Michael G. Kaplitt and Matthew During both cease to provide Consulting Services on behalf of the ConsultingEntity for any reason other than Cause; or (c) the equity ownership of the Consulting Entity changes from the equity ownership set forth on Schedule Bto this Agreement without the prior written approval of the Company.4.4 Effects of Termination.(a) In the event of termination by the Company pursuant to Section 4.1, or by the Consulting Entity pursuant to Section 4.2(b),the Consultation Period shall terminate immediately, and the following provisions shall survive: Sections 6 through 18 (inclusive) and Sections 4.1and 4.3 of Schedule A.(b) In the event of termination by the Company pursuant to Section 4.2(a) or for any reason pursuant to Section 4.3, theConsultation Period shall terminate immediately, and the following provisions shall survive: Sections 6 through 18 (inclusive).(c) Upon termination of the Consultation Period for any reason, the Consulting Entity and the Consultants (as applicable) shall beentitled to payment for expense reimbursements due under Section 3.2 for expenses incurred prior to the effective date of termination, and, except asexpressly provided in this Section 4.4, the Company shall have no further obligations to the Consulting Entity or any Consultant hereunder. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 3 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 5. Cooperation. The Consulting Entity and each Consultant shall use its, his or her commercially reasonable efforts in the performance of theConsulting Entity’s and each Consultant’s obligations under this Agreement. The Company shall provide such access to its information and propertyas may be reasonably required in order to permit the Consulting Entity and each Consultant to perform such person’s obligations hereunder. TheConsulting Entity and each Consultant shall reasonably cooperate with the Company’s personnel, shall not interfere with the conduct of theCompany’s business and shall observe all rules, regulations and security requirements of the Company concerning the safety of persons and property,provided that such rules, regulations and requirements shall have been (i) transmitted to the Consultants in writing (including by email) or(ii) otherwise made available to the Consultants in a manner reasonably expected to make the Consultants aware of such rules, regulations andrequirements.6. Inventions and Proprietary Information.6.1 Inventions.(a) All inventions, discoveries, computer programs, data, technology, designs, innovations and improvements (whether or notpatentable and whether or not copyrightable) which are made, conceived, reduced to practice, created, written, designed or developed by theConsulting Entity or any Consultant, solely or jointly with others and whether during normal business hours or otherwise, (i) during and prior to theConsultation Period if resulting from or directly related to the Project or the Services, (ii) during the Consultation Period if resulting from or directlyrelated to the business of the Company or (iii) after the Consultation Period if resulting or directly derived from Proprietary Information (as definedbelow) (collectively under clauses (i), (ii) and (iii), “Inventions”), shall be the sole property of the Company. The Consulting Entity and eachConsultant hereby assigns to the Company all Inventions and any and all related patents, copyrights, trademarks, trade names, and other industrial andintellectual property rights and applications therefor, in the United States and elsewhere and appoints any officer of the Company as the ConsultingEntity and each Consultant’s duly authorized attorney to execute, file, prosecute and protect the same before any government agency, court orauthority. Upon the request of the Company and at the Company’s expense, the Consulting Entity and each Consultant shall execute such furtherassignments, documents and other instruments as may be necessary or desirable to fully and completely assign all Inventions to the Company and toassist the Company in applying for, obtaining and enforcing patents or copyrights or other rights in the United States and in any foreign country withrespect to any Invention. The Consulting Entity and each Consultant also hereby waives all claims to moral rights in any Inventions.(b) The Consulting Entity and each Consultant shall promptly disclose to the Company all Inventions and will maintain adequateand current written records (in the form of notes, sketches, drawings and as may be specified by the Company) to document the conception and/or firstactual reduction to practice of any Invention. Such written records shall be available to and remain the sole property of the Company at all times. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 4 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 6.2 Proprietary Information.(a) The Consulting Entity and each Consultant acknowledges that its, his or her relationship with the Company is one of hightrust and confidence and that in the course of the Consulting Entity’s and such Consultant’s service to the Company it, he or she will have access toand contact with Proprietary Information. Subject to subsection (c) below, the Consulting Entity and each Consultant agrees that it, he or she will not,during the Consultation Period or at any time thereafter, disclose to others, or use for its, his or her benefit or the benefit of others, any ProprietaryInformation or Invention.(b) For purposes of this Agreement, Proprietary Information shall mean, by way of illustration and not limitation, all information(whether or not patentable and whether or not copyrightable) owned, possessed or used by the Company, including, without limitation, any Invention,formula, vendor information, customer information, apparatus, equipment, trade secret, process, research, report, technical data, know-how, computerprogram, software, software documentation, hardware design, technology, marketing or business plan, forecast, unpublished financial statement,budget, license, price, cost and employee list that is communicated to, learned of, developed or otherwise acquired by the Consulting Entity or anyConsultant in the course of such person’s service as a consultant to the Company.(c) The Consulting Entity’s and each Consultant’s obligations under this Section 6.2 shall not apply to any information that (i) isor becomes known to the general public under circumstances involving no breach by the Consulting Entity or any Consultant or agents of theConsulting Entity of the terms of this Section 6.2, (ii) is generally disclosed to third parties by the Company without restriction on such third parties,(iii) is approved for release by written authorization of an officer of the Company or (iv) is required to be disclosed pursuant to order or decision of anyjudicial, administrative or regulatory authority, pursuant to subpoena or similar legal compulsion, provided, that the Consulting Entity or Consultantsshall give the Company prior written notice thereof as far in advance of such disclosure as reasonably practicable and shall reasonably cooperate withthe Company in the event the Company seeks (at its own cost and expense) to prevent, limit or delay such disclosure.(d) Upon termination of this Agreement or at any other time upon request by the Company, the Consulting Entity and eachConsultant shall promptly deliver to the Company all records, files, memoranda, notes, designs, data, reports, price lists, customer lists, drawings, plans,computer programs, software, software documentation, sketches, laboratory and research notebooks and other documents (and all copies orreproductions of such materials) relating to the business of the Company.(e) The Consulting Entity and each Consultant represents that its, his or her retention as a consultant with the Company and suchperson’s performance under this Agreement does not, and shall not, breach any agreement that obligates it, him or her to keep in Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 5 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 confidence any trade secrets or confidential or proprietary information of the Consulting Entity or any Consultant or of any other party or to refrainfrom competing, directly or indirectly, with the business of any other party or otherwise conflict with any of his or her agreements or obligations to anyother party. The Consulting Entity and each Consultant shall not disclose to the Company any trade secrets or confidential or proprietary informationof any other party.(f) The Consulting Entity and each Consultant acknowledges that the Company from time to time may have agreements withother persons or with the United States Government, or agencies thereof, that impose obligations or restrictions on the Company regarding inventionsmade during the course of work under such agreements or regarding the confidential nature of such work. The Consulting Entity and each Consultantagrees to be bound by all such obligations and restrictions that are known to it, him or her and to take all action necessary to discharge the obligationsof the Company under such agreements (at the Company’s sole cost and expense, if any).(g) Defend Trade Secrets Act Notice of Immunity Rights. The Consulting Entity and each Consultant acknowledges that theCompany has provided the Consulting Entity and each Consultant with the following notice of immunity rights in compliance with the requirementsof the Defend Trade Secrets Act: (i) such Consulting Entity or Consultant will not be held criminally or civilly liable under any Federal or State tradesecret law for the disclosure of Proprietary Information that is made in confidence to a Federal, State, or local government official or to an attorneysolely for the purpose of reporting or investigating a suspected violation of law, (ii) such Consulting Entity or Consultant will not be held criminally orcivilly liable under any Federal or State trade secret law for the disclosure of Proprietary Information that is made in a complaint or other documentfiled in a lawsuit or other proceeding, if such filing is made under seal, and (iii) if such Consulting Entity or Consultant files a lawsuit for retaliation bythe Company for reporting a suspected violation of law, such Consulting Entity or Consultant may disclose the Proprietary Information to its attorneyand use the Proprietary Information in the court proceeding, if Consultant files any document containing the Proprietary Information under seal, anddoes not disclose the Proprietary Information, except pursuant to court order.6.3 Remedies. The Consulting Entity and each Consultant acknowledges that any breach of the provisions of this Section 6 shall result inserious and irreparable injury to the Company for which the Company cannot be adequately compensated by monetary damages alone. The ConsultingEntity and each Consultant agrees, therefore, that, in addition to any other remedy it may have, the Company shall be entitled to enforce the specificperformance of this Agreement by the Consulting Entity and any Consultant and to seek both temporary and permanent injunctive relief (to the extentpermitted by law) without the necessity of proving actual damages.6.4 Portfolio Reference. Upon termination of this Agreement, the Consulting Entity and each Consultant may disclose to prospectivethird party contractors or employers the fact that the Consulting Entity and each Consultant performed certain services for the Company, subject to thereceipt of prior written permission from the Company with respect to such disclosure and the scope thereof. Notwithstanding the foregoing, theConsulting Entity and each Consultant may freely disclose at any time only the fact that [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 6 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 7. Non-Solicitation. During the Consultation Period and for a period of twelve (12) months thereafter, the Consulting Entity and eachConsultant shall not, either alone or in association with others, (i) solicit, or permit any organization directly or indirectly controlled by the ConsultingEntity and any Consultant to solicit, any employee of the Company to leave the employ of the Company, or (ii) solicit for employment, hire or engageas an independent contractor, or permit any organization directly or indirectly controlled by the Consulting Entity and any Consultant to solicit foremployment, hire or engage as an independent contractor, any person who was employed by the Company at any time during the term of theConsulting Entity’s and the Consultant’s engagement with the Company; provided, that this clause (ii) shall not apply to (x) any individual whoseemployment with the Company has been terminated for a period of six months or longer or (y) general advertisements for employment or recruitmentefforts conducted by any recruitment agency, provided that such advertisements or recruitment efforts are not directed at any such individual.8. Other Agreements. The Consulting Entity and each Consultant hereby represents that, except as the Consulting Entity or any applicableConsultant has disclosed in writing to the Company, none of the Consulting Entity nor any Consultant is bound by the terms of any agreement withany current or prior employer or other party to refrain from using or disclosing any trade secret or confidential or proprietary information in the courseof the Consulting Entity’s and each Consultant’s relationship with the Company, to refrain from competing, directly or indirectly, with the business ofsuch employer or any other party or to refrain from soliciting employees, customers or suppliers of such employer or other party; in all cases, exceptwhere such terms would not prohibit or materially interfere with the ability of Consultants and the Consulting Entity to provide the Services andperform its and their obligations hereunder. The Consulting Entity and each Consultant agrees to furnish the Company with a copy of any suchagreement within or under its or their possession or control upon request, except to the extent such disclosure is prohibited by law or contractualobligation, in which case the Consulting Entity and each Consultant agrees to furnish the Company with a copy of such agreement redacted in order tocomply with such law or contractual obligation, and with respect to limitations based on contractual obligations, to request the other party to thecontract to waive such obligations.9. Independent Contractor Status; Benefits.9.1 The Consulting Entity and each Consultant shall perform all services under this Agreement as an “independent contractor” and not asan employee or agent of the Company and, as an independent contractor, the Consulting Entity and each Consultant will be solely responsible forcomplying with all applicable laws, rules and regulations concerning income, employment (or self-employment) and other taxes, social securitycontributions, pension fund contributions, unemployment contributions and similar matters and the Company shall not be required to withholdincome, employment or other taxes from payments to the Consulting Entity or the Consultants. None of the Consulting Entity nor any Consultant isauthorized to assume or create any obligation or responsibility, express or implied, on behalf of, or in the name of, the Company or to bind theCompany in any manner. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 7 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 9.2 None of the Consulting Entity nor the Consultants shall be entitled to any benefits, coverages or privileges, including, withoutlimitation, social security, unemployment, medical or pension payments, made available to employees of the Company. If any Consultant isreclassified by a state or federal agency or court as an employee of the Company, the Consultant will become a reclassified employee and will receiveno benefits except those mandated by state or federal law, even if by the terms of the Company’s benefit plans in effect at the time of suchreclassification, the Consultant would otherwise be eligible for such benefits.10. Notices. All notices, requests, demands, claims, and other communications hereunder shall be in writing. Any notice, request, demand,claim or other communication hereunder shall be deemed duly delivered four (4) Business Days after it is sent by registered or certified mail, returnreceipt requested, postage prepaid, or one business day after it is sent for next business day delivery via a reputable nationwide overnight courierservice, or on the same business day (if sent before 2 p.m. local time in the time zone of the recipient’s physical address (as specified below) andotherwise on the next business day) if sent by fax with electronic or telephonic confirmation of receipt, in each case to the intended recipient as setforth below: If to the Consulting Entity or Stephen B. Kaplitt: Stephen B. Kaplitt[contact information] If to Michael G. Kaplitt or Matthew During: Michael G. Kaplitt[contact information] Matthew During[contact information] If to the Company: MeiraGTx Holdings plc430 East 29th Street, 10th FloorNew York, NY 10016Attn: Richard GirouxTel: (646) 490-2971Email: rich@meiragtx.com With a copy (which shall not constitute notice) to: Latham & Watkins LLPJohn Hancock Tower200 Clarendon StreetBoston, MA 02116Attention: Peter N. Handrinos, Esq.Tel: (617) 948-6060Fax: (617) 948-6001Email: Peter.Handrinos@lw.comAny Party may give any notice, request, demand, claim or other communication hereunder using any other means (including personal delivery,expedited courier, messenger service, or ordinary mail), but no such notice, request, demand, claim or other communication that is given by such othermeans shall be deemed to have been duly given unless and until it actually is received by the party for whom it is intended. In addition, any notice,request, demand, claim or other communication hereunder delivered by email and acknowledged by reply Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 8 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 email from the recipient shall be deemed to be effective delivery hereunder. Any Party may change the address to which notices, requests, demands,claims, and other communications hereunder are to be delivered by giving the other Parties notice in the manner herein set forth.11. Pronouns. Whenever the context may require, any pronouns used in this Agreement shall include the corresponding masculine, feminine orneuter forms, and the singular forms of nouns and pronouns shall include the plural, and vice versa.12. Entire Agreement. The Merger Agreement and this Agreement constitute the entire agreement between the parties and supersedes all prioragreements and understandings, whether written or oral, relating to the subject matter of this Agreement.13. Amendment. This Agreement may be amended or modified only by a written instrument executed by all Parties.14. Governing Law. This Agreement shall be construed, interpreted and enforced in accordance with the laws of the State of New York withoutregard to conflict of law principles that would result in the application of any law other than the State of New York.15. Successors and Assigns. This Agreement shall be binding upon, and inure to the benefit of, both parties and their respective successors andassigns, including any affiliate of the Company or any corporation with which, or into which, the Company may be merged or which may succeed to itsassets or business, provided, however, that the obligations of the Consulting Entity and each Consultant are personal and shall not be assigned by it,him or her.16. Interpretation. If any restriction set forth in Section 1 or Section 7 is found by any court of competent jurisdiction to be unenforceablebecause it extends for too long a period of time or over too great a range of activities or in too broad a geographic area, it shall be interpreted to extendonly over the maximum period of time, range of activities or geographic area as to which it may be enforceable.17. [Reserved].18. Miscellaneous.18.1 No delay or omission by any Party in exercising any right under this Agreement shall operate as a waiver of that or any other right. Awaiver or consent given by a Party on any one occasion shall be effective only in that instance and shall not be construed as a bar or waiver of any righton any other occasion.18.2 The captions of the sections of this Agreement are for convenience of reference only and in no way define, limit or affect the scopeor substance of any section of this Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 9 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 18.3 In the event that any provision of this Agreement shall be invalid, illegal or otherwise unenforceable, the validity, legality andenforceability of the remaining provisions shall in no way be affected or impaired thereby.18.4 This Agreement may be executed in multiple counterparts by facsimile or other reliable electronic reproduction (including, withoutlimitation, transmission by pdf), each of which shall be taken together as one and the same instrument.18.5 Arbitration. Except for any dispute, controversy or claim described in Section 18.6 (which shall be handled exclusively inaccordance with Section 18.6), it is understood and agreed between the Parties hereto that any and all claims, grievances, demands, controversies,causes of action or disputes of any nature whatsoever (including, but not limited to, tort and contract claims, and claims upon any law, statute, order, orregulation) (hereinafter “Disputes”), arising out of, in connection with, or in relation to (a) this Agreement, or (b) questions of arbitrability under thisAgreement, shall be resolved by final, binding, nonjudicial arbitration in accordance with the Federal Arbitration Act, 9 U.S.C. Section 1, et seq.pursuant to the following procedures:(a) Any Party may send another Party or Parties written notice identifying the matter in dispute and invoking the procedures ofthis Section (the “Dispute Notice”). Within 14 days from delivery of the Dispute Notice, each Party involved in the dispute shall meet at a mutuallyagreed location in New York, New York, for the purpose of determining whether they can resolve the dispute themselves by written agreement, and, ifnot, whether they can agree upon an impartial third-party arbitrator (the “Arbitrator”) to whom to submit the matter in dispute for final and bindingarbitration.(b) If such Parties fail to resolve the dispute by written agreement or agree on the Arbitrator within the later of fourteen (14) daysfrom any such initial meeting or within thirty (30) days from the delivery of the Dispute Notice, any such Party may make written application to theJudicial Arbitration and Mediation Services (“JAMS”), in New York, New York for the appointment of a single Arbitrator to resolve the dispute byarbitration. At the request of JAMS the Parties involved in the dispute shall meet with JAMS at its offices within ten (10) days of such request to discussthe dispute and the qualifications and experience which each Party respectively believes the Arbitrator should have; provided, however, that theselection of the Arbitrator shall be the exclusive decision of JAMS and shall be made within thirty (30) days of the written application to JAMS. TheArbitrator shall be a disinterested party.(c) Within thirty (30) days of the selection of the Arbitrator, the Parties involved in the dispute shall meet in New York, New Yorkwith such Arbitrator at a place and time designated by such Arbitrator after consultation with such Parties and present their respective positions on thedispute. Each Party shall have no longer than one day to present its position, the entire proceedings before the Arbitrator shall be no more than threeconsecutive days, and the decision of the Arbitrator shall be made in writing no more than thirty (30) days following the end of the proceeding. Suchan award shall be a final and binding determination of the dispute and shall be fully enforceable as an arbitration decision in any court having Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 10 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 jurisdiction and venue over such Parties. The Parties shall bear their own legal fees and expenses, and shall share equally fees and expenses of theArbitrator and JAMS, regardless of the outcome. Except as may be required by applicable law (or as required to enter and enforce the judgment incourt), no Party (or its representative, witnesses or arbitrators) may disclose the content or result of any arbitration under this Agreement without theprior written consent of the other Party (or Parties) involved in such arbitration.18.6 Patents and Trademarks; Equitable Relief.(a) Any dispute, controversy or claim arising out of, relating to or in connection with the scope, validity, enforceability orinfringement of any patent rights shall in each case be submitted to a court of competent jurisdiction in the territory in which such patent rights weregranted or arose. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 11 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 (b) Any dispute, controversy or claim arising out of, relating to or in connection with the need to seek preliminary or injunctivemeasures or other equitable relief (e.g., in the event of a potential or actual breach of the confidentiality and non-use provisions in Section 6.2) neednot be resolved through the procedure described in Section 18.5 but may be immediately brought in a court of competent jurisdiction.[Remainder of Page Intentionally Left Blank] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 12 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 IN WITNESS WHEREOF, the parties hereto have executed this Agreement as of the day and year set forth above. THE COMPANY: MEIRAGTX HOLDINGS PLC By: /s/ Richard Giroux Name: Richard Giroux Title: Chief Operating OfficerCONSULTING ENTITY: VECTOR CONSULTING LLC By: /s/ Matthew During Name: Matthew During Title: PresidentCONSULTANT: /s/ Michael G. Kaplitt Michael G. KaplittCONSULTANT: /s/ Matthew During Matthew DuringCONSULTANT: /s/ Stephen B. Kaplitt Stephen B. Kaplitt Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 SCHEDULE ADescription of Services and Payments1. Certain Definitions. As used in this Agreement, the following terms shall have the following respective meanings:“Cause” means any of: (a) a breach by the Company of any of its material obligations under this Agreement; (b) a decision by theCompany to cease funding or other support for (i) the development activities related to the Services, or (ii) the development, marketing and sale ofGAD Products; (c) a decision by the Company to reduce, delay or suspend the funding or other support for the development activities related to theServices in a manner that would reasonably be expected to prevent or unreasonably delay Regulatory Approval for GAD Products; (d) death orDisability of both Michael G. Kaplitt and Matthew During; or (e) insolvency or liquidation of the Company.“Competitive Activity” means employment with, owning any interest in, or acting as a paid consultant, advisor, officer, director ormanager of or to any business engaged in: (a) the research, development, sale or marketing of any application of glutamic acid decarboxylase (GAD)gene therapy or neuropeptide (NPY) gene therapy; (b) any business activity conducted by the Company on the date of this Agreement; or (c) anybusiness activity relating to the Services conducted or contemplated to be conducted by the Company on the date of this Agreement or during theConsultation Period; provided, however, that notwithstanding the foregoing, “Competitive Activity” shall expressly exclude the following:(u) activities by Michael G. Kaplitt for or on behalf of Circuit Therapeutics, Inc. or its affiliates that do not directly compete with the Services or theCovered Products; (v) any passive investment by a Consultant (whether debt, equity or otherwise) in a publicly-traded security, where such investmentdoes not result in the Consultant owning 5.0% or more of the equity interests of such publicly-traded company; (w) any investments by a Consultant(whether debt, equity or otherwise) in any non-publicly-traded company which investments do not, in the aggregate, exceed 15.0% of the totalinvestment capital received by such company; (x) any activities conducted by or for the primary benefit of a charitable, not-for-profit or academicentity (including, without limitation, accredited hospitals and universities); (y) speaking, lecturing and writing for publication or at academic or trademeetings and conferences; and (z) any activities by Stephen B. Kaplitt acting in his capacity as an attorney.“Covered Products” means the GAD Products and the IP-related Covered Products.“FDA” means the United States Food and Drug Administration and any successor thereto. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. A-1 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 “Disability” means the inability to perform, with or without reasonable accommodation, the essential functions of the Services for a total ofthree months during any six month period as a result of incapacity due to mental or physical illness as determined by a physician selected in good faithby the Company. A Consultant’s refusal to submit to examination by such physician for the purpose of determining whether a Disability exists shall beconclusive evidence that that no such Disability exists.“EMA” means the European Medicines Agency and any successor thereto.“GAD Products” means any products produced by the Company or its Affiliates (directly or through any third party contractmanufacturers) that uses the adeno-associated virus gene therapy encoding glutamic acid decarboxylase (formerly known as NLX-P101) for thetreatment of Parkinson’s disease that was previously under development by Neurologix.“IP-related Covered Products” means any products (other than the GAD Products) produced by the Company or its Affiliates (directly orthrough any third party contract manufacturers) that (a) uses or incorporates adeno-associated virus gene therapy encoding glutamic aciddecarboxylase to treat a disease other than Parkinson’s disease; or (b)(i) uses any of the intellectual property listed on Appendix 1, and (ii) is coveredby a Valid Claim.“Project” means the Company’s project to develop, obtain Regulatory Approval for, and market and sell Covered Products.“Regulatory Approval” means, with respect to a Covered Product, receipt or issuance of all approvals, licenses, registrations, clearancesand authorizations by the FDA or EMA necessary to lawfully market and sell such GAD Product in the United States or Europe, as applicable.“Services” shall have the meaning set forth in Section 2 of this Schedule A.“Valid Claim” means a claim in an issued patent listed on Appendix 1 that has not: (a) expired or been canceled; (b) been declared invalidby an unreversed and unappealable or unappealed decision of a court or other appropriate body of competent jurisdiction; (c) been admitted to beinvalid or unenforceable through reissue, disclaimer or otherwise; or (d) been abandoned.2. Scope of Work. The following consulting, advisory and related services in support and furtherance of the Project (“Services”) shall beperformed by the Consulting Entity and the Consultants, jointly:2.1 advise and assist with clinical development strategies for Covered Products including Regulatory Approval in the U.S. and inEurope (with the assistance of regulatory consultants);2.2 prepare development plans to be presented to the Board of Directors; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. A-2 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 2.3 advise and assist with the timely preparation of documents to be submitted to the FDA and other health authorities for review,including for filing of one or more New Drug Applications (NDA), as dictated by Board-approved development plans (with the assistance of regulatoryconsultants);2.4 advise and assist with the strategic definition and tactical development of clinical trials programs, including protocol writing,interpretation of clinical data, and literature reviews;2.5 advise and assist with ensuring that all clinical trials are in keeping with approved timelines and budgets, with potential obstaclesidentified and solutions implemented to avoid delays in clinical trial implementation;2.6 advise and assist with ensuring the work with colleagues and collaborators are coordinated and that all people, systems, processesand materials required for clinical trials are available and appropriately prepared;2.7 advise and assist with ensuring that clinical trials are conducted in accordance with applicable regulatory requirements andguidelines;2.8 advise and assist with ensuring the identification, recruitment and selection of appropriate clinical investigators and contractresearch organizations, resulting in appropriate negotiation of contracts;2.9 advise and assist with ensuring the timely medical review and reporting of adverse events;2.10 advise and assist with ensuring the timely preparation of presentations reporting results of clinical trials to internal and externalaudiences;2.11 participate in meetings with the Company’s executive team and other employees from time to time as reasonably requested by theCompany; or2.12 provide such consulting, advisory and related services related to or in furtherance of the foregoing.3. Schedule. The Consulting Entity and each Consultant will coordinate the furnishing of Services pursuant to this Agreement with theCompany in order that such services can be provided in such a way as to generally conform to the business schedules of the Company, but the methodof performance, time of performance, place of performance, hours utilized in such performance, and other details of the manner of performance ofServices hereunder will be within the reasonable discretion of the Consultants and the Consulting Entity.4. Payments Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. A-3 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 4.1 Initial Equity Grants. Upon execution of this Agreement, the Consultants shall be granted the following options (“Options”) topurchase ordinary shares of the Company (“Company Shares”) under the Company’s 2018 Incentive Award Plan (the “Plan”), subject to the terms andprovisions of Section 4.3 of this Schedule A and a stock option agreement in substantially the form set forth in Annex 4.1. The exercise price per shareof each Option will be the Fair Market Value (as defined in the Plan) on the Option’s date of grant: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. A-4 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Consultant Number of Options Final Expiration DateMichael G. Kaplitt [***] 10 years from grant dateMatthew During [***] 10 years from grant dateStephen B. Kaplitt [***] 10 years from grant date4.2 Annual Equity Grants. During the Consultation Period, and subject to the approval of the Company’s Board of Directors, theConsultants shall be eligible to be granted, in the discretion of the Company’s Board of Directors, additional Options to purchase Company Sharesunder the Plan at a price per share equal to the Fair Market Value on the date of grant. The Options shall be granted annually, if at all, as determined bythe Board of Directors. The Options shall be subject to all terms, vesting schedules and other provisions set forth in the Plan and in a separate optionagreement, which shall be in substantially the same form as used for contemporaneous Option grants to the Company’s employees generally.4.3 Death or Permanent Disability; Termination. If, during the term of this Agreement, Michael G. Kaplitt, Stephen B. Kaplitt orMatthew During is no longer able to perform the Services due to death or Disability, the vesting of the Options granted pursuant to Section 4.1 andSection 4.2 to such Consultant that remain unvested at the time of death or Disability shall be accelerated and finally determined pursuant to the tablebelow with no further vesting: Timing of Death or Initial Occurrence of Disability Final VestedPercentage ofUnvested OptionsPrior to the first patient being enrolled in a Phase 3 clinical trial of any GAD Product or IP-related Covered Product 25%Following the first patient being enrolled in a Phase 3 clinical trial of any GAD Product or IP-related Covered Product, butprior to the submission by the Company (and acceptance by the FDA) of a new drug application to the FDA 50%Following Regulatory Approval by the FDA or EMA of any GAD Product or IP-related Covered Product 100% Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. A-5 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Appendix 1 to Schedule A[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. A-6 Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Annex 4.1 to Schedule AForm of Stock Option Agreement Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 MEIRAGTX HOLDINGS PLC2018 INCENTIVE AWARD PLANOPTION GRANT NOTICECapitalized terms not specifically defined in this Option Grant Notice (the “Grant Notice”) have the meanings given to them in the 2018Incentive Award Plan (as amended from time to time, the “Plan”) of MeiraGTx Holdings plc (the “Company”).The Company has granted to the participant listed below (“Participant”) the option described in this Grant Notice (the “Option”), subject to theterms and conditions of the Plan and the Option Agreement attached as Exhibit A (the “Agreement”), both of which are incorporated into this GrantNotice by reference. Participant: Grant Date: Exercise Price per Share: Shares Subject to the Option: Final Expiration Date: Vesting Commencement Date: Vesting Schedule: [To be specified in individual award agreements]Type of Option [Incentive Stock Option/Non-Qualified Stock Option]By Participant’s signature below, Participant agrees to be bound by the terms of this Grant Notice, the Plan and the Agreement. Participant hasreviewed the Plan, this Grant Notice and the Agreement in their entirety, has had an opportunity to obtain the advice of counsel prior to executing thisGrant Notice and fully understands all provisions of the Plan, this Grant Notice and the Agreement. Participant hereby agrees to accept as binding,conclusive and final all decisions or interpretations of the Administrator upon any questions arising under the Plan, this Grant Notice or the Agreement. MEIRAGTX HOLDINGS PLC PARTICIPANTBy: Name: [Participant Name]Title: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Exhibit AOPTION AGREEMENTCapitalized terms not specifically defined in this Agreement have the meanings specified in the Grant Notice or, if not defined in the GrantNotice, in the Plan.ARTICLE I.GENERAL1.1 Grant of Option. The Company has granted to Participant the Option effective as of the grant date set forth in the Grant Notice (the “GrantDate”).1.2 Incorporation of Terms of Plan. The Option is subject to the terms and conditions set forth in this Agreement and the Plan, which isincorporated herein by reference. In the event of any inconsistency between the Plan and this Agreement, the terms of the Plan will control.ARTICLE II.PERIOD OF EXERCISABILITY2.1 Commencement of Exercisability. The Option will vest and become exercisable according to the vesting schedule in the Grant Notice (the“Vesting Schedule”) except that any fraction of a Share as to which the Option would be vested or exercisable will be accumulated and will vest andbecome exercisable only when a whole Share has accumulated. Notwithstanding anything in the Grant Notice, the Plan or this Agreement to thecontrary, unless the Administrator otherwise determines, the Option will immediately expire and be forfeited as to any portion that is not vested andexercisable as of Participant’s Termination of Service for any reason.2.2 Duration of Exercisability. The Vesting Schedule is cumulative. Any portion of the Option which vests and becomes exercisable willremain vested and exercisable until the Option expires. The Option will be forfeited immediately upon its expiration.2.3 Expiration of Option. The Option may not be exercised to any extent by anyone after, and will expire on, the first of the following to occur:(a) The final expiration date in the Grant Notice;(b) Except as the Administrator may otherwise approve, the expiration of three (3) months from the date of Participant’s Termination ofService, unless Participant’s Termination of Service is for Cause or by reason of Participant’s death or Disability;(c) Except as the Administrator may otherwise approve, the expiration of one (1) year from the date of Participant’s Termination ofService by reason of Participant’s death or Disability; and(d) Except as the Administrator may otherwise approve, Participant’s Termination of Service for Cause. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 ARTICLE III.EXERCISE OF OPTION3.1 Person Eligible to Exercise. During Participant’s lifetime, only Participant may exercise the Option. After Participant’s death, anyexercisable portion of the Option may, prior to the time the Option expires, be exercised by Participant’s Designated Beneficiary as provided in thePlan.3.2 Partial Exercise. Any exercisable portion of the Option or the entire Option, if then wholly exercisable, may be exercised, in whole or inpart, according to the procedures in the Plan at any time prior to the time the Option or portion thereof expires, except that the Option may only beexercised for whole Shares.3.3 Tax Withholding.(a) The Company has the right and option, but not the obligation, to treat Participant’s failure to provide timely payment in accordancewith the Plan of any withholding tax arising in connection with the Option as Participant’s election to satisfy all or any portion of the withholding taxby requesting the Company retain Shares otherwise issuable under the Option.(b) Participant acknowledges that Participant is ultimately liable and responsible for all taxes owed in connection with the Option,regardless of any action the Company or any Subsidiary takes with respect to any tax withholding obligations that arise in connection with the Option.Neither the Company nor any Subsidiary makes any representation or undertaking regarding the treatment of any tax withholding in connection withthe awarding, vesting or exercise of the Option or the subsequent sale of Shares. The Company and the Subsidiaries do not commit and are under noobligation to structure the Option to reduce or eliminate Participant’s tax liability.ARTICLE IV.OTHER PROVISIONS4.1 Adjustments. Participant acknowledges that the Option is subject to adjustment, modification and termination in certain events as providedin this Agreement and the Plan.4.2 Notices. Any notice to be given under the terms of this Agreement to the Company must be in writing and addressed to the Company in careof the Company’s Secretary at the Company’s principal office or the Secretary’s then-current email address or facsimile number. Any notice to be givenunder the terms of this Agreement to Participant must be in writing and addressed to Participant (or, if Participant is then deceased, to the personentitled to exercise the Option) at Participant’s last known mailing address, email address or facsimile number in the Company’s personnel files. By anotice given pursuant to this Section, either party may designate a different address for notices to be given to that party. Any notice will be deemedduly given when actually received, when sent by email, when sent by certified mail (return receipt requested) and deposited with postage prepaid in apost office or branch post office regularly maintained by the United States Postal Service, when delivered by a nationally recognized express shippingcompany or upon receipt of a facsimile transmission confirmation.4.3 Titles. Titles are provided herein for convenience only and are not to serve as a basis for interpretation or construction of this Agreement.4.4 Conformity to Securities Laws. Participant acknowledges that the Plan, the Grant Notice and this Agreement are intended to conform to theextent necessary with all Applicable Laws and, to the extent Applicable Laws permit, will be deemed amended as necessary to conform to ApplicableLaws. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 4.5 Successors and Assigns. The Company may assign any of its rights under this Agreement to single or multiple assignees, and thisAgreement will inure to the benefit of the successors and assigns of the Company. Subject to the restrictions on transfer set forth in the Plan, thisAgreement will be binding upon and inure to the benefit of the heirs, legatees, legal representatives, successors and assigns of the parties hereto.4.6 Limitations Applicable to Section 16 Persons. Notwithstanding any other provision of the Plan or this Agreement, if Participant is subject toSection 16 of the Exchange Act, the Plan, the Grant Notice, this Agreement and the Option will be subject to any additional limitations set forth in anyapplicable exemptive rule under Section 16 of the Exchange Act (including any amendment to Rule 16b-3) that are requirements for the application ofsuch exemptive rule. To the extent Applicable Laws permit, this Agreement will be deemed amended as necessary to conform to such applicableexemptive rule.4.7 Entire Agreement. The Plan, the Grant Notice and this Agreement (including any exhibit hereto) constitute the entire agreement of theparties and supersede in their entirety all prior undertakings and agreements of the Company and Participant with respect to the subject matter hereof.4.8 Agreement Severable. In the event that any provision of the Grant Notice or this Agreement is held illegal or invalid, the provision will beseverable from, and the illegality or invalidity of the provision will not be construed to have any effect on, the remaining provisions of the GrantNotice or this Agreement.4.9 Limitation on Participant’s Rights. Participation in the Plan confers no rights or interests other than as herein provided. This Agreementcreates only a contractual obligation on the part of the Company as to amounts payable and may not be construed as creating a trust. Neither the Plannor any underlying program, in and of itself, has any assets. Participant will have only the rights of a general unsecured creditor of the Company withrespect to amounts credited and benefits payable, if any, with respect to the Option, and rights no greater than the right to receive the Shares as ageneral unsecured creditor with respect to the Option, as and when exercised pursuant to the terms hereof.4.10 Not a Contract of Employment. Nothing in the Plan, the Grant Notice or this Agreement confers upon Participant any right to continue inthe employ or service of the Company or any Subsidiary or interferes with or restricts in any way the rights of the Company and its Subsidiaries, whichrights are hereby expressly reserved, to discharge or terminate the services of Participant at any time for any reason whatsoever, with or without Cause,except to the extent expressly provided otherwise in a written agreement between the Company or a Subsidiary and Participant.4.11 Counterparts. The Grant Notice may be executed in one or more counterparts, including by way of any electronic signature, subject toApplicable Law, each of which will be deemed an original and all of which together will constitute one instrument.4.12 Incentive Stock Options. If the Option is designated as an Incentive Stock Option:(a) Participant acknowledges that to the extent the aggregate fair market value of shares (determined as of the time the option with respectto the shares is granted) with respect to which options intended to qualify as “incentive stock options” under Section 422 of the Code, including theOption, are exercisable for the first time by Participant during any calendar year exceeds $100,000 or if for any other reason such options do notqualify or cease to qualify for treatment as “incentive stock options” under Section 422 of the Code, such options (including the Option) will betreated as non-qualified stock options. Participant further acknowledges that the rule set forth in the preceding sentence will be applied by taking theOption and other options into account in the order in which they were granted, as determined Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 under Section 422(d) of the Code. Participant acknowledges that amendments or modifications made to the Option pursuant to the Plan that wouldcause the Option to become a Non-Qualified Stock Option will not materially or adversely affect Participant’s rights under the Option, and that anysuch amendment or modification shall not require Participant’s consent. Participant also acknowledges that if the Option is exercised more than three(3) months after Participant’s Termination of Service as an Employee, other than by reason of death or disability, the Option will be taxed as aNon-Qualified Stock Option.(b) Participant will give prompt written notice to the Company of any disposition or other transfer of any Shares acquired under thisAgreement if such disposition or other transfer is made (a) within two (2) years from the Grant Date or (b) within one (1) year after the transfer of suchShares to Participant. Such notice will specify the date of such disposition or other transfer and the amount realized, in cash, other property, assumptionof indebtedness or other consideration, by Participant in such disposition or other transfer.* * * * * Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plcPursuant to 17 C.F.R. Section 200.83 Schedule BEquity Ownership of Consulting Entity Michael G. Kaplitt 45% Matthew During 45% Stephen B. Kaplitt 10% Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Exhibit 10.20Confidential Treatment Requested by MeiraGTx Holdings plcLICENCE AGREEMENTbetweenUCL Business PlcandMeiraGTx UK II LimitedandMeiraGTx LimitedDated: 29 January 2019Ref: [***] (RPE65)Version: 9Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc INDEX 1. Definitions 2 2. Grant of Rights 5 3. Know-how and Confidential Information 8 4. Consideration 10 5. Commercialisation 14 6. Access to Medicines and Ethical Licensing 17 7. Compliance with Laws 17 8. Intellectual Property 18 9. Warranties and Liability 21 10. Duration and Termination 25 11. General 30 Schedule 1 Licensed Technology 35 Schedule 2 Appointment of Expert 36 Schedule 3 Definition of Tobacco Industry Funding (Revised 2009) 37 Schedule 4 Initial Development Plan 38 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. THIS AGREEMENT is made 29 January, 2019BETWEEN: (1)UCL BUSINESS PLC, a company incorporated in England and Wales under company registration number 02776963 whose registered office isThe Network Building, 97 Tottenham Court Road, London W1T 4TP (“UCLB”);and (2)MEIRAGTX UK II LIMITED, a company incorporated in England and Wales with registered number 09348737 and having its registeredoffice at 92 Britannia Walk, London, United Kingdom, N1 7NQ (the “Licensee”); and (3)MEIRAGTX LIMITED, a company incorporated in England and Wales with registered number 09501998 and having its registered office at92 Britannia Walk, London, United Kingdom, N1 7NQ (“Meira”).WHEREAS: A.University College London (“UCL”) has developed certain ocular gene technologies and owns certain intellectual property rights relating tothose gene therapies that the Licensee wishes to acquire rights to for the development and commercialisation of Licensed Products. B.UCL has assigned to UCLB all of its right, title and interest in and to such property. C.The Parties previously entered into a certain License Agreement dated as of February 4, 2015 (the “Original Commencement Date”), amendedon March 27, 2015, July 28, 2017, and December 14, 2017, as well as a certain Licence Addendum Number 5 dated December 15, 2017(collectively, the “Original Agreement”). D.The Parties now desire to amend the Original Agreement to exclude the Specified Technology [***] and to terminate the Licence AddendumNumber 5, and enter into a new agreement for the Specified Technology [***] on the terms set out in this Agreement. E.It is the policy of UCLB that its activities in licensing intellectual property take into consideration ethical and socially responsible licensingprinciples, including ensuring that Licensed Products are made available to fulfil unmet needs in developing countries, and the Licenseeacknowledges and agrees to carry out its activities under this Agreement in a manner which complies with ethical and socially responsiblelicensing principles and which is designed to fulfil such needs, all in accordance with the provisions of this Agreement.NOW IT IS AGREED as follows: 1.DEFINITIONS 1.1In this Agreement:Agreement means this agreement (including the Schedules);Affiliate in relation to a Party, means any entity or person that Controls, is Controlled by, or is under common Control with that Party;Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc Claims means all demands, claims and liability (whether criminal or civil, in contract, tort or otherwise) for losses, damages, costs and expensesof any nature whatsoever and all costs and expenses (including legal costs) incurred in connection therewith;Commencement Date means the date of this Agreement;Competing Product means any [***];Confidential Information means the Know-how, the Materials and all other technical or commercial information that: a)in respect of information provided in documentary form or by way of a model or in other tangible form, at the time of provision ismarked or otherwise designated to show expressly that it is imparted in confidence or which a reasonable person would expect to beconfidential; and b)in respect of information that is imparted orally, any information that the Disclosing Party or its representatives informed the ReceivingParty at the time of disclosure or which a reasonable person would expect to be confidential;Control means direct or indirect beneficial ownership of 50% (or, outside a Party’s home territory, such lesser percentage as is the maximumpermitted level of foreign investment) or more of the share capital, stock or other participating interest carrying the right to vote or todistribution of profits of that Party, as the case may be;Diligent Efforts means, with respect to efforts to be expended by the Licensee with respect to any objective under this Agreement, diligent,reasonable, good faith efforts to accomplish such objective [***], it being understood and agreed that with respect to the research,development, or commercialization of a product, such efforts will be substantially equivalent to those that would normally be exerted oremployed by [***], when utilizing sounds and reasonable scientific, medical and business practice and judgment;Disclosing Party has the meaning given in Clause 3.2;Field means ocular gene therapy;First Commercial Sale means the first sale to a third party of a Licensed Product in a given regulatory jurisdiction after all regulatory andmarketing approvals have been obtained for such Licensed Product in such jurisdiction. A sale shall not be deemed to have occurred if aLicensed Product is provided pursuant to an early access or compassionate use;Indemnitees has the meaning given in Clause 9.7; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 3Confidential Treatment Requested by MeiraGTx Holdings plc Intellectual Property means any and all patents, utility models, registered designs, unregistered design rights, copyright, database rights, rightsin respect of confidential information, rights under data exclusivity laws, rights under orphan drug laws, rights under unfair competition laws,property rights in biological or chemical materials, extension of the terms of any such rights (including supplementary protection certificates),applications for and the right to apply any of the foregoing registered property and rights, and similar or analogous rights in any part of theTerritory;Know-how means: a)the inventions claimed in the Patents; and b)the technical information relating to the inventions claimed in the Patents and data described in the Part B of Schedule 1;Licensed Products means any and all products that are developed, manufactured, used, or sold by or on behalf of the Licensee or its Affiliatesor Sub-licensees and which (a) are within (or are manufactured using a process described in) any claim of the Patents; and/or (b) incorporate, ortheir development or manufacture makes use of, any of the Know-how and/or the Materials;Licensed Technology means the Patents, the Know-how and the Materials set out in Schedule 1;Materials means any and all of the materials referred to in Part C of Schedule 1;Net Sales Value means in respect of the [***]Original Commencement Date has the meaning given in the Recitals;Original Agreement has the meaning given in the Recitals;Parties means UCLB, the Licensee and Meira, and “Party” shall mean either of them;Patent Costs means [***].Patents means any and all of the patents and patent applications referred to in Part A of Schedule 1;Principal Investigators means [***].Receiving Party has the meaning given in Clause 3.2;Regulatory Exclusivity means, with respect to a Licensed Product, any exclusive rights or protection which are recognised, afforded or grantedby any regulatory authority in any country or region with respect to the Licensed Product other than through patent rights;Sub-licensee means any third party (other than an Affiliate) to whom the Licensee grants a sub-licence of its rights under this Agreement inaccordance with Clause 2.3; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 4Confidential Treatment Requested by MeiraGTx Holdings plc Territory means worldwide;Valid Claim means a claim of a patent or patent application that has not been abandoned or allowed to lapse or expired or been held invalid orunenforceable by a court of competent jurisdiction in a final and non-appealable judgment. 2.GRANT OF RIGHTS 2.1LicenceUCLB hereby grants to the Licensee and its Affiliates, and the Licensee hereby accepts on its own behalf and on behalf of its Affiliates, subjectto the provisions of this Agreement: 2.1.1an exclusive (even as to UCL) licence under the Patents, the Know-how and the Materials, with the right to sub-license, subject toClause 2.3, to develop, commercialise, manufacture, have manufactured, use, sell and have sold Licensed Products only in the Field andin the Territory. 2.2UCLB shall at the Licensee’s request and cost execute such formal licences as may be necessary to enable the Licensee to register the licencesgranted to it under this Agreement with the Patent Offices in the relevant Territory. Such formal licence will reflect the terms of this Agreementwhere possible and for the avoidance of doubt if there is a conflict in the terms of such formal licence and this Agreement, the terms of thisAgreement shall prevail. [***] 2.3Sub-LicensingThe Licensee shall have the right to grant sub-licenses under the license in Clause 2.1 to its Affiliates or other third parties through one or morelevels of Sub-licensees except that the Licensee may not grant such a sub-license to any person or the Affiliates of any person involved in: thetobacco industry (as defined by the Cancer Research UK Code of Practice on Tobacco Industry Funding to Universities detailed in Schedule 3);arms dealing; gambling operations; the promotion of violence; child labour or any other illegal activity. A grant of any sub-licence shall beconditioned on the following: (a)The Licensee shall enter into a written agreement with each Sub-licensee and shall ensure that the provisions of eachsub-licence are consistent with the provisions of this Agreement, and the Licensee shall ensure that: (i)the sub-licence sets out all the proposed terms agreed between the Licensee and the Sub-licensee, including, inparticular, all terms as to remuneration; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 5Confidential Treatment Requested by MeiraGTx Holdings plc (ii)the Sub-licensee will maintain complete and accurate records in sufficient detail to permit UCLB to confirm theaccuracy of the calculation of royalty payments under this Agreement; and (iii)the sub-licence imposes obligations of confidentiality on the Sub-licensee which are no less onerous than those set outin Clause 3.2. (b)The Licensee shall procure that each Sub-licensee complies fully at all times with the provisions of its sub-licence. (c)The Licensee shall be liable for all acts and omissions of its Sub-licensees that, if committed by the Licensee, would constitutea breach of any of the provisions of this Agreement. (d)The Licensee shall provide UCLB with a copy of any sub-licence [***] ([***]) days after execution of such sub-licence,provided that the Licensee may redact confidential or proprietary terms from such copy, including financial terms. (e)Each sub-licence shall terminate automatically upon termination of this Agreement for any reason (but not expiry of thisAgreement under Clause 10.1), except where the Sub-licensee was not implicated in or at fault in any circumstances which ledto the termination of this Agreement, UCLB shall on receiving a written request from the relevant Sub-licensee within [***]([***]) days following the date of termination of this Agreement enter into a licence agreement with the Sub-licensee for theLicensed Technology on terms substantially the same as the terms set out in this Agreement (except that the Sub-licensee shallnot be obliged to pay to UCLB any sums equivalent to those sums set out in Clauses 4.1 or 4.3 which have already been paidto UCLB by the Licensee prior to the date of termination). 2.4Reservation of Rights 2.4.1UCLB reserves for itself and UCL the non-exclusive, irrevocable, worldwide, royalty-free right to: (a)Use the Licensed Technology in the Field solely for academic (non-commercially funded) research, publication and teaching;and (b)Grant licenses to academic third parties to use the Licensed Technology in academic research collaborations with UCL andsuch licenses shall not be sublicensable; and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 6Confidential Treatment Requested by MeiraGTx Holdings plc (c)Grant license of the Licensed Technology to post graduate student of UCL for the purpose of conducting a programme of postgraduate academic research and such licenses shall not by sublicensable.In exercising the rights described in Clause 2.4.1(b) and (c), UCL and UCLB shall comply with the provisions of Clause 3 as regardsconfidentiality of the Know-how. 2.4.2UCL and UCLB will refer a request from a third party for a licence to use the Patents in clinical trials or for diagnostic purposesinvolving human subjects to the Licensee, and the Licensee shall liaise directly with such third party. 2.4.3Except for the licences expressly granted by this Clause 2, UCLB grants no rights to the Licensee under this Agreement to or under anyintellectual property other than the Patents, the Know-how and the Materials and hereby reserves all rights under the Patents, theKnow-how and the Materials outside the Field. 2.4.4Nothing in this Agreement shall limit or otherwise affect UCL’s ability to apply for noncommercial grant funding or comply with suchgrant terms and conditions. In the event that any terms of this Agreement conflicts with the terms of any non-commercial grant funding,the Parties shall negotiate in good faith to amend the terms of this Agreement to allow UCL to access such funding provided thatnothing herein shall require the Licensee to agree to alter or modify the scope of the licence granted to it in this Clause 2. 2.5AffiliatesThe Licensee shall: 2.5.1ensure that its Affiliates comply fully with the terms of this Agreement; 2.5.2be responsible for any breach of or non-compliance with this Agreement by its Affiliates as if the breach or non-compliance had been abreach or non-compliance by the Licensee; 2.5.3indemnify in accordance with Clause 9.7 each of the Indemnitees against any Claims which are awarded against or suffered by any ofthe Indemnitees as a result of any breach of or non-compliance with this Agreement by its Affiliates; and 2.5.4ensure that if any Affiliate ceases to be an Affiliate as a result of a change of Control or otherwise, that unless a sub-licence agreement inaccordance with Clause 2.3 is entered into with such an Affiliate, such former Affiliate immediately upon such cessation: (a)ceases developing, manufacturing, having manufactured, using, selling and/ or having sold Licensed Products and ceases alluse or exploitation of the Licensed Technology, for as long as any of the relevant Patents remains in force and/or theKnow-how remains confidential; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 7Confidential Treatment Requested by MeiraGTx Holdings plc (b)returns to the Licensee or destroys any documents or other materials in the former Affiliate’s possession or under its control andthat contain Confidential Information provided under this Agreement relating to the Licensed Technology and/ or LicensedProducts; (c)to the extent possible, takes all action necessary to have any product licences, marketing authorisations, pricing and/ orreimbursement approvals (and any applications for any of the foregoing) which relate to Licensed Products transferred into thename of the Licensee. 2.6Use of Licensed Technology in CombinationUCLB acknowledges and agrees that the Licensee shall be entitled to use the Licensed Technology in combination with other technology,patents, know-how and materials licensed by UCLB to the Licensee under separate licence agreements and with any improvements to theLicensed Technology developed or generated by the Licensee. 3.KNOW-HOW AND CONFIDENTIAL INFORMATION 3.1Confidentiality of Know-how and MaterialsThe Licensee undertakes that for so long as the Know-how and/or the Materials remains confidential, it shall (and shall ensure that its Affiliatesand Sub-licensees) take all reasonable precautions to prevent unauthorised access to the Know-how and the Materials and protect theKnow-how and the Materials in the same manner as it (or they) protect(s) its (or their) own proprietary information, and shall not (and shallensure that its Affiliates and Sub-licensees do not) use the Know-how or the Materials for any purpose, except as expressly licensed hereby andin accordance with the provisions of this Agreement. For the avoidance of doubt, to the extent that any Materials, Know-how or informationrelating to the Patents falls within the public domain (without any breach of this Agreement or any other obligation of confidentiality), thenUCL, the Principal Investigators and UCLB shall be free to use such information without restriction in the same way that any third party wouldhave the freedom to use it. 3.2Confidentiality ObligationsEach Party (“Receiving Party”) undertakes: 3.2.1to maintain as secret and confidential all Confidential Information obtained from, in the case of UCLB, the Licensee or Meira asapplicable, and in the case of the Licensee and Meira, UCLB (“Disclosing Party”) in the course of or in anticipation of this Agreementand to respect the Disclosing Party’s rights therein; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 8Confidential Treatment Requested by MeiraGTx Holdings plc 3.2.2to use such Confidential Information only for the purposes of or as permitted by this Agreement; and 3.2.3subject to Clause 3.3, to disclose such Confidential Information only to those of its employees, contractors, Affiliates, and Sub-licensees(if any) to whom and to the extent that such disclosure is reasonably necessary for the purposes of this Agreement. 3.3Permitted Disclosure 3.3.1The Licensee shall have the right to disclose Confidential Information received from UCLB to: (a)potential or actual customers of Licensed Products to the extent reasonably necessary to promote the sale or use of LicensedProducts and provided that the customer has agreed to confidentiality provisions at least as restrictive as set forth herein; (b)to existing or potential Sub-licensees, collaborators, investors or lenders provided that such third parties have agreed toconfidentiality provisions at least as restrictive as set forth herein; and (c)to its Board of Directors (or similar governing body) and its counsel, accountants and other professional advisers. 3.4Exceptions to ObligationsThe provisions of Clause 3.2 shall not apply to Confidential Information which the Receiving Party can demonstrate by reasonable writtenevidence: 3.4.1was, prior to the Original Commencement Date, in the possession of the Receiving Party and at its free disposal and was not obtained orotherwise acquired directly or indirectly from the Disclosing Party or its Affiliates or their respective employees, students orrepresentatives; or 3.4.2is subsequently disclosed to the Receiving Party without any obligations of confidence by a third party; or 3.4.3is or becomes generally available to the public through no act or default of the Receiving Party or its agents, employees, Affiliates orSub-licensees; or 3.4.4the Receiving Party is required to disclose by or to the courts of any competent jurisdiction, or to any government regulatory agency orfinancial authority, provided that the Receiving Party shall: (a)inform the Disclosing Party as soon as is reasonably practicable; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 9Confidential Treatment Requested by MeiraGTx Holdings plc (b)at the Disclosing Party’s request and cost seek to persuade the court, agency or authority to have the information treated in aconfidential manner, where this is possible under the court, agency or authority’s procedures; and (c)where the disclosure is unavoidable, limit the disclosure of Confidential information to the minimum extent required by law; or 3.4.5which a Party is advised by its information officer that it is required to disclose under the Freedom of Information Act 2000 or theEnvironmental Information Regulations 2004. 3.5Disclosure to EmployeesThe Receiving Party shall procure that all of its employees, contractors, Affiliates and Sub-licensees who have access to any of the DisclosingParty’s Confidential Information to which Clause 3.2 applies, shall be made aware of the obligations of confidence and are bound byobligations of confidentiality at least as restrictive as those set forth herein (which it undertakes to enforce and for which it is legallyresponsible) and the Receiving Party shall only disclose the Disclosing Party’s Confidential Information to those of its subsidiaries, employees,and officers as need to have access thereto wholly necessarily and exclusively for the purposes of this Agreement. 4.CONSIDERATION 4.1Milestone PaymentsWithin [***] ([***]) days following achievement of each of the following milestone events by Licensee, its Affiliates or Sub-licensees, theLicensee shall notify UCLB in writing that the relevant milestone event has been achieved, provide documentary evidence of suchachievement as appropriate and pay to UCLB, within a period of [***] ([***]) days, the amount(s) set out next to such milestone event below: Milestone Event Amount to be paid[***] £[***][***] £[***] 4.2Annual Management FeesOn each date referred to in the following table, the Licensee shall pay to UCLB the annual management fee set out next to such date in thetable. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 10Confidential Treatment Requested by MeiraGTx Holdings plc Date Amount to be paidUpon each anniversary of the Original Commencement Date until[***] £50,000 4.3Sales Linked Milestone PaymentsUpon the first achievement of each of the sales linked milestones set out in the following table by the Licensee, its Affiliates or Sub-licensees,the Licensee shall notify UCLB in writing that the relevant sales linked milestone has been achieved, provide the relevant documentaryevidence and pay to UCLB the amount(s) set out next to such event in the table: Sales Linked Milestones Amount to be paidWhen Net Sales Value reaches £[***] £[***]On the next £[***] of Net Sales Value (when sales cumulatively reach£[***]) £[***]On the next £[***] of Net Sales Value (When sales cumulatively reach£[***]) £[***]On the next £[***] of Net Sales Value (when sales cumulatively reach£[***]) £[***]On the next £[***] of Net Sales Value (when sales cumulatively reach£[***]) £[***]On the next £[***] of Net Sales Value (when sales cumulatively reach£[***]) £[***] 4.4Royalties on Net SalesFor each Licensed Product in each country, the Licensee shall pay to UCLB a royalty of [***]% ([***] per cent) being a percentage of the NetSales Value of such Licensed Product sold by Licensee, its Affiliates or Sub-licensees. The Licensee’s obligations to pay such royalty for agiven Licensed Product in a given country shall begin after the First Commercial Sale of such Licensed Product in such country and shall endon the later to occur of the following: (a) Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 11Confidential Treatment Requested by MeiraGTx Holdings plc expiration of the last Valid Claim of a Patent claiming such Licensed Product in such country; or (b) the tenth (10th) anniversary of the date ofsuch First Commercial Sale in such country; or (c) the expiration of any Regulatory Exclusivity with respect to such Licensed Products in therelevant country. 4.5Combination ProductsIf any Licensed Products are incorporated in any other product (“Combination Product”) sold by the Licensee or its Affiliates and the LicensedProduct is not priced separately from the Combination Product, the Net Sales Value of such Licensed Product shall be deemed to be the fairmarket value of the Licensed Product in the country of sale when sold separately or if not sold separately in the country of sale, in comparablecountries and territories or if neither of the foregoing apply, a reasonable amount which fairly reflects the value of the Licensed Product withinthe Combination Product assuming the Licensed Product is not being sold as a loss leader. 4.6Payment FrequencyRoyalties due under this Agreement, except for the payments due under Clauses, 4.1, 4.2 and 4.3, which are payable upon the date/timespecified in Clauses 4.1, 4.2 and 4.3 as appropriate, shall be paid within [***] ([***]) days following the end of each calendar quarter ending on31 March, 30 June, 30 September and 31 December in each year, in respect of sales of Licensed Products made during such quarter, and within[***] ([***]) days following the termination of this Agreement. 4.7Payment termsAll sums due under this Agreement: 4.7.1are exclusive of Value Added Tax which where applicable will be paid by the Licensee to UCLB in addition; 4.7.2shall be paid in pounds sterling in cash by transferring an amount in aggregate to the following Account name: UCL Business Plc, SortCode: 20 10 53, Account number: 30782270, Address: Barclays Bank Plc, PO Box 11345, London, W12 8GG, and in the case of incomeor amounts received by the Licensee or its Affiliates in a currency other than pounds sterling, the royalty shall be calculated in the othercurrency and then converted into equivalent pounds sterling at the relevant daily spot rate for that currency as quoted in the FinancialTimes newspaper on the last business day of the quarter in relation to which the royalties are payable; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 12Confidential Treatment Requested by MeiraGTx Holdings plc 4.7.3will be made without any set-off, deduction or withholding except as may be required by law. If the Licensee is required by law to makeany deduction or to withhold any part of any amount due to UCLB under this Agreement, the Licensee will give to UCLB properevidence of the amount deducted or withheld and payment of that amount to the relevant taxation authority, and will do all things in itspower to enable or assist UCLB to claim exemption from or, if that is not possible, to obtain a credit for the amount deducted orwithheld under any applicable double taxation or similar agreement from time to time in force; and 4.7.4shall be made by the due date, failing which UCLB may charge interest on any outstanding amount on a daily basis at a rate equivalentto [***]% above the Bank of England pound sterling base rate then in force in London. 4.8Royalty StatementsThe Licensee shall send to UCLB, at the same time as each royalty payment is made in accordance with Clause 4.4, a statement setting out forthe relevant calendar quarter: 4.8.1in respect of each territory or region in which Licensed Products are sold; 4.8.2the types of Licensed Product sold; 4.8.3the quantity of each type sold; 4.8.4the total invoiced price for each type of Licensed Product sold; 4.8.5where relevant, details of any Licensed Products that have been sold other than on arm’s length terms for a cash consideration, includingthe relevant open market price or (if not available) the reasonable price attributed thereto; 4.8.6the amounts deducted from the Net Sales Value as referred to in paragraph (i) to (iv) of that definition (broken down on a product byproduct and category by category basis); and 4.8.7the aggregate royalties on Net Sales Value due to UCLB;in each case expressed both in local currency and pounds sterling and showing the conversion rates used, during the period to which the royaltypayment relates. 4.9RecordsThe Licensee shall keep at its normal place of business detailed and up to date records and accounts showing the quantity, description andinvoiced price or non-cash consideration for all Licensed Products sold by it or its Affiliates or on its or its Affiliates’ behalf, broken down ineach case on a country by country basis, and being sufficient to ascertain the payments due to UCLB under this Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 13Confidential Treatment Requested by MeiraGTx Holdings plc The Licensee shall make such records and accounts available, on reasonable notice, for inspection during business hours by an independentchartered accountant nominated by UCLB for the purpose of verifying the accuracy of any statement or report given by the Licensee to UCLBunder this Clause 4.9. The Licensee shall co-operate reasonably with any such accountant, and shall promptly provide all information andassistance reasonably requested by such accountant. The accountant shall be required to keep confidential all information learnt during anysuch inspection, and to disclose to UCLB only such details as may be necessary to report on the accuracy of the Licensee’s statement or report.UCLB shall be responsible for the accountant’s charges unless the accountant certifies that there is an inaccuracy of more than [***]% ([***]percent) in any royalty statement, in which case the Licensee shall pay his charges in respect of that inspection.The Licensee shall ensure that UCLB has the same rights as those set out in this Clause 4.9 in respect of the Licensee’s Affiliates andSub-licensees.The Licensee shall co-operate with UCLB in good faith to resolve any discrepancies identified during any such inspection and [***], togetherwith interest on late payment as specified in Clause 4.7.4, within [***] following receipt of a copy of the independent chartered accountant’sreport. 4.10Accounting StandardsWhere this Agreement requires a financial calculation to be made or an action to be taken by a party, such calculation will be made or taken inaccordance with the generally accepted accounting principles followed by such party. 5.COMMERCIALISATION 5.1General DiligenceThe Licensee shall use Diligent Efforts to develop and commercially exploit Licensed Products throughout the Territory (including obtainingall and any regulatory approvals which may be required to market and sell the Licensed Products) for the benefit of both Parties. 5.2Competing ActivitiesThe Licensee shall notify UCLB in confidence if it or any of its Affiliates or its Sub-licensees commences any marketing, sale orcommercialisation of any Competing Product or enters into an agreement with any other person with respect to any such activities. 5.3Development PlanThe Licensee’s initial plan for developing and commercialising Licensed Products is set out in Schedule 4 (the “Initial Development Plan”).The Licensee shall provide to UCLB on each anniversary of the Original Commencement Date a written update to the Initial Development Planthat shall: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 14Confidential Treatment Requested by MeiraGTx Holdings plc 5.3.1report on all activities conducted under this Agreement by the Licensee and its Affiliates and Sub-licensees since the OriginalCommencement Date or the date of the previous update (as appropriate); 5.3.2(where applicable) set out the milestone events achieved since the Original Commencement Date or the date of the previous update (asappropriate) and the Licensee’s reasonable estimate of the dates for achieving any future milestone events; 5.3.3set out the current and projected activities being taken or planned to be taken by the Licensee and its Affiliates and Sub-licensees tobring Licensed Products to market in the Territory; and 5.3.4set out the projected sales of Licensed Products (based on the Licensee’s or Sub-licensee’s current forecasts) for each of the next [***]([***]) years following the date of the report.UCLB’s receipt or approval of any update to the Updated Development Plan shall not be taken to waive or qualify the Licensee’s obligationsunder Clause 5.1. 5.4Annual MeetingIn respect of the Licensed Technology, the Licensee will on UCLB’s request meet with UCLB at least once per calendar year, following thesubmission of the update to the relevant Development Plan pursuant to Clause 5.3, to discuss progress with development andcommercialisation of the Licensed Technology. 5.5Development MilestonesIn addition to the Licensee’s obligations under Clause 5.1, the Licensee shall for each Licensed Technology use Diligent Efforts to achieve thedevelopment milestone events specified in Schedule 4 by the dates set out therein. In any instance in which it becomes apparent that aparticular development milestone will not be met due to regulatory, technical, safety or efficacy-related reasons or Force Majeure event(pursuant to Clause 11.1), the Parties in good faith will agree upon an appropriate adjust of such milestone and any subsequent milestones. 5.6Reporting of First Commercial SaleThe Licensee will promptly notify UCLB in writing of the First Commercial Sale of each Licensed Product on a commercial basis in eachcountry within the Territory. 5.7Reporting for Impact Purposes 5.7.1The Licensee acknowledges that part of UCLB’s purpose in licensing the Patents, Know-how and the Materials to the Licensee pursuantthis Agreement is to ensure that the Patents, Know-how and the Materials are made available for use and commercial Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 15Confidential Treatment Requested by MeiraGTx Holdings plc exploitation with the intention of benefitting society and the economy. In order to enable UCLB and UCL to monitor the benefit thatthey are providing, and to enable UCL to demonstrate the impact of its research activities, to society and the economy, the Licensee willupon request provide to UCLB [***], a written report describing in reasonable detail how it has used the Patents, Know-how and theMaterials and the societal and economic benefits generated therefrom. 5.7.2UCLB shall notify and seek permission from the Licensee in advance, in writing if it wishes to use any written reports received from theLicensee (and the information contained therein) pursuant to Clause 5.7.1 in applications for research or other grant related funding andin submissions to Higher Education funding bodies such as HEFCE and/ or HEIF (or any replacements for either of those entities) andlike entities, supplying a written copy of the application for research or other grant related funding or submission (or the relevantsections thereof). The Licensee will respond to UCLB in writing within [***] ([***]) days of receipt of such written information andsubject to the removal of any confidential information as notified in such written request by the Licensee, UCLB and UCL shall beentitled to submit the approved applications for research or other grant related funding and in submissions to Higher Education fundingbodies such as HEFCE and/ or HEIF (or any replacements for either of those entities) and like entities. 5.8QualityThe Licensee shall ensure that all of the Licensed Products marketed by it and its Affiliates and Sub-licensees are of satisfactory quality andcomply with all applicable laws and regulations in each part of the Territory. 5.9Marking of Licensed ProductsTo the extent permitted under the laws of any country, the Licensee shall mark and cause its Affiliates and Sub-Licensees to mark each LicensedProduct with the number of each issued Patent which applies to the Licensed Product and a statement that such Licensed Products are soldunder licence from UCL Business plc. 5.10Disposals of Licensed Products for FreeNotwithstanding the terms of Clause 5.1, the Licensee shall be entitled to supply a reasonable number of Licensed Products to third parties freeof charge as promotional items for the purpose of establishing a market for the Licensed Products in the relevant country or territory or forresearch, evaluation and testing purposes, or for clinical development, provided that the quantity of Licensed Products supplied for free (or forthe cost of manufacture) in each country or territory is not excessive and is in line with normal industry practice in such country or territory.Any Licensed Products disposed of to third parties in accordance with this Clause 5.10 shall not be taken into account for the purposes ofcalculating Net Sales Value. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 16Confidential Treatment Requested by MeiraGTx Holdings plc 5.11Referral to ExpertIf UCLB considers at any time during the period of this Agreement that the Licensee has failed to comply with its obligations under Clause 5.1or 5.3, then the matter shall be referred to an independent expert to answer the following questions: 5.11.1whether the Licensee has complied with its obligations under Clause 5.1 or 5.3; and if not 5.11.2what specific action the Licensee should have taken and/or now needs to take (“Specific Action”) in order to fulfil such obligations andwithin what period the Specific Action should be taken (“Action Period”).The independent expert shall be appointed in accordance with the provisions of Schedule 2 and his decision shall be final and binding on theParties. 5.12Consequences of Expert’s DecisionIf the expert determines that the Licensee has failed to comply with its obligations under Clause 5.1 or 5.3, and if the Licensee fails to take theSpecific Action within the Action Period, UCLB shall be entitled, by giving, at any time within [***] ([***]) months after the end of thatAction Period, not less than [***] ([***]) months’ notice, to (a) convert the licence granted under Clause 2.1 into a non-exclusive licence or(b) terminate this Agreement. 6.ACCESS TO MEDICINES AND ETHICAL LICENSINGLicensee shall use Diligent Efforts to carry out its activities under this Agreement in a manner which complies with ethical and sociallyresponsible licensing principles and which is designed to fulfil unmet needs in developing countries. 7.COMPLIANCE WITH LAWS 7.1General Compliance with LawsThe Licensee will at all times (and will ensure its Affiliates and Sub-licensees) comply with all legislation, rules, regulations and statutoryrequirements applying to and obtain any consents necessary for its use of the Patents, the Know-how and the Materials, the development,manufacture, and sale of Licensed Products in any country or territory. 7.2Bribery ActThe Licensee shall (and shall procure that any persons associated with it engaged in the performance of this Agreement including its Affiliatesand Sub-licensees shall): Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 17Confidential Treatment Requested by MeiraGTx Holdings plc 7.2.1comply with all applicable laws and codes of practice relating to anti-bribery and anti-corruption including the Bribery Act 2010 andwithout prejudice to the foregoing generality, shall not engage in any activity, practice or conduct which would constitute an offenceunder sections 1, 2 or 6 of the Bribery Act 2010 or do or omit to do any act that will cause or lead UCLB to be in breach of the BriberyAct 2010; 7.2.2comply with UCLB’s ethics, anti-bribery and anti-corruption policies as notified to the Licensee from time to time and have, maintain inplace and enforce throughout the term of this Agreement adequate procedures to ensure compliance with Clause 7.2.1; and 7.2.3promptly report to UCLB any request or demand for any undue financial or other advantage of any kind received in connection with theperformance of this Agreement.For the purpose of this Clause 7.2, the meaning of adequate procedures and whether a person is associated with another person shall bedetermined in accordance with the Bribery Act 2010 (and any guidance issued under section 9 of that Act). Breach of this Clause 7.2 shall bedeemed a material breach of this Agreement entitling UCLB to terminate under Clause 10.3.1. 7.3Export Control RegulationsThe Licensee shall ensure that, in using the Patents, Know-how or Materials and in selling Licensed Products, it and its Affiliates, employees,sub-contractors and Sub-licensees comply fully with any United Nations trade sanctions or EU or UK legislation or regulation, from time totime in force, which impose arms embargoes or control the export of goods, technology or software, including weapons of mass destruction andarms, military, paramilitary and security equipment and dual-use items (items designed for civil use but which can be used for militarypurposes) and certain drugs and chemicals. 8.INTELLECTUAL PROPERTY 8.1Obtain and Maintain the Patents 8.1.1The Licensee shall be responsible for the drafting, filing, prosecution and maintenance of all of the Patents at the Licensee’s cost andexpense. Subject to resource availability, UCLB shall use commercially reasonable efforts to provide such assistance as the Licenseemay request to prosecute and maintain the Patents[***]. 8.1.2The Patents will be filed, prosecuted and maintained in the countries and territories where Licensee normally files its patent applicationsand patents for other gene therapy products. The Licensee shall notify UCLB of any decisions as to which (if any) additional countriesto file and maintain Patents in. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 18Confidential Treatment Requested by MeiraGTx Holdings plc 8.1.3The Licensee shall consult with UCLB in relation to all material changes to the patent claims or specifications that would have theeffect of reducing or limiting the scope of the Patents, and not make any such changes without the prior written consent of UCLB. Suchconsent shall not be unreasonably withheld or delayed provided that UCLB has been given as much notice as is practicable, and in anyevent no less than [***] days’ notice (or such shorter period for response dictated by the relevant patent office) of such proposedchanges, and has been given an opportunity to file divisionals, continuations and/or such other types of protection to cover any claimsor subject matter that the Licensee intends to remove from the scope of the Patents. If UCLB fails to respond before the end of the [***]day period (or such shorter period for response dictated by the relevant patent office), the Licensee may proceed with the proposedchanges to the patent claims or specifications. The Licensee will ensure that UCLB receives copies of all correspondence to and fromPatent Offices in respect of the Patents, including copies of all documents generated in or with such correspondence, and shall be givenreasonable notice (or such shorter period for response dictated by the relevant patent office) of and the opportunity to participate in anyconference calls or meetings with the Licensee’s patent attorneys in relation to the drafting, filing, prosecution and maintenance of thePatents, so that UCLB may be continuously informed of progress with the drafting, filing, prosecution and maintenance of the Patents.Such involvement of UCLB under this Clause 8.1.3 shall be at UCLB’s cost and expense. 8.1.4If the Licensee wishes to abandon any application contained with the Patents or not to maintain any such Patent, it shall give [***]([***]) months’ prior written notice to UCLB and on the expiry of such notice period the licences of the relevant Patents granted to theLicensee under this Agreement shall cease. 8.1.5In the event that any of rights granted hereunder become non-exclusive, responsibility for the drafting, filing, prosecution andmaintenance of all of the Patents shall revert to UCLB. 8.2Infringement of the Patents, the Know-how and/or the Materials 8.2.1The Licensee and UCLB shall promptly give to each other written notice if it becomes aware of any infringement or potentialinfringement of any of the Patents or any unauthorised use of the Know-how or the Materials or any challenge to the validity orownership of the Patents, the Know-how or the Materials and the Licensee and UCLB shall consult with each other to decide the bestway to respond to such infringement, unauthorised use or challenge. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 19Confidential Treatment Requested by MeiraGTx Holdings plc 8.2.2The Licensee shall have the primary obligation and right to take action against any third party alleged to be infringing the Patents ormaking unauthorised use of the Know-how or the Materials and to defend the Patents against challenges to validity or ownership at itssole expense, provided that: (a)the Licensee and UCLB shall use their commercially reasonable efforts to eliminate the infringement without litigation. If theefforts of the Licensee, and UCLB are not successful in eliminating the infringement within [***]([***]) days after the infringerhas been formally notified of the infringement by the Licensee, the Licensee shall have the right after consulting with UCLB,to commence suit on its own account; (b)UCLB shall on the Licensee’s request cooperate with the Licensee in such action [***]; (c)the Licensee shall be solely responsible for the conduct of the action or for settlement thereof and shall be entitled to alldamages received from such action, subject to Clause 8.2.4; and (d)if the Licensee is unsuccessful in persuading the alleged infringer to desist within [***] ([***]) months of the Licensee firstbecoming aware of any potential infringement of the Patents for any Licensed Technology or fails without a commerciallyreasonable basis (relative to the Licensed Technology or other technology licenced to Licensee by UCLB) to initiate aninfringement action within [***] ([***]) months of becoming aware of such infringement, UCLB shall have the right, at its solediscretion, to prosecute such infringement under its sole control [***]. 8.2.3Before starting or defending or settling any legal action under Clause 8.2.2, the Licensee shall consult with UCLB as to the advisabilityof the action or defence or settlement, its effect on the good name of UCLB, the public interest, and how the action or defence should beconducted. 8.2.4The Licensee shall [***] in such action or defence. 8.2.5UCLB shall if reasonably requested by the Licensee agree to be joined in any suit to enforce such rights or will take such action in itsown name [***] and shall have the right to be separately represented by its own counsel [***]. Notwithstanding the foregoing, [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 20Confidential Treatment Requested by MeiraGTx Holdings plc 8.3Infringement of Third Party Rights 8.3.1If any warning letter or other notice of infringement is received by the Licensee or UCLB, or legal suit or other action is brought againstthe Licensee or UCLB, alleging infringement of third party rights in the manufacture, use or sale of any Licensed Product or use of anyPatents, Know-how or Materials, that Party shall (in the case of UCLB) promptly provide full details to the Licensee and (in the case ofthe Licensee) promptly provide full details to UCLB, and the Licensee and UCLB shall discuss the best way to respond. 8.3.2The Licensee shall have the right but not the obligation to defend such suit to the extent it relates to Licensee’s or its Affiliates’ orSub-licensee’s activities and shall have the right to settle with such third party, provided that [***]. In the event that the Licensee,Affiliates or Sub-licensees do not take forward an action, UCLB shall have the right, at its sole discretion, to defend such suit under itssole control and [***]. 9.WARRANTIES AND LIABILITY 9.1Warranties by UCLBUCLB warrants as of the Original Commencement Date and undertakes as follows to its reasonable knowledge and without having undertakenany due and careful enquires whether specific or general in nature: 9.1.1It is the owner of the Patents; 9.1.2it has the authority to grant the licences under this Agreement; and. 9.1.3so far as it is aware (having made no enquiry of any third parties or conducted any freedom to operate searches), use and exploitation ofthe Patents will not infringe the intellectual property rights of any third party.UCLB warrants and undertakes: 9.1.4it has full power and authority to enter into and perform this Agreement which, when executed, will constitute valid and legally bindingobligations on UCLB; and 9.2Warranties by the LicenseeThe Licensee warrants as of the Original Commencement Date and undertakes that in respect of the Licensed Technology that: 9.2.1full power and authority to enter into and perform this Agreement, which, when executed, will constitute valid and legally bindingobligations on the Licensee; 9.2.2entry into this Agreement will not result in any breach of, or violation of the terms or provisions of, the constitutional documents of theLicensee or any other agreement or instrument by which it is bound; 9.2.3so far as it is aware (having made no enquiry of any third parties), use and exploitation of the Patents will not infringe the intellectualproperty rights of any third party; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 21Confidential Treatment Requested by MeiraGTx Holdings plc 9.2.4neither it nor any of its Affiliates is currently researching, developing, marketing, selling or otherwise commercialising any CompetingProduct (“Competing Activities”), nor has any of them entered into an agreement with any other person with respect to any CompetingActivities; and 9.2.5it shall notify UCLB if it or any of its Affiliates or its Sub-licensees commences any Competing Activities or enters into an agreementwith any other person with respect to any Competing Activities. 9.3Warranties by MeiraLicensee warrants as of the Original Commencement Date and undertakes that full power and authority to enter into and perform this Agreementwhich, when executed, will constitute valid and legally binding obligations on Licensee. 9.4AcknowledgementsThe Licensee acknowledges that: 9.4.1the inventions claimed in the Patents, and the Know-how and the Materials are at an early stage of development. Accordingly, specificresults cannot be guaranteed and any results, materials, information or other items (together “Delivered Items”) provided under thisAgreement are provided “as is” and without any express or implied warranties, representations or undertakings. As examples, butwithout limiting the foregoing, UCLB does not give any warranty that Delivered Items are of merchantable or satisfactory quality, are fitfor any particular purpose, comply with any sample or description, or are viable, uncontaminated, safe or non-toxic. 9.4.2UCLB has not performed any searches or investigations into the existence of any third party rights that may affect any of the Patents,Know-how or Materials or the use and exploitation of any of the Patents, Know-how or Materials.9.5 No Other Warranties 9.5.1Each of the Parties acknowledges that, in entering into this Agreement, it does not do so in reliance on any representation, warranty orother provision except as expressly provided in this Agreement, and any conditions, warranties or other terms implied by statute orcommon law are excluded from this Agreement to the fullest extent permitted by law. 9.5.2Without limiting the scope of Clause 9.5.1, UCLB does not make any representation nor give any warranty or undertaking: (a)express or implied, including, without limitation, any implied warranties of merchantability or of fitness for a particularpurpose with respect to any Patent, trademark, software, non-public or other information, or tangible research property, licensedor otherwise provided to the Licensee hereunder and hereby disclaims the same; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 22Confidential Treatment Requested by MeiraGTx Holdings plc (b)as to the efficacy or usefulness of the Patents, Know-how or Materials; or (c)whatsoever with regard to the scope of any of the Patents or that any of the Patents is or will be valid or (in the case of anapplication) will proceed to grantor that such Patents may be exploited by the Licensee, Affiliate or Sub-licensee withoutinfringing other patents; or (d)that the Materials or the method used in making or using the Materials are free from liability for patent infringement; or (e)that the use of any of the Patents, Know-how or Materials, Licensed Technology, the manufacture, sale or use of the LicensedProducts, or the exercise of any of the rights granted under this Agreement will not infringe any intellectual property or otherrights of any other person; or (f)that the Know-how or any other information communicated by UCLB to the Licensee under or in connection with thisAgreement will produce Licensed Products of satisfactory quality or fit for the purpose for which the Licensee intended or thatany product will not have any defect, latent or otherwise, and whether or not discoverable by inspection; or (g)as imposing any obligation on UCLB to bring or prosecute actions or proceedings against third parties for infringement or todefend any action or proceedings for revocation of any of the Patents; or (h)as imposing any liability on UCLB in the event that any third party supplies Licensed Products to customers located in theTerritory; or (i)that there will be no similar or competitive products or services manufactured, used, sold or supplied by any third party in theTerritory. 9.6Responsibility for Development of Licensed ProductsThe Licensee shall be exclusively responsible for its and its Affiliates’ and Sub-licensees’ use of the Patents, Know-how and Materials, thetechnical and commercial development and manufacture of Licensed Products and for incorporating any modifications or developments theretothat may be necessary or desirable, for all Licensed Products sold or supplied, notwithstanding any consultancy services or other contributionsthat UCLB and/or UCL may provide in connection with such activities. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 23Confidential Treatment Requested by MeiraGTx Holdings plc 9.7IndemnityThe Licensee shall indemnify each of UCLB and UCL, and each of their respective officers, directors, Council members, employees andrepresentatives (together, the “Indemnitees”) against all third party Claims that may be asserted against or suffered by any of the Indemniteesand which relate to: 9.7.1the use by the Licensee or any of its Affiliates or Sub-licensees of any of the Patents, Know-how or Materials; or 9.7.2use of the Licensed Technology by or on behalf of the Licensee or any of its Affiliates or Sub-licensees; or 9.7.3the development, manufacture, use, marketing or sale of, or any other dealing in, any of the Licensed Products, by or on behalf of theLicensee or any of its Affiliates or Sub-licensees, or subsequently by any customer or any other person, including claims based onproduct liability laws.The indemnity given by the Licensee to each Indemnitee under this Clause 9.7 will not apply to any third party Claim to the extent that it isattributable to the negligence, gross negligence, reckless misconduct or intentional misconduct of any Indemnitee. 9.8Limitations of Liability 9.8.1To the extent that UCLB or any of its Affiliates has any liability in contract, tort, or otherwise under or in connection with thisAgreement, including any liability for breach of warranty, their liability shall be limited in accordance with the following provisions ofthis Clause 9.8. 9.8.2The aggregate liability of UCLB and any of its Affiliates shall be limited to the total income that UCLB has received from the Licenseepursuant to this Agreement (but excluding any other costs or expenses associated with drafting, filing, prosecuting, maintaining ordefending any Patents or providing any assistance to the Licensee) during the period of [***] ([***]) years preceding the date on whichthe liability arises, or fifty thousand pounds (£50,000) sterling, whichever is the higher. 9.8.3The liability of the Licensee to UCLB shall be limited to the limit of its insurance as set out in Clause 9.9.1, except that in the case ofproduct liability, the liability of the Licensee under this Agreement shall be unlimited. 9.8.4In no circumstances shall any Party or any Indemnitee be liable for any loss, damage, costs or expenses of any nature that is (a) of anindirect, special or consequential nature or (b) any loss of profits (whether direct or indirect), revenue, business opportunity or goodwill,which arises directly or indirectly from that Party’s breach or Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 24Confidential Treatment Requested by MeiraGTx Holdings plc nonperformance of this Agreement, or negligence in the performance of this Agreement or from any liability arising in any other wayout of the subject matter of this Agreement even if the Party bringing the claim has advised any other Party or the relevant Indemnitee ofthe possibility of those losses arising, or if such losses were within the contemplation of the Parties or the Indemnitee. 9.8.5Nothing in this Agreement excludes any Party’s liability to the extent that it may not be so excluded under applicable law, includingany such liability for death or personal injury caused by that Party’s negligence, or liability for fraud or fraudulent misrepresentation. 9.9Insurance 9.9.1The Licensee shall take out with a reputable insurance company and maintain at all times during the term of this Agreement public andproduct liability and professional indemnity insurance including against all loss of and damage to property (whether real, personal orintellectual) and injury to persons including death arising out of or in connection with this Agreement and the Licensee’s and itsAffiliates’ and Sub-licensees’ use of the Patents, Know-how or Materials and use, sale of or any other dealing in any of the LicensedProducts. Such insurances may be limited in respect of one claim provided that such limit must be at least [***] pounds (£[***])sterling, unless the Licensee commences any business in manufacturing, distribution, supply or otherwise make available to the publicany products, in which case such limit must be at least [***] pounds (£[***]) sterling. Such insurance shall continue to be maintainedfor a further [***] years from the end of this Agreement. 9.9.2The Licensee will produce to UCLB at all times upon demand proof that the insurance cover required pursuant to Clause 9.9.1 is in forceand evidence that all premiums have been paid up to date. If UCLB becomes aware that the Licensee has failed to maintain theinsurance required pursuant to Clause 9.9.1, UCLB may effect such insurance and the Licensee will reimburse UCLB for the reasonablecost of effecting and maintaining such insurance on demand. 10.DURATION AND TERMINATION 10.1Commencement and ExpiryThis Agreement shall commence as of the Commencement Date and, unless terminated earlier in accordance with this Clause 10, the licencesgranted hereunder shall continue in force on a country by country basis until the later of the last payment obligation of Licensee expires underthis Agreement. Upon such expiry, Licensee’s licenses under this Agreement shall become full-paid, perpetual and irrevocable. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 25Confidential Treatment Requested by MeiraGTx Holdings plc 10.2Termination of the Original AgreementUpon the Commencement Date, pursuant to Amendment No. 4 to the Original Agreement, the Licensed Technology shall be excluded from theOriginal Agreement, and Licence Addendum Number 5 shall be deemed to have terminated. All other provisions of the Original Agreement(including the options set out in Clause 2.6 of the Original Agreement which have not been exercised by the Licensee prior to theCommencement Date, i.e., the options in relation to Specified Technologies [***]) together with any accrued rights of the Parties under theOriginal Agreement prior to the Commencement Date shall continue in full force and effect. 10.3Early TerminationEach Party (the “Terminating Party”) may terminate this Agreement at any time by notice in writing to the other Parties (“Other Parties”),such notice to take effect as specified in the notice: 10.3.1If, in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, is in material breach of this Agreementand, in the case of a breach capable of remedy within thirty (30) days, the breach is not remedied within thirty (30) days of the OtherParties receiving notice specifying the breach and requiring its remedy or where the breach relates to non-payment of an undisputed sumdue under this Agreement, the sum is not paid in full within fourteen (14) days following the Other Party receiving notice specifying thenonpayment and requiring payment in full; provided however, that in respect on breaches not relating to non-payment, if such breach iscapable of being cured but cannot be cured within such thirty (30) day period and the Other Party initiates actions to cure such breachwithin such period and thereafter diligently pursues such actions, the Other Party shall have such additional period as is reasonableunder the circumstances to cure such breach; it being understood that no such extension shall apply with respect to any undisputedpayment obligations or extend beyond six (6) months from the end of such thirty (30) day period. In the event there is a genuine disputebetween the Parties with respect to any alleged breach hereunder, no purported termination of this Agreement pursuant to this Clause10.3.1 shall take effect while the Parties are actively working to resolve such dispute; or 10.3.2if: (a)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, becomes insolvent or unable topay its debts as and when they become due; (b)an order is made or a resolution is passed for the winding up of in the case of UCLB, either of the Other Parties, or in the case ofthe Licensee or Meira, UCLB (other than voluntarily for the purpose of solvent amalgamation or reconstruction); Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 26Confidential Treatment Requested by MeiraGTx Holdings plc (c)a liquidator, administrator, administrative receiver, receiver or trustee is appointed in respect of the whole or any part of, in thecase of UCLB, either of the Other Parties’, or in the case of the Licensee or Meira, UCLB’s, assets or business; (d)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, makes any composition with itscreditors; (e)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, ceases to continue its business;or (f)any event analogous to the events referred to in paragraphs (a) to (e) above occurs in any other jurisdiction. 10.4UCLB may terminate this Agreement by giving written notice to the Licensee and Meira, such termination to take effect forthwith or asotherwise stated in the notice: 10.4.1if there is any change of Control of the Licensee involving the categories of persons or Affiliates of persons prohibited by Clause 2.3; 10.4.2the Licensee is in persistent breach of the Agreement at least [***] ([***]) times in a calendar year, wherein the nature of each suchbreach is the same in each instance, and where the Parties have failed to agree on a mechanism to remedy the persistent nature of suchbreaches within a reasonable period following UCLB notifying Licensee of the persistent breaches and requesting that the Licenseeenters into discussions with UCLB as to mechanisms for remedying the persistent breaches or if the Parties have agreed to a mechanismto remedy the persistence of such breach by that mechanism if not fully complied with by the Licensee; or 10.4.3if the Licensee shall enter into any sub-licence with any of the categories of persons or Affiliates of persons prohibited by Clause 2.3which may, adversely affect UCL’s and/or UCLB’s reputation; 10.4.4subject to Clause 5.5, if the Licensee fails to achieve any of the milestone events described in Schedule 4 provided that if achievementof any of the milestone events should be compromised due to technical, legal or regulatory issues, the Parties shall first meet and UCLBwill work with the Licensee to manage the delivery schedule and provided that the Licensee is using Diligent Efforts to correct theissues, the applicable deadline in Schedule 4 shall be extended by six (6) months or such other time period as shall be agreed betweenthe Parties in writing after which if the Licensee has not achieved the milestone UCLB shall be entitled, subject to Clauses 5.11 and5.12, to terminate this Agreement by giving written notice to the Licensee. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 27Confidential Treatment Requested by MeiraGTx Holdings plc 10.5A Party’s right of termination under this Agreement, and the exercise of any such right, shall be without prejudice to any other right or remedy(including any right to claim damages) that such Party may have in the event of a breach of contract or other default by any other Party. 10.6Consequences of Termination 10.6.1Upon expiry of the period of this Agreement, and subject to all royalties and any other sums due to UCLB under this Agreement havingbeen duly paid, the Licensee shall have a fully paid up licence to the Patents, the Know-how and the Materials of the same scope as setforth in Clause 2.1 without any further obligation to pay any further sums to UCLB under Clause 4. Notwithstanding the foregoing theLicensee acknowledges that once each Patent expires or is abandoned or withdrawn or allowed to lapse in any country or territory, thirdparties in that country or territory will be entitled to use the inventions claimed in the Patent and that accordingly the licence granted tothe Licensee under Clause 2.1 will no longer be exclusive in that country or territory. 10.6.2Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4: (a)the Licensee and its Affiliates and Sub-licensees shall be entitled to sell, use or otherwise dispose of (subject to payment ofroyalties under Clause 4) any unsold or unused stocks of the Licensed Products for a period of [***] ([***]) months followingthe date of termination; (b)subject to paragraph (a) above, any license that has not become fully paid-up in accordance with Clause 10.1 shall terminateand the Licensee and its Affiliates (and subject to Clause 2.3, its Sub-licensees) shall no longer be licensed to use or otherwiseexploit the Patents and/or the Know-how and/or the Materials, in so far and for as long as any of the Patents remains in forceand the Know-how remains confidential; (c)the Licensee shall consent to the cancellation of any formal licence granted to it, or of any registration of it in any register, inrelation to any of the Patents; (d)the Licensee will, promptly on UCLB’s request, provide (and will ensure that its patent agents provide) to UCLB allinformation, documentation and assistance (including executing documents) which are in the possession of Licensee or itspatent agents relating to the Patents and which UCLB may reasonably require to enable it to continue with the drafting, filing,prosecution and maintenance of the Patents; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 28Confidential Treatment Requested by MeiraGTx Holdings plc (e)except as set out in Clause 2.3, all sub-licences of the Patents and/or the Know-how and/or the Materials granted by theLicensee pursuant to this Agreement will automatically terminate; (f)UCLB shall, upon the written request of either of the other Parties, and each of the Licensee and Meira shall, upon the writtenrequest of UCLB, return or destroy any documents or other materials that are in its or its Affiliates possession or under its ortheir control and that contain the requesting Party’s Confidential Information. 10.6.3Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4, in theevent that UCLB would be unable, absent a licence from the Licensee, to use or permit others to use or to exploit or permit others toexploit the relevant Licensed Technology without infringing intellectual property rights in any invention developed by the Licensee,whether solely or jointly with others (“Blocking Invention”), the Licensee shall be deemed to have granted UCLB the irrevocablenon-exclusive right to use, exploit and permit others to use and exploit the Blocking Invention only in conjunction with the relevantLicensed Technology. The Licensee shall at the request of UCLB provide (and will ensure that its patent agents provide) to UCLB allinformation, documentation and assistance (including executing documents) which UCLB may reasonably require to enable it tocontinue with the drafting, filing, prosecution and maintenance of the Patents licensed under this Agreement; 10.7Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4, the Licenseeshall, to the extent it is able to do so without being in breach of any obligation owed to a third party, disclose to UCLB full details of any andall Intellectual Property generated at any time by or on behalf of the Licensee as a result of the exercise of the Licensee’s rights under thisAgreement (“Licensee IP”) and, upon UCLB’s written request within [***] ([***]) days following such disclosure, negotiate in good faith toagree the terms of an exclusive or non-exclusive licence to UCLB (as UCLB may request) under the Licensee IP. If the Parties fail to agree theterms of such a licence within [***] days following commencement of such negotiation, despite negotiating in good faith, UCLB’s rights underthis Clause shall lapse. If Licensee may terminate this Agreement under Clause 10.3.1 (for UCLB or its Affiliates uncured material breach), thenLicensee may elect, in lieu of terminating the entire Agreement, to have all licenses granted to the Licensee under this Agreement continue inforce, subject to Licensee’s fulfilment of [***] percent ([***]%) of its payment obligations under Clause 4 after what would have been theeffective date of such termination. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 29Confidential Treatment Requested by MeiraGTx Holdings plc 10.8Upon termination of this Agreement for any reason, the provisions of Clauses 1, 2.3, 2.5, 3.1 to 3.5, 4 (in respect of amounts paid and payable toUCLB in respect of the period up to and including the date of termination), 5.7, 7, 9, 10.7, 10.8, 10.8 and 11 of this Agreement shall remain inforce. 11.GENERAL 11.1Force Majeure 11.1.1Any delays in or failure of performance by a Party under this Agreement will not be considered a breach of this Agreement and if and tothe extent that such delay or failure is caused by occurrences beyond the reasonable control of that Party including acts of God; acts,regulations and laws of any government; strikes or other concerted acts of workers; fire; floods; explosions; riots; wars; rebellion; andsabotage; and any time for performance hereunder will be extended by the actual time of delay caused by any such occurrence. 11.1.2If (a) UCLB or (b) the Licensee or Meira is prevented from carrying out its obligations: (a)under this Agreement for a continuous period of [***] ([***]) months, the Licensee (in the case of (a)) or UCLB (in the case of(b)), may terminate this Agreement on giving [***] ([***]) days prior written notice provided always that at the date uponwhich termination becomes effective the Party which was prevented from carrying out its obligations under this Agreementremains so prevented. 11.2AmendmentThis Agreement may only be amended in writing signed by duly authorised representatives of the Parties. 11.3Assignment and Third Party Rights 11.3.1Subject to Clause 11.3.3, the Licensee shall not assign, mortgage, charge or otherwise transfer any rights or obligations under thisAgreement, nor any of the Patents, Know-how or Materials, without the prior written consent of UCLB. 11.3.2UCLB may assign all its rights and obligations under this Agreement together with its rights in the Patents, Know-how and Materials toany third party. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 30Confidential Treatment Requested by MeiraGTx Holdings plc 11.3.3The Licensee, subject to obtaining the consent of UCLB which shall not be unreasonably withheld or delayed (except in relation tothose categories of persons or Affiliates of persons prohibited by Clause 2.3), may assign all its rights and obligations under thisAgreement together with its rights in the Patents, Know-how and Materials to any third party to which it transfers all or substantially allof its assets or business, provided that the assignee undertakes to UCLB to be bound by and perform the obligations of the assignorunder this Agreement. However, the Licensee shall not have such a right to assign this Agreement if it is insolvent. 11.3.4Meira shall not assign, mortgage, charge or otherwise transfer any rights or obligations under this Agreement without the prior writtenconsent of UCLB. 11.4WaiverAny waiver given under or in relation to this Agreement shall be in writing and signed by or on behalf of the relevant Party. No failure or delayon the part of a Party to exercise any right or remedy under this Agreement shall be construed or operate as a waiver thereof, nor shall any singleor partial exercise of any right or remedy preclude the further exercise of such right or remedy. 11.5Invalid ClausesIf any provision or part of this Agreement is held to be invalid, amendments to this Agreement may be made by the addition or deletion ofwording as appropriate to remove the invalid part or provision but otherwise retain the provision and the other provisions of this Agreement tothe maximum extent permissible under applicable law. 11.6No AgencyNo Party shall act or describe itself as the agent of the other, nor shall it make or represent that it has authority to make any commitments on theother’s behalf. 11.7InterpretationIn this Agreement: 11.7.1the headings are used for convenience only and shall not affect its interpretation; references to persons shall include incorporated andunincorporated persons; references to the singular include the plural and vice versa; and references to the masculine include thefeminine; 11.7.2references to Clauses and Schedules mean clauses of, and schedules to, this Agreement; 11.7.3references in this Agreement to termination shall include termination by expiry; 11.7.4where the word “including” is used it shall be understood as meaning “including without limitation”; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 31Confidential Treatment Requested by MeiraGTx Holdings plc 11.7.5any reference to any English law term for any action, remedy, method or judicial proceeding, legal document, legal status, court, officialor any legal concept or thing shall in respect of any jurisdiction other than England be deemed to include what most nearlyapproximates in that jurisdiction to the English law term; 11.7.6 where there is any conflict or inconsistency between the main body of this Agreement and any of the schedules, then the main body ofthe Agreement shall prevail; 11.7.7time shall be of the essence in relation to the performance of Meira’s and the Licensee’s obligations under this Agreement; and 11.7.8any reference to the sale of a Licensed Product by the Licensee or its Affiliates or Sub-licensees will be taken to include any supply orother disposal of Licensed Products, and the term sold shall be construed accordingly. 11.8Notices. Addresses for Service 11.8.1Any notice to be given under this Agreement shall be in English, in writing and shall be delivered by first class recorded delivery mail(if sent to an inland address) or by international courier (if sent to an address outside of the United Kingdom), to the address of therelevant Party set out at the head of this Agreement, or such other address as that Party may from time to time notify to the other Partiesin accordance with this Clause 11.8. 11.8.2Notices sent as above shall be deemed to have been received [***] ([***]) working day after the day of posting in the case of deliveryinland first class recorded delivery mail, or [***] ([***]) working days after the date of collection by the international courier. 11.9Law and JurisdictionThe validity, construction and performance of this Agreement, and any contractual and non-contractual claims arising hereunder, shall begoverned by English law and shall be subject to the exclusive jurisdiction of the English courts to which the Parties hereby submit, except thata Party may seek an interim injunction (or an equivalent remedy) in any court of competent jurisdiction. 11.10Entire AgreementThis Agreement, including its Schedules, sets out the entire agreement between the Parties relating to its subject matter and supersedes all priororal or written agreements, arrangements or understandings between them relating to such subject matter including, without limitation, the partsof the Original Agreement that pertains to the Specified Technology [***] and the Licence Addendum Number 5. Subject to Clause 9.8.5, theParties acknowledge that they are not relying on any representation, agreement, term or condition which is not set out in this Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 32Confidential Treatment Requested by MeiraGTx Holdings plc Agreement. For clarity, this Agreement is not intended to supersede or affect the effectiveness of the parts of the Original Agreement that doesnot pertain to the Specified Technology [***] and the Licence Addendum Number 5. Such parts of the Original Agreement that does not pertainto the Specified Technology [***] and the Licence Addendum Number 5 includes, without limitation, the parts that pertains to the SpecifiedTechnology [***] through [***]. 11.11Third PartiesExcept for the rights of UCL as provided in Clause 2.4, the rights of the Indemnitees as provided in Clause 9.7, the limitations of liabilityafforded to the Indemnitees pursuant to Clause 9.8, who may in their own right enforce and rely on the provisions of those Clauses, thisAgreement does not create any right enforceable by any person who is not a party to it (“Third Party”) under the Contracts (Rights of ThirdParties) Act 1999, but this Clause 11.11 does not affect any right or remedy of a Third Party which exists or is available apart from that Act. 11.12Non-use of Names; Announcements 11.12.1 The Licensee shall not use, and shall ensure that its Affiliates and Sub-licensees do not use, the name, any adaptation of the name, anylogo, trademark or other device of UCLB, nor of the inventors named on the Patents nor the Principal Investigators in any advertising,promotional or sales materials without prior written consent obtained from UCLB in each case, except that the Licensee may state that itis licensed by UCLB under the Patents. 11.12.2 Except as permitted under Clauses 3.3.1 and 5.7, no Party shall make any press or other public announcement concerning any aspect ofthis Agreement, or make any use of the name or trademarks of any other Party in connection with or in consequence of this Agreement,without the prior written consent of the relevant other Party. 11.13EscalationIf the Licensee or Meira on the one hand, and UCLB on the other, are unable to reach agreement on any issue concerning this Agreement or theProject within [***] days after one either has notified the other of that issue, they will refer the matter to the [***] in the case of UCLB, and tothe [***] in the case of the Licensee and Meira in an attempt to resolve the issue within the time specified elsewhere in this Agreement in thecase of other disputes. Any Party may bring proceedings in a court of competent jurisdiction if the matter has not been resolved within thatprescribed period, and any Party may apply to the court for an injunction, whether or not any issue has been escalated under this Clause 11.13.[Signature Page Follows] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 33Confidential Treatment Requested by MeiraGTx Holdings plc EXECUTED on the date set out at the head of this Agreement. For and on behalf of UCL Business PLC For and on behalf of MEIRAGTX UK II LIMITED /s/ [***] /s/ Zandy Forbes Signed Signed [***] Zandy Forbes Print name Print name [***] President and CEO Title Title 29 January 2019 Jan 29, 2019 Date Date For and on behalf of MEIRAGTX LIMITED/s/ Zandy ForbesSignedZandy ForbesPrint namePresident and CEOTitleJan 29, 2019Date Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 34Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 1LICENSED TECHNOLOGYPart A: The Patents[***]Part B: The Know-how[***]Part C: The Materials [***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 35Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 2APPOINTMENT OF EXPERTFor the purposes of this Schedule 2 only, the “Parties” shall mean the Licensee and UCLB. If either Party wishes to appoint an independent expert (the“Expert”) to determine any matter pursuant to any Clause of this Agreement, the following procedures will apply: 1.The Party wishing to appoint the Expert (the “Appointing Party”) will serve a written notice on the other Party (the “Responding Party”). Thewritten notice will specify the Clause pursuant to which the appointment is to be made and will contain reasonable details of the matter(s)which the Appointing Party wishes to refer to the Expert for determination. 2.The Parties shall within [***] ([***]) days following the date of the Appointing Party’s written notice use all reasonable efforts to agree who isto be appointed as the Expert to determine the relevant matter(s). If the Parties are unable to agree upon the identity of the Expert within thattimescale, the Expert shall be appointed by the President (for the time being) of the Licensing Executives Society Britain and Ireland uponwritten request of either Party. 3.Each Party will within [***] ([***]) days following appointment of the Expert, prepare and submit to the Expert and the other Party a detailedwritten statement setting out its position on the matter(s) in question and including any proposals which it may wish to make for settlement orresolution of the relevant matter. 4.Each Party will have [***] ([***]) days following receipt of the other Party’s written statement to respond in writing thereto. Any such responsewill be submitted to the other Party and the Expert. 5.The Expert will if he/ she deems appropriate be entitled to seek clarification from the Parties as to any of the statements or proposals made byeither Party in their written statement or responses. Each Party will on request make available all information in its possession and shall givesuch assistance to the Expert as may be reasonably necessary to permit the Expert to make his/ her determination. 6.The Expert will issue his/ her decision on the matter(s) referred to him/ her in writing as soon as reasonably possible, but at latest within [***]([***]) months following the date of his/ her appointment. The Expert’s decision shall (except in the case of manifest error) be final and bindingon the Parties. 7.The Expert will at all times act as an independent and impartial expert and not as an arbitrator. 8.The Expert’s charges will be borne as he/ she determines in his written decision. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 36Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 3DEFINITION OF TOBACCO INDUSTRY FUNDING (REVISED 2009)FROM THE CANCER RESEARCH UK CODE OF PRACTICE ON TOBACCO INDUSTRY FUNDING TO UNIVERSITIES.http://www.cancerresearchuk.org/science/funding/terms-conditions/funding-policies/policy-tobacco/A tobacco company is defined for the purposes of this policy as one that: • Derives over 5% of revenues from manufacturing tobacco products; • Derives 15%+ of revenues from the manufacture of products necessary for the production of tobacco products; • Derives 15% of revenues from the sale of tobacco products (and has 30 or more staff); • Owns a tobacco company (the company owns 50% or more of a tobacco company); • Is more than 50% owned by a company with tobacco involvement.The following do not constitute tobacco industry funding for the purposes of this Code: • legacies from tobacco industry investments (provided these are sold on immediately) • funding from a trust or foundation no longer having any connection with the tobacco industry even though it may bear a name that (forhistorical reasons) has tobacco industry associations. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 37Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 4DEVELOPMENT PLANThe Development Plan for the Licensed Technology is shown below Activity Timeline Phase I/II Start [***] Phase I/II Finish [***] Phase III /pivotal confirmatory study Start [***] Phase III /pivotal confirmatory study Finish [***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 38[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Exhibit 10.21Confidential Treatment Requested by MeiraGTx Holdings plcLICENCE AGREEMENTbetweenUCL Business PlcandMeiraGTx UK II LimitedandMeiraGTx LimitedandMeiraGTx Holdings plcDated: 29 January 2019Ref: [***] (CNGB3)Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc INDEX 1. Definitions 2 2. Grant of Rights 5 3. Know-how and Confidential Information 8 4. Consideration 11 5. Commercialisation 15 6. Access to Medicines and Ethical Licensing 18 7. Compliance with Laws 19 8. Intellectual Property 20 9. Warranties and Liability 22 10. Duration and Termination 27 11. General 31 Schedule 1 Licensed Technology 37 Schedule 2 Appointment of Expert 38 Schedule 3 Definition of Tobacco Industry Funding (Revised 2009) 39 Schedule 4 Initial Development Plan 40 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plcTHIS AGREEMENT is made 29 January, 2019BETWEEN: (1)UCL BUSINESS PLC, a company incorporated in England and Wales under company registration number 02776963 whose registered office isThe Network Building, 97 Tottenham Court Road, London W1T 4TP (“UCLB”);and (2)MEIRAGTX UK II LIMITED, a company incorporated in England and Wales with registered number 09348737 and having its registeredoffice at 92 Britannia Walk, London, United Kingdom, N1 7NQ (the “Licensee”); and (3)MEIRAGTX LIMITED, a company incorporated in England and Wales with registered number 09501998 and having its registered office at92 Britannia Walk, London, United Kingdom, N1 7NQ (“Meira”); and (4)for the purposes of Section 4.1 only, MEIRAGTX HOLDINGS PLC, a company incorporated in England and Wales with registered numberBR020669 and having its UK establishment address at 92 Britannia Walk, London, United Kingdom, N1 7NQ.WHEREAS: A.University College London (“UCL”) has developed certain ocular gene technologies and owns certain intellectual property rights relating tothose gene therapies that the Licensee wishes to acquire rights to for the development and commercialisation of Licensed Products. B.UCL has assigned to UCLB all of its right, title and interest in and to such property. C.The Parties previously entered into a certain License Agreement dated as of February 4, 2015 (the “Original Commencement Date”), amendedon March 27, 2015, July 28, 2017, and December 14, 2017, as well as a certain Licence Addendum Number 1 dated 4 February 2015(collectively, the “Original Agreement”). D.The Parties now desire to amend the Original Agreement to exclude the Specified Technology [***] and to terminate the Licence AddendumNumber 1, and enter into a new agreement for the Specified Technology [***] on the terms set out in this Agreement. E.It is the policy of UCLB that its activities in licensing intellectual property take into consideration ethical and socially responsible licensingprinciples, including ensuring that Licensed Products are made available to fulfil unmet needs in developing countries, and the Licenseeacknowledges and agrees to carry out its activities under this Agreement in a manner which complies with ethical and socially responsiblelicensing principles and which is designed to fulfil such needs, all in accordance with the provisions of this Agreement.NOW IT IS AGREED as follows: 1.DEFINITIONS 1.1In this Agreement:Agreement means this agreement (including the Schedules);Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc Affiliate in relation to a Party, means any entity or person that Controls, is Controlled by, or is under common Control with that Party;Claims means all demands, claims and liability (whether criminal or civil, in contract, tort or otherwise) for losses, damages, costs and expensesof any nature whatsoever and all costs and expenses (including legal costs) incurred in connection therewith;Commencement Date means the date of this Agreement;Competing Product means [***];Confidential Information means the Know-how, the Materials and all other technical or commercial information that: a)in respect of information provided in documentary form or by way of a model or in other tangible form, at the time of provision ismarked or otherwise designated to show expressly that it is imparted in confidence or which a reasonable person would expect to beconfidential; and b)in respect of information that is imparted orally, any information that the Disclosing Party or its representatives informed the ReceivingParty at the time of disclosure or which a reasonable person would expect to be confidential;Control means direct or indirect beneficial ownership of 50% (or, outside a Party’s home territory, such lesser percentage as is the maximumpermitted level of foreign investment) or more of the share capital, stock or other participating interest carrying the right to vote or todistribution of profits of that Party, as the case may be;Diligent Efforts means, with respect to efforts to be expended by the Licensee with respect to any objective under this Agreement, diligent,reasonable, good faith efforts to accomplish such objective [***], it being understood and agreed that with respect to the research,development, or commercialization of a product, such efforts will be substantially equivalent to those that would normally be exerted oremployed by [***], when utilizing sounds and reasonable scientific, medical and business practice and judgment;Disclosing Party has the meaning given in Clause 3.2;Field means ocular gene therapy;First Commercial Sale means the first sale to a third party of a Licensed Product in a given regulatory jurisdiction after all regulatory andmarketing approvals have been obtained for such Licensed Product in such jurisdiction. A sale shall not be deemed to have occurred if aLicensed Product is provided pursuant to an early access or compassionate use; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.3Confidential Treatment Requested by MeiraGTx Holdings plc Indemnitees has the meaning given in Clause 9.7;Intellectual Property means any and all patents, utility models, registered designs, unregistered design rights, copyright, database rights, rightsin respect of confidential information, rights under data exclusivity laws, rights under orphan drug laws, rights under unfair competition laws,property rights in biological or chemical materials, extension of the terms of any such rights (including supplementary protection certificates),applications for and the right to apply any of the foregoing registered property and rights, and similar or analogous rights in any part of theTerritory;Know-how means: a)the inventions claimed in the Patents; and b)the technical information relating to the inventions claimed in the Patents and data described in the Part B of Schedule 1;Licensed Products means any and all products that are developed, manufactured, used, or sold by or on behalf of the Licensee or its Affiliatesor Sub-licensees and which (a) are within (or are manufactured using a process described in) any claim of the Patents; and/or (b) incorporate, ortheir development or manufacture makes use of, any of the Know-how and/or the Materials;Licensed Technology means the Patents, the Know-how and the Materials set out in Schedule 1;Materials means any and all of the materials referred to in Part C of Schedule 1;Net Sales Value means in respect of [***]Original Commencement Date has the meaning given in the Recitals;Original Agreement has the meaning given in the Recitals;Parties means UCLB, the Licensee and Meira, and “Party” shall mean either of them;Patent Costs means [***].Patents means any and all of the patents and patent applications referred to in Part A of Schedule 1;Principal Investigators means [***].Receiving Party has the meaning given in Clause 3.2; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.4Confidential Treatment Requested by MeiraGTx Holdings plc Regulatory Exclusivity means, with respect to a Licensed Product, any exclusive rights or protection which are recognised, afforded or grantedby any regulatory authority in any country or region with respect to the Licensed Product other than through patent rights;Sub-licensee means any third party (other than an Affiliate) to whom the Licensee grants a sub-licence of its rights under this Agreement inaccordance with Clause 2.3;Territory means worldwide;Valid Claim means a claim of a patent or patent application that has not been abandoned or allowed to lapse or expired or been held invalid orunenforceable by a court of competent jurisdiction in a final and non-appealable judgment. 2.GRANT OF RIGHTS 2.1LicenceUCLB hereby grants to the Licensee and its Affiliates, and the Licensee hereby accepts on its own behalf and on behalf of its Affiliates, subjectto the provisions of this Agreement: 2.1.1an exclusive (even as to UCL) licence under the Patents, the Know-how and the Materials, with the right to sub-license, subject toClause 2.3, to develop, commercialise, manufacture, have manufactured, use, sell and have sold Licensed Products only in the Field andin the Territory. 2.2UCLB shall at the Licensee’s request and cost execute such formal licences as may be necessary to enable the Licensee to register thelicences granted to it under this Agreement with the Patent Offices in the relevant Territory. Such formal licence will reflect the terms ofthis Agreement where possible and for the avoidance of doubt if there is a conflict in the terms of such formal licence and this Agreement,the terms of this Agreement shall prevail. [***]. 2.3Sub-LicensingThe Licensee shall have the right to grant sub-licenses under the license in Clause 2.1 to its Affiliates or other third parties through one or morelevels of Sub-licensees except that the Licensee may not grant such a sub-license to any person or the Affiliates of any person involved in: thetobacco industry (as defined by the Cancer Research UK Code of Practice on Tobacco Industry Funding to Universities detailed in Schedule 3);arms dealing; gambling operations; the promotion of violence; child labour or any other illegal activity. A grant of any sub-licence shall beconditioned on the following: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.5Confidential Treatment Requested by MeiraGTx Holdings plc (a)The Licensee shall enter into a written agreement with each Sub-licensee and shall ensure that the provisions of each sub-licence are consistent with the provisions of this Agreement, and the Licensee shall ensure that: (i)the sub-licence sets out all the proposed terms agreed between the Licensee and the Sub-licensee, including, inparticular, all terms as to remuneration; (ii)the Sub-licensee will maintain complete and accurate records in sufficient detail to permit UCLB to confirm theaccuracy of the calculation of royalty payments under this Agreement; and (iii)the sub-licence imposes obligations of confidentiality on the Sub-licensee which are no less onerous than those set outin Clause 3.2. (b)The Licensee shall procure that each Sub-licensee complies fully at all times with the provisions of its sub-licence. (c)The Licensee shall be liable for all acts and omissions of its Sub-licensees that, if committed by the Licensee, would constitutea breach of any of the provisions of this Agreement. (d)The Licensee shall provide UCLB with a copy of any sub-licence [***] ([***]) days after execution of such sub-licence,provided that the Licensee may redact confidential or proprietary terms from such copy, including financial terms. (e)Each sub-licence shall terminate automatically upon termination of this Agreement for any reason (but not expiry of thisAgreement under Clause 10.1), except where the Sub-licensee was not implicated in or at fault in any circumstances which ledto the termination of this Agreement, UCLB shall on receiving a written request from the relevant Sub-licensee within [***]([***]) days following the date of termination of this Agreement enter into a licence agreement with the Sub-licensee for theLicensed Technology on terms substantially the same as the terms set out in this Agreement (except that the Sub-licensee shallnot be obliged to pay to UCLB any sums equivalent to those sums set out in Clauses 4.1 or 4.3 which have already been paidto UCLB by the Licensee prior to the date of termination). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.6Confidential Treatment Requested by MeiraGTx Holdings plc 2.4Reservation of Rights 2.4.1UCLB reserves for itself and UCL the non-exclusive, irrevocable, worldwide, royalty-free right to: (a)Use the Licensed Technology in the Field solely for academic (non-commercially funded) research, publication and teaching;and (b)Grant licenses to academic third parties to use the Licensed Technology in academic research collaborations with UCL andsuch licenses shall not be sublicensable; and (c)Grant license of the Licensed Technology to post graduate student of UCL for the purpose of conducting a programme of postgraduate academic research and such licenses shall not by sublicensable.In exercising the rights described in Clause 2.4.1(b) and (c), UCL and UCLB shall comply with the provisions of Clause 3 as regardsconfidentiality of the Know-how. 2.4.2UCL and UCLB will refer a request from a third party for a licence to use the Patents in clinical trials or for diagnostic purposesinvolving human subjects to the Licensee, and the Licensee shall liaise directly with such third party. 2.4.3Except for the licences expressly granted by this Clause 2, UCLB grants no rights to the Licensee under this Agreement to or under anyintellectual property other than the Patents, the Know-how and the Materials and hereby reserves all rights under the Patents, theKnow-how and the Materials outside the Field. 2.4.4Nothing in this Agreement shall limit or otherwise affect UCL’s ability to apply for noncommercial grant funding or comply with suchgrant terms and conditions. In the event that any terms of this Agreement conflicts with the terms of any non-commercial grant funding,the Parties shall negotiate in good faith to amend the terms of this Agreement to allow UCL to access such funding provided thatnothing herein shall require the Licensee to agree to alter or modify the scope of the licence granted to it in this Clause 2. 2.5AffiliatesThe Licensee shall: 2.5.1ensure that its Affiliates comply fully with the terms of this Agreement; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.7Confidential Treatment Requested by MeiraGTx Holdings plc 2.5.2be responsible for any breach of or non-compliance with this Agreement by its Affiliates as if the breach or non-compliance had been abreach or non-compliance by the Licensee; 2.5.3indemnify in accordance with Clause 9.7 each of the Indemnitees against any Claims which are awarded against or suffered by any ofthe Indemnitees as a result of any breach of or non-compliance with this Agreement by its Affiliates; and 2.5.4ensure that if any Affiliate ceases to be an Affiliate as a result of a change of Control or otherwise, that unless a sub-licence agreement inaccordance with Clause 2.3 is entered into with such an Affiliate, such former Affiliate immediately upon such cessation: (a)ceases developing, manufacturing, having manufactured, using, selling and/ or having sold Licensed Products and ceases alluse or exploitation of the Licensed Technology, for as long as any of the relevant Patents remains in force and/or theKnow-how remains confidential; (b)returns to the Licensee or destroys any documents or other materials in the former Affiliate’s possession or under its control andthat contain Confidential Information provided under this Agreement relating to the Licensed Technology and/ or LicensedProducts; (c)to the extent possible, takes all action necessary to have any product licences, marketing authorisations, pricing and/ orreimbursement approvals (and any applications for any of the foregoing) which relate to Licensed Products transferred into thename of the Licensee. 2.6Use of Licensed Technology in CombinationUCLB acknowledges and agrees that the Licensee shall be entitled to use the Licensed Technology in combination with other technology,patents, know-how and materials licensed by UCLB to the Licensee under separate licence agreements and with any improvements to theLicensed Technology developed or generated by the Licensee. 3.KNOW-HOW AND CONFIDENTIAL INFORMATION 3.1Confidentiality of Know-how and MaterialsThe Licensee undertakes that for so long as the Know-how and/or the Materials remains confidential, it shall (and shall ensure that its Affiliatesand Sub-licensees) take all reasonable precautions to prevent unauthorised access to the Know-how and the Materials and protect theKnow-how and the Materials in the same manner as it (or they) protect(s) its (or their) own Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.8Confidential Treatment Requested by MeiraGTx Holdings plc proprietary information, and shall not (and shall ensure that its Affiliates and Sub-licensees do not) use the Know-how or the Materials for anypurpose, except as expressly licensed hereby and in accordance with the provisions of this Agreement. For the avoidance of doubt, to the extentthat any Materials, Know-how or information relating to the Patents falls within the public domain (without any breach of this Agreement orany other obligation of confidentiality), then UCL, the Principal Investigators and UCLB shall be free to use such information withoutrestriction in the same way that any third party would have the freedom to use it. 3.2Confidentiality ObligationsEach Party (“Receiving Party”) undertakes: 3.2.1to maintain as secret and confidential all Confidential Information obtained from, in the case of UCLB, the Licensee or Meira asapplicable, and in the case of the Licensee and Meira, UCLB (“Disclosing Party”) in the course of or in anticipation of this Agreementand to respect the Disclosing Party’s rights therein; 3.2.2to use such Confidential Information only for the purposes of or as permitted by this Agreement; and 3.2.3subject to Clause 3.3, to disclose such Confidential Information only to those of its employees, contractors, Affiliates, and Sub-licensees(if any) to whom and to the extent that such disclosure is reasonably necessary for the purposes of this Agreement. 3.3Permitted Disclosure 3.3.1The Licensee shall have the right to disclose Confidential Information received from UCLB to: (a)potential or actual customers of Licensed Products to the extent reasonably necessary to promote the sale or use of LicensedProducts and provided that the customer has agreed to confidentiality provisions at least as restrictive as set forth herein; (b)to existing or potential Sub-licensees, collaborators, investors or lenders provided that such third parties have agreed toconfidentiality provisions at least as restrictive as set forth herein; and (c)to its Board of Directors (or similar governing body) and its counsel, accountants and other professional advisers. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.9Confidential Treatment Requested by MeiraGTx Holdings plc 3.4Exceptions to ObligationsThe provisions of Clause 3.2 shall not apply to Confidential Information which the Receiving Party can demonstrate by reasonable writtenevidence: 3.4.1was, prior to the Original Commencement Date, in the possession of the Receiving Party and at its free disposal and was not obtained orotherwise acquired directly or indirectly from the Disclosing Party or its Affiliates or their respective employees, students orrepresentatives; or 3.4.2is subsequently disclosed to the Receiving Party without any obligations of confidence by a third party; or 3.4.3is or becomes generally available to the public through no act or default of the Receiving Party or its agents, employees, Affiliates orSub-licensees; or 3.4.4the Receiving Party is required to disclose by or to the courts of any competent jurisdiction, or to any government regulatory agency orfinancial authority, provided that the Receiving Party shall: (a)inform the Disclosing Party as soon as is reasonably practicable; (b)at the Disclosing Party’s request and cost seek to persuade the court, agency or authority to have the information treated in aconfidential manner, where this is possible under the court, agency or authority’s procedures; and (c)where the disclosure is unavoidable, limit the disclosure of Confidential information to the minimum extent required by law; or 3.4.5which a Party is advised by its information officer that it is required to disclose under the Freedom of Information Act 2000 or theEnvironmental Information Regulations 2004. 3.5Disclosure to EmployeesThe Receiving Party shall procure that all of its employees, contractors, Affiliates and Sub-licensees who have access to any of the DisclosingParty’s Confidential Information to which Clause 3.2 applies, shall be made aware of the obligations of confidence and are bound byobligations of confidentiality at least as restrictive as those set forth herein (which it undertakes to enforce and for which it is legallyresponsible) and the Receiving Party shall only disclose the Disclosing Party’s Confidential Information to those of its subsidiaries, employees,and officers as need to have access thereto wholly necessarily and exclusively for the purposes of this Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.10Confidential Treatment Requested by MeiraGTx Holdings plc 4.CONSIDERATION 4.1Upfront PaymentsFurthermore, UCLB shall receive the following one-time payments in connection with the grant of the licence in Clause 2.1:(a) Licensee shall pay to UCLB £1,500,000 in cash within [***] ([***]) days following the Commencement Date.(b) MeiraGTx Holdings plc shall issue £1,500,000 in securities of MeiraGTx Holdings plc [***]. The number of such securities of will be equalto the quotient resulting from dividing: (x) £1,500,000; by (y) [***].Milestone PaymentsWithin [***] ([***]) days following achievement of each of the following milestone events by Licensee, its Affiliates or Sub-licensees, theLicensee shall notify UCLB in writing that the relevant milestone event has been achieved, provide documentary evidence of suchachievement as appropriate and pay to UCLB, within a period of [***] ([***]) days, the amount(s) set out next to such milestone event below: Milestone Event Amountto bepaid [***] £[***] [***] £[***] 4.2Annual Management FeesOn each date referred to in the following table, the Licensee shall pay to UCLB the annual management fee set out next to such date in thetable. Date Amountto bepaid Upon each anniversary of the Original Commencement Date until [***] £50,000 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.11Confidential Treatment Requested by MeiraGTx Holdings plc 4.3Sales Linked Milestone PaymentsUpon the first achievement of each of the sales linked milestones set out in the following table by the Licensee, its Affiliates or Sub-licensees,the Licensee shall notify UCLB in writing that the relevant sales linked milestone has been achieved, provide the relevant documentaryevidence and pay to UCLB the amount(s) set out next to such event in the table: Sales Linked Milestones Amountto bepaid When Net Sales Value reaches £[***] £[***] On the next £[***] of Net Sales Value (when sales cumulatively reach £[***]) £[***] On the next £[***] of Net Sales Value (When sales cumulatively reach £[***]) £[***] On the next £[***] of Net Sales Value (when sales cumulatively reach £[***]) £[***] On the next £[***] of Net Sales Value (when sales cumulatively reach £[***]) £[***] On the next £[***] of Net Sales Value (when sales cumulatively reach £[***]) £[***] 4.4Royalties on Net SalesFor each Licensed Product in each country, the Licensee shall pay to UCLB a royalty of [***]% ([***] per cent) being a percentage of the NetSales Value of such Licensed Product sold by Licensee, its Affiliates or Sub-licensees. The Licensee’s obligations to pay such royalty for agiven Licensed Product in a given country shall begin after the First Commercial Sale of such Licensed Product in such country and shall endon the later to occur of the following: (a) expiration of the last Valid Claim of a Patent claiming such Licensed Product in such country; or(b) the tenth (10th) anniversary of the date of such First Commercial Sale in such country; or (c) the expiration of any Regulatory Exclusivitywith respect to such Licensed Products in the relevant country. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.12Confidential Treatment Requested by MeiraGTx Holdings plc 4.5Combination ProductsIf any Licensed Products are incorporated in any other product (“Combination Product”) sold by the Licensee or its Affiliates and the LicensedProduct is not priced separately from the Combination Product, the Net Sales Value of such Licensed Product shall be deemed to be the fairmarket value of the Licensed Product in the country of sale when sold separately or if not sold separately in the country of sale, in comparablecountries and territories or if neither of the foregoing apply, a reasonable amount which fairly reflects the value of the Licensed Product withinthe Combination Product assuming the Licensed Product is not being sold as a loss leader. 4.6Payment FrequencyRoyalties due under this Agreement, except for the payments due under Clauses, 4.1, 4.2 and 4.3, which are payable upon the date/timespecified in Clauses 4.1, 4.2 and 4.3 as appropriate, shall be paid within [***] ([***]) days following the end of each calendar quarter ending on31 March, 30 June, 30 September and 31 December in each year, in respect of sales of Licensed Products made during such quarter, and within[***] ([***]) days following the termination of this Agreement. 4.7Payment termsAll sums due under this Agreement: 4.7.1are exclusive of Value Added Tax which where applicable will be paid by the Licensee to UCLB in addition; 4.7.2shall be paid in pounds sterling in cash by transferring an amount in aggregate to the following Account name: UCL Business Plc, SortCode: 20 10 53, Account number: 30782270, Address: Barclays Bank Plc, PO Box 11345, London, W12 8GG, and in the case of incomeor amounts received by the Licensee or its Affiliates in a currency other than pounds sterling, the royalty shall be calculated in the othercurrency and then converted into equivalent pounds sterling at the relevant daily spot rate for that currency as quoted in the FinancialTimes newspaper on the last business day of the quarter in relation to which the royalties are payable; 4.7.3will be made without any set-off, deduction or withholding except as may be required by law. If the Licensee is required by law to makeany deduction or to withhold any part of any amount due to UCLB under this Agreement, the Licensee will give to UCLB properevidence of the amount deducted or withheld and payment of that amount to the relevant taxation authority, and will do all things in itspower to enable or assist UCLB to claim exemption from or, if that is not possible, to obtain a credit for the amount deducted orwithheld under any applicable double taxation or similar agreement from time to time in force; and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.13Confidential Treatment Requested by MeiraGTx Holdings plc 4.7.4shall be made by the due date, failing which UCLB may charge interest on any outstanding amount on a daily basis at a rate equivalentto [***]% above the Bank of England pound sterling base rate then in force in London. 4.8Royalty StatementsThe Licensee shall send to UCLB, at the same time as each royalty payment is made in accordance with Clause 4.4, a statement setting out forthe relevant calendar quarter: 4.8.1in respect of each territory or region in which Licensed Products are sold; 4.8.2the types of Licensed Product sold; 4.8.3the quantity of each type sold; 4.8.4the total invoiced price for each type of Licensed Product sold; 4.8.5where relevant, details of any Licensed Products that have been sold other than on arm’s length terms for a cash consideration, includingthe relevant open market price or (if not available) the reasonable price attributed thereto; 4.8.6the amounts deducted from the Net Sales Value as referred to in paragraph (i) to (iv) of that definition (broken down on a product byproduct and category by category basis); and 4.8.7the aggregate royalties on Net Sales Value due to UCLB;in each case expressed both in local currency and pounds sterling and showing the conversion rates used, during the period to which the royaltypayment relates. 4.9RecordsThe Licensee shall keep at its normal place of business detailed and up to date records and accounts showing the quantity, description andinvoiced price or non-cash consideration for all Licensed Products sold by it or its Affiliates or on its or its Affiliates’ behalf, broken down ineach case on a country by country basis, and being sufficient to ascertain the payments due to UCLB under this Agreement.The Licensee shall make such records and accounts available, on reasonable notice, for inspection during business hours by an independentchartered accountant nominated by UCLB for the purpose of verifying the accuracy of any statement or report given by the Licensee to UCLBunder this Clause 4.9. The Licensee shall co-operate reasonably with any such accountant, and shall promptly provide all information andassistance reasonably requested by Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.14Confidential Treatment Requested by MeiraGTx Holdings plc such accountant. The accountant shall be required to keep confidential all information learnt during any such inspection, and to disclose toUCLB only such details as may be necessary to report on the accuracy of the Licensee’s statement or report. UCLB shall be responsible for theaccountant’s charges unless the accountant certifies that there is an inaccuracy of more than [***]% ([***] percent) in any royalty statement, inwhich case the Licensee shall pay his charges in respect of that inspection.The Licensee shall ensure that UCLB has the same rights as those set out in this Clause 4.9 in respect of the Licensee’s Affiliates andSub-licensees.The Licensee shall co-operate with UCLB in good faith to resolve any discrepancies identified during any such inspection and [***], togetherwith interest on late payment as specified in Clause 4.7.4, within [***] following receipt of a copy of the independent chartered accountant’sreport. 4.10Accounting StandardsWhere this Agreement requires a financial calculation to be made or an action to be taken by a party, such calculation will be made or taken inaccordance with the generally accepted accounting principles followed by such party. 5.COMMERCIALISATION 5.1General DiligenceThe Licensee shall use Diligent Efforts to develop and commercially exploit Licensed Products throughout the Territory (including obtainingall and any regulatory approvals which may be required to market and sell the Licensed Products) for the benefit of both Parties. 5.2Competing ActivitiesThe Licensee shall notify UCLB in confidence if it or any of its Affiliates or its Sub-licensees commences any marketing, sale orcommercialisation of any Competing Product or enters into an agreement with any other person with respect to any such activities. 5.3Development PlanThe Licensee’s initial plan for developing and commercialising Licensed Products is set out in Schedule 4 (the “Initial Development Plan”).The Licensee shall provide to UCLB on each anniversary of the Original Commencement Date a written update to the Initial Development Planthat shall: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.15Confidential Treatment Requested by MeiraGTx Holdings plc 5.3.1report on all activities conducted under this Agreement by the Licensee and its Affiliates and Sub-licensees since the OriginalCommencement Date or the date of the previous update (as appropriate); 5.3.2(where applicable) set out the milestone events achieved since the Original Commencement Date or the date of the previous update (asappropriate) and the Licensee’s reasonable estimate of the dates for achieving any future milestone events; 5.3.3set out the current and projected activities being taken or planned to be taken by the Licensee and its Affiliates and Sub-licensees tobring Licensed Products to market in the Territory; and 5.3.4set out the projected sales of Licensed Products (based on the Licensee’s or Sub-licensee’s current forecasts) for each of the next [***]([***]) years following the date of the report.UCLB’s receipt or approval of any update to the Updated Development Plan shall not be taken to waive or qualify the Licensee’s obligationsunder Clause 5.1. 5.4Annual MeetingIn respect of the Licensed Technology, the Licensee will on UCLB’s request meet with UCLB at least once per calendar year, following thesubmission of the update to the relevant Development Plan pursuant to Clause 5.3, to discuss progress with development andcommercialisation of the Licensed Technology. 5.5Development MilestonesIn addition to the Licensee’s obligations under Clause 5.1, the Licensee shall for each Licensed Technology use Diligent Efforts to achieve thedevelopment milestone events specified in Schedule 4 by the dates set out therein. In any instance in which it becomes apparent that aparticular development milestone will not be met due to regulatory, technical, safety or efficacy-related reasons or Force Majeure event(pursuant to Clause 11.1), the Parties in good faith will agree upon an appropriate adjust of such milestone and any subsequent milestones. 5.6Reporting of First Commercial SaleThe Licensee will promptly notify UCLB in writing of the First Commercial Sale of each Licensed Product on a commercial basis in eachcountry within the Territory. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.16Confidential Treatment Requested by MeiraGTx Holdings plc 5.7Reporting for Impact Purposes 5.7.1The Licensee acknowledges that part of UCLB’s purpose in licensing the Patents, Know-how and the Materials to the Licensee pursuantthis Agreement is to ensure that the Patents, Know-how and the Materials are made available for use and commercial exploitation withthe intention of benefitting society and the economy. In order to enable UCLB and UCL to monitor the benefit that they are providing,and to enable UCL to demonstrate the impact of its research activities, to society and the economy, the Licensee will upon requestprovide to UCLB [***], a written report describing in reasonable detail how it has used the Patents, Know-how and the Materials and thesocietal and economic benefits generated therefrom. 5.7.2UCLB shall notify and seek permission from the Licensee in advance, in writing if it wishes to use any written reports received from theLicensee (and the information contained therein) pursuant to Clause 5.7.1 in applications for research or other grant related funding andin submissions to Higher Education funding bodies such as HEFCE and/ or HEIF (or any replacements for either of those entities) andlike entities, supplying a written copy of the application for research or other grant related funding or submission (or the relevantsections thereof). The Licensee will respond to UCLB in writing within [***] ([***]) days of receipt of such written information andsubject to the removal of any confidential information as notified in such written request by the Licensee, UCLB and UCL shall beentitled to submit the approved applications for research or other grant related funding and in submissions to Higher Education fundingbodies such as HEFCE and/ or HEIF (or any replacements for either of those entities) and like entities. 5.8QualityThe Licensee shall ensure that all of the Licensed Products marketed by it and its Affiliates and Sub-licensees are of satisfactory quality andcomply with all applicable laws and regulations in each part of the Territory. 5.9Marking of Licensed ProductsTo the extent permitted under the laws of any country, the Licensee shall mark and cause its Affiliates and Sub-Licensees to mark each LicensedProduct with the number of each issued Patent which applies to the Licensed Product and a statement that such Licensed Products are soldunder licence from UCL Business plc. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.17Confidential Treatment Requested by MeiraGTx Holdings plc 5.10Disposals of Licensed Products for FreeNotwithstanding the terms of Clause 5.1, the Licensee shall be entitled to supply a reasonable number of Licensed Products to third parties freeof charge as promotional items for the purpose of establishing a market for the Licensed Products in the relevant country or territory or forresearch, evaluation and testing purposes, or for clinical development, provided that the quantity of Licensed Products supplied for free (or forthe cost of manufacture) in each country or territory is not excessive and is in line with normal industry practice in such country or territory.Any Licensed Products disposed of to third parties in accordance with this Clause 5.10 shall not be taken into account for the purposes ofcalculating Net Sales Value. 5.11Referral to ExpertIf UCLB considers at any time during the period of this Agreement that the Licensee has failed to comply with its obligations under Clause 5.1or 5.3, then the matter shall be referred to an independent expert to answer the following questions: 5.11.1whether the Licensee has complied with its obligations under Clause 5.1 or 5.3; and if not 5.11.2what specific action the Licensee should have taken and/or now needs to take (“Specific Action”) in order to fulfil such obligations andwithin what period the Specific Action should be taken (“Action Period”).The independent expert shall be appointed in accordance with the provisions of Schedule 2 and his decision shall be final and binding on theParties. 5.12Consequences of Expert’s DecisionIf the expert determines that the Licensee has failed to comply with its obligations under Clause 5.1 or 5.3, and if the Licensee fails to take theSpecific Action within the Action Period, UCLB shall be entitled, by giving, at any time within [***] ([***]) months after the end of thatAction Period, not less than [***] ([***]) months’ notice, to (a) convert the licence granted under Clause 2.1 into a non-exclusive licence or(b) terminate this Agreement. 6.ACCESS TO MEDICINES AND ETHICAL LICENSINGLicensee shall use Diligent Efforts to carry out its activities under this Agreement in a manner which complies with ethical and sociallyresponsible licensing principles and which is designed to fulfil unmet needs in developing countries. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.18Confidential Treatment Requested by MeiraGTx Holdings plc 7.COMPLIANCE WITH LAWS 7.1General Compliance with LawsThe Licensee will at all times (and will ensure its Affiliates and Sub-licensees) comply with all legislation, rules, regulations and statutoryrequirements applying to and obtain any consents necessary for its use of the Patents, the Know-how and the Materials, the development,manufacture, and sale of Licensed Products in any country or territory. 7.2Bribery ActThe Licensee shall (and shall procure that any persons associated with it engaged in the performance of this Agreement including its Affiliatesand Sub-licensees shall): 7.2.1comply with all applicable laws and codes of practice relating to anti-bribery and anti-corruption including the Bribery Act 2010 andwithout prejudice to the foregoing generality, shall not engage in any activity, practice or conduct which would constitute an offenceunder sections 1, 2 or 6 of the Bribery Act 2010 or do or omit to do any act that will cause or lead UCLB to be in breach of the BriberyAct 2010; 7.2.2comply with UCLB’s ethics, anti-bribery and anti-corruption policies as notified to the Licensee from time to time and have, maintain inplace and enforce throughout the term of this Agreement adequate procedures to ensure compliance with Clause 7.2.1; and 7.2.3promptly report to UCLB any request or demand for any undue financial or other advantage of any kind received in connection with theperformance of this Agreement.For the purpose of this Clause 7.2, the meaning of adequate procedures and whether a person is associated with another person shall bedetermined in accordance with the Bribery Act 2010 (and any guidance issued under section 9 of that Act). Breach of this Clause 7.2 shall bedeemed a material breach of this Agreement entitling UCLB to terminate under Clause 10.3.1. 7.3Export Control RegulationsThe Licensee shall ensure that, in using the Patents, Know-how or Materials and in selling Licensed Products, it and its Affiliates, employees,sub-contractors and Sub-licensees comply fully with any United Nations trade sanctions or EU or UK legislation or regulation, from time totime in force, which impose arms embargoes or control the export of goods, technology or software, including weapons of mass destruction andarms, military, paramilitary and security equipment and dual-use items (items designed for civil use but which can be used for militarypurposes) and certain drugs and chemicals. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.19Confidential Treatment Requested by MeiraGTx Holdings plc 8.INTELLECTUAL PROPERTY 8.1Obtain and Maintain the Patents 8.1.1The Licensee shall be responsible for the drafting, filing, prosecution and maintenance of all of the Patents at the Licensee’s cost andexpense. Subject to resource availability, UCLB shall use commercially reasonable efforts to provide such assistance as the Licenseemay request to prosecute and maintain the Patents[***]. 8.1.2The Patents will be filed, prosecuted and maintained in the countries and territories where Licensee normally files its patent applicationsand patents for other gene therapy products. The Licensee shall notify UCLB of any decisions as to which (if any) additional countriesto file and maintain Patents in. 8.1.3The Licensee shall consult with UCLB in relation to all material changes to the patent claims or specifications that would have theeffect of reducing or limiting the scope of the Patents, and not make any such changes without the prior written consent of UCLB. Suchconsent shall not be unreasonably withheld or delayed provided that UCLB has been given as much notice as is practicable, and in anyevent no less than [***] days’ notice (or such shorter period for response dictated by the relevant patent office) of such proposedchanges, and has been given an opportunity to file divisionals, continuations and/or such other types of protection to cover any claimsor subject matter that the Licensee intends to remove from the scope of the Patents. If UCLB fails to respond before the end of the [***]day period (or such shorter period for response dictated by the relevant patent office), the Licensee may proceed with the proposedchanges to the patent claims or specifications. The Licensee will ensure that UCLB receives copies of all correspondence to and fromPatent Offices in respect of the Patents, including copies of all documents generated in or with such correspondence, and shall be givenreasonable notice (or such shorter period for response dictated by the relevant patent office) of and the opportunity to participate in anyconference calls or meetings with the Licensee’s patent attorneys in relation to the drafting, filing, prosecution and maintenance of thePatents, so that UCLB may be continuously informed of progress with the drafting, filing, prosecution and maintenance of the Patents.Such involvement of UCLB under this Clause 8.1.3 shall be at UCLB’s cost and expense. 8.1.4If the Licensee wishes to abandon any application contained with the Patents or not to maintain any such Patent, it shall give [***]([***]) months’ prior written notice to UCLB and on the expiry of such notice period the licences of the relevant Patents granted to theLicensee under this Agreement shall cease. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.20Confidential Treatment Requested by MeiraGTx Holdings plc 8.1.5In the event that any of rights granted hereunder become non-exclusive, responsibility for the drafting, filing, prosecution andmaintenance of all of the Patents shall revert to UCLB. 8.2Infringement of the Patents, the Know-how and/or the Materials 8.2.1The Licensee and UCLB shall promptly give to each other written notice if it becomes aware of any infringement or potentialinfringement of any of the Patents or any unauthorised use of the Know-how or the Materials or any challenge to the validity orownership of the Patents, the Know-how or the Materials and the Licensee and UCLB shall consult with each other to decide the bestway to respond to such infringement, unauthorised use or challenge. 8.2.2The Licensee shall have the primary obligation and right to take action against any third party alleged to be infringing the Patents ormaking unauthorised use of the Know-how or the Materials and to defend the Patents against challenges to validity or ownership at itssole expense, provided that: (a)the Licensee and UCLB shall use their commercially reasonable efforts to eliminate the infringement without litigation. If theefforts of the Licensee, and UCLB are not successful in eliminating the infringement within [***] ([***]) days after theinfringer has been formally notified of the infringement by the Licensee, the Licensee shall have the right after consulting withUCLB, to commence suit on its own account; (b)UCLB shall on the Licensee’s request cooperate with the Licensee in such action [***]; (c)the Licensee shall be solely responsible for the conduct of the action or for settlement thereof and shall be entitled to alldamages received from such action, subject to Clause 8.2.4; and (d)if the Licensee is unsuccessful in persuading the alleged infringer to desist within [***] ([***]) months of the Licensee firstbecoming aware of any potential infringement of the Patents for any Licensed Technology or fails without a commerciallyreasonable basis (relative the Licensed Technology or to other technology licenced to Licensee by UCLB) to initiate aninfringement action within [***] ([***]) months of becoming aware of such infringement, UCLB shall have the right, at its solediscretion, to prosecute such infringement under its sole control and [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.21Confidential Treatment Requested by MeiraGTx Holdings plc 8.2.3Before starting or defending or settling any legal action under Clause 8.2.2, the Licensee shall consult with UCLB as to the advisabilityof the action or defence or settlement, its effect on the good name of UCLB, the public interest, and how the action or defence should beconducted. 8.2.4The Licensee shall [***] in such action or defence. 8.2.5UCLB shall if reasonably requested by the Licensee agree to be joined in any suit to enforce such rights or will take such action in itsown name [***] and shall have the right to be separately represented by its own counsel [***]. Notwithstanding the foregoing, [***]. 8.3Infringement of Third Party Rights 8.3.1If any warning letter or other notice of infringement is received by the Licensee or UCLB, or legal suit or other action is brought againstthe Licensee or UCLB, alleging infringement of third party rights in the manufacture, use or sale of any Licensed Product or use of anyPatents, Know-how or Materials, that Party shall (in the case of UCLB) promptly provide full details to the Licensee and (in the case ofthe Licensee) promptly provide full details to UCLB, and the Licensee and UCLB shall discuss the best way to respond. 8.3.2The Licensee shall have the right but not the obligation to defend such suit to the extent it relates to Licensee’s or its Affiliates’ orSub-licensee’s activities and shall have the right to settle with such third party, provided that [***]. In the event that the Licensee,Affiliates or Sub-licensees do not take forward an action, UCLB shall have the right, at its sole discretion, to defend such suit under itssole control and [***]. 9.WARRANTIES AND LIABILITY 9.1Warranties by UCLBUCLB warrants as of the Original Commencement Date and undertakes as follows to its reasonable knowledge and without having undertakenany due and careful enquires whether specific or general in nature: 9.1.1It is the owner of the Patents; 9.1.2it has the authority to grant the licences under this Agreement; and. 9.1.3so far as it is aware (having made no enquiry of any third parties or conducted any freedom to operate searches), use and exploitation ofthe Patents will not infringe the intellectual property rights of any third party. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.22Confidential Treatment Requested by MeiraGTx Holdings plc UCLB warrants and undertakes: 9.1.4it has full power and authority to enter into and perform this Agreement which, when executed, will constitute valid and legally bindingobligations on UCLB; and 9.2Warranties by the LicenseeThe Licensee warrants as of the Original Commencement Date and undertakes that in respect of the Licensed Technology that: 9.2.1full power and authority to enter into and perform this Agreement, which, when executed, will constitute valid and legally bindingobligations on the Licensee; 9.2.2entry into this Agreement will not result in any breach of, or violation of the terms or provisions of, the constitutional documents of theLicensee or any other agreement or instrument by which it is bound; 9.2.3so far as it is aware (having made no enquiry of any third parties), use and exploitation of the Patents will not infringe the intellectualproperty rights of any third party; 9.2.4neither it nor any of its Affiliates is currently researching, developing, marketing, selling or otherwise commercialising any CompetingProduct (“Competing Activities”), nor has any of them entered into an agreement with any other person with respect to any CompetingActivities; and 9.2.5it shall notify UCLB if it or any of its Affiliates or its Sub-licensees commences any Competing Activities or enters into an agreementwith any other person with respect to any Competing Activities. 9.3Warranties by MeiraLicensee warrants as of the Original Commencement Date and undertakes that full power and authority to enter into and perform this Agreementwhich, when executed, will constitute valid and legally binding obligations on Licensee. 9.4AcknowledgementsThe Licensee acknowledges that: 9.4.1the inventions claimed in the Patents, and the Know-how and the Materials are at an early stage of development. Accordingly, specificresults cannot be guaranteed and any results, materials, information or other items (together “Delivered Items”) provided under thisAgreement are provided “as is” and without any express or implied warranties, representations or undertakings. As examples, butwithout limiting the foregoing, UCLB does not give any warranty that Delivered Items are of merchantable or satisfactory quality, are fitfor any particular purpose, comply with any sample or description, or are viable, uncontaminated, safe or non-toxic. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.23Confidential Treatment Requested by MeiraGTx Holdings plc 9.4.2UCLB has not performed any searches or investigations into the existence of any third party rights that may affect any of the Patents,Know-how or Materials or the use and exploitation of any of the Patents, Know-how or Materials. 9.5No Other Warranties 9.5.1Each of the Parties acknowledges that, in entering into this Agreement, it does not do so in reliance on any representation, warranty orother provision except as expressly provided in this Agreement, and any conditions, warranties or other terms implied by statute orcommon law are excluded from this Agreement to the fullest extent permitted by law. 9.5.2Without limiting the scope of Clause 9.5.1, UCLB does not make any representation nor give any warranty or undertaking: (a)express or implied, including, without limitation, any implied warranties of merchantability or of fitness for a particularpurpose with respect to any Patent, trademark, software, non-public or other information, or tangible research property, licensedor otherwise provided to the Licensee hereunder and hereby disclaims the same; (b)as to the efficacy or usefulness of the Patents, Know-how or Materials; or (c)whatsoever with regard to the scope of any of the Patents or that any of the Patents is or will be valid or (in the case of anapplication) will proceed to grantor that such Patents may be exploited by the Licensee, Affiliate or Sub-licensee withoutinfringing other patents; or (d)that the Materials or the method used in making or using the Materials are free from liability for patent infringement; or (e)that the use of any of the Patents, Know-how or Materials, Licensed Technology, the manufacture, sale or use of the LicensedProducts, or the exercise of any of the rights granted under this Agreement will not infringe any intellectual property or otherrights of any other person; or (f)that the Know-how or any other information communicated by UCLB to the Licensee under or in connection with thisAgreement will produce Licensed Products of satisfactory quality or fit for the purpose for which the Licensee intended or thatany product will not have any defect, latent or otherwise, and whether or not discoverable by inspection; or Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.24Confidential Treatment Requested by MeiraGTx Holdings plc (g)as imposing any obligation on UCLB to bring or prosecute actions or proceedings against third parties for infringement or to defend anyaction or proceedings for revocation of any of the Patents; or (h)as imposing any liability on UCLB in the event that any third party supplies Licensed Products to customers located in the Territory; or (i)that there will be no similar or competitive products or services manufactured, used, sold or supplied by any third party in the Territory. 9.6Responsibility for Development of Licensed ProductsThe Licensee shall be exclusively responsible for its and its Affiliates’ and Sub-licensees’ use of the Patents, Know-how and Materials, thetechnical and commercial development and manufacture of Licensed Products and for incorporating any modifications or developments theretothat may be necessary or desirable, for all Licensed Products sold or supplied, notwithstanding any consultancy services or other contributionsthat UCLB and/or UCL may provide in connection with such activities. 9.7IndemnityThe Licensee shall indemnify each of UCLB and UCL, and each of their respective officers, directors, Council members, employees andrepresentatives (together, the “Indemnitees”) against all third party Claims that may be asserted against or suffered by any of the Indemniteesand which relate to: 9.7.1the use by the Licensee or any of its Affiliates or Sub-licensees of any of the Patents, Know-how or Materials; or 9.7.2use of the Licensed Technology by or on behalf of the Licensee or any of its Affiliates or Sub-licensees; or 9.7.3the development, manufacture, use, marketing or sale of, or any other dealing in, any of the Licensed Products, by or on behalf of theLicensee or any of its Affiliates or Sub-licensees, or subsequently by any customer or any other person, including claims based onproduct liability laws.The indemnity given by the Licensee to each Indemnitee under this Clause 9.7 will not apply to any third party Claim to the extent that it isattributable to the negligence, gross negligence, reckless misconduct or intentional misconduct of any Indemnitee. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.25Confidential Treatment Requested by MeiraGTx Holdings plc 9.8Limitations of Liability 9.8.1To the extent that UCLB or any of its Affiliates has any liability in contract, tort, or otherwise under or in connection with thisAgreement, including any liability for breach of warranty, their liability shall be limited in accordance with the following provisions ofthis Clause 9.8. 9.8.2The aggregate liability of UCLB and any of its Affiliates shall be limited to the total income that UCLB has received from the Licenseepursuant to this Agreement (but excluding any other costs or expenses associated with drafting, filing, prosecuting, maintaining ordefending any Patents or providing any assistance to the Licensee) during the period of [***] ([***]) years preceding the date on whichthe liability arises, or fifty thousand pounds (£50,000) sterling, whichever is the higher. 9.8.3The liability of the Licensee to UCLB shall be limited to the limit of its insurance as set out in Clause 9.9.1, except that in the case ofproduct liability, the liability of the Licensee under this Agreement shall be unlimited. 9.8.4In no circumstances shall any Party or any Indemnitee be liable for any loss, damage, costs or expenses of any nature that is (a) of anindirect, special or consequential nature or (b) any loss of profits (whether direct or indirect), revenue, business opportunity or goodwill,which arises directly or indirectly from that Party’s breach or nonperformance of this Agreement, or negligence in the performance ofthis Agreement or from any liability arising in any other way out of the subject matter of this Agreement even if the Party bringing theclaim has advised any other Party or the relevant Indemnitee of the possibility of those losses arising, or if such losses were within thecontemplation of the Parties or the Indemnitee. 9.8.5Nothing in this Agreement excludes any Party’s liability to the extent that it may not be so excluded under applicable law, includingany such liability for death or personal injury caused by that Party’s negligence, or liability for fraud or fraudulent misrepresentation. 9.9Insurance 9.9.1The Licensee shall take out with a reputable insurance company and maintain at all times during the term of this Agreement public andproduct liability and professional indemnity insurance including against all loss of and damage to property (whether real, personal orintellectual) and injury to persons including death arising out of or in connection with this Agreement and the Licensee’s and itsAffiliates’ and Sub-licensees’ use of the Patents, Know-how or Materials and use, sale of or any other Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.26Confidential Treatment Requested by MeiraGTx Holdings plc dealing in any of the Licensed Products. Such insurances may be limited in respect of one claim provided that such limit must be at least[***] pounds (£[***]) sterling, unless the Licensee commences any business in manufacturing, distribution, supply or otherwise makeavailable to the public any products, in which case such limit must be at least [***] pounds (£[***]) sterling. Such insurance shallcontinue to be maintained for a further [***] years from the end of this Agreement. 9.9.2The Licensee will produce to UCLB at all times upon demand proof that the insurance cover required pursuant to Clause 9.9.1 is in forceand evidence that all premiums have been paid up to date. If UCLB becomes aware that the Licensee has failed to maintain theinsurance required pursuant to Clause 9.9.1, UCLB may effect such insurance and the Licensee will reimburse UCLB for the reasonablecost of effecting and maintaining such insurance on demand. 10.DURATION AND TERMINATION 10.1Commencement and ExpiryThis Agreement shall commence as of the Commencement Date and, unless terminated earlier in accordance with this Clause 10, the licencesgranted hereunder shall continue in force on a country by country basis until the later of the last payment obligation of Licensee expires underthis Agreement. Upon such expiry, Licensee’s licenses under this Agreement shall become full-paid, perpetual and irrevocable. 10.2Termination of the Original AgreementUpon the Commencement Date, pursuant to Amendment No. 4 to the Original Agreement, the Licensed Technology shall be excluded from theOriginal Agreement, and Licence Addendum Number 1 shall be deemed to have terminated. All other provisions of the Original Agreement(including the options set out in Clause 2.6 of the Original Agreement which have not been exercised by the Licensee prior to theCommencement Date, i.e., the options in relation to Specified Technologies [***]) together with any accrued rights of the Parties under theOriginal Agreement prior to the Commencement Date shall continue in full force and effect. 10.3Early TerminationEach Party (the “Terminating Party”) may terminate this Agreement at any time by notice in writing to the other Parties (“Other Parties”),such notice to take effect as specified in the notice: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.27Confidential Treatment Requested by MeiraGTx Holdings plc 10.3.1If, in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, is in material breach of this Agreementand, in the case of a breach capable of remedy within thirty (30) days, the breach is not remedied within thirty (30) days of the OtherParties receiving notice specifying the breach and requiring its remedy or where the breach relates to non-payment of an undisputed sumdue under this Agreement, the sum is not paid in full within fourteen (14) days following the Other Party receiving notice specifying thenonpayment and requiring payment in full; provided however, that in respect on breaches not relating to non-payment, if such breach iscapable of being cured but cannot be cured within such thirty (30) day period and the Other Party initiates actions to cure such breachwithin such period and thereafter diligently pursues such actions, the Other Party shall have such additional period as is reasonableunder the circumstances to cure such breach; it being understood that no such extension shall apply with respect to any undisputedpayment obligations or extend beyond six (6) months from the end of such thirty (30) day period. In the event there is a genuine disputebetween the Parties with respect to any alleged breach hereunder, no purported termination of this Agreement pursuant to this Clause10.3.1 shall take effect while the Parties are actively working to resolve such dispute; or 10.3.2if: (a)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, becomes insolvent or unable topay its debts as and when they become due; (b)an order is made or a resolution is passed for the winding up of in the case of UCLB, either of the Other Parties, or in the case ofthe Licensee or Meira, UCLB (other than voluntarily for the purpose of solvent amalgamation or reconstruction); (c)a liquidator, administrator, administrative receiver, receiver or trustee is appointed in respect of the whole or any part of, in thecase of UCLB, either of the Other Parties’, or in the case of the Licensee or Meira, UCLB’s, assets or business; (d)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, makes any composition with itscreditors; (e)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, ceases to continue its business;or (f)any event analogous to the events referred to in paragraphs (a) to (e) above occurs in any other jurisdiction. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.28Confidential Treatment Requested by MeiraGTx Holdings plc 10.4UCLB may terminate this Agreement by giving written notice to the Licensee and Meira, such termination to take effect forthwith or asotherwise stated in the notice: 10.4.1if there is any change of Control of the Licensee involving the categories of persons or Affiliates of persons prohibited by Clause 2.3; 10.4.2the Licensee is in persistent breach of the Agreement at least [***] ([***]) times in a calendar year, wherein the nature of each suchbreach is the same in each instance, and where the Parties have failed to agree on a mechanism to remedy the persistent nature of suchbreaches within a reasonable period following UCLB notifying Licensee of the persistent breaches and requesting that the Licenseeenters into discussions with UCLB as to mechanisms for remedying the persistent breaches or if the Parties have agreed to a mechanismto remedy the persistence of such breach by that mechanism if not fully complied with by the Licensee; or 10.4.3if the Licensee shall enter into any sub-licence with any of the categories of persons or Affiliates of persons prohibited by Clause 2.3which may, adversely affect UCL’s and/or UCLB’s reputation; 10.4.4subject to Clause 5.5, if the Licensee fails to achieve any of the milestone events described in Schedule 4 provided that if achievementof any of the milestone events should be compromised due to technical, legal or regulatory issues, the Parties shall first meet and UCLBwill work with the Licensee to manage the delivery schedule and provided that the Licensee is using Diligent Efforts to correct theissues, the applicable deadline in Schedule 4 shall be extended by six (6) months or such other time period as shall be agreed betweenthe Parties in writing after which if the Licensee has not achieved the milestone UCLB shall be entitled, subject to Clauses 5.11 and5.12, to terminate this Agreement by giving written notice to the Licensee. 10.5A Party’s right of termination under this Agreement, and the exercise of any such right, shall be without prejudice to any other right or remedy(including any right to claim damages) that such Party may have in the event of a breach of contract or other default by any other Party. 10.6Consequences of Termination 10.6.1Upon expiry of the period of this Agreement, and subject to all royalties and any other sums due to UCLB under this Agreement havingbeen duly paid, the Licensee shall have a fully paid up licence to the Patents, the Know-how and the Materials of the same scope as setforth in Clause 2.1 without any further obligation to pay any further sums to UCLB under Clause 4. Notwithstanding the foregoing theLicensee acknowledges that once each Patent expires or is abandoned or withdrawn or allowed to lapse in any country or territory, thirdparties in that country or territory will be entitled to use the inventions claimed in the Patent and that accordingly the licence granted tothe Licensee under Clause 2.1 will no longer be exclusive in that country or territory. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.29Confidential Treatment Requested by MeiraGTx Holdings plc 10.6.2Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4: (a)the Licensee and its Affiliates and Sub-licensees shall be entitled to sell, use or otherwise dispose of (subject to payment of royaltiesunder Clause 4) any unsold or unused stocks of the Licensed Products for a period of [***] ([***]) months following the date oftermination; (b)subject to paragraph (a) above, any license that has not become fully paid-up in accordance with Clause 10.1 shall terminate and theLicensee and its Affiliates (and subject to Clause 2.3, its Sub-licensees) shall no longer be licensed to use or otherwise exploit thePatents and/or the Know-how and/or the Materials, in so far and for as long as any of the Patents remains in force and the Know-howremains confidential; (c)the Licensee shall consent to the cancellation of any formal licence granted to it, or of any registration of it in any register, in relation toany of the Patents; (d)the Licensee will, promptly on UCLB’s request, provide (and will ensure that its patent agents provide) to UCLB all information,documentation and assistance (including executing documents) which are in the possession of Licensee or its patent agents relating tothe Patents and which UCLB may reasonably require to enable it to continue with the drafting, filing, prosecution and maintenance ofthe Patents; (e)except as set out in Clause 2.3, all sub-licences of the Patents and/or the Know-how and/or the Materials granted by the Licenseepursuant to this Agreement will automatically terminate; (f)UCLB shall, upon the written request of either of the other Parties, and each of the Licensee and Meira shall, upon the written request ofUCLB, return or destroy any documents or other materials that are in its or its Affiliates possession or under its or their control and thatcontain the requesting Party’s Confidential Information. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.30Confidential Treatment Requested by MeiraGTx Holdings plc 10.6.3Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4, in theevent that UCLB would be unable, absent a licence from the Licensee, to use or permit others to use or to exploit or permit others toexploit the relevant Licensed Technology without infringing intellectual property rights in any invention developed by the Licensee,whether solely or jointly with others (“Blocking Invention”), the Licensee shall be deemed to have granted UCLB the irrevocablenon-exclusive right to use, exploit and permit others to use and exploit the Blocking Invention only in conjunction with the relevantLicensed Technology. The Licensee shall at the request of UCLB provide (and will ensure that its patent agents provide) to UCLB allinformation, documentation and assistance (including executing documents) which UCLB may reasonably require to enable it tocontinue with the drafting, filing, prosecution and maintenance of the Patents licensed under this Agreement; 10.7Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4, the Licenseeshall, to the extent it is able to do so without being in breach of any obligation owed to a third party, disclose to UCLB full details of any andall Intellectual Property generated at any time by or on behalf of the Licensee as a result of the exercise of the Licensee’s rights under thisAgreement (“Licensee IP”) and, upon UCLB’s written request within [***] ([***]) days following such disclosure, negotiate in good faith toagree the terms of an exclusive or non-exclusive licence to UCLB (as UCLB may request) under the Licensee IP. If the Parties fail to agree theterms of such a licence within [***] days following commencement of such negotiation, despite negotiating in good faith, UCLB’s rights underthis Clause shall lapse. If Licensee may terminate this Agreement under Clause 10.3.1 (for UCLB or its Affiliates uncured material breach), thenLicensee may elect, in lieu of terminating the entire Agreement, to have all licenses granted to the Licensee under this Agreement continue inforce, subject to Licensee’s fulfilment of [***] percent ([***]%) of its payment obligations under Clause 4 after what would have been theeffective date of such termination. 10.8Upon termination of this Agreement for any reason, the provisions of Clauses 1, 2.3, 2.5, 3.1 to 3.5, 4 (in respect of amounts paid and payable toUCLB in respect of the period up to and including the date of termination), 5.7, 7, 9, 10.7, 10.8, 10.8 and 11 of this Agreement shall remain inforce. 11.GENERAL 11.1Force Majeure 11.1.1Any delays in or failure of performance by a Party under this Agreement will not be considered a breach of this Agreement and if and tothe extent that such delay or failure is caused by occurrences beyond the reasonable control of that Party including acts of God; acts,regulations and laws of any government; strikes or other concerted acts of workers; fire; floods; explosions; riots; wars; rebellion; andsabotage; and any time for performance hereunder will be extended by the actual time of delay caused by any such occurrence. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.31Confidential Treatment Requested by MeiraGTx Holdings plc 11.1.2If (a) UCLB or (b) the Licensee or Meira is prevented from carrying out its obligations: (a)under this Agreement for a continuous period of [***] ([***]) months, the Licensee (in the case of (a)) or UCLB (in thecase of (b)), may terminate this Agreement on giving [***] ([***]) days prior written notice provided always that at thedate upon which termination becomes effective the Party which was prevented from carrying out its obligations underthis Agreement remains so prevented. 11.2AmendmentThis Agreement may only be amended in writing signed by duly authorised representatives of the Parties. 11.3Assignment and Third Party Rights 11.3.1Subject to Clause 11.3.3, the Licensee shall not assign, mortgage, charge or otherwise transfer any rights or obligations under thisAgreement, nor any of the Patents, Know-how or Materials, without the prior written consent of UCLB. 11.3.2UCLB may assign all its rights and obligations under this Agreement together with its rights in the Patents, Know-how and Materials toany third party. 11.3.3The Licensee, subject to obtaining the consent of UCLB which shall not be unreasonably withheld or delayed (except in relation tothose categories of persons or Affiliates of persons prohibited by Clause 2.3), may assign all its rights and obligations under thisAgreement together with its rights in the Patents, Know-how and Materials to any third party to which it transfers all or substantially allof its assets or business, provided that the assignee undertakes to UCLB to be bound by and perform the obligations of the assignorunder this Agreement. However, the Licensee shall not have such a right to assign this Agreement if it is insolvent. 11.3.4Meira shall not assign, mortgage, charge or otherwise transfer any rights or obligations under this Agreement without the prior writtenconsent of UCLB. 11.4WaiverAny waiver given under or in relation to this Agreement shall be in writing and signed by or on behalf of the relevant Party. No failure or delayon the part of a Party to exercise any right or remedy under this Agreement shall be construed or operate as a waiver thereof, nor shall any singleor partial exercise of any right or remedy preclude the further exercise of such right or remedy. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.32Confidential Treatment Requested by MeiraGTx Holdings plc 11.5Invalid ClausesIf any provision or part of this Agreement is held to be invalid, amendments to this Agreement may be made by the addition or deletion ofwording as appropriate to remove the invalid part or provision but otherwise retain the provision and the other provisions of this Agreement tothe maximum extent permissible under applicable law. 11.6No AgencyNo Party shall act or describe itself as the agent of the other, nor shall it make or represent that it has authority to make any commitments on theother’s behalf. 11.7InterpretationIn this Agreement: 11.7.1the headings are used for convenience only and shall not affect its interpretation; references to persons shall include incorporated andunincorporated persons; references to the singular include the plural and vice versa; and references to the masculine include thefeminine; 11.7.2references to Clauses and Schedules mean clauses of, and schedules to, this Agreement; 11.7.3references in this Agreement to termination shall include termination by expiry; 11.7.4where the word “including” is used it shall be understood as meaning “including without limitation”; 11.7.5any reference to any English law term for any action, remedy, method or judicial proceeding, legal document, legal status, court, officialor any legal concept or thing shall in respect of any jurisdiction other than England be deemed to include what most nearlyapproximates in that jurisdiction to the English law term; 11.7.6where there is any conflict or inconsistency between the main body of this Agreement and any of the schedules, then the main body ofthe Agreement shall prevail; 11.7.7time shall be of the essence in relation to the performance of Meira’s and the Licensee’s obligations under this Agreement; and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.33Confidential Treatment Requested by MeiraGTx Holdings plc 11.7.8any reference to the sale of a Licensed Product by the Licensee or its Affiliates or Sub-licensees will be taken to include any supply orother disposal of Licensed Products, and the term sold shall be construed accordingly. 11.8Notices. Addresses for Service 11.8.1Any notice to be given under this Agreement shall be in English, in writing and shall be delivered by first class recorded delivery mail(if sent to an inland address) or by international courier (if sent to an address outside of the United Kingdom), to the address of therelevant Party set out at the head of this Agreement, or such other address as that Party may from time to time notify to the other Partiesin accordance with this Clause 11.8. 11.8.2Notices sent as above shall be deemed to have been received [***] ([***]) working day after the day of posting in the case of deliveryinland first class recorded delivery mail, or [***] ([***]) working days after the date of collection by the international courier. 11.9Law and JurisdictionThe validity, construction and performance of this Agreement, and any contractual and non-contractual claims arising hereunder, shall begoverned by English law and shall be subject to the exclusive jurisdiction of the English courts to which the Parties hereby submit, except thata Party may seek an interim injunction (or an equivalent remedy) in any court of competent jurisdiction. 11.10Entire AgreementThis Agreement, including its Schedules, sets out the entire agreement between the Parties relating to its subject matter and supersedes all priororal or written agreements, arrangements or understandings between them relating to such subject matter including, without limitation, the partsof the Original Agreement that pertains to the Specified Technology [***] and the Licence Addendum Number 1. Subject to Clause 9.8.5, theParties acknowledge that they are not relying on any representation, agreement, term or condition which is not set out in this Agreement. Forclarity, this Agreement is not intended to supersede or affect the effectiveness of the parts of the Original Agreement that does not pertain to theSpecified Technology [***] and the Licence Addendum Number 1. Such parts of the Original Agreement that does not pertain to the SpecifiedTechnology [***] and the Licence Addendum Number 1 includes, without limitation, the parts that pertains to the Specified Technology [***]through [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.34Confidential Treatment Requested by MeiraGTx Holdings plc 11.11Third PartiesExcept for the rights of UCL as provided in Clause 2.4, the rights of the Indemnitees as provided in Clause 9.7, the limitations of liabilityafforded to the Indemnitees pursuant to Clause 9.8, who may in their own right enforce and rely on the provisions of those Clauses, thisAgreement does not create any right enforceable by any person who is not a party to it (“Third Party”) under the Contracts (Rights of ThirdParties) Act 1999, but this Clause 11.11 does not affect any right or remedy of a Third Party which exists or is available apart from that Act. 11.12Non-use of Names; Announcements 11.12.1 The Licensee shall not use, and shall ensure that its Affiliates and Sub-licensees do not use, the name, any adaptation of the name, anylogo, trademark or other device of UCLB, nor of the inventors named on the Patents nor the Principal Investigators in any advertising,promotional or sales materials without prior written consent obtained from UCLB in each case, except that the Licensee may state thatit is licensed by UCLB under the Patents. 11.12.2 Except as permitted under Clauses 3.3.1 and 5.7, no Party shall make any press or other public announcement concerning any aspect ofthis Agreement, or make any use of the name or trademarks of any other Party in connection with or in consequence of this Agreement,without the prior written consent of the relevant other Party. 11.13EscalationIf the Licensee or Meira on the one hand, and UCLB on the other, are unable to reach agreement on any issue concerning this Agreement or theProject within [***] days after one either has notified the other of that issue, they will refer the matter to the [***] in the case of UCLB, and tothe [***] in the case of the Licensee and Meira in an attempt to resolve the issue within the time specified elsewhere in this Agreement in thecase of other disputes. Any Party may bring proceedings in a court of competent jurisdiction if the matter has not been resolved within thatprescribed period, and any Party may apply to the court for an injunction, whether or not any issue has been escalated under this Clause 11.13.[Signature Page Follows] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.35Confidential Treatment Requested by MeiraGTx Holdings plc EXECUTED on the date set out at the head of this Agreement. For and on behalf ofMEIRAGTX LIMITED/s/ Zandy ForbesSignedZandy ForbesPrint namePresident and CEOTitleJan 29, 2019Date Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.36For and on behalf ofUCL Business PLC/s/ [***]Signed[***]Print name[***]Title29 January 2019DateFor and on behalf ofMEIRAGTX UK II LIMITED/s/ Zandy ForbesSignedZandy ForbesPrint namePresident and CEOTitleJan 29, 2019DateConfidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 1LICENSED TECHNOLOGYPart A: The Patents[***]Part B: The Know-how[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.37Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 2APPOINTMENT OF EXPERTFor the purposes of this Schedule 2 only, the “Parties” shall mean the Licensee and UCLB. If either Party wishes to appoint an independent expert (the“Expert”) to determine any matter pursuant to any Clause of this Agreement, the following procedures will apply: 1.The Party wishing to appoint the Expert (the “Appointing Party”) will serve a written notice on the other Party (the “Responding Party”). Thewritten notice will specify the Clause pursuant to which the appointment is to be made and will contain reasonable details of the matter(s)which the Appointing Party wishes to refer to the Expert for determination. 2.The Parties shall within [***] ([***]) days following the date of the Appointing Party’s written notice use all reasonable efforts to agree who isto be appointed as the Expert to determine the relevant matter(s). If the Parties are unable to agree upon the identity of the Expert within thattimescale, the Expert shall be appointed by the President (for the time being) of the Licensing Executives Society Britain and Ireland uponwritten request of either Party. 3.Each Party will within [***] ([***]) days following appointment of the Expert, prepare and submit to the Expert and the other Party a detailedwritten statement setting out its position on the matter(s) in question and including any proposals which it may wish to make for settlement orresolution of the relevant matter. 4.Each Party will have [***] ([***]) days following receipt of the other Party’s written statement to respond in writing thereto. Any such responsewill be submitted to the other Party and the Expert. 5.The Expert will if he/ she deems appropriate be entitled to seek clarification from the Parties as to any of the statements or proposals made byeither Party in their written statement or responses. Each Party will on request make available all information in its possession and shall givesuch assistance to the Expert as may be reasonably necessary to permit the Expert to make his/ her determination. 6.The Expert will issue his/ her decision on the matter(s) referred to him/ her in writing as soon as reasonably possible, but at latest within [***]([***]) months following the date of his/ her appointment. The Expert’s decision shall (except in the case of manifest error) be final and bindingon the Parties. 7.The Expert will at all times act as an independent and impartial expert and not as an arbitrator. 8.The Expert’s charges will be borne as he/ she determines in his written decision. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.38Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 3DEFINITION OF TOBACCO INDUSTRY FUNDING (REVISED 2009)FROM THE CANCER RESEARCH UK CODE OF PRACTICE ON TOBACCO INDUSTRY FUNDING TO UNIVERSITIES.http://www.cancerresearchuk.org/science/funding/terms-conditions/funding-policies/policy-tobacco/A tobacco company is defined for the purposes of this policy as one that: • Derives over 5% of revenues from manufacturing tobacco products; • Derives 15%+ of revenues from the manufacture of products necessary for the production of tobacco products; • Derives 15% of revenues from the sale of tobacco products (and has 30 or more staff); • Owns a tobacco company (the company owns 50% or more of a tobacco company); • Is more than 50% owned by a company with tobacco involvement.The following do not constitute tobacco industry funding for the purposes of this Code: • legacies from tobacco industry investments (provided these are sold on immediately) • funding from a trust or foundation no longer having any connection with the tobacco industry even though it may bear a name that (forhistorical reasons) has tobacco industry associations. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.39Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 4DEVELOPMENT PLANThe Development Plan for the Licensed Technology is shown below Activity TimelinePreclin/manufacture [***]Initiate Phase I/II [***]Initiate Phase II/III [***]Complete Phase II/III [***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.40Confidential Treatment Requested by MeiraGTx Holdings plc [***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Exhibit 10.22Confidential Treatment Requested by MeiraGTx Holdings plcLICENCE AGREEMENTbetweenUCL Business PlcandMeiraGTx UK II LimitedandMeiraGTx LimitedDated: 29 January 2019Ref: [***] (CNGA3) Confidential Treatment Requested by MeiraGTx Holdings plc INDEX 1. Definitions 2 2. Grant of Rights 5 3. Know-how and Confidential Information 8 4. Consideration 10 5. Commercialisation 15 6. Access to Medicines and Ethical Licensing 18 7. Compliance with Laws 18 8. Intellectual Property 19 9. Warranties and Liability 22 10. Duration and Termination 26 11. General 31 Schedule 1 Licensed Technology 36 Schedule 2 Appointment of Expert 37 Schedule 3 Definition of Tobacco Industry Funding (Revised 2009) 38 Schedule 4 Initial Development Plan 39 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc THIS AGREEMENT is made 29 January, 2019BETWEEN: (1)UCL BUSINESS PLC, a company incorporated in England and Wales under company registration number 02776963 whose registered office isThe Network Building, 97 Tottenham Court Road, London W1T 4TP (“UCLB”);and (2)MEIRAGTX UK II LIMITED, a company incorporated in England and Wales with registered number 09348737 and having its registeredoffice at 92 Britannia Walk, London, United Kingdom, N1 7NQ (the “Licensee”); and (3)MEIRAGTX LIMITED, a company incorporated in England and Wales with registered number 09501998 and having its registered office at92 Britannia Walk, London, United Kingdom, N1 7NQ (“Meira”).WHEREAS: A.University College London (“UCL”) has developed certain ocular gene technologies and owns certain intellectual property rights relating tothose gene therapies that the Licensee wishes to acquire rights to for the development and commercialisation of Licensed Products. B.UCL has assigned to UCLB all of its right, title and interest in and to such property. C.The Parties previously entered into a certain License Agreement dated as of March 15, 2018 (the “Original Commencement Date”) (the“Original Agreement”). D.The Parties now desire to amend the Original Agreement and enter into a new agreement for the Specified Technology [***] on the terms set outin this Agreement. E.It is the policy of UCLB that its activities in licensing intellectual property take into consideration ethical and socially responsible licensingprinciples, including ensuring that Licensed Products are made available to fulfil unmet needs in developing countries, and the Licenseeacknowledges and agrees to carry out its activities under this Agreement in a manner which complies with ethical and socially responsiblelicensing principles and which is designed to fulfil such needs, all in accordance with the provisions of this Agreement.NOW IT IS AGREED as follows: 1.DEFINITIONS 1.1In this Agreement:Agreement means this agreement (including the Schedules);Affiliate in relation to a Party, means any entity or person that Controls, is Controlled by, or is under common Control with that Party; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Claims means all demands, claims and liability (whether criminal or civil, in contract, tort or otherwise) for losses, damages, costs and expensesof any nature whatsoever and all costs and expenses (including legal costs) incurred in connection therewith;Commencement Date means the date of this Agreement;Competing Product means any [***];Confidential Information means the Know-how, the Materials and all other technical or commercial information that: a)in respect of information provided in documentary form or by way of a model or in other tangible form, at the time of provision ismarked or otherwise designated to show expressly that it is imparted in confidence or which a reasonable person would expect to beconfidential; and b)in respect of information that is imparted orally, any information that the Disclosing Party or its representatives informed the ReceivingParty at the time of disclosure or which a reasonable person would expect to be confidential;Control means direct or indirect beneficial ownership of 50% (or, outside a Party’s home territory, such lesser percentage as is the maximumpermitted level of foreign investment) or more of the share capital, stock or other participating interest carrying the right to vote or todistribution of profits of that Party, as the case may be;Diligent Efforts means, with respect to efforts to be expended by the Licensee with respect to any objective under this Agreement, diligent,reasonable, good faith efforts to accomplish such objective [***] it being understood and agreed that with respect to the research, development,or commercialization of a product, [***], when utilizing sounds and reasonable scientific, medical and business practice and judgment;Disclosing Party has the meaning given in Clause 3.2;Field means ocular gene therapy;First Commercial Sale means the first sale to a third party of a Licensed Product in a given regulatory jurisdiction after all regulatory andmarketing approvals have been obtained for such Licensed Product in such jurisdiction. A sale shall not be deemed to have occurred if aLicensed Product is provided pursuant to an early access or compassionate use;Indemnitees has the meaning given in Clause 9.7; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 3Confidential Treatment Requested by MeiraGTx Holdings plc Intellectual Property means any and all patents, utility models, registered designs, unregistered design rights, copyright, database rights, rightsin respect of confidential information, rights under data exclusivity laws, rights under orphan drug laws, rights under unfair competition laws,property rights in biological or chemical materials, extension of the terms of any such rights (including supplementary protection certificates),applications for and the right to apply any of the foregoing registered property and rights, and similar or analogous rights in any part of theTerritory;Know-how means: a)the inventions claimed in the Patents; and b)the technical information relating to the inventions claimed in the Patents and data described in the Part B of Schedule 1;Licensed Products means any and all products that are developed, manufactured, used, or sold by or on behalf of the Licensee or its Affiliatesor Sub-licensees and which (a) are within (or are manufactured using a process described in) any claim of the Patents; and/or (b) incorporate, ortheir development or manufacture makes use of, any of the Know-how and/or the Materials;Licensed Technology means the Patents, the Know-how and the Materials set out in Schedule 1;Materials means any and all of the materials referred to in Part C of Schedule 1;Net Sales Value means in respect of [***]Original Commencement Date has the meaning given in the Recitals;Original Agreement has the meaning given in the Recitals;Parties means UCLB, the Licensee and Meira, and “Party” shall mean either of them;Patent Costs means [***].Patents means any and all of the patents and patent applications referred to in Part A of Schedule 1;Principal Investigators means [***].Receiving Party has the meaning given in Clause 3.2;Regulatory Exclusivity means, with respect to a Licensed Product, any exclusive rights or protection which are recognised, afforded or grantedby any regulatory authority in any country or region with respect to the Licensed Product other than through patent rights; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 4Confidential Treatment Requested by MeiraGTx Holdings plc Sub-licensee means any third party (other than an Affiliate) to whom the Licensee grants a sub-licence of its rights under this Agreement inaccordance with Clause 2.3;Territory means worldwide;Valid Claim means a claim of a patent or patent application that has not been abandoned or allowed to lapse or expired or been held invalid orunenforceable by a court of competent jurisdiction in a final and non-appealable judgment. 2.GRANT OF RIGHTS 2.1LicenceUCLB hereby grants to the Licensee and its Affiliates, and the Licensee hereby accepts on its own behalf and on behalf of its Affiliates, subjectto the provisions of this Agreement: 2.1.1an exclusive (even as to UCL) licence under the Patents, the Know-how and the Materials, with the right to sub-license, subject toClause 2.3, to develop, commercialise, manufacture, have manufactured, use, sell and have sold Licensed Products only in the Field andin the Territory. 2.2UCLB shall at the Licensee’s request and cost execute such formal licences as may be necessary to enable the Licensee to register the licencesgranted to it under this Agreement with the Patent Offices in the relevant Territory. Such formal licence will reflect the terms of this Agreementwhere possible and for the avoidance of doubt if there is a conflict in the terms of such formal licence and this Agreement, the terms of thisAgreement shall prevail. [***] 2.3Sub-LicensingThe Licensee shall have the right to grant sub-licenses under the license in Clause 2.1 to its Affiliates or other third parties through one or morelevels of Sub-licensees except that the Licensee may not grant such a sub-license to any person or the Affiliates of any person involved in: thetobacco industry (as defined by the Cancer Research UK Code of Practice on Tobacco Industry Funding to Universities detailed in Schedule 3);arms dealing; gambling operations; the promotion of violence; child labour or any other illegal activity. A grant of any sub-licence shall beconditioned on the following: (a)The Licensee shall enter into a written agreement with each Sub-licensee and shall ensure that the provisions of each sub-licence are consistent with the provisions of this Agreement, and the Licensee shall ensure that: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 5Confidential Treatment Requested by MeiraGTx Holdings plc (i)the sub-licence sets out all the proposed terms agreed between the Licensee and the Sub-licensee, including, inparticular, all terms as to remuneration; (ii)the Sub-licensee will maintain complete and accurate records in sufficient detail to permit UCLB to confirm theaccuracy of the calculation of royalty payments under this Agreement; and (iii)the sub-licence imposes obligations of confidentiality on the Sub-licensee which are no less onerous than those set outin Clause 3.2. (b)The Licensee shall procure that each Sub-licensee complies fully at all times with the provisions of its sub-licence. (c)The Licensee shall be liable for all acts and omissions of its Sub-licensees that, if committed by the Licensee, would constitutea breach of any of the provisions of this Agreement. (d)The Licensee shall provide UCLB with a copy of any sub-licence [***] ([***]) days after execution of such sub-licence,provided that the Licensee may redact confidential or proprietary terms from such copy, including financial terms. (e)Each sub-licence shall terminate automatically upon termination of this Agreement for any reason (but not expiry of thisAgreement under Clause 10.1), except where the Sub-licensee was not implicated in or at fault in any circumstances which ledto the termination of this Agreement, UCLB shall on receiving a written request from the relevant Sub-licensee within [***]([***]) days following the date of termination of this Agreement enter into a licence agreement with the Sub-licensee for theLicensed Technology on terms substantially the same as the terms set out in this Agreement (except that the Sub-licensee shallnot be obliged to pay to UCLB any sums equivalent to those sums set out in Clauses 4.1 or 4.3 which have already been paidto UCLB by the Licensee prior to the date of termination). 2.4Reservation of Rights 2.4.1UCLB reserves for itself and UCL the non-exclusive, irrevocable, worldwide, royalty-free right to: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 6Confidential Treatment Requested by MeiraGTx Holdings plc (a)Use the Licensed Technology in the Field solely for academic (non-commercially funded) research, publication and teaching;and (b)Grant licenses to academic third parties to use the Licensed Technology in academic research collaborations with UCL andsuch licenses shall not be sublicensable; and (c)Grant license of the Licensed Technology to post graduate student of UCL for the purpose of conducting a programme of postgraduate academic research and such licenses shall not by sublicensable.In exercising the rights described in Clause 2.4.1(b) and (c), UCL and UCLB shall comply with the provisions of Clause 3 as regardsconfidentiality of the Know-how. 2.4.2UCL and UCLB will refer a request from a third party for a licence to use the Patents in clinical trials or for diagnostic purposesinvolving human subjects to the Licensee, and the Licensee shall liaise directly with such third party. 2.4.3Except for the licences expressly granted by this Clause 2, UCLB grants no rights to the Licensee under this Agreement to or under anyintellectual property other than the Patents, the Know-how and the Materials and hereby reserves all rights under the Patents, theKnow-how and the Materials outside the Field. 2.4.4Nothing in this Agreement shall limit or otherwise affect UCL’s ability to apply for noncommercial grant funding or comply with suchgrant terms and conditions. In the event that any terms of this Agreement conflicts with the terms of any non-commercial grant funding,the Parties shall negotiate in good faith to amend the terms of this Agreement to allow UCL to access such funding provided thatnothing herein shall require the Licensee to agree to alter or modify the scope of the licence granted to it in this Clause 2. 2.5AffiliatesThe Licensee shall: 2.5.1ensure that its Affiliates comply fully with the terms of this Agreement; 2.5.2be responsible for any breach of or non-compliance with this Agreement by its Affiliates as if the breach or non-compliance had been abreach or non-compliance by the Licensee; 2.5.3indemnify in accordance with Clause 9.7 each of the Indemnitees against any Claims which are awarded against or suffered by any ofthe Indemnitees as a result of any breach of or non-compliance with this Agreement by its Affiliates; and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 7Confidential Treatment Requested by MeiraGTx Holdings plc 2.5.4ensure that if any Affiliate ceases to be an Affiliate as a result of a change of Control or otherwise, that unless a sub-licence agreement inaccordance with Clause 2.3 is entered into with such an Affiliate, such former Affiliate immediately upon such cessation: (a)ceases developing, manufacturing, having manufactured, using, selling and/ or having sold Licensed Products and ceases alluse or exploitation of the Licensed Technology, for as long as any of the relevant Patents remains in force and/or theKnow-how remains confidential; (b)returns to the Licensee or destroys any documents or other materials in the former Affiliate’s possession or under its control andthat contain Confidential Information provided under this Agreement relating to the Licensed Technology and/ or LicensedProducts; (c)to the extent possible, takes all action necessary to have any product licences, marketing authorisations, pricing and/ orreimbursement approvals (and any applications for any of the foregoing) which relate to Licensed Products transferred into thename of the Licensee. 2.6Use of Licensed Technology in CombinationUCLB acknowledges and agrees that the Licensee shall be entitled to use the Licensed Technology in combination with other technology,patents, know-how and materials licensed by UCLB to the Licensee under separate licence agreements and with any improvements to theLicensed Technology developed or generated by the Licensee. 3.KNOW-HOW AND CONFIDENTIAL INFORMATION 3.1Confidentiality of Know-how and MaterialsThe Licensee undertakes that for so long as the Know-how and/or the Materials remains confidential, it shall (and shall ensure that its Affiliatesand Sub-licensees) take all reasonable precautions to prevent unauthorised access to the Know-how and the Materials and protect theKnow-how and the Materials in the same manner as it (or they) protect(s) its (or their) own proprietary information, and shall not (and shallensure that its Affiliates and Sub-licensees do not) use the Know-how or the Materials for any purpose, except as expressly licensed hereby andin accordance with the provisions of this Agreement. For the avoidance of doubt, to the extent that any Materials, Know-how or informationrelating to the Patents falls within the public domain (without any breach of this Agreement or any other obligation of confidentiality), thenUCL, the Principal Investigators and UCLB shall be free to use such information without restriction in the same way that any third party wouldhave the freedom to use it. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 8Confidential Treatment Requested by MeiraGTx Holdings plc 3.2Confidentiality ObligationsEach Party (“Receiving Party”) undertakes: 3.2.1to maintain as secret and confidential all Confidential Information obtained from, in the case of UCLB, the Licensee or Meira asapplicable, and in the case of the Licensee and Meira, UCLB (“Disclosing Party”) in the course of or in anticipation of this Agreementand to respect the Disclosing Party’s rights therein; 3.2.2to use such Confidential Information only for the purposes of or as permitted by this Agreement; and 3.2.3subject to Clause 3.3, to disclose such Confidential Information only to those of its employees, contractors, Affiliates, and Sub-licensees(if any) to whom and to the extent that such disclosure is reasonably necessary for the purposes of this Agreement. 3.3Permitted Disclosure 3.3.1The Licensee shall have the right to disclose Confidential Information received from UCLB to: (a)potential or actual customers of Licensed Products to the extent reasonably necessary to promote the sale or use of LicensedProducts and provided that the customer has agreed to confidentiality provisions at least as restrictive as set forth herein; (b)to existing or potential Sub-licensees, collaborators, investors or lenders provided that such third parties have agreed toconfidentiality provisions at least as restrictive as set forth herein; and (c)to its Board of Directors (or similar governing body) and its counsel, accountants and other professional advisers. 3.4Exceptions to ObligationsThe provisions of Clause 3.2 shall not apply to Confidential Information which the Receiving Party can demonstrate by reasonable writtenevidence: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 9Confidential Treatment Requested by MeiraGTx Holdings plc 3.4.1was, prior to the Original Commencement Date, in the possession of the Receiving Party and at its free disposal and was not obtained orotherwise acquired directly or indirectly from the Disclosing Party or its Affiliates or their respective employees, students orrepresentatives; or 3.4.2is subsequently disclosed to the Receiving Party without any obligations of confidence by a third party; or 3.4.3is or becomes generally available to the public through no act or default of the Receiving Party or its agents, employees, Affiliates orSub-licensees; or 3.4.4the Receiving Party is required to disclose by or to the courts of any competent jurisdiction, or to any government regulatory agency orfinancial authority, provided that the Receiving Party shall: (a)inform the Disclosing Party as soon as is reasonably practicable; (b)at the Disclosing Party’s request and cost seek to persuade the court, agency or authority to have the information treated in aconfidential manner, where this is possible under the court, agency or authority’s procedures; and (c)where the disclosure is unavoidable, limit the disclosure of Confidential information to the minimum extent required by law; or 3.4.5which a Party is advised by its information officer that it is required to disclose under the Freedom of Information Act 2000 or theEnvironmental Information Regulations 2004. 3.5Disclosure to EmployeesThe Receiving Party shall procure that all of its employees, contractors, Affiliates and Sub-licensees who have access to any of the DisclosingParty’s Confidential Information to which Clause 3.2 applies, shall be made aware of the obligations of confidence and are bound byobligations of confidentiality at least as restrictive as those set forth herein (which it undertakes to enforce and for which it is legallyresponsible) and the Receiving Party shall only disclose the Disclosing Party’s Confidential Information to those of its subsidiaries, employees,and officers as need to have access thereto wholly necessarily and exclusively for the purposes of this Agreement. 4.CONSIDERATION 4.1Milestone PaymentsWithin [***] ([***]) days following achievement of each of the following milestone events by Licensee, its Affiliates or Sub-licensees, theLicensee shall notify UCLB in writing that the relevant milestone event has been achieved, provide documentary evidence of suchachievement as appropriate and pay to UCLB, within a period of [***] ([***]) days, the amount(s) set out next to such milestone event below: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 10Confidential Treatment Requested by MeiraGTx Holdings plc Milestone Event Amountto bepaid[***] £[***][***] £[***] 4.2Annual Management FeesOn each date referred to in the following table, the Licensee shall pay to UCLB the annual management fee set out next to such date in thetable. Date Amountto bepaidUpon each anniversary of the Original Commencement Date until [***] £50,000 4.3Sales Linked Milestone PaymentsUpon the first achievement of each of the sales linked milestones set out in the following table by the Licensee, its Affiliates or Sub-licensees,the Licensee shall notify UCLB in writing that the relevant sales linked milestone has been achieved, provide the relevant documentaryevidence and pay to UCLB the amount(s) set out next to such event in the table: Sales Linked Milestones Amountto bepaidWhen Net Sales Value reaches £[***] £[***]On the next £[***] of Net Sales Value (when sales cumulatively reach £[***]) £[***]On the next £[***] of Net Sales Value (When sales cumulatively reach £[***]) £[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 11Confidential Treatment Requested by MeiraGTx Holdings plc On the next £[***] of Net Sales Value (when sales cumulatively reach£[***]) £[***]On the next £[***] of Net Sales Value (when sales cumulatively reach£[***]) £[***]On the next £[***] of Net Sales Value (when sales cumulatively reach£[***]) £[***] 4.4Royalties on Net SalesFor each Licensed Product in each country, the Licensee shall pay to UCLB a royalty of [***]% ([***] per cent) being a percentage of the NetSales Value of such Licensed Product sold by Licensee, its Affiliates or Sub-licensees. The Licensee’s obligations to pay such royalty for agiven Licensed Product in a given country shall begin after the First Commercial Sale of such Licensed Product in such country and shall endon the later to occur of the following: (a) expiration of the last Valid Claim of a Patent claiming such Licensed Product in such country; or(b) the tenth (10th) anniversary of the date of such First Commercial Sale in such country; or (c) the expiration of any Regulatory Exclusivitywith respect to such Licensed Products in the relevant country. 4.5Combination ProductsIf any Licensed Products are incorporated in any other product (“Combination Product”) sold by the Licensee or its Affiliates and the LicensedProduct is not priced separately from the Combination Product, the Net Sales Value of such Licensed Product shall be deemed to be the fairmarket value of the Licensed Product in the country of sale when sold separately or if not sold separately in the country of sale, in comparablecountries and territories or if neither of the foregoing apply, a reasonable amount which fairly reflects the value of the Licensed Product withinthe Combination Product assuming the Licensed Product is not being sold as a loss leader. 4.6Payment FrequencyRoyalties due under this Agreement, except for the payments due under Clauses, 4.1, 4.2 and 4.3, which are payable upon the date/timespecified in Clauses 4.1, 4.2 and 4.3 as appropriate, shall be paid within [***] ([***]) days following the end of each calendar quarter ending on31 March, 30 June, 30 September and 31 December in each year, in respect of sales of Licensed Products made during such quarter, and within[***] ([***]) days following the termination of this Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 12Confidential Treatment Requested by MeiraGTx Holdings plc 4.7Payment termsAll sums due under this Agreement: 4.7.1are exclusive of Value Added Tax which where applicable will be paid by the Licensee to UCLB in addition; 4.7.2shall be paid in pounds sterling in cash by transferring an amount in aggregate to the following Account name: UCL Business Plc, SortCode: 20 10 53, Account number: 30782270, Address: Barclays Bank Plc, PO Box 11345, London, W12 8GG, and in the case of incomeor amounts received by the Licensee or its Affiliates in a currency other than pounds sterling, the royalty shall be calculated in the othercurrency and then converted into equivalent pounds sterling at the relevant daily spot rate for that currency as quoted in the FinancialTimes newspaper on the last business day of the quarter in relation to which the royalties are payable; 4.7.3will be made without any set-off, deduction or withholding except as may be required by law. If the Licensee is required by law to makeany deduction or to withhold any part of any amount due to UCLB under this Agreement, the Licensee will give to UCLB properevidence of the amount deducted or withheld and payment of that amount to the relevant taxation authority, and will do all things in itspower to enable or assist UCLB to claim exemption from or, if that is not possible, to obtain a credit for the amount deducted orwithheld under any applicable double taxation or similar agreement from time to time in force; and 4.7.4shall be made by the due date, failing which UCLB may charge interest on any outstanding amount on a daily basis at a rate equivalentto [***]% above the Bank of England pound sterling base rate then in force in London. 4.8Royalty StatementsThe Licensee shall send to UCLB, at the same time as each royalty payment is made in accordance with Clause 4.4, a statement setting out forthe relevant calendar quarter: 4.8.1in respect of each territory or region in which Licensed Products are sold; 4.8.2the types of Licensed Product sold; 4.8.3the quantity of each type sold; 4.8.4the total invoiced price for each type of Licensed Product sold; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 13Confidential Treatment Requested by MeiraGTx Holdings plc 4.8.5where relevant, details of any Licensed Products that have been sold other than on arm’s length terms for a cash consideration, includingthe relevant open market price or (if not available) the reasonable price attributed thereto; 4.8.6the amounts deducted from the Net Sales Value as referred to in paragraph (i) to (iv) of that definition (broken down on a product byproduct and category by category basis); and 4.8.7the aggregate royalties on Net Sales Value due to UCLB;in each case expressed both in local currency and pounds sterling and showing the conversion rates used, during the period to which the royaltypayment relates. 4.9RecordsThe Licensee shall keep at its normal place of business detailed and up to date records and accounts showing the quantity, description andinvoiced price or non-cash consideration for all Licensed Products sold by it or its Affiliates or on its or its Affiliates’ behalf, broken down ineach case on a country by country basis, and being sufficient to ascertain the payments due to UCLB under this Agreement.The Licensee shall make such records and accounts available, on reasonable notice, for inspection during business hours by an independentchartered accountant nominated by UCLB for the purpose of verifying the accuracy of any statement or report given by the Licensee to UCLBunder this Clause 4.9. The Licensee shall co-operate reasonably with any such accountant, and shall promptly provide all information andassistance reasonably requested by such accountant. The accountant shall be required to keep confidential all information learnt during anysuch inspection, and to disclose to UCLB only such details as may be necessary to report on the accuracy of the Licensee’s statement or report.UCLB shall be responsible for the accountant’s charges unless the accountant certifies that there is an inaccuracy of more than [***]% ([***]percent) in any royalty statement, in which case the Licensee shall pay his charges in respect of that inspection.The Licensee shall ensure that UCLB has the same rights as those set out in this Clause 4.9 in respect of the Licensee’s Affiliates andSub-licensees.The Licensee shall co-operate with UCLB in good faith to resolve any discrepancies identified during any such inspection and [***], togetherwith interest on late payment as specified in Clause 4.7.4, within [***] following receipt of a copy of the independent chartered accountant’sreport. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 14Confidential Treatment Requested by MeiraGTx Holdings plc 4.10Accounting StandardsWhere this Agreement requires a financial calculation to be made or an action to be taken by a party, such calculation will be made or taken inaccordance with the generally accepted accounting principles followed by such party. 5.COMMERCIALISATION 5.1General DiligenceThe Licensee shall use Diligent Efforts to develop and commercially exploit Licensed Products throughout the Territory (including obtainingall and any regulatory approvals which may be required to market and sell the Licensed Products) for the benefit of both Parties. 5.2Competing ActivitiesThe Licensee shall notify UCLB in confidence if it or any of its Affiliates or its Sub-licensees commences any marketing, sale orcommercialisation of any Competing Product or enters into an agreement with any other person with respect to any such activities. 5.3Development PlanThe Licensee’s initial plan for developing and commercialising Licensed Products is set out in Schedule 4 (the “Initial Development Plan”).The Licensee shall provide to UCLB on each anniversary of the Original Commencement Date a written update to the Initial Development Planthat shall: 5.3.1report on all activities conducted under this Agreement by the Licensee and its Affiliates and Sub-licensees since the OriginalCommencement Date or the date of the previous update (as appropriate); 5.3.2(where applicable) set out the milestone events achieved since the Original Commencement Date or the date of the previous update (asappropriate) and the Licensee’s reasonable estimate of the dates for achieving any future milestone events; 5.3.3set out the current and projected activities being taken or planned to be taken by the Licensee and its Affiliates and Sub-licensees tobring Licensed Products to market in the Territory; and 5.3.4set out the projected sales of Licensed Products (based on the Licensee’s or Sub-licensee’s current forecasts) for each of the next [***]([***]) years following the date of the report. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 15Confidential Treatment Requested by MeiraGTx Holdings plc UCLB’s receipt or approval of any update to the Updated Development Plan shall not be taken to waive or qualify the Licensee’s obligationsunder Clause 5.1. 5.4Annual MeetingIn respect of the Licensed Technology, the Licensee will on UCLB’s request meet with UCLB at least once per calendar year, following thesubmission of the update to the relevant Development Plan pursuant to Clause 5.3, to discuss progress with development andcommercialisation of the Licensed Technology. 5.5Development MilestonesIn addition to the Licensee’s obligations under Clause 5.1, the Licensee shall for each Licensed Technology use Diligent Efforts to achieve thedevelopment milestone events specified in Schedule 4 by the dates set out therein. In any instance in which it becomes apparent that aparticular development milestone will not be met due to regulatory, technical, safety or efficacy-related reasons or Force Majeure event(pursuant to Clause 11.1), the Parties in good faith will agree upon an appropriate adjust of such milestone and any subsequent milestones. 5.6Reporting of First Commercial SaleThe Licensee will promptly notify UCLB in writing of the First Commercial Sale of each Licensed Product on a commercial basis in eachcountry within the Territory. 5.7Reporting for Impact Purposes 5.7.1The Licensee acknowledges that part of UCLB’s purpose in licensing the Patents, Know-how and the Materials to the Licensee pursuantthis Agreement is to ensure that the Patents, Know-how and the Materials are made available for use and commercial exploitation withthe intention of benefitting society and the economy. In order to enable UCLB and UCL to monitor the benefit that they are providing,and to enable UCL to demonstrate the impact of its research activities, to society and the economy, the Licensee will upon requestprovide to UCLB [***], a written report describing in reasonable detail how it has used the Patents, Know-how and the Materials and thesocietal and economic benefits generated therefrom. 5.7.2UCLB shall notify and seek permission from the Licensee in advance, in writing if it wishes to use any written reports received from theLicensee (and the information contained therein) pursuant to Clause 5.7.1 in applications for research or other grant related funding andin submissions to Higher Education funding bodies such as HEFCE and/ or HEIF (or any replacements for either of those entities) andlike entities, supplying a written copy of the application for research or other grant related funding Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 16Confidential Treatment Requested by MeiraGTx Holdings plc or submission (or the relevant sections thereof). The Licensee will respond to UCLB in writing within [***] ([***]) days of receipt ofsuch written information and subject to the removal of any confidential information as notified in such written request by the Licensee,UCLB and UCL shall be entitled to submit the approved applications for research or other grant related funding and in submissions toHigher Education funding bodies such as HEFCE and/ or HEIF (or any replacements for either of those entities) and like entities. 5.8QualityThe Licensee shall ensure that all of the Licensed Products marketed by it and its Affiliates and Sub-licensees are of satisfactory quality andcomply with all applicable laws and regulations in each part of the Territory. 5.9Marking of Licensed ProductsTo the extent permitted under the laws of any country, the Licensee shall mark and cause its Affiliates and Sub-Licensees to mark each LicensedProduct with the number of each issued Patent which applies to the Licensed Product and a statement that such Licensed Products are soldunder licence from UCL Business plc. 5.10Disposals of Licensed Products for FreeNotwithstanding the terms of Clause 5.1, the Licensee shall be entitled to supply a reasonable number of Licensed Products to third parties freeof charge as promotional items for the purpose of establishing a market for the Licensed Products in the relevant country or territory or forresearch, evaluation and testing purposes, or for clinical development, provided that the quantity of Licensed Products supplied for free (or forthe cost of manufacture) in each country or territory is not excessive and is in line with normal industry practice in such country or territory.Any Licensed Products disposed of to third parties in accordance with this Clause 5.10 shall not be taken into account for the purposes ofcalculating Net Sales Value. 5.11Referral to ExpertIf UCLB considers at any time during the period of this Agreement that the Licensee has failed to comply with its obligations under Clause 5.1or 5.3, then the matter shall be referred to an independent expert to answer the following questions: 5.11.1whether the Licensee has complied with its obligations under Clause 5.1 or 5.3; and if not Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 17Confidential Treatment Requested by MeiraGTx Holdings plc 5.11.2what specific action the Licensee should have taken and/or now needs to take (“Specific Action”) in order to fulfil such obligations andwithin what period the Specific Action should be taken (“Action Period”).The independent expert shall be appointed in accordance with the provisions of Schedule 2 and his decision shall be final and binding on theParties. 5.12Consequences of Expert’s DecisionIf the expert determines that the Licensee has failed to comply with its obligations under Clause 5.1 or 5.3, and if the Licensee fails to take theSpecific Action within the Action Period, UCLB shall be entitled, by giving, at any time within [***] ([***]) months after the end of thatAction Period, not less than [***] ([***]) months’ notice, to (a) convert the licence granted under Clause 2.1 into a non-exclusive licence or(b) terminate this Agreement. 6.ACCESS TO MEDICINES AND ETHICAL LICENSINGLicensee shall use Diligent Efforts to carry out its activities under this Agreement in a manner which complies with ethical and sociallyresponsible licensing principles and which is designed to fulfil unmet needs in developing countries. 7.COMPLIANCE WITH LAWS 7.1General Compliance with LawsThe Licensee will at all times (and will ensure its Affiliates and Sub-licensees) comply with all legislation, rules, regulations and statutoryrequirements applying to and obtain any consents necessary for its use of the Patents, the Know-how and the Materials, the development,manufacture, and sale of Licensed Products in any country or territory. 7.2Bribery ActThe Licensee shall (and shall procure that any persons associated with it engaged in the performance of this Agreement including its Affiliatesand Sub-licensees shall): 7.2.1comply with all applicable laws and codes of practice relating to anti-bribery and anti-corruption including the Bribery Act 2010 andwithout prejudice to the foregoing generality, shall not engage in any activity, practice or conduct which would constitute an offenceunder sections 1, 2 or 6 of the Bribery Act 2010 or do or omit to do any act that will cause or lead UCLB to be in breach of the BriberyAct 2010; 7.2.2comply with UCLB’s ethics, anti-bribery and anti-corruption policies as notified to the Licensee from time to time and have, maintain inplace and enforce throughout the term of this Agreement adequate procedures to ensure compliance with Clause 7.2.1; and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 18Confidential Treatment Requested by MeiraGTx Holdings plc 7.2.3promptly report to UCLB any request or demand for any undue financial or other advantage of any kind received in connection with theperformance of this Agreement.For the purpose of this Clause 7.2, the meaning of adequate procedures and whether a person is associated with another person shall bedetermined in accordance with the Bribery Act 2010 (and any guidance issued under section 9 of that Act). Breach of this Clause 7.2 shall bedeemed a material breach of this Agreement entitling UCLB to terminate under Clause 10.3.1. 7.3Export Control RegulationsThe Licensee shall ensure that, in using the Patents, Know-how or Materials and in selling Licensed Products, it and its Affiliates, employees,sub-contractors and Sub-licensees comply fully with any United Nations trade sanctions or EU or UK legislation or regulation, from time totime in force, which impose arms embargoes or control the export of goods, technology or software, including weapons of mass destruction andarms, military, paramilitary and security equipment and dual-use items (items designed for civil use but which can be used for militarypurposes) and certain drugs and chemicals. 8.INTELLECTUAL PROPERTY 8.1Obtain and Maintain the Patents 8.1.1The Licensee shall be responsible for the drafting, filing, prosecution and maintenance of all of the Patents at the Licensee’s cost andexpense. Subject to resource availability, UCLB shall use commercially reasonable efforts to provide such assistance as the Licenseemay request to prosecute and maintain the Patents[***]. 8.1.2The Patents will be filed, prosecuted and maintained in the countries and territories where Licensee normally files its patent applicationsand patents for other gene therapy products. The Licensee shall notify UCLB of any decisions as to which (if any) additional countriesto file and maintain Patents in. 8.1.3The Licensee shall consult with UCLB in relation to all material changes to the patent claims or specifications that would have theeffect of reducing or limiting the scope of the Patents, and not make any such changes without the prior written consent of UCLB. Suchconsent shall not be unreasonably withheld or delayed provided that UCLB has been given as much notice as is practicable, and in anyevent no less than [***] days’ notice (or such shorter period for response dictated by the relevant patent office) of such proposedchanges, and has been given an opportunity to file Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 19Confidential Treatment Requested by MeiraGTx Holdings plc divisionals, continuations and/or such other types of protection to cover any claims or subject matter that the Licensee intends toremove from the scope of the Patents. If UCLB fails to respond before the end of the [***] day period (or such shorter period for responsedictated by the relevant patent office), the Licensee may proceed with the proposed changes to the patent claims or specifications. TheLicensee will ensure that UCLB receives copies of all correspondence to and from Patent Offices in respect of the Patents, includingcopies of all documents generated in or with such correspondence, and shall be given reasonable notice (or such shorter period forresponse dictated by the relevant patent office) of and the opportunity to participate in any conference calls or meetings with theLicensee’s patent attorneys in relation to the drafting, filing, prosecution and maintenance of the Patents, so that UCLB may becontinuously informed of progress with the drafting, filing, prosecution and maintenance of the Patents. Such involvement of UCLBunder this Clause 8.1.3 shall be at UCLB’s cost and expense. 8.1.4If the Licensee wishes to abandon any application contained with the Patents or not to maintain any such Patent, it shall give [***]([***]) months’ prior written notice to UCLB and on the expiry of such notice period the licences of the relevant Patents granted to theLicensee under this Agreement shall cease. 8.1.5In the event that any of rights granted hereunder become non-exclusive, responsibility for the drafting, filing, prosecution andmaintenance of all of the Patents shall revert to UCLB. 8.2Infringement of the Patents, the Know-how and/or the Materials 8.2.1The Licensee and UCLB shall promptly give to each other written notice if it becomes aware of any infringement or potentialinfringement of any of the Patents or any unauthorised use of the Know-how or the Materials or any challenge to the validity orownership of the Patents, the Know-how or the Materials and the Licensee and UCLB shall consult with each other to decide the bestway to respond to such infringement, unauthorised use or challenge. 8.2.2The Licensee shall have the primary obligation and right to take action against any third party alleged to be infringing the Patents ormaking unauthorised use of the Know-how or the Materials and to defend the Patents against challenges to validity or ownership at itssole expense, provided that: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 20Confidential Treatment Requested by MeiraGTx Holdings plc (a)the Licensee and UCLB shall use their commercially reasonable efforts to eliminate the infringement without litigation. If theefforts of the Licensee, and UCLB are not successful in eliminating the infringement within [***] ([***]) days after theinfringer has been formally notified of the infringement by the Licensee, the Licensee shall have the right after consulting withUCLB, to commence suit on its own account; (b)UCLB shall on the Licensee’s request cooperate with the Licensee in such action [***]; (c)the Licensee shall be solely responsible for the conduct of the action or for settlement thereof and shall be entitled to alldamages received from such action, subject to Clause 8.2.4; and (d)if the Licensee is unsuccessful in persuading the alleged infringer to desist within [***] ([***]) months of the Licensee firstbecoming aware of any potential infringement of the Patents for any Licensed Technology or fails without a commerciallyreasonable basis (relative to the Licensed Technology or other technology licenced to Licensee by UCLB) to initiate aninfringement action within [***] ([***]) months of becoming aware of such infringement, UCLB shall have the right, at its solediscretion, to prosecute such infringement under its sole control [***]. 8.2.3Before starting or defending or settling any legal action under Clause 8.2.2, the Licensee shall consult with UCLB as to the advisabilityof the action or defence or settlement, its effect on the good name of UCLB, the public interest, and how the action or defence should beconducted. 8.2.4The Licensee shall [***] in such action or defence. 8.2.5UCLB shall if reasonably requested by the Licensee agree to be joined in any suit to enforce such rights or will take such action in itsown name [***] and shall have the right to be separately represented by its own counsel [***] Notwithstanding the foregoing, [***]. 8.3Infringement of Third Party Rights 8.3.1If any warning letter or other notice of infringement is received by the Licensee or UCLB, or legal suit or other action is brought againstthe Licensee or UCLB, alleging infringement of third party rights in the manufacture, use or sale of any Licensed Product or use of anyPatents, Know-how or Materials, that Party shall (in the case of UCLB) promptly provide full details to the Licensee and (in the case ofthe Licensee) promptly provide full details to UCLB, and the Licensee and UCLB shall discuss the best way to respond. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 21Confidential Treatment Requested by MeiraGTx Holdings plc 8.3.2The Licensee shall have the right but not the obligation to defend such suit to the extent it relates to Licensee’s or its Affiliates’ orSub-licensee’s activities and shall have the right to settle with such third party, provided that [***]. In the event that the Licensee,Affiliates or Sub-licensees do not take forward an action, UCLB shall have the right, at its sole discretion, to defend such suit under itssole control and [***]. 9.WARRANTIES AND LIABILITY 9.1Warranties by UCLBUCLB warrants as of the Original Commencement Date and undertakes as follows to its reasonable knowledge and without having undertakenany due and careful enquires whether specific or general in nature: 9.1.1It is the owner of the Patents; 9.1.2it has the authority to grant the licences under this Agreement; and. 9.1.3so far as it is aware (having made no enquiry of any third parties or conducted any freedom to operate searches), use and exploitation ofthe Patents will not infringe the intellectual property rights of any third party.UCLB warrants and undertakes: 9.1.4it has full power and authority to enter into and perform this Agreement which, when executed, will constitute valid and legally bindingobligations on UCLB; and 9.2Warranties by the LicenseeThe Licensee warrants as of the Original Commencement Date and undertakes that in respect of the Licensed Technology that: 9.2.1full power and authority to enter into and perform this Agreement, which, when executed, will constitute valid and legally bindingobligations on the Licensee; 9.2.2entry into this Agreement will not result in any breach of, or violation of the terms or provisions of, the constitutional documents of theLicensee or any other agreement or instrument by which it is bound; 9.2.3so far as it is aware (having made no enquiry of any third parties), use and exploitation of the Patents will not infringe the intellectualproperty rights of any third party; 9.2.4neither it nor any of its Affiliates is currently researching, developing, marketing, selling or otherwise commercialising any CompetingProduct (“Competing Activities”), nor has any of them entered into an agreement with any other person with respect to any CompetingActivities; and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 22Confidential Treatment Requested by MeiraGTx Holdings plc 9.2.5it shall notify UCLB if it or any of its Affiliates or its Sub-licensees commences any Competing Activities or enters into an agreementwith any other person with respect to any Competing Activities. 9.3Warranties by MeiraLicensee warrants as of the Original Commencement Date and undertakes that full power and authority to enter into and perform this Agreementwhich, when executed, will constitute valid and legally binding obligations on Licensee. 9.4AcknowledgementsThe Licensee acknowledges that: 9.4.1the inventions claimed in the Patents, and the Know-how and the Materials are at an early stage of development. Accordingly, specificresults cannot be guaranteed and any results, materials, information or other items (together “Delivered Items”) provided under thisAgreement are provided “as is” and without any express or implied warranties, representations or undertakings. As examples, butwithout limiting the foregoing, UCLB does not give any warranty that Delivered Items are of merchantable or satisfactory quality, are fitfor any particular purpose, comply with any sample or description, or are viable, uncontaminated, safe or non-toxic. 9.4.2UCLB has not performed any searches or investigations into the existence of any third party rights that may affect any of the Patents,Know-how or Materials or the use and exploitation of any of the Patents, Know-how or Materials. 9.5No Other Warranties 9.5.1Each of the Parties acknowledges that, in entering into this Agreement, it does not do so in reliance on any representation, warranty orother provision except as expressly provided in this Agreement, and any conditions, warranties or other terms implied by statute orcommon law are excluded from this Agreement to the fullest extent permitted by law. 9.5.2Without limiting the scope of Clause 9.5.1, UCLB does not make any representation nor give any warranty or undertaking: (a)express or implied, including, without limitation, any implied warranties of merchantability or of fitness for a particularpurpose with respect to any Patent, trademark, software, non-public or other information, or tangible research property, licensedor otherwise provided to the Licensee hereunder and hereby disclaims the same; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 23Confidential Treatment Requested by MeiraGTx Holdings plc (b)as to the efficacy or usefulness of the Patents, Know-how or Materials; or (c)whatsoever with regard to the scope of any of the Patents or that any of the Patents is or will be valid or (in the case of anapplication) will proceed to grantor that such Patents may be exploited by the Licensee, Affiliate or Sub-licensee withoutinfringing other patents; or (d)that the Materials or the method used in making or using the Materials are free from liability for patent infringement; or (e)that the use of any of the Patents, Know-how or Materials, Licensed Technology, the manufacture, sale or use of the LicensedProducts, or the exercise of any of the rights granted under this Agreement will not infringe any intellectual property or otherrights of any other person; or (f)that the Know-how or any other information communicated by UCLB to the Licensee under or in connection with thisAgreement will produce Licensed Products of satisfactory quality or fit for the purpose for which the Licensee intended or thatany product will not have any defect, latent or otherwise, and whether or not discoverable by inspection; or (g)as imposing any obligation on UCLB to bring or prosecute actions or proceedings against third parties for infringement or todefend any action or proceedings for revocation of any of the Patents; or (h)as imposing any liability on UCLB in the event that any third party supplies Licensed Products to customers located in theTerritory; or (i)that there will be no similar or competitive products or services manufactured, used, sold or supplied by any third party in theTerritory. 9.6Responsibility for Development of Licensed ProductsThe Licensee shall be exclusively responsible for its and its Affiliates’ and Sub-licensees’ use of the Patents, Know-how and Materials, thetechnical and commercial development and manufacture of Licensed Products and for incorporating any modifications or developments theretothat may be necessary or desirable, for all Licensed Products sold or supplied, notwithstanding any consultancy services or other contributionsthat UCLB and/or UCL may provide in connection with such activities. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 24Confidential Treatment Requested by MeiraGTx Holdings plc 9.7IndemnityThe Licensee shall indemnify each of UCLB and UCL, and each of their respective officers, directors, Council members, employees andrepresentatives (together, the “Indemnitees”) against all third party Claims that may be asserted against or suffered by any of the Indemniteesand which relate to: 9.7.1the use by the Licensee or any of its Affiliates or Sub-licensees of any of the Patents, Know-how or Materials; or 9.7.2use of the Licensed Technology by or on behalf of the Licensee or any of its Affiliates or Sub-licensees; or 9.7.3the development, manufacture, use, marketing or sale of, or any other dealing in, any of the Licensed Products, by or on behalf of theLicensee or any of its Affiliates or Sub-licensees, or subsequently by any customer or any other person, including claims based onproduct liability laws.The indemnity given by the Licensee to each Indemnitee under this Clause 9.7 will not apply to any third party Claim to the extent that it isattributable to the negligence, gross negligence, reckless misconduct or intentional misconduct of any Indemnitee. 9.8Limitations of Liability 9.8.1To the extent that UCLB or any of its Affiliates has any liability in contract, tort, or otherwise under or in connection with thisAgreement, including any liability for breach of warranty, their liability shall be limited in accordance with the following provisions ofthis Clause 9.8. 9.8.2The aggregate liability of UCLB and any of its Affiliates shall be limited to the total income that UCLB has received from the Licenseepursuant to this Agreement (but excluding any other costs or expenses associated with drafting, filing, prosecuting, maintaining ordefending any Patents or providing any assistance to the Licensee) during the period of [***] ([***]) years preceding the date on whichthe liability arises, or fifty thousand pounds (£50,000) sterling, whichever is the higher. 9.8.3The liability of the Licensee to UCLB shall be limited to the limit of its insurance as set out in Clause 9.9.1, except that in the case ofproduct liability, the liability of the Licensee under this Agreement shall be unlimited. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 25Confidential Treatment Requested by MeiraGTx Holdings plc 9.8.4In no circumstances shall any Party or any Indemnitee be liable for any loss, damage, costs or expenses of any nature that is (a) of anindirect, special or consequential nature or (b) any loss of profits (whether direct or indirect), revenue, business opportunity or goodwill,which arises directly or indirectly from that Party’s breach or nonperformance of this Agreement, or negligence in the performance ofthis Agreement or from any liability arising in any other way out of the subject matter of this Agreement even if the Party bringing theclaim has advised any other Party or the relevant Indemnitee of the possibility of those losses arising, or if such losses were within thecontemplation of the Parties or the Indemnitee. 9.8.5Nothing in this Agreement excludes any Party’s liability to the extent that it may not be so excluded under applicable law, includingany such liability for death or personal injury caused by that Party’s negligence, or liability for fraud or fraudulent misrepresentation. 9.9Insurance 9.9.1The Licensee shall take out with a reputable insurance company and maintain at all times during the term of this Agreement public andproduct liability and professional indemnity insurance including against all loss of and damage to property (whether real, personal orintellectual) and injury to persons including death arising out of or in connection with this Agreement and the Licensee’s and itsAffiliates’ and Sub-licensees’ use of the Patents, Know-how or Materials and use, sale of or any other dealing in any of the LicensedProducts. Such insurances may be limited in respect of one claim provided that such limit must be at least [***] pounds (£[***])sterling, unless the Licensee commences any business in manufacturing, distribution, supply or otherwise make available to the publicany products, in which case such limit must be at least [***] pounds (£[***]) sterling. Such insurance shall continue to be maintainedfor a further [***] years from the end of this Agreement. 9.9.2The Licensee will produce to UCLB at all times upon demand proof that the insurance cover required pursuant to Clause 9.9.1 is in forceand evidence that all premiums have been paid up to date. If UCLB becomes aware that the Licensee has failed to maintain theinsurance required pursuant to Clause 9.9.1, UCLB may effect such insurance and the Licensee will reimburse UCLB for the reasonablecost of effecting and maintaining such insurance on demand. 10.DURATION AND TERMINATION 10.1Commencement and ExpiryThis Agreement shall commence as of the Commencement Date and, unless terminated earlier in accordance with this Clause 10, the licencesgranted hereunder shall continue in force on a country by country basis until the later of the last payment obligation of Licensee expires underthis Agreement. Upon such expiry, Licensee’s licenses under this Agreement shall become full-paid, perpetual and irrevocable. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 26Confidential Treatment Requested by MeiraGTx Holdings plc 10.2Termination of the Original AgreementUpon the Commencement Date, pursuant to Amendment No. 4 to the Original Agreement, the Original Agreement shall be deemed to haveterminated. 10.3Early TerminationEach Party (the “Terminating Party”) may terminate this Agreement at any time by notice in writing to the other Parties (“Other Parties”),such notice to take effect as specified in the notice: 10.3.1If, in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, is in material breach of this Agreementand, in the case of a breach capable of remedy within thirty (30) days, the breach is not remedied within thirty (30) days of the OtherParties receiving notice specifying the breach and requiring its remedy or where the breach relates to non-payment of an undisputed sumdue under this Agreement, the sum is not paid in full within fourteen (14) days following the Other Party receiving notice specifying thenonpayment and requiring payment in full; provided however, that in respect on breaches not relating to non-payment, if such breach iscapable of being cured but cannot be cured within such thirty (30) day period and the Other Party initiates actions to cure such breachwithin such period and thereafter diligently pursues such actions, the Other Party shall have such additional period as is reasonableunder the circumstances to cure such breach; it being understood that no such extension shall apply with respect to any undisputedpayment obligations or extend beyond six (6) months from the end of such thirty (30) day period. In the event there is a genuine disputebetween the Parties with respect to any alleged breach hereunder, no purported termination of this Agreement pursuant to this Clause10.3.1 shall take effect while the Parties are actively working to resolve such dispute; or 10.3.2if: (a)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, becomes insolvent or unable topay its debts as and when they become due; (b)an order is made or a resolution is passed for the winding up of in the case of UCLB, either of the Other Parties, or in the case ofthe Licensee or Meira, UCLB (other than voluntarily for the purpose of solvent amalgamation or reconstruction); Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 27Confidential Treatment Requested by MeiraGTx Holdings plc (c)a liquidator, administrator, administrative receiver, receiver or trustee is appointed in respect of the whole or any part of, in thecase of UCLB, either of the Other Parties’, or in the case of the Licensee or Meira, UCLB’s, assets or business; (d)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, makes any composition with itscreditors; (e)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, ceases to continue its business;or (f)any event analogous to the events referred to in paragraphs (a) to (e) above occurs in any other jurisdiction. 10.4UCLB may terminate this Agreement by giving written notice to the Licensee and Meira, such termination to take effect forthwith or asotherwise stated in the notice: 10.4.1if there is any change of Control of the Licensee involving the categories of persons or Affiliates of persons prohibited by Clause 2.3; 10.4.2the Licensee is in persistent breach of the Agreement at least [***] ([***]) times in a calendar year, wherein the nature of each suchbreach is the same in each instance, and where the Parties have failed to agree on a mechanism to remedy the persistent nature of suchbreaches within a reasonable period following UCLB notifying Licensee of the persistent breaches and requesting that the Licenseeenters into discussions with UCLB as to mechanisms for remedying the persistent breaches or if the Parties have agreed to a mechanismto remedy the persistence of such breach by that mechanism if not fully complied with by the Licensee; or 10.4.3if the Licensee shall enter into any sub-licence with any of the categories of persons or Affiliates of persons prohibited by Clause 2.3which may, adversely affect UCL’s and/or UCLB’s reputation; 10.4.4subject to Clause 5.5, if the Licensee fails to achieve any of the milestone events described in Schedule 4 provided that if achievementof any of the milestone events should be compromised due to technical, legal or regulatory issues, the Parties shall first meet and UCLBwill work with the Licensee to manage the delivery schedule and provided that the Licensee is using Diligent Efforts to correct theissues, the applicable deadline in Schedule 4 shall be extended by six (6) months or such other time period as shall be agreed betweenthe Parties in writing after which if the Licensee has not achieved the milestone UCLB shall be entitled, subject to Clauses 5.11 and5.12, to terminate this Agreement by giving written notice to the Licensee. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 28Confidential Treatment Requested by MeiraGTx Holdings plc 10.5A Party’s right of termination under this Agreement, and the exercise of any such right, shall be without prejudice to any other right or remedy(including any right to claim damages) that such Party may have in the event of a breach of contract or other default by any other Party. 10.6Consequences of Termination 10.6.1Upon expiry of the period of this Agreement, and subject to all royalties and any other sums due to UCLB under this Agreement havingbeen duly paid, the Licensee shall have a fully paid up licence to the Patents, the Know-how and the Materials of the same scope as setforth in Clause 2.1 without any further obligation to pay any further sums to UCLB under Clause 4. Notwithstanding the foregoing theLicensee acknowledges that once each Patent expires or is abandoned or withdrawn or allowed to lapse in any country or territory, thirdparties in that country or territory will be entitled to use the inventions claimed in the Patent and that accordingly the licence granted tothe Licensee under Clause 2.1 will no longer be exclusive in that country or territory. 10.6.2Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4: (a)the Licensee and its Affiliates and Sub-licensees shall be entitled to sell, use or otherwise dispose of (subject to payment ofroyalties under Clause 4) any unsold or unused stocks of the Licensed Products for a period of [***] ([***]) months followingthe date of termination; (b)subject to paragraph (a) above, any license that has not become fully paid-up in accordance with Clause 10.1 shall terminateand the Licensee and its Affiliates (and subject to Clause 2.3, its Sub-licensees) shall no longer be licensed to use or otherwiseexploit the Patents and/or the Know-how and/or the Materials, in so far and for as long as any of the Patents remains in forceand the Know-how remains confidential; (c)the Licensee shall consent to the cancellation of any formal licence granted to it, or of any registration of it in any register, inrelation to any of the Patents; (d)the Licensee will, promptly on UCLB’s request, provide (and will ensure that its patent agents provide) to UCLB allinformation, documentation and assistance (including executing documents) which are in the possession of Licensee or itspatent agents relating to the Patents and which UCLB may reasonably require to enable it to continue with the drafting, filing,prosecution and maintenance of the Patents; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 29Confidential Treatment Requested by MeiraGTx Holdings plc (e)except as set out in Clause 2.3, all sub-licences of the Patents and/or the Know-how and/or the Materials granted by theLicensee pursuant to this Agreement will automatically terminate; (f)UCLB shall, upon the written request of either of the other Parties, and each of the Licensee and Meira shall, upon the writtenrequest of UCLB, return or destroy any documents or other materials that are in its or its Affiliates possession or under its ortheir control and that contain the requesting Party’s Confidential Information. 10.6.3Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4, in theevent that UCLB would be unable, absent a licence from the Licensee, to use or permit others to use or to exploit or permit others toexploit the relevant Licensed Technology without infringing intellectual property rights in any invention developed by the Licensee,whether solely or jointly with others (“Blocking Invention”), the Licensee shall be deemed to have granted UCLB the irrevocablenon-exclusive right to use, exploit and permit others to use and exploit the Blocking Invention only in conjunction with the relevantLicensed Technology. The Licensee shall at the request of UCLB provide (and will ensure that its patent agents provide) to UCLB allinformation, documentation and assistance (including executing documents) which UCLB may reasonably require to enable it tocontinue with the drafting, filing, prosecution and maintenance of the Patents licensed under this Agreement; 10.7Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4, the Licenseeshall, to the extent it is able to do so without being in breach of any obligation owed to a third party, disclose to UCLB full details of any andall Intellectual Property generated at any time by or on behalf of the Licensee as a result of the exercise of the Licensee’s rights under thisAgreement (“Licensee IP”) and, upon UCLB’s written request within [***] ([***]) days following such disclosure, negotiate in good faith toagree the terms of an exclusive or non-exclusive licence to UCLB (as UCLB may request) under the Licensee IP. If the Parties fail to agree theterms of such a licence within [***] days following commencement of such negotiation, despite negotiating in good faith, UCLB’s rights underthis Clause shall lapse. If Licensee may terminate this Agreement under Clause 10.3.1 (for UCLB or its Affiliates uncured material breach), thenLicensee may elect, in lieu of terminating the entire Agreement, to have all licenses granted to the Licensee under this Agreement continue inforce, subject to Licensee’s fulfilment of [***] percent ([***]%) of its payment obligations under Clause 4 after what would have been theeffective date of such termination. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 30Confidential Treatment Requested by MeiraGTx Holdings plc 10.8Upon termination of this Agreement for any reason, the provisions of Clauses 1, 2.3, 2.5, 3.1 to 3.5, 4 (in respect of amounts paid and payable toUCLB in respect of the period up to and including the date of termination), 5.7, 7, 9, 10.6, 10.7, 10.8 and 11 of this Agreement shall remain inforce. 11.GENERAL 11.1Force Majeure 11.1.1Any delays in or failure of performance by a Party under this Agreement will not be considered a breach of this Agreement and if and tothe extent that such delay or failure is caused by occurrences beyond the reasonable control of that Party including acts of God; acts,regulations and laws of any government; strikes or other concerted acts of workers; fire; floods; explosions; riots; wars; rebellion; andsabotage; and any time for performance hereunder will be extended by the actual time of delay caused by any such occurrence. 11.1.2If (a) UCLB or (b) the Licensee or Meira is prevented from carrying out its obligations: (a)under this Agreement for a continuous period of [***] ([***]) months, the Licensee (in the case of (a)) or UCLB (in the case of(b)), may terminate this Agreement on giving [***] ([***]) days prior written notice provided always that at the date uponwhich termination becomes effective the Party which was prevented from carrying out its obligations under this Agreementremains so prevented. 11.2AmendmentThis Agreement may only be amended in writing signed by duly authorised representatives of the Parties. 11.3Assignment and Third Party Rights 11.3.1Subject to Clause 11.3.3, the Licensee shall not assign, mortgage, charge or otherwise transfer any rights or obligations under thisAgreement, nor any of the Patents, Know-how or Materials, without the prior written consent of UCLB. 11.3.2UCLB may assign all its rights and obligations under this Agreement together with its rights in the Patents, Know-how and Materials toany third party. 11.3.3The Licensee, subject to obtaining the consent of UCLB which shall not be unreasonably withheld or delayed (except in relation tothose categories of persons or Affiliates of persons prohibited by Clause 2.3), may assign all its rights and obligations under thisAgreement together with its rights in the Patents, Know-how and Materials to any third party to which it transfers all or substantially allof its assets or business, provided that the assignee undertakes to UCLB to be bound by and perform the obligations of the assignorunder this Agreement. However, the Licensee shall not have such a right to assign this Agreement if it is insolvent. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 31Confidential Treatment Requested by MeiraGTx Holdings plc 11.3.4Meira shall not assign, mortgage, charge or otherwise transfer any rights or obligations under this Agreement without the prior writtenconsent of UCLB. 11.4WaiverAny waiver given under or in relation to this Agreement shall be in writing and signed by or on behalf of the relevant Party. No failure or delayon the part of a Party to exercise any right or remedy under this Agreement shall be construed or operate as a waiver thereof, nor shall any singleor partial exercise of any right or remedy preclude the further exercise of such right or remedy. 11.5Invalid ClausesIf any provision or part of this Agreement is held to be invalid, amendments to this Agreement may be made by the addition or deletion ofwording as appropriate to remove the invalid part or provision but otherwise retain the provision and the other provisions of this Agreement tothe maximum extent permissible under applicable law. 11.6No AgencyNo Party shall act or describe itself as the agent of the other, nor shall it make or represent that it has authority to make any commitments on theother’s behalf. 11.7InterpretationIn this Agreement: 11.7.1the headings are used for convenience only and shall not affect its interpretation; references to persons shall include incorporated andunincorporated persons; references to the singular include the plural and vice versa; and references to the masculine include thefeminine; 11.7.2references to Clauses and Schedules mean clauses of, and schedules to, this Agreement; 11.7.3references in this Agreement to termination shall include termination by expiry; 11.7.4where the word “including” is used it shall be understood as meaning “including without limitation”; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 32Confidential Treatment Requested by MeiraGTx Holdings plc 11.7.5any reference to any English law term for any action, remedy, method or judicial proceeding, legal document, legal status, court, officialor any legal concept or thing shall in respect of any jurisdiction other than England be deemed to include what most nearlyapproximates in that jurisdiction to the English law term; 11.7.6where there is any conflict or inconsistency between the main body of this Agreement and any of the schedules, then the main body ofthe Agreement shall prevail; 11.7.7time shall be of the essence in relation to the performance of Meira’s and the Licensee’s obligations under this Agreement; and 11.7.8any reference to the sale of a Licensed Product by the Licensee or its Affiliates or Sub-licensees will be taken to include any supply orother disposal of Licensed Products, and the term sold shall be construed accordingly. 11.8Notices. Addresses for Service 11.8.1Any notice to be given under this Agreement shall be in English, in writing and shall be delivered by first class recorded delivery mail(if sent to an inland address) or by international courier (if sent to an address outside of the United Kingdom), to the address of therelevant Party set out at the head of this Agreement, or such other address as that Party may from time to time notify to the other Partiesin accordance with this Clause 11.8. 11.8.2Notices sent as above shall be deemed to have been received [***] ([***]) working day after the day of posting in the case of deliveryinland first class recorded delivery mail, or [***] ([***]) working days after the date of collection by the international courier. 11.9Law and JurisdictionThe validity, construction and performance of this Agreement, and any contractual and non-contractual claims arising hereunder, shall begoverned by English law and shall be subject to the exclusive jurisdiction of the English courts to which the Parties hereby submit, except thata Party may seek an interim injunction (or an equivalent remedy) in any court of competent jurisdiction. 11.10Entire AgreementThis Agreement, including its Schedules, sets out the entire agreement between the Parties relating to its subject matter and supersedes all priororal or written agreements, arrangements or understandings between them relating to such subject matter including, without limitation, the partsof the Original Agreement that pertains to the Specified Technology [***]. Subject to Clause 9.8.5, the Parties acknowledge that they are notrelying on any representation, agreement, term or condition which is not set out in this Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 33Confidential Treatment Requested by MeiraGTx Holdings plc 11.11Third PartiesExcept for the rights of UCL as provided in Clause 2.4, the rights of the Indemnitees as provided in Clause 9.7, the limitations of liabilityafforded to the Indemnitees pursuant to Clause 9.8, who may in their own right enforce and rely on the provisions of those Clauses, thisAgreement does not create any right enforceable by any person who is not a party to it (“Third Party”) under the Contracts (Rights of ThirdParties) Act 1999, but this Clause 11.11 does not affect any right or remedy of a Third Party which exists or is available apart from that Act. 11.12Non-use of Names; Announcements 11.12.1 The Licensee shall not use, and shall ensure that its Affiliates and Sub-licensees do not use, the name, any adaptation of the name, anylogo, trademark or other device of UCLB, nor of the inventors named on the Patents nor the Principal Investigators in any advertising,promotional or sales materials without prior written consent obtained from UCLB in each case, except that the Licensee may state thatit is licensed by UCLB under the Patents. 11.12.2 Except as permitted under Clauses 3.3.1 and 5.7, no Party shall make any press or other public announcement concerning any aspect ofthis Agreement, or make any use of the name or trademarks of any other Party in connection with or in consequence of this Agreement,without the prior written consent of the relevant other Party. 11.13EscalationIf the Licensee or Meira on the one hand, and UCLB on the other, are unable to reach agreement on any issue concerning this Agreement or theProject within [***] days after one either has notified the other of that issue, they will refer the matter to the [***] in the case of UCLB, and tothe [***] in the case of the Licensee and Meira in an attempt to resolve the issue within the time specified elsewhere in this Agreement in thecase of other disputes. Any Party may bring proceedings in a court of competent jurisdiction if the matter has not been resolved within thatprescribed period, and any Party may apply to the court for an injunction, whether or not any issue has been escalated under this Clause 11.13.[Signature Page Follows] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 34Confidential Treatment Requested by MeiraGTx Holdings plc EXECUTED on the date set out at the head of this Agreement. For and on behalf of UCL Business PLC For and on behalf of MEIRAGTX UK II LIMITED/s/ [***] /s/ Zandy ForbesSigned Signed[***] Zandy ForbesPrint name Print name[***] President and CEOTitle Title29 January 2019 Jan 29, 2019Date Date For and on behalf of MEIRAGTX LIMITED/s/ Zandy ForbesSignedZandy ForbesPrint namePresident and CEOTitleJan 29, 2019Date Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 35Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 1LICENSED TECHNOLOGYPart A: The Patents[***]Part B: The Know-how[***]Part C: The Materials[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 36Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 2APPOINTMENT OF EXPERTFor the purposes of this Schedule 2 only, the “Parties” shall mean the Licensee and UCLB. If either Party wishes to appoint an independent expert (the“Expert”) to determine any matter pursuant to any Clause of this Agreement, the following procedures will apply: 1.The Party wishing to appoint the Expert (the “Appointing Party”) will serve a written notice on the other Party (the “Responding Party”). Thewritten notice will specify the Clause pursuant to which the appointment is to be made and will contain reasonable details of the matter(s)which the Appointing Party wishes to refer to the Expert for determination. 2.The Parties shall within [***] ([***]) days following the date of the Appointing Party’s written notice use all reasonable efforts to agree who isto be appointed as the Expert to determine the relevant matter(s). If the Parties are unable to agree upon the identity of the Expert within thattimescale, the Expert shall be appointed by the President (for the time being) of the Licensing Executives Society Britain and Ireland uponwritten request of either Party. 3.Each Party will within [***] ([***]) days following appointment of the Expert, prepare and submit to the Expert and the other Party a detailedwritten statement setting out its position on the matter(s) in question and including any proposals which it may wish to make for settlement orresolution of the relevant matter. 4.Each Party will have [***] ([***]) days following receipt of the other Party’s written statement to respond in writing thereto. Any such responsewill be submitted to the other Party and the Expert. 5.The Expert will if he/ she deems appropriate be entitled to seek clarification from the Parties as to any of the statements or proposals made byeither Party in their written statement or responses. Each Party will on request make available all information in its possession and shall givesuch assistance to the Expert as may be reasonably necessary to permit the Expert to make his/ her determination. 6.The Expert will issue his/ her decision on the matter(s) referred to him/ her in writing as soon as reasonably possible, but at latest within [***]([***]) months following the date of his/ her appointment. The Expert’s decision shall (except in the case of manifest error) be final and bindingon the Parties. 7.The Expert will at all times act as an independent and impartial expert and not as an arbitrator. 8.The Expert’s charges will be borne as he/ she determines in his written decision. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 37Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 3DEFINITION OF TOBACCO INDUSTRY FUNDING (REVISED 2009)FROM THE CANCER RESEARCH UK CODE OF PRACTICE ON TOBACCO INDUSTRY FUNDING TO UNIVERSITIES.http://www.cancerresearchuk.org/science/funding/terms-conditions/funding-policies/policy-tobacco/A tobacco company is defined for the purposes of this policy as one that: • Derives over 5% of revenues from manufacturing tobacco products; • Derives 15%+ of revenues from the manufacture of products necessary for the production of tobacco products; • Derives 15% of revenues from the sale of tobacco products (and has 30 or more staff); • Owns a tobacco company (the company owns 50% or more of a tobacco company); • Is more than 50% owned by a company with tobacco involvement.The following do not constitute tobacco industry funding for the purposes of this Code: • legacies from tobacco industry investments (provided these are sold on immediately) • funding from a trust or foundation no longer having any connection with the tobacco industry even though it may bear a name that (forhistorical reasons) has tobacco industry associations. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 38Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 4DEVELOPMENT PLANThe Development Plan for the Licensed Technology is shown below Activity TimelinePhase I/II Start [***]Phase I/II Finish [***]Phase III /pivotal confirmatory study Start [***]Phase III /pivotal confirmatory study Finish [***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 39Confidential Treatment Requested by MeiraGTx Holdings plc [***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Exhibit 10.23Confidential Treatment Requested by MeiraGTx Holdings plcLICENCE AGREEMENTbetweenUCL Business PlcandMeiraGTx UK II LimitedandMeiraGTx LimitedDated: 5 February 2019Ref: [***] (RPGR)Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc INDEX 1. Definitions 2 2. Grant of Rights 5 3. Know-how and Confidential Information 10 4. Consideration 13 5. Commercialisation 17 6. Access to Medicines and Ethical Licensing 20 7. Compliance with Laws 20 8. Intellectual Property 21 9. Warranties and Liability 25 10. Duration and Termination 31 11. General 35 Schedule 1 Licensed Technology 41 Schedule 2 Appointment of Expert 42 Schedule 3 Definition of Tobacco Industry Funding (Revised 2009) 43 Schedule 4 Initial Development Plan 44 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plcTHIS AGREEMENT is made 5 February, 2019BETWEEN: (1)UCL BUSINESS PLC, a company incorporated in England and Wales under company registration number 02776963 whose registered office isThe Network Building, 97 Tottenham Court Road, London W1T 4TP (“UCLB”);and (2)MEIRAGTX UK II LIMITED, a company incorporated in England and Wales with registered number 09348737 and having its registeredoffice at 92 Britannia Walk, London, United Kingdom, N1 7NQ (the “Licensee”); and (3)MEIRAGTX LIMITED, a company incorporated in England and Wales with registered number 09501998 and having its registered office at92 Britannia Walk, London, United Kingdom, N1 7NQ (“Meira”).WHEREAS: A.University College London (“UCL”), the Medical Research Council (“MRC”), Massachusetts Eye and Ear Infirmary (“MEEI”), and NationalInstitutes of Health which is a part of the U.S. Department of Health and Human Services (“NIH”) developed certain technology and ownedcertain intellectual property rights relating to a gene therapy for treating retinitis pigmentosa using an abbreviated form of a human RetinitisPigmentosa GTPase Regulator (RPGR) gene that the Licensee wishes to acquire rights to for the development and commercialisation ofLicensed Products. B.UCL has assigned to UCLB all of its right, title and interest in and to such property. The NIH has licensed, maintaining certain reserved rights,all of its right, title and interest in and to the Patents, the Know-how and the Materials to MEEI. MEEI has granted to UCLB, maintainingcertain reserved rights, an exclusive licence under the Patents and to use the Know-how and Materials. C.The Licensee and UCLB previously entered into a certain License Agreement dated as of July 28, 2017 (the “Original Commencement Date”)(the “Original Agreement”). D.The Parties now desire to amend the Original Agreement and enter into a new agreement for the Specified Technology [***] on the terms set outin this Agreement. E.It is the policy of UCLB that its activities in licensing intellectual property take into consideration ethical and socially responsible licensingprinciples, including ensuring that Licensed Products are made available to fulfil unmet needs in developing countries, and the Licenseeacknowledges and agrees to carry out its activities under this Agreement in a manner which complies with ethical and socially responsiblelicensing principles and which is designed to fulfil such needs, all in accordance with the provisions of this Agreement.NOW IT IS AGREED as follows: 1.DEFINITIONS 1.1In this Agreement:Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc Agreement means this agreement (including the Schedules);Affiliate in relation to a Party, means any entity or person that Controls, is Controlled by, or is under common Control with that Party;Claims means all demands, claims and liability (whether criminal or civil, in contract, tort or otherwise) for losses, damages, costs and expensesof any nature whatsoever and all costs and expenses (including legal costs) incurred in connection therewith;Commencement Date means the date of this Agreement;Competing Product means any [***];Confidential Information means the Know-how, the Materials and all other technical or commercial information that: a)in respect of information provided in documentary form or by way of a model or in other tangible form, at the time of provision ismarked or otherwise designated to show expressly that it is imparted in confidence or which a reasonable person would expect to beconfidential; and b)in respect of information that is imparted orally, any information that the Disclosing Party or its representatives informed the ReceivingParty at the time of disclosure or which a reasonable person would expect to be confidential;Control means direct or indirect beneficial ownership of 50% (or, outside a Party’s home territory, such lesser percentage as is the maximumpermitted level of foreign investment) or more of the share capital, stock or other participating interest carrying the right to vote or todistribution of profits of that Party, as the case may be;Diligent Efforts means, with respect to efforts to be expended by the Licensee with respect to any objective under this Agreement, diligent,reasonable, good faith efforts to accomplish such objective [***], it being understood and agreed that with respect to the research,development, or commercialization of a product, such efforts will be substantially equivalent to those that would normally be exerted oremployed by [***], when utilizing sounds and reasonable scientific, medical and business practice and judgment;Disclosing Party has the meaning given in Clause 3.2;Field means ocular gene therapy;First Commercial Sale means the first sale to a third party of a Licensed Product in a given regulatory jurisdiction after all regulatory andmarketing approvals have been obtained for such Licensed Product in such jurisdiction. A sale shall not be deemed to have occurred if aLicensed Product is provided pursuant to an early access or compassionate use; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 3Confidential Treatment Requested by MeiraGTx Holdings plc Indemnitees has the meaning given in Clause 9.7;Intellectual Property means any and all patents, utility models, registered designs, unregistered design rights, copyright, database rights, rightsin respect of confidential information, rights under data exclusivity laws, rights under orphan drug laws, rights under unfair competition laws,property rights in biological or chemical materials, extension of the terms of any such rights (including supplementary protection certificates),applications for and the right to apply any of the foregoing registered property and rights, and similar or analogous rights in any part of theTerritory;Know-how means: a)the inventions claimed in the Patents; and b)the technical information relating to the inventions claimed in the Patents and data described in the Part B of Schedule 1;Licensed Products means any and all products that are developed, manufactured, used, or sold by or on behalf of the Licensee or its Affiliatesor Sub-licensees and which (a) are within (or are manufactured using a process described in) any claim of the Patents; and/or (b) incorporate, ortheir development or manufacture makes use of, any of the Know-how and/or the Materials;Licensed Technology means the Patents, the Know-how and the Materials set out in Schedule 1;Materials means any and all of the materials referred to in Part C of Schedule 1;MEEI lndemnitees has the meaning given in Clause 10.7.1;Net Sales Value means in respect of [***]Original Commencement Date has the meaning given in the Recitals;Original Agreement has the meaning given in the Recitals;Parties means UCLB, the Licensee and Meira, and “Party” shall mean either of them;Patent Costs means [***].Patents means any and all of the patents and patent applications referred to in Part A of Schedule 1; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 4Confidential Treatment Requested by MeiraGTx Holdings plc Principal Investigators means [***].Receiving Party has the meaning given in Clause 3.2;Regulatory Exclusivity means, with respect to a Licensed Product, any exclusive rights or protection which are recognised, afforded or grantedby any regulatory authority in any country or region with respect to the Licensed Product other than through patent rights;Sub-licensee means any third party (other than an Affiliate) to whom the Licensee grants a sub-licence of its rights under this Agreement inaccordance with Clause 2.3;Territory means worldwide;Valid Claim means a claim of a patent or patent application that has not been abandoned or allowed to lapse or expired or been held invalid orunenforceable by a court of competent jurisdiction in a final and non-appealable judgment. 2.GRANT OF RIGHTS 2.1LicenceUCLB hereby grants to the Licensee and its Affiliates, and the Licensee hereby accepts on its own behalf and on behalf of its Affiliates, subjectto the provisions of this Agreement: 2.1.1an exclusive (even as to UCL) licence under the Patents, the Know-how and the Materials, with the right to sub-license, subject toClause 2.3, to develop, commercialise, manufacture, have manufactured, use, sell and have sold Licensed Products only in the Field andin the Territory. 2.2UCLB shall at the Licensee’s request and cost execute such formal licences as may be necessary to enable the Licensee to register thelicences granted to it under this Agreement with the Patent Offices in the relevant Territory. Such formal licence will reflect the terms ofthis Agreement where possible and for the avoidance of doubt if there is a conflict in the terms of such formal licence and this Agreement,the terms of this Agreement shall prevail. [***]. 2.3Sub-LicensingThe Licensee shall have the right to grant sub-licenses under the license in Clause 2.1 to its Affiliates or other third parties through one or morelevels of Sub-licensees except that the Licensee may not grant such a sub-license to any person or the Affiliates of any person involved in: thetobacco industry (as defined by the Cancer Research UK Code of Practice on Tobacco Industry Funding to Universities detailed in Schedule 3);arms dealing; gambling operations; the promotion of violence; child labour or any other illegal activity. A grant of any sub-licence shall beconditioned on the following: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 5Confidential Treatment Requested by MeiraGTx Holdings plc (a)The Licensee shall enter into a written agreement with each Sub-licensee and shall ensure that the provisions of eachsub-licence are consistent with the provisions of this Agreement, and the Licensee shall ensure that: (i)the sub-licence sets out all the proposed terms agreed between the Licensee and the Sub-licensee, including, inparticular, all terms as to remuneration; (ii)the Sub-licensee will maintain complete and accurate records in sufficient detail to permit UCLB to confirm theaccuracy of the calculation of royalty payments under this Agreement; and (iii)the sub-licence imposes obligations of confidentiality on the Sub-licensee which are no less onerous than those set outin Clause 3.2. (b)The Licensee shall procure that each Sub-licensee complies fully at all times with the provisions of its sub-licence. (c)The Licensee shall be liable for all acts and omissions of its Sub-licensees that, if committed by the Licensee, would constitutea breach of any of the provisions of this Agreement. (d)The Licensee shall provide UCLB with a copy of any sub-licence [***] ([***]) days after execution of such sub-licence,provided that the Licensee may redact confidential or proprietary terms from such copy, including financial terms. (e)Each sub-licence shall terminate automatically upon termination of this Agreement for any reason (but not expiry of thisAgreement under Clause 10.1), except where the Sub-licensee was not implicated in or at fault in any circumstances which ledto the termination of this Agreement, UCLB shall on receiving a written request from the relevant Sub-licensee within [***]([***]) days following the date of termination of this Agreement enter into a licence agreement with the Sub-licensee for theLicensed Technology on terms substantially the same as the terms set out in this Agreement (except that the Sub-licensee shallnot be obliged to pay to UCLB any sums equivalent to those sums set out in Clauses 4.1 or 4.3 which have already been paidto UCLB by the Licensee prior to the date of termination). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 6Confidential Treatment Requested by MeiraGTx Holdings plc 2.4Reservation of Rights 2.4.1UCLB reserves for itself and UCL the non-exclusive, irrevocable, worldwide, royalty-free right to: (a)Use the Licensed Technology in the Field solely for academic (non-commercially funded) research, publication and teaching;and (b)Grant licenses to academic third parties to use the Licensed Technology in academic research collaborations with UCL andsuch licenses shall not be sublicensable; and (c)Grant license of the Licensed Technology to post graduate student of UCL for the purpose of conducting a programme of postgraduate academic research and such licenses shall not by sublicensable.In exercising the rights described in Clause 2.4.1(b) and (c), UCL and UCLB shall comply with the provisions of Clause 3 as regardsconfidentiality of the Know-how. 2.4.2UCLB reserves for MEEI the non-exclusive, irrevocable, worldwide, royalty-free right to: (a)use the Patents, the Know-how and the Materials solely for MEEl’s own internal non-commercially funded research,publication and teaching, excluding use in human subjects, clinical trials or for diagnostic purposes involving human subjects; (b)to license other academic institutions to use the Patents, the Know-how and the Materials solely in non-commercially fundedacademic research collaborations with MEEI, excluding research for use in human subjects, clinical trials or research fordiagnostic purposes involving human subjects; and (c)to grant licences of the Patents, the Know-how and the Materials to other academic, governmental or not-for-profitorganisations to use the Patents, the Know-how and the Materials solely for non-commercial research purposes and not for usein human subjects, clinical trials or for diagnostic purposes involving human subjects. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 7Confidential Treatment Requested by MeiraGTx Holdings plc 2.4.3MEEI and UCLB will refer a request from a third party for a licence to use the Patents in clinical trials or for diagnostic purposesinvolving human subjects to the Licensee, and the Licensee shall liaise directly with such third party. 2.4.4UCLB reserves for the U.S. Government only the irrevocable, royalty-free, paid-up right to practice and have practiced the rights underthe Patents throughout the world by or on behalf of the U.S. Government and on behalf of any foreign government or internationalorganisation pursuant to any existing or future treaty or agreement to which the U.S. Government is a signatory. 2.4.5UCLB reserves the right, if required by the NIH, to grant sub-licenses of the rights under the Patents to responsible applicants, on termsthat are reasonable under the circumstances when necessary to fulfill health or safety needs or when necessary to meet requirements forpublic use specified by U.S. Federal regulations. 2.4.6UCLB reserves for the NIH only the right to require the Licensee, to grant sub-licenses of the rights under the Patents to responsibleapplicants, on terms that are reasonable under the circumstances when necessary to fulfill health or safety needs or when necessary tomeet requirements for public use specified by U.S. Federal regulations. 2.4.7In addition to the reserved rights of Clause 2.4.5, UCLB reserves the right, should it be required by the NIH, to grant a non-transferable,non-exclusive license to make and to use any tangible embodiment of the Patents and to practice any process(es) included within thePatents for purposes of internal research and not for purposes of commercial manufacture or distribution or in lieu of purchase, onreasonable terms and conditions. 2.4.8In addition to the reserved rights of Clause 2.4.6, UCLB reserves for the NIH only the right to require the Licensee to grant anon-transferable, non-exclusive license to make and to use any tangible embodiment of the Patents and to practice any process(es)included within the Patents for purposes of internal research and not for purposes of commercial manufacture or distribution or in lieu ofpurchase, on reasonable terms and conditions. 2.4.9UCLB reserves for the MRC the non-exclusive right to: (a)use the Patents for its own internal, not-for-profit and non-commercially funded research, teaching and publicity, excluding usein human subjects, clinical trials or for diagnostic purposes involving human subjects; and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 8Confidential Treatment Requested by MeiraGTx Holdings plc (b)licence academic institutions who employ [***] to use the Patents in not-for-profit and non-commercially funded research,excluding research for use in human subjects, clinical trials or research for diagnostic purposes involving human subjects. 2.4.10Except for the licences expressly granted by this Clause 2, UCLB grants no rights to the Licensee under this Agreement to or under anyintellectual property other than the Patents, the Know-how and the Materials and hereby reserves all rights under the Patents, theKnow-how and the Materials outside the Field. 2.4.11Nothing in this Agreement shall limit or otherwise affect UCL’s ability to apply for non-commercial grant funding or comply with suchgrant terms and conditions. In the event that any terms of this Agreement conflicts with the terms of any non-commercial grant funding,the Parties shall negotiate in good faith to amend the terms of this Agreement to allow UCL to access such funding provided thatnothing herein shall require the Licensee to agree to alter or modify the scope of the licence granted to it in this Clause 2. 2.5AffiliatesThe Licensee shall: 2.5.1ensure that its Affiliates comply fully with the terms of this Agreement; 2.5.2be responsible for any breach of or non-compliance with this Agreement by its Affiliates as if the breach or non-compliance had been abreach or non-compliance by the Licensee; 2.5.3indemnify in accordance with Clause 9.7 each of the Indemnitees against any Claims which are awarded against or suffered by any ofthe Indemnitees as a result of any breach of or non-compliance with this Agreement by its Affiliates; and 2.5.4ensure that if any Affiliate ceases to be an Affiliate as a result of a change of Control or otherwise, that unless a sub-licence agreement inaccordance with Clause 2.3 is entered into with such an Affiliate, such former Affiliate immediately upon such cessation: (a)ceases developing, manufacturing, having manufactured, using, selling and/ or having sold Licensed Products and ceases alluse or exploitation of the Licensed Technology, for as long as any of the relevant Patents remains in force and/or theKnow-how remains confidential; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 9Confidential Treatment Requested by MeiraGTx Holdings plc (b)returns to the Licensee or destroys any documents or other materials in the former Affiliate’s possession or under its control andthat contain Confidential Information provided under this Agreement relating to the Licensed Technology and/ or LicensedProducts; (c)to the extent possible, takes all action necessary to have any product licences, marketing authorisations, pricing and/ orreimbursement approvals (and any applications for any of the foregoing) which relate to Licensed Products transferred into thename of the Licensee. 2.6Use of Licensed Technology in CombinationUCLB acknowledges and agrees that the Licensee shall be entitled to use the Licensed Technology in combination with other technology,patents, know-how and materials licensed by UCLB to the Licensee under separate licence agreements and with any improvements to theLicensed Technology developed or generated by the Licensee. 3.KNOW-HOW AND CONFIDENTIAL INFORMATION 3.1Confidentiality of Know-how and MaterialsThe Licensee undertakes that for so long as the Know-how and/or the Materials remains confidential, it shall (and shall ensure that its Affiliatesand Sub-licensees) take all reasonable precautions to prevent unauthorised access to the Know-how and the Materials and protect theKnow-how and the Materials in the same manner as it (or they) protect(s) its (or their) own proprietary information, and shall not (and shallensure that its Affiliates and Sub-licensees do not) use the Know-how or the Materials for any purpose, except as expressly licensed hereby andin accordance with the provisions of this Agreement. For the avoidance of doubt, to the extent that any Materials, Know-how or informationrelating to the Patents falls within the public domain (without any breach of this Agreement or any other obligation of confidentiality), thenUCL, the Principal Investigators and UCLB shall be free to use such information without restriction in the same way that any third party wouldhave the freedom to use it. 3.2Confidentiality ObligationsEach Party (“Receiving Party”) undertakes: 3.2.1to maintain as secret and confidential all Confidential Information obtained from, in the case of UCLB, the Licensee or Meira asapplicable, and in the case of the Licensee and Meira, UCLB (“Disclosing Party”) in the course of or in anticipation of this Agreementand to respect the Disclosing Party’s rights therein; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 10Confidential Treatment Requested by MeiraGTx Holdings plc 3.2.2to use such Confidential Information only for the purposes of or as permitted by this Agreement; and 3.2.3subject to Clause 3.3, to disclose such Confidential Information only to those of its employees, contractors, Affiliates, and Sub-licensees(if any) to whom and to the extent that such disclosure is reasonably necessary for the purposes of this Agreement. 3.3Permitted Disclosure 3.3.1UCLB shall have the right to provide the MRC and MEEI with brief annual updates on the status of the commercial exploitation of thePatents, the Materials and the Know-how. 3.3.2UCLB shall have the right to disclose to the MRC under binding obligations of confidentiality: (a)that it has entered into this Agreement with the Licensee; (b)details of all milestone payments and royalty payments provided for in this Agreement; (c)a copy of the Initial Development Plan; (d)details of any development milestones provided for in this Agreement. 3.3.3The Licensee shall have the right to disclose Confidential Information received from UCLB to: (a)potential or actual customers of Licensed Products to the extent reasonably necessary to promote the sale or use of LicensedProducts and provided that the customer has agreed to confidentiality provisions at least as restrictive as set forth herein; (b)to existing or potential Sub-licensees, collaborators, investors or lenders provided that such third parties have agreed toconfidentiality provisions at least as restrictive as set forth herein; and (c)to its Board of Directors (or similar governing body) and its counsel, accountants and other professional advisers. 3.4Exceptions to ObligationsThe provisions of Clause 3.2 shall not apply to Confidential Information which the Receiving Party can demonstrate by reasonable writtenevidence: 3.4.1was, prior to the Original Commencement Date, in the possession of the Receiving Party and at its free disposal and was not obtained orotherwise acquired directly or indirectly from the Disclosing Party or its Affiliates or their respective employees, students orrepresentatives; or Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 11Confidential Treatment Requested by MeiraGTx Holdings plc 3.4.2is subsequently disclosed to the Receiving Party without any obligations of confidence by a third party; or 3.4.3is or becomes generally available to the public through no act or default of the Receiving Party or its agents, employees, Affiliates orSub-licensees; or 3.4.4the Receiving Party is required to disclose by or to the courts of any competent jurisdiction, or to any government regulatory agency orfinancial authority, provided that the Receiving Party shall: (a)inform the Disclosing Party as soon as is reasonably practicable; (b)at the Disclosing Party’s request and cost seek to persuade the court, agency or authority to have the information treated in aconfidential manner, where this is possible under the court, agency or authority’s procedures; and (c)where the disclosure is unavoidable, limit the disclosure of Confidential information to the minimum extent required by law; or 3.4.5which a Party is advised by its information officer that it is required to disclose under the Freedom of Information Act 2000 or theEnvironmental Information Regulations 2004. 3.5Disclosure to EmployeesThe Receiving Party shall procure that all of its employees, contractors, Affiliates and Sub-licensees who have access to any of the DisclosingParty’s Confidential Information to which Clause 3.2 applies, shall be made aware of the obligations of confidence and are bound byobligations of confidentiality at least as restrictive as those set forth herein (which it undertakes to enforce and for which it is legallyresponsible) and the Receiving Party shall only disclose the Disclosing Party’s Confidential Information to those of its subsidiaries, employees,and officers as need to have access thereto wholly necessarily and exclusively for the purposes of this Agreement. 3.6Response to NIHIf the Licensee is notified of a determination of a conflict of interest regarding the Patents by the NIH it shall provide a response to suchdetermination to the NIH within the period given by NIH to respond to such determination. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 12Confidential Treatment Requested by MeiraGTx Holdings plc 4.CONSIDERATION 4.1Milestone PaymentsWithin [***] ([***]) days following achievement of each of the following milestone events by Licensee, its Affiliates or Sub-licensees, theLicensee shall notify UCLB in writing that the relevant milestone event has been achieved, provide documentary evidence of suchachievement as appropriate and pay to UCLB, within a period of [***] ([***]) days, the amount(s) set out next to such milestone event below: Milestone Event Amountto bepaid [***] £[***] [***] £[***] 4.2Annual Management FeesOn each date referred to in the following table, the Licensee shall pay to UCLB the annual management fee set out next to such date in thetable. Date Amountto bepaid Upon each anniversary of the Original Commencement Date until [***] £50,000 4.3Sales Linked Milestone PaymentsUpon the first achievement of each of the sales linked milestones set out in the following table by the Licensee, its Affiliates or Sub-licensees,the Licensee shall notify UCLB in writing that the relevant sales linked milestone has been achieved, provide the relevant documentaryevidence and pay to UCLB the amount(s) set out next to such event in the table: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 13Confidential Treatment Requested by MeiraGTx Holdings plc Sales Linked Milestones Amountto bepaid When Net Sales Value reaches £[***] £[***] On the next £[***] of Net Sales Value (when sales cumulatively reach £[***]) £[***] On the next £[***] of Net Sales Value (When sales cumulatively reach £[***]) £[***] On the next £[***] of Net Sales Value (when sales cumulatively reach £[***]) £[***] On the next £[***] of Net Sales Value (when sales cumulatively reach £[***]) £[***] On the next £[***] of Net Sales Value (when sales cumulatively reach £[***]) £[***] 4.4Royalties on Net SalesFor each Licensed Product in each country, the Licensee shall pay to UCLB a royalty of [***]% ([***] per cent) being a percentage of the NetSales Value of such Licensed Product sold by Licensee, its Affiliates or Sub-licensees. The Licensee’s obligations to pay such royalty for agiven Licensed Product in a given country shall begin after the First Commercial Sale of such Licensed Product in such country and shall endon the later to occur of the following: (a) expiration of the last Valid Claim of a Patent claiming such Licensed Product in such country; or(b) the tenth (10th) anniversary of the date of such First Commercial Sale in such country; or (c) the expiration of any Regulatory Exclusivitywith respect to such Licensed Products in the relevant country. 4.5Combination ProductsIf any Licensed Products are incorporated in any other product (“Combination Product”) sold by the Licensee or its Affiliates and the LicensedProduct is not priced separately from the Combination Product, the Net Sales Value of such Licensed Product shall be deemed to be the fairmarket value of the Licensed Product in the country of sale when sold separately or if not sold separately in the country of sale, in comparablecountries and territories or if neither of the foregoing apply, a reasonable amount which fairly reflects the value of the Licensed Product withinthe Combination Product assuming the Licensed Product is not being sold as a loss leader. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 14Confidential Treatment Requested by MeiraGTx Holdings plc 4.6Payment FrequencyRoyalties due under this Agreement, except for the payments due under Clauses, 4.1, 4.2 and 4.3, which are payable upon the date/timespecified in Clauses 4.1, 4.2 and 4.3 as appropriate, shall be paid within [***] ([***]) days following the end of each calendar quarter ending on31 March, 30 June, 30 September and 31 December in each year, in respect of sales of Licensed Products made during such quarter, and within[***] ([***]) days following the termination of this Agreement. 4.7Payment termsAll sums due under this Agreement: 4.7.1are exclusive of Value Added Tax which where applicable will be paid by the Licensee to UCLB in addition; 4.7.2shall be paid in pounds sterling in cash by transferring an amount in aggregate to the following Account name: UCL Business Plc, SortCode: 20 10 53, Account number: 30782270, Address: Barclays Bank Plc, PO Box 11345, London, W12 8GG, and in the case of incomeor amounts received by the Licensee or its Affiliates in a currency other than pounds sterling, the royalty shall be calculated in the othercurrency and then converted into equivalent pounds sterling at the relevant daily spot rate for that currency as quoted in the FinancialTimes newspaper on the last business day of the quarter in relation to which the royalties are payable; 4.7.3will be made without any set-off, deduction or withholding except as may be required by law. If the Licensee is required by law to makeany deduction or to withhold any part of any amount due to UCLB under this Agreement, the Licensee will give to UCLB properevidence of the amount deducted or withheld and payment of that amount to the relevant taxation authority, and will do all things in itspower to enable or assist UCLB to claim exemption from or, if that is not possible, to obtain a credit for the amount deducted orwithheld under any applicable double taxation or similar agreement from time to time in force; and 4.7.4shall be made by the due date, failing which UCLB may charge interest on any outstanding amount on a daily basis at a rate equivalentto [***] above the Bank of England pound sterling base rate then in force in London. 4.8Royalty StatementsThe Licensee shall send to UCLB, at the same time as each royalty payment is made in accordance with Clause 4.4, a statement setting out forthe relevant calendar quarter: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 15Confidential Treatment Requested by MeiraGTx Holdings plc 4.8.1in respect of each territory or region in which Licensed Products are sold; 4.8.2the types of Licensed Product sold; 4.8.3the quantity of each type sold; 4.8.4the total invoiced price for each type of Licensed Product sold; 4.8.5where relevant, details of any Licensed Products that have been sold other than on arm’s length terms for a cash consideration, includingthe relevant open market price or (if not available) the reasonable price attributed thereto; 4.8.6the amounts deducted from the Net Sales Value as referred to in paragraph (i) to (iv) of that definition (broken down on a product byproduct and category by category basis); and 4.8.7the aggregate royalties on Net Sales Value due to UCLB;in each case expressed both in local currency and pounds sterling and showing the conversion rates used, during the period to which the royaltypayment relates. 4.9RecordsThe Licensee shall keep at its normal place of business detailed and up to date records and accounts showing the quantity, description andinvoiced price or non-cash consideration for all Licensed Products sold by it or its Affiliates or on its or its Affiliates’ behalf, broken down ineach case on a country by country basis, and being sufficient to ascertain the payments due to UCLB under this Agreement.The Licensee shall make such records and accounts available, on reasonable notice, for inspection during business hours by an independentchartered accountant nominated by UCLB for the purpose of verifying the accuracy of any statement or report given by the Licensee to UCLBunder this Clause 4.9. The Licensee shall co-operate reasonably with any such accountant, and shall promptly provide all information andassistance reasonably requested by such accountant. The accountant shall be required to keep confidential all information learnt during anysuch inspection, and to disclose to UCLB only such details as may be necessary to report on the accuracy of the Licensee’s statement or report.UCLB shall be responsible for the accountant’s charges unless the accountant certifies that there is an inaccuracy of more than [***]% ([***]percent) in any royalty statement, in which case the Licensee shall pay his charges in respect of that inspection.The Licensee shall ensure that UCLB has the same rights as those set out in this Clause 4.9 in respect of the Licensee’s Affiliates andSub-licensees. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 16Confidential Treatment Requested by MeiraGTx Holdings plc The Licensee shall co-operate with UCLB in good faith to resolve any discrepancies identified during any such inspection and [***], togetherwith interest on late payment as specified in Clause 4.7.4, within [***] following receipt of a copy of the independent chartered accountant’sreport. 4.10Accounting StandardsWhere this Agreement requires a financial calculation to be made or an action to be taken by a party, such calculation will be made or taken inaccordance with the generally accepted accounting principles followed by such party. 5.COMMERCIALISATION 5.1General DiligenceThe Licensee shall use Diligent Efforts to develop and commercially exploit Licensed Products throughout the Territory (including obtainingall and any regulatory approvals which may be required to market and sell the Licensed Products) for the benefit of both Parties. 5.2Competing ActivitiesThe Licensee shall notify UCLB in confidence if it or any of its Affiliates or its Sub-licensees commences any marketing, sale orcommercialisation of any Competing Product or enters into an agreement with any other person with respect to any such activities. 5.3Development PlanThe Licensee’s initial plan for developing and commercialising Licensed Products is set out in Schedule 4 (the “Initial Development Plan”).The Licensee shall provide to UCLB on each anniversary of the Original Commencement Date a written update to the Initial Development Planthat shall: 5.3.1report on all activities conducted under this Agreement by the Licensee and its Affiliates and Sub-licensees since the OriginalCommencement Date or the date of the previous update (as appropriate); 5.3.2(where applicable) set out the milestone events achieved since the Original Commencement Date or the date of the previous update (asappropriate) and the Licensee’s reasonable estimate of the dates for achieving any future milestone events; 5.3.3set out the current and projected activities being taken or planned to be taken by the Licensee and its Affiliates and Sub-licensees tobring Licensed Products to market in the Territory; and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 17Confidential Treatment Requested by MeiraGTx Holdings plc 5.3.4set out the projected sales of Licensed Products (based on the Licensee’s or Sub-licensee’s current forecasts) for each of the next [***]([***]) years following the date of the report.UCLB’s receipt or approval of any update to the Updated Development Plan shall not be taken to waive or qualify the Licensee’s obligationsunder Clause 5.1. 5.4Annual MeetingIn respect of the Licensed Technology, the Licensee will on UCLB’s request meet with UCLB at least once per calendar year, following thesubmission of the update to the relevant Development Plan pursuant to Clause 5.3, to discuss progress with development andcommercialisation of the Licensed Technology. 5.5Development MilestonesIn addition to the Licensee’s obligations under Clause 5.1, the Licensee shall for each Licensed Technology use Diligent Efforts to achieve thedevelopment milestone events specified in Schedule 4 by the dates set out therein. In any instance in which it becomes apparent that aparticular development milestone will not be met due to regulatory, technical, safety or efficacy-related reasons or Force Majeure event(pursuant to Clause 11.1), the Parties in good faith will agree upon an appropriate adjust of such milestone and any subsequent milestones. 5.6Reporting of First Commercial SaleThe Licensee will promptly notify UCLB in writing of the First Commercial Sale of each Licensed Product on a commercial basis in eachcountry within the Territory. 5.7Reporting for Impact Purposes 5.7.1The Licensee acknowledges that part of UCLB’s purpose in licensing the Patents, Know-how and the Materials to the Licensee pursuantthis Agreement is to ensure that the Patents, Know-how and the Materials are made available for use and commercial exploitation withthe intention of benefitting society and the economy. In order to enable UCLB and UCL to monitor the benefit that they are providing,and to enable UCL to demonstrate the impact of its research activities, to society and the economy, the Licensee will upon requestprovide to UCLB [***], a written report describing in reasonable detail how it has used the Patents, Know-how and the Materials and thesocietal and economic benefits generated therefrom. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 18Confidential Treatment Requested by MeiraGTx Holdings plc 5.7.2UCLB shall notify and seek permission from the Licensee in advance, in writing if it wishes to use any written reports received from theLicensee (and the information contained therein) pursuant to Clause 5.7.1 in applications for research or other grant related funding andin submissions to Higher Education funding bodies such as HEFCE and/ or HEIF (or any replacements for either of those entities) andlike entities, supplying a written copy of the application for research or other grant related funding or submission (or the relevantsections thereof). The Licensee will respond to UCLB in writing within [***] ([***]) days of receipt of such written information andsubject to the removal of any confidential information as notified in such written request by the Licensee, UCLB and UCL shall beentitled to submit the approved applications for research or other grant related funding and in submissions to Higher Education fundingbodies such as HEFCE and/ or HEIF (or any replacements for either of those entities) and like entities. 5.8QualityThe Licensee shall ensure that all of the Licensed Products marketed by it and its Affiliates and Sub-licensees are of satisfactory quality andcomply with all applicable laws and regulations in each part of the Territory. 5.9Marking of Licensed ProductsTo the extent permitted under the laws of any country, the Licensee shall mark and cause its Affiliates and Sub-Licensees to mark each LicensedProduct with the number of each issued Patent which applies to the Licensed Product and a statement that such Licensed Products are soldunder licence from UCL Business plc. 5.10Disposals of Licensed Products for FreeNotwithstanding the terms of Clause 5.1, the Licensee shall be entitled to supply a reasonable number of Licensed Products to third parties freeof charge as promotional items for the purpose of establishing a market for the Licensed Products in the relevant country or territory or forresearch, evaluation and testing purposes, or for clinical development, provided that the quantity of Licensed Products supplied for free (or forthe cost of manufacture) in each country or territory is not excessive and is in line with normal industry practice in such country or territory.Any Licensed Products disposed of to third parties in accordance with this Clause 5.10 shall not be taken into account for the purposes ofcalculating Net Sales Value. 5.11Referral to ExpertIf UCLB considers at any time during the period of this Agreement that the Licensee has failed to comply with its obligations under Clause 5.1or 5.3, then the matter shall be referred to an independent expert to answer the following questions: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 19Confidential Treatment Requested by MeiraGTx Holdings plc 5.11.1whether the Licensee has complied with its obligations under Clause 5.1 or 5.3; and if not 5.11.2what specific action the Licensee should have taken and/or now needs to take (“Specific Action”) in order to fulfil such obligations andwithin what period the Specific Action should be taken (“Action Period”).The independent expert shall be appointed in accordance with the provisions of Schedule 2 and his decision shall be final and binding on theParties. 5.12Consequences of Expert’s DecisionIf the expert determines that the Licensee has failed to comply with its obligations under Clause 5.1 or 5.3, and if the Licensee fails to take theSpecific Action within the Action Period, UCLB shall be entitled, by giving, at any time within [***] ([***]) months after the end of thatAction Period, not less than [***] ([***]) months’ notice, to (a) convert the licence granted under Clause 2.1 into a non-exclusive licence or(b) terminate this Agreement. 5.13Use and sale in United StatesThe Licensee agrees that for use and sale of the rights under the Patents in the United States, any products embodying the Patents, or producedthrough use of the Patents, shall be manufactured substantially in the United States, unless a waiver is granted by the NIH. UCLB shall usereasonable efforts, and shall ensure that MEEI use reasonable efforts to assist in the preparation and obtaining of such a waiver, if requested inwriting by Licensee and at Licensee’s reasonable expense. 6.ACCESS TO MEDICINES AND ETHICAL LICENSINGLicensee shall use Diligent Efforts to carry out its activities under this Agreement in a manner which complies with ethical and sociallyresponsible licensing principles and which is designed to fulfil unmet needs in developing countries. 7.COMPLIANCE WITH LAWS 7.1General Compliance with LawsThe Licensee will at all times (and will ensure its Affiliates and Sub-licensees) comply with all legislation, rules, regulations and statutoryrequirements applying to and obtain any consents necessary for its use of the Patents, the Know-how and the Materials, the development,manufacture, and sale of Licensed Products in any country or territory. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 20Confidential Treatment Requested by MeiraGTx Holdings plc 7.2Bribery ActThe Licensee shall (and shall procure that any persons associated with it engaged in the performance of this Agreement including its Affiliatesand Sub-licensees shall): 7.2.1comply with all applicable laws and codes of practice relating to anti-bribery and anti-corruption including the Bribery Act 2010 andwithout prejudice to the foregoing generality, shall not engage in any activity, practice or conduct which would constitute an offenceunder sections 1, 2 or 6 of the Bribery Act 2010 or do or omit to do any act that will cause or lead UCLB to be in breach of the BriberyAct 2010; 7.2.2comply with UCLB’s ethics, anti-bribery and anti-corruption policies as notified to the Licensee from time to time and have, maintain inplace and enforce throughout the term of this Agreement adequate procedures to ensure compliance with Clause 7.2.1; and 7.2.3promptly report to UCLB any request or demand for any undue financial or other advantage of any kind received in connection with theperformance of this Agreement.For the purpose of this Clause 7.2, the meaning of adequate procedures and whether a person is associated with another person shall bedetermined in accordance with the Bribery Act 2010 (and any guidance issued under section 9 of that Act). Breach of this Clause 7.2 shall bedeemed a material breach of this Agreement entitling UCLB to terminate under Clause 10.3.1. 7.3Export Control RegulationsThe Licensee shall ensure that, in using the Patents, Know-how or Materials and in selling Licensed Products, it and its Affiliates, employees,sub-contractors and Sub-licensees comply fully with any United Nations trade sanctions or EU or UK legislation or regulation, from time totime in force, which impose arms embargoes or control the export of goods, technology or software, including weapons of mass destruction andarms, military, paramilitary and security equipment and dual-use items (items designed for civil use but which can be used for militarypurposes) and certain drugs and chemicals. 8.INTELLECTUAL PROPERTY 8.1Obtain and Maintain the Patents 8.1.1The Licensee shall be responsible for the drafting, filing, prosecution and maintenance of all of the Patents at the Licensee’s cost andexpense. Subject to resource availability, UCLB shall use commercially reasonable efforts to provide such assistance as the Licenseemay request to prosecute and maintain the Patents[***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 21Confidential Treatment Requested by MeiraGTx Holdings plc 8.1.2The Patents will be filed, prosecuted and maintained in the countries and territories where Licensee normally files its patent applicationsand patents for other gene therapy products. The Licensee shall notify UCLB of any decisions as to which (if any) additional countriesto file and maintain Patents in. 8.1.3The Licensee shall consult with UCLB in relation to all material changes to the patent claims or specifications that would have theeffect of reducing or limiting the scope of the Patents, and not make any such changes without the prior written consent of UCLB. Suchconsent shall not be unreasonably withheld or delayed provided that UCLB has been given as much notice as is practicable, and in anyevent no less than [***] days’ notice (or such shorter period for response dictated by the relevant patent office) of such proposedchanges, and has been given an opportunity to file divisionals, continuations and/or such other types of protection to cover any claimsor subject matter that the Licensee intends to remove from the scope of the Patents. If UCLB fails to respond before the end of the [***]day period (or such shorter period for response dictated by the relevant patent office), the Licensee may proceed with the proposedchanges to the patent claims or specifications. The Licensee will ensure that UCLB receives copies of all correspondence to and fromPatent Offices in respect of the Patents, including copies of all documents generated in or with such correspondence, and shall be givenreasonable notice (or such shorter period for response dictated by the relevant patent office) of and the opportunity to participate in anyconference calls or meetings with the Licensee’s patent attorneys in relation to the drafting, filing, prosecution and maintenance of thePatents, so that UCLB may be continuously informed of progress with the drafting, filing, prosecution and maintenance of the Patents.Such involvement of UCLB under this Clause 8.1.3 shall be at UCLB’s cost and expense. 8.1.4If the Licensee wishes to abandon any application contained with the Patents or not to maintain any such Patent, it shall give [***]([***]) months’ prior written notice to UCLB and on the expiry of such notice period the licences of the relevant Patents granted to theLicensee under this Agreement shall cease. 8.1.5In the event that any of rights granted hereunder become non-exclusive, responsibility for the drafting, filing, prosecution andmaintenance of all of the Patents shall revert to UCLB. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 22Confidential Treatment Requested by MeiraGTx Holdings plc 8.2Infringement of the Patents, the Know-how and/or the Materials 8.2.1The Licensee and UCLB shall promptly give to each other (and MEEI) written notice if it becomes aware of any infringement orpotential infringement of any of the Patents or any unauthorised use of the Know-how or the Materials or any challenge to the validityor ownership of the Patents, the Know-how or the Materials and the Licensee and UCLB shall consult with each other (and MEEI) todecide the best way to respond to such infringement, unauthorised use or challenge. 8.2.2The Licensee shall have the primary obligation and right to take action against any third party alleged to be infringing the Patents ormaking unauthorised use of the Know-how or the Materials and to defend the Patents against challenges to validity or ownership at itssole expense, provided that: (a)the Licensee, UCLB and MEEI, in cooperation with the NIH, shall use their commercially reasonable efforts to eliminate theinfringement without litigation. If the efforts of the Licensee, UCLB and MEEI are not successful in eliminating theinfringement within [***] ([***]) days after the infringer has been formally notified of the infringement by the Licensee, theLicensee shall have the right after consulting with MEEI, NIH and UCLB, to commence suit on its own account; (b)UCLB shall procure that MEEI shall and that MEEI shall procure that the NIH shall on the Licensee’s or UCLB’s requestcooperate with the Licensee in such action [***]; (c)UCLB shall on the Licensee’s request cooperate with the Licensee in such action and [***]; (d)the Licensee shall be solely responsible for the conduct of the action or for settlement thereof and shall be entitled to alldamages received from such action, subject to Clause 8.2.4; and (e)if the Licensee is unsuccessful in persuading the alleged infringer to desist within [***] ([***]) months of the Licensee firstbecoming aware of any potential infringement of the Patents for any Licensed Technology or fails without a commerciallyreasonable basis (relative to the Licensed Technology or other technology licenced to Licensee by UCLB) to initiate aninfringement action within [***] ([***]) months of becoming aware of such infringement, UCLB shall have the right, at its solediscretion, to prosecute such infringement under its sole control and [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 23Confidential Treatment Requested by MeiraGTx Holdings plc 8.2.3Before starting or defending or settling any legal action under Clause 8.2.2, the Licensee shall consult with UCLB as to the advisabilityof the action or defence or settlement, its effect on the good name of UCLB, the public interest, and how the action or defence should beconducted. 8.2.4The Licensee shall [***] in such action or defence. 8.2.5UCLB shall if reasonably requested by the Licensee agree to be joined in any suit to enforce such rights or will take such action in itsown name [***] and shall have the right to be separately represented by its own counsel [***]. Notwithstanding the foregoing, [***]. 8.2.6UCLB shall procure that MEEI shall and that MEEI shall procure that the NIH shall if reasonably requested by UCLB or the Licenseeagree to be joined in any suit to enforce such rights or will take such action in its or their own name(s) [***]. Notwithstanding theforegoing, [***]. 8.2.7If within [***] ([***]) months of the Licensee or UCLB giving to the other and MEEI written notice or receiving written notice from theother or MEEI of any potential infringement of the Patents, the Licensee and UCLB are both unsuccessful in persuading the allegedinfringer to desist or fail to initiate an infringement action, MEEI shall have the right, at its sole discretion, to prosecute suchinfringement under its sole control and [***]. 8.3Infringement of Third Party Rights 8.3.1If any warning letter or other notice of infringement is received by the Licensee or UCLB, or legal suit or other action is brought againstthe Licensee or UCLB, alleging infringement of third party rights in the manufacture, use or sale of any Licensed Product or use of anyPatents, Know-how or Materials, that Party shall (in the case of UCLB) promptly provide full details to the Licensee and (in the case ofthe Licensee) promptly provide full details to UCLB, and the Licensee and UCLB shall discuss the best way to respond with MEEI. 8.3.2The Licensee shall have the right but not the obligation to defend such suit to the extent it relates to Licensee’s or its Affiliates’ orSub-licensee’s activities and shall have the right to settle with such third party, provided that [***]. In the event that the Licensee,Affiliates or Sub-licensees do not take forward an action, UCLB shall have the right, at its sole discretion, to defend such suit under itssole control and [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 24Confidential Treatment Requested by MeiraGTx Holdings plc 9.WARRANTIES AND LIABILITY 9.1Warranties by UCLBUCLB warrants as of the Original Commencement Date and undertakes as follows to its reasonable knowledge and without having undertakenany due and careful enquires whether specific or general in nature: 9.1.1It, MEEI and NIH are the owners of the Patents; 9.1.2it has the authority to grant the licences under this Agreement; and. 9.1.3so far as it is aware (having made no enquiry of any third parties or conducted any freedom to operate searches), use and exploitation ofthe Patents will not infringe the intellectual property rights of any third party. UCLBwarrants and undertakes: 9.1.4it has full power and authority to enter into and perform this Agreement which, when executed, will constitute valid and legally bindingobligations on UCLB; and 9.2Warranties by the LicenseeThe Licensee warrants as of the Original Commencement Date and undertakes that in respect of the Licensed Technology that: 9.2.1full power and authority to enter into and perform this Agreement, which, when executed, will constitute valid and legally bindingobligations on the Licensee; 9.2.2entry into this Agreement will not result in any breach of, or violation of the terms or provisions of, the constitutional documents of theLicensee or any other agreement or instrument by which it is bound; 9.2.3so far as it is aware (having made no enquiry of any third parties), use and exploitation of the Patents will not infringe the intellectualproperty rights of any third party; 9.2.4neither it nor any of its Affiliates is currently researching, developing, marketing, selling or otherwise commercialising any CompetingProduct (“Competing Activities”), nor has any of them entered into an agreement with any other person with respect to any CompetingActivities; and 9.2.5it shall notify UCLB if it or any of its Affiliates or its Sub-licensees commences any Competing Activities or enters into an agreementwith any other person with respect to any Competing Activities. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 25Confidential Treatment Requested by MeiraGTx Holdings plc 9.3Warranties by MeiraLicensee warrants as of the Original Commencement Date and undertakes that full power and authority to enter into and perform this Agreementwhich, when executed, will constitute valid and legally binding obligations on Licensee. 9.4AcknowledgementsThe Licensee acknowledges that: 9.4.1the inventions claimed in the Patents, and the Know-how and the Materials are at an early stage of development. Accordingly, specificresults cannot be guaranteed and any results, materials, information or other items (together “Delivered Items”) provided under thisAgreement are provided “as is” and without any express or implied warranties, representations or undertakings. As examples, butwithout limiting the foregoing, UCLB does not give any warranty that Delivered Items are of merchantable or satisfactory quality, are fitfor any particular purpose, comply with any sample or description, or are viable, uncontaminated, safe or non-toxic. 9.4.2UCLB has not performed any searches or investigations into the existence of any third party rights that may affect any of the Patents,Know-how or Materials or the use and exploitation of any of the Patents, Know-how or Materials. 9.5No Other Warranties 9.5.1Each of the Parties acknowledges that, in entering into this Agreement, it does not do so in reliance on any representation, warranty orother provision except as expressly provided in this Agreement, and any conditions, warranties or other terms implied by statute orcommon law are excluded from this Agreement to the fullest extent permitted by law. 9.5.2Without limiting the scope of Clause 9.5.1, UCLB does not make any representation nor give any warranty or undertaking: (a)express or implied, including, without limitation, any implied warranties of merchantability or of fitness for a particularpurpose with respect to any Patent, trademark, software, non-public or other information, or tangible research property, licensedor otherwise provided to the Licensee hereunder and hereby disclaims the same; (b)as to the efficacy or usefulness of the Patents, Know-how or Materials; or Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 26Confidential Treatment Requested by MeiraGTx Holdings plc (c)whatsoever with regard to the scope of any of the Patents or that any of the Patents is or will be valid or (in the case of anapplication) will proceed to grantor that such Patents may be exploited by the Licensee, Affiliate or Sub-licensee withoutinfringing other patents; or (d)that the Materials or the method used in making or using the Materials are free from liability for patent infringement; or (e)that the use of any of the Patents, Know-how or Materials, Licensed Technology, the manufacture, sale or use of the LicensedProducts, or the exercise of any of the rights granted under this Agreement will not infringe any intellectual property or otherrights of any other person; or (f)that the Know-how or any other information communicated by UCLB to the Licensee under or in connection with thisAgreement will produce Licensed Products of satisfactory quality or fit for the purpose for which the Licensee intended or thatany product will not have any defect, latent or otherwise, and whether or not discoverable by inspection; or (g)as imposing any obligation on UCLB to bring or prosecute actions or proceedings against third parties for infringement or todefend any action or proceedings for revocation of any of the Patents; or (h)as imposing any liability on UCLB in the event that any third party supplies Licensed Products to customers located in theTerritory; or (i)that there will be no similar or competitive products or services manufactured, used, sold or supplied by any third party in theTerritory. 9.6Responsibility for Development of Licensed ProductsThe Licensee shall be exclusively responsible for its and its Affiliates’ and Sub-licensees’ use of the Patents, Know-how and Materials, thetechnical and commercial development and manufacture of Licensed Products and for incorporating any modifications or developments theretothat may be necessary or desirable, for all Licensed Products sold or supplied, notwithstanding any consultancy services or other contributionsthat UCLB and/or UCL may provide in connection with such activities. 9.7IndemnityThe Licensee shall indemnify each of UCLB and UCL, and each of their respective officers, directors, Council members, employees andrepresentatives (together, the “Indemnitees”) against all third party Claims that may be asserted against or suffered by any of the Indemniteesand which relate to: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 27Confidential Treatment Requested by MeiraGTx Holdings plc 9.7.1the use by the Licensee or any of its Affiliates or Sub-licensees of any of the Patents, Know-how or Materials; or 9.7.2use of the Licensed Technology by or on behalf of the Licensee or any of its Affiliates or Sub-licensees; or 9.7.3the development, manufacture, use, marketing or sale of, or any other dealing in, any of the Licensed Products, by or on behalf of theLicensee or any of its Affiliates or Sub-licensees, or subsequently by any customer or any other person, including claims based onproduct liability laws.The indemnity given by the Licensee to each Indemnitee under this Clause 9.7 will not apply to any third party Claim to the extent that it isattributable to the negligence, gross negligence, reckless misconduct or intentional misconduct of any Indemnitee.Licensee shall indemnify, defend and hold harmless MEEI and its trustees, officers, medical and professional staff, employees and agents andtheir respective successors, heirs and assigns (the “MEEI lndemnitees”), against any liability, damage, loss or expense (including reasonableattorney’s fees and expenses of litigation) incurred by or imposed upon the MEEI lndemnitees or any one of them in connection with any thirdparty claims, suits, actions, demands or judgments: arising out of any theory of product liability (including, but not limited to, actions in theform of contract, tort, warranty, or strict liability) concerning any product, process or service made, used or sold or any right or license grantedunder this Agreement.Licensee’s indemnification of the MEEI Indemnitees under this Clause 9.7 shall not apply to liability, damage, loss or expense to the extentthat it is directly attributable to the negligent activities, reckless misconduct or intentional misconduct of the MEEI lndemnitees.Licensee agrees, at its own expense, to provide attorneys reasonably acceptable to MEEI to defend against any actions brought or filed againstany party indemnified hereunder with respect to the subject of indemnity contained herein, whether or not such actions are rightfully brought.Licensee’s indemnification of the MEEI Indemnitees under this Clause 9.7 shall survive expiration or termination of this Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 28Confidential Treatment Requested by MeiraGTx Holdings plc 9.8Limitations of Liability 9.8.1To the extent that UCLB or any of its Affiliates has any liability in contract, tort, or otherwise under or in connection with thisAgreement, including any liability for breach of warranty, their liability shall be limited in accordance with the following provisions ofthis Clause 9.8. 9.8.2The aggregate liability of UCLB and any of its Affiliates shall be limited to the total income that UCLB has received from the Licenseepursuant to this Agreement (but excluding any other costs or expenses associated with drafting, filing, prosecuting, maintaining ordefending any Patents or providing any assistance to the Licensee) during the period of [***] ([***]) years preceding the date on whichthe liability arises, or fifty thousand pounds (£50,000) sterling, whichever is the higher. 9.8.3The liability of the Licensee to UCLB shall be limited to the limit of its insurance as set out in Clause 9.9.1, except that in the case ofproduct liability, the liability of the Licensee under this Agreement shall be unlimited. 9.8.4In no circumstances shall any Party or any Indemnitee be liable for any loss, damage, costs or expenses of any nature that is (a) of anindirect, special or consequential nature or (b) any loss of profits (whether direct or indirect), revenue, business opportunity or goodwill,which arises directly or indirectly from that Party’s breach or nonperformance of this Agreement, or negligence in the performance ofthis Agreement or from any liability arising in any other way out of the subject matter of this Agreement even if the Party bringing theclaim has advised any other Party or the relevant Indemnitee of the possibility of those losses arising, or if such losses were within thecontemplation of the Parties or the Indemnitee. 9.8.5Nothing in this Agreement excludes any Party’s liability to the extent that it may not be so excluded under applicable law, includingany such liability for death or personal injury caused by that Party’s negligence, or liability for fraud or fraudulent misrepresentation. 9.9Insurance 9.9.1The Licensee shall take out with a reputable insurance company and maintain at all times during the term of this Agreement public andproduct liability and professional indemnity insurance including against all loss of and damage to property (whether real, personal orintellectual) and injury to persons including death arising out of or in connection with this Agreement and the Licensee’s and itsAffiliates’ and Sub-licensees’ use of the Patents, Know-how or Materials and use, sale of or any other Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 29Confidential Treatment Requested by MeiraGTx Holdings plc dealing in any of the Licensed Products. Such insurances may be limited in respect of one claim provided that such limit must be at least[***] pounds (£[***]) sterling, unless the Licensee commences any business in manufacturing, distribution, supply or otherwise makeavailable to the public any products, in which case such limit must be at least [***] pounds (£[***]) sterling. Such insurance shallcontinue to be maintained for a further [***] years from the end of this Agreement. 9.9.2The Licensee will produce to UCLB at all times upon demand proof that the insurance cover required pursuant to Clause 9.9.1 is in forceand evidence that all premiums have been paid up to date. If UCLB becomes aware that the Licensee has failed to maintain theinsurance required pursuant to Clause 9.9.1, UCLB may effect such insurance and the Licensee will reimburse UCLB for the reasonablecost of effecting and maintaining such insurance on demand. 9.9.3Beginning no later than the time any Licensed Products are being commercially distributed or sold (other than for the purpose ofobtaining regulatory approvals) by Licensee or by a Sub-licensee, affiliate or agent of Licensee, Licensee shall, at its own cost andexpense procure and maintain Commercial General Liability (CGL) insurance or other coverage acceptable to MEEI in amounts not lessthan [***] and naming the MEEI lndemnitees as additional insureds. Such CGL or other insurance shall provide: (a)Product liability coverage, and (b)Contractual liability coverage for Licensee’s indemnification under Clause 9.7 of this Agreement. 9.9.4If Licensee elects to self-insure all or parts of the limits described above (including deductibles or retentions which are in excess of$[***] annual aggregate) such self-insurance program must be acceptable to MEEI and CRICO. The minimum amount of insurancecoverage required under this Clause 9.9.4 shall not be construed to create a limit of Licensee’s liability with respect to itsindemnification under Clause 10.7 of this Agreement. Licensee shall provide MEEI with written evidence of such insurance uponrequest of MEEI. Licensee shall provide MEEI with written notice at least [***] ([***]) days prior to the cancellation, non-renewal ormaterial change in such insurance, if (a)Licensee does not obtain replacement insurance providing comparable coverage within such [***] ([***]) day period, MEEIshall have the right to terminate this Agreement effective at the end of such [***] ([***]) days without notice of any additionalwaiting period. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 30Confidential Treatment Requested by MeiraGTx Holdings plc 9.9.5Licensee shall maintain such CGL or other insurance during: (a)the period that any Licensed Products are being commercially distributed or sold (other than for the purpose of obtainingregulatory approvals) by Licensee or by a Sub-licensee, affiliate or agent of Licensee; (b)reasonable period after the period referred to in Clause 9.9.5(a) above, which in no event shall be less than [***] ([***]) years. 9.9.6This Clause 9.9 shall survive expiration or termination of this Agreement. 10.DURATION AND TERMINATION 10.1Commencement and ExpiryThis Agreement shall commence as of the Commencement Date and, unless terminated earlier in accordance with this Clause 10, the licencesgranted hereunder shall continue in force on a country by country basis until the later of the last payment obligation of Licensee expires underthis Agreement. Upon such expiry, Licensee’s licenses under this Agreement shall become full-paid, perpetual and irrevocable. 10.2Termination of the Original AgreementUpon the Commencement Date, pursuant to Amendment No. 4 to the Original Agreement, the Original Agreement shall be deemed to haveterminated. 10.3Early TerminationEach Party (the “Terminating Party”) may terminate this Agreement at any time by notice in writing to the other Parties (“Other Parties”),such notice to take effect as specified in the notice: 10.3.1If, in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, is in material breach of this Agreementand, in the case of a breach capable of remedy within thirty (30) days, the breach is not remedied within thirty (30) days of the OtherParties receiving notice specifying the breach and requiring its remedy or where the breach relates to non-payment of an undisputed sumdue under this Agreement, the sum is not paid in full within fourteen (14) days following the Other Party receiving notice specifying thenonpayment and requiring payment in full; provided however, that in respect on breaches not relating to non-payment, if such breach iscapable of being cured but cannot be cured within such thirty (30) day period and the Other Party initiates actions to cure such breachwithin such period and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 31Confidential Treatment Requested by MeiraGTx Holdings plc thereafter diligently pursues such actions, the Other Party shall have such additional period as is reasonable under the circumstances tocure such breach; it being understood that no such extension shall apply with respect to any undisputed payment obligations or extendbeyond six (6) months from the end of such thirty (30) day period. In the event there is a genuine dispute between the Parties withrespect to any alleged breach hereunder, no purported termination of this Agreement pursuant to this Clause 10.3.1 shall take effectwhile the Parties are actively working to resolve such dispute; or 10.3.2if: (a)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, becomes insolvent or unable topay its debts as and when they become due; (b)an order is made or a resolution is passed for the winding up of in the case of UCLB, either of the Other Parties, or in the case ofthe Licensee or Meira, UCLB (other than voluntarily for the purpose of solvent amalgamation or reconstruction); (c)a liquidator, administrator, administrative receiver, receiver or trustee is appointed in respect of the whole or any part of, in thecase of UCLB, either of the Other Parties’, or in the case of the Licensee or Meira, UCLB’s, assets or business; (d)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, makes any composition with itscreditors; (e)in the case of UCLB, either of the Other Parties, or in the case of the Licensee or Meira, UCLB, ceases to continue its business;or (f)any event analogous to the events referred to in paragraphs (a) to (e) above occurs in any other jurisdiction. 10.4UCLB may terminate this Agreement by giving written notice to the Licensee and Meira, such termination to take effect forthwith or asotherwise stated in the notice: 10.4.1if there is any change of Control of the Licensee involving the categories of persons or Affiliates of persons prohibited by Clause 2.3; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 32Confidential Treatment Requested by MeiraGTx Holdings plc 10.4.2the Licensee is in persistent breach of the Agreement at least [***] ([***]) times in a calendar year, wherein the nature of each suchbreach is the same in each instance, and where the Parties have failed to agree on a mechanism to remedy the persistent nature of suchbreaches within a reasonable period following UCLB notifying Licensee of the persistent breaches and requesting that the Licenseeenters into discussions with UCLB as to mechanisms for remedying the persistent breaches or if the Parties have agreed to a mechanismto remedy the persistence of such breach by that mechanism if not fully complied with by the Licensee; or 10.4.3if the Licensee shall enter into any sub-licence with any of the categories of persons or Affiliates of persons prohibited by Clause 2.3which may, adversely affect UCL’s and/or UCLB’s reputation; 10.4.4subject to Clause 5.5, if the Licensee fails to achieve any of the milestone events described in Schedule 4 provided that if achievementof any of the milestone events should be compromised due to technical, legal or regulatory issues, the Parties shall first meet and UCLBwill work with the Licensee to manage the delivery schedule and provided that the Licensee is using Diligent Efforts to correct theissues, the applicable deadline in Schedule 4 shall be extended by six (6) months or such other time period as shall be agreed betweenthe Parties in writing after which if the Licensee has not achieved the milestone UCLB shall be entitled, subject to Clauses 5.11 and5.12, to terminate this Agreement by giving written notice to the Licensee. 10.5A Party’s right of termination under this Agreement, and the exercise of any such right, shall be without prejudice to any other right or remedy(including any right to claim damages) that such Party may have in the event of a breach of contract or other default by any other Party. 10.6Consequences of Termination 10.6.1Upon expiry of the period of this Agreement, and subject to all royalties and any other sums due to UCLB under this Agreement havingbeen duly paid, the Licensee shall have a fully paid up licence to the Patents, the Know-how and the Materials of the same scope as setforth in Clause 2.1 without any further obligation to pay any further sums to UCLB under Clause 4. Notwithstanding the foregoing theLicensee acknowledges that once each Patent expires or is abandoned or withdrawn or allowed to lapse in any country or territory, thirdparties in that country or territory will be entitled to use the inventions claimed in the Patent and that accordingly the licence granted tothe Licensee under Clause 2.1 will no longer be exclusive in that country or territory. 10.6.2Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 33Confidential Treatment Requested by MeiraGTx Holdings plc (a)the Licensee and its Affiliates and Sub-licensees shall be entitled to sell, use or otherwise dispose of (subject to payment ofroyalties under Clause 4) any unsold or unused stocks of the Licensed Products for a period of [***] ([***]) months followingthe date of termination; (b)subject to paragraph (a) above, any license that has not become fully paid-up in accordance with Clause 10.1 shall terminateand the Licensee and its Affiliates (and subject to Clause 2.3, its Sub-licensees) shall no longer be licensed to use or otherwiseexploit the Patents and/or the Know-how and/or the Materials, in so far and for as long as any of the Patents remains in forceand the Know-how remains confidential; (c)the Licensee shall consent to the cancellation of any formal licence granted to it, or of any registration of it in any register, inrelation to any of the Patents; (d)the Licensee will, promptly on UCLB’s request, provide (and will ensure that its patent agents provide) to UCLB allinformation, documentation and assistance (including executing documents) which are in the possession of Licensee or itspatent agents relating to the Patents and which UCLB may reasonably require to enable it to continue with the drafting, filing,prosecution and maintenance of the Patents; (e)except as set out in Clause 2.3, all sub-licences of the Patents and/or the Know-how and/or the Materials granted by theLicensee pursuant to this Agreement will automatically terminate; (f)UCLB shall, upon the written request of either of the other Parties, and each of the Licensee and Meira shall, upon the writtenrequest of UCLB, return or destroy any documents or other materials that are in its or its Affiliates possession or under its ortheir control and that contain the requesting Party’s Confidential Information. 10.6.3Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4, in theevent that UCLB would be unable, absent a licence from the Licensee, to use or permit others to use or to exploit or permit others toexploit the relevant Licensed Technology without infringing intellectual property rights in any invention developed by the Licensee,whether solely or jointly with others (“Blocking Invention”), the Licensee shall be deemed to have granted UCLB the irrevocablenon-exclusive right to use, exploit and permit others to use and exploit the Blocking Invention only in conjunction with the relevantLicensed Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 34Confidential Treatment Requested by MeiraGTx Holdings plc Technology. The Licensee shall at the request of UCLB provide (and will ensure that its patent agents provide) to UCLB allinformation, documentation and assistance (including executing documents) which UCLB may reasonably require to enable it tocontinue with the drafting, filing, prosecution and maintenance of the Patents licensed under this Agreement; 10.7Upon termination of this Agreement by UCLB under Clause 10.3.1 (for Licensee’s uncured material breach) or under Clause 10.4, the Licenseeshall, to the extent it is able to do so without being in breach of any obligation owed to a third party, disclose to UCLB full details of any andall Intellectual Property generated at any time by or on behalf of the Licensee as a result of the exercise of the Licensee’s rights under thisAgreement (“Licensee IP”) and, upon UCLB’s written request within [***] ([***]) days following such disclosure, negotiate in good faith toagree the terms of an exclusive or non-exclusive licence to UCLB (as UCLB may request) under the Licensee IP. If the Parties fail to agree theterms of such a licence within [***] days following commencement of such negotiation, despite negotiating in good faith, UCLB’s rights underthis Clause shall lapse. If Licensee may terminate this Agreement under Clause 10.3.1 (for UCLB or its Affiliates uncured material breach), thenLicensee may elect, in lieu of terminating the entire Agreement, to have all licenses granted to the Licensee under this Agreement continue inforce, subject to Licensee’s fulfilment of [***] percent ([***]%) of its payment obligations under Clause 4 after what would have been theeffective date of such termination. 10.8Upon termination of this Agreement for any reason, the provisions of Clauses 1, 2.3, 2.5, 3.1 to 3.5, 4 (in respect of amounts paid and payable toUCLB in respect of the period up to and including the date of termination), 5.7, 7, 9, 10.7, 10.8, 10.8 and 11 of this Agreement shall remain inforce. 11.GENERAL 11.1Force Majeure 11.1.1Any delays in or failure of performance by a Party under this Agreement will not be considered a breach of this Agreement and if and tothe extent that such delay or failure is caused by occurrences beyond the reasonable control of that Party including acts of God; acts,regulations and laws of any government; strikes or other concerted acts of workers; fire; floods; explosions; riots; wars; rebellion; andsabotage; and any time for performance hereunder will be extended by the actual time of delay caused by any such occurrence. 11.1.2If (a) UCLB or (b) the Licensee or Meira is prevented from carrying out its obligations: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 35Confidential Treatment Requested by MeiraGTx Holdings plc (a)under this Agreement for a continuous period of [***] ([***]) months, the Licensee (in the case of (a)) or UCLB (in the case of (b)), mayterminate this Agreement on giving [***] ([***]) days prior written notice provided always that at the date upon which terminationbecomes effective the Party which was prevented from carrying out its obligations under this Agreement remains so prevented. 11.2AmendmentThis Agreement may only be amended in writing signed by duly authorised representatives of the Parties. 11.3Assignment and Third Party Rights 11.3.1Subject to Clause 11.3.3, the Licensee shall not assign, mortgage, charge or otherwise transfer any rights or obligations under thisAgreement, nor any of the Patents, Know-how or Materials, without the prior written consent of UCLB. 11.3.2UCLB may assign all its rights and obligations under this Agreement together with its rights in the Patents, Know-how and Materials toany third party. 11.3.3The Licensee, subject to obtaining the consent of UCLB which shall not be unreasonably withheld or delayed (except in relation tothose categories of persons or Affiliates of persons prohibited by Clause 2.3), may assign all its rights and obligations under thisAgreement together with its rights in the Patents, Know-how and Materials to any third party to which it transfers all or substantially allof its assets or business, provided that the assignee undertakes to UCLB to be bound by and perform the obligations of the assignorunder this Agreement. However, the Licensee shall not have such a right to assign this Agreement if it is insolvent. 11.3.4Meira shall not assign, mortgage, charge or otherwise transfer any rights or obligations under this Agreement without the prior writtenconsent of UCLB. 11.4WaiverAny waiver given under or in relation to this Agreement shall be in writing and signed by or on behalf of the relevant Party. No failure or delayon the part of a Party to exercise any right or remedy under this Agreement shall be construed or operate as a waiver thereof, nor shall any singleor partial exercise of any right or remedy preclude the further exercise of such right or remedy. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 36Confidential Treatment Requested by MeiraGTx Holdings plc 11.5Invalid ClausesIf any provision or part of this Agreement is held to be invalid, amendments to this Agreement may be made by the addition or deletion ofwording as appropriate to remove the invalid part or provision but otherwise retain the provision and the other provisions of this Agreement tothe maximum extent permissible under applicable law. 11.6No AgencyNo Party shall act or describe itself as the agent of the other, nor shall it make or represent that it has authority to make any commitments on theother’s behalf. 11.7InterpretationIn this Agreement: 11.7.1the headings are used for convenience only and shall not affect its interpretation; references to persons shall include incorporated andunincorporated persons; references to the singular include the plural and vice versa; and references to the masculine include thefeminine; 11.7.2references to Clauses and Schedules mean clauses of, and schedules to, this Agreement; 11.7.3references in this Agreement to termination shall include termination by expiry; 11.7.4where the word “including” is used it shall be understood as meaning “including without limitation”; 11.7.5any reference to any English law term for any action, remedy, method or judicial proceeding, legal document, legal status, court, officialor any legal concept or thing shall in respect of any jurisdiction other than England be deemed to include what most nearlyapproximates in that jurisdiction to the English law term; 11.7.6where there is any conflict or inconsistency between the main body of this Agreement and any of the schedules, then the main body ofthe Agreement shall prevail; 11.7.7time shall be of the essence in relation to the performance of Meira’s and the Licensee’s obligations under this Agreement; and 11.7.8any reference to the sale of a Licensed Product by the Licensee or its Affiliates or Sub-licensees will be taken to include any supply orother disposal of Licensed Products, and the term sold shall be construed accordingly. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 37Confidential Treatment Requested by MeiraGTx Holdings plc 11.8Notices. Addresses for Service 11.8.1Any notice to be given under this Agreement shall be in English, in writing and shall be delivered by first class recorded delivery mail(if sent to an inland address) or by international courier (if sent to an address outside of the United Kingdom), to the address of therelevant Party set out at the head of this Agreement, or such other address as that Party may from time to time notify to the other Partiesin accordance with this Clause 11.8. 11.8.2Notices sent as above shall be deemed to have been received [***] ([***]) working day after the day of posting in the case of deliveryinland first class recorded delivery mail, or [***] ([***]) working days after the date of collection by the international courier. 11.9Law and JurisdictionThe validity, construction and performance of this Agreement, and any contractual and non-contractual claims arising hereunder, shall begoverned by English law and shall be subject to the exclusive jurisdiction of the English courts to which the Parties hereby submit, except thata Party may seek an interim injunction (or an equivalent remedy) in any court of competent jurisdiction. 11.10Entire AgreementThis Agreement, including its Schedules, sets out the entire agreement between the Parties relating to its subject matter and supersedes all priororal or written agreements, arrangements or understandings between them relating to such subject matter including, without limitation, the partsof the Original Agreement that pertains to the Specified Technology [***]. Subject to Clause 9.8.5, the Parties acknowledge that they are notrelying on any representation, agreement, term or condition which is not set out in this Agreement. 11.11Third PartiesExcept for the rights of UCL, MEEI and NIH as provided in Clause 2.4, the rights of the Indemnitees and MEEI Indemnitees as provided inClause 9.7, the limitations of liability afforded to the Indemnitees pursuant to Clause 9.8, the rights of MEEI under Clause 9.9 and the rights ofMEEI and NIH under Clause 8, who may in their own right enforce and rely on the provisions of those Clauses, this Agreement does not createany right enforceable by any person who is not a party to it (“Third Party”) under the Contracts (Rights of Third Parties) Act 1999, but thisClause 11.11 does not affect any right or remedy of a Third Party which exists or is available apart from that Act. The Parties may amend, renew,terminate or otherwise vary all or any of the provisions of this Agreement, including Clauses 2.4, 8, 9.7, and 9.8, without the consent of MEEI,NIH and/or the MEEI lndemnitees Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 38Confidential Treatment Requested by MeiraGTx Holdings plc 11.12Non-use of Names; Announcements 11.12.1The Licensee shall not use, and shall ensure that its Affiliates and Sub-licensees do not use, the name, any adaptation of the name,any logo, trademark or other device of UCLB, nor of the inventors named on the Patents nor the Principal Investigators in anyadvertising, promotional or sales materials without prior written consent obtained from UCLB in each case, except that the Licenseemay state that it is licensed by UCLB under the Patents. 11.12.2Except as permitted under Clauses 3.3.1 and 5.7, no Party shall make any press or other public announcement concerning any aspectof this Agreement, or make any use of the name or trademarks of any other Party in connection with or in consequence of thisAgreement, without the prior written consent of the relevant other Party. 11.13EscalationIf the Licensee or Meira on the one hand, and UCLB on the other, are unable to reach agreement on any issue concerning this Agreement or theProject within [***] days after one either has notified the other of that issue, they will refer the matter to the [***] in the case of UCLB, and tothe [***] in the case of the Licensee and Meira in an attempt to resolve the issue within the time specified elsewhere in this Agreement in thecase of other disputes. Any Party may bring proceedings in a court of competent jurisdiction if the matter has not been resolved within thatprescribed period, and any Party may apply to the court for an injunction, whether or not any issue has been escalated under this Clause 11.13.[Signature Page Follows] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 39Confidential Treatment Requested by MeiraGTx Holdings plc EXECUTED on the date set out at the head of this Agreement. For and on behalf of UCL Business PLC For and on behalf of MEIRAGTX UK II LIMITED/s/ [***] /s/ Zandy ForbesSigned Signed[***] Zandy ForbesPrint name Print name[***] CEOTitle Title5 February 2019 2.5.19Date Date For and on behalf of MEIRAGTX LIMITED/s/ Zandy ForbesSignedZandy ForbesPrint nameCEOTitle2.5.19Date Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 40Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 1LICENSED TECHNOLOGYPart A: The Patents[***]Part B: The Know-how[***]Part C: The Materials[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 41Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 2APPOINTMENT OF EXPERTFor the purposes of this Schedule 2 only, the “Parties” shall mean the Licensee and UCLB. If either Party wishes to appoint an independent expert (the“Expert”) to determine any matter pursuant to any Clause of this Agreement, the following procedures will apply: 1.The Party wishing to appoint the Expert (the “Appointing Party”) will serve a written notice on the other Party (the “Responding Party”). Thewritten notice will specify the Clause pursuant to which the appointment is to be made and will contain reasonable details of the matter(s)which the Appointing Party wishes to refer to the Expert for determination. 2.The Parties shall within [***] ([***]) days following the date of the Appointing Party’s written notice use all reasonable efforts to agree who isto be appointed as the Expert to determine the relevant matter(s). If the Parties are unable to agree upon the identity of the Expert within thattimescale, the Expert shall be appointed by the President (for the time being) of the Licensing Executives Society Britain and Ireland uponwritten request of either Party. 3.Each Party will within [***] ([***]) days following appointment of the Expert, prepare and submit to the Expert and the other Party a detailedwritten statement setting out its position on the matter(s) in question and including any proposals which it may wish to make for settlement orresolution of the relevant matter. 4.Each Party will have [***] ([***]) days following receipt of the other Party’s written statement to respond in writing thereto. Any such responsewill be submitted to the other Party and the Expert. 5.The Expert will if he/ she deems appropriate be entitled to seek clarification from the Parties as to any of the statements or proposals made byeither Party in their written statement or responses. Each Party will on request make available all information in its possession and shall givesuch assistance to the Expert as may be reasonably necessary to permit the Expert to make his/ her determination. 6.The Expert will issue his/ her decision on the matter(s) referred to him/ her in writing as soon as reasonably possible, but at latest within [***]([***]) months following the date of his/ her appointment. The Expert’s decision shall (except in the case of manifest error) be final and bindingon the Parties. 7.The Expert will at all times act as an independent and impartial expert and not as an arbitrator. 8.The Expert’s charges will be borne as he/ she determines in his written decision. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 42Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 3DEFINITION OF TOBACCO INDUSTRY FUNDING (REVISED 2009)FROM THE CANCER RESEARCH UK CODE OF PRACTICE ON TOBACCO INDUSTRY FUNDING TO UNIVERSITIES.http://www.cancerresearchuk.org/science/funding/terms-conditions/funding-policies/policy-tobacco/A tobacco company is defined for the purposes of this policy as one that: • Derives over 5% of revenues from manufacturing tobacco products; • Derives 15%+ of revenues from the manufacture of products necessary for the production of tobacco products; • Derives 15% of revenues from the sale of tobacco products (and has 30 or more staff); • Owns a tobacco company (the company owns 50% or more of a tobacco company); • Is more than 50% owned by a company with tobacco involvement.The following do not constitute tobacco industry funding for the purposes of this Code: • legacies from tobacco industry investments (provided these are sold on immediately) • funding from a trust or foundation no longer having any connection with the tobacco industry even though it may bear a name that (forhistorical reasons) has tobacco industry associations. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 43Confidential Treatment Requested by MeiraGTx Holdings plc SCHEDULE 4DEVELOPMENT PLAN[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. 44Confidential Treatment Requested by MeiraGTx Holdings plc [***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Exhibit 10.24Confidential Treatment Requested by MeiraGTx Holdings plcAMENDMENT NO. 4 TO EXCLUSIVE LICENSE AGREEMENTThis AMENDMENT NO. 4 TO EXCLUSIVE LICENSE AGREEMENT (this “Amendment”), effective as of January 29, 2019 (“AmendmentEffective Date”), is entered into by and between MeiraGTx Limited (registered number 9501998), having a place of business located at 92 BritanniaWalk, London, United Kingdom, N1 7NQ United Kingdom (“MeiraGTx”) and UCL Business PLC (registered number 02776963), whose registeredoffice is The Network Building, 97 Tottenham Court Road, London W1T 4TP United Kingdom (“UCLB”). MeiraGTx and UCLB are each sometimesreferred to herein as a “Party” and collectively referred to herein as the “Parties”.WITNESSETH:WHEREAS, UCLB and Athena Vison Ltd., a company incorporated under the laws of England and Wales under company registration number09348737 (“Athena”) entered into a License Agreement dated 4 February 2015, as amended by Amendment No. 1 to Exclusive License Agreement,effective as of 27 March 2015, Amendment No. 2, effective as of July 28, 2017, and Amendment No.3, effective as of December 14, 2017 (as amended,the “Licence Agreement”), pursuant to which UCLB licensed to Athena certain technology and intellectual property relating to ocular gene therapy,which UCLB acquired by assignment from University College London, for development and commercialization by Athena;WHEREAS, UCLB, Athena, and MeiraGTx entered into a Deed of Novation and Amendment in 2016 (“Deed of Novation and Amendment”) pursuantto which the parties thereto agreed to, among other things, novate Athena’s rights, obligations, and liabilities under the Licence Agreement toMeiraGTx on the terms and conditions of the Deed of Novation and Amendment;WHEREAS, UCLB and MeiraGTx UK II Limited (an affiliate of MeiraGTx) entered into a separate licence agreement, dated July 28, 2017 toconsolidate UCLB’s rights to an ocular gene therapy for RPGR (“RPGR Licence Agreement”);WHEREAS, UCLB, MeiraGTx and MeiraGTx UK II Limited (an affiliate of MeiraGTx) entered into a separate licence agreement, dated March 15,2018 to consolidate UCLB’s rights to an ocular gene therapy for CNGA3 (“CNGA3 Licence Agreement”); andWHEREAS, UCLB and MeiraGTx further wish to amend the Licence Agreement in certain respects.NOW, THEREFORE, in consideration of the foregoing statements and the mutual agreements and covenants herein contained, and for other good andvaluable consideration, the sufficiency of which are hereby acknowledged, the Parties hereby agree as follows.Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc Section 1. Definitions. Except as set for the herein, capitalized terms not otherwise defined or amended in this Amendment shall have themeaning ascribed to them in the Licence Agreement, as amended by this Amendment. References to Articles, Clauses or Schedules are to the same withall their subparts as they appear in the Licence Agreement. References to Sections are to the numbered paragraphs with all their subparts as they appearin this Amendment, and references to Exhibits are to the exhibits attached to this Amendment, and incorporated herein by reference.Section 2. Amendment to the Licence Agreement.(a) CNGB3. Licence Addendum Number 1, dated 4 February 2015 for technology [***] (CNGB3), is hereby deleted in its entirety as of theAmendment Effective Date and the Parties have entered into the stand-alone licence agreement attached as Exhibit 1. For clarity, the Partiesacknowledge and agree that such licence agreement supersedes the rights and obligations of the Parties set out in Licence Addendum Number 1 as ofthe Amendment Effective Date, and MeiraGTx or its affiliates will not owe UCLB any payments for any licensed products under Licence AddendumNumber 1 or the related Licence Agreement.(b) RPE65. Licence Addendum Number 5, dated December 2017 for technology [***] (RPE65), is hereby deleted in its entirety as of theAmendment Effective Date, and Parties have entered into the stand-alone licence agreement attached as Exhibit 2. For clarity, the Parties acknowledgeand agree that such licence agreement supersedes the rights and obligations of the Parties set out in Licence Addendum Number 5 as of the AmendmentEffective Date, and MeiraGTx or its affiliates will not owe UCLB any payments for any licensed products under Licence Addendum Number 5 or therelated Licence Agreement.(c) RPGR. The RPGR Licence Agreement shall terminate in its entirety as of the date on which UCLB notifies MeiraGTx in writing that ithas obtained approval from MEEI and MRC for the new stand alone licence agreement attached as Exhibit 3, and Parties shall enter into the stand-alone licence agreement attached as Exhibit 3. For clarity, the Parties acknowledge and agree that such licence agreement (once it becomes effective)shall supersede the rights and obligations of the Parties set out in Licence Addendum Number 3, for technology [***] (RPGR) as of July 28, 2017, andthat Licence Addendum Number 3 shall be deleted from the Licence Agreement from such date, and MeiraGTx or its affiliates will not owe UCLB anypayments for any licensed products under Licence Addendum Number 3, the related Licence Agreement or the RPGR Licence Agreement.(d) CNGA3. The CNGA3 Licence Agreement is hereby terminated in its entirety as of the Amendment Effective Date, and Parties haveentered into the stand-alone licence agreement attached as Exhibit 4. For clarity, the Parties acknowledge and agree that such licence agreementsupersedes the rights and obligations of the Parties set out in Licence Addendum Number 2, dated 4 February 2015 for technology [***] (CNGA3) asof March 15, 2018, and that Licence Addendum Number 2 has been deleted from the Licence Agreement, and MeiraGTx or its affiliates will not oweUCLB any payments for any licensed products under Licence Addendum Number 2, the related Licence Agreement or the RPGR Licence Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc (e) Option Rights. All other provisions of the Licence Agreement (including without limitation the options set out in Clause 2.6 of theLicence Agreement which have not been exercised by MeiraGTx prior to the date of termination, i.e., the options in relation to Specified Technologies[***]) together with any accrued rights of the Parties under the Licence Agreement prior to the Commencement Date shall continue in full force andeffect.Section 3. Other.(a) Effect of Amendment. From and after the Amendment Effective Date, all references to the Licence Agreement shall mean the LicenceAgreement as amended by this Amendment.(b) Counterparts. This Amendment may be executed in two or more counterparts, each of which shall be deemed an original, but all ofwhich together shall constitute one and the same instrument. Signature to his Amendment transmitted by fax, by email in “portable document format”(“.pdf”) or by any other electronic means intended to preserve the original graphic and pictorial appearance of this Amendment shall have the sameeffect as physical delivery of the paper document bearing original signature.(c) Entire Amendment. This Amendment contains the entire understanding of the Parties with respect to the subject matter of thisAmendment. Except as specifically modified and amended hereby, all of the terms, provisions, requirements and specifications contained in theLicence Agreement remain in full force and effect. Except as otherwise expressly provided herein, the Parties do not intend to, and the execution of thisAmendment shall not, in any manner, impair the Agreement, the purpose of this Amendment being simply to amend certain specific provision of theAgreement only and to confirm and carry forward the Licence Agreement, as hereby amended, in full force and effect. This Amendment may beamended, or any term hereof modified, only by a written instrument executed by both Parties.(d) Notices. Any notices, requests and other communications hereunder shall be in writing and shall be personally delivered or sent by faxtransmission (and promptly confirmed by personal delivery, registered or certified mail or overnight courier) or by registered or certified mail, returnreceipt required, postage prepaid, or sent by internally-recognized overnight courier, in each case to the respective address specified below, or suchother address as may be specified in writing to the other Party hereto:If to MeiraGTx to:MeiraGTx Limited92 Britannia Walk, London Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc United Kingdom, N1 7NQ United KingdomFax No.: [***]Attention: Chief Operating OfficerIf to UCLB to:UCL Business PLCThe Network Building97 Tottenham Court RoadLondon W1T 4TP United KingdomFax No.: [***]Attention: Director of Legal Affairs(e) Law and Jurisdiction. The validity, construction and performance of this Amendment, and any contractual and non-contractual claimsarising hereunder, shall be governed by English law and shall be subject to the exclusive jurisdiction of the English courts to which the Parties herebysubmit, except that a Party may seek an interim injunction (or an equivalent remedy) in any court of competent jurisdiction.[Signature Page Follows] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc IN WITNESS WHEREOF, the Parties have caused this Amendment to be effective as of the Amendment Effective Date. MEIRAGTX LIMITEDBy [***] Name: [***] Title: [***]UCL BUSINESS PLCBy /s/ Zandy Forbes Name: Zandy Forbes Title: President and CEO Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 1Coverpage for New standalone Licence Agreement for CNGB3 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 2Coverpage for New standalone Licence Agreement for RPE65 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 3Coverpage for New standalone Licence Agreement for CNGA3 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 4Coverpage for New standalone Licence Agreement for RPGR Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission.Exhibit 10.25Confidential Treatment Requested by MeiraGTx Holdings plcEXECUTION VERSION COLLABORATION, OPTION AND LICENSE AGREEMENTBY AND BETWEENJANSSEN PHARMACEUTICALS, INC.,MEIRAGTX UK II LIMITEDANDMEIRAGTX HOLDINGS PLC Confidential Treatment Requested by MeiraGTx Holdings plc TABLE OF CONTENTS 1. DEFINITIONS 1 2. OVERVIEW OF COLLABORATION 27 2.1. Overview of Clinical Development Plan Activities 27 2.2. Overview of Research Plan Activities 28 2.3. Overview of CMC Development Plan Activities and Other Manufacturing Activities 29 3. NON-CLINICAL RESEARCH 29 3.1. Research IRD Targets 29 3.2. Research Plans 30 3.3. Conduct of Research Activities 30 3.4. Research Budget 30 3.5. Research Records 31 3.6. Audits 31 3.7. Research Reports and Materials 32 3.8. Research Costs 33 3.9. Option 33 4. LICENSES 34 4.1. Licenses to Janssen 34 4.2. Licenses to MeiraGTx 35 4.3. Sublicense Rights 36 4.4. Subcontractors 36 4.5. Third Party Licenses 37 4.6. Exclusivity 37 4.7. No Other Rights 38 4.8. MeiraGTx Know-How Transfer 39 5. GOVERNANCE 39 5.1. Alliance Managers 39 5.2. Joint Steering Committee 39 5.3. Joint Research Committee 40 5.4. Joint Development Committee 40 5.5. Joint Manufacturing Committee 41 5.6. Operational Teams 42 5.7. Committee Representatives and Meetings 42 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. iConfidential Treatment Requested by MeiraGTx Holdings plc 5.8. Resolution of Committee Disputes 43 6. DEVELOPMENT 44 6.1. Clinical IRD Products 44 6.2. Janssen Research IRD Products 47 7. REGULATORY 48 7.1. Clinical IRD Products 48 7.2. Research IRD Products 50 7.3. Pharmacovigilance 51 8. MANUFACTURING 52 8.1. Product Manufacturing outside Supply Agreements 52 8.2. Clinical Supply Agreement 52 8.3. Commercial Supply Agreement 52 8.4. Collaboration on CMC Development 53 8.5. [***] Manufacturing Services 53 9. COMMERCIALIZATION 56 10. FINANCIAL PROVISIONS 56 10.1. Upfront Payment 56 10.2. Research Costs 56 10.3. Clinical Development Costs 56 10.4. Clinical Development Manufacturing Costs 57 10.5. Process Development Costs 58 10.6. Development Milestone Payments 59 10.7. Commercial Milestone Payments 60 10.8. Royalties 62 10.9. Additional Royalty Provisions 63 10.10. Third Party Obligations 64 10.11. Reports and Royalty Payments 65 10.12. Disclaimer. 66 10.13. Payment Terms 66 10.14. Records and Audits 67 11. INTELLECTUAL PROPERTY RIGHTS 69 11.1. Ownership under the Clinical IRD Programs and Research IRD Programs 69 11.2. Ownership under the CMC Development Collaboration 70 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -ii-Confidential Treatment Requested by MeiraGTx Holdings plc 11.3. Duties to the Other Party 70 11.4. Patent Prosecution and Maintenance 71 11.5. Third Party Infringement; Patent Actions 73 11.6. Product Infringement 76 11.7. Patents Licensed From Third Parties 77 11.8. Trademarks 77 12. CONFIDENTIALITY 77 12.1. Duty of Confidence 77 12.2. Exceptions 77 12.3. Authorized Disclosures 78 12.4. Terms of this Agreement 79 12.5. Use of Residuals 79 12.6. Data Privacy 79 13. PUBLICATIONS AND PUBLICITY 79 13.1. Use of Names 79 13.2. Press Releases and Publicity Related to this Agreement 80 13.3. Public Disclosures and Publications Related to the Programs or Products 80 13.4. Publications 80 13.5. Disclosures Required By Law 81 13.6. Publication 81 14. EFFECTIVENESS 82 14.1. Effective Date 82 14.2. Filings 82 14.3. Outside Date 82 15. TERM AND TERMINATION 83 15.1. Term 83 15.2. Termination 83 15.3. Rights in Insolvency 85 15.4. Effects of Expiration or Termination 86 15.5. Post-Termination Royalties to Janssen 89 15.6. Survival 89 15.7. Termination Not Sole Remedy 90 16. REPRESENTATIONS, WARRANTIES AND COVENANTS 90 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -iii-Confidential Treatment Requested by MeiraGTx Holdings plc 16.1. Representations and Warranties by Each Party 90 16.2. Representations, Warranties and Covenants by MeiraGTx 91 16.3. Mutual Covenants 93 16.4. No Other Warranties 93 17. INDEMNIFICATION; LIABILITY; INSURANCE 94 17.1. Indemnification by MeiraGTx 94 17.2. Indemnification by Janssen 94 17.3. Indemnification Procedure 95 17.4. Mitigation of Loss 97 17.5. Limited Liability 97 17.6. Insurance Obligations 97 18. DISPUTE RESOLUTION 98 18.1. Dispute Resolution 98 18.2. Governing Law 100 18.3. Exclusions 100 18.4. Cumulative Remedies 101 18.5. Injunctive Relief 101 19. GENERAL PROVISIONS 101 19.1. Assignment 101 19.2. Extension to Affiliates 102 19.3. Severability 102 19.4. Force Majeure 102 19.5. Waivers and Amendments 102 19.6. Relationship of the Parties 103 19.7. Notices 103 19.8. Further Assurances 104 19.9. No Third Party Beneficiary Rights 104 19.10. English Language 104 19.11. Interpretation 104 19.12. Entire Agreement 105 19.13. Counterparts 106 Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -iv-Confidential Treatment Requested by MeiraGTx Holdings plc ExhibitsExhibit 1.34: Clinical Development Plans, Including Anticipated Costs and TimelinesExhibit 1.34-1: Anticipated Costs and Timelines – CNGA3 ProductExhibit 1.34-2: Anticipated Costs and Timelines – CNGB3 ProductExhibit 1.34-3: Anticipated Costs and Timelines – RPGR ProductExhibit 1.51: CMC Development PlansExhibit 1.103: Final Report RequirementsExhibit 1.136: J&J Universal Calendar for 2019 and 2020Exhibit 1.168: MeiraGTx PatentsExhibit 1.221: IRD GenesExhibit 1.223: Research PlansExhibit 3.4: Research Budget CapExhibit 3.5: Janssen Data PoliciesExhibit 4.4: MeiraGTx SubcontractorsExhibit 8.2: Key Terms of Clinical Supply Agreement and Clinical Quality Assurance AgreementExhibit 8.3: Key Terms of Commercial Supply Agreement and Commercial Quality Assurance AgreementExhibit 8.4(a): CMC Development Plan Activities – BreakdownExhibit 16.2(b): Third Party RightsExhibit 16.2(g): MeiraGTx Existing Third Party Obligations Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -v-Confidential Treatment Requested by MeiraGTx Holdings plc COLLABORATION, OPTION AND LICENSE AGREEMENTThis Collaboration, Option and License Agreement (this “Agreement”) is made as of January 30, 2019 (the “Execution Date”), by and betweenJanssen Pharmaceuticals, Inc., a Pennsylvania corporation located at 1125 Trenton-Harbourton Road, Titusville, NJ 08560, United States of America(“Janssen”), on the one hand, and MeiraGTx UK II Limited, a company organized and existing under the laws of England, located at 25 Provost Street,London N1 7NH, United Kingdom and MeiraGTx Holdings plc, a Cayman Islands corporation located at 430 East 29th Street, 10th Floor, New York,NY 10016, United States of America (MeiraGTx UK II Limited and MeiraGTx Holdings plc, individually or collectively, “MeiraGTx”), on the otherhand. Janssen and MeiraGTx are each referred to individually as a “Party” and together as the “Parties.”RECITALSWHEREAS, MeiraGTx has technology and expertise in gene therapy, including viral vector design and optimization and gene therapymanufacturing, and is applying such technology to develop therapeutic products, including the Clinical IRD Products (as defined below);WHEREAS, Janssen, together with its Affiliates, is engaged in the Research, Development, and Commercialization of biopharmaceuticalproducts globally;WHEREAS, MeiraGTx and Janssen desire to enter into a collaboration to jointly Develop the Clinical IRD Products to permit Janssen toCommercialize such Clinical IRD Products under an exclusive license from MeiraGTx, all under the terms and conditions set forth herein;WHEREAS, MeiraGTx and Janssen also desire to enter into a collaboration where Janssen would co-fund MeiraGTx’s research on Research IRDProducts (as defined below), and in return, MeiraGTx would grant Janssen an exclusive option to obtain an exclusive license to further Develop andCommercialize certain Research IRD Products, all under the terms and conditions set forth herein; andWHEREAS, MeiraGTx and Janssen desire to collaborate on the Development of certain manufacturing and technical improvements and providecertain additional Manufacturing services, under the terms and conditions set forth herein.NOW, THEREFORE, in consideration of the mutual covenants and agreements herein contained, the Parties agree as follows. 1.DEFINITIONSUnless the context otherwise requires, the capitalized terms in this Agreement have the meanings set forth below or the meaning as designated in theindicated places throughout this Agreement. 1.1.“Accounting Standards” means GAAP, as generally and consistently applied throughout each Party’s organization. 1.2.“Acquired Party” has the meaning set forth in Section 4.6(d). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc 1.3.“Act” means the United States Federal Food, Drug and Cosmetic Act, 21 U.S.C. §§ 301 et seq. and the United States Public Health Service (PHS)Act, 42 U.S.C. §§ 201 et seq. 1.4.“Advancement Criteria” means the specific, objective criteria and standards that need to be fulfilled for a successful IND Submission inaccordance with 21 C.F.R. §312 and any foreign counterpart to such regulation and any other requirements of any Regulatory Authority. 1.5.“Adverse Event” means (a) any untoward medical occurrence in a Clinical Study subject or in a patient who is administered a Product, whetheror not considered related to such Product, including any undesirable sign (including abnormal laboratory findings of clinical concern),symptom, or disease associated with the use of a Product or (b) finding from any animal or in vitro testing or toxicology study, whether or notconducted by the sponsor, that suggests a significant risk in humans exposed to the drug. 1.6.“Affiliate” means, with respect to a Party, any Person that, directly or indirectly, controls, is controlled by, or is under common control with thatParty, for so long as such control exists. For the purpose of this definition, “control” means any of the following: (a) direct or indirect ownershipof fifty percent (50%) or more of the shares of stock entitled to vote for the election of directors, in the case of a corporation, or fifty percent(50%) or more of the equity interest in the case of any other type of legal entity, (b) status as a general partner in any partnership, or (c) anyother arrangement whereby the entity or person controls or has the right to control the board of directors or equivalent governing body of acorporation or other entity, or the ability to cause the direction of the management or policies of a corporation or other entity, whether throughownership of voting securities, by contract or otherwise. In the case of entities organized under the laws of certain countries, the maximumpercentage ownership permitted by law for a foreign investor may be less than fifty percent (50%), and in such case, such lower percentage shallbe substituted in the preceding sentence, provided that such foreign investor has the power to direct the management and policies of suchentity. 1.7.“Affordable Basis” means selling a Product for [***]. In determining Affordable Basis, the Parties recognize that, to the extent that a Partyengages a Third Party in the Commercialization of a Product on an Affordable Basis, [***]. 1.8.“Agreement” has the meaning set forth in the first paragraph of this Agreement. 1.9.“Alliance Manager” has the meaning set forth in Section 5.1. 1.10.“Annual Net Sales” means Net Sales of Product(s) in a Calendar Year. 1.11.“Anti-Corruption Laws” means all Applicable Laws and international financial institution rules regarding corruption, bribery, ethical businessconduct, money laundering, political contributions, gifts and gratuities, or lawful expenses to public officials, healthcare professionals, andprivate persons, agency relationships, commissions, lobbying, books and records, and financial controls, including the FCPA. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -2-Confidential Treatment Requested by MeiraGTx Holdings plc 1.12.“Applicable Law” means any applicable law, statute, code, ordinance, rule or regulation, enforceable guideline or other requirement, order,injunction, judgment, writ, stipulation, award, arbitration award, decree, other pronouncement having the effect of law, constitution or treatyenacted, promulgated, issued, enforced or entered by any Governmental Authority applicable to any Party or such Party’s businesses, propertiesor assets, as may be amended from time to time. 1.13.“Audited Party” has the meaning set forth in Section 10.14(b). 1.14.“Auditing Party” has the meaning set forth in Section 10.14(b). 1.15.“Auditor” has the meaning set forth in Section 10.14(b). 1.16.“Biosimilar Product” means, in a particular country with respect to a particular Product, any biopharmaceutical product that: (a) has receivedall necessary approvals by the applicable Regulatory Authorities in such country to market and sell such product as a biopharmaceuticalproduct; (b) is marketed or sold by a Third Party who is not a licensee or Sublicensee of Janssen or its Affiliates regarding suchbiopharmaceutical product; and (c) is approved as (i) a “biosimilar” (in the United States) of such Product, (ii) a “similar biological medicinalproduct” (in the EU) with respect to which such Product is the “reference medicinal product,” or (iii) if not in the US or EU, the foreignequivalent of a “biosimilar” or “similar biological medicinal product” of such Product, in each case, for use in such country pursuant to aregulatory approval process governing approval of generic biologics based on the then-current standards for regulatory approval in suchcountry (e.g., the Biologics Price Competition and Innovation Act of 2009 or an equivalent under foreign law). 1.17.“BLA” means: (a) a Biologics License Application as defined in the Act and the regulations promulgated thereunder; (b) an MAA in the EU; or(c) any equivalent or comparable application, registration or certification in any other country or region. 1.18.“Business Day” means any day that is not a Saturday, Sunday, or other day on which commercial banks are authorized or required to be closedin New York, New York. 1.19.“Calendar Quarter” means a financial quarter based on the J&J Universal Calendar for that year that is used by Janssen and Affiliates forinternal and external reporting purposes; provided, however, that the first Calendar Quarter of the Term extends from the Effective Date to theend of the then-current Calendar Quarter, and the last Calendar Quarter extends from the first day of such Calendar Quarter until the effectivedate of the termination or expiration of this Agreement. 1.20.“Calendar Year” means a year based on the J&J Universal Calendar for that year; provided, however, that the first Calendar Year of the Termextends from the Effective Date to the end of the then-current Calendar Year, and the last Calendar Year extends from the first day of suchCalendar Year for the year during which termination or expiration of this Agreement will occur until the effective date of the termination orexpiration of this Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -3-Confidential Treatment Requested by MeiraGTx Holdings plc 1.21.“CAPA” has the meaning set forth in Section 3.6. 1.22.“cGCP” means the then-current ethical, scientific and quality standards, practices and procedures required by FDA for designing, conducting,recording and reporting trials that involve the participation of human subjects, as set forth in FDA regulations in 21 C.F.R. Parts 11, 50, 54, 56,and 312 and related FDA guidance documents, including the guidelines entitled “Guidance for Industry E6 Good Clinical Practice:Consolidated Guidance” and by the International Conference on Harmonization E6: Good Clinical Practices Consolidated Guideline, or theequivalent Applicable Law of an applicable Regulatory Authority. 1.23.“cGLP” means the then-current good laboratory practice standards as required by the FDA under 21 C.F.R. Part 58 and all applicable FDArules, regulations, orders and guidance, and the requirements with respect to current good laboratory practices prescribed by the EuropeanCommunity, the OECD (Organization for Economic Cooperation and Development Council) and the ICH Guidelines, or the equivalentApplicable Law of an applicable Regulatory Authority. 1.24.“cGMP” means the then-current good manufacturing practices as required by the FDA under provisions of 21 C.F.R. Parts 210 and 211 and allapplicable FDA rules, regulations, orders and guidance, and the requirements with respect to current good manufacturing practices prescribedby the European Community under provisions of “The Rules Governing Medicinal Products in the European Community, Volume 4, GoodManufacturing Practices, Annex 13, Manufacture of Investigational Medicinal Products, July 2003,” or the equivalent Applicable Law of anapplicable Regulatory Authority. 1.25.“Challenge” or “Challenging” means, with respect to any MeiraGTx Patents, MeiraGTx Research Patents or Joint Patents, to contest thevalidity or enforceability of any such Patents, in whole or in part, in any court, arbitration proceeding or other tribunal, including the UnitedStates Patent and Trademark Office, the European Patent Office, and the United States International Trade Commission. As used in this term“Challenge”, the term “contest” includes [***]. 1.26.“Challenged Patent” has the meaning set forth in Section 15.2(c). 1.27.“Change of Control” means, with respect to a Party, (a) a merger or consolidation of such Party with a Third Party that results in the votingsecurities of such Party outstanding immediately prior thereto, or any securities into which such voting securities have been converted orexchanged, ceasing to represent at least fifty percent (50%) of the combined voting power of the surviving entity or the parent of the survivingentity immediately after such merger or consolidation; (b) a transaction or series of related transactions in which a Third Party, together with itsAffiliates, becomes the beneficial owner of fifty percent (50%) or more of the combined voting power of the outstanding securities of suchParty; or (c) the sale or other transfer to a Third Party of all or substantially all of such Party’s assets or business to which the subject matter ofthis Agreement relates. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -4-Confidential Treatment Requested by MeiraGTx Holdings plc 1.28.“Claims” means all Third Party demands, claims, actions, suits, causes of action and proceedings. 1.29.“Clinical Development Budget” has the meaning set forth in Section 6.1(d). Each Clinical Development Budget, upon approval in accordancewith the terms and conditions herewith, will be automatically incorporated into this Agreement by reference and form a part of this Agreement. 1.30.“Clinical Development Costs” means, with respect to each Clinical IRD Product [***] all costs and expenses incurred on or after the EffectiveDate in connection with the performance of any Clinical Development Plan Activities for such Clinical IRD Product in accordance with theapplicable Clinical Development Plan, including FTE Costs and fees charged by Third Party service providers, but excluding (a) overhead costsand capital expenditures and (b) all Clinical Development Manufacturing Costs. 1.31.“Clinical Development Manufacturing Activities” has the meaning set forth in Section 8.4(a). 1.32.“Clinical Development Manufacturing Budget” has the meaning set forth in Section 8.4(b). Each Clinical Development ManufacturingBudget, upon approval in accordance with the terms and conditions herewith, will be automatically incorporated into this Agreement byreference and form a part of this Agreement. 1.33.“Clinical Development Manufacturing Costs” means the fully-burdened cost incurred on or after the Effective Date (and, solely with respect tothe Clinical Supply of the CNGA3 Product, such costs incurred prior to the Effective Date and as specified on Exhibit 1.34-1) by either Party orany of their respective Affiliates in connection with the performance of any Clinical Development Manufacturing Activities in accordance withthis Agreement and consistent with (a) the applicable CMC Development Plan, (b) the Clinical Supply Agreement, and (c) all componentsdefined in Commercial Manufacturing Costs, including in each case ((a)-(c)), all FTE Costs, Out-of-Pocket Costs, and depreciation of capitalexpenditures. 1.34.“Clinical Development Plan” means the strategic plan for Developing a Clinical IRD Product, as such plan may be agreed to, approved,amended or updated from time to time in accordance with Section 6.1(c). Each Clinical Development Plan will be automatically incorporatedinto this Agreement by reference and form a part of Exhibit 1.34 in accordance with Section 6.1(c). The anticipated costs and timelines foractivities under the Clinical Development Plan for the CNGA3 Product are attached hereto as Exhibit 1.34-1. The anticipated costs andtimelines for activities under the Clinical Development Plan for the CNGB3 Product are attached hereto as Exhibit 1.34-2. The anticipated costsand timelines for activities under the Clinical Development Plan for the RPGR Product are attached hereto as Exhibit 1.34-3. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -5-Confidential Treatment Requested by MeiraGTx Holdings plc 1.35.“Clinical Development Plan Activities” has the meaning set forth in Section 6.1(c). 1.36.“Clinical Development Plan Term” means the term of a Clinical Development Plan, as set forth in such Clinical Development Plan, providedthat in no event will the Clinical Development Plan Term exceed the Term. 1.37.“Clinical Development Records” has the meaning set forth in Section 6.1(g). 1.38.“Clinical IRD Patents” has the meaning set forth in Section 11.4(b). 1.39.“Clinical IRD Products” means: (a) the CNGB3 Product; (b) the CNGA3 Product; (c) the RPGR Product; and (d) [***]. 1.40.“Clinical IRD Target” means: (a) the CNGB3 Target; (b) the CNGA3 Target; (c) the RPGR Target; and (d) [***]. 1.41.“Clinical IRD Target Indication” means: (a) the CNGB3 Target Indication; (b) the CNGA3 Target Indication; (c) the RPGR Target Indication;and (d) [***]. 1.42.“Clinical Quality Assurance Agreement” has the meaning set forth in Section 8.2. 1.43.“Clinical Study” means a (a) Phase 1 Study, (b) Phase 1/2 Study, (c) Phase 2 Study, (d) Phase 3 Study, (e) Pivotal Study or (f) other prospectivestudy (including a non-interventional study or Natural History Study) or post-Regulatory Approval study, in each case of this subsection (f) inhumans to obtain information regarding a disease state or product, including information relating to the safety, tolerability, pharmacologicalactivity, pharmacokinetics, dose ranging or efficacy of the product. 1.44.“Clinical Supply” means, with respect to a Clinical IRD Product or Janssen Research IRD Product, such Product Manufactured for use in aClinical Study of such Product under this Agreement. 1.45.“Clinical Supply Agreement” has the meaning set forth in Section 8.2. 1.46.“CMC” means chemistry, manufacturing and controls. 1.47.“CMC Development Collaboration” has the meaning set forth in Section 8.4(a). 1.48.“CMC Development Inventions” means all Inventions, including CMC data, arising out of the CMC Development Collaboration, whethermade solely by or on behalf of a Party or jointly with the other Party under this Agreement. 1.49.“CMC Development Know-How” means all Know-How, including CMC data, arising out of the CMC Development Collaboration, whethermade solely by or on behalf of a Party or jointly with the other Party under this Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -6-Confidential Treatment Requested by MeiraGTx Holdings plc 1.50.“CMC Development Patents” mean all Patents Covering patentable CMC Development Inventions or CMC Development Know-How. 1.51.“CMC Development Plan” means the strategic plan for the CMC Development Collaboration, as such plan may be agreed to, approved,amended or updated from time to time in accordance with Section 8.4(a). Each CMC Development Plan will be automatically incorporated intothis Agreement by reference and form a part of Exhibit 1.51. 1.52.“CMC Development Plan Activities” has the meaning set forth in Section 8.4(a). 1.53.“CMC Development Technology” means the CMC Development Patents, CMC Development Inventions and CMC Development Know-How. 1.54.“CMC Development Term” has the meaning set forth in Section 2.3. 1.55.“CMO” means a Third Party contract Manufacturing organization. 1.56.“CNGA3 Product” means: (a) MeiraGTx’s Gene Therapy Product [***] for the treatment of the CNGA3 Target Indication by expressing theCNGA3 Target; [***]. 1.57.“CNGA3 Target” means the [***]. 1.58.“CNGA3 Target Indication” means the inherited retinal disease resulting from the loss of function of the CNGA3 Target. 1.59.“CNGB3 Product” means: (a) MeiraGTx’s Gene Therapy Product [***] for the treatment of the CNGB3 Target Indication by expressing theCNGB3 Target; [***]. 1.60.“CNGB3 Target” means the [***]. 1.61.“CNGB3 Target Indication” means the inherited retinal disease resulting from the loss of function of the CNGB3 Target. 1.62.“Code” means the United States Bankruptcy Code, 11 U.S.C. §§ 101 et seq. 1.63.“Commercial Manufacturing Costs” means [***]. 1.64.“Commercial Milestone Event” has the meaning set forth in Section 10.7(a). 1.65.“Commercial Milestone Payment” has the meaning set forth in Section 10.7(a). 1.66.“Commercial Quality Assurance Agreement” has the meaning set forth in Section 8.3. 1.67.“Commercial Supply” means a Product Manufactured for Commercialization of such Product, including for Product launch. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -7-Confidential Treatment Requested by MeiraGTx Holdings plc 1.68.“Commercial Supply Agreement” has the meaning set forth in Section 8.3. 1.69.“Commercialize” or “Commercialization” means to market, promote, detail, conduct Medical Affairs, distribute, import, export, offer to sell,use or sell biopharmaceutical products or conduct other commercialization activities, including activities directed to obtaining PricingApprovals, conducting pre- and post-Regulatory Approval activities and launching and promoting such biopharmaceutical products in eachcountry, as applicable. 1.70.“Commercially Reasonable Efforts” means, with respect to the efforts to be expended by a Party with respect to any objective under thisAgreement, those reasonable, good faith efforts normally used by such Party under similar circumstances for similar products or productcandidates owned or controlled by such Party, or to which such Party has similar rights, which product or product candidate is of similar marketpotential in such country and is at a similar stage in its development or product life, taking into account all relevant scientific, technical,operational, commercial, economic and other factors that may affect the Development, Regulatory Approval, Manufacturing orCommercialization of a product, including (as applicable): actual and potential issues of safety, efficacy or stability; expected and actualproduct profile (including product modality, category and mechanism of action); stage of development or life cycle status; actual and projectedDevelopment, Regulatory Approval, Manufacturing, and Commercialization costs, timelines and budgets; any issues regarding the ability toManufacture or have Manufactured the product; the likelihood of obtaining Regulatory Approvals; the timing of such approvals; labeling oranticipated labeling; the then-current competitive environment and the likely competitive environment at the time of projected entry into themarket, including the expected and actual competitiveness of alternative products; past performance of the product or similar products; presentand future market potential; existing or projected pricing, sales, reimbursement and profitability; and expected and actual proprietary position,strength and duration of patent protection and anticipated regulatory or other exclusivity as such company would normally use to accomplish asimilar objective under similar circumstances. With respect to a Party’s obligations, Commercially Reasonable Efforts requires that the Party, tothe extent doing so would be required by this Agreement and the definition of “Commercially Reasonable Efforts” to do so: [***] To the extentthat [***]. To the extent that [***]. 1.71.“Committee” means the Joint Steering Committee, the Joint Research Committee, the Joint Development Committee, the Joint ManufacturingCommittee or any other subcommittee established under Section 5.2(b), as applicable. 1.72.“Competing Product” means, with respect to any Product, any Gene Therapy Product that [***]. 1.73.“Completion Notice” has the meaning set forth in Section 3.7(c). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -8-Confidential Treatment Requested by MeiraGTx Holdings plc 1.74.“Confidential Information” means all confidential Know-How and other confidential information and data of a Party that is disclosed by or onbehalf of a Party or any of its Affiliates or otherwise made available to the other Party or its Affiliates, whether made available orally, in writingor in electronic form, including information comprising or relating to concepts, discoveries, inventions, data, designs or formulae in relation tothis Agreement or of a financial, commercial, business, operational or technical nature. For clarity, the terms and conditions of this Agreementshall constitute the Confidential Information of both Parties and each Party will be deemed a Receiving Party with respect thereto, and allresearch results shall constitute the Confidential Information of both Parties during the Term (subject to Section 15.4(b)). 1.75.“Control” or “Controlled” means, with respect to any Intellectual Property Rights, the legal authority or right (whether by ownership, licenseor otherwise, other than pursuant to this Agreement) of a Party to grant a license or a sublicense of or under such Intellectual Property Rights toanother Person, or to otherwise disclose such Intellectual Property Rights to another Person, without violating any Applicable Law, breachingthe terms of any agreement with a Third Party or misappropriating the proprietary or trade secret information of a Third Party, and, subject toSection 10.10, without incurring payment obligations by reason of licensing, sublicensing, or providing access to the other Party with respectthereto (unless such other Party agrees in writing to bear all such costs arising from the license, sublicense, or access to such item by such otherParty). Notwithstanding anything to the contrary in this Agreement, in the event of a Change of Control of a Party, (a) any Intellectual PropertyRights Controlled by any acquiring entity (and not Controlled by such Party or its Affiliates) immediately prior to the effective date of suchChange of Control and (b) any Intellectual Property Rights independently developed or acquired by or on behalf of any acquiring entitywithout access to or use of any Intellectual Property Rights used or made available under this Agreement or pre-acquisition employees of suchParty or its pre-acquisition Affiliates, in each case ((a) and (b)) shall not be deemed to be Controlled by such Party or its Affiliates after theeffective date of such Change of Control for purposes of this Agreement. 1.76.“Controlling Party” has the meaning set forth in Section 11.5(d). 1.77.“Cover” or “Covered” means that, but for a license granted to a Person under a Valid Claim of a Patent, the act of Developing, Manufacturing,or Commercializing by such Person would infringe, or contribute to or induce the infringement of, such Valid Claim. 1.78.“CPR Mediation Procedure” has the meaning set forth in Section 18.1(b). 1.79.“CPR Rules” has the meaning set forth in Section 18.1(c)(i). 1.80.“Damages” means all losses, liabilities, damages, taxes, costs and expenses of every kind and nature (including reasonable attorneys’ fees). 1.81.“Data Exclusivity Right” means any data exclusivity rights or exclusive marketing rights conferred by any Regulatory Authority with respectto a Product (other than Patents), including orphan drug exclusivity, market exclusivity, data exclusivity, or pediatric exclusivity. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -9-Confidential Treatment Requested by MeiraGTx Holdings plc 1.82.“Debarred Person” means a Person that is: (a) debarred from or disqualified under the Act or any other governmental program; (b) on any of theFDA clinical investigator enforcement lists (including, the (i) Disqualified/Totally Restricted List, (ii) Restricted List and (iii) AdequateAssurances List); (c) excluded from participation in any governmental healthcare program or other federal or state program, convicted of anoffense under 42 U.S.C. § 1320a-7, or otherwise deemed ineligible for participation in health care or federal or state programs; or (d) is thesubject of a conviction described in 21 U.S.C. 335a or is subject to any similar sanction. 1.83.“Deemed Sublicensee Distributor” means any Third Party appointed by Janssen or any of its Affiliates or its or their sublicensees to distribute,market, and sell Product, with or without packaging rights, in one or more countries in the Territory, in circumstances where: (a) the Third Partypurchases Product from Janssen or its Affiliates or its or their sublicensees and otherwise makes an upfront, royalty or other payment (separatefrom a payment for supply of Product) to Janssen or its Affiliates or its or their sublicensees with respect to Product; or (b) the Third Partyengages in material promotional activity under a co-promotion agreement with Janssen with respect to Product. For clarity, a DeemedSublicensee Distributor shall be deemed a Sublicensee for the purpose of Net Sales and corresponding Royalty calculations. 1.84.“Develop” or “Development” means any and all drug development activities, other than Research activities, conducted before or afterobtaining Regulatory Approval that are reasonably related to or leading to the development, preparation and submission of data andinformation to a Regulatory Authority for the purpose of obtaining, supporting or expanding Regulatory Approval or to the appropriate bodyfor obtaining, supporting or expanding Pricing Approval, including all activities related to pharmacokinetic profiling, design and conduct ofClinical Studies, regulatory affairs, regulatory strategy, safety matters, statistical analysis, report writing, and Regulatory Filing creation andsubmission (including the services of outside advisors and consultants in connection therewith). 1.85.“Development Milestone Event” has the meaning set forth in Section 10.6. 1.86.“Development Milestone Payment” has the meaning set forth in Section 10.6. 1.87.“Disclosing Party” has the meaning set forth in Section 12.1. 1.88.“Dispute” has the meaning set forth in Section 18.1(a). 1.89.“Distributor” means any Third Party appointed by Janssen or any of its Affiliates or its or their sublicensees to distribute, market, and sellProduct, with or without packaging rights, in one or more countries in the Territory, in circumstances where: (a) the Third Party purchasesProduct from Janssen or its Affiliates or its or their sublicensees, but does not otherwise make any upfront, royalty or other payment (separatefrom a payment for supply of Product) to Janssen or its Affiliates or its or their sublicensees with respect to Product; and (b) the Third Party doesnot engage in any material promotional activity under a co-promotion agreement with Janssen with respect to Product. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -10-Confidential Treatment Requested by MeiraGTx Holdings plc 1.90.“Dollar” or “Dollars” or “$” means the legal tender of the United States of America. 1.91.“Drug Master File” means, for each Product, the drug master file that includes detailed information about such Product, processes, facilities,articles or Materials used in the Manufacture of such Product, which is or is intended to be submitted to a Regulatory Authority, including“Drug Master Files” as defined in 21 C.F.R. § 314.420, any non-United States equivalents and any equivalent information applicable for a GeneTherapy Product. 1.92.“Effective Date” has the meaning set forth in Section 14.1 1.93.“EMA” means the European Medicines Agency or any successor entity thereto. 1.94.“End-of-Phase 2 Meeting” means any “End-of-Phase 2 Meeting” as described in 21 CFR 312.47, or, with respect to the EU, a similar meetingwith the EMA. 1.95.“EU” means the European Union, as its membership may be constituted from time to time, and any successor thereto; except that, for purposesof this Agreement, the EU will be deemed to include [***]. 1.96.“European Commission” means the executive of the EU that promotes its general interest. 1.97.“Execution Date” has the meaning set forth in the first paragraph of this Agreement. 1.98.“Executive Officers” means the Chief Executive Officer of MeiraGTx and the Global Head of Janssen Research and Development of Janssen. 1.99.“Existing Third Party Obligations” has the meaning set forth in Section 10.10(a). 1.100.“FCPA” means the U.S. Foreign Corrupt Practices Act (15 U.S.C. § 78dd-1, et seq.). 1.101.“FDA” means the United States Food and Drug Administration or any successor entity thereto. 1.102.“Field” means the diagnosis, prevention or treatment of diseases and other conditions in all Indications in humans. 1.103.“Final Report” means, with respect to each Research Plan, a final report containing, at a minimum, the contents of Exhibit 1.103, including[***]. A Final Report must contain the results of Research Plan Activities that [***]. 1.104.“Final Report Delivery Date” has the meaning set forth in Section 3.7(c). 1.105.“First Commercial Sale” means, with respect to any Product, and on a country-by-country basis, the first commercial sale in an arm’s lengthtransaction of such Product to a Third Party by Janssen, its Affiliates or Sublicensees in such country following receipt of applicable RegulatoryApproval and Pricing Approval of such Product in such country. For Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -11-Confidential Treatment Requested by MeiraGTx Holdings plc clarity, the First Commercial Sale of a Product shall not include: (a) any distribution or other sale solely where the Product is supplied withoutcharge or at the actual Manufacturing cost thereof (without allocation of indirect costs or any markup); (b) any sale by Janssen to its Affiliatesor Sublicensees for further re-sale by such Affiliate or Sublicensee; or (c) sales for clinical trial purposes, early access or compassionate useprograms. 1.106.[***] 1.107.“Force Majeure” has the meaning set forth in Section 19.4. 1.108.“FTE” means a full-time person, or more than one person working the equivalent of a full-time person, working directly on performingResearch Plan Activities, Clinical Development Plan Activities or CMC Development Plan Activities, where “full-time” is considered [***] perCalendar Year. For clarity, indirect personnel (including support functions such as managerial, financial, legal or business development) shallnot constitute FTEs. 1.109.“FTE Costs” means, for any period, the FTE Rate multiplied by the number of FTEs in such period. 1.110.“FTE Rate” means [***] The FTE Rate shall be adjusted annually in proportion to the Consumer Price Index for all Urban Consumers for [***]to [***], as published by the U.S. Department of Labor, Bureau of Statistics (national CPI-U; Base Period: 1982-84=100; available athttp://www.bls.gov/cpi/home.htm). Rate changes shall be effective as of the first (1st) day of the first (1st) Calendar Quarter of the Calendar Year.Notwithstanding the foregoing, for any time period during the Term that is less than a full Calendar Year, the above referenced rate will beproportionately reduced to reflect such portion of FTEs for such full Calendar Year. 1.111.“Future Third Party Obligations” means any Intellectual Property Rights of a Third Party that Cover the composition of matter, use orManufacture of, or are necessary or useful to use or make, a Product, excluding any Intellectual Property Rights under any Existing Third PartyObligation. 1.112.“GAAP” means accounting principles generally accepted in the United States of America, as in effect from time to time, consistently applied. 1.113.“Gene Therapy Product” means any [***] product that delivers [***] for purposes of [***]. 1.114.“Governing Law” has the meaning set forth in Section 18.2. 1.115.“Governmental Authority” means any applicable government authority, court, tribunal, arbitrator, agency, department, legislative body,commission or other instrumentality of: (a) any government of any country or territory; (b) any nation, state, province, county, city or otherpolitical subdivision thereof; (c) any supranational body; or (d) any arbitrator with binding authority. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -12-Confidential Treatment Requested by MeiraGTx Holdings plc 1.116.“HSR Act” means the Hart-Scott-Rodino Act of 1976. 1.117.“HSR Clearance Date” means the earliest date on which the Parties have actual knowledge that all applicable waiting periods under the HSRAct with respect to the transactions contemplated under this Agreement have expired or have been terminated. 1.118.“HSR Filing” means filings by Janssen and MeiraGTx with the United States Federal Trade Commission and the United States Department ofJustice of a Notification and Report Form for Certain Mergers and Acquisitions (as that term is defined in the HSR Act) with respect to thematters set forth in this Agreement, together with all required documentary attachments thereto, or any foreign form substantially equivalentthereto. 1.119.“Improved Clinical IRD Product” means, for any Clinical IRD Product, any Gene Therapy Product that: (a) contains [***]; and (b) [***]. 1.120.“Improved Janssen Research IRD Product” means, for any Janssen Research IRD Product, any Gene Therapy Product that: (a) contains [***];and (b) [***]. 1.121.“IND” means an Investigational New Drug Application (including any amendments thereto) filed with the FDA pursuant to 21 CFR Part 312before the commencement of a clinical trial of a Product, or a similar application filed with an applicable Regulatory Authority outside of theUnited States such as a clinical trial application or a clinical trial exemption, or any other equivalent or related regulatory submission, licenseor authorization. 1.122.“IND Submission” means the submission of an IND to a Regulatory Authority in a Major Market Country. 1.123.“Indemnification Claim Notice” has the meaning set forth in Section 17.3(b). 1.124.“Indemnified Party” has the meaning set forth in Section 17.3(b). 1.125.“Indemnifying Party” has the meaning set forth in Section 17.3(b). 1.126.“Indemnitee” means a MeiraGTx Indemnitee or a Janssen Indemnitee, as the context requires. 1.127.“Indication” means (a) any disease, condition or syndrome, or (b) any sign or symptom of or associated with a disease, condition or syndrome. 1.128.“Initial Research Plan Term” has the meaning set forth in Section 2.2(b). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -13-Confidential Treatment Requested by MeiraGTx Holdings plc 1.129.“Insolvency Event” means, in relation to either Party, any one of the following: (a) that Party becomes insolvent according to Applicable Law;(b) that Party is the subject of voluntary or involuntary bankruptcy proceedings instituted on behalf of or against such Party (except forinvoluntary bankruptcy proceedings, which are dismissed within [***]; (c) an administrative receiver, receiver and manager, interim receiver,custodian, sequestrator, or similar officer is appointed in respect of that Party; (d) a notice shall have been issued to convene a meeting for thepurpose of passing a resolution to wind up that Party, or such a resolution shall have been passed other than a resolution for the solventreconstruction or reorganization of that Party; (e) a resolution shall have been passed by that Party or that Party’s directors to make anapplication for an administration order or to appoint an administrator; or (f) that Party proposes or makes any general assignment, composition,or arrangement with or for the benefit of all or some of that Party’s creditors or makes or suspends or threatens to suspend making payments toall or some of that Party’s creditors. 1.130.“Intellectual Property Rights” means any Know-How, Inventions, Patents, and copyrights. 1.131.“Interest Rate” has the meaning set forth in Section 10.13(f). 1.132.“Interim Report” has the meaning set forth in Section 3.7(b). 1.133.“Internal Research” means non-commercial research activities conducted by Janssen. 1.134.“Invention” means any process, method, composition of matter, article of manufacture, discovery, improvement, or finding, includingKnow-How, patentable or otherwise, that is first developed, generated, conceived or reduced to practice as a result of a Party (acting solely orjointly with the other Party) exercising its rights or carrying out its obligations in accordance with this Agreement, whether directly or via itsAffiliates, employees, agents or independent contractors, including all rights, title and interests in and to the intellectual property rights in andto any of the foregoing. 1.135.“IRD Gene” means a gene whose loss of function is responsible for an inherited retinal disease. 1.136.“J&J Universal Calendar” means the Johnson & Johnson universal calendar for a given year. The J&J Universal Calendar for 2019 and 2020is set forth on Exhibit 1.136 attached hereto, and the J&J Universal Calendar for subsequent years will be provided to MeiraGTx by Janssenupon MeiraGTx’s request in writing from time to time. 1.137.“Janssen” has the meaning set forth in the first paragraph of this Agreement. 1.138.“Janssen Indemnitees” has the meaning set forth in Section 17.1. 1.139.“Janssen Quality Requirements” means, with respect to (a) MeiraGTx’s Manufacturing or supply obligations for any Products under thisAgreement and (b) the Clinical Studies to be conducted pursuant to this Agreement, in each case ((a) and (b)) the standards and requirementsthat meet Janssen’s or any Regulatory Authorities’ quality requirements. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -14-Confidential Treatment Requested by MeiraGTx Holdings plc 1.140.“Janssen Research IRD Patents” has the meaning set forth in Section 11.4(d)(ii). 1.141.“Janssen Research IRD Product” means (a) any Research IRD Product for which Janssen has exercised the applicable Option in accordancewith Section 3.9(b), and (b) any Improved Janssen Research IRD Product. 1.142.“Janssen Technology” means all Patents, Inventions and Know-How Controlled by Janssen or its Affiliates during the Term that are necessaryto conduct the Research Plan Activities for a Research IRD Product in accordance with the respective Research Plan, conduct the ClinicalDevelopment Plan Activities for a Clinical IRD Product in accordance with the respective Clinical Development Plan or conduct the CMCDevelopment Plan Activities in accordance with the respective CMC Development Plan, in each case of the foregoing to the extent the resultsof such activities are or will be incorporated into, with Janssen’s prior written permission, any Clinical IRD Product, Research IRD Product orManufacturing process therefor. 1.143.“Joint Development Committee” or “JDC” means the committee established as set forth in Section 5.4(a). 1.144.“Joint Inventions” has the meaning set forth in Section 11.1(c). 1.145.“Joint Manufacturing Committee” or “JMC” means the committee established as set forth in Section 5.5(a). 1.146.“Joint Patents” has the meaning set forth in Section 11.1(c). 1.147.“Joint Product Patents” mean all Joint Patents that Cover (a) one or more components of a Product; or (b) any approved use of a Product; [***]. 1.148.“Joint Research Committee” or “JRC” means the committee established as set forth in Section 5.3(a). 1.149.“Joint Steering Committee” or “JSC” means the committee established as set forth in Section 5.2(a). 1.150.“Joint Technology” means Joint Patents and Joint Inventions. 1.151.“JRD” has the meaning set forth in Section 10.13(g). 1.152.“Know-How” means any non-public or proprietary information and all other proprietary rights (including technical and scientific information)that may exist or be created under the laws of any jurisdiction in the world, including technical information, know-how, data (includingpharmacological, toxicological, non-clinical and clinical data, analytical and quality control data, Manufacturing data and descriptions, marketdata, financial data or descriptions), Materials, research results, inventions (whether patentable or not), discoveries, trade secrets, specifications,instructions, processes, formulae, expertise, other Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -15-Confidential Treatment Requested by MeiraGTx Holdings plc technology applicable to compounds, formulations, compositions or products, to their Manufacture, Development, registration, use orCommercialization, methods of assaying or testing them or processes for their Manufacture, formulations containment, compositionsincorporating or comprising them, including all biological, chemical, pharmacological, biochemical, toxicological, pharmaceutical, physicaland analytical, safety, quality control, Manufacturing, preclinical and clinical data, Regulatory Filings or Regulatory Materials and copiesthereof, relevant to the Development, Manufacture, use or Commercialization of or which may be useful in studying, testing, development,production or formulation of products, or intermediates for the synthesis thereof, but excluding Patents. 1.153.“Licensed Patent Action” has the meaning set forth in Section 11.5(a). 1.154.“Loss of Market Exclusivity Due to a Biosimilar Product” means, with respect to any Product in any country, that both of the followingevents have occurred: (a) [***] and (b) one or more Biosimilar Product(s) of such Product are marketed or sold by one or more Third Parties whoare not licensees or Sublicensees of Janssen or its Affiliates in such country. 1.155.“Loss of Market Exclusivity Due to a Competing Product” means, with respect to any [***] in any country, that all of the following eventshave occurred: (a) one or more Competing Product(s) of such [***] (other than a Biosimilar Product of such [***]) are marketed or sold by oneor more Third Parties who are not licensees or Sublicensees of Janssen or its Affiliates in such country; [***] 1.156.“MAA” means an application for the authorization or approval to market Product(s) in any country or group of countries outside the UnitedStates, as defined by Applicable Law and filed with the Regulatory Authority of a given country or group of countries. 1.157.“Major European Countries” means [***]. 1.158.“Major Market Countries” means the [***]. 1.159.“Manufacture” or “Manufacturing” means activities directed to producing, manufacturing, processing, sourcing of materials, filling, finishing,packaging, labeling, quality assurance testing and release, shipping and storage of a product. 1.160.“Manufacturing Costs” means Clinical Development Manufacturing Costs or Commercial Manufacturing Costs, as applicable to the Product.For clarity, in no event shall [***]. 1.161.“Manufacturing Services Agreement” has the meaning set forth in Section 8.5. 1.162.“Materials” means any tangible compositions of matter, articles of manufacture, assays, chemical, biological or physical materials, and othersimilar materials, including media composition. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -16-Confidential Treatment Requested by MeiraGTx Holdings plc 1.163.“Medical Affairs” means activities conducted by a Party’s or its Affiliate’s medical affairs department, including communications with keyopinion leaders, medical education, symposia, advisory boards (to the extent related to medical affairs or clinical guidance), activitiesperformed in connection with patient registries, and other medical programs and communications, including educational grants, research grants(including conducting investigator-initiated studies), and charitable donations to the extent related to medical affairs and not to other activitiesthat do not involve the promotion, marketing, sale, or other Commercialization of Products and are not conducted by a Party’s medical affairsdepartment. 1.164.“MeiraGTx” has the meaning set forth in the first paragraph of this Agreement. 1.165.“MeiraGTx Facility” means the Manufacturing facility operated by MeiraGTx or its Affiliate located at 92 Britannia Walk, London, N1 7NQ,United Kingdom, or such other Manufacturing facility operated by MeiraGTx or one of its Affiliates as mutually agreed by the Parties inwriting. 1.166.“MeiraGTx Indemnitees” has the meaning set forth in Section 17.2. 1.167.“MeiraGTx Know-How” means any Know-How or Inventions Controlled by MeiraGTx or any of its Affiliates as of the Execution Date orthereafter until this Agreement expires or terminates, which are: (a) used in or otherwise relating to any Clinical IRD Product; or (b) otherwiseused by MeiraGTx in the Development, Manufacture, or Commercialization of any Clinical IRD Product in the Field. The MeiraGTxKnow-How excludes (y) any Know-How or Inventions embodied solely in the MeiraGTx [***] Technology and (z) any Joint Inventions. 1.168.“MeiraGTx Patents” means any Patents Controlled by MeiraGTx or any of its Affiliates as of the Execution Date or thereafter until thisAgreement expires or terminates, which Cover any Clinical IRD Products, Clinical IRD Targets, MeiraGTx Know-How in the Field or ClinicalDevelopment Plan Activities, including such Patents set forth on Exhibit 1.168. The MeiraGTx Patents exclude (a) any Patents that solelyCover the MeiraGTx [***] Technology and (b) any Joint Patents. 1.169.“MeiraGTx Research Know-How” means any Know-How or Inventions Controlled by MeiraGTx or any of its Affiliates as of the ExecutionDate or thereafter until this Agreement expires or terminates that are used in or otherwise relating to any Research IRD Product (including anyJanssen Research IRD Product). The MeiraGTx Research Know-How excludes (a) any Know-How or Inventions embodied solely in theMeiraGTx [***] Technology and (b) any Joint Inventions. 1.170.“MeiraGTx Research Patents” means any Patents Controlled by MeiraGTx or any of its Affiliates as of the Execution Date or thereafter untilthis Agreement expires or terminates that claim or otherwise Cover any Research IRD Products (including any Janssen Research IRD Products),Research IRD Targets or MeiraGTx Research Know-How in the Field or otherwise Cover any Research Plan Activities. The MeiraGTx ResearchPatents exclude (a) any Patents that solely Cover the MeiraGTx [***] Technology and (b) any Joint Patents. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -17-Confidential Treatment Requested by MeiraGTx Holdings plc 1.171.“MeiraGTx Research Technology” means the MeiraGTx Research Know-How and the MeiraGTx Research Patents. 1.172.“MeiraGTx [***] Technology” means MeiraGTx’s [***] technology, which directly relates to [***]. 1.173.“MeiraGTx Technology” means the MeiraGTx Know-How and the MeiraGTx Patents. 1.174.“Missing Information Notice” has the meaning set forth in Section 3.7(c). 1.175.“Natural History Study” means a human clinical study that follows a group of people over time who have, or are at risk of developing, aspecific medical condition or disease. 1.176.“Net Sales” means the gross amounts invoiced on sales of a Product by Janssen, or any of its Affiliates or Sublicensees, to a Third Partypurchaser in an arms-length transaction, less the following customary and commercially reasonable deductions, determined in accordance withJanssen’s Accounting Standards and internal policies and actually taken, paid, accrued, allocated or allowed based on good faith estimates:(a) trade, cash or quantity discounts, allowances and credits, excluding commissions for commercialization;(b) excise taxes, use taxes, tariffs, sales taxes and customs duties, or other government charges imposed on the sale of Product (including valueadded tax, but only to the extent that such value added taxes are not reimbursable or refundable), and specifically excluding, for clarity, anyincome taxes assessed against the income arising from such sale;(c) compulsory or negotiated payments and cash rebates or other expenditures to governmental authorities (or designated beneficiaries thereof)in the context of any national or local health insurance programs or similar programs; including pay-for-performance agreements, risk sharingagreements and government levied fees as a result of the Affordable Care Act;(d) rebates, chargebacks, administrative fees and discounts (or equivalent thereof) to managed health care organizations, group purchasingorganizations, insurers, pharmacy benefit managers (or equivalent thereof), specialty pharmacy providers, governmental authorities or theiragencies or purchasers, reimbursers or trade customers, as well as amounts owed to patients through co-pay assistance cards or similar forms ofrebate to the extent the latter are directly related to the prescribing of Product;(e) outbound freight, shipment and insurance costs to the extent included in the price and separately itemized on the invoice price; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -18-Confidential Treatment Requested by MeiraGTx Holdings plc (f) retroactive price reductions, credits or allowances actually granted upon claims, rejections or returns of Product, including for recalls ordamaged or expired goods, billing errors and reserves for returns;(g) any invoiced amounts which are not collected by the selling Party or its Affiliates or Sublicensees, including bad debts; and(h) any deductions in the context of payments that are due or collected significantly after invoice issuance.All aforementioned deductions shall only be allowable to the extent they are commercially reasonable by Janssen and shall be determined, on acountry-by-country basis, as incurred in the ordinary course of business in type and amount verifiable based on Janssen’s and its Affiliates’reporting system. All such discounts, allowances, credits, rebates, and other deductions shall be fairly and equitably allocated to Product andother products of Janssen and its Affiliates and Sublicensees such that Product does not bear a disproportionate portion of such deductions.The following shall be excluded from Net Sales calculations for all purposes: (a) sales of Product for the use in conducting Clinical Studies orother scientific testing of Product in a country, (b) compassionate and named patient sales or sales on an Affordable Basis and (c) anydisposition of Product as free samples, donations, patient assistance, test marketing programs or other similar programs or studies. 1.177.“Non-Withholding Party” has the meaning set forth in Section 10.13(d). 1.178.“Operational Team” has the meaning set forth in Section 5.6. 1.179.“Option” has the meaning set forth in Section 3.9(b). 1.180.“Option Election Date” has the meaning set forth in Section 3.9(b). 1.181.“Option Exercise Notice” has the meaning set forth in Section 3.9(b). 1.182.“Option Fee” has the meaning set forth in Section 3.9(b). 1.183.“Option Opt-Out” has the meaning set forth in Section 3.9(b). 1.184.“Option Opt-Out Date” has the meaning set forth in Section 3.9(b). 1.185.“Option Period” means, for a given Research IRD Product, the period (a) beginning on the date that the Parties finalize a Research Plan for suchResearch IRD Target associated with such Research IRD Product and (b) expiring on (i) such date that is [***] after the Final Report DeliveryDate with respect to such Research IRD Target associated with such Research IRD Product or (ii) such date that the Parties otherwise mutuallyagree to in writing. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -19-Confidential Treatment Requested by MeiraGTx Holdings plc 1.186.“Out-of-Pocket Costs” means, with respect to certain activities hereunder, direct expenses paid or payable by either Party or its Affiliates toThird Parties for services or materials specifically identifiable and incurred to conduct such activities for such Product in the Territory,including payments to contract personnel (including contractors, consultants and subcontractors) in each case, pursuant to the applicableClinical Development Plan, Research Plan or CMC Development Plan, and provided that such expenses shall have been recorded as incomestatement items in accordance with such Party’s Accounting Standards and shall not include any pre-paid amounts, capital expenditures, oritems intended to be covered by the FTE Rate. 1.187.“Party” or “Parties” has the meaning set forth in the first paragraph of this Agreement. 1.188.“Patents” means any and all (a) patents, (b) pending patent applications, including all provisionals applications, divisionals, continuations,substitutions, continuations-in-part, divisions and renewals, and all patents granted thereon, (c) all patents-of-addition, reissues, reexaminationsand extensions or restorations by existing or future extension or restoration mechanisms, including supplementary protection certificates or theequivalent thereof, (d) inventor’s certificates, (e) any other form of government-issued right substantially similar to any of the foregoing, and(f) all United States and foreign counterparts of any of the foregoing. 1.189.“Person” means any individual, limited liability or general partnership, limited liability company, joint venture, firm, corporation, association,trust, unincorporated organization or other entity or body, including a Governmental Authority. 1.190.“Phase 1 Study” means a clinical study of an investigational product in patients with the primary objective of characterizing its safety,tolerability, and pharmacokinetics and identifying a recommended dose and regimen for future studies. The investigational product can beadministered to patients as a single agent or in combination with other investigational or marketed agents and shall be deemed commencedwhen the first patient in such study has received his or her initial dose of a product. A “Phase 1 Study” shall include any clinical trial that wouldsatisfy the requirements of 21 C.F.R. § 312.21(a), or a comparable clinical study prescribed by the relevant Regulatory Authority in a countryother than the United States. 1.191.“Phase 1/2 Study” means a combined Phase 1 Study and Phase 2 Study. 1.192.“Phase 2 Study” means a clinical study of an investigational product in patients with the primary objective of characterizing its activity in aspecific disease state as well as generating more detailed safety, tolerability, and pharmacokinetics information. The investigational productcan be administered to patients as a single agent or in combination with other investigational or marketed agents and shall be deemedcommenced when the first patient in such study has received his or her initial dose of a product. A “Phase 2 Study” shall include any clinicaltrial that would satisfy the requirements of 21 C.F.R. § 312.21(b), or a comparable clinical study prescribed by the relevant RegulatoryAuthority in a country other than the United States. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -20-Confidential Treatment Requested by MeiraGTx Holdings plc 1.193.“Phase 3 Study” means a clinical study of an investigational product in patients that incorporates accepted endpoints for confirmation ofstatistical significance of efficacy and safety with the aim to obtain Regulatory Approval in any country as described in 21 C.F.R. § 312.21(c),or a comparable clinical study prescribed by the relevant Regulatory Authority in a country other than the United States. The investigationalproduct can be administered to patients as a single agent or in combination with other investigational or marketed agents and shall be deemedcommenced when the first patient in such study has received his or her initial dose of a product. For clarity, Phase 3 Studies include clinicalstudies of approved products for unapproved Indications. 1.194.“Pivotal Study” means a human clinical study in any country that is prospectively designed to generate data intended to satisfy therequirements of 21 C.F.R. § 312.21(c) in the U.S. or a similar clinical study prescribed by a Regulatory Authority from another country, fromtime to time, pursuant to Applicable Law. For clarity, a Pivotal Study may be a Phase 2 Study, a Phase 1/2 Study, or a Phase 3 Study. 1.195.“PO” has the meaning set forth in Section 10.13(c). 1.196.[***] 1.197.“Pricing Approval” means, with respect to a product and any country or regulatory jurisdiction, any pricing and reimbursement approvals thatare commercially necessary to conduct a launch of such product in such country or regulatory jurisdiction (even if such approvals are notlegally required to launch such product in such country or regulatory jurisdiction). For purposes of illustration, with respect to the MajorEuropean Countries, the following pricing and reimbursement approvals are examples of those that are currently necessary to conduct a launchof a drug or biological product: in France, publication of the reimbursed price level in the official journal and registration on a reimbursementlist by or on behalf of Comité Economique des Produits de Santé or Haute Autorité de Santé (or a successor agency); in Italy, publication ofreimbursement in the Government’s Official Gazette (by Agenzia Italiana del Farmaco or a successor agency); in Germany, execution ofcontract with the head association of sick funds (GKV-Spitzenverband, Gesetzlichen Krankenversicherung, or a successor agency); in Spain,authorization by La Comisión Interministerial de Precios de los Medicamentos or La Comisión Nacional para el Uso Racional de losMedicamentos (or a successor agency) for national patient access to reimbursement by or on behalf of a Governmental Authority; and in theUnited Kingdom, a recommendation by the National Institute for Health and Care Excellence (or a successor agency) to obtain mandatoryfunding to enable broad market access. 1.198.“Prior CDA” means that certain Confidential Disclosure Agreement, dated [***]. 1.199.“Priority Review Voucher” means a priority review voucher issued by the United States Department of Health and Human Services thatentitles the holder of such voucher to Priority Review of a single human drug application submitted under Section 505(b)(1) of the Act orSection 351(a) of the United States Public Health Service Act, as further defined in Section 529(a)(2) of the Act (21 U.S.C. § 360ff(a)(2)). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -21-Confidential Treatment Requested by MeiraGTx Holdings plc 1.200.“Privacy and Data Security Laws” means all applicable privacy, security and data protection laws, rules, regulations, and guidelines withrespect to privacy, security and data protection including the collection, processing, storage, protection and disclosure of Sensitive Information. 1.201.“Process Development Activities” has the meaning set forth in Section 8.4(a). 1.202.“Process Development Budget” has the meaning set forth in Section 8.4(c). Each Process Development Budget, upon approval in accordancewith the terms and conditions herewith, will be automatically incorporated into this Agreement by reference and form a part of this Agreement. 1.203.“Process Development Costs” means the fully-burdened cost incurred on or after the Effective Date by either Party or any of their respectiveAffiliates in connection with the performance of any Process Development Activities in accordance with this Agreement and consistent with theapplicable CMC Development Plan, including all FTE Costs, Out-of-Pocket Costs, and depreciation of capital expenditures. 1.204.“Product” means, as applicable, any Clinical IRD Product or Janssen Research IRD Product. 1.205.“Product Infringement” has the meaning set forth in Section 11.6. 1.206.“Product Marks” has the meaning set forth in Section 11.8. 1.207.“Qualified Change of Control” means, with respect to MeiraGTx, a Change of Control in which the acquiring Person, as of the time of suchChange of Control, (a) [***] or (b) [***]. 1.208.“Quality Agreement” means, as applicable, the Clinical Quality Assurance Agreement or the Commercial Quality Assurance Agreement. 1.209.“Receiving Party” has the meaning set forth in Section 12.1. 1.210.“Regulatory Approval” means, with respect to each product in any country or jurisdiction, the approval of the applicable RegulatoryAuthority necessary for the marketing and sale of such product in such country or jurisdiction by the relevant Regulatory Authority, excludingseparate pricing or reimbursement approvals that may be required, as it may be amended or updated from time to time. 1.211.“Regulatory Authority” means any Governmental Authority responsible for granting Regulatory Approvals for Products, including the FDA,EMA, European Commission and any corresponding national or regional regulatory authorities. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -22-Confidential Treatment Requested by MeiraGTx Holdings plc 1.212.“Regulatory Filings” means, with respect to any product, any application or submission to a Regulatory Authority of any appropriateregulatory application, and shall include any submission to a regulatory advisory board, MAA, and any supplement or amendment thereto. Forthe avoidance of doubt, Regulatory Filings shall include any BLA or the corresponding application in any other country or group of countries. 1.213.“Regulatory Lead Party” means, on a Clinical IRD Product-by-Clinical IRD Product basis, the Party allocated primary responsibility for allregulatory matters relating to such Clinical IRD Product including all Regulatory Filings and related Regulatory Materials in accordance withSection 7.1(a). 1.214.“Regulatory Materials” means any notifications, communication, correspondence, registrations, approvals, or other filings made to, receivedfrom or otherwise conducted with a Regulatory Authority related to Developing, Manufacturing, or otherwise Commercializing abiopharmaceutical product in a particular country or jurisdiction, other than Regulatory Filings. 1.215.“Research” or “Researching” means activities, other than Development, Manufacturing and Commercialization, related to the advance,design, delivery, discovery, generation, identification, optimization, profiling, characterization, production, process development, cell linedevelopment, pre-clinical development or non-clinical or pre-clinical studies of drug candidates and products, including such non-clinicalstudies and other material Development activities to be undertaken to generate data sufficient to enable the filing of an IND. 1.216.“Research Budget” has the meaning set forth in Section 3.4. Each Research Budget, upon approval in accordance with the terms andconditions herewith, will be automatically incorporated into this Agreement by reference and form a part of this Agreement. 1.217.“Research Budget Cap” has the meaning set forth in Section 3.4. 1.218.“Research Costs” has the meaning set forth in Section 3.8. 1.219.“Research IRD Patents” has the meaning set forth in Section 11.4(d)(i). 1.220.“Research IRD Product” has the meaning set forth in Section 3.2. 1.221.“Research IRD Target” means any IRD Gene nominated and approved by the JRC in accordance with Section 3.1. 1.222.“Research IRD Target Indication” means, with respect to any Research IRD Target, the inherited retinal disease resulting from the loss offunction of such Research IRD Target, as specified in a Research Plan. 1.223.“Research Plan” means the strategic plan for Researching a Research IRD Product, as such plan may be agreed to, approved, amended orupdated from time to time in accordance with Section 3.2. Each Research Plan, upon approval in accordance with the terms and conditionsherewith, will be automatically incorporated into this Agreement by reference and form a part of Exhibit 1.223. In addition, each initialResearch Plan will be attached hereto as a sequentially numbered part of Exhibit 1.223 upon approval in accordance with this Agreement (e.g.,Exhibit 1.223-1, Exhibit 1.223-2). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -23-Confidential Treatment Requested by MeiraGTx Holdings plc 1.224.“Research Plan Activities” has the meaning set forth in Section 3.2. 1.225.“Research Plan Term” means, subject to Section 2.2(b), the Initial Research Plan Term, together with if applicable, the Research Tail Period. 1.226.“Research Records” has the meaning set forth in Section 3.5. 1.227.“Research Results” mean all tangible Materials, and copies of all material data, results, and research records relating to a Research IRD Productgenerated in connection with a Research Plan. 1.228.“Research Tail Notice” has the meaning set forth in Section 2.2(b). 1.229.“Research Tail Period” has the meaning set forth in Section 2.2(b). 1.230.“Residuals” means [***] hereunder. 1.231.“Review Period” has the meaning set forth in Section 13.4(a). 1.232.“Right of Reference” means the “right of reference or use” as that term is defined in 21 C.F.R. § 314.3(b) and any foreign counterpart to suchregulation. 1.233.“Royalty” or “Royalties” has the meaning set forth in Section 10.8. 1.234.“Royalty Term” has the meaning set forth in Section 10.9(a). 1.235.“[***] Product” means: (a) MeiraGTx’s Gene Therapy Product [***] for the treatment of the [***] Target Indication by expressing the [***]Target; [***]. 1.236.“[***] Product Opt-In Date” has the meaning set forth in Section 2.1(b)(iii). 1.237.“[***] Target” means the [***]. 1.238.“[***] Target Indication” means the inherited retinal disease resulting from the loss of function of the [***] Target. 1.239.“RPGR Product” means: (a) MeiraGTx’s Gene Therapy Product [***] for the treatment of the RPGR Target Indication by expressing the RPGRTarget; [***]. 1.240.“RPGR Target” means the [***]. 1.241.“RPGR Target Indication” means the inherited retinal disease resulting from the loss of function of the RPGR Target. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -24-Confidential Treatment Requested by MeiraGTx Holdings plc 1.242.“Sales & Royalty Report” means a written report or reports showing each of: (a) the Net Sales of each Product in the Territory, on a US andex-US basis, during the reporting period by Janssen and its Affiliates and Sublicensees; and (b) the Royalties payable, in United States Dollars,which shall have accrued hereunder with respect to such Net Sales. 1.243.[***] 1.244.“Senior Officers” means, for Janssen, the Therapeutic Area Head for Cardio Vascular and Metabolism or another senior executive designee withresponsibilities and seniority comparable thereto, and for MeiraGTx, the Chief Executive Officer of MeiraGTx. 1.245.“Sensitive Information” means personally identifiable information, which information may include names, address, other contact information,social security number, date of birth, passwords, protected health information, biometrics, personal identification numbers and codes or otherinformation or data that is protected by Applicable Laws or can be used for identity theft. 1.246.“Sole Inventions” has the meaning set forth in Section 11.1(b). 1.247.“Sponsorship Transfer Date” means, on a Clinical IRD Product-by-Clinical IRD Product basis and country-by-country basis, the date, [***],on which Janssen shall become the sponsor of the IND for such Clinical IRD Product. 1.248.“Sublicensee” means any Person, other than an Affiliate or a Distributor (but not a Deemed Sublicensee Distributor), to which a Party grants asublicense of any right granted to such Party hereunder in accordance with the terms of this Agreement. 1.249.“Supply Agreement” means, as applicable, the Clinical Supply Agreement or the Commercial Supply Agreement. 1.250.“Target” means, as applicable, any Clinical IRD Target or Research IRD Target. 1.251.“Target Indication” means, with respect to any IRD Gene, the inherited retinal disease resulting from the loss of function of such IRD Gene. 1.252.“Technology” means Patents and Know-How. 1.253.“Term” has the meaning set forth in Section 15.1. 1.254.“Terminated Product” means any Product pursuant to which this Agreement is expired or terminated in accordance with the terms andconditions herein. 1.255.“Territory” means all countries and territories of the world. 1.256.“Third Party” means any Person other than a Party or an Affiliate of a Party. 1.257.“Third Party Infringement” has the meaning set forth in Section 11.5(a). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -25-Confidential Treatment Requested by MeiraGTx Holdings plc 1.258.“Third Party License” means a written agreement between a Party or its Affiliates and a Third Party to license or acquire Third PartyIntellectual Property Rights, including any such agreement entered into as a result of settlement of any claims for infringement of Third PartyIntellectual Property Rights. 1.259.“[***] Clinical Development Budget Period” has the meaning set forth in Section 6.1(d). 1.260.“[***] Clinical Development Manufacturing Budget Period” has the meaning set forth in Section 8.4(b). 1.261.“[***] Process Development Budget Period” has the meaning set forth in Section 8.4(c). 1.262.“[***] Research Budget Period” has the meaning set forth in Section 3.4. 1.263.“Trade Control Laws” mean all applicable statutory and regulatory requirements related to export controls, economic sanctions, tradeembargoes, imports of goods and payment of custom duties. 1.264.“Trademarks” mean all registered and unregistered trademarks, service marks, trade dress, trade names, logos, insignias, symbols, designs, andall other indicia of ownership, and combinations thereof. 1.265.“[***]” means [***]. 1.266.“United States” or “U.S.” means the United States of America, its territories and possessions. 1.267.“U.S. Export Control Laws” means all applicable U.S. laws and regulations relating to the export or re-export of commodities, technologies orservices, including the Export Administration Act of 1979, 24 U.S.C. §§ 2401-2420, the International Emergency Economic Powers Act, 50U.S.C. §§ 1701-1706, the Trading with the Enemy Act, 50 U.S.C. §§ 1 et seq., the Arms Export Control Act, 22 U.S.C. §§ 2778-2779, theInternational Boycott Provisions of Section 999 of the U.S. Internal Revenue Code of 1986, the U.S. Department of Commerce’s ExportAdministration Regulations, the U.S. Department of State’s International Traffic in Arms Regulations, and the economic sanctions programsadministered by the U.S. Department of Treasury’s Office of Foreign Asset Controls. 1.268.“Valid Claim” means: (a) a claim of any issued and unexpired Patent that (i) has not been dedicated to the public, disclaimed, revoked or heldunenforceable or invalid by a decision of a Governmental Authority of competent jurisdiction from which no appeal can be taken, or a decisionof a Governmental Authority of competent jurisdiction that can be appealed, but with respect to which an appeal has not been taken within thetime allowed for appeal, and (ii) has not been disclaimed or admitted to be invalid or unenforceable through reissue, disclaimer or otherwise; or(b) a claim of any pending patent application that (i) has not been cancelled, withdrawn or abandoned, without being re-filed in anotherapplication in Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -26-Confidential Treatment Requested by MeiraGTx Holdings plc the applicable jurisdiction, (ii) has not been finally rejected by an administrative agency or other governmental action from which no appealcan be taken and (iii) has not been pending or filed more than [***] from the earliest possible priority date for such patent application; providedthat if such claim is later issued, it shall from the issuance date forward be deemed to be a Valid Claim. 1.269.“Withholding Party” has the meaning set forth in Section 10.13(d). 2.OVERVIEW OF COLLABORATION 2.1.Overview of Clinical Development Plan Activities.(a) General Scope. The Parties will jointly Develop Clinical IRD Products through seeking Regulatory Approval for each of the Clinical IRDProducts in accordance with the terms and conditions of this Agreement. MeiraGTx will have the primary responsibility to Develop each Clinical IRDProduct in accordance with the Clinical Development Plan for each such Clinical IRD Product, including where applicable, conducting any necessaryDevelopment in order to submit the applicable Regulatory Filings to Regulatory Authorities. With respect to any Clinical IRD Product, followingRegulatory Approval of such Clinical IRD Product in a country, Janssen will be responsible for conducting, at its cost and expense, Commercializationand post-approval Development of such Clinical IRD Product in such country. This Section 2.1(a) is qualified in its entirety by the more detailedprovisions of this Agreement set forth below (including Section 2.1(b) and Section 6.1).(b) [***] Product.(i) [***] Meetings. As of the Execution Date, (A) the [***] Product is deemed not to be a Clinical IRD Product or Research IRD Productunder this Agreement, (B) the [***] Target is deemed not to be a Clinical IRD Target or a Research IRD Target under this Agreement and (C) the [***]Target Indication is deemed not to be a Clinical IRD Target Indication under this Agreement. To the extent not prohibited by Applicable Law, Janssenshall have the right to participate in any [***] in each case relating to the [***] Product, and MeiraGTx shall provide the [***] to Janssen. Prior to any[***], MeiraGTx shall also provide Janssen for review and comment at least [***] in advance of any deadline for comments [***] copies [***] by or onbehalf of MeiraGTx prior to the relevant submission with respect to the [***] Product to Janssen, and incorporate reasonable comments theretoprovided by Janssen.(ii) Meeting Between Parties. Within [***] following the date that MeiraGTx provides to Janssen [***] with respect to the [***] Product,the Parties shall meet to discuss MeiraGTx’s plans for Developing the [***] Product. At or prior to such meeting between the Parties, MeiraGTx shallprovide to Janssen all information in MeiraGTx’s possession or control reasonably necessary for Janssen to evaluate whether to make the [***] Producta Clinical IRD Product hereunder, including MeiraGTx’s [***] with respect to the [***] Product, information regarding [***] of the [***] Product,profile and full statistical analyses of the efficacy and safety of the [***] Product [***], MeiraGTx’s good faith forecasts of all future ClinicalDevelopment Costs of the [***] Product (including a proposed Clinical Development Plan and proposed Clinical Development Budget for the [***]Product as contemplated in Section 6.1(c) and Section 6.1(d)) and [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -27-Confidential Treatment Requested by MeiraGTx Holdings plc (iii) Opt-In Process. Within [***] following the date that the Parties meet to discuss MeiraGTx’s plans for Developing the [***] Productpursuant to Section 2.1(b)(ii), Janssen will notify MeiraGTx in writing whether Janssen will make the [***] Product a Clinical IRD Product hereunder.If Janssen notifies MeiraGTx that Janssen elects to make the [***] Product a Clinical IRD Product hereunder (such date, the “[***] Product Opt-InDate”), then (A) the [***] Product will automatically become a Clinical IRD Product, (B) the [***] Target will automatically become a Clinical IRDTarget, (C) the [***] Target Indication will automatically become a Clinical IRD Target Indication, (D) the Development, Manufacturing andCommercialization terms applicable to Clinical IRD Products shall immediately go into effect in accordance herewith with respect to the [***] Productand (E) Janssen shall [***].(iv) Declining to Opt-In. If Janssen notifies MeiraGTx in writing that Janssen will not elect to make the[***] Product a Clinical IRDProduct hereunder, or if Janssen fails to notify MeiraGTx during such [***] period set forth in Section 2.1(b)(iii) that Janssen will elect to make the[***] Product a Clinical IRD Product hereunder, then, as of such date of notice or as of the end of such [***] period, as applicable: (A) the [***]Product will not become a Clinical IRD Product or Research IRD Product under this Agreement, the [***] Target will not become a Clinical IRD Targetor Research IRD Target under this Agreement, and the [***] Target Indication will not become a Clinical IRD Target Indication or a Research IRDTarget Indication under this Agreement; (B) MeiraGTx will retain its rights with regard to the Research, Development, Manufacture orCommercialization of the [***] Product and [***] Target; (C) MeiraGTx may continue the Research, Development, Manufacture andCommercialization of the [***] Product and [***] Target outside of this Agreement, [***]; (D) Janssen shall have [***]; (E) Janssen shall have noobligation to Develop, Manufacture or Commercialize the [***] Product; (F) Janssen shall have [***]; and (G) each of MeiraGTx and Janssen shall notbe subject to the exclusivity obligations set forth in Section 4.6 with respect to the [***] Target. 2.2.Overview of Research Plan Activities.(a) General Scope. During the applicable Research Plan Term, and in accordance with the terms and conditions of this Agreement, MeiraGTx willconduct Research on each Research IRD Target and design, synthesize, test and optimize Research IRD Products designed to treat the Research IRDTarget Indication associated with such Research IRD Target in accordance with the applicable Research Plan and Janssen’s feedback, with the aim ofachieving IND Submission for each of the Research IRD Products. As part of any Research Plan, upon the Parties’ mutual agreement, MeiraGTx mayinitiate Natural History Studies prior to any IND Submission with respect to the applicable Research IRD Target Indication associated with suchResearch IRD Target. Each Research IRD Target will be Researched according to a separate Research Plan, and Janssen will have an exclusive Optionto select Research IRD Products arising out of any Research Plan Activities to advance such Research IRD Products, at Janssen’s sole cost and expense,into Development and Commercialization in accordance with Section 3.9(b). This Section 2.2(a) is qualified in its entirety by the more detailedprovisions of this Agreement set forth below (including Article 3). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -28-Confidential Treatment Requested by MeiraGTx Holdings plc (b) Research Plan Term. With respect to each Research Plan, MeiraGTx will begin the respective Research Plan Activities on or [***] by the JSCpursuant to Section 3.2. MeiraGTx will continue such Research Plan Activities for [***] thereafter (the “Initial Research Plan Term”). Subject to allthe other terms and conditions of this Agreement, Janssen may elect, at the end of the Initial Research Plan Term, to continue funding any ongoing andpreviously approved Research Plan Activities in accordance with the previously approved Research Budget and Section 3.8, until the respective FinalReport Delivery Date or such other reasonable date mutually agreed by the Parties, by so notifying MeiraGTx in writing at least [***] before the end ofthe Initial Research Plan Term (such additional period, the “Research Tail Period,” and such notice, the “Research Tail Notice”). Any changes duringthe Research Tail Period to the respective ongoing and previously approved Research Plan and Research Plan Activities from the end of the InitialResearch Plan Term shall require the Parties’ mutual written agreement. If MeiraGTx does not receive the Research Tail Notice from Janssen at least[***] before the end of the Initial Research Plan Term, then the Research Plan Activities shall cease [***] after the date that such Research Plan is firstapproved by the JSC. Notwithstanding the foregoing, nothing shall obligate MeiraGTx to conduct any further Research Plan Activities as of (a) theFinal Report Delivery Date with respect to such Research Plan or (b) such date that this Agreement terminates with respect to such Research Plan. 2.3.Overview of CMC Development Plan Activities and Other Manufacturing Activities.During the Term, and in accordance with the terms and conditions of this Agreement and the applicable Supply Agreements: (a) MeiraGTx willManufacture certain Products for Janssen; and (b) [***] Beginning on such date that the Parties finalize a CMC Development Plan and for [***]thereafter (“CMC Development Term”), the Parties will collaborate together to Develop the Manufacturing process used for each clinical programundertaken hereunder in connection with each Clinical IRD Product and Research IRD Product. This Section 2.3 is qualified in its entirety by the moredetailed provisions of this Agreement set forth below (including Article 8). 3.NON-CLINICAL RESEARCH 3.1.Research IRD Targets.During the [***] period beginning on the Effective Date (or, if Janssen provides written notice to MeiraGTx at least [***] prior to the expiration ofsuch [***] period of Janssen’s election to extend such period by another [***], during the [***] period beginning on the Effective Date), the JRC willnominate and approve IRD Genes (including, at the JRC’s discretion, any IRD Gene set forth in Exhibit 1.221) as Research IRD Targets. Subject toSection 3.2, any IRD Gene approved by the JRC as a Research IRD Target will become the subject of a Research Plan. During the applicable ResearchPlan Term or as otherwise mutually agreed by the Parties, MeiraGTx will conduct Research on each such Research IRD Target, subject to the terms andconditions herein. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -29-Confidential Treatment Requested by MeiraGTx Holdings plc 3.2.Research Plans.[***], the Parties will, through the JRC, jointly create a Research Plan for each Research IRD Target. Each Research Plan will set forth for suchResearch IRD Target: [***] The JRC shall develop and submit each initial Research Plan to the JSC for its review and approval, [***] in accordanceherewith, and each initial Research Plan will be automatically attached hereto and form a part of Exhibit 1.223. From time to time, and at least on anannual basis, the JRC will develop, review, and approve amendments or updates to each Research Plan, subject to the terms and conditions of thisAgreement. Each such amended or updated Research Plan will be deemed to be automatically replacing the respective Research Plan previously ineffect, and will be automatically incorporated into this Agreement by reference and form a part of this Agreement. Each Research Plan shall also beconsistent with the terms of this Agreement. 3.3.Conduct of Research Activities.Following approval of each Research Plan as set forth in Section 3.2, with respect to each Research Plan, MeiraGTx will use Commercially ReasonableEfforts to perform (itself or through its Affiliates or subject to Section 4.4, permitted subcontractors) the Research Plan Activities associated with suchResearch Plan in accordance with the applicable Research Plan (including the timelines set forth therein) for the duration of the Research Plan Term. Inperforming its respective Research Plan Activities, MeiraGTx: (a) will conduct such activities in a good scientific manner and in compliance with allApplicable Laws in all material respects, including, where applicable, cGMP, cGLP, cGCP, good pharmacovigilance practices and current internationalregulatory standards; (b) will not employ or use any Debarred Person; and (c) will not use any MeiraGTx [***] Technology during the course ofperforming Research Plan Activities or incorporate any MeiraGTx [***] Technology into any Research IRD Product [***]. 3.4.Research Budget.Each Research Plan will be subject to a rolling budget covering Research Costs associated with the anticipated Research Plan Activities under suchResearch Plan to be performed during the [***] (such [***] period, the “[***] Research Budget Period”), in each case broken down by CalendarQuarter and broken out on a line item basis to show Out-of-Pocket Costs (including for Manufacturing related activities) and FTE Costs of FTEsdirectly engaged to perform each such Research Plan Activity (each, as agreed to, approved, amended or updated from time to time in accordance withthis Section 3.4, a “Research Budget”). No Research Budget for any Calendar Year shall exceed in any event the applicable Calendar Year capprovided in Exhibit 3.4 attached hereto without the prior written consent of both Parties (such cap, as may be amended by the Parties in writing, the“Research Budget Cap”). [***], the JRC shall develop and submit to the JSC for its review and approval, a Research Budget for each Research Plan,and upon the JSC’s approval, such Research Budget will be automatically incorporated into this Agreement by reference and form a part of thisAgreement. Each Research Budget will be reviewed by the JRC and approved by the JSC (a) [***] based on: (i) the Parties’ good faith estimation of theanticipated Research Plan Activities to be conducted during the relevant [***] Research Budget Period; and (ii) information prepared by the Parties ingood faith for their own internal planning processes relating to anticipated Research Plan Activities for such Research IRD Product; or (b) whenever thetotal Research Costs for any given Calendar Quarter are reasonably expected to be at least [***] percent ([***]%) higher than the Research Budget forsuch Calendar Quarter, whether as a result of any amendments to the Research Plan or increases in costs for the Research Plan Activities Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -30-Confidential Treatment Requested by MeiraGTx Holdings plc already planned for such Calendar Quarter. Each such amended or updated Research Budget will automatically replace the respective Research Budgetpreviously in effect, and will be automatically incorporated into this Agreement by reference and form a part of this Agreement. With respect to anyCalendar Quarter, in no event will a Party be responsible for any Research Costs that exceed the respective portion of the then-current Research Budgetrecommended and proposed by the JRC and approved by the JSC, except as otherwise provided in Section 10.2(b) or mutually agreed by the Parties inwriting. 3.5.Research Records.MeiraGTx will prepare and maintain, and cause its Affiliates and their respective employees and permitted subcontractors to prepare and maintain,records, accounts, notes, reports, data and laboratory notebooks with respect to the Research Plan Activities hereunder (“Research Records”), insufficient detail and in a good scientific manner appropriate for scientific, regulatory and intellectual property protection purposes and in compliancewith Applicable Law, which Research Records shall: (a) be segregated from other research activities not performed under this Agreement; and (b) becomplete and accurate, and fully and accurately reflect all work done, data and developments made, and results achieved in the performance of theResearch Plan Activities. MeiraGTx shall retain, and cause its Affiliates and their respective employees and permitted subcontractors to retain,Research Records for at least [***] or such longer period as may be required by Applicable Law. MeiraGTx shall comply with Janssen’s data policiesset forth on Exhibit 3.5 attached hereto with regard to Research Records. 3.6.Audits.With respect to any facility or site at which MeiraGTx conducts any Research Plan Activities, Janssen shall have the right, [***] upon reasonablewritten notice by Janssen, and during normal business hours, to inspect such site and facility of MeiraGTx or to accompany MeiraGTx to inspect anysubcontractor site once per year and also for cause, to verify MeiraGTx’s compliance with Applicable Law in carrying out its obligations under thisAgreement, including those relating to cGMP, cGLP, cGCP and good pharmacovigilance practices. In the event that any such facility or site is found tobe non-compliant with cGMP, cGLP, cGCP or good pharmacovigilance practices during such an audit, and such non-compliance relates to or impactsany Research Plan Activities hereunder, MeiraGTx shall submit to Janssen proposed Corrective and Preventative Actions (“CAPA”) within [***] afterJanssen provides notice of such non-compliance. Janssen shall have the right to review and comment on such CAPA, which comments MeiraGTx shallconsider in good faith. MeiraGTx shall use Commercially Reasonable Efforts to implement such CAPA [***] after review and comment by Janssen.Except as may otherwise be provided in a Supply Agreement, if any Regulatory Authority or any other Governmental Authority conducts or givesnotice of its intent to conduct any audit or inspection at any offices or facilities (including Research facilities) of MeiraGTx or any applicablepermitted subcontractor where such audit or inspection relates to any Research IRD Product, then MeiraGTx will [***] notify Janssen and, to the extentsuch audit or inspection relates to a Research IRD Product and to the extent practicable and not prohibited by Applicable Law, secure for Janssen theright to participate in any such audit or inspection. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -31-Confidential Treatment Requested by MeiraGTx Holdings plc 3.7.Research Reports and Materials.(a) General. MeiraGTx will keep Janssen reasonably informed regarding the status, progress, and results of its Research Plan Activities under eachResearch Plan, including a review of results (including Manufacturing related campaign reports) and progress against timelines in such Research Planthrough regularly scheduled JRC (and, if applicable, Operational Team) meetings. Through the course of such discussions, and notwithstandinganything herein to the contrary, with respect to each Research Plan, the Parties will mutually agree on the lead Research IRD Product to treat theResearch IRD Target Indication with respect to any Research IRD Target (and on the plan for pre-IND Manufacturing of such lead Research IRDProduct for the respective IND).(b) Interim Reports. On [***], with respect to each Research Plan, MeiraGTx shall create and submit to the JRC (and, if applicable, theOperational Team) for its review and discussion, a written report (such report, an “Interim Report”) that includes: (i) a summary of the Research PlanActivities completed during the most recently completed [***] under such Research Plan; (ii) a copy of all results and data generated during suchperiod related to such Research Plan; (iii) progress against the timeline set forth in such Research Plan, with appropriate documentation to substantiateall such activities and results; and (iv) Research Costs incurred in the most recently completed [***] under such Research Plan. MeiraGTx shall alsomake its employees and consultants available for an in-person or telephonic meeting with Janssen at least [***] every [***] to discuss MeiraGTx’sprogress with respect to the conduct of Research Plan Activities.(c) Final Report. MeiraGTx shall provide Janssen with a written Final Report within [***] after the completion or earlier termination of eachResearch Plan. Janssen shall have [***] following receipt of the Final Report to notify MeiraGTx in writing if Janssen in good faith believes that anyinformation is missing from the Final Report (a “Missing Information Notice”), which Missing Information Notice shall identify with reasonable detailsuch required information that Janssen believes is missing. If Janssen delivers a Missing Information Notice to MeiraGTx, MeiraGTx shall deliver themissing required information identified in such Missing Information Notice as soon as practicable to Janssen, and Janssen will [***] notify MeiraGTxin writing upon Janssen’s confirmation that MeiraGTx has provided to Janssen all such missing required information that MeiraGTx must provide (a“Completion Notice”). The Final Report will be deemed complete on the date on which MeiraGTx receives the Completion Notice. If Janssen does notdeliver a timely Missing Information Notice to MeiraGTx within the [***] period described above in this Section 3.7(c), the initial Final Report will bedeemed complete on the date such [***] period lapses. For purposes of this Agreement, “Final Report Delivery Date” means the date upon whichJanssen has received a complete Final Report in accordance with this Section 3.7(c). During the [***] period following the Final Report Delivery Date,MeiraGTx shall: (i) promptly update the Final Report if any new data relating to any Research IRD Product or Research IRD Target becomes available;and (ii) upon Janssen’s reasonable request, provide to Janssen and its representatives reasonable access during normal business hours to MeiraGTx’spersonnel to discuss such updated Final Report. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -32-Confidential Treatment Requested by MeiraGTx Holdings plc 3.8.Research Costs.Subject to Section 10.2, the Parties will be responsible for the documented FTE Costs and Out-of-Pocket Costs, in each case, incurred by or on behalf ofMeiraGTx during the Research Plan Term in accordance with the then-current Research Plan, including the cost of any Natural History Studies that arepart of such Research Plan (collectively, the “Research Costs”) as follows: Janssen will be responsible for [***] percent ([***]%) of all Research Costsunder each Research Plan, and MeiraGTx will be responsible for [***] percent ([***]%) of all Research Costs under each Research Plan, in each case tothe extent that such Research Costs are incurred [***]. All Research Costs will be reconciled and paid in accordance with the procedure described inSection 10.2. Notwithstanding anything herein to the contrary, Janssen will [***]. 3.9.Option.(a) Provision of Information. During the applicable Research Plan Term, Janssen and the JRC and JSC will assess the results provided in eachInterim Report provided under a Research Plan, and the JSC will review and determine whether the Advancement Criteria for the respective ResearchIRD Product have been met. Janssen will determine (based on the Interim Reports or the Final Report for a given Research IRD Product) whether itwishes to further Develop any such Research IRD Product by exercising the Option as set forth in Section 3.9(b).(b) Exercise of Option. Subject to the terms and conditions of this Agreement, with respect to each Research IRD Target, MeiraGTx hereby grantsto Janssen an exclusive (even as to MeiraGTx) option (each, an “Option”), exercisable during the applicable Option Period, to obtain, on a ResearchIRD Target-by-Research IRD Target basis, the exclusive license (as set forth in Section 4.1(b)(ii)) to each Research IRD Product that treats the ResearchIRD Target Indication resulting from the loss of function of such Research IRD Target. With respect to each Research IRD Target, during the applicableOption Period, Janssen may exercise the applicable Option for such Research IRD Target by delivering a notice of Janssen’s exercise of such Option toMeiraGTx (each, an “Option Exercise Notice”) and paying MeiraGTx [***], which fee shall be non-creditable and non-refundable (each, an “OptionFee,” and such date of receipt of notice, the “Option Election Date”). After receipt of such Option Exercise Notice, MeiraGTx shall submit an invoiceto Janssen for such Option Fee, and Janssen shall make such Option Fee payment to MeiraGTx within [***] after Janssen’s receipt of such invoice. Forclarity, the [***]. As of such Option Election Date, each such Research IRD Product for which Janssen has exercised an Option will become a JanssenResearch IRD Product, the exclusive license granted to Janssen in Section 4.1(b)(ii) for such Janssen Research IRD Product will become effective, andJanssen shall assume all responsibility, at Janssen’s sole cost and expense, for the further Development, Manufacture, and Commercialization of suchResearch IRD Product in accordance with this Agreement. In addition, with respect to any Option, and on a Research IRD Target-by-Research IRDTarget basis, Janssen may deliver a written notice to MeiraGTx of its intention not to exercise such Option. If with respect to any given Option Janssenfails to provide an Option Exercise Notice before the expiration of the applicable Option Period, or if Janssen provides written notice to MeiraGTx thatJanssen does not wish to exercise the applicable Option, then such Option will expire on a Research IRD Target-by-Research IRD Target basis (each, an“Option Opt-Out,” and each of such dates, an “Option Opt-Out Date”), Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -33-Confidential Treatment Requested by MeiraGTx Holdings plc and: (i) the exclusivity obligations applicable to MeiraGTx under Section 4.6(c) shall expire, and the exclusivity obligations applicable to Janssenunder 4.6(b) shall not take effect, in each case with respect to each Research IRD Product that treats the Research IRD Target Indication resulting fromthe loss of function of such Research IRD Target; (ii) MeiraGTx may freely Develop, Manufacture and Commercialize, or grant to any Third Partyrights to Develop, Manufacture and Commercialize, at MeiraGTx’s sole cost and expense, each such Research IRD Product without further obligationto Janssen; (iii) the non-exclusive license granted from MeiraGTx to Janssen in Section 4.1(b)(i) will terminate with respect to the Research PlanActivities applicable to each such Research IRD Product; (iv) Janssen shall have [***] except as otherwise expressly provided herein; (v) Janssen shallhave no obligation to Develop or Commercialize any such Research IRD Product; and (vi) Janssen shall have [***]. 4.LICENSES 4.1.Licenses to Janssen.(a) Clinical IRD Products. Subject to the terms and conditions of this Agreement and each Supply Agreement, MeiraGTx hereby grants, on behalfof itself and its Affiliates, to Janssen an exclusive (even as to MeiraGTx), royalty-bearing, sublicensable (through multiple tiers, solely as provided inSection 4.3), transferable (in accordance with Section 19.1) license under the MeiraGTx Technology and MeiraGTx’s interest in the Joint Technologyduring the Term, to Develop, make, have made, use, have used, sell, have sold, offer for sale, have offered for sale, import, have imported, and otherwiseexploit, Manufacture and Commercialize Clinical IRD Products in the Field in the Territory. For clarity, Janssen may not exercise its rights to make,have made (except by MeiraGTx or its Affiliates or permitted CMOs) or Manufacture Clinical IRD Products in the Field in the Territory except asexpressly permitted in a Supply Agreement.(b) Research IRD Products.(i) Non-Exclusive License to Perform Obligations. Subject to the terms and conditions of this Agreement, MeiraGTx hereby grants, onbehalf of itself and its Affiliates, to Janssen a non-exclusive, sublicensable (through multiple tiers, solely as provided in Section 4.3), transferable (inaccordance with Section 19.1) license under the MeiraGTx Research Technology and MeiraGTx’s interest in the Joint Technology during the Term, forpurposes of Janssen conducting its obligations under this Agreement, including any Research Plan Activities assigned to Janssen in the respectiveResearch Plan.(ii) Exclusive License Following Option Exercise. Subject to the terms and conditions of this Agreement and each Supply Agreement, withrespect to each Janssen Research IRD Product, MeiraGTx hereby grants, on behalf of itself and its Affiliates, to Janssen an exclusive (even as toMeiraGTx), royalty-bearing, sublicensable (through multiple tiers, solely as provided in Section 4.3), transferable (in accordance with Section 19.1)license, under the MeiraGTx Research Technology and MeiraGTx’s interest in the Joint Technology during the Term, to Research, Develop, make,have made, use, have used, sell, have sold, offer for sale, have offered for sale, import, have imported, and otherwise exploit, Manufacture andCommercialize such Janssen Research IRD Product in the Field in the Territory. For clarity, Janssen may not exercise its rights to make, have made(except by MeiraGTx or its Affiliates or permitted CMOs) or Manufacture Janssen Research IRD Products in the Field in the Territory except asexpressly permitted in a Supply Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -34-Confidential Treatment Requested by MeiraGTx Holdings plc (c) Internal Research License. Subject to the terms and conditions of this Agreement, MeiraGTx hereby grants, on behalf of itself and itsAffiliates, to Janssen a non-exclusive, royalty-free, sublicensable (but only to its Affiliates) license, under the MeiraGTx Technology, MeiraGTxResearch Technology and MeiraGTx’s interest in the Joint Technology to conduct Internal Research in the Field in the Territory. The license grantedunder this Section 4.1(c) shall be perpetual and survive any expiration of this Agreement and any termination of this Agreement by Janssen underSection 15.2(a)(i) for MeiraGTx’s uncured material breach or under Section 15.2(b) for a MeiraGTx Insolvency Event. In addition, upon an OptionOpt-Out for a Research IRD Product pursuant to Section 3.9(b), the license granted under this Section 4.1(c) shall be perpetual and survive with respectto such Research IRD Product, except as otherwise expressly provided in this Section 4.1(c). [***] 4.2.Licenses to MeiraGTx.(a) Clinical IRD Products.(i) License Back for Performance of Obligations and Manufacture. Subject to the terms and conditions of this Agreement, with respect toeach Clinical IRD Product, Janssen hereby grants, on behalf of itself and its Affiliates, to MeiraGTx a non-exclusive, royalty-free, sublicensable (solelyas provided in Section 4.3), transferable (in accordance with Section 19.1) license, under the MeiraGTx Technology and both Parties’ interest in theJoint Technology to: (A) perform MeiraGTx’s obligations under this Agreement with respect to such Clinical IRD Product; and (B) make, have madeand otherwise Manufacture such Clinical IRD Product in accordance with the terms herewith. (ii) Non-exclusive License to Janssen Technology.Subject to the terms and conditions of this Agreement, with respect to each Clinical IRD Product, Janssen hereby grants, on behalf of itself and itsAffiliates, to MeiraGTx, a non-exclusive, royalty-free, sublicensable (solely as provided in Section 4.3), transferable (in accordance with Section 19.1)license under the Janssen Technology during the Term, for purposes of MeiraGTx conducting its obligations under this Agreement, including anyClinical Development Plan Activities assigned to MeiraGTx in the respective Clinical Development Plan or the Manufacture of any Clinical IRDProducts in accordance with the terms herewith.(b) Research IRD Products.(i) Non-Exclusive License Back for Manufacture. Subject to the terms and conditions of this Agreement, with respect to each JanssenResearch IRD Product, Janssen hereby grants, on behalf of itself and its Affiliates, to MeiraGTx a non-exclusive, royalty-free, sublicensable (solely asprovided in Section 4.3), transferable (in accordance with Section 19.1) license, under the MeiraGTx Research Technology and Janssen’s interest in theJoint Technology to make, have made and otherwise Manufacture such Janssen Research IRD Product in accordance with the terms herewith. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -35-Confidential Treatment Requested by MeiraGTx Holdings plc (ii) Non-Exclusive License to Janssen Technology. Subject to the terms and conditions of this Agreement, with respect to each ResearchIRD Product (including any Janssen Research IRD Product), Janssen hereby grants, on behalf of itself and its Affiliates, to MeiraGTx, a non-exclusive,royalty-free, sublicensable (solely as provided in Section 4.3), transferable (in accordance with Section 19.1) license under the Janssen Technologyduring the Term, for purposes of MeiraGTx conducting its obligations under this Agreement, including any Research Plan Activities assigned toMeiraGTx in the respective Research Plan or the Manufacture of any Research IRD Products in accordance with the terms herewith. 4.3.Sublicense Rights.Janssen may sublicense through multiple tiers the rights granted to it by MeiraGTx under Section 4.1(a) and Section 4.1(b) at any time [***]; providedthat: (a) each such sublicense shall be consistent with the terms and conditions of this Agreement (including Section 4.1 and Article 12) and shall besubject to any Third Party License requirements with respect to any Technology in-licensed by MeiraGTx and (b) each sublicense to a Third Partycollaborator shall be in writing and shall contain terms prohibiting the Third Party collaborator from using any MeiraGTx Confidential Information,MeiraGTx Know-How or MeiraGTx Research Know-How for any purpose inconsistent with this Agreement. In the case of any sublicense by Janssen,Janssen shall remain primarily responsible for, and directly liable to MeiraGTx for, the payment of all royalties payable based on Net Sales of Productby any Janssen Sublicensee and the payment of all Development Milestone Payments and Commercial Milestone Payments even if a Sublicensee isresponsible for the achievement of any such milestone with respect to any Product. Either Party may exercise its rights and perform its rights andobligations under this Agreement itself or through any of its Affiliates. Except for MeiraGTx sublicensing to an Affiliate or as permitted underSection 4.4, MeiraGTx may not sublicense the rights granted to it by Janssen under this Agreement without first obtaining, in each case, Janssen’s priorwritten consent (which shall not be unreasonably withheld, delayed, or conditioned), and any such sublicense must be consistent with the terms of thisAgreement (including Section 4.2 and Article 12). 4.4.Subcontractors.Each Party may engage subcontractors to perform any obligations assigned to it under this Agreement, except that: (a) [***]; (b) prior to [***]; (c) priorto [***]; (d) the subcontracting Party shall remain fully responsible for the work allocated to, and payment to, such subcontractors to the same extent itwould if it had done such work itself; (e) each contract between a Party and a subcontractor shall be consistent with the provisions of this Agreement,including (i) obligations of confidentiality and non-use applicable to Confidential Information that are at least as stringent as those set forth in Article12 and (ii) obligations of assignment of all Inventions and other Intellectual Property Rights developed in the course of performing any such workunder this Agreement to the subcontracting Party and obligations of cooperation to execute any documents to confirm or perfect such assignment; and(f) the subcontracting Party shall remain at all times fully liable for all acts or omissions of such subcontractor. Any reference to a Party’s performancehereunder shall include references to any performance by its subcontractors permitted in accordance with this Section 4.4. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -36-Confidential Treatment Requested by MeiraGTx Holdings plc 4.5.Third Party Licenses.All rights licensed to a Party from a Third Party and sublicensed to the other Party under this Agreement will be subject to and subordinate to the termsof the applicable Third Party License to the extent such terms apply to a sublicensee of such Third Party Intellectual Property Rights, except that noParty shall be obligated to comply with any such Third Party License until such Third Party License has been disclosed to such Party. 4.6.Exclusivity.(a) Janssen Exclusivity for Clinical IRD Targets. On a Clinical IRD Target-by-Clinical IRD Target basis, beginning on (i) the Effective Date withrespect to the CNGA3 Target, CNGB3 Target and RPGR Target and (ii) the [***] Product Opt-In Date with respect to the [***] Target, and ending onthe earlier of (A) [***] of the Effective Date or (B) the expiration or termination of this Agreement with respect to the Clinical IRD Product associatedwith such Clinical IRD Target, [***] other than Researching, Developing, Manufacturing, and Commercializing Clinical IRD Products in accordancewith the terms and conditions of this Agreement. In addition, on a Clinical IRD Product-by-Clinical IRD Product basis, beginning on [***] and for[***] thereafter, [***](b) Janssen Exclusivity for Research IRD Targets. On a Research IRD Target-by-Research IRD Target basis, beginning on the Option ElectionDate associated with the Janssen Research IRD Product intended to treat the Research IRD Target Indication resulting from the loss of function of suchResearch IRD Target and ending on the earlier of (i) the [***] of the Option Election Date associated with the Janssen Research IRD Product intendedto treat such Research IRD Target Indication resulting from the loss of function of such Research IRD Target or (ii) the expiration or termination of thisAgreement with respect to the Janssen Research IRD Product intended to treat such Research IRD Target Indication resulting from the loss of functionof such Research IRD Target, [***] other than Researching, Developing, Manufacturing, and Commercializing Janssen Research IRD Products inaccordance with the terms and conditions of this Agreement.(c) MeiraGTx Exclusivity Obligations. Beginning on the Effective Date, [***] other than Researching, Developing, Manufacturing andCommercializing Gene Therapy Products in accordance with the terms and conditions of this Agreement. The prohibition contained in theimmediately preceding sentence shall end:[***]provided, however, that if both subsections (ii) and (iii) above apply to any one particular case, the longer period described in subsection (ii) or (iii)shall apply thereto. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -37-Confidential Treatment Requested by MeiraGTx Holdings plc (d) Mergers and Acquisitions Involving Competing Products. Subject to Section 4.6(e), in the event that a Third Party merges with a Party orbecomes an Affiliate of a Party or assets of a Third Party are combined with a Party or an Affiliate of a Party (“Acquired Party”) during the Termthrough merger, acquisition, consolidation or other similar transaction, and as of the closing date of such merger, acquisition, consolidation or othersimilar transaction, such Third Party is engaged in the Research, Development, Manufacture or Commercialization of a Competing Product, then,unless the Parties mutually agree otherwise, the Acquired Party or its new Affiliate (as applicable) shall have [***] from the closing date of suchtransaction to wind down or complete the Divestiture of such Competing Product, and its new Affiliate’s conduct of the Research, Development,Manufacture and Commercialization of such Competing Product during such [***] period shall not be deemed a breach of the Acquired Party’sexclusivity obligations set forth in this Section 4.6; provided that such new Affiliate conducts the Research, Development, Manufacture andCommercialization of such Competing Product during such [***] period independently of the activities of this Agreement and does not use any of theother Party’s Intellectual Property Rights or Confidential Information (except as may be separately licensed by such other Party to such new Affiliate)in such conduct. “Divestiture”, as used in this Section 4.6(d), means the sale or transfer of rights to the Research, Development, Manufacture andCommercialization of a Competing Product to a Third Party without receiving a continuing share of profit, royalty payment or other economic interestin the success of such Competing Product.(e) Qualified Change of Control. In the event of a Qualified Change of Control involving MeiraGTx, Janssen shall have the right to terminate[***], by providing MeiraGTx with written notice at least [***] after the announcement of such Qualified Change of Control, with such termination tobe effective within [***] of such notice; provided that all other obligations under this Agreement, including under Section 4.1 and Article 10, shallremain in full force and effect. For clarity, upon Janssen’s termination notice to MeiraGTx in accordance with the preceding sentence, (i) allCommittees shall be disbanded and Janssen shall have sole and exclusive control with respect to any activities previously conducted pursuant to thisAgreement and (ii) Janssen shall not have any further obligations to share any information, updates or reports with MeiraGTx with respect to anyactivities previously conducted pursuant to this Agreement, except with respect to Section 10.6, Section 10.7, Section 10.8, Section 10.9,Section 10.11 (including Sales & Royalty Reports) and any other applicable payment obligations. In the event that Janssen elects to not exercise itsrights under this Section 4.6(e), the terms of Section 4.6(d) shall apply. 4.7.No Other Rights.Each Party expressly reserves and retains all Patents, Know-How, and other intellectual property rights not expressly granted herein, and no right orlicense under any Intellectual Property Rights of either Party is granted or shall be granted by implication, estoppel or otherwise. Each licensee Partycovenants to the licensor Party that the licensee Party shall not (and shall require that any of its Affiliates or Sublicensees shall not) use any of thelicensor Party’s Intellectual Property Rights licensed under this Agreement for any purpose other than as expressly provided under this Agreement forsuch licensee Party. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -38-Confidential Treatment Requested by MeiraGTx Holdings plc 4.8.MeiraGTx Know-How Transfer.(a) Clinical IRD Products. Within [***] following the Effective Date, MeiraGTx will deliver to Janssen all copies of: (i) MeiraGTx Know-Howreasonably necessary or useful for the Development or Commercialization of the Clinical IRD Products; and (ii) documents and files related to theMeiraGTx Patents Covering the Clinical IRD Products. Thereafter, on a [***] basis during the Term or as otherwise mutually agreed by the Parties,MeiraGTx shall [***] disclose to Janssen (through the JSC, JDC, or JRC, as applicable) any additional MeiraGTx Know-How reasonably necessary oruseful for the Development or Commercialization of any Clinical IRD Product that came into existence after the prior disclosure, will providereasonable assistance to Janssen in connection with understanding and using all such MeiraGTx Know-How for purposes consistent with the licensesand rights granted to Janssen under Section 4.1 and will provide any new documents or files related to the MeiraGTx Patents Covering the ClinicalIRD Products that came into MeiraGTx’s possession or control after the prior disclosure. All costs of the transfer set forth in this Section 4.8(a) shall bedeemed Clinical Development Costs hereunder and paid in accordance with the terms and conditions set forth in Section 6.1(f).(b) Research IRD Products. With respect to any Janssen Research IRD Product, within [***] after Janssen’s exercise of the respective Option,MeiraGTx will deliver to Janssen all copies of: (i) MeiraGTx Research Know-How reasonably necessary or useful for the Development orCommercialization of the applicable Janssen Research IRD Product; and (ii) documents and files related to the MeiraGTx Research Patents Coveringthe Janssen Research IRD Product. Thereafter, on a [***] basis during the Term or as otherwise mutually agreed by the Parties, MeiraGTx shall [***]disclose to Janssen (through the JSC, JDC, or JRC, as applicable) any additional MeiraGTx Research Know-How reasonably necessary or useful for theDevelopment or Commercialization of any Janssen Research IRD Product that came into existence after the prior disclosure, will provide reasonableassistance to Janssen in connection with understanding and using all such MeiraGTx Research Know-How for purposes consistent with the licensesand rights granted to Janssen under Section 4.1 and will [***]. All costs of the transfer set forth in this Section 4.8(b) shall be [***]. 5.GOVERNANCE 5.1.Alliance Managers.Within [***] following the Effective Date, each Party shall designate an individual employee to facilitate communicationand coordination of the Parties’ activities under this Agreement and to provide support and guidance to the JSC (each, an “AllianceManager”). Each Alliance Manager may not serve as a representative of its respective Party on any Committee. 5.2.Joint Steering Committee.(a) Purpose; Formation. Within [***] following the Effective Date, the Parties shall establish a joint steering committee (the “JSC” or “JointSteering Committee”). The JSC shall monitor, make certain decisions, and provide strategic oversight of the activities under this Agreement andfacilitate communications between the Parties with respect to the Research, Development, Manufacturing, and Commercialization of the Products. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -39-Confidential Treatment Requested by MeiraGTx Holdings plc (b) Specific Responsibilities. In addition to providing general oversight of all activities hereunder, the JSC shall in particular have the followingresponsibilities, in each case to the extent the applicable decisions are not made at the level of another Committee: (i) reviewing and approving eachinitial Clinical Development Plan; (ii) reviewing and approving the overall regulatory strategy for the Clinical IRD Products; (iii) approving allmaterial Regulatory Filings and Regulatory Materials as necessary, subject to Section 7.1(c); (iv) reviewing and approving each initial Research Planfor a Research IRD Target; (v) reviewing and approving each initial CMC Development Plan; (vi) approving, upon recommendation and proposal bythe applicable Committee, Research Budgets, Clinical Development Budgets, Clinical Development Manufacturing Budgets and ProcessDevelopment Budgets as provided herein (provided that no Research Budget for a Calendar Year shall exceed the respective Research Budget Cap forsuch Calendar Year); (vii) facilitating the flow of information with respect to the Development and Commercialization of the Products; (viii) receivingand discussing reports from the other Committees; (ix) providing guidance to the other Committees on all significant strategic issues that fall withinthe scope of such Committees; (x) establishing such additional joint subcommittees as it deems necessary to achieve the objectives and intent of thisAgreement; (xi)facilitating the prosecution and maintenance with respect to Patents pursuant to Section 11.4 and resolving disputes with respect tosuch Patents; (xii) resolving disputes for which it is responsible as provided in this Agreement; and (xiii) performing such other functions as expresslyprovided in this Agreement. 5.3.Joint Research Committee.(a) Purpose; Formation. Within [***] following the Effective Date, the Parties shall establish a committee to oversee the Research Plan Activitiesand otherwise facilitate the flow of information between the Parties with respect to, and provide a forum to discuss, Research Plan Activities (the “JRC”or “Joint Research Committee”).(b) Specific Responsibilities. The JRC shall be responsible for: (i) nominating and approving IRD Genes to become Research IRD Targets;(ii) discussing, preparing, and recommending for submission to the JSC for approval, each initial Research Plan and discussing and approving asapplicable amendments thereto; (iii) discussing, preparing, and recommending for submission to the JSC for approval, each Research Budget;(iv) overseeing and directing the Research Plan Activities, including Natural History Studies; (v) reviewing and discussing all reports describing theResearch Plan Activities and the Research Results (including Interim Reports and Final Reports); (vi) performing such other functions as may bedelegated to it by the JSC; and (vii) performing such other functions expressly delegated to it in this Agreement. 5.4.Joint Development Committee.(a) Purpose; Formation. Within [***] following the Effective Date, the Parties shall establish a committee to oversee the Clinical DevelopmentPlan Activities of the Parties with respect to each Clinical IRD Product and otherwise facilitate the flow of information between the Parties with respectto, and provide a forum to discuss, the Development of Products (the “JDC” or “Joint Development Committee”). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -40-Confidential Treatment Requested by MeiraGTx Holdings plc (b) Specific Responsibilities. The JDC shall in particular have the following responsibilities: (i) reviewing, amending, and recommending forapproval by the JSC, each initial Clinical Development Plan and discussing and approving as applicable amendments thereto; (ii) discussing,preparing, and recommending for submission to the JSC for approval, each Clinical Development Budget; (iii) reviewing and monitoring the Parties’Clinical Development Plan Activities and progress against each Clinical Development Plan, including facilitating discussions between the Partiesregarding the Development of each Clinical IRD Product; (iv) reviewing and recommending to the JSC for approval all material Regulatory Filings andall Regulatory Materials as necessary or as requested by the JSC, subject to Section 7.1(c); (v) discussing and recommending to the JSC for approvalthe overall regulatory strategy for each Clinical IRD Product; (vi) discussing the Development reports; (vii) determining the timing of the transitionbetween the Parties with respect to regulatory matters pursuant to Section 7.1(a)(iii); (viii) reviewing and monitoring the Parties’ Developmentactivities with respect to safety matters; (ix) developing and reviewing the overall publication strategy for the Parties in connection with thisAgreement, subject to Section 13.4; (x) performing such other functions as may be delegated to it by the JSC; and (xi) performing such other functionsexpressly delegated to it in this Agreement. 5.5.Joint Manufacturing Committee.(a) Purpose; Formation. Within [***] following the Effective Date, the Parties shall establish a committee to oversee and coordinate theManufacturing activities of the Parties with respect to each Clinical IRD Product or Research IRD Product and otherwise facilitate the flow ofinformation between the Parties with respect to, and provide a forum to discuss, the Manufacturing of Products and the CMC DevelopmentCollaboration (the “JMC” or “Joint Manufacturing Committee”).(b) Specific Responsibilities. The JMC shall in particular have the following responsibilities: (i) reviewing, amending, and recommending forapproval by the JSC initial Manufacturing plans, initial CMC Development Plans and discussing and approving as applicable amendments thereto,including with respect to capacity; (ii) discussing, preparing, and recommending for submission to the JSC for approval each Clinical DevelopmentManufacturing Budget and Process Development Budget; (iii) overseeing the Manufacturing of Products used in Development activities, includingdiscussing any potential supply issues, interruptions, the outcome of any Regulatory Authority inspection of Manufacturing facilities used by or onbehalf of MeiraGTx, and any remedial actions required, if applicable, as a result of such inspection; (iv) overseeing the CMC Development PlanActivities for all Clinical IRD Products, Research IRD Products, and Janssen Research IRD Products; (v) collaborating on the creation of theManufacturing-related Regulatory Filings and all Regulatory Materials for any Clinical IRD Product and any Research IRD Product beingManufactured by or on behalf of MeiraGTx; (vi) overseeing the Know-How transfer of Manufacturing technology to Janssen in accordance with anySupply Agreement; (vii) reviewing and approving specifications for Clinical IRD Products and Research IRD Products; (viii) performing such otherfunctions as may be delegated to it by the JSC; and (ix) performing such other functions expressly delegated to it in this Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -41-Confidential Treatment Requested by MeiraGTx Holdings plc 5.6.Operational Teams.From time-to-time, each of the JSC, JRC, JDC or JMC may establish and delegate specific matters or duties within its responsibilities to directed teams(each, an “Operational Team”), the composition, operation, and responsibilities of which will be determined by the applicable establishingCommittee. Operational Teams may be established on an ad hoc basis for purposes of a specific activity or on such other basis as the applicableestablishing Committee may determine. Each Operational Team will report to, and its activities will be subject to the oversight of, the applicableestablishing Committee and the JSC, and no Operational Team’s authority may exceed that specified for the applicable establishing Committee. Anydisagreement between the representatives of the Parties on any Operational Teams will be referred to the applicable establishing Committee forresolution in accordance with Section 5.8. 5.7.Committee Representatives and Meetings.(a) Committee Representatives. Each Party shall initially appoint [***], and no more than [***] representatives to each Committee, with theexception of the JSC, which shall have [***] representatives from each Party. Each Committee representative shall have appropriate knowledge andexpertise within the applicable Party to make decisions or recommendations arising within the scope of the applicable Committee’s responsibilities.Each Party may replace its representatives on any Committee upon written notice to the other Party. Each Party shall appoint [***] of itsrepresentatives on each Committee to act as a co-chairperson of such Committee. The responsibility for running each meeting of each Committee shallalternate between the co-chairpersons of such Committee from meeting-to-meeting, with Janssen’s co-chairperson running the first meeting of eachCommittee. The co-chairpersons of each Committee shall jointly prepare and circulate agendas to such Committee’s representatives before each suchCommittee meeting and shall direct the preparation of reasonably detailed documentation for each such Committee meeting, which shall be approvedby the Committee’s co-chairpersons and circulated to Committee representatives within [***] of such meeting. Unless mutually agreed upon by theParties, any member of one Committee shall not be a member of another Committee under this Agreement, and Alliance Managers and Senior Officersmay not serve on any Committee.(b) Non-Committee Representatives. Each Party may [***] invite [***], in addition to its representatives, to attend Committee meetings in anon-voting capacity; provided that if either Party intends to have any Third Party attend such a meeting, such Party shall obtain the other Party’s priorwritten consent for such Third Party to attend such meeting, which consent shall not be unreasonably withheld, conditioned, or delayed; and providedfurther that each Party’s co-chairperson of the JMC may attend any JDC meeting in a non-voting capacity and each Party’s co-chairperson of the JDCmay attend any JMC meeting in a non-voting capacity, in each case without the consent of the other Party. Each Party shall be responsible for ensuringthat each Committee member that it appoints and each Third Party that it invites to a Committee meeting complies with the confidentiality andnon-use obligations set forth in this Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -42-Confidential Treatment Requested by MeiraGTx Holdings plc (c) Meetings. Each Committee shall hold meetings at such times as it elects to do so, but at least [***] in the case of the JRC, the JDC and theJMC, and at least [***] in the case of the JSC; provided that the JSC shall hold its first meeting no later than [***] from the Effective Date. Meetings ofany Committee may be held in person or by audio or video teleconference; provided that unless otherwise agreed by the Parties, at least [***] meetingper [***] for each Committee shall be held in person. Notwithstanding Section 5.7(a), upon the Parties’ mutual agreement, any meeting of a Committeemay be combined with any meeting of another Committee. Each Party shall be responsible for all of its own costs and expenses of participating in anyCommittee meetings. No action taken at any meeting of a Committee shall be effective unless at least [***] Committee representative of each Party isparticipating in such meeting.(d) Dissolution. Each Committee will continue to exist until the earlier of completion of such Committee’s obligations under this Agreement ormutual agreement of the Parties to disband such Committee; provided that following the dissolution of the JSC, the JSC may, upon the Parties’agreement, continue to meet on a [***] basis (or more or less frequently, if mutually agreed by the Parties) solely to serve as a forum for sharing anddiscussing information. 5.8.Resolution of Committee Disputes.(a) Votes. All decisions of each Committee shall be made by [***] vote, with each Party’s representatives on the respective Committeecollectively having [***] vote.(b) Disputes. If, after reasonable discussion and good-faith consideration of each Party’s view on a particular matter before any Committee otherthan the JSC and within the scope of its authority, the representatives of the Parties on such Committee cannot reach an agreement as to such matterwithin [***] after such matter was brought to such Committee for resolution, such disagreement shall be referred to the JSC for resolution. If, afterreasonable discussion and good-faith consideration of each Party’s view on a particular matter before the JSC and within the scope of its authority, therepresentatives of the Parties on the JSC cannot reach an agreement as to such matter within [***] after such matter was brought to the JSC forresolution or after such matter has been referred to the JSC from another Committee, such disagreement shall be referred to the Senior Officers forresolution.(c) Final Decision-Making Authority. If the Senior Officers cannot in good faith resolve a particular matter within [***] after such matter has beenreferred to them, then: [***](d) No Other Powers. Notwithstanding anything herein to the contrary, each Committee shall have only the powers assigned expressly to it in thisArticle 5 and elsewhere in this Agreement, and no Committee shall have any power to amend, modify or waive compliance with this Agreement, or toimpose additional obligations on a Party beyond those provided in this Agreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -43-Confidential Treatment Requested by MeiraGTx Holdings plc 6.DEVELOPMENT6.1. Clinical IRD Products.(a) Clinical IRD Products. Subject to the terms and conditions of this Agreement (including Section 2.1(b)), the Parties will collaborate with oneanother to Develop the Clinical IRD Products.(b) Responsibility. Subject to the oversight of the JSC and JDC, the Parties will jointly collaborate with one another on the Development of eachClinical IRD Product in accordance with this Agreement and the applicable Clinical Development Plan for such Clinical IRD Product, includingconducting any necessary Research to support BLA approval for such Clinical IRD Product.(c) Clinical Development Plans. The anticipated costs and timelines for activities under the Clinical Development Plans for the CNGA3 Product,the CNGB3 Product and the RPGR Product are attached hereto as parts of Exhibit 1.34. [***] with respect to the CNGA3 Product, CNGB3 Product andRPGR Product, the JDC shall develop and submit to the JSC for its review and approval, a Clinical Development Plan for each such Clinical IRDProduct, and upon the JSC’s approval, each such initial Clinical Development Plan will be attached hereto and form a part of Exhibit 1.34. If Janssenelects to make the [***] Product a Clinical IRD Product in accordance with Section 2.1(b), no later than [***] following the [***] Product Opt-In Date,the JDC shall develop and submit to the JSC for its review and approval, a Clinical Development Plan for the [***] Product, and upon the JSC’sapproval, such initial Clinical Development Plan shall be attached hereto and form a part of Exhibit 1.34. In the event that any Clinical DevelopmentPlan for a Clinical IRD Product conflicts with the anticipated costs and timelines set forth in Exhibit 1.34 for such Clinical IRD Product, the ClinicalDevelopment Plan shall control. Each Clinical Development Plan will set forth in reasonable detail at all times (i) all activities that are necessary oruseful to be undertaken to achieve and maintain Regulatory Approval in the relevant Major Market Country for such Clinical IRD Product (the“Clinical Development Plan Activities”), (ii) an estimated timeline for completing such activities and the respective Clinical Development Plan Term,(iii) the respective deliverables for such activities and (iv) the allocation of responsibilities between the Parties for performance of each such activity.The terms of, and Clinical Development Plan Activities set forth in, each Clinical Development Plan will at all times be designed to expediteRegulatory Approval, reimbursement and Commercialization of the Clinical IRD Product that is the subject of such Clinical Development Plan, and tobe in compliance with all Applicable Laws and in accordance with professional and ethical standards customary in the biopharmaceutical industry.From time to time, and at least on an annual basis, the JDC will develop, review and approve amendments or updates to each Clinical DevelopmentPlan for a Clinical IRD Product, subject to the remaining terms and conditions of this Agreement. Each such amended or updated ClinicalDevelopment Plan will automatically replace the respective Clinical Development Plan previously in effect, and will be automatically incorporatedinto this Agreement by reference and form a part of this Agreement. Each Clinical Development Plan shall also be consistent with the terms of thisAgreement. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -44-Confidential Treatment Requested by MeiraGTx Holdings plc (d) Clinical Development Budgets. Each Clinical Development Plan, when approved by the Parties in accordance with Section 6.1(c), will besubject to a rolling budget covering Clinical Development Costs associated with the anticipated Clinical Development Plan Activities for suchClinical IRD Product to be performed during the [***] (such [***] period, the “[***] Clinical Development Budget Period”), in each case brokendown by Calendar Quarter and broken out on a line item basis to show Out-of-Pocket Costs and FTE Costs of FTEs directly engaged to perform eachsuch Development activity including for each Development activity with respect to each Clinical Study conducted thereunder (each, as agreed to,approved, amended or updated from time to time in accordance with this Section 6.1(d), a “Clinical Development Budget”). [***] with respect to theCNGA3 Product, CNGB3 Product and RPGR Product, and [***] in the event that Janssen elects to make the [***] Product a Clinical IRD Product inaccordance with Section 2.1(b), the JDC shall develop and submit to the JSC for its review and approval, a Clinical Development Budget for therespective Clinical IRD Product, and upon the JSC’s approval, each such Clinical Development Budget will be automatically incorporated into thisAgreement by reference and form a part of this Agreement. Each Clinical Development Budget will be reviewed by the JDC and approved by the JSC(i) at least [***] (and [***] of the applicable [***] Clinical Development Budget Period) based on: (A) the Parties’ good faith estimation of theanticipated Clinical Development Plan Activities to be conducted during the relevant [***] Clinical Development Budget Period; and (B) informationprepared by the Parties in good faith for their own internal planning processes relating to anticipated Clinical Development Plan Activities for suchClinical IRD Product; or (ii) whenever the total Clinical Development Costs for any given Calendar Quarter are reasonably expected to be at least [***]percent ([***]%) higher than the Clinical Development Budget for such Calendar Quarter, whether as a result of any amendments to the ClinicalDevelopment Plan or increases in costs for the Clinical Development Plan Activities already planned for such Calendar Quarter (provided, however,that following the beginning of a Calendar Year, the JSC may not increase the Clinical Development Budget for such Calendar Year by more than[***] percent ([***]%) without the Parties’ prior written approval). Each such amended or updated Clinical Development Budget will automaticallyreplace the respective Clinical Development Budget previously in effect, and will be automatically incorporated into this Agreement by reference andform a part of this Agreement. With respect to any Calendar Quarter, in no event will a Party be responsible for any Clinical Development Costs thatexceed the respective portion of the then-current Clinical Development Budget recommended and proposed by the JDC and approved by the JSC,except as otherwise provided in Section 10.3(b).(e) Conduct of Clinical Development Activities. On a Clinical IRD Product-by-Clinical IRD Product basis, Janssen and MeiraGTx will each useCommercially Reasonable Efforts to perform their respective Clinical Development Plan Activities in accordance with each Clinical Development Plan(including the timelines set forth therein). In performing its respective Clinical Development Plan Activities, each Party: (i) will conduct such activitiesin good scientific manner and in compliance with all Applicable Laws in all material respects, including, where applicable, those relating to cGMP,cGLP, cGCP, good pharmacovigilance practices and requirements for protection of human subjects; (ii) will not employ or use any Debarred Person;and (iii) in the case of MeiraGTx, will not use any MeiraGTx [***] Technology during the course of performing any Clinical Development PlanActivities or incorporate any MeiraGTx [***] Technology into any Clinical IRD Product unless otherwise mutually agreed to by the Parties. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -45-Confidential Treatment Requested by MeiraGTx Holdings plc (f) Clinical Development Costs. Janssen will be responsible for one hundred percent (100%) of all Clinical Development Costs for each ClinicalIRD Product; provided, however, that in no event will any Clinical Development Costs for any Calendar Quarter exceed the applicable ClinicalDevelopment Budget for such Calendar Quarter, except as otherwise provided in Section 10.3(b). Notwithstanding anything herein to the contrary, inno event [***].(g) Clinical Development Records. Each Party will prepare and maintain, and cause its Affiliates and their respective employees and permittedsubcontractors to prepare and maintain, records, accounts, notes, reports and data with respect to the Clinical Development Plan Activities hereunder(“Clinical Development Records”), in sufficient detail and in a good scientific manner appropriate for scientific, regulatory, and intellectual propertyprotection purposes and in compliance with Applicable Law and such Party’s standard practices (including cGLP with respect to activities that requirecGLP compliance to be submitted in Regulatory Filings including INDs and BLAs), which Clinical Development Records will: (i) be segregated fromother development activities not performed under this Agreement; and (ii) be complete and accurate, and fully and accurately reflect all work done,data and developments made, and results achieved in the performance of the Clinical Development Plan Activities. Each Party shall retain, and causeits Affiliates and their respective and permitted subcontractors to retain, Clinical Development Records for at least [***] or such longer period as maybe required by Applicable Law. Each Party shall comply with Janssen’s data policies set forth on Exhibit 3.5 attached hereto with regard to ClinicalDevelopment Records.(h) Audits. With respect to any facility or site at which a Party conducts any Clinical Development Plan Activities, and subject to the terms of anyagreement between such Party and any applicable permitted subcontractor with respect to any facility or site of such subcontractor, the other Partyshall have the right, at its own expense, upon reasonable written notice by such other Party, and during normal business hours, to inspect such site andfacility of such Party or to accompany such Party to inspect any subcontractor site once per year and also for cause, to verify such Party’s compliancewith Applicable Law in carrying out its obligations under this Agreement, including those relating to cGMP, cGLP, cGCP, good pharmacovigilancepractices and requirements for protection of human subjects. In the event that any such facility or site is found to be non-compliant with cGMP, cGLP,cGCP, good pharmacovigilance practices and requirements for protection of human subjects during such an audit, and such non-compliance relates toor impacts any Clinical Development Plan Activities hereunder, the audited Party shall submit to the auditing Party proposed CAPA within [***] afterthe auditing Party provides notice of such non-compliance. The auditing Party shall have the right to review and comment on such CAPA, whichcomments the audited Party shall consider in good faith. The audited Party shall use Commercially Reasonable Efforts to implement such CAPA [***]after review and comment by the auditing Party. Except as may otherwise be provided in a Supply Agreement, if any Regulatory Authority or any otherGovernmental Authority conducts or gives notice of its intent to conduct any audit or inspection at any offices or facilities (including Developmentfacilities) of MeiraGTx or any applicable permitted subcontractor where such audit or inspection relates to any Clinical IRD Product, then MeiraGTxwill [***] notify Janssen and, to the extent such audit or inspection relates to a Clinical IRD Product and to the extent practicable and not prohibitedby Applicable Law, secure for Janssen the right to participate in any such audit or inspection. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -46-Confidential Treatment Requested by MeiraGTx Holdings plc (i) Reports. Each Party will: (i) provide to the JDC, on a [***], or [***] by the JDC, an update regarding any Clinical Development PlanActivities conducted by or on behalf of such Party hereunder; and (ii) promptly (and where safety is concerned, immediately within [***]) share withthe other Party all material developments and information that it comes to possess relating to the Development of each Clinical IRD Product,including: (A) safety concerns (including Adverse Events and any planned or actual inspection of any MeiraGTx facility by a Regulatory Authority oraudit of any subcontractor providing material services to MeiraGTx by MeiraGTx or any Regulatory Authority); (B) study reports and data generatedfrom Clinical Studies and (C) any significant Clinical Development Costs incurred by MeiraGTx. Each Party shall also make its employees andconsultants available for an in-person or telephonic meeting with the other Party at least once every Calendar Quarter to discuss such Party’s progresswith respect to the conduct of the Clinical Development Plan Activities hereunder.(j) Improved Clinical IRD Products. The Parties will only Develop an Improved Clinical IRD Product in accordance with the terms herewith withthe written consent of both Parties; provided, however, that [***], then [***]. 6.2.Janssen Research IRD Products.(a) Responsibility and Costs. Janssen will be solely responsible for conducting, at its sole cost and expense, Development of each JanssenResearch IRD Product, except that Janssen will use Commercially Reasonable Efforts to Develop [***].(b) Reports. Janssen will provide to the JDC, on a [***] basis for its review and discussion, a high-level report summarizing: (i) any materialDevelopment and regulatory activities for each Janssen Research IRD Product under Development by or on behalf of Janssen over the prior CalendarQuarter; and (ii) any planned future Development and regulatory activities for each Janssen Research IRD Product (if any), including those activities itanticipates to initiate or has initiated for the following Calendar Year.(c) Additional Support for Janssen Research IRD Products. On a Janssen Research IRD Product-by-Janssen Research IRD Product basis, Janssenmay request that MeiraGTx assist in subsequent Development of such Janssen Research IRD Product. At MeiraGTx’s sole discretion, it may elect toprovide such assistance, in which case it will so notify Janssen in writing and thereafter MeiraGTx’s costs of Developing such Janssen Research IRDProduct shall be [***].(d) Improved Janssen Research IRD Products. The Parties will only Research or Develop an Improved Janssen Research IRD Product inaccordance with the terms herewith with the written consent of both Parties, provided, however, that [***], then [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -47-Confidential Treatment Requested by MeiraGTx Holdings plc 7.REGULATORY 7.1.Clinical IRD Products.(a) Responsibility for Regulatory Matters.(i) Regulatory Lead Party. Subject to the review and approval of the JDC and JSC, on a Clinical IRD Product-by-Clinical IRD Product andcountry-by-country basis, (A) MeiraGTx will be the Regulatory Lead Party prior to [***]; and (B) Janssen will be the Regulatory Lead Party on andafter [***]. On and after [***] for a Clinical IRD Product, Janssen will have responsibility for all regulatory matters relating to such Clinical IRDProduct, including with respect to Regulatory Filings and meetings with Regulatory Authorities; provided that MeiraGTx will reasonably cooperatewith Janssen to provide any reasonable additional assistance or materials reasonably requested by Janssen.(ii) General. Subject to the review and approval of the JDC and this Section 7.1, on a Clinical IRD Product-by-Clinical IRD Product andcountry-by-country basis, until [***], MeiraGTx as the Regulatory Lead Party for such Clinical IRD Product shall be responsible for (A) overseeing,monitoring and coordinating all regulatory actions, communications, and filings with, and submissions to, each Regulatory Authority with respectthereto, (B) interfacing, corresponding and meeting with each Regulatory Authority with respect thereto and (C) seeking and maintaining allRegulatory Filings with respect to such Clinical IRD Product.(iii) Transition. With respect to each Clinical IRD Product, within a reasonable period following the Effective Date as determined by theJDC, MeiraGTx shall take all actions required by the FDA (and, where relevant, other Regulatory Authorities) to designate Janssen as the express andauthorized regulatory agent of record for MeiraGTx in the United States (and, as applicable, other countries) for the purposes of Development andregulatory activities for such Clinical IRD Product in the United States (and, as applicable, such other countries). On a Clinical IRDProduct-by-Clinical IRD Product basis and country-by-country basis, upon a date prior to the applicable Sponsorship Transfer Date as reasonablydetermined by the JDC, MeiraGTx will [***] assign and transfer to Janssen or its designee all existing Regulatory Filings and other RegulatoryMaterials with respect to such Clinical IRD Product, including all Drug Master Files and all written correspondence, minutes of meetings andmemoranda of oral communications with any Regulatory Authority, on electronic media (including rendered source documents when available), to theextent not already provided to Janssen previously; provided, however, that Regulatory Filings and other Regulatory Materials with respect to suchClinical IRD Product generated on or after the Effective Date shall be provided in accordance with Janssen’s instructions (including Janssen’sformatting and Electronic Common Technical Document (eCTD) requirements). Each Party will submit to the applicable Regulatory Authority allfilings, letters and other documentation necessary to effect such assignment and transfer as soon as practicable, in an efficient and seamless manner forsuch Clinical IRD Product. Each Party shall provide to the other Party a copy of any and all notices received by such Party from such RegulatoryAuthority confirming such assignment and transfer. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -48-Confidential Treatment Requested by MeiraGTx Holdings plc (iv) Right of Reference. For each Clinical IRD Product, each Party hereby grants and will cause its Affiliates, licensees, and Sublicensees togrant to the other Party, a Right of Reference to, and a right to access, copy and use, all information and data (including all CMC information) includedin or used in support of any Drug Master File maintained by or on behalf of such Party that relates to such Clinical IRD Product to the extent necessaryfor the other Party to Develop or Manufacture such Clinical IRD Product in accordance with the applicable Clinical Development Plan or CMCDevelopment Plan. From and after the Sponsorship Transfer Date, MeiraGTx hereby grants and will cause its Affiliates, licensees, and Sublicensees togrant to Janssen, a Right of Reference to, and a right to copy, access, and otherwise use, all information and data (including all CMCinformation) included in or used in support of any Drug Master File maintained by or on behalf of MeiraGTx that relates to such Clinical IRD Productto the extent not transferred to Janssen pursuant to Section 7.1(a)(iii). Notwithstanding anything to the contrary in this Agreement, MeiraGTx will not,and will cause its Affiliates, licensees, and Sublicensees not to, withdraw or inactivate any Regulatory Filing that Janssen, its Affiliates or Sublicenseesreference or otherwise use pursuant to this Section 7.1(a)(iv). In addition, Janssen hereby grants and will cause its Affiliates, licensees, and Sublicenseesto grant to MeiraGTx a Right of Reference to, and a right to access, copy and use, all information and data (including CMC information) included in orused in support of any Drug Master File maintained by or on behalf of Janssen that relate to the RPGR Product to the extent necessary to Research,Develop, Manufacture or Commercialize the [***] Product, solely in the event that Janssen elects to not make the [***] Product a Clinical IRD Productin accordance with Section 2.1(b). [***](b) Regulatory Meetings. Until the Sponsorship Transfer Date for any Clinical IRD Product, MeiraGTx will: (i) provide Janssen with reasonableadvance notice of all substantive meetings, conferences, and discussions (whether in person or by telephonic or video conference) with any RegulatoryAuthorities pertaining to such Clinical IRD Product; (ii) provide Janssen with draft briefing materials and meeting presentations for review reasonablyin advance and consider in the preparation of such meetings, conferences or discussions any reasonable input timely provided by Janssen; and (iii) tothe extent not prohibited by Applicable Law, grant Janssen the right to participate in any such meetings, conferences or discussions and facilitate suchparticipation (such number of representatives to attend to be determined by Janssen). If Janssen elects not to participate in such meetings, conferencesor discussions, MeiraGTx shall provide Janssen, upon Janssen’s request, with written summaries of such meetings, conferences or discussions inEnglish after the conclusion thereof.(c) Regulatory Filings. Until the Sponsorship Transfer Date of any Clinical IRD Product, MeiraGTx will: (i) provide to Janssen for review andcomment in accordance with such timeframes specified by the JDC (such timeframe to be no less than [***] in advance of any regulatory deadline, or ifany Regulatory Authority deadline is sooner, as reasonably in advance as possible), copies in English of all Regulatory Filings and RegulatoryMaterials to be submitted (other than routine correspondence and administrative documents and documents related to Pricing Approval) by or onbehalf of MeiraGTx prior to the relevant submission with respect to such Clinical IRD Product; (ii) incorporate reasonable comments thereto providedby Janssen; and (iii) [***] notify and provide Janssen any Regulatory Materials (other than routine correspondence and administrative documents anddocuments related to Pricing Approval) received from any Regulatory Authority with respect to such Clinical IRD Product. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -49-Confidential Treatment Requested by MeiraGTx Holdings plc (d) Regulatory Vouchers. If a Priority Review Voucher is issued to MeiraGTx for any Clinical IRD Product, then MeiraGTx will transfer suchPriority Review Voucher to Janssen. If Janssen elects to transfer to a Third Party or otherwise monetize such Priority Review Voucher, the proceedsthereof will be allocated [***], with [***] percent ([***]%) to Janssen and [***] percent ([***]%) to MeiraGTx. If Janssen uses such Priority ReviewVoucher for one of its programs or for the programs of one of its Affiliates, then: (i) the value of such Priority Review Voucher will be determined by:(A) taking the values of the [***] most recent publicly disclosed sales or transfers of a Priority Review Voucher as of the time that the Party was grantedsuch Priority Review Voucher; (B) removing the highest and lowest of such [***] publicly disclosed values; and (C) averaging the values of theremaining [***] publicly disclosed values; and (ii) the determined value of such Priority Review Voucher will be allocated [***], with [***] percent([***]%) to Janssen and [***] percent ([***]%) to MeiraGTx.(e) [***] Product. In the event that Janssen notifies MeiraGTx that Janssen does not elect to make the [***] Product a Clinical IRD Product orthat Janssen fails to notify MeiraGTx that Janssen will make the [***] Product a Clinical IRD Product, in each case as set forth in Section 2.1(b), thenMeiraGTx shall [***] notify Janssen of [***] that directly relates to or impacts any Clinical IRD Product or Janssen Research IRD Product. In addition,if Janssen becomes aware of [***], MeiraGTx shall [***] provide any [***]. 7.2.Research IRD Products.(a) Responsibility and Costs for Regulatory Matters. For each Janssen Research IRD Product, Janssen will be solely responsible, [***] fordetermining the regulatory plans and strategies and other regulatory matters relating to such Janssen Research IRD Product, including: (i) overseeing,monitoring and coordinating all regulatory actions, communications and filings with, and submissions to, each Regulatory Authority with respect tosuch Janssen Research IRD Product; and (ii) interfacing, corresponding, and meeting with each Regulatory Authority. Janssen shall invite MeiraGTx toattend, in an observational role or, to the extent permitted by Applicable Law, in a participatory role, material meetings and telephone conferences withregulators regarding such Janssen Research IRD Product. MeiraGTx will cooperate with and provide reasonable assistance to Janssen and its designeesupon Janssen’s request in connection with filings to any Regulatory Authority relating to such Janssen Research IRD Product, including by executingany required documents, providing access to personnel and providing Janssen with copies of all reasonably required documentation (such documentsto be generated and provided to Janssen in accordance with Janssen’s formatting requirements and Electronic Common Technical Document (eCTD)requirements, to the extent that such documents are generated on or after the Effective Date).(b) Ownership of Regulatory Filings. For each Janssen Research IRD Product, Janssen or its designee will own all Regulatory Filings and relatedRegulatory Materials with respect to each such Janssen Research IRD Product, including any Drug Master Files maintained by or on behalf ofMeiraGTx primarily related to and reasonably necessary for the Development of such Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -50-Confidential Treatment Requested by MeiraGTx Holdings plc Janssen Research IRD Product. At Janssen’s request, MeiraGTx will [***] assign and transfer to Janssen, all Regulatory Filings and related RegulatoryMaterials with respect to such Janssen Research IRD Product that is in the possession or control of MeiraGTx and exclusively related to such JanssenResearch IRD Product (such Regulatory Materials to be generated and provided to Janssen in rendered source document format when available and inaccordance with Janssen’s formatting requirements and Electronic Common Technical Document (eCTD) requirements, to the extent that suchdocuments are generated on or after the Effective Date), and each Party will submit all filings, letters and other documentation necessary to effect suchassignment and transfer to the applicable Regulatory Authority as soon as reasonably practicable, but no later than [***] after such request for suchJanssen Research IRD Product. Each Party shall provide to the other Party a copy of any and all notices received by such Party from such RegulatoryAuthority confirming such assignment and transfer.(c) Right of Reference. MeiraGTx hereby grants, and will cause its Affiliates, licensees, and Sublicensees to grant, to Janssen, at the request ofJanssen or its Affiliates or Sublicensees, a Right of Reference to, and a right to copy, access, and otherwise use, all information and data (including allCMC information) included in or used in support of any Drug Master File maintained by or on behalf of MeiraGTx that relates to a Janssen ResearchIRD Product to the extent necessary to Research, Develop, Manufacture or Commercialize such Janssen Research IRD Product, in each case, nottransferred to Janssen pursuant to Section 7.2(b). Notwithstanding anything to the contrary in this Agreement, MeiraGTx will not, and will cause itsAffiliates, licensees, and Sublicensees not to, withdraw or inactivate any Regulatory Filing that Janssen, its Affiliates or Sublicensees reference orotherwise use pursuant to this Section 7.2(c). [***](d) Regulatory Vouchers. Janssen shall have the sole right to apply for any Priority Review Voucher for any Janssen Research IRD Product. [***]. 7.3.Pharmacovigilance.On a Product-by-Product basis, the Parties shall negotiate in good faith and mutually agree upon a written pharmacovigilance agreement for eachProduct no later than the earliest of (a) close of the Phase 3 Study for such Product, (b) the Sponsorship Transfer Date for such Product, or(c) engagement in Commercialization for such Product. Each such written pharmacovigilance agreement shall contain provisions to ensure that thesafety data exchange and other pharmacovigilance responsibilities of both Parties with respect to regulatory reporting requirements are fulfilled inaccordance with Applicable Laws with respect to such Product. Janssen will take the lead for safety and hold the global safety database on behalf ofboth Parties for all Clinical IRD Products no later than [***], Janssen will take the lead for safety and hold the global safety database on behalf of bothParties for all Janssen Research IRD Products as of such date determined by the JDC, and the respective pharmacovigilance agreement shall containsafety lead and global safety database provisions consistent with the foregoing. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -51-Confidential Treatment Requested by MeiraGTx Holdings plc 7.4.Clinical Study Quality Agreement.The Parties shall use good faith efforts to negotiate and execute [***] following the Effective Date an agreement to govern the conduct and quality ofClinical Studies, consistent with good pharmacovigilance practices and Janssen Quality Requirements. 8.MANUFACTURING 8.1.Product Manufacturing outside Supply Agreements.(a) For Research. Subject to the terms and conditions of this Agreement and the oversight of the JSC and JMC, MeiraGTx will Manufacture (i)Clinical IRD Products required for Research under Section 6.1(b) in accordance with the applicable CMC Development Plans and (ii) Research IRDProducts for Research in accordance with the applicable CMC Development Plans, in each case ((i) and (ii)) in accordance with Janssen QualityRequirements.(b) For Development. Subject to the terms and conditions of this Agreement and the oversight of the JSC and JMC, MeiraGTx will ManufactureClinical IRD Products for use in respective Clinical Studies in accordance with Janssen Quality Requirements. (c) MeiraGTx Facility. All Products tobe Manufactured by MeiraGTx as set forth in this Section 8.1 will be Manufactured at the MeiraGTx Facility. If MeiraGTx wishes to use a CMO toManufacture any Product, MeiraGTx shall first offer such Manufacturing opportunity to Janssen, and Janssen shall have [***] to respond regardingwhether it wishes to be engaged as a CMO of such Product for MeiraGTx. If Janssen responds to MeiraGTx within such [***] period that Janssenwishes to be engaged as a CMO, then the Parties will [***] negotiate in good faith and agree upon the terms of such Manufacturing. If Janssen respondsto MeiraGTx within such [***] period that Janssen does not wish to be engaged as a CMO, or if Janssen fails to respond within such [***] period, thenMeiraGTx may use a Third Party as its CMO, provided that MeiraGTx obtains Janssen’s prior written consent thereto (not to be unreasonably withheld,conditioned or delayed) prior to engaging such CMO for such services.(d) Price for Supply of Products. The costs for Manufacturing Products in accordance with Section 8.1(a) shall be set forth in the ClinicalDevelopment Manufacturing Budget. The Manufacturing of Clinical IRD Products in accordance with Section 8.1(b) shall be [***]. Janssen shall[***]. 8.2.Clinical Supply Agreement.At such time as directed by the JSC and subject to the oversight of the JMC, the Parties will [***] negotiate in good faith a definitive clinical supplyagreement for MeiraGTx to Manufacture and supply Janssen Research IRD Products for use in respective Clinical Studies (“Clinical SupplyAgreement”), along with the associated quality agreement for such Janssen Research IRD Products (“Clinical Quality Assurance Agreement”). TheClinical Supply Agreement and the Clinical Quality Assurance Agreement will contain such terms provided in Exhibit 8.2 attached hereto. TheClinical Supply Agreement and Clinical Quality Assurance Agreement will each also Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -52-Confidential Treatment Requested by MeiraGTx Holdings plc be consistent with the terms and conditions of this Agreement and contain other terms and conditions customary for agreements of such nature. TheClinical Supply Agreement and the Clinical Quality Assurance Agreement will each be executed prior to the initiation of any Clinical Studyapplicable to the Products supplied thereunder. 8.3.Commercial Supply Agreement.At such time as directed by the JSC and subject to the oversight of the JMC, the Parties will [***] negotiate in good faith a definitive commercialsupply agreement for MeiraGTx to Manufacture and supply Clinical IRD Products for Commercial Supply (“Commercial Supply Agreement”), alongwith the associated quality agreement for such Clinical IRD Products (“Commercial Quality Assurance Agreement”). The Commercial SupplyAgreement and Commercial Quality Assurance Agreement will contain such terms provided in Exhibit 8.3 attached hereto. The Commercial SupplyAgreement and Commercial Quality Assurance Agreement will each also be consistent with the terms and conditions of this Agreement and containother terms and conditions customary for agreements of such nature. The Commercial Supply Agreement and Commercial Quality AssuranceAgreement will each be executed by the Parties at least [***] prior to the reasonably anticipated date of the First Commercial Sale of any Clinical IRDProduct. 8.4.Collaboration on CMC Development.(a) CMC Development Plan. Subject to the oversight of the JMC and JSC, during the CMC Development Term, Janssen and MeiraGTx will eachcollaborate with one another to Develop the Manufacturing process used for each clinical program undertaken hereunder in connection with anyClinical IRD Product or Research IRD Product, to identify certain technical Manufacturing process improvements and efficiencies and to share datapackages necessary for regulatory filings in connection therewith (“CMC Development Collaboration”). The Parties will undertake the CMCDevelopment Collaboration in accordance with this Agreement and one or more CMC Development Plans, which plan will set forth (i) all activitiesthat are necessary or useful to be undertaken by each Party in connection with such CMC Development Collaboration (the “CMC Development PlanActivities”), (ii) an estimated timeline for completing such activities, (iii) the respective deliverables for such activities and (iv) the allocation ofresponsibilities between the Parties for performance of each such activity. In addition, the CMC Development Plan Activities will comprise of:(x) those specific activities listed under “Clinical Development Manufacturing Activities” in Exhibit 8.4(a) attached hereto (“Clinical DevelopmentManufacturing Activities”) and (y) those specific activities listed under “Process Development Activities” in Exhibit 8.4(a) attached hereto (“ProcessDevelopment Activities”). In the event that any CMC Development Plan Activity does not fall under one of the specific activities listed in Exhibit8.4(a), the JMC shall discuss and determine whether such CMC Development Plan Activity should be (1) treated as a Clinical DevelopmentManufacturing Activity hereunder and subject to the cost provisions set forth in Section 8.4(d) or (2) treated as a Process Development Activityhereunder and subject to the cost provisions set forth in Section 8.4(e). The JMC shall develop and submit an initial CMC Development Plan to theJSC for its review and approval, [***], and upon the JSC’s approval, such initial CMC Development Plan will be automatically attached hereto andform a part of Exhibit 1.51. From time to time, and at least on an annual basis, Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -53-Confidential Treatment Requested by MeiraGTx Holdings plc the JMC shall propose, review and approve amendments or updates to each CMC Development Plan, subject to the remaining terms and conditions ofthis Agreement. Each such amended or updated CMC Development Plan will automatically replace the respective CMC Development Plan previouslyin effect, and will be automatically incorporated into this Agreement by reference and form a part of this Agreement. Each CMC Development Planshall also be consistent with the terms of this Agreement. The Parties will use Commercially Reasonable Efforts to carry out their respective obligationsunder each CMC Development Plan.(b) Clinical Development Manufacturing Budget. The CMC Development Collaboration will be subject to a rolling budget covering ClinicalDevelopment Manufacturing Costs associated with the anticipated Clinical Development Manufacturing Activities to be performed during the [***](such [***] period, the “[***] Clinical Development Manufacturing Budget Period”), in each case broken down by Calendar Quarter and broken outon a line item basis to show Out-of-Pocket Costs and FTE Costs of FTEs directly engaged to perform each such Clinical Development ManufacturingActivity and further broken down by Party (the “Clinical Development Manufacturing Budget”). [***] after the Effective Date, the ClinicalDevelopment Manufacturing Budget will be developed by the JMC, and recommended and proposed to the JSC for review and approval, and uponapproval by the JSC, each such Clinical Development Manufacturing Budget will be automatically incorporated into this Agreement by reference andform a part of this Agreement. The Clinical Development Manufacturing Budget will be reviewed by the JMC and approved by the JSC (i) at least[***] (and [***] for the first Calendar Year of the applicable [***] Clinical Development Manufacturing Budget Period) based on: (A) the Parties’good faith estimation of the anticipated Clinical Development Manufacturing Activities to be conducted during the relevant [***] ClinicalDevelopment Manufacturing Budget Period; and (B) information prepared by the Parties in good faith for their own internal planning processesrelating to anticipated Clinical Development Manufacturing Activities; or (ii) whenever the total Clinical Development Manufacturing Costs for anygiven Calendar Quarter are reasonably expected to be at least [***] percent ([***]%) higher than the Clinical Development Manufacturing Budget forsuch Calendar Quarter, whether as a result of any amendments to the CMC Development Plan or increases in costs for the Clinical DevelopmentManufacturing Activities already planned for such Calendar Quarter (provided, however, that following the beginning of a Calendar Year, the JSC maynot increase the Clinical Development Manufacturing Budget for such Calendar Year by more than [***] percent ([***]%) without the Parties’ priorwritten approval). Each such amended or updated Clinical Development Manufacturing Budget will automatically replace the respective ClinicalDevelopment Manufacturing Budget previously in effect, and will be automatically incorporated into this Agreement by reference and form a part ofthis Agreement. With respect to any Calendar Quarter, in no event will a Party be responsible for any Clinical Development Manufacturing Costs thatexceed the respective portion of the then-current Clinical Development Manufacturing Budget recommended and proposed by the JMC and approvedby the JSC, except as otherwise provided in Section 10.4(b). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -54-Confidential Treatment Requested by MeiraGTx Holdings plc (c) Process Development Budget. The CMC Development Collaboration will be subject to a rolling budget covering Process Development Costsassociated with the anticipated Process Development Activities to be performed during the [***] (such [***] period, the “[***] Process DevelopmentBudget Period”), in each case broken down by Calendar Quarter and broken out on a line item basis to show Out-of-Pocket Costs and FTE Costs ofFTEs directly engaged to perform each such Process Development Activity and further broken down by Party (the “Process Development Budget”).[***] following the Effective Date, the Process Development Budget will be developed by the JMC, and recommended and proposed to the JSC forreview and approval, and upon approval by the JSC, each such Process Development Budget will be automatically incorporated into this Agreementby reference and form a part of this Agreement. The Process Development Budget will be reviewed by the JMC and approved by the JSC (i) at least[***] (and [***] for the first Calendar Year of the applicable [***] Process Development Budget Period) based on: (A) the Parties’ good faithestimation of the anticipated Process Development Activities to be conducted during the relevant [***] Process Development Budget Period; and(B) information prepared by the Parties in good faith for their own internal planning processes relating to anticipated Process Development Activities;or (ii) whenever the total Process Development Costs for any given Calendar Quarter are reasonably expected to be at least [***] percent ([***]%)higher than the Process Development Budget for such Calendar Quarter, whether as a result of any amendments to the CMC Development Plan orincreases in costs for the Process Development Activities already planned for such Calendar Quarter (provided, however, that following the beginningof a Calendar Year, the JSC may not increase the Process Development Budget for such Calendar Year by more than [***] percent ([***]%) without theParties’ prior written approval). Each such amended or updated Process Development Budget will automatically replace the respective ProcessDevelopment Budget previously in effect, and will be automatically incorporated into this Agreement by reference and form a part of this Agreement.With respect to any Calendar Quarter, in no event will a Party be responsible for any Process Development Costs that exceed the respective portion ofthe then-current Process Development Budget recommended and proposed by the JMC and approved by the JSC, except as otherwise provided inSection 10.5(b).(d) Clinical Development Manufacturing Costs. Janssen will be responsible for [***] of the documented Clinical Development ManufacturingCosts; provided, however, that in no event will any Clinical Development Manufacturing Costs for any Calendar Quarter exceed the applicableClinical Development Manufacturing Budget for such Calendar Quarter, except as otherwise provided in Section 10.4(b). All Clinical DevelopmentManufacturing Costs shall be reconciled and paid in accordance with the procedure described in Section 10.4. Notwithstanding anything herein to thecontrary, [***].(e) Process Development Costs. Janssen and MeiraGTx will each be responsible for [***] of the documented Process Development Costs;provided, however, that in no event will a Party’s portion of any Process Development Costs for any Calendar Quarter exceed its portion of theapplicable Process Development Budget for such Calendar Quarter, except as otherwise provided in Section 10.5(b). All Process Development Costsshall be reconciled and paid in accordance with the procedure described in Section 10.5. Notwithstanding anything herein to the contrary, in no eventwill [***] of any Process Development Costs [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -55-Confidential Treatment Requested by MeiraGTx Holdings plc 8.5.[***] Manufacturing Services.During the [***] after the Effective Date, if MeiraGTx intends to grant any Third Party the right to reserve for such Third Party one (1) vectorManufacturing suite at the MeiraGTx Facility for Manufacturing of any product other than a Product, then MeiraGTx shall [***]. If Janssen notifiesMeiraGTx in writing within [***] of receipt of MeiraGTx’s notice that Janssen desires to [***], then the Parties [***] (the “Manufacturing ServicesAgreement”), which agreement will contain customary terms and conditions, including [***], except that, if Janssen [***] for Manufacturing. IfJanssen either responds to MeiraGTx that [***]. 9.COMMERCIALIZATIONJanssen will be solely responsible, at its sole cost and expense, for all aspects of Commercialization in the Territory of each Clinical IRD Product andeach Janssen Research IRD Product, including planning and implementation, distribution, booking of sales, pricing and reimbursement, except thatJanssen shall use Commercially Reasonable Efforts, at its expense, to Commercialize (a) for each Clinical IRD Target, [***] and (b) for each ResearchIRD Target for which Janssen has exercised an Option, [***], in each case ((a) and (b)) following the obtaining of Regulatory Approval for suchClinical IRD Product or Janssen Research IRD Product (as applicable) and [***] such Regulatory Approval has been obtained. 10.FINANCIAL PROVISIONS 10.1.Upfront Payment.Provided that this Agreement is not terminated in accordance with Section 14.3, Janssen shall pay to MeiraGTx within [***], a one-time,non-refundable, non-creditable payment of One Hundred Million Dollars ($100,000,000) in partial consideration of the collaborations, options,licenses and rights granted by MeiraGTx to Janssen under this Agreement (“Upfront Payment”). For clarity, Janssen shall have no obligation to paythe Upfront Payment in the event that this Agreement is terminated in accordance with Section 14.3. 10.2.Research Costs.(a) Invoices. For each Research Plan, commencing upon the first Calendar Quarter immediately following the date that such Research Plan is firstapproved by the JSC pursuant to Section 3.2 and continuing thereafter during the applicable Research Plan Term so long as MeiraGTx incurs ResearchCosts under this Agreement, MeiraGTx will submit to Janssen within [***] after the conclusion of each Calendar Quarter an invoice setting forth theResearch Costs that MeiraGTx incurred in performing its Research Plan Activities under such Research Plan in such Calendar Quarter. Each invoiceshall include the nature and amount of Research services rendered or deliverables provided, and each invoice shall provide proper support for expensesincluded on the invoice. Reasonable support documents for Out-of-Pocket Costs include invoices or pro forma invoices from Third Party vendors. ForFTE reimbursement, proper support includes an FTE time report break down by function. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -56-Confidential Treatment Requested by MeiraGTx Holdings plc (b) Research Costs. Within [***] after receipt of such invoice, Janssen shall make a reconciliation payment to MeiraGTx equal to [***] percent([***]%) of all Research Costs incurred during such Calendar Quarter; provided, however, that if the total Research Costs for such Calendar Quarterexceeds the Research Budget for such Calendar Quarter, then MeiraGTx will provide written notice to Janssen as far in advance as reasonably possibleof such cost overrun along with a reasonably detailed explanation of such cost overrun. Janssen shall pay for such excess only if: (i) the JSC approvesof such excess; or (ii) such excess exceeds the Research Budget for such Calendar Quarter by [***] percent ([***]%) or less and is attributable to achange in Applicable Law; provided, however, that in no event will any total Research Costs for a Calendar Year exceed the Research Budget Cap forsuch Calendar Year, which excess may only be approved by the Parties in writing. Janssen shall not be obligated to pay for any Research Costs inexcess of the Research Budget except as otherwise set forth in this Section 10.2(b) or with the Parties’ written approval therefor. All such reconciliationpayments shall be non-creditable and non-refundable. In addition, subject to the terms and conditions herein, if any Research Plan Activities willexceed the respective portion of the applicable Research Budget or extend beyond the applicable Research Plan Term, then (1) MeiraGTx shallprovide written notice to Janssen, as far in advance as reasonably possible, that MeiraGTx intends to not perform such Research Plan Activities (withreasonable detail regarding such Research Plan Activities and Research Costs), (2) MeiraGTx shall afford the JSC or the Parties, as applicable, areasonable opportunity to approve additional funding or reimburse such Research Plan Activities as permitted in this Section 10.2(b), and(3) MeiraGTx shall not be obligated to undertake such Research Plan Activities unless the JSC or the Parties, as applicable, agree to provide suchadditional funding as permitted in this Section 10.2(b). 10.3.Clinical Development Costs.(a) Invoices. For each Clinical IRD Product (and any Janssen Research IRD Product subject to Section 6.2(c)), commencing upon the firstCalendar Quarter immediately following the Effective Date and continuing thereafter so long as MeiraGTx incurs Clinical Development Costs underthis Agreement, MeiraGTx will submit to Janssen within [***] after the conclusion of such Calendar Quarter an invoice setting forth the ClinicalDevelopment Costs MeiraGTx incurred in such Calendar Quarter. Each invoice shall include the nature and amount of Development services renderedor deliverables provided, and each invoice shall provide proper support for expenses included on the invoice. Reasonable support documents forOut-of-Pocket Costs include invoices or pro forma invoices from Third Party vendors. For FTE reimbursement, proper support includes an FTE timereport break down by function.(b) Clinical Development Costs. Within [***] after receipt of such invoice and report, Janssen will pay to MeiraGTx all the ClinicalDevelopment Costs set forth in each such invoice; provided, however, that if the total Clinical Development Costs for such Calendar Quarter exceedsthe Clinical Development Budget for such Calendar Quarter, then MeiraGTx will provide written notice to Janssen as far in advance as reasonablypossible of such cost overrun along with a reasonably detailed explanation of such cost overrun. Janssen shall pay for such excess only if: (i) the JSCapproves of such excess; or (ii) such excess exceeds the Clinical Development Budget for such Calendar Quarter by [***] percent ([***]%) or less andis attributable to (A) a change in Applicable Law, (B) a variation in actual patient enrollment from projected patient enrollment or (C) a change to aclinical trial protocol required or requested by any Regulatory Authority; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -57-Confidential Treatment Requested by MeiraGTx Holdings plc provided, however, that in no event will total Clinical Development Costs in any given Calendar Year exceed the Clinical Development Budget forsuch Calendar Year by more than [***] percent ([***]%), which excess may only be approved by the Parties in writing. Janssen shall not be obligatedto pay for any Clinical Development Costs in excess of the Clinical Development Budget except as otherwise set forth in this Section 10.3(b). All suchpayments of MeiraGTx’s Clinical Development Costs shall be non-creditable and non-refundable. In addition, subject to the terms and conditionsherein, if any Clinical Development Plan Activities will exceed the respective portion of the applicable Clinical Development Budget or extendbeyond the applicable Clinical Development Plan Term, then (x) MeiraGTx shall provide written notice to Janssen, as far in advance as reasonablypossible, that MeiraGTx intends to not perform such Clinical Development Plan Activities (with reasonable detail regarding such ClinicalDevelopment Plan Activities and Clinical Development Costs), (y) MeiraGTx shall afford the JSC or the Parties, as applicable, a reasonableopportunity to approve additional funding or reimburse such Clinical Development Plan Activities as permitted in this Section 10.3(b), and(z) MeiraGTx shall not be obligated to undertake such Clinical Development Plan Activities unless the JSC or the Parties, as applicable, agree toprovide such additional funding as permitted in this Section 10.3(b). 10.4.Clinical Development Manufacturing Costs.(a) Invoices. For each Clinical IRD Product (and any Janssen Research IRD Product subject to Section 6.2(c)), commencing upon the [***]following the Effective Date and continuing thereafter so long as MeiraGTx incurs Clinical Development Manufacturing Costs under this Agreement,MeiraGTx will submit to Janssen within [***] after the conclusion of such Calendar Quarter an invoice setting forth the Clinical DevelopmentManufacturing Costs MeiraGTx incurred in such Calendar Quarter. Each invoice shall include the nature and amount of services rendered ordeliverables provided in connection with Clinical Development Manufacturing Activities, and each invoice shall provide proper support for expensesincluded on the invoice. Reasonable support documents for Out-of-Pocket Costs include invoices or pro forma invoices from Third Party vendors. ForFTE reimbursement, proper support includes an FTE time report break down by function.(b) Clinical Development Manufacturing Costs. Within [***] after receipt of such invoice and report, Janssen will pay to MeiraGTx [***] theClinical Development Manufacturing Costs set forth in each such invoice; provided, however, that if the total Clinical Development ManufacturingCosts for such Calendar Quarter exceeds the Clinical Development Manufacturing Budget for such Calendar Quarter, then MeiraGTx will providewritten notice to Janssen as far in advance as reasonably possible of such cost overrun along with a reasonably detailed explanation of such costoverrun. Janssen shall pay for such excess only if: (i) the JSC approves of such excess; or (ii) such excess exceeds the Clinical DevelopmentManufacturing Budget for such Calendar Quarter by [***] percent ([***]%) or less and is attributable to (A) a change in Applicable Law, (B) avariation in actual patient enrollment from projected patient enrollment or (C) a change to a clinical trial protocol required or requested by anyRegulatory Authority; provided, however, that in no event will total Clinical Development Manufacturing Costs in any given Calendar Year exceedthe Clinical Development Manufacturing Budget for such Calendar Year by more than [***] percent ([***]%), which excess may only be approved bythe Parties in writing. Janssen Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -58-Confidential Treatment Requested by MeiraGTx Holdings plc shall not be obligated to pay for any Clinical Development Manufacturing Costs in excess of the Clinical Development Manufacturing Budget exceptas otherwise set forth in this Section 10.4(b). All such payments of MeiraGTx’s Clinical Development Manufacturing Costs shall be non-creditable andnon-refundable. In addition, subject to the terms and conditions herein, if any Clinical Development Manufacturing Activities will exceed therespective portion of the applicable Clinical Development Manufacturing Budget or extend beyond the applicable CMC Development Term, then(x) MeiraGTx shall provide written notice to Janssen, as far in advance as reasonably possible, that MeiraGTx intends to not perform such ClinicalDevelopment Manufacturing Activities (with reasonable detail regarding such Clinical Development Manufacturing Activities and ClinicalDevelopment Manufacturing Costs), (y) MeiraGTx shall afford the JSC or the Parties, as applicable, a reasonable opportunity to approve additionalfunding or reimburse such Clinical Development Manufacturing Activities as permitted in this Section 10.4(b), and (z) MeiraGTx shall not beobligated to undertake such Clinical Development Manufacturing Activities unless the JSC or the Parties, as applicable, agree to provide suchadditional funding as permitted in this Section 10.4(b). 10.5.Process Development Costs.(a) Invoices. Within [***] after the conclusion of each Calendar Quarter, MeiraGTx and Janssen each will submit to the other Party an invoicesetting forth the Process Development Costs it incurred in such Calendar Quarter under the CMC Development Plan. Each invoice shall include thenature and amount of services rendered or deliverables provided in connection with Process Development Activities, and each invoice shall provideproper support for expenses included on the invoice. Reasonable support documents for Out-of-Pocket Costs include invoices or pro forma invoicesfrom Third Party vendors. For FTE reimbursement, proper support includes an FTE time report break down by function.(b) Process Development Costs. Within [***] after receipt of such invoices, the Parties will confer and agree in writing on whether areconciliation payment is due from MeiraGTx to Janssen or Janssen to MeiraGTx, and if so, the amount of such reconciliation payment, so that [***]Process Development Costs; provided, however, that if the Process Development Costs incurred by a Party for such Calendar Quarter exceeds suchParty’s Process Development Budget for such Calendar Quarter, then such Party will provide written notice to the other Party as far in advance asreasonably possible of such cost overrun along with a reasonably detailed explanation of such cost overrun. The other Party shall pay for its portion ofsuch excess only if: (i) the JSC approves of such excess; or (ii) such excess exceeds the Process Development Budget for such Calendar Quarter by[***] percent ([***]%) or less and is attributable to a change in Applicable Law; provided, however, that in no event will total Process DevelopmentCosts in any given Calendar Year exceed the Process Development Budget for such Calendar Year by more than [***] percent ([***]%), which excessmay only be approved by the Parties in writing. The Party owed a reconciliation in connection with any Process Development Costs shall provide aninvoice for the amount of such reconciliation payment to the paying Party, and such paying Party will make such reconciliation payment to the otherParty, within [***] following receipt of the other Party’s invoice for such amount. Janssen shall not be obligated to pay for any portion of ProcessDevelopment Costs in excess of the applicable portion of the applicable Process Development Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -59-Confidential Treatment Requested by MeiraGTx Holdings plc Budget except as otherwise set forth in this Section 10.5(b). All such payments shall be non-creditable and non-refundable. In addition, subject to theterms and conditions herein, if any Process Development Activities will exceed the respective portion of the applicable Process Development Budgetor extend beyond the applicable CMC Development Term, then (x) MeiraGTx shall provide written notice to Janssen, as far in advance as reasonablypossible, that MeiraGTx intends not to perform such Process Development Activities (with reasonable detail regarding such Process DevelopmentActivities and Process Development Costs), (y) MeiraGTx shall afford the JSC or the Parties, as applicable, a reasonable opportunity to approveadditional funding or reimburse such Process Development Activities as permitted in this Section 10.5(b), and (z) MeiraGTx shall not be obligated toundertake such Process Development Activities unless the JSC or the Parties, as applicable, agree to provide such additional funding as permitted inthis Section 10.5(b). 10.6.Development Milestone Payments.In partial consideration of the cooperation of MeiraGTx in conducting the Clinical Development Plan Activities hereunder, and subject toSection 10.10(b), on a Product-by-Product basis, Janssen shall make milestone payments to MeiraGTx (each, a “Development MilestonePayment”) upon the first (1st) achievement of each milestone event set forth in this Section 10.6 (each, a “Development Milestone Event”) as set forthin the applicable tables below. Each Development Milestone Payment shall be non-refundable and non-creditable.(a) Clinical IRD Products. Subject to the terms and conditions of this Section 10.6, on a Clinical IRD Product-by-Clinical IRD Product basis,Janssen shall make the Development Milestone Payments provided below to MeiraGTx upon the first (1st) achievement of the correspondingDevelopment Milestone Event for the applicable Clinical IRD Product. Each Development Milestone Payment for a Clinical IRD Product will bepayable only once with respect to the respective Clinical IRD Product, even if the corresponding Development Milestone Event occurs: (i) more thanonce; (ii) with respect to more than one (1) Gene Therapy Product that treats the same Clinical IRD Target Indication as such Clinical IRD Product,including if such Clinical IRD Product [***]; or (iii) for Development Milestone Events set forth in the tables below, with respect to more than one(1) Indication. The aggregate total of all Development Milestone Payments made with respect to each Clinical IRD Product shall not exceed theamount identified as the Development Milestone Cap for such Clinical IRD Product in the applicable table below.(i) RPGR Product Development Milestone Event Development Milestone Payment (USD)[***] [***][***] [***][***] [***][***] [***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -60-Confidential Treatment Requested by MeiraGTx Holdings plc (ii) CNGB3 Product Development Milestone Event Development Milestone Payment (USD)[***] [***][***] [***][***] [***][***] [***](iii) CNGA3 Product Development Milestone Event Development Milestone Payment (USD)[***] [***][***] [***][***] [***][***] [***](iv) [***] Product (subject to Section 2.1(b)) Development Milestone Event Development Milestone Payment (USD)[***] [***][***] [***][***] [***][***] [***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -61-Confidential Treatment Requested by MeiraGTx Holdings plc (b) Research IRD Products. Subject to the terms and conditions of this Section 10.6, on a Janssen Research IRD Product-by-Janssen Research IRDProduct basis, Janssen shall make the Development Milestone Payments provided below to MeiraGTx upon the first (1st) achievement of thecorresponding Development Milestone Event for each Janssen Research IRD Product. Each Development Milestone Payment for a Janssen ResearchIRD Product will be payable only once, even if the corresponding Development Milestone Event occurs: (i) more than once; (ii) with respect to morethan one (1) Gene Therapy Product that treats the same Research IRD Target Indication as such Janssen Research IRD Product, including if suchJanssen Research IRD Product [***]; or (iii) for Development Milestone Events set forth in the table below, with respect to more than one Indication.The aggregate total of all Development Milestone Payments made with respect to each Janssen Research IRD Product shall not exceed the amountidentified as the Development Milestone Cap for such Janssen Research IRD Product in the table below.(i) Each Janssen Research IRD Product Development Milestone Event Development Milestone Payment (USD)[***] [***][***] [***][***] [***][***] [***](c) Payment Terms for Development Milestone Payments. Janssen shall provide MeiraGTx with written notice of the achievement of eachDevelopment Milestone Event for which payment is due hereunder within [***] after the Calendar Quarter in which such Development MilestoneEvent has been achieved. After receipt of such notice, MeiraGTx shall submit an invoice to Janssen for the corresponding Development MilestonePayment, and Janssen shall make the corresponding Development Milestone Payment to MeiraGTx within [***] after Janssen’s receipt of such invoice. 10.7.Commercial Milestone Payments.(a) Commercial Milestone Payments and Events for Clinical IRD Products. In partial consideration of the cooperation provided by MeiraGTx toJanssen under this Agreement, and subject to Section 10.10(b), on a Clinical IRD Product-by-Clinical IRD Product basis, Janssen shall make one (1)-time payments of each of the sales milestone payments indicated below (each, a “Commercial Milestone Payment”) to MeiraGTx when theworldwide, aggregate (i.e., cumulative since First Commercial Sale) Net Sales of a Clinical IRD Product first achieves the Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -62-Confidential Treatment Requested by MeiraGTx Holdings plc Dollar thresholds indicated in the table below (each, a “Commercial Milestone Event”). Each Commercial Milestone Payment will be payable onlyonce with respect to a Clinical IRD Product, notwithstanding the number of times a Dollar threshold indicated in the table below is reached. EachCommercial Milestone Payment shall be non-refundable and non-creditable. Commercial Milestone Event Commercial Milestone Payment (USD)[***] [***][***] [***][***] [***](b) Payment Terms for Commercial Milestone Payments. Janssen shall provide MeiraGTx with written notice of the achievement of eachCommercial Milestone Event within [***] after the Calendar Quarter in which such Commercial Milestone Event is achieved. Janssen shall make thecorresponding Commercial Milestone Payment to MeiraGTx within [***] following the end of the Calendar Quarter in which such CommercialMilestone Event was achieved.(c) Commercial Milestone Terms for Janssen Research IRD Products. For the avoidance of doubt, Janssen does not owe MeiraGTx anycommercial milestone payments for Research IRD Products. 10.8.Royalties.In partial consideration of the collaborations, options, licenses and rights granted by MeiraGTx to Janssen under this Agreement, Janssen shall makeroyalty payments to MeiraGTx, on a Product-by-Product basis, based on Annual Net Sales of the applicable Product within the Field in the Territory, asreported by Janssen or its Affiliates or Sublicensees for each Calendar Quarter, at the applicable rates set forth below during the applicable RoyaltyTerm and subject to Section 10.9, Section 10.10, and where applicable, Section 15.2(a)(ii) (such payments, “Royalties”).(a) Clinical IRD Products. Janssen shall pay to MeiraGTx Royalties, on a Clinical IRD Product-by-Clinical IRD Product basis, on Annual NetSales for each Clinical IRD Product within the Field in the Territory equal to twenty percent (20%) of Annual Net Sales of such Clinical IRD Product inthe Territory.(b) Janssen Research IRD Products. Janssen shall pay to MeiraGTx Royalties, on a Janssen Research IRD Product-by-Janssen Research IRDProduct basis, on Annual Net Sales for each such Janssen Research IRD Product within the Field in the Territory equal to [***] percent ([***]%) ofAnnual Net Sales of such Janssen Research IRD Product in the Territory. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -63-Confidential Treatment Requested by MeiraGTx Holdings plc 10.9.Additional Royalty Provisions.(a) Royalty Term. Subject to this Section 10.9, on a Product-by-Product and country-by-country basis, the Royalties due under Section 10.8 shallbe payable on Annual Net Sales commencing from the First Commercial Sale of such Product in a country until the later of: (i) expiration of the lastValid Claim of: [***] in each case ((A)-(D)) in such country; (ii) ten (10) years from the date of the First Commercial Sale of such Product in suchcountry; or (iii) expiration of the last Data Exclusivity Right applicable to such Product in such country (the “Royalty Term”).(b) Royalty Step-Down. For each Product and for any period during the Royalty Term in which the sale of such Product in a given country isneither: (i) Covered by any Valid Claim described in Section 10.9(a)(i); nor (ii) protected by any Data Exclusivity Right applicable to such Product insuch country, then the Royalty rate applicable to Net Sales of such Product in such country during such period shall be equal to [***] percent([***]%) of the applicable Royalty rate set forth in Section 10.8 on Net Sales (i.e., [***] percent ([***]%) for Clinical IRD Products and [***] percent([***]%) for Janssen Research IRD Products).(c) Loss of Market Exclusivity of a Product Due to a Biosimilar Product. On a country-by-country basis, if a Loss of Market Exclusivity Due to aBiosimilar Product occurs with respect to a Product in a country, the Royalty rate applicable to Net Sales of such Product in such country shall be equalto [***] percent ([***]%) of the applicable Royalty rate set forth in Section 10.8 on Net Sales from the date such Loss of Market Exclusivity Due to aBiosimilar Product occurs until the end of the Royalty Term (i.e., [***] percent ([***]%) for Clinical IRD Products and [***] percent ([***]%) forJanssen Research IRD Products).(d) Loss of Market Exclusivity of a Clinical IRD Product Due to a Competing Product. On a country-by-country basis, if a Loss of MarketExclusivity Due to a Competing Product occurs with respect to a Clinical IRD Product in a country, the Royalty rate applicable to Net Sales of suchClinical IRD Product in such country shall be equal to [***] percent ([***]%) of the applicable Royalty rate set forth in Section 10.8 on Net Sales from[***] until [***] (i.e., [***]% for Clinical IRD Products).(e) Single Royalty. Only a single Royalty shall be due under this Agreement: (i) with respect to the sale of the same unit of Product; and (ii) onthe sale of a Product even if the Manufacture or Commercialization of such Product Covered more than one (1) Valid Claim described inSection 10.9(a)(i).(f) Royalty Minimum. Notwithstanding anything to the contrary in this Agreement, in no event will the applicable Royalty otherwise due toMeiraGTx in a Calendar Quarter be reduced by more than [***] due to the deductions contemplated in this Agreement (i.e., not below [***] forClinical IRD Products and [***] percent ([***]%) for Janssen Research IRD Products); provided, however, that [***].(g) Compulsory Licenses and Other Step-In Rights. If Janssen, its Affiliates or any of its Sublicensees are required to grant any licenses or otherrights to a Third Party (including any Governmental Authority) to Develop, Manufacture, or Commercialize a Product because of the actions of anyGovernmental Authority, then the Royalty rates set forth in Section 10.8 shall not apply, and instead, [***] for each such Product reflecting theapplicable market for such Product in such country, subject to [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -64-Confidential Treatment Requested by MeiraGTx Holdings plc (h) Combination Products. In the event that a Party reasonably determines that any Product will be incorporated into any other product sold byJanssen or its Affiliates or Sublicensees and such Product will not be priced separately from such other product sold by Janssen or its Affiliates, suchParty shall [***] provide written notice thereof to the other Party. Upon receipt of such notice, the Parties shall [***] enter into good faith discussionsto determine the reasonable Net Sales value with respect to such Product, which shall be [***]. 10.10.Third Party Obligations.(a) Existing Third Party Obligations. [***] shall remain responsible for the payment of royalty, milestone, and other payment obligations, if any,due to Third Parties in connection with any Third Party License that exists as of the Effective Date and under which MeiraGTx Technology orMeiraGTx Research Technology has been licensed to MeiraGTx and is sublicensed to Janssen under Section 4.1 for Clinical IRD Products and JanssenResearch IRD Products (the “Existing Third Party Obligations”). All such payments in respect of the Existing Third Party Obligations shall be made[***] in accordance with the terms of its agreements with the applicable Third Party License.(b) Future Third Party Obligations. If either Party reasonably determines that licensing Future Third Party Obligations would be necessary oruseful to Develop, Manufacture, or Commercialize any Product in the Field in the Territory under this Agreement, then MeiraGTx shall have the firstright to negotiate and acquire (subject to Janssen’s approval; and, for clarity, if MeiraGTx declines to exercise its first right to negotiate and acquiresuch Future Third Party Obligations or otherwise fails to do so within [***] of any request therefor by Janssen, Janssen shall have the right to do so) aThird Party License to such Future Third Party Obligations. If either Party negotiates and acquires a Third Party License to such Future Third PartyObligations, then: (i) [***]; (ii) [***]; provided, however, that Janssen shall have the right to [***] for such Product [***] percent ([***]%) of the [***]paid to such Third Party under such Third Party License, and to the extent that Janssen cannot [***] paid to such Third Party exceed any [***] owed toMeiraGTx for such Product at the time of payment to such Third Party, then Janssen may [***] payable for such Product; and (iii) Janssen thereaftershall have the right to [***] up to [***] percent ([***]%) of the [***] subject to the following conditions: (A) Janssen may [***] and [***]; (B) such[***]; (C) if any such Third Party License either: (1) includes additional Intellectual Property Rights other than Intellectual Property Rights that areControlled by a Third Party and that are necessary or useful to Develop, Manufacture, or Commercialize any Product in the Field in the Territory; or(2) Covers products other than such Product, then any such [***] would be equitably allocated by Janssen in good faith among all products andprograms to which such Third Party License applies; and (D) to the extent that Janssen cannot [***], then Janssen may [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -65-Confidential Treatment Requested by MeiraGTx Holdings plc (c) Breaches, Amendments and Terminations. Neither MeiraGTx nor its Affiliates will breach or default under any Existing Third PartyObligation or any Third Party License for a Future Third Party Obligation. Neither MeiraGTx nor its Affiliates will terminate any such Third PartyLicense in a manner that would terminate any rights that are licensed to Janssen hereunder or otherwise diminish the scope or exclusivity of suchIntellectual Property Rights licensed to Janssen hereunder. In the event that MeiraGTx or one of its Affiliates receives notice of a potential dispute inconnection with or an alleged breach by MeiraGTx or one of its Affiliates under any such Third Party License, where termination of such Third PartyLicense or any diminishment of the scope or exclusivity of such Intellectual Property Rights licensed to Janssen hereunder is or could be sought by thecounterparty, then MeiraGTx will [***], but in no event less than [***] thereafter, provide written notice thereof to Janssen and grant Janssen the right(but not the obligation) to cure such alleged breach, to the extent not prohibited by such Third Party License. In the event that MeiraGTx receives anotice of termination of such a Third Party License by a counterparty, MeiraGTx will [***], but in any event within [***] following receipt of suchnotice, notify Janssen of such termination in writing. In the event that MeiraGTx or one of its Affiliates intends to materially amend such a Third PartyLicense, then MeiraGTx will [***], but in no event less than [***] before, provide written notice thereof to Janssen. Janssen will have the right (but notthe obligation), acting reasonably, to reject any amendment that would increase Janssen’s obligations under this Agreement (including any financialobligations) or diminish the licenses or rights granted to Janssen under this Agreement and in the event of any such rejection, neither MeiraGTx nor itsAffiliates will enter into any such amendment unless and until Janssen approves such amendment. 10.11.Reports and Royalty Payments.For as long as Royalties are due under Section 10.8, Janssen shall furnish to MeiraGTx a draft Sales & Royalty Report, within [***] after the end ofeach Calendar Quarter, showing the estimated amount of Annual Net Sales of Products and Royalties due for such Calendar Quarter. Janssen shallfurnish to MeiraGTx a final Sales & Royalty Report and pay such Royalties contained in such final Sales & Royalty Report within [***] following theend of the applicable Calendar Quarter. 10.12.Disclaimer.MeiraGTx expressly acknowledges and agrees that, despite the efforts and obligations required by this Agreement which may result in the achievementof a Development Milestone Event, Commercial Milestone Event or Net Sales of Product, such event or Net Sales of Product may not be achieved andMeiraGTx, in that case, would not be entitled to receive such further payments hereunder (the Development Milestone Payment, CommercialMilestone Payment or Royalties, as the case may be), other than (i) the Upfront Payment, (ii) Research Costs to be paid as set forth in Section 10.2, (iii)Clinical Development Costs to be paid as set forth in Section 10.3, (iv) Clinical Development Manufacturing Costs to be paid as set forth inSection 10.4, (v) Process Development Costs to be paid as set forth in Section 10.5, (vi) any Option Fee (if and when the respective Option is exercisedas set forth in Section 3.9(b)), (vii) such portion of the proceeds of Priority Review Vouchers to be paid to MeiraGTx as set forth in Section 7.1(d) and(viii) such other costs to be paid by Janssen for Manufacturing of Products as set forth in Section 8.1(d). Any Development Milestone Payment,Commercial Milestone Payment or Royalties are contingent upon satisfaction of the conditions provided for herein which may not be satisfied. Exceptas otherwise expressly provided herein (but without waiving the implied covenant of good faith and fair dealing), Janssen will be under no obligationto use its Commercially Reasonable Efforts, best efforts or any other standard of diligence with respect to satisfying the conditions to any paymenthereunder. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -66-Confidential Treatment Requested by MeiraGTx Holdings plc 10.13.Payment Terms.(a) Manner of Payment. All payments to be made by a Party hereunder will be made in Dollars by wire transfer to such bank account as the otherParty may timely designate to the other in writing. Any payment which falls due on a date which is not a Business Day in the location from which thepayment will be made may be made on the next succeeding Business Day in such location. For the avoidance of doubt, no payment obligations shallbe incurred by either Party under or in connection with this Agreement unless and until the Effective Date.(b) Currency Exchange. With respect to Annual Net Sales invoiced in Dollars, the Annual Net Sales and the amounts due to MeiraGTx under thisAgreement shall be expressed in Dollars. When the conversion of payments from any foreign currency is required to be undertaken by Janssen, theDollar equivalent shall be calculated using Janssen’s then-current standard exchange rate methodology as applied in its external reporting for theconversion of foreign currency sales into Dollars.(c) Invoices. All invoices hereunder to be paid by a Party must reference a valid purchase order (“PO”) number which the other Party shallprovide to such Party within [***] after the Effective Date and invoices shall include the nature and amount of services rendered or deliverablesprovided and such other terms and conditions specified herein. Invoices to Janssen must be sent to the Johnson & Johnson Accounts PayableDepartment via [***] if MeiraGTx establishes a web invoice account or sent by postal mail to the address indicated on the PO. Invoices to MeiraGTxmust be sent to [***], with a cc to [***]. The other Party reserves the right to return to such Party unprocessed and unpaid those invoices that do notreference the applicable PO number. For clarity, no invoices shall be required with respect to any Royalties due under Section 10.8.(d) Withholding Taxes. Either Party (the “Withholding Party”) may withhold from payments due to the other Party (the “Non-WithholdingParty”) amounts for payment of any withholding tax that is required by Applicable Law to be paid to any taxing authority with respect to suchpayments, which shall be remitted in accordance with Applicable Law. Any such tax required to be withheld will be an expense of and borne by theNon-Withholding Party. If any such tax is assessed against and paid by the Withholding Party, then the Non-Withholding Party will indemnify andhold harmless the Withholding Party from and against such tax, including interest. The Withholding Party shall provide to the Non-Withholding Partyall relevant documentation and correspondence, and shall also provide to the Non-Withholding Party any other cooperation or assistance on areasonable basis as may be necessary to enable the Non-Withholding Party to claim exemption from such withholding taxes and to receive a refund ofsuch withholding tax or claim a foreign tax credit. The Withholding Party shall give proper evidence from time to time as to the payment of any suchtax. The Parties shall cooperate with each other in seeking deductions under any double taxation or other similar treaty or agreement from time to timein force. Such cooperation may include the Withholding Party making payments from a single source, where possible. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -67-Confidential Treatment Requested by MeiraGTx Holdings plc (e) Tax Forms. On the date of execution of this Agreement, MeiraGTx will deliver to Janssen an accurate and complete Internal Revenue ServiceForm W-8BEN-E certifying that MeiraGTx is entitled to the applicable benefits under the Income Tax Treaty between the United Kingdom and theUnited States.(f) Late Payments. Any undisputed payments or portions thereof due hereunder which are not paid when due will bear interest at the rate perannum equal to the lesser of: [***] or (ii) the highest rate permitted by Applicable Law, calculated on the number of days such payment is paid after thedate such payment is due, and compounded monthly (the “Interest Rate”).(g) Janssen Entities. [***] acting as paying agent for Janssen, may make certain payments due under this Agreement, and Janssen shall reimburse[***] for all such payments.10.14. Records and Audits.(a) Records. Each Party shall keep complete, true, and accurate books and records in accordance with its Accounting Standards in relation to thisAgreement, including with respect to Research Costs, Clinical Development Costs, Clinical Development Manufacturing Costs, Process DevelopmentCosts, Manufacturing Costs, Net Sales, and such other information contained in Sales & Royalty Reports. Each Party shall keep such books and recordsfor at least [***] following the Calendar Year to which they pertain.(b) Audits. Each Party (the “Auditing Party”) may, upon written request, cause an internationally-recognized independent accounting firm (the“Auditor”), which is reasonably acceptable to the other Party (the “Audited Party”), to inspect the relevant records of such Audited Party and itsAffiliates to verify the payments made and amounts reported by the Audited Party and the related reports, statements, and books of accounts, asapplicable. Such audit shall be limited to a period of time no more than [***] immediately preceding the year in which the audit is requested, and anaudit of the records relating to a particular Calendar Year may be conducted once and not more than once. Before beginning its audit, the Auditor shallexecute a written undertaking acceptable to the Audited Party by which the Auditor shall agree to keep confidential all information made available tothe Auditor during the audit. Each Party and its Affiliates shall make their records available for inspection by the Auditor during regular business hoursat such place or places where such records are customarily kept, upon receipt of reasonable advance notice from the Auditing Party. The records shallbe reviewed to verify the accuracy of the Audited Party’s Sales & Royalty Report and other payment obligations and compliance with the financialterms of this Agreement. Such inspection right shall not be exercised more than [***], unless the audit reveals a non-compliance by the Audited Partywith the terms of this Agreement in which case the audit may be repeated within [***] to confirm compliance. The Auditing Party agrees to hold inconfidence all information received and learned in the course of any audit in accordance with Article 12. The Auditor shall provide a draft audit reportand basis for any determination to the Audited Party prior to distributing the final report so that the Audited Party can provide comment on the draftreport. The final audit report will be provided to the Audited Party at the time such report is provided to the Auditing Party. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -68-Confidential Treatment Requested by MeiraGTx Holdings plc (c) Overpayments and Underpayments. If the final result of the inspection reveals an underpayment or an overpayment by either Party, theunderpaid or overpaid amount shall be settled within [***] of the Audited Party’s receipt of the final report. The Auditing Party shall pay for any audit,as well as its expenses associated with enforcing its rights with respect to any payments under this Agreement, except that, if an underpayment ofamounts due by the Audited Party of more than [***] percent ([***]%) of the total payments due under this Agreement for the applicable year isdiscovered, reasonable and necessary fees and expenses charged by the Auditor shall be paid by the Audited Party, subject to reasonable substantiationthereof. 11.INTELLECTUAL PROPERTY RIGHTS 11.1.Ownership under the Clinical IRD Programs and Research IRD Programs.(a) Ownership. As between the Parties, and subject to the licenses granted under this Agreement, each Party retains all rights, title, and interests inand to all Intellectual Property Rights that such Party owns or Controls as of the Effective Date or that it develops or otherwise acquires after theEffective Date outside the performance of the activities under this Agreement.(b) Sole Inventions. Each Party shall solely own any and all Inventions and Know-How developed, conceived, generated or reduced to practicesolely by it, its Affiliates or its or their employees, agents or independent contractors in the course of performing activities under any Research Plan orClinical Development Plan hereunder during the Term (“Sole Inventions”). Inventorship shall be determined in accordance with U.S. patent laws.(c) Joint Inventions. Except as set forth in Section 11.2, the Parties shall jointly own any and all Inventions and Know-How developed,conceived, generated or reduced to practice jointly by a Party, its Affiliates or its or their employees, agents or independent contractors together withanother Party, its Affiliates or its or their employees, agents or independent contractors in the course of performing activities under this Agreementduring the Term (“Joint Inventions”). All Patents Covering Joint Inventions shall be referred to herein as “Joint Patents”. Except to the extent a Partyis expressly limited by the terms of this Agreement, including via exclusive license to the other Party, each Party shall be entitled to practice, license(through multiple tiers), assign (their respective interest only) and otherwise exploit the Joint Inventions and Joint Patents in all countries andjurisdictions without the duty of accounting or seeking consent from the other Party. Upon the reasonable request of either Party, the other Party shallexecute documents that evidence or confirm the requesting Party’s right to engage in such activities. Inventorship shall be determined in accordancewith U.S. patent laws. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -69-Confidential Treatment Requested by MeiraGTx Holdings plc 11.2.Ownership under the CMC Development Collaboration.The Parties shall jointly own any and all CMC Development Inventions, CMC Development Know-How and CMC Development Patents regardless ofinventorship. Except to the extent a Party is expressly limited by the terms of this Agreement, including by exclusive license to the other Party, eachParty shall be entitled to practice, license (through multiple tiers), assign (their respective interest only) and otherwise exploit the CMC DevelopmentInventions, CMC Development Know-How and CMC Development Patents in all countries and jurisdictions without the duty of accounting or seekingconsent from the other Party. Upon the reasonable request of either Party, the other Party shall execute documents that evidence or confirm therequesting Party’s right to engage in such activities. 11.3.Duties to the Other Party.(a) Assignment. Each Party hereby assigns to the other Party, one-half of its interest in and to any CMC Development Inventions, CMCDevelopment Know-How and CMC Development Patents, including all rights of action and claims for damages and benefits arising due to past andpresent infringement of said rights, such that each Party owns an undivided joint interest in and to such CMC Development Inventions, CMCDevelopment Know-How and CMC Development Patents. Each Party shall and shall cause its Affiliates and contractors to, execute and take suchfurther actions reasonably necessary to effectuate such joint ownership in and to such CMC Development Inventions, CMC Development Know-Howand CMC Development Patents.(b) Disclosure. Each Party shall [***] disclose to the JSC all Inventions made by or on behalf of such Party and its Affiliates and subcontractors inthe course of performing activities under this Agreement during the Term, including all invention disclosures or other similar documents submitted tosuch Party by it, its Affiliates or its or their employees, agents or contractors relating to such Invention, and shall also respond to reasonable requestsfrom the other Party for additional information relating to any such Inventions.(c) Personnel Obligations. Each Party shall cause each employee, agent or contractor of such Party to, and shall cause each of its respectiveAffiliates or Sublicensees performing work under this Agreement to, prior to commencing such work, be bound by invention assignment obligations,including: (i) [***] reporting any Intellectual Property Right arising from such work; (ii) presently assigning to the applicable Party all of his, her or itsrights, title and interests in and to any Intellectual Property Right arising from such work (excluding any agreements with academic universities orother governmental entities, for which a non-exclusive license, or an option for an exclusive license may be obtained); (iii) reasonably cooperating inthe preparation, filing, prosecution, maintenance, defense, and enforcement of any Patent; and (iv) performing all acts and signing, executing,acknowledging and delivering any and all documents required for effecting the obligations and purposes of this Agreement.(d) Common Ownership under Joint Research Agreements. Notwithstanding anything to the contrary in this Agreement, neither Party will havethe right to invoke Common Ownership under a Joint Research Agreement pursuant to 35 U.S.C. § 102(c) (AIA) when exercising its rights under thisAgreement without the prior written consent of the other Party, such consent not to be unreasonably withheld, conditioned, or delayed. If a Party ispermitted to invoke the 35 U.S.C. § 102(c) (AIA) as required by the preceding sentence, the Parties will reasonably cooperate and coordinate theiractivities with respect to any submissions, filings or other activities in support thereof. The Parties acknowledge and agree that this Agreement is a“joint research agreement” as defined in 35 U.S.C. § 102(c) (AIA). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -70-Confidential Treatment Requested by MeiraGTx Holdings plc (e) Updated Exhibit. On each anniversary of the Effective Date during the Term, MeiraGTx shall provide to the JSC an updated copy of Exhibit1.168, setting forth a true, complete and accurate list of all Patents Controlled by MeiraGTx or any of its Affiliates as of such date, which claim orotherwise Cover any Clinical IRD Products, Clinical IRD Targets or MeiraGTx Know-How in the Field. 11.4.Patent Prosecution and Maintenance.(a) Sole Patents. Each Party shall have the sole and exclusive right to prosecute and maintain all Patents Covering each Party’s Sole Inventions[***], except to the extent the prosecution of such Patents is otherwise covered by another provision of this Section 11.4.(b) Clinical IRD Product Patents. Subject to the terms of this Section 11.4, the Parties shall jointly prosecute and maintain (i) the MeiraGTxPatents in MeiraGTx’s name and (ii) the Joint Patents Covering Clinical IRD Products in both Parties’ names (collectively, (i) and (ii), “Clinical IRDPatents”). The Parties shall [***]. The Parties shall [***]. Each Party shall be reasonably informed of the status of the Clinical IRD Patents, shall beprovided with all material correspondences received from any patent authorities in connection therewith and shall be entitled to consultation andreasonable input with respect to proposed filings and correspondence. In addition, the Parties shall [***] to consult with and consider the reasonableinput of [***] with respect to those MeiraGTx Patents licensed from [***] to MeiraGTx. In the event of a dispute, the Parties shall follow the process ofSection 5.8.(c) CMC Development Patents. Subject to the terms of this Section 11.4, as between the Parties, Janssen shall have the first right, but not theobligation, to prosecute and maintain all CMC Development Patents in both Parties’ names, subject to consultation and reasonable input fromMeiraGTx. The Parties shall [***] and will be joint owners of the CMC Development Patents. The Parties shall [***]. Janssen shall keep MeiraGTxreasonably informed of the status of the CMC Development Patents, shall provide MeiraGTx with all material correspondences received from anypatent authorities in connection therewith and shall in good faith consult with and consider reasonable input from MeiraGTx with respect to proposedfilings and correspondence. If Janssen decides not to prosecute or maintain any CMC Development Patent, Janssen shall notify MeiraGTx thereof inwriting, and MeiraGTx shall thereupon have the right, but not the obligation, to assume the prosecution and maintenance thereof in both Parties’names, [***], and [***]. In the event of a dispute, the Parties shall follow the process of Section 5.8.(d) Research IRD Product Patents.(i) Prior to the Receipt of the Applicable Option Exercise Notice. Prior to MeiraGTx’s receipt of an applicable Option Exercise Notice withrespect to a Research IRD Product, subject to the terms of this Section 11.4, as between the Parties, MeiraGTx shall have the first right, but not theobligation, to prosecute and maintain (A) the MeiraGTx Research Patents in MeiraGTx’s name and (B) Joint Patents Covering such Research IRDProduct in both Parties’ Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -71-Confidential Treatment Requested by MeiraGTx Holdings plc names (collectively, (A) and (B), “Research IRD Patents”), [***], subject to consultation and reasonable input from Janssen. MeiraGTx shall keepJanssen reasonably informed of the status of the Research IRD Patents and shall provide Janssen with all material correspondences received from anypatent authorities in connection therewith. If MeiraGTx decides not to prosecute or maintain any Research IRD Patent, MeiraGTx shall notify Janssenthereof in writing, and Janssen shall thereupon have the right, but not the obligation, to assume the prosecution and maintenance of such Research IRDPatent [***] (with such MeiraGTx Patents in MeiraGTx’s name and such Joint Patents in both Parties’ names).(ii) After Exercise of the Applicable Option. On or after Janssen’s exercise of an Option with respect to a Research IRD Product, subject tothe terms of this Section 11.4, as between the Parties, Janssen shall have the first right, but not the obligation, to prosecute and maintain (A) theMeiraGTx Research Patents Covering such Janssen Research IRD Product in MeiraGTx’s name and (B) the Joint Patents Covering such JanssenResearch IRD Product in both Parties’ names (collectively, (A) and (B), “Janssen Research IRD Patents”), subject to consultation and reasonable inputfrom MeiraGTx. [***] shall [***] the cost of and expenses thereof. The Parties shall [***] to facilitate the prosecution and maintenance of such JanssenResearch IRD Patents. Janssen shall keep MeiraGTx reasonably informed of the status of the Janssen Research IRD Patents, shall provide MeiraGTxwith all material correspondences received from any patent authorities in connection therewith and shall in good faith consult with and considerreasonable input from MeiraGTx with respect to proposed filings and correspondence. If Janssen decides not to prosecute or maintain any JanssenResearch IRD Patent, Janssen shall notify MeiraGTx thereof in writing, and MeiraGTx shall thereupon have the right, but not the obligation, to assumethe prosecution and maintenance thereof (in MeiraGTx’s name with respect to any MeiraGTx Research Patent and both Parties’ names with respect toany Joint Patent Covering Janssen Research IRD Products)] [***], and [***] shall [***] the costs and expenses thereof. In addition, each Party shall[***] to consult with and consider the reasonable input of [***] with respect to those MeiraGTx Research Patents licensed from [***] to MeiraGTx andCovering such Janssen Research IRD Product. In the event of a dispute, the Parties shall follow the process of Section 5.8.(e) Cooperation. Upon a Party’s request, the other Party shall provide the prosecuting and maintaining Party with all reasonable assistance andcooperation in connection with its prosecution and maintenance of the applicable Patents, including by providing access to relevant persons andexecuting all documentation reasonably requested by the prosecuting and maintaining Party.(f) Patent Term Extension. The Parties shall cooperate with one another in seeking and obtaining patent term extensions (including any pediatricexclusivity extensions as may be available) or supplemental protection certificates or their equivalents in any country with respect to the Clinical IRDPatents, CMC Development Patents, Research IRD Patents and Janssen Research IRD Patents. [***] shall have [***] to obtain patent term extensionsor supplemental protection certificates or their equivalents with respect to such Patents that Cover Products and shall report to [***] on the statusthereof. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -72-Confidential Treatment Requested by MeiraGTx Holdings plc (g) Recordation. To the extent permitted by Applicable Law, if MeiraGTx is responsible for prosecuting any Clinical IRD Patent, CMCDevelopment Patent, Research IRD Patent or Janssen Research IRD Patent, as applicable, under this Section 11.4, then (i) [***] following the EffectiveDate, MeiraGTx shall record with each Governmental Authority (where such right is available with such Governmental Authority and suchGovernmental Authority permits the filing of a form of short notice of license) the licenses granted in Section 4.1 against each such Clinical IRDPatent, CMC Development Patent, Research IRD Patent or Janssen Research IRD Patent then issued by or filed with such Governmental Authority andbeing prosecuted by MeiraGTx, as applicable, and (ii) thereafter on a reasonably regular basis as determined by the JSC or upon Janssen’s reasonablewritten request, MeiraGTx shall record with each Governmental Authority (where such right is available with such Governmental Authority and suchGovernmental Authority permits the filing of a form of short notice of license) the licenses granted in Section 4.1 against each Clinical IRD Patent,CMC Development Patent, Research IRD Patent or Janssen Research IRD Patent newly filed with such Governmental Authority and being prosecutedby MeiraGTx, as applicable, in each case ((i)-(ii)) by using a form of short notice of license mutually agreed to by the Parties (but shall not, for theavoidance of doubt, file or publicly disclose this Agreement itself for such purposes). To the extent permitted by Applicable Law, if Janssen isresponsible for prosecuting any Clinical IRD Patent, CMC Development Patent, Research IRD Patent or Janssen Research IRD Patent, as applicable,under this Section 11.4, then Janssen shall have the right to record with a Governmental Authority (where such right is available with suchGovernmental Authority and such Governmental Authority permits the filing of a form of short notice of license) the licenses granted in Section 4.1against any such Clinical IRD Patent, CMC Development Patent, Research IRD Patent or Janssen Research IRD Patent then issued by or filed with suchGovernmental Authority and prosecuted by Janssen, as applicable, by using a form of short notice of license mutually agreed by the Parties (but shallnot, for the avoidance of doubt, file or publicly disclose this Agreement itself for such purposes). For clarity, for purposes of this Section 11.4(g), a formof short notice of license may include a short notice form provided by a Governmental Authority or, where permitted by such Governmental Authority,any other short notice of license drafted by the Parties (e.g., a custom short statement that the Parties confirm that certain scheduled Patents are subjectto a license grant). 11.5.Third Party Infringement; Patent Actions.(a) Notice. If either Party becomes aware of: (i) existing or threatened infringement, misappropriation or other violation by a Third Party of any ofthe MeiraGTx Technology, MeiraGTx Research Technology, Janssen Technology, CMC Development Technology or any Joint Patents (“Third PartyInfringement”); (ii) request for declaratory judgment, opposition, nullity action, interference, inter partes reexamination, inter partes review, post-grantreview, derivation proceeding or similar action alleging the invalidity, unenforceability or non-infringement of any of the MeiraGTx Patents,MeiraGTx Research Patents, Janssen Technology, CMC Development Patents or any Joint Patents (each, a “Licensed Patent Action”); or (iii) a BLAfor a Biosimilar Product referencing a Product submitted to a Party or a Regulatory Authority, it shall [***] notify the other Party in writing to thateffect, and the Parties will consult with each other regarding any actions to be taken. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -73-Confidential Treatment Requested by MeiraGTx Holdings plc (b) Enforcement Rights for MeiraGTx Know-How, CMC Development Technology and Clinical IRD Patents.(i) Janssen’s First Right of Enforcement. Janssen shall have the first right, but not the obligation, in the case of the MeiraGTx Know-How,CMC Development Technology or Clinical IRD Patents to bring and control an appropriate suit or other action or defend against any Third PartyInfringement or Licensed Patent Action with respect to any MeiraGTx Know-How, CMC Development Technology or Clinical IRD Patents. MeiraGTxshall provide reasonable assistance to Janssen in such enforcement or defense, at Janssen’s request and expense, including joining such action as aparty plaintiff to ensure legal standing if required by Applicable Laws to pursue such action or if requested by Janssen. Janssen shall consult withMeiraGTx and keep MeiraGTx reasonably informed of the status of the enforcement or defense of such MeiraGTx Know-How, CMC DevelopmentTechnology or Clinical IRD Patents. Janssen shall consider MeiraGTx’s comments with respect to the enforcement or defense of such MeiraGTxKnow-How, CMC Development Technology or Clinical IRD Patents, as applicable, in good faith. Prior to settling any such suit or action, Janssen shallnotify MeiraGTx in writing as to the terms of such proposed settlement to the extent relating to MeiraGTx Know-How, CMC Development Technologyor Clinical IRD Patents and shall not execute such settlement without MeiraGTx’s written consent if MeiraGTx identifies to Janssen in reasonabledetail a material risk of a material negative impact on the MeiraGTx Know-How, CMC Development Technology or Clinical IRD Patents, taking intoaccount the potential impact on the value of the Products worldwide as a result of such settlement. If Janssen recovers monetary damages in such claim,suit or action with respect to the MeiraGTx Know-How, CMC Development Technology or Clinical IRD Patents, after Janssen recoups its costs andexpenses associated with such litigation, with respect to any portion of such recovery remaining, [***].(ii) MeiraGTx’s Back-Up Right of Enforcement. If Janssen does not, within the earlier of [***] after learning about a Third PartyInfringement or Licensed Patent Action or [***] before the expiration date for filing an appropriate suit or responding to or taking any action (asapplicable), initiate and prosecute any legal action to enforce or defend the MeiraGTx Know-How, CMC Development Technology or Clinical IRDPatents, as applicable, then MeiraGTx shall have the right, but not the obligation, to commence such a suit or take such an action to enforce or defendthe applicable MeiraGTx Know-How, CMC Development Technology or Clinical IRD Patents, as applicable. In such event, Janssen shall takeappropriate actions in order to enable MeiraGTx to commence a suit or take the actions set forth in the preceding sentence. Prior to settling any suchsuit or action, MeiraGTx shall notify Janssen in writing as to the terms of such proposed settlement and shall not execute such settlement withoutJanssen’s written consent if Janssen identifies to MeiraGTx in reasonable detail a material risk of a material negative impact on the MeiraGTxKnow-How, CMC Development Technology or Clinical IRD Patents, taking into account the potential impact on the value of the Products worldwideas a result of such settlement. Prior to MeiraGTx commencing such a suit or action, MeiraGTx shall consider in good faith any reasonable Janssenbusiness concerns. If Janssen identifies to MeiraGTx in reasonable detail a material risk of a material negative impact on the MeiraGTx Know-How,CMC Development Technology or Clinical IRD Patents resulting directly from such a suit or action, taking into account the potential impact on thevalue of the Products worldwide, then MeiraGTx Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -74-Confidential Treatment Requested by MeiraGTx Holdings plc shall not commence any such suit or action. If MeiraGTx recovers monetary damages in such claim, suit or action with respect to the MeiraGTxKnow-How, CMC Development Technology or Clinical IRD Patents, any portion of such recovery remaining after MeiraGTx recoups its costs andexpenses associated with such litigation, shall be [***].(c) Enforcement Rights for MeiraGTx Research Know-How and Research IRD Patents.(i) Parties’ First Right of Enforcement. Prior to Janssen’s exercise of the applicable Option, MeiraGTx shall have the sole right to bring andcontrol an appropriate suit or other action against any Third Party Infringement or Licensed Patent Action with respect to the MeiraGTx ResearchKnow-How or Research IRD Patents; provided, however, that in the event that any such Third Party Infringement or Licensed Patent Action also affectsMeiraGTx Know-How, CMC Development Technology or Clinical IRD Patents, Janssen shall have the first right, but not the obligation, to bring andcontrol such suit or other action pursuant to Section 11.5(b)(i). On or after Janssen’s exercise of the applicable Option, Janssen shall have the first right,but not the obligation, in the case of the applicable MeiraGTx Research Know-How and Research IRD Patents to bring and control an appropriate suitor other action against any Third Party Infringement or Licensed Patent Action with respect to such MeiraGTx Research Know-How or Research IRDPatents. MeiraGTx shall provide reasonable assistance to Janssen in such enforcement or defense, at Janssen’s request and expense, including joiningsuch action as a party plaintiff to ensure legal standing if required by Applicable Laws to pursue such action or if requested by Janssen. Janssen shallconsult with MeiraGTx and keep MeiraGTx reasonably informed of the status of the enforcement or defense of such MeiraGTx Research Know-Howand Research IRD Patents. Janssen shall consider MeiraGTx’s comments with respect to the enforcement or defense of such MeiraGTx ResearchKnow-How or Research IRD Patents in good faith. Prior to settling any such suit or action, Janssen shall notify MeiraGTx in writing as to the terms ofsuch proposed settlement to the extent relating to MeiraGTx Research Know-How and Research IRD Patents and shall not execute such settlementwithout MeiraGTx’s written consent if MeiraGTx identifies to Janssen in reasonable detail a material risk of a material negative impact on theMeiraGTx Research Know-How or Research IRD Patents, taking into account the potential impact on the value of the Products worldwide as a result ofsuch settlement. If Janssen recovers monetary damages in such claim, suit or action with respect to the MeiraGTx Research Know-How or Research IRDPatents, after Janssen recoups its costs and expenses associated with such litigation, with respect to any portion of such recovery remaining, [***].(ii) MeiraGTx’s Back-Up Right of Enforcement. If Janssen does not, within [***] after learning about a Third Party Infringement orLicensed Patent Action or [***] before the expiration date for filing an appropriate suit or responding to or taking any action (as applicable), initiateand prosecute any legal action to enforce or defend the MeiraGTx Research Know-How or Research IRD Patents, as applicable, then MeiraGTx shallhave the right, but not the obligation, to commence such a suit or take such an action to enforce the applicable MeiraGTx Research Know-How orResearch IRD Patents. In such event, Janssen shall take appropriate actions in order to enable MeiraGTx to commence a suit or take the actions set forthin the preceding sentence. Prior to settling any such suit or action, MeiraGTx shall notify Janssen in Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -75-Confidential Treatment Requested by MeiraGTx Holdings plc writing as to the terms of such proposed settlement and shall not execute such settlement without Janssen’s written consent if Janssen identifies toMeiraGTx in reasonable detail a material risk of a material negative impact on the MeiraGTx Research Know-How or Research IRD Patents, taking intoaccount the potential impact on the value of the Products worldwide as a result of such settlement. Prior to MeiraGTx commencing such a suit oraction, MeiraGTx shall consider in good faith any reasonable Janssen business concerns. If Janssen identifies to MeiraGTx in reasonable detail amaterial risk or a material negative impact on the MeiraGTx Research Know-How or Research IRD Patents resulting directly from such a suit or action,taking into account the potential impact on the value of the Research IRD Products worldwide, then MeiraGTx shall not commence any such suit oraction. If MeiraGTx recovers monetary damages in such claim, suit or action with respect to the MeiraGTx Research Know-How or Research IRDPatents, any portion of such recovery remaining after MeiraGTx recoups its costs and expenses associated with such litigation shall be [***].(d) Cooperation. At the request of the Party bringing and controlling any Third Party Infringement or defending any Licensed Patent Action, asapplicable (“Controlling Party”), the other Party shall provide reasonable assistance in connection with such action, including by executingreasonably appropriate documents, providing access to such Party’s premises and employees, cooperating reasonably in discovery, and joining as aparty to the action if requested by the Controlling Party. The Controlling Party will keep the other Party reasonably informed of all materialdevelopments in connection with any such suit, and the other Party shall have the right to consult with the Controlling Party and to participate in and,if appropriate, be represented by independent but mutually agreed upon counsel in such litigation at such other Party’s own cost and expense. 11.6.Product Infringement.If a Party becomes aware of any actual or potential Claim alleging that the Research, Development, Manufacture, or Commercialization of any Productsunder this Agreement infringes, misappropriates, or otherwise violates any Intellectual Property Rights of a Third Party (or would if carried out)(“Product Infringement”), then such Party will notify the other Party [***] following the receipt of service of process in such action, suit, orproceeding, or the date on which such Party becomes aware that such action, suit, or proceeding has been instituted, and the Parties will meet as soon aspossible to discuss the overall strategy for defense of such matter. Janssen shall have the first right (but not the obligation) to defend any Claims ofProduct Infringement. If Janssen does not intend to defend such Product Infringement or determines to cease defending any such Product Infringementand informs MeiraGTx that it wishes for MeiraGTx to defend such Product Infringement, then MeiraGTx will have the right, but not the obligation,upon written notice to Janssen, to defend such Product Infringement or take over defense of the Product Infringement from Janssen, as applicable.Notwithstanding anything to the contrary, if either Party has an obligation to indemnify the other Party with respect to such Claim of ProductInfringement, then the provisions of Article 17 will apply with respect thereto. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -76-Confidential Treatment Requested by MeiraGTx Holdings plc 11.7.Patents Licensed From Third Parties.Each Party’s rights under this Article 11 with respect to the prosecution and maintenance, enforcement, and defense of any Patent that is licensed froma Third Party shall be subject to the rights retained by such Third Party with respect to such Patent. 11.8.Trademarks.Janssen shall have the right to brand any and all Product(s) using Janssen related Trademarks it determines appropriate for such Product(s), which mayvary by country or within a country (“Product Marks”). Subject to Section 15.4(c)(iii), Janssen shall own all rights in Product Marks and shall have thesole right to register and maintain Product Marks in the countries and regions it determines reasonably necessary, and Janssen shall have the sole right,in its discretion and at its expense, to defend and enforce such Product Marks. 12.CONFIDENTIALITY 12.1.Duty of Confidence.Except to the extent expressly authorized by this Agreement or otherwise agreed in writing by the Parties, the Parties agree that, during the Term andfor [***] after the expiration or earlier termination of this Agreement, subject to the other provisions of this Article 12, each Party will, as a receivingParty (the “Receiving Party”), and will cause its Affiliates to, maintain in confidence, not publish or otherwise disclose and otherwise safeguard, anyand all Confidential Information disclosed by or on behalf of the other Party (the “Disclosing Party”) or its Affiliates under this Agreement to it, usingsuch degree of care that such Party uses with respect to its own confidential information (which shall in no event be less than a reasonable degree ofcare). During the Term and for [***] after the expiration or earlier termination of this Agreement, subject to the other provisions of this Article 12, theReceiving Party may only use such Confidential Information for the purposes of this Agreement and in connection with the performance of itsobligations or exercise of rights granted or reserved in this Agreement. 12.2.Exceptions.The obligations under this Article 12 shall not apply to any information to the extent that such information:(a) is (at the time of disclosure) or becomes (after the time of disclosure) known to the public or part of the public domain through no breach ofthis Agreement by the Receiving Party or its Affiliates;(b) was known to, or was otherwise in the possession of, the Receiving Party or its Affiliates, as evidenced by written records of the ReceivingParty and its Affiliates kept in the ordinary course of business, prior to the time of disclosure by the Disclosing Party or any of its Affiliates; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -77-Confidential Treatment Requested by MeiraGTx Holdings plc (c) is disclosed to the Receiving Party or any of its Affiliates on a non-confidential basis by a Third Party who is entitled to disclose it withoutbreaching any confidentiality obligation to the Disclosing Party or any of its Affiliates; or(d) is independently developed by or on behalf of the Receiving Party or its Affiliates outside of its performance under this Agreement, asevidenced by written records of the Receiving Party and its Affiliates kept in the ordinary course of business, without the use of the ConfidentialInformation disclosed by the Disclosing Party or its Affiliates to the Receiving Party or its Affiliates under this Agreement.Specific aspects or details of Confidential Information shall not be deemed to be within the public domain or in the possession of the ReceivingParty merely because the Confidential Information is embraced by more general information in the public domain or in the possession of the ReceivingParty. Further, any combination of Confidential Information shall not be considered in the public domain or in the possession of the Receiving Partymerely because individual elements of such Confidential Information are in the public domain or in the possession of the Receiving Party, unless thecombination and its principles are in the public domain or in the possession of the Receiving Party. 12.3.Authorized Disclosures.(a) Disclosures. Except as expressly permitted by this Agreement, in addition to disclosures allowed under Section 12.2 or Section 12.4, theReceiving Party and its Affiliates may only disclose to Third Parties the Disclosing Party’s Confidential Information to the extent such disclosure isnecessary in the following instances: (i) in connection with the performance of its obligations or exercise of rights granted or reserved in thisAgreement, under non-disclosure and non-use provisions no less restrictive than those in this Agreement; (ii) in connection with the prosecution andmaintenance of Patents as permitted by this Agreement; (iii) in connection with Regulatory Filings or audits by Regulatory Authorities for anyProduct; (iv) in connection with prosecuting or defending litigation as permitted by this Agreement; (v) in complying with applicable court orders orgovernmental regulations (including securities regulations); (vi) in the case of any Party, in communication with its employees, directors, officers,agents, contractors, consultants, and professional advisers; Affiliates; potential or actual collaborators, partners, and licensees (including potentialco-marketing and co-promotion contractors); and potential or actual investment bankers, acquirers, lenders or investors, each of the foregoing whom,on a need-to-know basis and prior to disclosure, must be bound by similar obligations of confidentiality and non-use no less restrictive than thosecontained in this Article 12; (vii) as permitted in accordance with Article 13; or (viii) as mutually agreed to in writing by the Parties.(b) Disclosures pursuant to Applicable Law. If the Receiving Party is required to disclose Confidential Information of the Disclosing Partypursuant to Applicable Law (including the rules of the Securities and Exchange Commission or any stock exchange) or in connection with any bonafide legal process, including disclosures of the type contemplated by Section 12.3(a), such disclosure to the extent reasonably necessary shall not bedeemed a breach of this Agreement; provided, however, that the Receiving Party, except where reasonably impracticable or legally impermissible, will:(i) inform the Disclosing Party as soon as reasonably practicable following it becoming aware of the required disclosure; (ii) limit the disclosure to therequired purpose; and (iii) at the Disclosing Party’s request and reasonable expense, assist in attempting to object to, limit or seek to secureconfidential treatment of the required disclosure. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -78-Confidential Treatment Requested by MeiraGTx Holdings plc 12.4.Terms of this Agreement.The Parties acknowledge and agree that this Agreement and all of the respective terms of this Agreement shall be treated as Confidential Information ofeach Party, subject to Section 13.5. 12.5.Use of Residuals.Residuals shall not be considered Confidential Information of MeiraGTx and the restrictions set forth in this Article 12 shall not apply to the use ofResiduals. 12.6.Data Privacy.(a) Privacy and Data Security Laws. Without limiting a Party’s other obligations under this Agreement, each Party shall, and shall cause itsAffiliates and permitted subcontractors to, implement and maintain reasonable security procedures and practices appropriate to the nature of SensitiveInformation such Party is processing and take such other actions as are reasonably necessary to protect the security and confidentiality of suchSensitive Information against any threats or hazards to the security or integrity of such Sensitive Information in accordance with Privacy and DataSecurity Laws.(b) Data Breaches. If a Party learns of any security breach involving Sensitive Information of the other Party collected, prepared or developed inconnection with this Agreement, such Party shall immediately notify the owning Party of the same, and shall, at the owning Party’s expense,reasonably cooperate with the owning Party in investigating and responding to the foregoing. The Party whose Sensitive Information has beenbreached (or allegedly breached) shall have the sole right to determine the content, timing and other details of any notices to affected individuals orGovernmental Authorities in connection with such breach. 13.PUBLICATIONS AND PUBLICITY 13.1.Use of Names.Except as otherwise expressly permitted herein or to the extent required pursuant to Section 13.5, neither Party may use the names or Trademarks of theother Party or its Affiliates for any purpose, including in any press release, publication, or other form of public disclosure, without first obtaining, ineach case, the prior written consent of the other Party (such consent not to be unreasonably withheld, conditioned, or delayed), except for thosedisclosures for which consent has already been obtained or which are required by Applicable Law. Notwithstanding the foregoing, Janssen shall beentitled to use the name of MeiraGTx and its Affiliates to the extent necessary under Applicable Law in connection with the Development orCommercialization of any Product. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -79-Confidential Treatment Requested by MeiraGTx Holdings plc 13.2.Press Releases and Publicity Related to this Agreement.The Parties shall agree on a form of a press release that may be issued by each, if desired, on or shortly following the Execution Date. Thereafter, exceptto the extent required to comply with Applicable Law or otherwise pursuant to Section 13.5, neither Party shall issue any press releases or other publicstatements, whether oral or written, disclosing the existence of this Agreement, or the terms hereof, without first obtaining, in each case, the priorwritten consent of the other Party, such consent not to be unreasonably withheld, conditioned, or delayed. Notwithstanding the foregoing, to the extentinformation in connection with this Agreement has already been publicly disclosed in accordance with this Agreement, either Party (or its Affiliates)may subsequently disclose the same information to the public without the consent of the other Party. 13.3.Public Disclosures and Publications Related to the Programs or Products.Except to the extent required pursuant to Section 13.5, and subject to this Section 13.3, each Party shall not issue any press releases or other publicstatements, whether oral or written, regarding the Research Plan Activities, Research IRD Targets, Research IRD Products, CMC Development PlanActivities, Clinical Development Plan Activities, Clinical IRD Products and Clinical IRD Targets, without first obtaining, in each case, the priorwritten consent of the other Party, such consent not to be unreasonably withheld, conditioned, or delayed. MeiraGTx shall be permitted to providepublic statements with respect to certain Clinical IRD Products pursuant to obligations incurred prior to the Execution Date; provided that Janssen hasactual knowledge as of the Execution Date of such obligations; and provided further that such public statements shall be approved by Janssen prior totheir release (such approval not to be unreasonably withheld, conditioned, or delayed). In addition, and notwithstanding the foregoing, on or after therespective Sponsorship Transfer Date with respect to any Clinical IRD Product and on or after the respective Option Election Date with respect to anyJanssen Research IRD Product, Janssen may publish a press release or other public statement about such Clinical IRD Product or Janssen Research IRDProduct, as applicable; provided that Janssen shall provide MeiraGTx with a copy of any such press release or public statement reasonably in advanceof such publication and consider in good faith any comments timely provided by MeiraGTx to Janssen with respect to such press release or publicstatement, as applicable. 13.4.Publications.(a) Review Process. The Parties may wish to publish or present the results of the programs or Products hereunder. Either Party may publish theresults or observations of its work on a program or Product hereunder after providing the other Party [***] to review the proposed publication, abstractor presentation (“Review Period”) to determine whether the proposed publication, abstract or presentation contains any Confidential Information ofthe non-submitting Party. If within the Review Period, the non-submitting Party determines that the proposed publication, abstract or presentationcontains any such Confidential Information, the non-submitting Party will notify the submitting Party in writing of the presence of the ConfidentialInformation in the proposed publication, abstract or presentation and the submitting Party will delete such Confidential Information from the proposedpublication, abstract or presentation. If, within the Review Period, the non-submitting Party identifies material in such publication, abstract Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -80-Confidential Treatment Requested by MeiraGTx Holdings plc or presentation which, if published, would disclose an Invention of the non-submitting Party that has not yet been protected through the filing of aPatent or would adversely affect the Intellectual Property Rights owned by the non-submitting Party, then at the non-submitting Party’s election, (i) thesubmitting Party will delay submission of its publication, abstract or presentation for an additional period, not to exceed [***] or such other periodmutually agreed to by the Parties, in order to allow for the filing of a Patent or other appropriate Intellectual Property Rights protection for suchmaterial or (ii) the submitting Party will remove the identified material prior to publication.(b) Acknowledgments. Proper acknowledgement will be made for contributions, if any, of each Party to the results or other information andmaterial disclosed in any publication, abstract or presentation under this Section 13.4. Authorship of scientific publications will be determined inaccordance with customary academic and industry policies and procedures, the scientific contribution, and the standards of the applicable journal. 13.5.Disclosures Required By Law.Notwithstanding Section 13.1, Section 13.2, and Section 13.3, each Party may make any disclosures required to comply with any duty of disclosure itmay have pursuant to Applicable Law or the requirements of any Governmental Authority or Regulatory Authority or pursuant to the rules of anyrecognized stock exchange (including the rules of the Securities and Exchange Commission). If a disclosure required by Applicable Law, therequirements of any Governmental Authority or Regulatory Authority, or the rules of any recognized stock exchange arises, the Parties shall coordinatewith each other with respect to the timing, form, and content of such required disclosure. If so requested by the other Party, except where impracticableor not legally permitted, the Party subject to such obligation shall use commercially reasonable efforts to obtain an order protecting the confidentialityof such provisions of this Agreement, as determined by the Disclosing Party in consultation with its legal counsel. Without limiting the foregoing,MeiraGTx shall use commercially reasonable efforts to provide Janssen with each proposed filing by MeiraGTx with the United States Securities andExchange Commission (or any recognized stock exchange, including Nasdaq, or any similar regulatory agency in any country other than the UnitedStates) describing the terms of this Agreement (including any filings of this Agreement) at least [***] prior to submission of such filing, and shall seekconfidential treatment of portions of this Agreement or such terms or information as may be reasonably requested by Janssen in a timely manner. 13.6.Publication and Transparency of Clinical Research Results.All research results of clinical trials hereunder will be posted on clinicalstudyresults.org and on any other registry with requirements consistent with theregistration and publication guidelines of the International Committee of Medical Journal Editors, to the extent required. All data and informationposted on clinicaltrial.gov, clinicalstudyresults.org or any other registry will be subject to prior review by the other Party pursuant to Section 13.4(a).All research results generated from clinical trials hereunder may be made available to Third Parties pursuant to data transparency policies andmechanisms of Janssen or its Affiliates then in effect. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -81-Confidential Treatment Requested by MeiraGTx Holdings plc 14.EFFECTIVENESS 14.1.Effective Date.Except for the Parties’ obligations under Article 12, Article 13 and this Article 14, which shall be effective as of the Execution Date, this Agreementshall not become effective until the first date (the “Effective Date”) on which (a) expiration or early termination of all applicable waiting periods underthe HSR Act has occurred, (b) each of the representations of MeiraGTx set forth in Section 16 are true and correct in all material respects, (c) an officerof MeiraGTx has delivered a written certificate with respect to (b) to Janssen, (d) an updated copy of Exhibit 1.168, setting forth a true, complete andaccurate list of all Patents Controlled by MeiraGTx or any of its Affiliates as of the Effective Date, which claim or otherwise Cover any Clinical IRDProducts, Clinical IRD Targets or MeiraGTx Know-How in the Field, has been delivered to Janssen and (e) [***] has entered into a bindingcommitment with MeiraGTx to provide to Janssen the right to undertake a back-up license under [***]’s license grant to MeiraGTx; provided,however, that Janssen may waive the conditions described in clause (b), (c), (d) or (e) above. 14.2.Filings.The Parties shall cooperate with one another in the preparation and execution of all documents that are required to be filed pursuant to the HSR Act.Each Party shall, within [***] following the Execution Date (or such later time as may be agreed to in writing by the Parties), file with the United StatesFederal Trade Commission and the Antitrust Division of the United States Department of Justice, and/or with equivalent foreign authorities, any HSRFiling required of it in the reasonable opinion of either Party with respect to the transactions contemplated hereby. Neither Party shall seek expeditedtreatment of any HSR Filing without the other Party’s prior written consent. Each Party will use reasonable efforts to do, or cause to be done, all thingsnecessary, proper and advisable to, [***] take all actions necessary to make the filings required of such Party or its Affiliates under the HSR Act. TheParties shall cooperate with one another to the extent necessary in the preparation of any such HSR Filing. Each Party shall be responsible for its owncosts, expenses, and filing fees associated with any HSR Filing; provided, however, that Janssen shall be solely responsible for any fees (other thanpenalties that may be incurred as a result of actions or omissions on the part of MeiraGTx) required to be paid to any Governmental Authority inconnection with making any such HSR Filing. 14.3.Outside Date.This Agreement shall terminate (a) at the election of either Party, immediately upon written notice to the other Party, if the U.S. Federal TradeCommission or the U.S. Department of Justice, or an equivalent authority in the European Union, seeks a preliminary injunction under applicableantitrust and non-competition laws against Janssen and MeiraGTx to enjoin the transactions contemplated by this Agreement; or (b) at the election ofeither Party, immediately upon written notice to the other Party, in the event that the HSR Clearance Date shall not have occurred on or prior to onehundred eighty (180) days after the effective date of the HSR Filing. In the event of such termination, this Agreement shall be of no further force andeffect. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -82-Confidential Treatment Requested by MeiraGTx Holdings plc 15.TERM AND TERMINATION 15.1.Term.The term of this Agreement shall commence upon the Effective Date and, unless terminated pursuant to Section 15.2, shall continue in full force andeffect, on a Product-by-Product and country-by-country basis, until such time as the Royalty Term expires in such country (the “Term”). On aProduct-by-Product and country-by-country basis, effective upon the expiration of the Royalty Term for such Product in such country (but not for anytermination of this Agreement), the licenses granted to Janssen will each become fully paid-up, royalty-free, irrevocable, and perpetual in such countrywith respect to such Product. 15.2.Termination.This Agreement may be terminated as follows:(a) Termination for Breach.(i) General. Subject to Section 15.2(a)(ii), if either Janssen or MeiraGTx is in material breach of this Agreement, the non-breaching Partymust give written notice to the breaching Party within [***] following the alleged breach specifying the claimed particulars of such breach in sufficientdetail to put the allegedly breaching party on reasonable notice of the nature of the alleged breach, and in the event such material breach is not curedwithin [***] after such notice (or in the case of any undisputed payment obligations, [***] after such notice), the non-breaching Party shall have theright thereafter to terminate this Agreement immediately, in whole or with respect to the applicable Product (except with respect to a material breach byJanssen arising under Section 6.1(e), Section 6.2(a), Section 8.4(a), Article 9 and Section 19.4, which must be terminated on a Product-by-Productbasis), by giving written notice to the breaching Party to such effect; provided, however, that if such breach is capable of being cured but cannot becured within the period referenced above and the breaching Party initiates actions to cure such breach within such period and thereafter diligentlypursues such actions, the breaching Party shall have such additional period as is reasonable under the circumstances to cure such breach; it beingunderstood that no such extension shall apply with respect to any undisputed payment obligations. If arbitration is commenced with respect to anyalleged breach hereunder pursuant to Section 18.1, no purported termination of this Agreement pursuant to this Section 15.2(a)(i) shall take effectuntil: (A) a final resolution of such arbitration; and (B) the losing Party in such arbitration fails to satisfy the arbitration award within the cure timeframes referenced above.(ii) Janssen Special Remedy. If Janssen would have the right to terminate this Agreement under Section 15.2(a)(i), in whole or in part, for anuncured material breach by MeiraGTx in connection with a Product, then Janssen may, in its sole discretion, elect: (A) to either exercise suchtermination right or (B) in lieu of exercising such termination right, and without limiting Janssen’s rights otherwise set under this Agreement,subsequently reduce any payment amounts due to MeiraGTx under [***] for such Product by [***] percent ([***]%) as a result of this Section 15.2(a)(ii). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -83-Confidential Treatment Requested by MeiraGTx Holdings plc (b) Termination for Insolvency. This Agreement may be immediately terminated in its entirety by a Party by providing written notice oftermination to the other Party in the event of an Insolvency Event of the other Party.(c) Termination for Patent Challenge. To the extent permitted by Applicable Law, if Janssen or any of its Affiliates Challenges any Patentincluded in the MeiraGTx Patents, MeiraGTx Research Patents or Joint Patents in any country in the Territory (such Patent, a “Challenged Patent”),then MeiraGTx may, following written notice to Janssen and provided that Janssen or its Affiliate does not withdraw such Challenge within [***] afterreceipt of such notice, terminate this Agreement in its entirety by providing written notice of termination to Janssen; provided that this Section 15.2(c)will not apply to, and MeiraGTx may not terminate this Agreement with respect to (i) any claim or proceeding that would otherwise be a Challengehereunder to the extent commenced by a Third Party that after the Effective Date acquires or is acquired by Janssen or its Affiliates or its or theirbusiness or assets, whether by stock purchase, merger, asset purchase, or otherwise; provided that such proceeding commenced prior to the closing ofsuch acquisition; (ii) any claim or proceeding by a licensor of a product licensed by Janssen for which the licensor has an existing challenge, whetherin a court or administrative proceeding, against any MeiraGTx Patent, MeiraGTx Research Patent or Joint Patent; (iii) any Challenge required to becommenced pursuant to an order of a Governmental Authority or Applicable Law; (iv) any Challenge that is commenced by a Sublicensee; providedthat Janssen demands that such Sublicensee withdraw such Challenge [***] after Janssen becomes aware of such Challenge and terminates thesublicense agreement with the applicable Sublicensee if such Sublicensee does not withdraw such Challenge within [***] after receipt of such demandfrom Janssen; (v) any proceeding not initiated, directed or controlled by or on behalf of Janssen or one of its Affiliates, for which Janssen or theAffiliate, as the case may be, opposes, or assists any Third Party to oppose, the grant of a MeiraGTx Patent, MeiraGTx Research Patent or Joint Patentpursuant to any application in relation thereto in an administrative proceeding, such as a patent re-examination, inter partes review, or other post grantproceeding or opposition; (vi) challenges by an open forum entity or other industry group in which Janssen or its Affiliates or Sublicensees do notdirect or control the action of such entity; (vii) general activities not specifically directed to a particular Patent, such as amicus briefs on cases notinvolving any MeiraGTx Patent, MeiraGTx Research Patent or Joint Patent; (viii) lobbying or other efforts directed to patent issues generally and notto any specific Patent; (ix) any affirmative defense or other validity, enforceability, or non-infringement challenge, whether in the same action or in anyother agency or forum of competent jurisdiction, advanced by Janssen, any of its Affiliates or Sublicensees in response to any claim or action broughtin the first instance by, or on behalf of, MeiraGTx or any Third Party; (x) providing documents or testimony in response to any discovery requests orcourt order in a valid legal process not directed to Challenging any Challenged Patent; or (xi) any Challenge related to any Clinical IRD Patent, CMCDevelopment Patent, Research IRD Patent or Janssen Research IRD Patent for which Janssen’s reasonable input with respect to such Patent’sprosecution, maintenance or enforcement pursuant to Section 11.4 or Section 11.5 was not included in any filings or correspondence with patentauthorities or otherwise accepted or pursued by MeiraGTx (as applicable).(d) Termination by Janssen At Will. Beginning on [***], Janssen may terminate this Agreement at will in its entirety or on a Product-by-Productbasis at any time on: (i) [***] prior written notice to MeiraGTx, if prior to the First Commercial Sale of such Product; and (ii) on [***] prior writtennotice to MeiraGTx, if following the First Commercial Sale of such Product. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -84-Confidential Treatment Requested by MeiraGTx Holdings plc 15.3.Rights in Insolvency.The Parties agree that this Agreement constitutes an executory contract under Section 365 of the Code for the license of “intellectual property” asdefined under Section 101 of the Code and constitutes a license of “intellectual property” for purposes of any similar laws in any other country in theTerritory. The Parties further agree that Janssen, as licensee of such rights under this Agreement, will retain and may fully exercise all of its protections,rights and elections under the Code, including under Section 365(n) of the Code, and any similar laws in any other country in the Territory and thatJanssen cannot be compelled to accept a money satisfaction of its interests in the intellectual property licensed pursuant to this Agreement, and thatany such sale therefore may not be made to a purchaser “free and clear” of Janssen’s rights under this Agreement and Section 365(n) without theexpress, contemporaneous consent of Janssen. The Parties further agree that, in the event of an Insolvency Event by or against MeiraGTx under theCode and any similar laws in any other country in the Territory, Janssen may be entitled to a complete duplicate of (or complete access to, asappropriate) any such intellectual property and all embodiments of such intellectual property (including Materials and Research Results), and thesame, if not already in its possession, will be [***] delivered to it: (a) upon any such commencement of an Insolvency Event upon its written requesttherefor, unless MeiraGTx elects to continue to perform all of its obligations under this Agreement; or (b) if not delivered under clause (a) above,following the rejection of this Agreement by or on behalf of MeiraGTx upon written request therefor by Janssen. Whenever MeiraGTx or any of itssuccessors or assigns provides to Janssen any of the intellectual property licensed hereunder (or any embodiment thereof including Materials andResearch Results) pursuant to this Section 15.3, Janssen shall have the right to perform MeiraGTx’s obligations hereunder with respect to suchintellectual property, but neither such provision nor such performance by Janssen shall release MeiraGTx from liability resulting from rejection of thelicense or the failure to perform such obligations. MeiraGTx and Janssen each acknowledges and agrees that Option Fees, Development MilestonePayments, Commercial Milestone Payments and Royalties constitute royalties within the meaning of Section 365(n) of the Bankruptcy Code.All rights, powers and remedies of Janssen provided herein are in addition to and not in substitution for any and all other rights, powers and remediesnow or hereafter existing at law or in equity (including the Bankruptcy Code) in the event of the commencement of a case under the Bankruptcy Codewith respect to MeiraGTx. The Parties agree that they intend the following rights to extend to the maximum extent permitted by law, and to beenforceable under Bankruptcy Code Section 365(n):(i) the right of access to any intellectual property rights (including all embodiments thereof) of MeiraGTx licensed to Janssen hereunder, orany Third Party with whom MeiraGTx contracts to perform an obligation of MeiraGTx under this Agreement, and, in the case of the Third Party, whichis necessary for the Manufacture, use, sale, import or export of Products; and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -85-Confidential Treatment Requested by MeiraGTx Holdings plc (ii) the right to contract directly with any Third Party to complete the contracted work. 15.4.Effects of Expiration or Termination.(a) Licenses. Upon any expiration or termination of this Agreement after the Effective Date, in whole or in part, any licenses granted by a Partyunder Section 4.1 with respect to a Terminated Product will terminate, except to the extent necessary for Janssen to perform any of its obligations thatsurvive such termination, to conduct wind-down activities in compliance with applicable legal and ethical obligations, or to the extent set forth inSection 4.1(c) or Section 15.1, and each of the Parties’ respective obligations with respect to such Terminated Product shall terminate, except as setforth in Section 15.1, Section 15.4, Section 15.5 and Section 15.6. For clarity, upon any termination in part of this Agreement, the licenses grantedunder Article 4 shall survive with respect to the Products not affected by such partial termination.(b) Confidential Information. Upon any expiration or termination of this Agreement, in whole or in part, each Party will destroy all written,electronic, or other materials containing Confidential Information of the other Party, including all copies thereof relating to such Terminated Productwithin [***] of such termination and provide certification of such destruction to the owning Party; provided, that: (i) a Party may retain one (1) copy ofany materials containing Confidential Information of the other Party in its archives solely for the purpose of monitoring its ongoing confidentialityobligations hereunder; and (ii) a Party will not be obligated to destroy such materials containing Confidential Information of the other Party that arenecessary for the Party to exercise any other license or right of such Party that survives such termination of this Agreement; provided, however, that thereceiving Party’s use of such Confidential Information will continue to be subject to the requirements and restrictions set forth in Article 12.(c) Wind-Down Activities. Upon any termination of this Agreement, in whole or in part, by MeiraGTx for Janssen’s uncured material breachunder Section 15.2(a), by MeiraGTx under Section 15.2(b) or Section 15.2(c), or by Janssen under Section 15.2(d), then the following shall occur:(i) Janssen hereby grants to MeiraGTx, with respect to the Terminated Product(s), a non-exclusive, perpetual and irrevocable right andlicense, with the right to grant sublicenses (through multiple tiers), under Janssen’s Sole Inventions as necessary to Develop, Manufacture andCommercialize such Terminated Product(s) in the Field in the Territory;(ii) Effective as of such termination date, Janssen hereby assigns, and shall [***] transfer (at MeiraGTx’s reasonable expense, unlessMeiraGTx terminated this Agreement, in whole or in part, for Janssen’s uncured material breach under Section 15.2(a), in which case such transfer shallbe at Janssen’s reasonable expense) on an as-is, where-is basis, to MeiraGTx or MeiraGTx’s designee possession and ownership of all governmental orregulatory correspondence, conversation logs, filings and approvals (including all Regulatory Approvals and Pricing Approvals), and global safetydatabase, in each case relating to the Development, Manufacture or Commercialization of the Terminated Product(s) and to the extent permitted underApplicable Law, and Janssen shall reasonably cooperate, at no additional out-of-pocket cost to Janssen, with requests by MeiraGTx for assistancenecessary to facilitate MeiraGTx’s assumption of regulatory responsibilities for the Terminated Product(s) in the applicable countries in which directtransfer is not permitted during the [***] following such termination date; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -86-Confidential Treatment Requested by MeiraGTx Holdings plc (iii) Effective as of such termination date, Janssen hereby assigns (and shall ensure that its Affiliates or Sublicensees hereby assign) toMeiraGTx all Trademarks used solely with respect to the Terminated Product(s) in the Territory (together with the goodwill associated with theforegoing); and(iv) Effective as of such termination date, Janssen shall [***] provide MeiraGTx with a summary of all Third Party agreements relating tothe Development, Manufacture or Commercialization of the Terminated Product(s) to which Janssen is a party (other than those to be assigned toMeiraGTx) and shall assign to MeiraGTx any Third Party agreements solely relating to the Development, Manufacture or Commercialization of theTerminated Product(s), and MeiraGTx shall accept such assignment, including responsibility for all liabilities and obligations accruing under suchagreements from and after the date of termination, in each case to the extent permitted thereunder. With respect to each agreement relating to theDevelopment, Manufacture or Commercialization of Terminated Product(s) that is not transferred to MeiraGTx, at MeiraGTx’s request, Janssen shalluse reasonable efforts to facilitate a direct introduction between MeiraGTx and the Third Party counterparty to such agreement.(d) Clinical Studies. If Janssen is conducting any Clinical Study on the date of notice of termination for any reason under this Agreement, thenMeiraGTx shall notify Janssen within [***] after the notice of termination with regard to any Clinical Study, whether MeiraGTx elects to have Janssen(i) complete such Clinical Study on behalf of MeiraGTx (unless Janssen reasonably believes there is a material safety issue that should prevent thecontinuation of such Clinical Study), (ii) wind down such Clinical Study as soon as practicable, subject to compliance with ethical and legalrequirements, or (iii) transfer such Clinical Study to MeiraGTx as soon as practicable. Notwithstanding the foregoing, if Janssen terminates thisAgreement pursuant to Section 15.2(a) or 15.2(b), then this Section 15.4(d) shall not apply and Janssen, at its sole discretion, shall determine whetherto continue or wind down any ongoing Clinical Study, subject to compliance with ethical and legal requirements; and each Party shall bear its ownexpenses incurred pursuant to such wind down. In addition, and subject to the foregoing:(i) If MeiraGTx notifies Janssen of its election to have Janssen complete a Clinical Study on behalf of MeiraGTx, Janssen and MeiraGTxwill, as necessary, negotiate in good faith a separate agreement pursuant to which Janssen would complete such Clinical Study. If the Parties fail toreach agreement within [***] after MeiraGTx makes such election, Janssen may wind down such Clinical Study, subject to compliance with ethicaland legal requirements or, if requested by MeiraGTx, transfer such Clinical Study to MeiraGTx.(ii) If MeiraGTx notifies Janssen of its election to have Janssen wind down such Clinical Study (or fails to provide notice within such [***]period), then Janssen shall wind-down such Clinical Study as soon as practicable, subject to compliance with ethical and legal requirements. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -87-Confidential Treatment Requested by MeiraGTx Holdings plc (iii) If MeiraGTx notifies Janssen of its election to have Janssen transfer such Clinical Study to MeiraGTx, then Janssen shall usecommercially reasonable efforts to transfer, and MeiraGTx shall use commercially reasonable efforts to assume, such Clinical Study as [***] aspracticable (and, in any event, within [***]) after the effective date of termination.(iv) The costs of ongoing Clinical Studies under this Section 15.4(d) shall be borne as follows:(A) By [***] after the effective date of termination, if [***]. [***] shall reimburse [***] for any such costs incurred by [***] after theeffective date of termination.(B) By [***] after the effective date of termination, if [***]. [***] shall reimburse [***] after the effective date of termination.(C) By [***] as follows after the effective date of termination, if [***]: (x) by [***] until the earlier of (i) completion of such wind-down or(ii) [***] after the effective date of termination for such Clinical Study for such Clinical IRD Product; and (y) by [***] until the completion of suchwind-down; provided that both Parties shall cooperate to wind-down such Clinical Study for such Clinical IRD Product as soon as reasonablypracticable, including through discussions with the applicable Regulatory Authorities to minimize the compliance and legal requirement period forsuch wind-down.(D) By [***] after the effective date of termination, if [***] until the completion of such wind-down.(e) Further Actions. Janssen shall take all such further actions within the [***] following such termination of this Agreement, in whole or in part,as may be reasonably requested by MeiraGTx in order to give effect to the foregoing clauses in this Section 15.4, [***].(f) Rights Terminated. Except as set forth in Section 15.1, Section 15.4, Section 15.5 and Section 15.6, the rights and obligations of the Partieshereunder shall terminate as of the date of such termination of this Agreement, in whole or in part, with respect to the Terminated Product(s). For clarity,termination of this Agreement, in whole or part, will not affect a Party’s rights to freely exploit CMC Development Inventions or CMC DevelopmentPatents in accordance with Article 11. In addition, upon any expiration or termination of this Agreement after the Effective Date, in whole or in part,each Party shall not undertake any further actions under this Agreement following such expiration or termination except in accordance with thisSection 15.4. Subject to Section 15.2(a)(ii), Section 15.4(d) and Section 15.5, in the event of any expiration or termination of this Agreement, in wholeor in part, (i) Janssen will pay all amounts then due and owing to MeiraGTx in accordance with Article 10 as of the effective date of such expiration ortermination; (ii) each Party will use reasonable efforts to mitigate and avoid any costs incurred by or on behalf of such Party on or after such date ofexpiration and termination, to the extent to be reimbursed by the other Party in accordance with the express terms herewith (including underSection 15.4(d)(iv)(C)); and (iii) no further payment amounts shall be due to MeiraGTx with respect to the Terminated Product(s) after the effectivedate of such expiration or termination. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -88-Confidential Treatment Requested by MeiraGTx Holdings plc 15.5.Post-Termination Royalties to Janssen.Following termination of this Agreement, in whole or in part, with respect to a Product, MeiraGTx shall be obligated to pay Janssen a royalty on NetSales of such Product in the amount of: (a) [***] percent ([***]%) of Net Sales if a Phase 2 Study has not been initiated for such Product as of theeffective date of termination; (b) [***] percent ([***]%) of Net Sales if a Phase 2 Study has been initiated as of the effective date of termination but aPivotal Study has not been initiated for such Product as of the effective date of termination; (c) [***] percent ([***]%) of Net Sales if a Pivotal Studyhas been initiated for such Product as of the effective date of termination but Pricing Approval in at least one (1) country has not been obtained forsuch Product as of the effective date of termination; or (d) [***] percent ([***]%) of Net Sales if Pricing Approval in at least one (1) country has beenobtained for such Product as of the effective date of termination. The royalty reporting, payment, record-keeping, audit and withholding tax provisionsset forth in Sections 10.8, 10.9, 10.11, 10.13(d) and 10.14 and “Royalty Term” definition set forth in Section 10.9(a) shall apply mutatis mutandis toroyalties payable pursuant to this Section 15.5. For purposes of this Section 15.5, “Net Sales” shall have the meaning given to it in Section 1.176, as ifthe references to Janssen in such definition were to MeiraGTx. 15.6.Survival.Expiration or termination of this Agreement shall not relieve the Parties of any obligation accruing prior to such expiration or termination. Subject tothe other terms and conditions regarding the termination and survival of obligations under this Agreement in the event of expiration or termination ofthis Agreement, upon expiration or termination of this Agreement, all provisions of this Agreement will cease to have any effect, except that thefollowing provisions will survive any such expiration or termination for any reason for the period of time specified therein, or if not specified, thenthey will survive indefinitely: Article 1 (Definitions), Section 3.5 (Research Records), Section, 3.7(c) (Final Report), Section 4.1(c) (Internal ResearchLicense), Section 4.7 (No Other Rights), Section 6.1(g) (Clinical Development Records), Section 10.1 (Upfront Payment) (only with respect to the lastsentence), Section 10.11 (Reports and Royalty Payments), Section 10.12 (Disclaimer), Section 10.13 (Payment Terms), Section 10.14 (Records andAudits), Section 11.1 (Ownership under the Clinical IRD Programs and Research IRD Programs), Section 11.2 (Ownership under the CMCDevelopment Collaboration), Section 11.3 (Duties to the Other Party), Section 11.8 (Trademarks) (only with respect to the second sentence), Article 12(Confidentiality), Section 13.1 (Use of Names), Section 13.2 (Press Releases and Publicity Related to this Agreement), Section 13.4 (Publications)(provided, however, that Section 13.4 shall not survive in the event of any expiration or termination of this Agreement in its entirety), Section 13.5(Publications Required by Law), Section 14.3 (Outside Date), Section 15.1 (Term), Section 15.3 (Rights in Insolvency), Section 15.4 (Effects ofExpiration or Termination), Section 15.5 (Post-Termination Royalties to Janssen), Section 15.6 (Survival), Section 15.7 (Termination Not SoleRemedy), Article 17 (Indemnification; Liability; Insurance), Article 18 (Dispute Resolution) and Article 19 (General Provisions). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -89-Confidential Treatment Requested by MeiraGTx Holdings plc 15.7.Termination Not Sole Remedy.Termination is not the sole remedy under this Agreement and, whether or not termination is effected and notwithstanding anything contained in thisAgreement to the contrary, all other remedies will remain available except as agreed to otherwise herein, and except that termination shall beMeiraGTx’s sole remedy with respect to any failure by Janssen to discharge its obligations under Section 6.1(e), Section 6.2(a), Section 8.4(a) andArticle 9. 16.REPRESENTATIONS, WARRANTIES AND COVENANTS 16.1.Representations and Warranties by Each Party.Each Party hereby represents and warrants to the other Party, that as of the Execution Date and the Effective Date:(a) such Party is a company duly organized, validly existing, and in good standing under the laws of its jurisdiction of formation orincorporation;(b) such Party has full power and authority to execute, deliver, and perform this Agreement, and has taken all action required by Applicable Lawand its organizational documents to authorize the execution and delivery of this Agreement by such Party and the performance of all obligations ofsuch Party as contemplated by this Agreement;(c) this Agreement constitutes a legal, valid, and binding agreement enforceable against such Party in accordance with its terms;(d) the person or persons executing this Agreement on such Party’s behalf have been duly authorized to do so by all requisite corporate action;(e) all consents, approvals and authorizations from all Governmental Authorities or other Third Parties required to be obtained by such Party inconnection with entering into this Agreement have been obtained, except in the case of the Execution Date as required pursuant to the HSR Act; and(f) the execution and delivery of this Agreement and all other instruments and documents required to be executed pursuant to this Agreement, theperformance of such Party’s obligations hereunder and the licenses, options, rights of negotiation and other rights to be granted by such Party pursuantto this Agreement, and the consummation of the transactions contemplated hereby do not and shall not: (i) conflict with or result in a breach of anyprovision of its organizational documents; (ii) conflict with, violate, result in a breach or constitute a default under any contractual obligations of suchParty or any of its Affiliates existing as of the Execution Date or Effective Date, including with respect to applicable Third Party Licenses; or(iii) conflict with or violate any requirements of Applicable Laws existing as of the Execution Date or Effective Date and applicable to such Party. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -90-Confidential Treatment Requested by MeiraGTx Holdings plc 16.2.Representations, Warranties and Covenants by MeiraGTx.In addition to the representations and warranties made by MeiraGTx in Section 16.1 above and Section 16.3 below, MeiraGTx hereby represents,warrants and covenants to Janssen that, (1) as of the Execution Date, (2) except as otherwise expressly provided, the Effective Date, and (3) solely withrespect to Sections 16.2(a), (b), (d), (f), (g), (i), (k), (l), and (m), and solely with respect to the [***] Product as a Research IRD Product, as of the OptionElection Date:(a) MeiraGTx has, and will have during the Term of this Agreement, the full right, power and authority to grant the licenses, exclusivity, options,right of first negotiation and other rights granted to Janssen hereunder, including under (i) the MeiraGTx Patents and MeiraGTx Know-How; and (ii) toMeiraGTx’s knowledge, the MeiraGTx Research Patents and MeiraGTx Research Know-How that each Cover Research IRD Products;(b) MeiraGTx has not entered, and during the Term, will not enter, into any written agreement that grants or would grant to any Affiliate or ThirdParty, including any academic organization or agency or other Person, any rights to (i) any Clinical IRD Target or Clinical IRD Product; or (ii) toMeiraGTx’s knowledge, any Research IRD Product or related Research IRD Target, or that otherwise conflicts or would conflict with the rights grantedto Janssen hereunder or MeiraGTx’s ability to fully perform its obligations hereunder, apart from those rights set forth in Exhibit 16.2(b);(c) Exhibit 1.168 sets forth a true, complete and accurate list of all Patents Controlled by MeiraGTx or any of its Affiliates as of the ExecutionDate, which claim or otherwise Cover any Clinical IRD Products, Clinical IRD Targets or MeiraGTx Know-How in the Field, and each such updatedcopy of Exhibit 1.168 provided by MeiraGTx to Janssen as of the Effective Date and to the JSC on each anniversary of the Effective Date thereafter setsforth a true, complete and accurate list of all Patents Controlled by MeiraGTx or any of its Affiliates as of such respective dates, which claim orotherwise Cover any Clinical IRD Products, Clinical IRD Targets or MeiraGTx Know-How in the Field.(d) the MeiraGTx Technology comprises all of the Intellectual Property Rights used by MeiraGTx and its Affiliates and consultants in theResearch, Development and Manufacturing of the Clinical IRD Products prior to the Execution Date, and no MeiraGTx [***] Technology is used byMeiraGTx or its Affiliates or consultants, or is otherwise necessary, for the Research, Development, Manufacture or Commercialization of Clinical IRDProducts;(e) to MeiraGTx’s knowledge, (i) the MeiraGTx Research Technology comprises all of the Intellectual Property Rights used by MeiraGTx and itsAffiliates and consultants in the Research, Development and Manufacturing of Research IRD Products prior to the Execution Date, and (ii) noMeiraGTx [***] Technology is used by MeiraGTx or its Affiliates or consultants, or is otherwise necessary, for the Research, Development,Manufacture or Commercialization of Research IRD Products;(f) neither MeiraGTx nor its Affiliates is or was engaged in the Commercialization of any Clinical IRD Product or Research IRD Product on orprior to the Execution Date; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -91-Confidential Treatment Requested by MeiraGTx Holdings plc (g) Exhibit 16.2(g) sets forth a complete and accurate list of all Existing Third Party Obligations as of the Execution Date, and besides ExistingThird Party Obligations as set forth in Exhibit 16.2(g), neither MeiraGTx nor any of its Affiliates is subject to any royalty, milestone or other paymentobligation to any Third Party in connection with the practice, or the grant of rights to Janssen to practice, (i) any of the MeiraGTx Technology,including with respect to the Commercialization of Clinical IRD Products under this Agreement and (ii) to MeiraGTx’s knowledge, any of theMeiraGTx Research Technology, including with respect to the Commercialization of Research IRD Products;(h) MeiraGTx has provided Janssen with complete, true and accurate copies of all agreements in effect between MeiraGTx and [***] relating tothis Agreement;(i) MeiraGTx has not materially breached, or received any notice of breach or default under, any Existing Third Party Obligation, and eachExisting Third Party Obligation is valid, binding and in full force and effect;(j) Exhibit 4.4 sets forth a complete and accurate list of all subcontractors that MeiraGTx has existing relationships with as of the Execution Date,such subcontractors’ activities or obligations in connection with this Agreement and if applicable, any MeiraGTx commitments specifically requiringeither of the Parties to engage any such subcontractors in connection with any Clinical Development Plan Activities or Research Plan Activities on orafter the Effective Date;(k) to MeiraGTx’s knowledge, the Research, Development, Manufacture and Commercialization of the Products, do not, or will not, infringe ormisappropriate the Intellectual Property Rights of any Third Party, and MeiraGTx or any of its Affiliates or licensees or Sublicensees of any MeiraGTxTechnology or MeiraGTx Research Technology Covering Research IRD Products have not received any written notice alleging such infringement ormisappropriation, except that [***];(l) there are no claims, judgments, orders, decrees, or settlements against or owed by MeiraGTx or any of its Affiliates, and there is no writtenaction or proceeding of any nature, civil, criminal, regulatory or otherwise, pending or, to the knowledge of MeiraGTx, threatened against MeiraGTx orany of its Affiliates, in each case that would prevent MeiraGTx from performing its obligations under this Agreement or from granting the licenses andother rights set forth hereunder;(m) none of MeiraGTx, its Affiliates, or licensees or Sublicensees of any MeiraGTx Technology, have initiated or been involved in anyproceeding or other Claims in which it alleges that any Third Party is or was infringing or misappropriating any (i) MeiraGTx Technology or (ii) toMeiraGTx’s knowledge, MeiraGTx Research Technology Covering Research IRD Products, nor have any such proceedings been threatened byMeiraGTx, its Affiliates, or licensees or Sublicensees, nor does MeiraGTx or its Affiliates know of any valid basis for any such proceedings; and Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -92-Confidential Treatment Requested by MeiraGTx Holdings plc (n) MeiraGTx has provided Janssen with true, accurate and complete copies of all agreements listed in Exhibit 16.2(b) and Exhibit 16.2(g)attached hereto (including amendments and addendums to such agreements). 16.3.Mutual Covenants.(a) Compliance. Each Party shall comply, and shall cause its Affiliates, subcontractors and Sublicensees to comply, in all material respects withall Applicable Laws applicable to the Development, Manufacture and Commercialization of Products and performance of its obligations under thisAgreement, including, to the extent applicable, the statutes, regulations and written directives of the FDA (including cGCP, cGLP, and cGMP), theEMA and any Regulatory Authority having jurisdiction in the Territory, the FD&C Act, the Prescription Drug Marketing Act, the Federal Health CarePrograms Anti-Kickback Law, 42 U.S.C. § 1320a-7b(b), the statutes, regulations and written directives of Medicare, Medicaid and all other health careprograms, as defined in 42 U.S.C. § 1320a-7b(f), Anti-Corruption Laws, Privacy and Data Security Laws, Trade Control Laws and U.S. Export ControlLaws.(b) No Debarred Person. In the course of the Research, Development, Manufacture and Commercialization of the Products, neither Party nor itsAffiliates, Sublicensees or subcontractors shall use any employee, officer, director, agent or consultant who is or has been a Debarred Person, or, to suchParty’s or its Affiliate’s knowledge, is the subject of debarment proceedings by a Regulatory Authority. Each Party shall notify the other Party [***] inwriting upon becoming aware that any person performing services under this Agreement has become a Debarred Person or is the subject of debarmentproceedings or pending or threatened debarment proceedings by any Regulatory Authority.(c) Know-How and IP Protection. All of a Party’s employees and other Persons acting on such Party’s behalf, in each case with access toKnow-How and Inventions as contemplated herein, are obligated to maintain the confidentiality of such Know-How and Inventions and not use suchKnow-How or Inventions for any purpose inconsistent with the terms and conditions of this Agreement. All of a Party’s employees and other Personsperforming activities hereunder on behalf of such Party in accordance herewith will be obligated to assign all rights, title and interests in and to anyInventions developed by them in connection with such activities, whether or not patentable, to such Party. 16.4.No Other Warranties.EXCEPT AS EXPRESSLY STATED HEREIN, (A) NO REPRESENTATION, CONDITION OR WARRANTY WHATSOEVER IS MADE OR GIVEN BYOR ON BEHALF OF JANSSEN OR MEIRAGTX; AND (B) ALL OTHER CONDITIONS AND WARRANTIES WHETHER ARISING BY OPERATIONOF LAW OR OTHERWISE ARE HEREBY EXPRESSLY EXCLUDED, INCLUDING ANY CONDITIONS AND WARRANTIES OFMERCHANTABILITY, FITNESS FOR A PARTICULAR PURPOSE OR NON-INFRINGEMENT. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -93-Confidential Treatment Requested by MeiraGTx Holdings plc 17.INDEMNIFICATION; LIABILITY; INSURANCE 17.1.Indemnification by MeiraGTx.MeiraGTx shall indemnify, defend and hold Janssen, its Affiliates and Sublicensees, and their respective officers, directors, employees and agents(“Janssen Indemnitees”) harmless from and against any Damages arising out of or resulting from any Claims of Third Parties against them to the extentarising or resulting from:(a) the negligence, gross negligence or willful misconduct of any MeiraGTx Indemnitee or contractor in connection with this Agreement;(b) the breach of any of the covenants, agreements, warranties or representations made by MeiraGTx to Janssen under this Agreement;(c) MeiraGTx’s Research activities with respect to any Research IRD Products prior to the respective Option Election Date, including pursuant toSection 2.2(a) and Section 3.3;(d) MeiraGTx’s Research or Development activities with respect to the [***] Product unless and until Janssen elects to make the [***] Product aClinical IRD Product pursuant to Section 2.1(b);(e) MeiraGTx’s Research or Development activities with respect to any Clinical IRD Product, including its Clinical Development Plan Activitiespursuant to Section 6.1(c) and Section 6.1(e), and, prior to the Sponsorship Transfer Date, regulatory activities pursuant to Section 7.1(a); or(f) MeiraGTx’s Manufacturing activities with respect to any Product, including Manufacturing Products pursuant to Section 8.1, any SupplyAgreement or any Quality Agreement, and conducting CMC Development Plan Activities pursuant to Section 8.4(a);except that MeiraGTx shall not be obliged to so indemnify the Janssen Indemnitees for any Claim to the extent that Janssen has an obligation toindemnify MeiraGTx Indemnitees pursuant to Section 17.2 for such Claim. 17.2.Indemnification by Janssen.Janssen shall indemnify, defend and hold MeiraGTx, its Affiliates and Sublicensees, and their respective officers, directors, employees and agents(“MeiraGTx Indemnitees”) harmless from and against any Damages arising out of or resulting from any Claims of Third Parties against them to theextent arising or resulting from:(a) the negligence, gross negligence or willful misconduct of any Janssen Indemnitee or contractor in connection with this Agreement;(b) the breach of any of the covenants, agreements, warranties or representations made by Janssen to MeiraGTx under this Agreement; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -94-Confidential Treatment Requested by MeiraGTx Holdings plc (c) Janssen’s Research, Development or Commercialization activities with respect to any Janssen Research IRD Product pursuant toSection 2.2(a), Section 6.2(a), Section 7.2(a) or Article 9;(d) Janssen’s Development or Commercialization activities with respect to any Clinical IRD Product, including conducting its ClinicalDevelopment Plan Activities pursuant to Section 6.1(c) and Section 6.1(e), regulatory activities after [***] pursuant to Section 7.1(a) andCommercialization activities pursuant to Article 9; or(e) Janssen’s Manufacturing activities with respect to any Product, including Manufacturing Products pursuant to the applicable SupplyAgreements and conducting its CMC Development Plan Activities pursuant to Section 8.4(a);except that Janssen shall not be obliged to so indemnify the MeiraGTx Indemnitees for any Claim to the extent that MeiraGTx has an obligation toindemnify Janssen Indemnitees pursuant to Section 17.1 for such Claim. 17.3.Indemnification Procedure.(a) For the avoidance of doubt, all indemnification claims in respect of a Janssen Indemnitee or MeiraGTx Indemnitee shall be made solely byJanssen or MeiraGTx, respectively.(b) A Party seeking indemnification hereunder (the “Indemnified Party”) shall notify the other Party (the “Indemnifying Party”) in writingreasonably [***] after the assertion against the Indemnified Party of any Claim or fact in respect of which the Indemnified Party intends to base a claimfor indemnification hereunder (an “Indemnification Claim Notice”); provided, that the failure or delay to so notify the Indemnifying Party shall notrelieve the Indemnifying Party of any obligation or liability that it may have to the Indemnified Party, except to the extent that the Indemnifying Partydemonstrates that its ability to defend or resolve such Claim is adversely affected thereby. The Indemnification Claim Notice shall contain adescription of the Claim and the nature and amount of the Claim (to the extent that the nature and amount of such Claim is known at such time). Uponthe request of the Indemnifying Party, the Indemnified Party shall furnish [***] to the Indemnifying Party copies of all correspondence,communications, and official documents (including court documents) received or sent in respect of such Claim.(c) Subject to Section 17.3(d) and Section 17.3(e), the Indemnifying Party shall have the right, upon written notice given to the IndemnifiedParty, to assume the defense and handling of such Claim, at the Indemnifying Party’s sole expense, in which case Section 17.3(d) shall govern. Theassumption of the defense of a Claim by the Indemnifying Party shall be construed as acknowledgement that the Indemnifying Party is liable toindemnify any Indemnitee with respect to the Claim, and shall constitute a waiver by the Indemnifying Party of any defenses it may assert against anyIndemnified Party’s claim for indemnification. Until the Indemnifying Party gives such written notice to the Indemnified Party, Section 17.3(e) shallgovern. An Indemnifying Party will not have the right to conduct the defense of any Claim (a) brought by a Governmental Authority, (b) where suchClaim involves injunctive relief (which cannot reasonably be severed from the proceeding for damages) or (c) where the Indemnified Party reasonablybelieves that the Indemnifying Party does not have the financial resources to discharge its indemnification obligation in full, unless the IndemnifyingParty posts a bond to cover such potential indemnification obligations. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -95-Confidential Treatment Requested by MeiraGTx Holdings plc (d) Upon assumption of the defense of a Claim by the Indemnifying Party: (i) the Indemnifying Party shall have the right to and shall assume solecontrol and responsibility for defending and handling the Claim; (ii) the Indemnifying Party may, at its own cost, appoint as counsel in connectionwith conducting the defense and handling of such Claim any law firm or counsel selected by the Indemnifying Party which is reasonably acceptable tothe Indemnified Party; (iii) the Indemnifying Party shall keep the Indemnified Party informed of the status of such Claim; and (iv) the IndemnifyingParty shall have the right to settle such Claim on any terms the Indemnifying Party chooses; provided, however, that it shall not, without the priorwritten consent of the Indemnified Party (such consent not to be unreasonably withheld, conditioned, or delayed), agree to a settlement of any Claimwhich could lead to liability or create any financial or other obligation on the part of the Indemnified Party for which the Indemnified Party is notentitled to indemnification under this Agreement or which admits any wrongdoing or responsibility for the Claim on behalf of the Indemnified Party orwhich involves a conduct restriction or obligation of any kind. The Indemnified Party shall cooperate with the Indemnifying Party and shall be entitledto participate in, but not control, the defense of such Claim with its own counsel and at its own expense. In particular, the Indemnified Party shallfurnish such records, information, and testimony, provide witnesses, and attend such conferences, discovery proceedings, hearings, trials, and appealsas may be reasonably requested in connection therewith. Such cooperation shall include access during normal business hours by the IndemnifyingParty to, and reasonable retention by the Indemnified Party of, records and information that are reasonably relevant to such Claim, and making theIndemnified Party, the Indemnitees, and its and their employees and agents available on a mutually convenient basis to provide additional informationand explanation of any records or information provided.(e) If the Indemnifying Party does not assume the defense of the Indemnified Party in accordance with Section 17.3(c), the Indemnified Party may,at the Indemnifying Party’s expense, select counsel reasonably acceptable to the Indemnifying Party in connection with conducting the defense andhandling of such Claim and defend or handle such Claim in such manner as it may deem appropriate. In such event, the Indemnified Party shall keepthe Indemnifying Party reasonably informed of the status of such Claim and shall not settle such Claim without the prior written consent of theIndemnifying Party, which consent shall not be unreasonably withheld, conditioned, or delayed. If the Indemnified Party defends or handles suchClaim, the Indemnifying Party shall cooperate with the Indemnified Party, at the Indemnified Party’s request but at no expense to the IndemnifiedParty, and shall be entitled to participate in the defense and handling of such Claim with its own counsel and at its own expense.(f) Neither Party shall have the obligation to indemnify the other Party in connection with any settlement made without the Indemnifying Party’swritten consent, which consent shall not be unreasonably withheld, conditioned or delayed. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -96-Confidential Treatment Requested by MeiraGTx Holdings plc 17.4.Mitigation of Loss.Each Indemnified Party will take and will procure that the other Janssen Indemnitees (where Janssen is the Indemnified Party) or the MeiraGTxIndemnitees (where MeiraGTx is the Indemnified Party) take all such reasonable steps and actions in order to mitigate any potential Damages underthis Article 17. 17.5.Limited Liability.NEITHER PARTY SHALL BE LIABLE TO THE OTHER PARTY FOR ANY SPECULATIVE OR PUNITIVE DAMAGES OF ANY KIND ARISINGFROM OR RELATING TO THIS AGREEMENT, THE NEGOTIATION OF THIS AGREEMENT OR THE ACTIVITIES CONDUCTED HEREUNDER,OR ANY OTHER DAMAGES NOT REASONABLY FORESEEABLE AS A PROXIMATE RESULT OF THE BREACH OF A PARTY OF THISAGREEMENT, ON ANY THEORY OF LIABILITY (WHETHER IN CONTRACT, TORT, STRICT LIABILITY OR OTHERWISE), REGARDLESS OFANY NOTICE, AWARENESS OR ADVICE OF THE LIKELIHOOD OF SUCH DAMAGES. NOTWITHSTANDING THE FOREGOING, NOTHING INTHIS SECTION 17.5 IS INTENDED TO OR SHALL LIMIT OR RESTRICT INDEMNIFICATION FOR DAMAGES OF THE NATURE DESCRIBEDABOVE THAT ARE PAYABLE TO A THIRD PARTY FOLLOWING A FINAL ADJUDICATION OF THE MATTER IN QUESTION OR ASETTLEMENT EFFECTED IN ACCORDANCE WITH THIS AGREEMENT. 17.6.Insurance ObligationsEach Party, at its own expense, shall procure and maintain during the Term and for a period of [***] thereafter product liability insurance adequate tocover the activities to be conducted by such Party and its obligations under this Agreement that are consistent with normal business practices ofprudent companies similarly situated; provided, however, that in no event shall such product liability insurance be written in amounts less than [***]per claim or per occurrence and annual aggregate. All such insurance shall include worldwide coverage. Prior to the initiation of any Clinical Study ofa Product, the Party responsible for such Clinical Study shall secure, and maintain in full force and effect, clinical trial insurance as required byApplicable Law in those territories where such Clinical Study shall be conducted. It is understood that such insurance shall not be construed to create alimit of either Party’s liability with respect to its indemnification obligations under Section 17.1 and Section 17.2. Each Party shall provide the otherParty with written evidence of such insurance upon request. Each Party shall provide the other Party with written notice at least [***] prior to thecancellation, non-renewal or material change of such insurance that could materially adversely affect the rights of such other Party hereunder.Notwithstanding the foregoing, either Party’s failure to maintain adequate insurance shall not relieve that Party of its obligations set forth in thisAgreement. The Parties acknowledge and agree that Janssen may meet its obligations under this Section 17.6 through self-insurance consistent with thelevels set forth herein with prior written notice to MeiraGTx. In such event, Janssen shall provide a written certification of such self-insurance toMeiraGTx upon request. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -97-Confidential Treatment Requested by MeiraGTx Holdings plc 18.DISPUTE RESOLUTION 18.1.Dispute Resolution.(a) Escalation; Decision-Making Authority. In the case of any dispute, claim or controversy between the Parties arising from or related to thisAgreement, or the interpretation, application, breach, termination or validity of this Agreement and which are not subject to Section 5.8(c) (a“Dispute”), the Parties will discuss and negotiate in good faith a solution acceptable to the Parties and in the spirit of this Agreement. If, afternegotiating in good faith pursuant to the foregoing sentence, the Parties fail to reach agreement within [***] (or such longer period as agreed in writingby the Parties), then the Dispute may be referred to the Executive Officers for resolution at the request of either Party. If, after negotiating in good faith,the Executive Officers fail to reach agreement within [***] of submission to the Executive Officers (or such longer period as agreed in writing by theParties), then either Party may upon written notice to the other submit the Dispute to non-binding mediation pursuant to Section 18.1(b).(b) Mediation.(i) If the Parties fail to resolve the Dispute pursuant to Section 18.1(a), the Parties shall attempt in good faith to resolve any Dispute byconfidential mediation in accordance with the then-current Mediation Procedure of the International Institute for Conflict Prevention and Resolution(“CPR Mediation Procedure”) (www.cpradr.org) before initiating arbitration. The CPR Mediation Procedure shall control, except where it conflictswith these provisions, in which case these provisions control. The mediator shall be chosen pursuant to CPR Mediation Procedure. The mediation shallbe held in New York, New York.(ii) Either Party may initiate mediation by written notice to the other Party of the existence of a Dispute. The Parties agree to select amediator within [***] of the notice and the mediation will begin [***] after the selection. The mediation will continue until the mediator, or eitherParty, declares in writing, no sooner than after the conclusion of [***] of a substantive mediation conference attended on behalf of each Party by asenior business person with authority to resolve the Dispute, that the Dispute cannot be resolved by mediation. In no event, however, shall mediationcontinue more than [***] from the initial notice by a Party to initiate meditation unless the Parties agree in writing to extend that period.(iii) Any period of limitations that would otherwise expire between the initiation of mediation and its conclusion shall be extended until[***] after the conclusion of the mediation.(c) Arbitration. If the Parties fail to resolve the Dispute pursuant to Section 18.1(a) or Section 18.1(b), and a Party desires to pursue resolution ofthe Dispute, subject to Section 18.5, the Dispute shall be resolved by final and binding arbitration as follows: Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -98-Confidential Treatment Requested by MeiraGTx Holdings plc (i) The Dispute shall be finally resolved by arbitration in accordance with the then-current CPR Rules for Non-Administered ArbitrationRules (“CPR Rules”) (www.cpradr.org), except where they conflict with these provisions, in which case these provisions control. The place ofarbitration shall be New York, New York. All aspects of the arbitration shall be treated as confidential.(ii) Whenever a Party decides to institute such arbitration proceedings, such Party shall as [***] as practicable, give written notice to thateffect to the other Party. The law of this arbitration clause shall be the law of New York, USA, the seat and venue of the arbitration shall be New York,USA, and the arbitration proceedings shall be conducted in the English language. The arbitrators will be chosen from the CPR Panel of DistinguishedNeutrals, unless a candidate not on such panel is approved by both Parties. Each arbitrator shall be a lawyer with at least fifteen (15) years’ experiencewith a law firm or corporate law department of over twenty-five (25) lawyers or who was a judge of a court of general jurisdiction. There shall be three(3) arbitrators for the Dispute, selected as follows: each Party shall select an arbitrator in accordance with the “screened” appointment procedureprovided in CPR Rule 5.4, and the chair will be chosen in accordance with CPR Rule 6.4. If, however, the aggregate award sought by the Parties is lessthan [***] and equitable relief is not sought, a single arbitrator shall be appointed in accordance with the CPR Rules. Candidates for the arbitratorposition(s) may be interviewed by representatives of the Parties in advance of their selection, provided that all Parties are represented.(iii) The hearing on the merits shall be concluded within [***] after the initial prehearing conference and the award shall be renderedwithin [***] of the conclusion of the hearing, or of any post-hearing briefing, which briefing shall be completed by both sides within [***] after theconclusion of the hearing, unless the arbitrator(s) determine(s), in a reasoned decision, that the interest of justice or the complexity of the case requiresthat the time limit for concluding the hearing on the merits or rendering the award be extended. In the event the Parties cannot agree upon a schedule,then the arbitrator(s) shall set the schedule following the time limits set forth above as closely as practical.(iv) The hearing on the merits will be concluded in [***] or less, unless the arbitrator(s) determine(s), in a reasoned decision, that theinterest of justice or the complexity of the case requires that the time limit for concluding the hearing on the merits or rendering the award be extended.Multiple hearing days will be scheduled consecutively to the greatest extent possible. A transcript of the testimony adduced at the hearing shall bemade and shall be made available to each Party.(v) The Parties shall allow and participate in discovery in accordance with the United States Federal Rules of Civil Procedure. Unresolveddiscovery disputes shall be submitted to the arbitrator(s).(vi) The arbitrator(s) shall decide the merits of any Dispute in accordance with the law governing this Agreement, without application ofany principle of conflict of laws that would result in reference to a different law. The arbitrator(s) may not apply principles such as “amiablecompositeur” or “natural justice and equity.” Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -99-Confidential Treatment Requested by MeiraGTx Holdings plc (vii) The arbitrator(s) are expressly empowered to decide dispositive motions in advance of any hearing and shall endeavor to decide suchmotions as would a United States District Court Judge sitting in the jurisdiction whose substantive law governs.(viii) The arbitrator(s) shall render a written opinion stating the reasons upon which the award is based. The Parties consent to the entry ofjudgment on any award rendered hereunder. Judgment on the award may be entered in any court of competent jurisdiction.(ix) The award of the arbitral tribunal shall be final and binding upon the Parties, and the prevailing Party may apply to a court ofcompetent jurisdiction for enforcement of such award. When any Dispute occurs and when any Dispute is under arbitration, except for the matters inDispute, the Parties shall continue to fulfill their respective obligations and shall be entitled to exercise their rights under this Agreement. Except in aproceeding to enforce the results of the arbitration or as otherwise required by Applicable Law, neither Party nor any arbitrator may disclose theexistence, content or results of any arbitration hereunder without the prior written consent of both Parties. Notwithstanding anything to the contrarycontained in this Agreement, each Party to the Dispute shall be entitled to seek provisional remedies, interim measures of protection and emergencyrelief, if possible, such as attachment, preliminary injunction, replevin or other equitable relief, from the arbitral tribunal or any court of competentjurisdiction in accordance with the Applicable Laws of that jurisdiction in order to avoid irreparable harm, maintain the status quo, preserve its statusand priority as a creditor or preserve the subject matter of the Dispute.(d) Waivers. EACH PARTY HERETO WAIVES: (I) ITS RIGHT TO TRIAL OF ANY ISSUE BY JURY AND (II) ANY CLAIM FOR ATTORNEYFEES, COSTS AND PREJUDGMENT INTEREST; PROVIDED, HOWEVER, THAT THE FOREGOING WILL NOT LIMIT A PARTY’S OBLIGATIONSIN RESPECT OF DAMAGES CLAIMED BY A THIRD PARTY. 18.2.Governing Law.This Agreement shall be governed by and construed under the laws of the State of New York, without giving effect to the conflicts or choice of lawsprovision thereof (“Governing Law”). The United Nations Convention on Contracts for the International Sale of Goods (1980) shall not apply to theinterpretation of this Agreement. 18.3.Exclusions.Nothing in this Article 18 shall preclude a Party from: (a) seeking and obtaining in any competent court injunctive or equitable relief to preserve thestatus quo or prevent immediate harm to the Party; or (b) submitting any dispute, controversy or Claim relating to the scope, validity, enforceability orinfringement of any Patents or Trademarks to adjudication in accordance with the Applicable Laws of the country or jurisdiction in which the relevantpatent is pending or has been issued, including before any patent or trademark administrative body in the country in which such Patent or Trademarkwas granted or arose. The Parties agree that the venue of any such adjudication involving a patent pending in or issued by the United States will be aU.S. federal Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -100-Confidential Treatment Requested by MeiraGTx Holdings plc district court sitting in New York, New York or an appellate body of such court, and for a patent pending in or issued by any other country, anycompetent court having jurisdiction over the subject of the Patent controversy or Claim sitting in the capital of such country (or if there is not any suchcompetent court in the capital, a location reasonably proximate to the capital), and each Party hereby irrevocably submits to the jurisdiction of suchcourts or administrative bodies. 18.4.Cumulative Remedies.Unless expressly stated otherwise in this Agreement, no remedy referred to in this Agreement is intended to be exclusive, but each shall be cumulativeand in addition to, and not in lieu of, any other remedy referred to in this Agreement or otherwise available to either Party under Applicable Law. 18.5.Injunctive Relief.Notwithstanding anything to the contrary set forth in this Agreement, the Parties each stipulate and agree that given the nature of the ConfidentialInformation and Intellectual Property Rights and the competitive damage and irreparable harm that may result to a Party upon unauthorized disclosure,use or transfer of its Confidential Information or Intellectual Property Rights to any Third Party, monetary damages may not be a sufficient remedy forany breach of Section 4.4, Article 11, Article 12 or Article 13 by a Party. In the case of any such breach or threatened breach, in addition to all otherremedies, the non-breaching Party will be entitled to seek equitable relief (including temporary or permanent restraining orders, specific performance orother injunctive relief) for any such breach or threatened breach from any court of competent jurisdiction without first submitting to the disputeresolution procedures set forth in Section 18.1. 19.GENERAL PROVISIONS 19.1.Assignment.Subject to Section 4.6(d) and Section 4.6(e), neither Party may assign its rights or obligations under this Agreement, in whole or in part, without theother Party’s prior written consent, except that either Party may assign this Agreement, in whole or in part, without the other Party’s consent to: (a) anAffiliate of the assigning Party; or (b) any Third Party successor or purchaser of all or substantially all of its business or assets to which this Agreementrelates, whether in a merger, sale of stock, sale of assets or other similar transaction. In addition, Janssen may, without the prior written consent ofMeiraGTx, assign its rights and obligations, in whole or in part, under this Agreement to a Third Party, where Janssen or its Affiliate is required, ormakes a good faith determination based on advice of counsel, to divest any Products in order to comply with Applicable Law or the order of anyGovernmental Authority as a result of a merger or acquisition or similar transaction. In any such case, Janssen shall provide MeiraGTx with advancenotice of any such assignment. Any permitted assignee will assume all obligations of its assignor under this Agreement (or related to the assignedportion in case of a partial assignment). For clarity: (i) an assignment to an Affiliate will terminate, and all rights so assigned will revert to the assigningParty, if and when such Affiliate ceases to be an Affiliate of the assigning Party; and (ii) sublicensing of any licenses granted under this Agreement willbe governed by Section 4.1(c) and Section 4.3. Any attempted assignment in contravention of the foregoing will be null and void. Subject to the termsof this Agreement, this Agreement will be binding upon and inure to the benefit of the Parties and their respective successors and permitted assigns. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -101-Confidential Treatment Requested by MeiraGTx Holdings plc 19.2.Extension to Affiliates.Each Party may discharge any obligations and exercise any rights under this Agreement through delegation of its obligations or rights to any of itsAffiliates. Each Party hereby guarantees the performance by its Affiliates of such Party’s obligations under this Agreement, and will cause its Affiliatesto comply with the provisions of this Agreement in connection with such performance. Any breach by a Party’s Affiliate of any of such Party’sobligations under this Agreement will be deemed a breach by such Party. Any reference to performance by a Party hereunder includes any performanceby its Affiliates. 19.3.Severability.If any of the provisions of this Agreement become void or unenforceable as a matter of law, then this Agreement shall be construed as if such provisionwere not contained herein, the remainder of this Agreement shall be in full force and effect, and the Parties will negotiate in good faith to substitute anyinvalid or unenforceable provision with a valid and enforceable provision such that this Agreement conforms as nearly as possible with the originalintent of the Parties. 19.4.Force Majeure.If either Party is prevented from performing its obligations under this Agreement due to any contingency beyond its reasonable control (“ForceMajeure”), including acts of Governmental Authorities, any war, terrorism, hostilities between nations, civil commotions, riots, national industrystrikes, sabotage, fire, floods and acts of nature such as typhoons, hurricanes, earthquakes, or tsunamis, the Party so affected shall not be responsible tothe other Party for any delay or failure of performance of its obligations hereunder, for so long as and to the extent that such Force Majeure preventssuch performance. If a Force Majeure arises, the Party immediately affected thereby shall give prompt written notice to the other Party specifying theForce Majeure event complained of, and shall use Commercially Reasonable Efforts to avoid or remove such causes of non-performance and tomitigate the effect of such occurrence to resume performance of its obligations with reasonable dispatch. 19.5.Waivers and Amendments.The delay or failure of any Party to assert a right hereunder or to insist upon compliance with any term or condition of this Agreement shall notconstitute a waiver of that right, term or condition or excuse a similar subsequent failure to perform any such term or condition by the other Party, andno waiver shall be effective unless it has been given in writing and signed by the Party giving such waiver. No waiver by either Party of any conditionor of the breach of any term contained in this Agreement, in any one (1) or more instances, will be deemed to be, or considered as, a further orcontinuing waiver of any such condition or of the breach of such term or any other term of this Agreement. No provision of this Agreement may beamended or modified other than by a written document signed by authorized representatives of each Party. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -102-Confidential Treatment Requested by MeiraGTx Holdings plc 19.6.Relationship of the Parties.Nothing contained in this Agreement shall be deemed to constitute a partnership, joint venture, or legal entity of any type between MeiraGTx andJanssen or their respective Affiliates, or to constitute one as the agent of the other. Moreover, each Party agrees not to construe this Agreement, or anyof the transactions contemplated hereby, as a partnership for any tax purposes. Each Party shall act solely as an independent contractor, and nothing inthis Agreement shall be construed to give any Party the power or authority to assume or create any obligations on behalf of or in the name of the otherParty or to bind the other Party to any contract, agreement or undertaking with any Third Party. 19.7.Notices.All notices, consents, waivers, and other communications under this Agreement must be in writing, in the English language, and will be deemed tohave been duly given when: (a) delivered by hand; (b) when sent by an internationally recognized overnight delivery service (receipt requested); or(c) when sent by confirmed email, in each case, to the appropriate addresses set forth below (or to such other addresses as a Party may designate bynotice in accordance with this Section 19.7):If to MeiraGTx:MeiraGTx UK II Limited25 Provost StreetLondon N1 7NH Email: [***]Attn: Chief Operating Officerwith a copy to:MeiraGTx - US430 East 29th Street, 10th FloorNew York, NY 10016Attn: Chief Operating OfficerIf to Janssen:Janssen Pharmaceuticals, Inc.1125 Trenton-Harbourton RoadTitusville, NJ 08560Attention: President Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -103-Confidential Treatment Requested by MeiraGTx Holdings plc with a copy to:Johnson & JohnsonOne Johnson & Johnson PlazaNew Brunswick, New Jersey 08933Attn: General Counsel, PharmaceuticalsAny such notice shall be deemed to have been given on the date delivered. A Party may add, delete (so long as at least one (1) person is remaining) orchange the person or address to which notices should be sent at any time upon written notice delivered to the other Party in accordance with thisSection 19.7. 19.8.Further Assurances.Janssen and MeiraGTx hereby covenant and agree without the necessity of any further consideration, to execute, acknowledge and deliver, and tocause to be executed, acknowledged, and delivered, any and all such other documents and take any such other action as may be reasonably necessaryto carry out the intent and purposes of this Agreement. 19.9.No Third Party Beneficiary Rights.The provisions of this Agreement are for the sole benefit of the Parties and their successors and permitted assigns, and they shall not be construed asconferring any rights to any Third Party (including any third party beneficiary rights), except with respect to certain Janssen Indemnitees and certainMeiraGTx Indemnitees, who are Third Parties, solely with respect to Article 17. 19.10.English Language.This Agreement was prepared and executed in the English language, which language shall govern the interpretation of, and any disputes regarding, theterms of this Agreement. Any translation into any other language shall not be an official version of this Agreement and in the event of any conflict ininterpretation between the English version and such translation, the English version shall prevail. 19.11.Interpretation.Unless the context of this Agreement otherwise requires:(a) references to an Article, Section or Exhibit means an Article or Section of, or Exhibit to, this Agreement, unless another agreement isspecified;(b) the term “including” (in its various forms) means “including without limitation”;(c) a particular statute or statutory instrument, regulation or any of their provisions shall include all rules and regulations thereunder and shall beconstrued as a reference to that statute or statutory instrument, regulation or such provision as the same may have been or may from time to timehereafter be modified, amended or re-enacted; Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -104-Confidential Treatment Requested by MeiraGTx Holdings plc (d) words denoting the singular shall include the plural and vice versa, and words denoting any gender shall include all genders;(e) references to a particular Person include such Person’s successors and assigns to the extent not prohibited by this Agreement;(f) the Exhibits and other attachments form part of the operative provisions of this Agreement and references to this Agreement shall, unless thecontext otherwise requires, include references to the Exhibits and attachments;(g) the headings in this Agreement, in any Exhibit to this Agreement and in the table of contents to this Agreement are for information andconvenience only and shall not in any way affect the construction of or be considered in the interpretation of this Agreement;(h) the word “notice” means notice in writing (whether or not specifically stated) and will include notices, consents, approvals and other writtencommunications contemplated under this Agreement;(i) the word “or” will be interpreted in the inclusive sense commonly associated with the term “and/or”;(j) “days” refers to calendar days;(k) the terms “hereof”, “herein”, “hereby”, and derivative or similar words refer to this entire Agreement;(l) general words shall not be given a restrictive interpretation by reason of their being preceded or followed by words indicating a particularclass of acts, matters or things;(m) the words “shall” and “will” have the same meaning; and(n) the Parties agree that each Party has been represented by legal counsel in connection with this Agreement, the terms and conditions of thisAgreement are the result of negotiations between the Parties, each Party has participated in the drafting hereof, and the terms and provisions of thisAgreement shall not be construed and applied in favor of or against any Party by reason of the extent to which any Party participated in the preparationof this Agreement. 19.12.Entire Agreement.This Agreement, together with its Exhibits, which are incorporated by reference herein, sets forth the entire agreement and understanding between theParties as to the subject matter hereof and supersedes all prior discussions, representations, understandings, agreements, proposals, oral or written, andall other prior communications between the Parties with respect to such subject matter; provided, however, that the Prior CDA shall survive inaccordance with its terms, except that any Information (as defined in the Prior CDA) that was disclosed by MeiraGTx LLC under the Prior CDA and thatrelates to the subject matter of this Agreement will be deemed Confidential Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -105-Confidential Treatment Requested by MeiraGTx Holdings plc Information of MeiraGTx under this Agreement and treated in accordance with the terms and conditions of Article 12 and Article 13. If any conflictbetween a substantive provision of this Agreement and any Exhibit hereto arises, the substantive provisions of this Agreement shall prevail. 19.13.Counterparts.This Agreement may be executed in one or more counterparts, each of which shall be deemed an original, and all of which taken together shall bedeemed to constitute one and the same single instrument. Signature pages of this Agreement exchanged by facsimile or other electronic transmissionwill be deemed to be as effective as an original executed signature page.[Signature Pages Follow] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. -106-Confidential Treatment Requested by MeiraGTx Holdings plc IN WITNESS WHEREOF, the Parties have caused this Agreement to be executed by their duly authorized representatives. MEIRAGTX UK II LTDBy: /s/ Alexandria ForbesName: Alexandria ForbesTitle: Chief Executive OfficerSignature Page to Collaboration, Option and License Agreement Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc IN WITNESS WHEREOF, the Parties have caused this Agreement to be executed by their duly authorized representatives. MEIRAGTX HOLDINGS PLCBy: /s/ Alexandria ForbesName: Alexandria ForbesTitle: Chief Executive OfficerSignature Page to Collaboration, Option and License Agreement Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc IN WITNESS WHEREOF, the Parties have caused this Agreement to be executed by their duly authorized representatives. JANSSEN PHARMACEUTICALS, INC.By: /s/ Jeffrey N. SmithName: Jeffrey N. SmithTitle: Vice PresidentSignature Page to Collaboration, Option and License Agreement Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 1.34Clinical Development Plans, Including Anticipated Costs and TimelinesSee attached for anticipated costs and timelines.Initial Clinical Development Plans to be attached upon approval as Exhibits 1.34-4, -5, -6 and (as applicable) -7 in accordance with Section 6.1(c). Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 1.34-1Anticipated Costs and Timelines – CNGA3 Product[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 1.34-2Anticipated Costs and Timelines – CNGB3 Product[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 1.34-3Anticipated Costs and Timelines – RPGR Product[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 1.51CMC Development PlansTo be attached upon approval. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 1.103Final Report RequirementsRequired[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 1.136J&J Universal Calendar for 2019 and 2020[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 1.168MeiraGTx Patents[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 1.221IRD Genes[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 1.223Research PlansTo be attached upon approval. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 3.4Research Budget Cap Calendar Year 2019 2020 2021Research Budget Cap* [***] [***] [***] *Total budget to be [***] in accordance with Section 3.8. Includes all pre-clinical work required to [***]. Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 3.5Janssen Data PoliciesJANSSEN DATA GENERATION, PROCESSING AND STORAGE POLICIES[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 4.4MeiraGTx Subcontractors Subcontractor Activities[***] MeiraGTx Commitments to Engage Scheduled Subcontractors: [***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 8.2Key Terms of Clinical Supply Agreement and Clinical Quality Assurance AgreementThe Clinical Supply Agreement will substantially contain the following provisions:[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 8.3Key Terms of Commercial Supply Agreement and Commercial Quality AssuranceAgreementThe Commercial Supply Agreement will substantially contain the following provisions:[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 8.4(a)CMC Development Plan Activities – Breakdown[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 16.2(b)Third Party Rights[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Confidential Treatment Requested by MeiraGTx Holdings plc Exhibit 16.2(g)MeiraGTx Existing Third Party Obligations[***] Confidential Portions of this Exhibit marked as [***] have been omitted pursuant to a request for confidential treatment and have been filed separatelywith the Securities and Exchange Commission. Exhibit 21SUBSIDIARIES OF MEIRAGTX HOLDINGS PLC Legal Name of Subsidiary Jurisdiction of Organization BRI-Alzan, Inc. Delaware MeiraGTx B.V Netherlands MeiraGTx Limited England and Wales MeiraGTx, LLC Delaware MeiraGTx UK Limited England and Wales MeiraGTx UK II Limited England and Wales MeiraGTx Neurosciences, Inc. Delaware EXHIBIT 23.1Consent of Independent Registered Public Accounting FirmWe consent to the incorporation by reference in Registration Statement (Form S-8 No. 333-225535) pertaining to the 2016 Equity Incentive Plan, 2018Incentive Award Plan and 2018 Employee Share Purchase Plan of MeiraGTx Holdings plc of our report dated March 26, 2019, with respect to theconsolidated financial statements of MeiraGTx Holdings plc included in this Annual Report (Form 10-K) of MeiraGTx Holdings plc for the year endedDecember 31, 2018./s/ Ernst & Young LLPStamford, ConnecticutMarch 26, 2019Exhibit 31.1CERTIFICATIONI, Alexandria Forbes, certify that: 1.I have reviewed this Annual Report on Form 10-K for the fiscal year ended December 31, 2018 of MeiraGTx Holdings plc; 2.Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make thestatements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by thisreport; 3.Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respectsthe financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report; 4.The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined inExchange Act Rules 13a-15(e) and 15d-15(e)) for the registrant and have: (a)Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision,to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others withinthose entities, particularly during the period in which this report is being prepared; (b)[intentionally omitted]; (c)Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about theeffectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and (d)Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s mostrecent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likelyto materially affect, the registrant’s internal control over financial reporting; and 5.The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, tothe registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions): (a)All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which arereasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and (b)Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internalcontrol over financial reporting. Date: March 26, 2019 By: /s/ Alexandria Forbes Alexandria Forbes President and Chief Executive Officer (Principal Executive Officer)Exhibit 31.2CERTIFICATIONI, Katherine Breedis, certify that: 1.I have reviewed this Annual Report on Form 10-K for the fiscal year ended December 31, 2018 of MeiraGTx Holdings plc; 2.Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make thestatements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by thisreport; 3.Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respectsthe financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report; 4.The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined inExchange Act Rules 13a-15(e) and 15d-15(e)) for the registrant and have: (a)Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision,to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others withinthose entities, particularly during the period in which this report is being prepared; (b)[intentionally omitted]; (c)Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about theeffectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and (d)Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s mostrecent fiscal quarter (the registrant’s fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likelyto materially affect, the registrant’s internal control over financial reporting; and 5.The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, tothe registrant’s auditors and the audit committee of the registrant’s board of directors (or persons performing the equivalent functions): (a)All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which arereasonably likely to adversely affect the registrant’s ability to record, process, summarize and report financial information; and (b)Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internalcontrol over financial reporting. Date: March 26, 2019 By: /s/ Katherine Breedis Katherine Breedis Chief Financial Officer (Principal Financial Officer)Exhibit 32.1CERTIFICATION PURSUANT TO18 U.S.C. SECTION 1350, AS ADOPTED PURSUANT TOSECTION 906 OF THE SARBANES-OXLEY ACT OF 2002In connection with the Annual Report of MeiraGTx Holdings plc (the “Company”) on Form 10-K for the fiscal year ended December 31, 2018, asfiled with the Securities and Exchange Commission on the date hereof (the “Report”), I certify, pursuant to 18 U.S.C. § 1350, as adopted pursuant to§ 906 of the Sarbanes-Oxley Act of 2002, that to the best of my knowledge: (1)The Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and (2)The information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of theCompany. Date: March 26, 2019 By: /s/ Alexandria Forbes Alexandria Forbes President and Chief Executive Officer (Principal Executive Officer)Exhibit 32.2CERTIFICATION PURSUANT TO18 U.S.C. SECTION 1350, AS ADOPTED PURSUANT TOSECTION 906 OF THE SARBANES-OXLEY ACT OF 2002In connection with the Annual Report of MeiraGTx Holdings plc (the “Company”) on Form 10-K for the fiscal year ended December 31, 2018, asfiled with the Securities and Exchange Commission on the date hereof (the “Report”), I certify, pursuant to 18 U.S.C. § 1350, as adopted pursuant to§ 906 of the Sarbanes-Oxley Act of 2002, that to the best of my knowledge: (1)The Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934; and (2)The information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of theCompany. Date: March 26, 2019 By: /s/ Katherine Breedis Katherine Breedis Chief Financial Officer (Principal Financial Officer)

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