uniQure
Annual Report 2013

Plain-text annual report

Use these links to rapidly review the documentTABLE OF CONTENTS UNIQURE N.V. INDEX TO CONSOLIDATED FINANCIAL STATEMENTSTable of ContentsSECURITIES AND EXCHANGE COMMISSIONWashington D.C. 20549FORM 20-FuniQure N.V.(Exact name of Registrant as specified in its charter and translation of Registrant's name into English)The Netherlands(Jurisdiction of incorporation or organization)Meibergdreef 61, 1105BA Amsterdam, The Netherlands(Address of principal executive offices)Jörn AldagChief Executive OfficerTel: +31 20 566 7394Fax: +31 20 566 9272Meibergdreef 61, 1105BA Amsterdam, The Netherlands(Name, Telephone, E-mail and/or Facsimile number and Address of Company Contact Person)Securities registered or to be registered pursuant to Section 12(b) of the Act:Securities registered or to be registered pursuant to Section 12(g) of the Act: NoneSecurities for which there is a reporting obligation pursuant to Section 15(d) of the Act: Ordinary Shareso REGISTRATION STATEMENT PURSUANT TO SECTION 12(b) OR (g) OF THE SECURITIES EXCHANGE ACTOF 1934OR ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934For the fiscal year ended December 31, 2013ORo TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934For the transition period from to o SHELL COMPANY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934Date of event requiring this shell company reportCommission file number: 001-36294Title of each class Name of each exchange on which registeredOrdinary Shares NASDAQ Global Select MarketIndicate the number of outstanding shares of each of the issuer's classes of capital or common stock as of the close of the period covered by the annual report:12,194,906 Ordinary Shares(as of December 31, 2013)Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act.o Yes  NoIf this report is an annual or transition report, indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934.o Yes  NoIndicate 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 preceding 12 months (orfor such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes o NoIndicate by check mark whether the registrant has submitted electronically and posted on its corporate Web site, if any, every Interactive Data File required to be submitted and postedpursuant 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 and post suchfiles). Yes o NoIndicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, or a non-accelerated filer. See definition of "accelerated filer and large accelerated filer" inRule 12b-2 of the Exchange Act. (Check one):Indicate by check mark which basis of accounting the registrant has used to prepare the financial statements included in this filing:If "Other" has been checked in response to the previous question, indicate by check mark which financial statement item the registrant has elected to follow:o Item 17 o Item 18If this is an annual report, indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act).o Yes  NoIndicate by check mark whether the registrant has filed all documents and reports required to be filed by Sections 12, 13 or 15(d) of the Securities Exchange Act of 1934 subsequent to thedistribution of securities under a plan confirmed by a court.o Yes o NoLarge accelerated filer o Accelerated filer o Non-accelerated filer U.S. GAAP o International Financial Reporting Standards as issuedby the International Accounting Standards Board  Other oTable of ContentsTABLE OF CONTENTS Page General 1 Forward-Looking Statements 1 PART I Item 1 Identity of Directors, Senior Management and Advisers 1 Item 2 Offer Statistics and Expected Timetable 1 Item 3 Key Information 1 Item 4 Information on the Company 34 Item 4A Unresolved Staff Comments 63 Item 5 Operating and Financial Review and Prospects 63 Item 6 Directors, Senior Management and Employees 86 Item 7 Major Shareholders and Related Party Transactions 93 Item 8 Financial Information 98 Item 9 The Offer and Listing 98 Item 10 Additional Information 99 Item 11 Quantitative and Qualitative Disclosures About Market Risk 110 Item 12 Description of Securities Other than Equity Securities 110 PART II Item 13 Defaults, Dividend Arrearages and Delinquencies 110 Item 14 Material Modification to the Rights of Security Holders and Use of Proceeds 110 Item 15 Control and Procedures 111 Item 16A Audit Committee Financial Expert 112 Item 16B Code of Ethics 112 Item 16C Principal Accountant Fees and Services 112 Item 16D Exemptions From the Listing Requirements and Standards for Audit Committees 112 Item 16E Purchases of Equity Securities by the Issuer and Affiliated Purchasers 112 Item 16F Change in Registrant's Certifying Accountant 113 Item 16G Corporate Governance 113 Item 16H Mine Safety Disclosure 113 PART III Item 17 Financial Statements 113 Item 18 Financial Statements 113 Item 19 Exhibits 114 Table of ContentsGeneral As used herein, references to "we", "us", the "company", "uniQure", "uniQure B.V." or the "Group", or similar terms in this Form 20-F shallmean uniQure N.V. and, as the context requires, its subsidiaries. Effective February 10, 2014, we converted from a private company with limitedliability (besloten vennootschap met beperkte aansprakelijkheid) to a public company with limited liability (naamloze vennootschap) under the laws ofthe Netherlands. In connection with this conversion, our legal name changed from uniQure B.V. to uniQure N.V. Our financial statements are presented in Euros except where otherwise indicated, and are prepared in accordance with International FinancialReporting Standards ("IFRS") as issued by the International Accounting Standards Board ("IASB"). All references in this annual report to "Dollars"and "$" are to US Dollars, and all references to "Euro" or "€" are to European Union Euro. For presentation purposes all financial information,including comparative figures from prior periods, have been stated in round thousands.Forward-Looking Statements This annual report on Form 20-F contains forward-looking statements. The Private Securities Litigation Reform Act of 1995 provides a safeharbor from civil litigation for forward-looking statements accompanied by meaningful cautionary statements. Except for historical information, thisreport contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of theSecurities Exchange Act of 1934, which may be identified by words such as "estimates", "anticipates", "projects", "plans", "seeks", "may", "will","expects", "intends", "believes", "should" and similar expressions, or the negative versions thereof, and which also may be identified by their context.Such statements, whether expressed or implied, are based upon our current expectations and speak only as of the date made. We assume no obligationto update or revise any forward-looking statements even if experience or future changes make it clear that any projected results expressed or impliedtherein will not be realized. These statements are subject to various risks, uncertainties and assumptions. Our actual results of operations may differ materially from thosestated in or implied by such forward-looking statements as a result of a variety of factors, including those described under "Risk Factors" and elsewherein this annual report.PART I Item 1 Identity of Directors, Senior Management and Advisers Not applicable.Item 2 Offer Statistics and Expected Timetable Not applicable.Item 3 Key InformationA. Selected Financial Data The selected consolidated financial data as of December 31, 2013 and 2012 and for each of the years ended December 31, 2013, 2012 and 2011have been derived from our the audited consolidated financial statements and notes thereto set forth in Item 18 of this annual report. The selectedconsolidated financial data as of December 31, 2011, and 2010 are derived from the audited consolidated financial statements not appearing in thisannual report.1Table of Contents The following selected consolidated financial data should be read in conjunction with our "Operating and Financial Review and Prospects" and ourconsolidated financial statements and related notes appearing elsewhere in this annual report. Our financial statements are prepared in accordance withIFRS. Note 1 to the financial statements contains additional information relating to the business combination between uniQure and Amsterdam MolecularTherapeutics, and the presentation of a continuous trading history. The total number of ordinary shares outstanding at December 31, 2011, 2012 and 2013 was 4,749,625; 9,653,495 and 12,194,906, respectively.The share capital at December 31, 2011, 2012 and 2013 was €237,000; €483,000 and €610,000, respectively.2 YEARS ENDED DECEMBER 31 € in thousands (except share and per share data) 2011 2012 2013 License revenues — — 440 Collaboration revenues — — 2,503 Total revenues — — 2,943 Cost of goods sold — — (800)Other income 2,192 649 585 Research and development expenses (15,500) (10,231) (13,182)Selling, general and administrative expenses (3,807) (4,564) (11,628)Other losses, net (26) (45) (453) Total Operating Costs (19,333) (14,840) (25,263) Operating result (17,141) (14,191) (22,535) Finance income 277 22 102 Finance expense (436) (547) (4,387) Finance income/(expense)—net (159) (525) (4,285) Result before corporate income taxes (17,300) (14,716) (26,820)Corporate income taxes — — — Net Loss (17,300) (14,716) (26,820) Items that may be subsequently reclassified to profit or loss — — 12 Other comprehensive income — — — Total comprehensive loss* (17,300) (14,716) (26,808) Loss per share attributable to the equity holders of the companyduring the year Basic and diluted loss per share (3.65) (1.70) (2.48)*Total comprehensive loss is fully attributable to equity holders of the groupTable of Contents The following table sets forth selected balance sheet data as of the dates indicated:Consolidated Balance Sheet Data:Exchange Rate Information Our business is primarily conducted in the European Union, and we maintain our books and records in Euro. We have presented results ofoperations in Euro. In this annual report, translations from Euro to US dollars were made at a rate of €0.725 to $1.00, the official exchange rate quotedby the European Central Bank at the close of business on December 31, 2013. As of April 24, 2014, the official exchange rate of Euro to US dollarswas 0.724 to $1.00. Such US dollar amounts are not necessarily indicative of the actual amounts of US dollars which could have been actuallypurchased on exchange of Euro on the dates indicated.B. Capitalization and Indebtedness Not applicable.C. Reasons for the Offer and Use of Proceeds Not applicable.3 AS OF DECEMBER 31, (€ in thousands) 2010 2011 2012 2013 Cash and cash equivalents 17,859 1,100 263 23,810 Total assets 22,703 5,804 5,567 38,969 Total debt 4,621 4,544 1,498 7,864 Accumulated deficit (88,205) (105,505) (117,234) (144,041)Total shareholders' equity (deficit) 13,659 (2,593) (448) 5,564 Period-end Averagefor period Low High (€ per U.S. dollar) Year Ended December 31 2010 0.748 0.754 0.687 0.837 2011 0.773 0.718 0.672 0.776 2012 0.758 0.778 0.743 0.827 2013 0.725 0.753 0.724 0.783 2014 (through April 24) 0.724 0.725 0.721 0.729 Month Ended October 31, 2013 0.733 0.733 0.724 0.741 November 30, 2013 0.735 0.741 0.735 0.748 December 31, 2013 0.725 0.730 0.724 0.739 January 31, 2014 0.740 0.735 0.731 0.740 February 28, 2014 0.724 0.732 0.724 0.741 March 31, 2014 0.725 0.723 0.717 0.728 Table of ContentsD. Risk FactorsRisks Related to Our Financial Position and Need for Additional Capital We have incurred significant losses to date, expect to incur losses over the next several years and may never achieve or maintain profitability. We have incurred significant losses to date. We had a net loss of €26.8 million in 2013, €14.7 million in 2012 and €17.3 million in 2011. As ofDecember 31, 2013, we had an accumulated deficit of €144.0 million. To date, we have financed our operations primarily through the sale of equitysecurities and convertible debt and, to a lesser extent, through milestone payments, subsidies and grants from governmental agencies and fees forservices. We have devoted substantially all of our financial resources and efforts to research and development, including preclinical studies and clinicaltrials. Our product, Glybera, received marketing approval under exceptional circumstances from the European Commission in October 2012. We plan inthe future to apply for marketing approval for Glybera in the United States and other countries and will be required to conduct one or more additionalclinical trials of Glybera. We are still in the early stages of development of the other product candidates in our pipeline. We expect to continue to incursignificant expenses and losses over the next several years. Our net losses may fluctuate significantly from quarter to quarter and year to year. Weanticipate that our expenses will increase substantially as we:•complete our EMA-mandated post-approval clinical trial of Glybera and implement an LPLD patient registry; •conduct a clinical trial of Glybera, either as part of the EMA-mandated post-approval clinical trial or separately, to obtain data needed tofile a BLA for Glybera with the FDA; •seek marketing approval for Glybera in the United States and other countries; •initiate a Phase I/II clinical trial of AMT-060 for hemophilia B in collaboration with Chiesi; •advance the preclinical and clinical development of our other product candidates, most of which are at relatively early stages ofdevelopment, and seek to discover and develop additional product candidates; •seek marketing approval for any product candidates that successfully complete clinical trials; •establish a sales, marketing and medical affairs infrastructure in the United States; •complete the building out and equipping of our manufacturing facility in Lexington, Massachusetts to expand our manufacturingcapabilities for Glybera and our pipeline of product candidates; •fund the ongoing operations of our Lexington facility; •maintain, expand and protect our intellectual property portfolio, including in-licensing additional intellectual property rights from thirdparties; •hire additional personnel, particularly in our manufacturing, research, clinical development, medical affairs, commercial and qualitycontrol groups; •add operational, financial and management information systems and related finance and compliance personnel; and •operate as a public company. We are only in the preliminary stages of most of these activities. We and our collaborators may never succeed in these activities and, even if we do,may never generate revenues that are significant enough to achieve profitability.4Table of Contents Even if we do achieve profitability, we may not be able to sustain or increase profitability. Our failure to become and remain profitable woulddepress the value of our company and could impair our ability to expand our business, maintain our research and development efforts, diversify ourproduct offerings or even continue our operations.Our financial results will substantially depend on the commercial success of sales of Glybera. We anticipate that our collaborator Chiesi will commercially launch Glybera in the European Union in mid 2014 and that revenues from sales ofGlybera will be one of the principal sources of funds for our business for at least the next several years, although such revenues may be modest initially.A number of factors, some of which are out of our control, may adversely affect the commercial success of Glybera, including the following:•our collaborator Chiesi may not successfully commercialize Glybera in the European Union and other specified countries in the Chiesiterritory; •the post-approval requirements imposed by the EMA in connection with Glybera's approval under exceptional circumstances may becostly or may eventually lead to withdrawal of approval; •we may never be able to obtain marketing approval for Glybera in the United States or other countries; •Glybera may fail to achieve market acceptance by physicians, patients, third party payors and others in the medical community; •other alternative treatments for LPLD may be developed and gain commercial acceptance, eroding Glybera's market share; •the limited label we have received for Glybera in the European Union may limit our addressable market, and other regulatory agenciesmay approve Glybera only with a similarly limited label; •we may be unable to establish or maintain sales, marketing and medical affairs capabilities for the commercialization of Glybera in theUnited States, even if we receive FDA approval; and •coverage, pricing and reimbursement levels may be lower than we expect. Because our business is currently dependent on Glybera, failure to achieve anticipated revenues from this product would have an adverse effect onour results of operations and cause the value of our ordinary shares to decline.Even if our commercialization of Glybera or other product candidates for which we obtain marketing approval is successful, we may not befinancially successful due to our obligations to third parties. We have obtained exclusive or non-exclusive rights from third parties under a range of patents and other technology that we are exploiting inGlybera and our development programs. Our agreements with these third parties generally grant us a license to make, use, sell, offer to sell and importproducts covered by the licensed patent rights in exchange for our payment of some combination of an upfront amount, annual fees, royalties, a portionof amounts we receive from our sublicensees and payments upon the achievement of specified development, regulatory or commercial milestones. Forexample, we are contractually obligated to pay royalties and other obligations to third parties on net sales of Glybera by us, Chiesi or other sublicenseesor on other amounts we receive, including from Chiesi or other sublicensees for their sales of Glybera. We also received a technical development loanfrom the Dutch government, which requires repayment based on the timing and amount of revenues we receive from the sale of Glybera. Thesefinancial obligations to third parties are an expense to us, which could adversely affect our financial position.5Table of ContentsWe will likely need to raise additional funding, particularly if we experience delays in implementing our development programs orcommercialization efforts. Additional funding may not be available on acceptable terms, or at all, and any failure to obtain capital when neededmay force us to delay, limit or terminate our product development efforts or other operations. We expect to incur significant expenses in connection with our ongoing activities and expect that we will likely need to obtain substantial additionalfunding in connection with our continuing operations. We have based our estimate of our financing requirements on assumptions that may prove to bewrong, and we could use our capital resources sooner than we currently expect. Adequate capital may not be available to us when needed or may not be available on acceptable terms. Our ability to obtain debt financing may belimited by covenants we have made under our Loan and Security Agreement with Hercules Technology Growth Capital, Inc., or Hercules, and ourpledge to Hercules of substantially all of our assets as collateral to the extent that we raise additional capital through the sale of equity or convertible debtsecurities, our shareholders' ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences thatadversely affect the rights of shareholders of ordinary shares. If we raise additional funds through collaborations, strategic alliances or marketing,distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, productsor product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise capital when needed or on attractive terms, wecould be forced to delay, reduce or eliminate our research and development programs or any future commercialization efforts, which would have anegative impact on our financial condition.Our existing and any future indebtedness could adversely affect our ability to operate our business. As of December 31, 2013, we had a liability of €7.5 million ($10.0 million) of outstanding borrowings under our Loan and Security Agreementwith Hercules, which we are required to repay in monthly installments through October 1, 2016. We could in the future incur additional debt obligationsbeyond our borrowings from Hercules. Our existing loan obligations, together with other similar obligations that we may incur in the future, could havesignificant adverse consequences, including:•requiring us to dedicate a portion of our cash resources to the payment of interest and principal, reducing money available to fundworking capital, capital expenditures, product development and other general corporate purposes; •increasing our vulnerability to adverse changes in general economic, industry and market conditions; •subjecting us to restrictive covenants that may reduce our ability to take certain corporate actions or obtain further debt or equityfinancing; •limiting our flexibility in planning for, or reacting to, changes in our business and the industry in which we compete; and •placing us at a competitive disadvantage compared to our competitors that have less debt or better debt servicing options. We may not have sufficient funds, and may be unable to arrange for additional financing, to pay the amounts due under our existing loanobligations. Failure to make payments or comply with other covenants under our existing debt could result in an event of default and acceleration ofamounts due. Under our agreement with Hercules, the occurrence of an event that would reasonably be expected to have a material adverse effect on ourbusiness, operations, assets or condition is an event of default. If an event of default occurs and the lender accelerates the amounts due, we may not beable to make6Table of Contentsaccelerated payments, and the lender could seek to enforce security interests in the collateral securing such indebtedness, which includes substantially allof our assets.Our business operations may be negatively affected by the strategic restructuring we undertook in 2012. At the end of 2011, following the initial rejection of our application for marketing approval for Glybera in the European Union, our predecessorentity, Amsterdam Molecular Therapeutics, or AMT, initiated a strategic restructuring in order to conserve resources and improve its financial position.As part of this effort, AMT significantly reduced personnel, programs and spending. As a result, we lost many talented employees, includingemployees with an extensive understanding of our clinical programs as well as our regulatory and financial affairs. In the fourth quarter of 2011, totalstaff was reduced from 92 to 49. Since that time, we have hired a number of new staff, and total employee headcount as of December 31, 2013 was 87.In addition, we have engaged 33 consultants and contract workers. Nevertheless, this loss of talent and institutional knowledge has adversely affectedour operations during the past year and may result in delays in preparing regulatory filings, completing clinical trials and other related activities, andcould negatively impact our future business operations.Risks Related to the Development of Our Product Candidates We may not be successful in our efforts to use our gene therapy technology platform to build a pipeline of additional product candidates. A key element of our strategy is to use our gene therapy technology platform to expand our pipeline of gene therapies and to progress theseproduct candidates through clinical development together with our collaborators. Although we currently have a pipeline of programs at various stages ofdevelopment, we may not be able to identify or develop product candidates that are safe and effective. Even if we are successful in continuing to buildour pipeline, the potential product candidates that we identify may not be suitable for clinical development. Research programs to identify new productcandidates require substantial technical, financial and human resources. We or our collaborators may focus our efforts and resources on potentialprograms or product candidates that ultimately prove to be unsuccessful. If we do not continue to successfully develop and commercialize productcandidates based upon our technology, we may face difficulty in obtaining product revenues in future periods, which could result in significant harm toour financial position and adversely affect our share price.Our strategy of obtaining rights to key technologies through in-licenses may not be successful. We seek to expand our product pipeline in part by in-licensing the rights to key technologies, including those related to gene delivery and genecassettes. The future growth of our business will depend in significant part on our ability to in-license or otherwise acquire the rights to additionalproduct candidates or technologies, particularly through our collaborations with academic research institutions. However, we may be unable to in-license or acquire the rights to any such product candidates or technologies from third parties on acceptable terms or at all. The in-licensing andacquisition of these technologies is a competitive area, and a number of more established companies are also pursuing strategies to license or acquireproduct candidates or technologies that we may consider attractive. These established companies may have a competitive advantage over us due to theirsize, cash resources and greater clinical development and commercialization capabilities. In addition, companies that perceive us to be a competitor maybe unwilling to license rights to us. Furthermore, we may be unable to identify suitable product candidates or technologies within our area of focus. Ifwe are unable to successfully obtain rights to suitable product candidates or technologies, our business, financial condition and prospects could suffer.7Table of ContentsWe may encounter substantial delays in and impediments to the progress of our clinical trials or fail to demonstrate the safety and efficacy of ourproduct candidates. Clinical development is expensive, time-consuming and uncertain as to outcome. Our product candidates are in early clinical or preclinicaldevelopment, and there is a significant risk of failure or delay in each of these programs. In several of our programs, we intend to transition acollaborator's program to a different viral vector or to our insect-cell based manufacturing process, which could result in additional developmentchallenges and delays. We cannot guarantee that any clinical trials will be conducted as planned or completed on schedule, if at all. A failure of one ormore clinical trials can occur at any stage of testing. Events that may prevent successful or timely completion of clinical development include, but are notlimited to:•delays in reaching a consensus with regulatory agencies on study design; •delays in reaching agreement on acceptable terms with prospective clinical research organizations, or CROs, and clinical trial sites; •delays in obtaining required Institutional Review Board, or IRB, approval at each clinical trial site; •imposition of a clinical hold by regulatory agencies after an inspection of our clinical trial operations or trial sites; •failure by CROs, other third parties or us to adhere to clinical trial requirements or otherwise properly manage the clinical trial process,including the retention of proper case files; •failure to perform in accordance with the FDA's good clinical practices, or GCP, or applicable regulatory guidelines in other countries; •delays in the testing, validation, manufacturing and delivery of our product candidates to the clinical sites; •delays in having patients complete participation in a study or return for post-treatment follow-up; •clinical trial sites or patients dropping out of a study; •occurrence of serious adverse events associated with a product candidate that are viewed to outweigh its potential benefits; or •changes in regulatory requirements and guidance that require amending or submitting new clinical protocols. Before obtaining marketing approval from regulatory authorities for the sale of our product candidates, we must conduct extensive clinical trials todemonstrate the safety and efficacy of the product candidates in humans. It is impossible to predict when or if any of our clinical trials will demonstratethat product candidates are effective or safe in humans. Because of the nature of the gene therapies we are developing, regulators may also require us to demonstrate long-term gene expression or clinicalefficacy, which may require longer clinical trial periods or longer patient follow-up than is typically required in the case of other therapies.8Table of Contents We or our collaborators may not be able to locate and enroll a sufficient number of eligible patients to participate in these trials as required by theFDA, the EMA or similar regulatory authorities outside the United States and the European Union. This may result in our failure to initiate or continueclinical trials for our product candidates, or may cause us to abandon one or more clinical trials altogether. In particular, because several of our programsare focused on the treatment of patients with orphan diseases, our ability to enroll eligible patients in these trials may be limited or slower than weanticipate in light of the small patient populations involved. For example, we reduced the number of patients enrolled in our second Phase II/III clinicaltrial of Glybera from the 16 patients originally planned to five patients due to slow recruitment. In addition, our potential competitors, including majorpharmaceutical, specialty pharmaceutical and biotechnology companies, academic institutions and governmental agencies and public and private researchinstitutions, may seek to develop competing therapies, which would further limit the small patient pool available for our studies. Any inability to successfully initiate or complete preclinical and clinical development could result in additional costs to us or impair our ability toreceive marketing approval, to generate revenues from product sales, or obtain regulatory and commercialization milestones and royalties. In addition, ifwe make manufacturing or formulation changes to our product candidates, including changes in the vector or manufacturing process used, we may needto conduct additional studies to bridge our modified product candidates to earlier versions. Clinical trial delays could also shorten any periods duringwhich we may have the exclusive right to commercialize our product candidates or allow our competitors to bring products to market before we do,which could impair our ability to successfully commercialize our product candidates and may harm our business and results of operations.Our progress in early-stage clinical trials may not be indicative of long-term efficacy in late-stage clinical trials, and our progress in trials for oneproduct candidate may not be indicative of progress in trials for other product candidates. With the exception of Glybera, the product candidates in our pipeline are at early-stages of development. Study designs and results from previousstudies are not necessarily predictive of our future clinical study designs or results, and initial results may not be confirmed upon full analysis of thecomplete study data. Our product candidates may fail to show the desired safety and efficacy in later stages of clinical development despite havingsuccessfully advanced through initial clinical studies. A number of companies in the pharmaceutical and biotechnology industries have suffered significant setbacks in late-stage clinical trials even afterachieving promising results in early-stage clinical trials. If a larger population of patients does not experience positive results, if these results are notreproducible, or if our products show diminishing activity over time, our products may not receive approval from the EMA or FDA. Data obtainedfrom preclinical and clinical activities are subject to varying interpretations, which may delay, limit or prevent regulatory approval. In addition, we mayencounter regulatory delays or rejections as a result of many factors, including changes in regulatory policy during the period of product development.Failure to confirm favorable results from earlier trials by demonstrating the safety and effectiveness of our products in late stage clinical trials with largerpatient populations could have a material adverse effect on our business that would cause our share price to decline. Progress in trials of Glybera and its approval in the European Union do not indicate that we will make similar progress in additional trials forGlybera or in trials for our other product candidates. While Glybera uses an AAV1 vector for gene delivery, the rest of the product candidates in ourpipeline use other AAV vector variants, such as AAV5 or AAV2. Also, while Glybera is injected directly into the muscles of the leg, the rest of theproducts in our pipeline target other tissues. Due to these variations, trials for our other product candidates may be less successful than the trials forGlybera.9Table of ContentsNegative public opinion and increased regulatory scrutiny of gene therapy and genetic research may damage public perception of Glybera andour product candidates or adversely affect our ability to conduct our business or obtain further marketing approvals for Glybera and marketingapprovals for our product candidates. Public perception may be influenced by claims that gene therapy is unsafe, and gene therapy may not gain the acceptance of the public or themedical community. A generalized public backlash developed against gene therapy following the death in September 1999 of an 18-year-old who hadvolunteered for a gene therapy experiment at the University of Pennsylvania. Researchers at the university had infused the volunteer's liver with a geneaimed at reversing a rare metabolic disease of the liver. The procedure triggered an extreme immune-system reaction that caused multiple-organ failure ina very short time, leading to the first death to occur as a direct result of a gene therapy experiment. In addition, two gene therapy studies in 2003 wereterminated after five subjects developed leukemia. Although none of our current product candidates utilize the gamma-retroviruses used in the 2003 studies, our product candidates do use a viralvector delivery system. The risk of cancer remains a concern for gene therapy and we cannot assure that it will not occur in any of our planned or futureclinical studies. In addition, there is the potential risk of delayed adverse events following exposure to gene therapy products due to persistent biologicalactivity of the genetic material or other components of products used to carry the genetic material. Glybera or our product candidates may prove to have undesirable or unintended side effects, toxicities or other characteristics that may precludeour obtaining additional marketing approval or prevent or limit commercial use. In our clinical development program for Glybera, there were a total of48 serious adverse events, two of which were determined to be related to Glybera, a pulmonary embolism and fever. In our partner's clinicaldevelopment program for AIP, there was one serious adverse event that was determined by the investigator not to be treatment-related. Adverse events in our clinical trials or those conducted by other parties, even if not ultimately attributable to our product candidates, and theresulting publicity could result in increased governmental regulation, unfavorable public perception, failure of the medical community to accept andprescribe gene therapy treatments, potential regulatory delays in the testing or approval of our product candidates, stricter labeling requirements for thoseproduct candidates that are approved and a decrease in demand for any such product candidates. If any such adverse events occur, commercialization ofGlybera or further advancement of our clinical trials could be halted or delayed, which would have a material adverse effect on our business andoperations.Risks Related to the Regulatory Approval of Glybera and Our Product Candidates We cannot predict when or if we will obtain marketing approval to commercialize a product candidate, or in the case of Glybera, furthermarketing approval in jurisdictions outside the EU, and any approval we receive may be for a more narrow indication than we expect. We cannot commercialize a product until the appropriate regulatory authorities have reviewed and approved the product candidate. Thedevelopment and commercialization of our product candidates, including their design, testing, manufacture, safety, efficacy, purity, recordkeeping,labeling, storage, approval, advertising, promotion, sale and distribution, are subject to comprehensive regulation by the EMA and other regulatoryagencies of the member states of the European Union, by the FDA and other regulatory agencies in the United States, and similar regulatory authoritiesoutside the European Union and the United States. Failure to obtain marketing approval for a product candidate in a specific jurisdiction will prevent usfrom commercializing the product candidate in that jurisdiction.10Table of Contents We have not received approval to market any of our products or product candidates from regulatory authorities in the United States. We receivedmarketing authorization for Glybera from the European Commission in October 2012 under exceptional circumstances for a subset of LPLD patients,after our initial application was rejected in June 2011. The FDA does not maintain a regulatory approval process similar to the EMA's marketingauthorization under exceptional circumstances, which may make it more difficult to obtain marketing authorization for Glybera or other productcandidates in the United States. Given the differences between the regulatory schemes for approval of new products in Europe and the United States,approval of Glybera in the European Union does not assure or increase the likelihood of approval of the product in the United States. We plan to file anIND with the FDA for Glybera in the first half of 2014. The results of our prior clinical trials of Glybera will not be sufficient to obtain FDA approval,and the FDA may not ultimately approve Glybera for marketing in the United States. Based on our meetings with the FDA in August and December2013, we believe that to obtain marketing approval for Glybera in the United States, we will need to successfully conduct an adequate and appropriatelycontrolled clinical trial. We have not yet completed the design of this trial or prepared or submitted a protocol for this trial to the FDA. We will seek toamend the protocol for our EU post-approval trial of Glybera so that such trial also could serve as such a trial. The FDA may require preclinical testingor clinical trials beyond this clinical trial as a basis for marketing approval of Glybera, which would be expensive and time consuming. If we fail toobtain marketing approval of Glybera in the United States on our anticipated timeframe, or obtain only limited approval for a specific patient population,our business could be materially adversely affected. The process of obtaining marketing approval for our product candidates in the European Union, the United States and other countries is expensiveand may take many years, if approval is obtained at all. Changes in marketing approval policies during the development period, changes in or theenactment of additional statutes or regulations, or changes in regulatory review for each submitted product application, may cause delays in the approvalor rejection of an application. Regulatory authorities have substantial discretion in the approval process and may refuse to accept any application, maydecide that our data are insufficient for approval, may require additional preclinical, clinical or other studies and may not complete their review in atimely manner. Further, any marketing approval we ultimately obtain may be for only limited indications, or be subject to stringent labeling or otherrestrictions or post-approval commitments that render the approved product not commercially viable. If we experience delays in obtaining marketing approval or fail to obtain approval of Glybera in the United States or elsewhere or of any of ourproduct candidates in the United States or other countries, the commercial prospects for Glybera or our other product candidates may be harmed and ourability to generate revenues will be materially impaired.The FDA will require us to conduct comparability studies evaluating the products manufactured at our Amsterdam facility with those to bemanufactured at our Lexington, Massachusetts facility, which is currently under construction. Those studies and their results could substantiallydelay or preclude our ability to commercialize Glybera and our product candidates in the United States. The FDA maintains strict requirements governing the manufacturing process for biologics. When a manufacturer seeks to modify or change thatprocess, the FDA typically requires the applicant to conduct non-clinical and, depending on the magnitude of the changes, potentially clinicalcomparability studies that evaluate the potential differences in the product resulting from the change in the manufacturing process. In connection withany application we may file with the FDA seeking marketing approval for Glybera or any of our other product candidates in the United States, we willbe required to conduct comparability studies assessing product manufactured at our facility in Amsterdam with product to be manufactured at ourfacility in Lexington, Massachusetts, which we are currently building out and equipping. The FDA may be especially concerned about the need for sucha comparability study for Glybera if the clinical studies on which we rely for approval of our application only involved11Table of Contentsproduct manufactured at our facility in the Netherlands and if we intend to market only product manufactured in Lexington in the United States. Delays in designing and completing a comparability study to the satisfaction of the FDA could delay or preclude our development andcommercialization plans and, thereby, limit our revenues and growth. For example, for Glybera, we may attempt to show comparability of the productmanufactured at the different facilities through the use of non-clinical data, such as potency assays and animal studies. In the event that the FDA doesnot accept such non-clinical comparability data, we may need to conduct a study involving dosing of patients with product from our Lexington facility.That potential study may result in a delay of the approval or launch of Glybera in the United States.We are subject to potentially costly post-approval requirements in the European Union, and any of our product candidates for which we obtainmarketing approval in the future could be subject to similar post-approval or other regulatory requirements. Such requirements may restrict oreliminate the commercial success of Glybera or our other product candidates. Glybera and any of our product candidates for which we obtain marketing approval in the future, as well as the manufacturing process, post-approval studies and measures, labeling, advertising and promotional activities for such products, will be subject to continued requirements of andreview by the FDA, EMA and other regulatory authorities. As part of our marketing approval under exceptional circumstances in the European Union, the EMA has imposed ongoing requirements for apotentially costly post-approval study and market surveillance. Specifically, as a condition to approval of Glybera we are required to complete a post-approval clinical trial and implement a disease registry for long term surveillance of patients, as well as implement risk management procedures,distribute educational materials to healthcare professionals and patients, comply with certain notification obligations and undergo annual reassessment,the outcome of which could eventually lead to a withdrawal of marketing approval for Glybera. The expense and uncertain result of these post-approvalrequirements may delay, limit or terminate our commercialization plan for Glybera and adversely affect our financial position. Non-compliance with EU requirements regarding safety monitoring or pharmacovigilance can also result in significant financial penalties.Similarly, failure to comply with the European Union's requirements regarding the protection of personal information can also lead to significantpenalties and sanctions. Should we receive FDA approval of Glybera or any of our other product candidates in the future the FDA may also impose requirements forcostly post-marketing studies or clinical trials and surveillance to monitor the safety or efficacy of a product. The FDA and other agencies, including theDepartment of Justice, closely regulate and monitor the post-approval marketing and promotion of products to ensure that they are manufactured,marketed and distributed only for the approved indications and in accordance with the provisions of the approved label. Any government investigation of alleged violations of law could require us to expend significant time and resources and could generate negativepublicity. The occurrence of any event or penalty may inhibit our ability or that of our collaborators to commercialize Glybera and any other productsand generate revenues or may lead to withdrawal of marketing approval, which would have a material adverse effect on our business.The risks associated with the marketing approval process are heightened by our products' status as gene therapies. Glybera has been evaluated as a gene therapy by the EMA. We believe that all of our current product candidates, including Glybera, will be viewedas gene therapy products by the EMA, FDA and12Table of Contentsother regulatory authorities. Gene therapies are relatively new treatments and regulators do not have extensive experience or standard review andapproval processes for gene therapies. The FDA has never approved a gene therapy product as safe and effective and, unlike the EMA, does not havean exceptional circumstances approval pathway. The EMA has approved only one gene therapy, Glybera, for a subset of LPLD patients, underexceptional circumstances, and only did so by a vote of 17 to 15 and after twice denying approval. The EMA and FDA have demonstrated caution in their regulation of gene therapy treatments, and ethical and legal concerns about gene therapyand genetic testing may result in additional regulations or restrictions on the development and commercialization of our product candidates that aredifficult to predict. For example, in 2003, the FDA suspended 27 gene therapy trials involving several hundred patients after learning that a child treatedin France had developed a condition resembling leukemia. Although the FDA was not aware that any of the patients treated in the 27 American trialshad suffered illnesses similar to that of the infant in France, it nevertheless took precautions. This temporary halt, the largest such action involving genetherapy trials, was a setback for the field. The FDA and the EMA have issued various guidance documents pertaining to gene therapy products, withwhich we likely must comply to gain regulatory approval of any of our product candidates in the United States or European Union, respectively. Theclose regulatory scrutiny of gene therapy products may result in delays and increased costs, and may ultimately lead to any gene therapy product notbeing approved. Regulatory requirements affecting gene therapy have changed frequently and may continue to change. For example, the European Commissionconducted a public consultation in early 2013 on the application of EU legislation that governs advanced therapy medicinal products, including genetherapy products, that could result in changes in the data we need to submit to the EMA in order for our product candidates to gain regulatory approval.In addition, divergent scientific opinions among the various bodies involved in the review process may result in delays and require additional resourcesand may ultimately result in rejection. For further discussion about the regulation we face in Europe and the United States, please see "Business—Government Regulation and Reimbursement." Agencies at both the U.S. federal and state level, as well as congressional committees and foreign governments, have sometimes expressed interestin further regulating biotechnology. In the United States, the FDA has established the Office of Cellular, Tissue and Gene Therapies within theAgency's Center for Biologics Evaluation and Research, or CBER, to consolidate the review of gene therapy and related products, and the Cellular,Tissue and Gene Therapies Advisory Committee to advise CBER on its review. Gene therapy clinical trials conducted at institutions that receive fundingfor recombinant DNA research from the NIH are also subject to review by the NIH Office of Biotechnology Activities' Recombinant DNA AdvisoryCommittee, or the RAC. Also, before a clinical trial can begin at an NIH-funded institution, that institution's institutional review board, or IRB, and itsInstitutional Biosafety Committee will review the proposed clinical trial to assess the safety of the study. These regulatory agencies, committees and advisory groups and the new regulations and guidelines they promulgate may lengthen the regulatoryreview process, require us to perform additional studies, increase our development costs, lead to changes in regulatory positions and interpretations,delay or prevent approval and commercialization of our product candidates or lead to significant post- approval limitations or restrictions. Delay orfailure to obtain, or unexpected costs in obtaining, the regulatory approval necessary to bring a potential product to market could decrease our ability togenerate sufficient product revenues to maintain our business.13Table of ContentsIf we are not able to obtain or maintain orphan product exclusivity for any of our product candidates for which we seek this status, or if ourcompetitors are able to obtain orphan product exclusivity before we do, we may not be able to obtain approval for our competing products for asignificant period of time. Regulatory authorities in some jurisdictions, including the European Union and the United States, may designate drugs for relatively small patientpopulations as orphan drugs. Generally, if a product with an orphan drug designation subsequently receives the first marketing approval for theindication for which it has such designation, the product is entitled to a period of market exclusivity, which precludes the EMA or FDA from approvinganother marketing application for the same drug for the same indication for that time period. However, the same drug can subsequently be approved forthe same condition if the competent regulatory agency concludes that the later drug is clinically superior in that it is shown to be safer, more effective ormakes a major contribution to patient care.. Orphan drug exclusivity may be lost if the EMA or FDA determines that the request for designation was materially defective, or if themanufacturer is unable to assure sufficient quantity of the drug to meet the needs of patients with the rare disease or condition or if the incidence andprevalence of patients who are eligible to receive the drug in these markets materially increase. We have obtained orphan designation for Glybera in the European Union and the United States. If we lose orphan drug exclusivity for Glybera orif our competitors obtain orphan drug exclusivity in indications related to our other product candidates before we do, we may be precluded fromobtaining marketing authorization or we may lose out on the potential benefits of market exclusivity.Risks Related to the Commercialization of Glybera and Our Product Candidates If we or our collaborators are unable to commercialize Glybera or our other product candidates or experience significant delays in doing so, ourbusiness will be materially harmed. Our ability to generate product revenues will depend heavily on the successful commercialization of Glybera and development and eventualcommercialization of other product candidates. The success of our product candidates will depend on several factors, including the following:•successful completion of preclinical studies and clinical trials; •receipt of marketing approvals from applicable regulatory authorities; •obtaining and maintaining patent and trade secret protection and non-patent, orphan drug exclusivity for our product candidates; •completing the build-out of, and obtaining regulatory approval for, our new manufacturing facility in Lexington, Massachusetts; •launching commercial sales of our products, if and when approved, whether alone or in collaboration with others; •identifying and engaging effective distributors or other third party resellers on acceptable terms in certain jurisdiction where we plan toutilize third parties for the marketing and sale of Glybera or other candidate products; •acceptance of our products, if and when approved, by patients, the medical community and third party payors; •effectively competing with other therapies; •obtaining and maintaining healthcare coverage and adequate reimbursement; and •complying with post-approval requirements of the EMA and maintaining a continued acceptable overall safety profile based on theEMA's risk-benefit analysis.14Table of Contents Failure to achieve or implement any of these elements could result in significant delays or an inability to successfully commercialize Glybera or ourproduct candidates, which could materially harm our business.The affected populations for Glybera and our other product candidates may be smaller than we or third parties currently project, which may affectthe addressable markets for Glybera and our other 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 diseases whohave the potential to benefit from treatment with Glybera or our product candidates, are estimates based on our knowledge and understanding of thesediseases. The total addressable market opportunity for Glybera and our product candidates will ultimately depend upon a number of factors includingthe diagnosis and treatment criteria included in the final label, if approved for sale in specified indications, acceptance by the medical community, patientaccess and product pricing and reimbursement. Prevalence estimates are frequently based on information and assumptions that are not exact and may not be appropriate, and the methodology isforward-looking and speculative. The use of such data involves risks and uncertainties and is subject to change based on various factors. Our estimatesmay prove to be incorrect and new studies may change the estimated incidence or prevalence of the diseases we seek to address. The number of patientswith the diseases we are targeting in the European Union, the United States and elsewhere may turn out to be lower than expected or may not beotherwise amenable to treatment with our products, or new patients may become increasingly difficult to identify or access, all of which wouldadversely affect our results of operations and our business.Glybera, and any other product candidate that receives marketing approval in the future, may fail to achieve the degree of market acceptance byphysicians, patients, third-party payors and others in the medical community necessary for commercial success. Doctors may be reluctant to accept a gene therapy as a treatment option or, where available, choose to continue to rely on existing symptomatictreatments. The degree of market acceptance of Glybera, as well as of any of our product candidates that receive marketing approval in the future, willdepend on a number of factors, including:•the efficacy and potential advantages of our therapies compared with alternative treatments; •our ability to convince payors of the long-term cost-effectiveness of our therapies and, consequently, the availability of third- partycoverage and adequate reimbursement; •the limitations on use and label requirements imposed by regulators; •the convenience and ease of administration of our gene therapies, which in the case of Glybera requires spinal anaesthesia and multipleintramuscular injections, compared to alternative treatments; •the willingness of the target patient population to try new therapies, especially a gene therapy, and of physicians to administer thesetherapies; •the strength of marketing and distribution support; •the prevalence and severity of any side effects; and •any restrictions on the use of our products. In the case of Glybera in the European Union, we are required to put in place a restricted access program to ensure that the product is usedappropriately when the diagnosis is confirmed, mandating that the product only be supplied to doctors who have received the appropriate educationalmaterials15Table of Contentsand only be used to treat patients participating in a registry to monitor the outcome of patients treated with Glybera. If Glybera does not achieve anadequate level of acceptance, we may not generate significant revenues from this product and we may never achieve profitability.If our collaboration with Chiesi is not successful, we may not effectively commercialize Glybera in the European Union and other countriescovered by our partnership with Chiesi. We have entered into a collaboration with Chiesi for the commercialization of Glybera in the European Union, China, Russia and other specifiedcountries. As a result, we are dependent on the efforts of Chiesi to successfully commercialize Glybera in these countries. There is a risk that Chiesi:•may not perform its obligations as expected; •may have difficulties gaining acceptance of the use of Glybera in the clinical community and achieving satisfactory pricing andreimbursement of Glybera; •may terminate, or may elect not to continue or renew, our commercialization arrangements based on changes in its strategic focus oravailable funding, or external factors, such as an acquisition, that divert resources or create competing priorities; and •may not commit sufficient resources to the marketing and distribution of Glybera. In addition, we are required to manufacture Glybera for sale by Chiesi. Should we encounter manufacturing problems, we may fail to adequatelysupply Glybera to Chiesi. If any of these circumstances related to our collaboration with Chiesi are realized, they may adversely affect the commercialsuccess of Glybera in the European Union and other countries covered by our partnership with Chiesi.We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully thanwe do. The development and commercialization of new biotechnology and biopharmaceutical products, including gene therapies, is highly competitive. Wemay face competition with respect to Glybera and our current product candidates, as well as with respect to any product candidates that we may seek todevelop or commercialize in the future, from large and specialty pharmaceutical companies and biotechnology companies worldwide, who currentlymarket and sell products or are pursuing the development of products for the treatment of many of the disease indications for which we are developingour product candidates. Potential competitors also include academic institutions, government agencies and other public and private researchorganizations that conduct research, seek patent protection and establish collaborative arrangements for research, development, manufacturing andcommercialization. In recent years, there has been a significant increase in commercial and scientific interest and financial investment in gene therapy asa therapeutic approach, which has intensified the competition in this area. We are aware of several companies focused on developing gene therapies in various indications, including AGTC, Asklepios, AudentesTherapeutics, BioMarin, bluebird bio, Dimension/Regen X, Oxford BioSciences, Sangamo BioScience, and Spark Therapeutics, as well as severalcompanies addressing other methods for modifying genes and regulating gene expression. We may also face competition with respect to the treatment ofsome of the diseases that we are seeking to target with our gene therapies from protein pharmaceuticals under development at pharmaceutical andbiotechnology companies, including Pfizer, Baxter, Bayer, Novo Nordisk, Genzyme, Shire, BioMarin and Biogen Idec. We must also compete withexisting standards of care, therapies and symptomatic treatments, as well as any new therapies that may become available in the future for the indicationswe are targeting.16Table of Contents Our commercial opportunity could be reduced or eliminated if our competitors develop and commercialize products that are safer, more effective,have fewer or less severe side effects, are more convenient or are less expensive than the products that we develop. Our competitors also may obtainEMA, FDA or other regulatory approval for their products more rapidly than we do, which could result in our competitors establishing a strong marketposition before we are able to enter the market. Because we expect that gene therapy patients may generally require only a single administration, webelieve that the first gene therapy product to enter the market for a particular indication will likely enjoy a significant commercial advantage, and mayalso obtain market exclusivity under applicable orphan drug regimes. Many of the companies with which we are competing or may compete in the future have significantly greater financial resources and expertise thanwe do in research and development, manufacturing, preclinical testing, conducting clinical trials, obtaining regulatory approvals and marketing approvedproducts. Mergers and acquisitions in the pharmaceutical and biotechnology industries may result in more resources being concentrated among a smallernumber of our competitors. Smaller and other early-stage companies may also prove to be significant competitors, particularly through collaborativearrangements with large and established companies. These third parties compete with us in recruiting and retaining qualified scientific and managementpersonnel, establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies complementary to, or necessaryfor, our programs.Risks Related to Our Dependence on Third Parties for Glybera and our Product Pipeline We rely on third parties for important aspects of our development programs. If these parties do not perform successfully or if we are unable tomaintain any of our collaboration arrangements, our business could be adversely affected. We have entered into collaborations with other companies and academic research institutions with respect to important elements of our commercialand development programs. For example, we have collaboration agreements with Chiesi, for both commercialization of Glybera in the European Unionand certain other countries and co-development and commercialization of our hemophilia B program, and development programs with Digna Biotech,Institut Pasteur and UCSF. Our existing collaborations, and any future collaborations we enter into, may pose a number of risks, including the following:•collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations; •in our current collaborations, we generally have limited or no control over the design or conduct of clinical trials sponsored by ourcollaborators; •if our collaborators do not conduct the clinical trials they sponsor in accordance with regulatory requirements or stated protocols, we willnot be able to rely on the data produced in such trials in our further development efforts; •collaborators may not perform their obligations as expected; •collaborators may also have relationships with other entities, some of which may be our competitors; •collaborators may not pursue development and commercialization of any product candidates or may elect not to continue or renewdevelopment or commercialization programs based on clinical trial results, changes in the collaborators' strategic focus or availablefunding, or external factors, such as an acquisition, that divert resources or create competing priorities;17Table of Contents•collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a productcandidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing; •collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our products orproduct candidates if the collaborators believe that competitive products are more likely to be successfully developed or can becommercialized under terms that are more economically attractive than ours; •our collaboration arrangements may impose restrictions on our ability to undertake other development efforts that may appear to beattractive to us; •product candidates discovered in collaboration with us may be viewed by our collaborators as competitive with their own productcandidates or products, which may cause collaborators to cease to devote resources to the commercialization of our product candidates; •a collaborator with marketing and distribution rights to Glybera or one or more of our product candidates that achieve regulatoryapproval may not commit sufficient resources to the marketing and distribution of such product or products; •disagreements with collaborators, including disagreements over proprietary rights, contract interpretation or the preferred course ofdevelopment, could cause delays or termination of the research, development or commercialization of product candidates, lead toadditional responsibilities for us, or result in litigation or arbitration, any of which would be time-consuming and expensive; •collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a wayas to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potentiallitigation; •collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability; and •collaborations may in some cases be terminated for the convenience of the collaborator and, if terminated, we could be required toexpend additional funds to pursue further development or commercialization of the applicable product or product candidates. If our collaborations do not result in the successful development and commercialization of products or if one of our collaborators terminates itsagreement with us, we may not receive future research funding or milestone or royalty payments under the collaboration, and we may lose access toimportant technologies and capabilities of the collaboration. All of the risks relating to product development, regulatory approval and commercializationherein also apply to the activities of our development collaborators.If we are unable to enter into additional collaborations in the future, or if our new collaborations are not successful, we may not be able todevelop or market our product candidates or obtain a strategic position in the development of new gene therapies. We believe collaborations enable us to gain access to early-stage clinical programs and related data, as well as to promising transgenes and otherintellectual property, with limited financial investment by us. Part of our strategy is to leverage our experience and expertise in gene therapy research anddevelopment, as well as our proprietary manufacturing capabilities, to be an attractive collaborator for academic research institutions and biotechnologyand pharmaceutical companies seeking to advance their programs into larger, late-stage clinical trials that require commercial-scale manufacturing. Weface significant competition and we may be unable to attract suitable collaborators or reach agreements with them on acceptable terms, which could limitour access to attractive development programs.18Table of Contents Many of our agreements with our licensors, including our agreements with the NIH, require us to obtain consent from the licensor before we canenter into arrangements involving the sublicensing of technology we have licensed from such licensors. Our licensors may withhold such consent, ormay provide such consent only if we agree to reduce our rights or increase our financial or other obligations to them. Obtaining such consent may alsohamper our ability to enter into collaboration arrangements on a timely basis. If we are unable to reach agreements with suitable collaborators on a timely basis, on acceptable terms, or at all, we may have to curtail thedevelopment of a product candidate, reduce or delay its development or one or more of our other development programs, delay its potentialcommercialization or reduce the scope of any sales or marketing activities, or increase our expenditures and undertake development or commercializationactivities at our own expense. We do not currently have a sales or marketing infrastructure and have no experience in the sale, marketing or distribution of pharmaceuticalproducts. We may not be successful in entering into arrangements with third parties in the future to sell, market and distribute our product candidates,including Glybera in territories outside the European Union and certain other countries, or we may be unable to do so on terms that are favorable to us.We likely will have little control over such third parties, and any of them may fail to devote the necessary resources and attention to sell and market ourproducts effectively.Risks Related to Our Manufacturing Gene therapies, including Glybera, are complex and difficult to manufacture. We could experience production problems that result in delays inour development or commercialization schedules or otherwise adversely affect our business. We manufacture Glybera and clinical supplies of our product candidates ourselves in our facility in Amsterdam and plan to commence productionin the facility we are currently building out in Lexington, Massachusetts. The insect-cell based manufacturing process we use to produce Glybera andour other product candidates is highly complex and in the normal course is subject to production difficulties. Problems with the manufacturing process,even minor deviations from the normal process, could result in product defects or manufacturing failures that result in lot failures, product recalls,product liability claims and insufficient inventory. We may encounter problems achieving adequate or clinical-grade materials that meet EMA, FDA orother applicable standards or specifications with consistent and acceptable production yields and costs. Additionally, a number of factors could causeproduction interruptions, including equipment malfunctions, facility contamination, labor problems, raw material shortages or contamination, naturaldisasters, disruption in utility services, terrorist activities, human error or disruptions in the operations of our suppliers. We also may encounter problems hiring and retaining the experienced specialist personnel needed to operating our manufacturing process, whichcould result in delays in our production or difficulties in maintaining compliance with applicable regulatory requirements. Any problems in our manufacturing process or facilities could make us a less attractive collaborator for academic research institutions and otherparties, which could limit our access to additional attractive development programs, could result in delays in our clinical development or marketingschedules and could harm our business.Delays in completing and receiving regulatory approvals for our new U.S. manufacturing facility could delay our development andcommercialization plans and thereby limit our revenues and growth. We are expending significant funds for the build-out of our leased 53,000 square foot manufacturing facility in Lexington, Massachusetts. Thisproject may result in unanticipated delays and19Table of Contentscost more than expected due to a number of factors, including regulatory requirements. If construction or regulatory approval of our new facility isdelayed, we may not be able to manufacture sufficient quantities of Glybera or our product candidates, which would limit our commercialization anddevelopment activities and our opportunities for growth. Cost overruns associated with this facility could also require us to raise additional funds fromexternal sources, which may be unavailable on favorable terms or at all.Our manufacturing facility in Amsterdam is, and our facility in Lexington will be, subject to significant government regulations and approvals,which are often costly. If we fail to comply with these regulations or maintain these approvals, our business will be materially harmed. Our manufacturing facility in Amsterdam is, and our new facility in Lexington will be, subject to ongoing regulation and periodic inspection by theEMA, FDA and other regulatory bodies to ensure compliance with current Good Manufacturing Practices, or cGMP. Any failure to follow anddocument our adherence to such cGMP regulations or other regulatory requirements may lead to significant delays in the availability of products forcommercial sale or clinical study, may result in the termination of or a hold on a clinical study, or may delay or prevent filing or approval of marketingapplications for our products. Failure to comply with applicable regulations could also result in the EMA, FDA or other applicable authorities taking various actions, includinglevying fines and other civil penalties; imposing consent decrees or injunctions; requiring us to suspend or put on hold one or more of our clinical trials;suspending or withdrawing regulatory approvals; delaying or refusing to approve pending applications or supplements to approved applications;requiring us to suspend manufacturing activities or product sales, imports or exports; requiring us to communicate with physicians and other customersabout concerns related to actual or potential safety, efficacy, and other issues involving our products; mandating product recalls or seizing products;imposing operating restrictions; and seeking criminal prosecutions. Any of the foregoing could materially harm our business.Our use of viruses, chemicals and other hazardous materials requires us to comply with regulatory requirements and exposes us to significantpotential liabilities. Our development and manufacturing processes involve the use of viruses, chemicals and other hazardous materials, and produce waste products.Accordingly, we will be subject to federal, state and local laws and regulations in the United States, and are subject to comparable regulations in theNetherlands, governing the use, manufacture, distribution, storage, handling, treatment and disposal of these materials. In addition to ensuring the safehandling of these materials, applicable requirements require increased safeguards and security measures for many of these agents, including controllingaccess and screening of entities and personnel who have access to them, and establishing a comprehensive national database of registered entities. In theevent of an accident or failure to comply with environmental, occupational health and safety and export control laws and regulations, we could be heldliable for damages that result, and any such liability could exceed our assets and resources.Risks Related to Our Intellectual Property We license intellectual property from third parties, and such licenses may not provide adequate rights, may not be available in the future oncommercially reasonable terms or at all, or our licensors may be unable to obtain and maintain patent protection for the technology or productsthat we license from them. We currently are heavily reliant upon licenses of proprietary technology from third parties that is important or necessary to the development of ourtechnology and products, including technology related to our manufacturing process, our vector platform, our gene cassettes and the therapeutic genesof interest we are using. These and other licenses may not provide adequate rights to use such20Table of Contentstechnology in all relevant fields of use. Licenses to additional third-party technology that may be required for our development programs may not beavailable in the future or may not be available on commercially reasonable terms, which could have a material adverse effect on our business andfinancial condition. In some circumstances, we may not have the right to control the preparation, filing and prosecution of patent applications, or to maintain thepatents, covering technology that we license from third parties. In addition, some of our agreements with our licensors require us to obtain consent fromthe licensor before we can enforce patent rights, and our licensor may withhold such consent or may not provide it on a timely basis. Therefore, wecannot be certain that these patents and applications will be prosecuted and enforced in a manner consistent with the best interests of our business. Inaddition, if third parties who license patents to us fail to maintain such patents, or lose rights to those patents, the rights we have licensed may bereduced or eliminated.Our intellectual property licenses with third parties may be subject to disagreements over contract interpretation, which could narrow the scope ofour rights to the relevant intellectual property or technology or increase our financial or other obligations to our licensors. The agreements under which we currently license intellectual property or technology from third parties are complex, and certain provisions in suchagreements may be susceptible to multiple interpretations. The resolution of any contract interpretation disagreement that may arise could narrow whatwe believe to be the scope of our rights to the relevant intellectual property or technology, or increase what we believe to be our financial or otherobligations under the relevant agreement, either of which could have a material adverse effect on our business and financial condition. For example, wehave an exclusive license from the NIH for "the development and sale of AAV5 based therapeutic products to be delivered to the brain or liver fortreatment of human diseases originating in the brain or liver," other than arthritis-related diseases. We also have a non-exclusive license from the NIHfor the development and sale of AAV5 based therapeutic products to treat human diseases other than those covered by our exclusive license. We believe that our exclusive license from the NIH includes the systemic administration of AAV5-based therapeutic products so long as suchtherapeutic products are "to be delivered to the brain or liver for treatment of human diseases originating in the brain or liver." However, SangamoBioSciences, Inc., or Sangamo, has announced that it has broad worldwide licenses to use AAV vectors, including AAV5 and AAV6, for research,development and commercialization of therapies for hemophilia A and B, Huntington's disease and other targets. We believe Sangamo's view may bethat our exclusive license excludes systemic administration because Sangamo interprets the phrase "to be delivered to" to require direct administrationinto the brain or liver. Our view is that the phrase "to be delivered to" indicates the ultimate destination of the therapy and not the location where it isfirst introduced into the body. Although we think our interpretation is correct, there can be no assurance that a court would agree with our interpretationregarding the meaning of this phrase. If our interpretation of the phrase "to be delivered to" is incorrect, then others may obtain licenses from the NIHthat may enable them to compete with us in the systemic administration of AAV5-based therapeutics for treatment of human diseases originating in thebrain or liver, which could harm our business.If we fail to comply with our obligations in our intellectual property licenses with third parties, we could lose rights that are important to ourbusiness. We in-license intellectual property from third parties that is material to Glybera and all of our product candidates, including technology related toour manufacturing process, our vector platform, and the therapeutic gene cassettes we are using. Our licensing arrangements with third parties imposediligence, development and commercialization timelines, milestone payment, royalty, insurance and21Table of Contentsother obligations on us. If we fail to comply with these obligations, our counterparties may have the right to terminate these agreements, in which casewe might not be able to develop, manufacture or market any product that is covered by these agreements or may face other penalties under theagreements. Such an occurrence could materially adversely affect the value of the product candidate being developed under any such agreement.Termination of these agreements or reduction or elimination of our rights under these agreements may result in our having to negotiate new or amendedagreements with less favorable terms, or cause us to lose our rights under these agreements, including our rights to important intellectual property ortechnology.If we are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection is notsufficiently broad, our ability to successfully commercialize our products may be impaired. We rely upon a combination of in-licensed and owned patents, trade secret protection and confidentiality agreements to protect our intellectualproperty. Our success depends in large part on our ability to obtain and maintain this protection in the European Union, the United States and othercountries, in part by filing patent applications related to our novel technologies and product candidates. Our patents may not provide us with anymeaningful commercial protection, prevent competitors from competing with us or otherwise provide us with any competitive advantage. Ourcompetitors may be able to circumvent our owned or licensed patents by developing similar or alternative technologies or products in a non-infringingmanner. Successful challenges to our patent may result in loss of exclusivity or freedom to operate or in patent claims being narrowed, invalidated orheld unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar or identical technology andproducts, or limit the duration of the patent protection of our technology and products. The patent prosecution process is expensive, time-consuming and uncertain, and we may not be able to file and prosecute all necessary or desirablepatent applications at a reasonable cost or in a timely manner. It is also possible that we will fail to identify patentable aspects of our research anddevelopment output before it is too late to obtain patent protection. Additionally, given the amount of time required for the development, testing andregulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized.As a result, our owned and licensed patent portfolio may not provide us with sufficient rights to exclude others from commercializing products similaror identical to ours. The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves complex legal and factual questions andhas in recent years been the subject of much litigation. In addition, the laws of foreign countries may not protect our rights to the same extent as the lawsof the United States. For example, EU patent law with respect to the patentability of methods of treatment of the human body is more limited than U.S.law. Publications of discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the United States and otherjurisdictions are typically not published until 18 months after their priority date, or in some cases at all. Therefore, we cannot know with certaintywhether we were the first to make the inventions or that we were the first to file for patent protection of the inventions claimed in our owned or licensedpatents or pending patent applications. As a result, the issuance, scope, validity, enforceability and commercial value of our patent rights are highlyuncertain. Our pending and future patent applications may not result in patents being issued that protect our technology or products, in whole or in part,or which effectively prevent others from commercializing competitive technologies and products. Changes in either the patent laws or interpretation ofthe patent laws in the European Union, the United States or other countries may diminish the value of our patents or narrow the scope of our patentprotection.22Table of ContentsWe may become involved in lawsuits to protect or enforce our patents or other intellectual property or third parties may assert their intellectualproperty rights against us, which could be expensive, time consuming and unsuccessful. Competitors may infringe our owned or licensed patents or other intellectual property. To counter infringement or unauthorized use, we may berequired to file infringement claims, which can be expensive and time consuming. An adverse result in any litigation proceeding could put one or moreof our patents at risk of being invalidated or interpreted narrowly. Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses,increase our operating losses, reduce available resources and could distract our technical and management personnel from their normal responsibilities.In addition, there could be public announcements of the results of hearings, motions or other interim proceedings or developments, which could have anadverse effect on the price of our ordinary shares. Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would beuncertain and could have a material adverse effect on the success of our business. If we are found to infringe a third party's intellectual property rights,we could be required to obtain a license from such third party to continue developing and marketing our products and technology. We may not be ableto obtain the required license on commercially reasonable terms or at all. Even if we were able to obtain a license, it could be non-exclusive, therebygiving our competitors access to the same technologies licensed to us. We could be forced, including by court order, to cease commercializing theinfringing technology or product. In addition, we could be found liable for monetary damages, including treble damages and attorneys' fees if we arefound to have wilfully infringed a patent. A finding of infringement could prevent us from commercializing our product candidates or force us to ceasesome of our business operations, which could materially harm our business. Claims that we have misappropriated the confidential information or tradesecrets of third parties could have a similar negative impact on our business.Our reliance on third parties may require us to share our trade secrets, which could increase the possibility that a competitor will discover them orthat our trade secrets will be misappropriated or disclosed. Because we collaborate with various organizations and academic research institutions on the advancement of our gene therapy platform, we must,at times, share trade secrets with them. We seek to protect our proprietary technology in part by entering into confidentiality agreements and, ifapplicable, materials transfer agreements, collaborative research agreements, consulting agreements or other similar agreements with our collaborators,advisors and consultants prior to beginning research or disclosing proprietary information. These agreements typically limit the rights of the third partiesto use or disclose our confidential information, such as trade secrets. Despite the contractual provisions employed when working with third parties, theneed to share trade secrets and other confidential information increases the risk that such trade secrets become known by our competitors, areinadvertently incorporated into the technology of others, or are disclosed or used in violation of these agreements. Given that our proprietary position isbased, in part, on our know-how and trade secrets, a competitor's discovery of our trade secrets or other unauthorized use or disclosure would impairour competitive position and may have a material adverse effect on our business. In addition, these agreements typically restrict the ability of our collaborators, advisors and consultants to publish data potentially relating to ourtrade secrets. Our academic collaborators typically have rights to publish data, provided that we are notified in advance and may delay publication for aspecified time in order to secure our intellectual property rights arising from the collaboration. In other cases, publication rights are controlledexclusively by us, although in some cases we may share these rights with other parties. We also conduct joint research and development23Table of Contentsprograms that may require us to share trade secrets under the terms of our research and development partnerships or similar agreements. Some courts inside and outside the United States are less willing or unwilling to protect trade secrets. If any of our trade secrets were to belawfully obtained or independently developed by a competitor, we would have no right to prevent them, or those to whom they communicate it, fromusing that technology or information to compete with us.Risks Related to Pricing and Reimbursement We face uncertainty related to insurance coverage of and pricing and reimbursement for Glybera and any product candidates for which we mayreceive marketing approval. We anticipate that the cost of treatment using Glybera or our other product candidates will be significant. We expect that most patients and theirfamilies will not be capable of paying for our products themselves. There will be no commercially viable market for Glybera or our other productcandidates without reimbursement from third-party payors, such as government health administration authorities, private health insurers and otherorganizations. Even if there is a commercially viable market, if the level of third-party reimbursement is below our expectations, our revenues and grossmargins will be adversely affected and our business will be harmed. Government authorities and other third party payors, such as private health insurers and health maintenance organizations, decide whichmedications they will pay for and establish reimbursement levels. Reimbursement systems vary significantly by country and by region, andreimbursement approvals must be obtained on a country-by-country basis. Government authorities and third party payors have attempted to controlcosts by limiting coverage and the amount of reimbursement for particular medications and procedures. Increasingly, third party payors require drugcompanies to provide them with predetermined discounts from list prices, are exerting influence on decisions regarding the use of particular treatmentsand are limiting covered indications. Additionally, in the United States and some foreign jurisdictions, legislative and regulatory changes regarding thehealthcare system and insurance coverage could result in more rigorous coverage criteria and downward pressure on drug prices, and may affect ourability to profitably sell any products for which we obtain marketing approval. The pricing review period and pricing negotiations for new medicines take considerable time and have uncertain results. Pricing review andnegotiation often begins only after the receipt of regulatory marketing approval, and some authorities require approval of the sale price of a productbefore it can be marketed. In some markets, particularly the countries of the European Union, prescription pharmaceutical pricing remains subject tocontinuing direct governmental control and to drug reimbursement programs even after initial approval is granted and price reductions may be imposed.Prices of medical products may also be subject to varying price control mechanisms or limitations as part of national health systems if products areconsidered not cost-effective or where a drug company's profits are deemed excessive. In addition, pricing and reimbursement decisions in certaincountries can lead to mandatory price reductions or additional reimbursement restrictions in other countries. As a result of these restrictions, Glybera, aswell as any product candidates for which we may obtain marketing approval in the future, may be subject to price regulations that delay or prohibit ouror our partners' commercial launch of the product. In addition, we or our collaborators may elect to reduce the price of our products in order to increasethe likelihood of obtaining reimbursement approvals. In the event that countries impose prices which are not sufficient to allow us or our collaboratorsto generate a profit, we or our collaborators may refuse to launch the product in such countries or withdraw the product from the market. If pricing is setat unsatisfactory levels, or if the price decreases, our business could be harmed, possibly materially. If we fail to obtain and sustain an adequate level of24Table of Contentscoverage and reimbursement for our products by third party payors, our ability to market and sell our products would be adversely affected and ourbusiness would be harmed.Due to the generally limited addressable market for our target orphan indications and the potential for Glybera and our product candidates tooffer therapeutic benefit in a single administration, we face uncertainty related to pricing and reimbursement for these product candidates. The relatively small market size for orphan indications and the potential for long-term therapeutic benefit from a single administration presentparticular challenges to pricing review and negotiation for Glybera and our product candidates for which we may obtain marketing authorization. Thepatient populations for Glybera and our product candidates targeted at orphan disease are relatively small. If we are unable to obtain adequate levels ofreimbursement relative to the small market size in our target orphan indications, our ability to support our development and commercial infrastructureand to successfully market and sell Glybera and other product candidates for which we may obtain marketing approval will be adversely affected. We also anticipate that Glybera and many or all of our gene therapy product candidates may provide long-term, and potentially curative benefit witha single administration. This is a different paradigm than that of other pharmaceutical therapies, which often require an extended course of treatment orfrequent administration. As a result, governments and other payors may be reluctant to provide the significant level of reimbursement that we seek at thetime of administration of our gene therapies or may seek to tie reimbursement to clinical evidence of continuing therapeutic benefit over time. Althoughwe anticipate that Glybera will need to be administered only once, there may be situations in which we may need to readminister Glybera, which mayfurther complicate the pricing and reimbursement for Glybera. In addition, in light of the anticipated cost of these therapies, governments and otherpayors may be particularly restrictive in making coverage decisions. These factors could limit our commercial success and harm our business.Risks Related to Other Legal Compliance Matters Our relationships with customers and third-party payors will be subject to applicable anti-kickback, fraud and abuse and other healthcare lawsand regulations, which could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm and diminished profits andfuture earnings. Healthcare providers, physicians and third-party payors will play a primary role in the recommendation and prescription of any products for whichwe obtain marketing approval. Our future arrangements with third-party payors and customers may expose us to broadly applicable fraud and abuse andother healthcare laws and regulations that may constrain the business or financial arrangements and relationships through which we market, sell anddistribute any products for which we obtain marketing approval. Within the European Union, the control of unlawful marketing activities is a matter of national law in each of the member states. Industryassociations also closely monitor the activities of member companies. Similarly, failure to comply with the European Union's requirements regarding theprotection of personal information can also lead to significant penalties and sanctions. If we market a product in the United States in the future, we will be subject to various federal and state laws and regulations including, the federalAnti-Kickback Statute, the federal False Claims Act, the federal Health Insurance Portability and Accountability Act of 1996, federal law that requiresapplicable manufacturers of covered drugs to report payments and other transfers of value to physicians and teaching hospitals, the U.S. ForeignCorrupt Practices Act and certain state and local laws applicable to pharmaceutical companies.25Table of Contents Efforts to ensure that our business arrangements with third parties will comply with applicable laws and regulations will involve substantial costs.If our operations, or the activities of our collaborators, distributors or other third-party agents are found to be in violation of any of these laws or anyother governmental regulations that may apply to us, we may be subject to significant civil, criminal and administrative penalties, damages, fines,imprisonment, exclusion of products from government funded healthcare programs and the curtailment or restructuring of our operations.If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs thatcould harm our business. We are subject to numerous environmental, health and safety laws and regulations, including those governing laboratory procedures and thehandling, use, storage, treatment and disposal of hazardous materials and wastes. Our operations involve the use of hazardous and flammable materials,including chemicals and biological materials. Our operations also produce hazardous waste products. We cannot eliminate the risk of contamination orinjury from these materials. In the event of contamination or injury resulting from our use of hazardous materials, we could be held liable for anyresulting damages, and any liability could exceed our resources. We also could incur significant costs associated with civil or criminal fines andpenalties for failure to comply with such laws and regulations. Although we maintain employer's liability insurance to cover us for costs and expenses we may incur due to injuries to our employees resultingfrom the use of hazardous materials, this insurance may not provide adequate coverage against potential liabilities. We do not maintain insurance forenvironmental liability or toxic tort claims that may be asserted against us in connection with our storage or disposal of biological, hazardous orradioactive materials. In addition, we may incur substantial costs in order to comply with current or future environmental, health and safety laws and regulations. Thesecurrent or future laws and regulations may impair our research, development or production efforts. Our failure to comply with these laws andregulations also may result in substantial fines, penalties or other sanctions.Product liability lawsuits against us could cause us to incur substantial liabilities and to limit commercialization of Glybera and any products thatwe may develop in the future. We face an inherent risk of product liability related to the testing of our product candidates in human clinical trials and will face an even greater riskwhen we commercially sell Glybera and any other products that we may develop. If we cannot successfully defend ourselves against claims that ourproduct candidates or products caused injuries, we will incur substantial liabilities. Regardless of merit or eventual outcome, liability claims may resultin:•decreased demand for any product candidates or products that we develop or sell; •injury to our reputation and significant negative media attention; •negative publicity or public opinion surrounding gene therapy; •withdrawal of clinical trial participants; •significant costs to defend the related litigation; •substantial monetary awards to trial participants or patients; •loss of revenue; •reduced resources of our management to pursue our business strategy; and •the inability to further develop or commercialize any products that we develop.26Table of Contents We currently hold €6,000,000 in clinical trial insurance coverage in the aggregate, with a per incident limit of €400,000 to €450,000, with respectto the clinical studies we conduct. Such coverage may not be adequate to cover all liabilities that we may incur. We may need to increase our insurancecoverage as we expand our clinical trials and commercialize Glybera. In addition, insurance coverage is increasingly expensive. We may not be able tomaintain insurance coverage at a reasonable cost or in an amount adequate to satisfy any liability that may arise.Risks Related to Employee Matters and Managing Growth Our future success depends on our ability to retain key executives and technical staff and to attract, retain and motivate qualified personnel. We are highly dependent on the research and development, clinical and business development expertise of our Chief Executive Officer, JörnAldag, our Chief Medical Officer, Christian Meyer, M.D., and our Chief Scientific Officer, Harald Petry, as well as the other principal members of ourmanagement, scientific and clinical team. Although we have entered into employment agreements with our senior management, each of them mayterminate their employment on relatively short notice. We do not maintain "key person" insurance for any of our senior management or employees. The loss of the services of our senior management or other key employees could impede the achievement of our research, development andcommercialization objectives and seriously harm our ability to successfully implement our business strategy. Furthermore, replacing senior managementand key employees may be difficult and may take an extended period of time because of the limited number of individuals in our industry with thebreadth and depth of skills and experience required to successfully develop, gain regulatory approval of and commercialize gene therapy products.Competition to hire from this limited pool is intense, and we may be unable to hire, train, retain or motivate these key personnel on acceptable terms. If we are unable to continue to attract and retain high quality personnel, our ability to pursue our growth strategy will be limited.We plan to expand our key capabilities and, as a result, may encounter difficulties in managing our growth, which could disrupt our operations.If we are unable to establish such capabilities we may not be successful in commercializing Glybera or our other product candidates in the UnitedStates or other countries, even if we receive marketing approval. If we receive marketing approval, we intend to build a sales, marketing and medical affairs infrastructure to market Glybera and potentially otherproduct candidates in the United States and other countries. We currently have no experience building and training an internal sales force. We expect inthe future to experience significant growth in the number of our employees and the scope of our operations, particularly in the areas of manufacturing,clinical development, regulatory affairs and sales, marketing and distribution. To manage our anticipated future growth, we will be required toimplement and improve our managerial, operational and financial systems, expand our facilities and recruit and train additional qualified personnel.Recruiting and training a sales force is expensive and time-consuming and could delay any ultimate launch of Glybera or other product candidates forwhich we are able to obtain marketing approval in the United States and other markets. Due to our limited financial resources and the limited experienceof our management team in running a company with this level of anticipated growth, we may not be able to effectively manage the expansion of ouroperations or recruit and train additional qualified personnel. If we do not successfully establish sales, marketing and medical affairs capabilities, eitheron our own or in collaboration with third parties, we will not be successful in commercializing Glybera or other product candidates in the United Statesor other countries in which we may receive marketing approval.27Table of ContentsRisks Related to our Ordinary Shares The price of our ordinary shares has been and may in the future be volatile and fluctuate substantially. Our share price has been and may in the future be volatile. Since initial trading of our ordinary shares began on the NASDAQ Global SelectMarket on February 4, 2013 through April 23, 2014, the sale price of our ordinary shares has ranged from a high of $18.05 to a low of $8.90. Thestock market in general and the market for smaller biopharmaceutical companies in particular have experienced extreme volatility that has often beenunrelated to the operating performance of particular companies. The market price for our ordinary shares may be influenced by many factors, including:•the success of competitive products or technologies; •results of clinical trials of our product candidates or those of our competitors; •public perception of gene therapy; •regulatory delays and greater government regulation of potential products due to adverse events; •regulatory or legal developments in the European Union, the United States and other countries; •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; •the results of our efforts to discover, develop, acquire or in- license additional product candidates or products; •actual or anticipated 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; and •general economic, industry and market conditions.An active trading market for our ordinary shares may not be sustained. Although our ordinary shares are listed on The NASDAQ Global Select Market, an active trading market for our shares may not be sustained. Ifan active market for our ordinary shares does not continue, it may be difficult for our shareholders to sell their shares without depressing the marketprice for the shares or sell their shares at all. Any inactive trading market for our ordinary shares may also impair our ability to raise capital to continueto fund our operations by selling shares and may impair our ability to acquire other companies or technologies by using our shares as consideration. Wehave no plans to list our ordinary shares in any other jurisdiction. As a result, a holder of our ordinary shares outside the United States may not be ableto effect transactions in our ordinary shares as readily as the holder may if our securities were listed on an exchange in that holder's home jurisdiction.Our senior managers, directors and major shareholders, if they choose to act together, will continue to have the ability to control all matterssubmitted to shareholders for approval. Our management board and supervisory board members, senior management, and our shareholders and their affiliates who own more than 5% ofour outstanding ordinary shares, in the aggregate, beneficially own approximately 50% of our share capital. As a result, if these shareholders28Table of Contentswere to choose to act together, they would be able to control all matters submitted to our shareholders for approval, as well as our management andaffairs. For example, these persons, if they choose to act together, would control the election of supervisory board directors and approval of any merger,consolidation or sale of all or substantially all of our assets.Provisions of our articles of association or Dutch corporate law might deter acquisition bids for us that might be considered favorable and preventor frustrate any attempt to replace or remove the management board and supervisory board. Certain provisions of our articles of association may make it more difficult for a third party to acquire control of us or effect a change in ourmanagement board or supervisory board. These provisions include:•staggered three-year terms of our supervisory directors; •a provision that our managing directors and supervisory directors may only be removed at a general meeting of shareholders by a two-thirds majority of votes cast representing more than half of the issued share capital of the company (unless the removal was proposed bythe supervisory board); and •a requirement that certain matters, including an amendment of our articles of association, may only be brought to our shareholders for avote upon a proposal by our management board that has been approved by our supervisory board.We do not expect to pay dividends in the foreseeable future. We have not paid any dividends since our incorporation. Even if future operations lead to significant levels of distributable profits, we currentlyintend that earnings, if any, will be reinvested in our business and that dividends will not be paid until we have an established revenue stream to supportcontinuing dividends. Under Dutch law, we may only pay dividends if our shareholders' equity exceeds the sum of the paid-up and called-up sharecapital plus the reserves required to be maintained by Dutch law or by our articles of association. In addition, our loan agreement with Herculescontains, and any other loan facilities that we may enter into may contain, restrictions on our ability, or that of our subsidiaries, to pay dividends. Subjectto such restrictions, a proposal for the payment of cash dividends in the future, if any, will be at the discretion of our management board, subject to theapproval of our supervisory board, and will depend upon such factors as earnings levels, capital requirements, contractual restrictions, our overallfinancial condition and any other factors deemed relevant by our management board. Accordingly, shareholders cannot rely on dividend income fromour ordinary shares and any returns on an investment in our ordinary shares will likely depend entirely upon any future appreciation in the price of ourordinary shares.The sale of a substantial number of our ordinary shares following expiration of lockup agreements to which certain shares are subject, maycause the market price of our ordinary shares to decline. Sales of a substantial number of shares in the public market may occur at any time after the expiration of the lock-up agreements entered into bycertain shareholders in connection with our recent initial public offering. The sale or the resale by our shareholders of such shares, or a marketexpectation of such sales, may cause the market price of our ordinary shares to decline. Currently, 13,434,612 ordinary shares, or 76% of ouroutstanding shares, are currently restricted as a result of securities laws or lock-up agreements but will be able to be sold, subject to any applicablevolume limitations under U.S. federal securities laws with respect to affiliate sales and subject to the expiration of the relevant lock-up agreements, in thefuture.29Table of ContentsWe are an "emerging growth company," and the reduced disclosure requirements applicable to emerging growth companies may make ourordinary shares less attractive to investors. We are an "emerging growth company," as defined in the Jumpstart Our Business Startups Act of 2012, or the JOBS Act, and may remain anemerging growth company for up to five years. For so long as we remain an emerging growth company, we are permitted and intend to rely onexemptions from certain disclosure requirements that are applicable to other public companies that are not emerging growth companies. Theseexemptions include:•not being required to comply with the auditor attestation requirements in the assessment of our internal control over financial reporting;and •not being required to comply with any requirement that may be adopted by the Public Company Accounting Oversight Board regardingmandatory audit firm rotation or a supplement to the auditor's report providing additional information about the audit and the financialstatements; 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 share price may be more volatile.We are a foreign private issuer and, as a result, we are not subject to U.S. proxy rules and are subject to Exchange Act reporting obligations that,to some extent, are more lenient and less frequent than those of a U.S. domestic public company. We report under the Securities Exchange Act of 1934, as amended, or the Exchange Act, as a non-U.S. company with foreign private issuer status.Because we qualify as a foreign private issuer under the Exchange Act and although we are subject to Dutch laws and regulations with regard to suchmatters and intend to furnish quarterly financial information to the SEC, we are exempt from certain provisions of the Exchange Act that are applicableto U.S. domestic public companies, including:•the sections of the Exchange Act regulating the solicitation of proxies, consents or authorizations in respect of a security registered underthe Exchange Act; •the sections of the Exchange Act requiring insiders to file public reports of their stock ownership and trading activities and liability forinsiders who profit from trades made in a short period of time; and •the rules under the Exchange Act requiring the filing with the Securities and Exchange Commission of quarterly reports on Form 10-Qcontaining unaudited financial and other specified information, or current reports on Form 8-K, upon the occurrence of specifiedsignificant events. In addition, foreign private issuers are not be required to file their annual report on Form 20-F until 120 days after the end of each fiscal year, whileU.S. domestic issuers that are accelerated filers are required to file their annual report on Form 10-K within 75 days after the end of each fiscal year.Foreign private issuers are also exempt from the Regulation Fair Disclosure, aimed at preventing issuers from making selective disclosures of materialinformation. As a result of the above, our shareholders may not have the same protections afforded to shareholders of companies that are not foreignprivate issuers. We cannot predict whether investors will find our ordinary shares less attractive if we rely on these exemptions. If some investors findour ordinary shares less attractive as a result, there may be a less active trading market for our ordinary shares and our share price may be more volatile.30Table of ContentsWe may lose our foreign private issuer status which would then require us to comply with the Exchange Act's domestic reporting regime andcause us to incur significant legal, accounting and other expenses. If we lose foreign private issuer status we would be required to comply with the Exchange Act reporting and other requirements applicable to U.S.domestic issuers, which are more detailed and extensive than the requirements for foreign private issuers. We may also be required to make changes inour corporate governance practices in accordance with various SEC and NASDAQ rules. The regulatory and compliance costs to us under U.S.securities laws if we are required to comply with the reporting requirements applicable to a U.S. domestic issuer may be significantly higher than thecost we would incur as a foreign private issuer. As a result, we expect that any loss of foreign private issuer status would increase our legal andfinancial compliance costs and would make some activities more time consuming and costly. We also expect that if we were required to comply with therules and regulations applicable to U.S. domestic issuers, it would make it more difficult and expensive for us to obtain director and officer liabilityinsurance, and we may be required to accept reduced coverage or incur substantially higher costs to obtain coverage. These rules and regulations couldalso make it more difficult for us to attract and retain qualified members of our supervisory board.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, we will incur significant legal, accounting and otherexpenses that we did not incur as a private company. We currently estimate that we will incur incremental annual costs of approximately $1.5 millionassociated with operating as a public company, although it is possible that our actual incremental annual costs will be higher than we currently estimate.The Sarbanes-Oxley Act of 2002, the Dodd-Frank Wall Street Reform and Consumer Protection Act, the listing requirements of The NASDAQ GlobalSelect Market and other applicable securities rules and regulations impose various requirements on public companies, including establishment andmaintenance of effective disclosure and financial controls and corporate governance practices. Our management and other personnel will need to devotea substantial amount of time to these compliance initiatives. 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 and controlenvironment process improvements.If we fail to implement and maintain an effective system of internal controls, we may be unable to accurately report our results of operations orprevent fraud or fail to meet our reporting obligations, and investor confidence and the market price of our ordinary shares may be materially andadversely affected. Prior to our initial public offering that closed February 10, 2014, we were a private company with limited accounting personnel and other resourceswith which to address our internal controls and procedures. Our independent registered public accounting firm has not conducted an audit of ourinternal control over financial reporting. However, in connection with the audit of our consolidated financial statements as of and for year endedDecember 31, 2012 and 2013, we and our independent registered public accounting firm identified three material weaknesses in our internal controlover financial reporting. A significant deficiency is a control deficiency, or a combination of control deficiencies, that adversely affects our ability toinitiate, authorize, record, process, or report external financial data reliably in accordance with IFRS such that there is more than a remote likelihood thata misstatement of our annual or interim financial statements that is more than inconsequential will not be prevented or detected by our employees. Amaterial weakness is a significant deficiency, or combination of significant deficiencies, that results in more than a remote likelihood that a material31Table of Contentsmisstatement of our annual or interim financial statement will not be prevented or detected by our employees. In response, we have begun the process ofevaluating our internal control over financial reporting. We have also taken several remedial actions to address these material weaknesses. For details,see "Operating and Financial Review and Prospects—Internal Control Over Financial Reporting." Furthermore, it is possible that, had our independent registered public accounting firm conducted an audit of our internal control over financialreporting, such firm might have identified additional material weaknesses and deficiencies. As a public company in the United States we are subject tothe Sarbanes-Oxley Act of 2002. Section 404 of the Sarbanes-Oxley Act of 2002, or Section 404, will require that we include a report of managementon our internal control over financial reporting in our annual report on Form 20-F beginning with our annual report for the fiscal year endingDecember 31, 2015. In addition, once we cease to be an "emerging growth company" as such term is defined in the JOBS Act, our independentregistered public accounting firm must attest to and report on the effectiveness of our internal control over financial reporting. Our management mayconclude that our internal control over financial reporting is not effective. Moreover, even if our management concludes that our internal control overfinancial reporting is effective, our independent registered public accounting firm, after conducting its own independent testing, may issue a report that isqualified if it is not satisfied with our internal controls or the level at which our controls are documented, designed, operated or reviewed, or if itinterprets the relevant requirements differently from us. In addition, our reporting obligations may place a significant strain on our management,operational and financial resources and systems for the foreseeable future. We may be unable to timely complete our evaluation, testing and any requiredremediation. During the course of documenting and testing our internal control procedures in order to satisfy the requirements of Section 404, we may identifyother weaknesses and deficiencies in our internal control over financial reporting. In addition, if we fail to maintain the adequacy of our internal controlover financial reporting, as these standards are modified, supplemented or amended from time to time, we may not be able to conclude on an ongoingbasis that we have effective internal control over financial reporting in accordance with Section 404. If we fail to achieve and maintain an effectiveinternal control environment, we could experience material misstatements in our financial statements and fail to meet our reporting obligations, whichwould likely cause investors to lose confidence in our reported financial information. This could in turn limit our access to capital markets, harm ourresults of operations, and lead to a decline in the trading price of our ordinary shares. Additionally, ineffective internal control over financial reportingcould expose us to increased risk of fraud or misuse of corporate assets and subject us to potential delisting from The NASDAQ Global Select Market,regulatory investigations and civil or criminal sanctions. We may also be required to restate our financial statements for prior periods.We rely on NASDAQ Stock Market rules that permit us to comply with applicable Dutch corporate governance practices, rather than thecorresponding domestic U.S. corporate governance practices, and therefore the rights of our shareholders will differ from the rights ofshareholders of a domestic U.S. issuer. As a foreign private issuer whose ordinary shares are listed on The NASDAQ Global Select Market, we are permitted in certain cases to followDutch corporate governance practices instead of the corresponding requirements of the NASDAQ Marketplace Rules. A foreign private issuer thatelects to follow a home country practice instead of NASDAQ requirements must submit to NASDAQ in advance a written statement from anindependent counsel in such issuer's home country certifying that the issuer's practices are not prohibited by the home country's laws. In addition, aforeign private issuer must disclose in its annual reports filed with the Securities and Exchange Commission each such requirement that it does notfollow and describe the home country practice followed instead of any such requirement. We follow Dutch corporate governance practices with regardto the quorum32Table of Contentsrequirements applicable to meetings of shareholders and the provision of proxy statements for general meetings of shareholders, rather than thecorresponding domestic U.S. corporate governance practices. In accordance with Dutch law and generally accepted business practices, our articles ofassociation do not provide quorum requirements generally applicable to general meetings of shareholders. Although we do provide shareholders with anagenda and other relevant documents for the general meeting of shareholders, Dutch law does not have a regulatory regime for the solicitation of proxiesand the solicitation of proxies is not a generally accepted business practice in the Netherlands. Accordingly, our shareholders may not be afforded thesame protection as provided under NASDAQ's corporate governance rules.We do not comply with all the provisions of the Dutch Corporate Governance Code. As a Dutch company we are subject to the Dutch Corporate Governance Code, or DCGC. The DCGC contains both principles and best practiceprovisions for management boards, supervisory boards, shareholders and general meetings of shareholders, financial reporting, auditors, disclosure,compliance and enforcement standards. The DCGC applies to all Dutch companies listed on a government-recognized stock exchange, whether in theNetherlands or elsewhere, including The NASDAQ Global Select Market. The principles and best practice provisions apply to our management boardand supervisory board, in relation to their role and composition, conflicts of interest and independence requirements, board committees andremuneration, shareholders and the general meeting of shareholders, for example, regarding anti-takeover protection and obligations of the company toprovide information to its shareholders; and financial reporting, including external auditor and internal audit requirements. Because we do not complywith all the provisions of the DCGC, shareholders may not have the same level of protection as a shareholder in a Dutch company that fully complieswith the DCGC.Risks for U.S. Holders We may be classified as a passive foreign investment company for any taxable year, which may result in adverse U.S. federal income taxconsequence to U.S. holders. Based on our estimated gross income and average value of our gross assets, the expected price of our shares, and the nature of our business, we donot expect to be considered a "passive foreign investment company," or PFIC, for U.S. federal income tax for the 2013 tax year or in the foreseeablefuture. A corporation organized outside the United States generally will be classified as a PFIC for U.S. federal income tax purposes in any taxable yearin which at least 75% of its gross income is passive income or on average at least 50% of the gross value of its assets is attributable to assets thatproduce passive income or are held for the production of passive income. Passive income for this purpose generally includes dividends, interest,royalties, rents and gains from commodities and securities transactions. Our status in any taxable year will depend on our assets and activities in eachyear, and because this is a factual determination made annually after the end of each taxable year, there can be no assurance that we will not beconsidered a PFIC for the current taxable year or any future taxable year. The market value of our assets may be determined in large part by reference tothe market price of our ordinary shares, which is likely to fluctuate, and may fluctuate considerably given that market prices of biotechnology companieshave been especially volatile. If we were to be treated as a PFIC for any taxable year during which a U.S. holder held our ordinary shares, however,certain adverse U.S. federal income tax consequences could apply to the U.S. holder. See "Taxation—Taxation in the United States—Passive foreigninvestment company considerations."Any U.S. or other foreign judgments may be difficult to enforce against us in the Netherlands. We are incorporated under the laws of the Netherlands. We currently have only limited operations in the United States. Most of our assets arecurrently located in the Netherlands.33Table of Contents The majority of our managing directors, supervisory directors and senior management reside outside the United States. As a result, it may not bepossible for shareholders to effect service of process within the United States upon such persons or to enforce judgments against them or us in U.S.courts, including judgments predicated upon the civil liability provisions of the federal securities laws of the United States. In addition, it is not clearwhether a Dutch court would impose civil liability on us or any of our managing directors or supervisory directors in an original action based solelyupon the federal securities laws of the United States brought in a court of competent jurisdiction in the Netherlands. The United States and the Netherlands currently do not have a treaty providing for the reciprocal recognition and enforcement of judgments, otherthan arbitration awards, in civil and commercial matters. Consequently, a final judgment for payment given by a court in the United States, whether ornot predicated solely upon U.S. securities laws, would not automatically be recognized or enforceable in the Netherlands. Therefore U.S. investors may not be able to enforce against us or our management board or supervisory board members, representatives or certainexperts named herein who are residents of the Netherlands or countries other than the United States any judgments obtained in U.S. courts in civil andcommercial matters, including judgments under the U.S. federal securities laws.Item 4 Information on the CompanyA. History and Development of the Company uniQure was incorporated on January 9, 2012 as a private company with limited liability (besloten vennootschap met beperkte aansprakelijkheid)under the laws of the Netherlands. Our business was founded in 1998 and was initially operated through our predecessor company, AmsterdamMolecular Therapeutics (AMT) Holding N.V, or AMT. In 2011, AMT undertook a corporate reorganization, pursuant to which uniQure B.V. acquiredthe entire business and assets of AMT and completed a share-for-share exchange with the shareholders of AMT in the first half of 2012. EffectiveFebruary 10, 2014, in connection with our initial public offering and pursuant to a deed of amendment and conversion, we converted into a publiccompany with limited liability (naamloze vennootschap). Our legal name changed from uniQure B.V. to uniQure N.V. at the time of the conversion. Our company is registered with the Dutch Trade Register of the Chamber of Commerce (handelsregister van de Kamer van Koophandel enFabrieken) in Amsterdam, the Netherlands under number 54385229. Our corporate seat is in Amsterdam, the Netherlands, and our registered officeislocated at Meibergdreef 61, Amsterdam 1105 BA, the Netherlands, and our telephone number is +31 20 566 7394. Our website address iswww.uniqure.com. Information on our website is not incorporated by reference into this annual report or any other report we file with or furnish to theSEC. Our ordinary shares are traded on the NASDAQ Global Select Market under the symbol QURE.B. Business Overview We are a leader in the field of gene therapy and have developed Glybera, the first and currently the only gene therapy product to receive regulatoryapproval in the European Union. We are developing a pipeline of additional gene therapies through multiple collaborations that are designed to acceleratethe development and commercialization of these programs. Our pipeline includes product candidates targeting diseases for which either the efficacy ofexisting treatments is limited or the administration regimen is burdensome, such as hemophilia B, as well as diseases for which there are currently notreatments, such as Sanfilippo B syndrome. We initially intend to focus on orphan diseases but believe that we will also be able to leverage ourtechnology to develop gene therapies targeting chronic and degenerative diseases that affect larger populations. We develop our gene therapies using34Table of Contentsour innovative, modular technology platform, which consists of a suite of components that may be applied to multiple gene therapies and includes ourproprietary, cost-effective manufacturing process.Our Gene Therapy Platform Our gene therapy approach seeks to treat the causes of genetic diseases by enabling patients to effectively express a missing or deficient protein. Toaccomplish this, Glybera and our product candidates are designed to deliver a functional gene, or transgene, through a delivery system called a vector.Our approach is designed to be modular, in that it may allow us to efficiently develop, manufacture and seek regulatory approval for multiple genetherapies generally using the same principal components. The key components of our gene therapy approach are:•Therapeutic genes. We design our gene therapies to deliver into the body's cells a transgene that encodes, or provides the blueprint forthe expression of, a therapeutic protein. The transgene is carried in a gene cassette, or DNA sequence that encodes the specific gene, thatincludes DNA promoters that direct expression in specific tissues. We either develop our own gene cassettes or in-license them, often aspart of our collaborations with academic research institutions and biotechnology and pharmaceutical companies. In-licensing genecassettes provides us access to key intellectual property and allows us to build upon our collaborators' scientific expertise and financialinvestment, as well as their preclinical and, in some cases, clinical development efforts. •AAV-based vector delivery system. We deliver the gene cassette to the target tissue using an engineered, non-replicating viral vectordelivery system based on AAV, a common virus that affects humans but does not cause disease. We believe that AAV is the vector ofchoice for most in vivo gene therapy applications, such as ours, in which the functional gene is introduced directly into the patient's body.We use different variants, or serotypes, of AAV, including AAV1, AAV2 and AAV5, each of which selectively targets particulartissues. In the case of diseases for which relatively modest levels of gene expression may result in therapeutic benefit, we expect that wewill be able to achieve adequate levels of expression using existing, naturally derived AAV serotypes. In the case of diseases for whichhigher levels of gene expression may be required for therapeutic benefit, however, we believe we may need access to more potentvectors than are currently available. To complement our internal development efforts in this regard, in January 2014 we entered into acollaboration and license agreement with 4D Molecular Therapeutics, or 4D, a recently formed, private biotechnology company with ateam that we believe is a leader in AAV vector discovery and optimization. 4D uses directed evolution techniques, which involve aniterative selection process in which researchers screen libraries of mutant AAV variants to identify those that are expected to haveoptimal properties for achieving higher levels of gene expression. In more than 80 gene therapy clinical studies conducted by us or thirdparties, AAV-based vectors raised no material safety concerns. AAV-based vectors have also demonstrated sustained expression intarget tissue in non-human primates for more than five years. In the hemophilia B Phase I/II clinical trial described below, St. JudeChildren's Research Hospital in Memphis, Tennessee, or St. Jude, has reported expression in target tissue in humans for more than threeyears after a single treatment. •Administration technologies. We and our collaborators are developing expertise in utilizing a variety of administration technologies tooptimize the introduction of our gene therapy vectors to effectively deliver the transgene into the tissues and organs relevant to theindications we are targeting. •Scalable, proprietary manufacturing process. We produce our AAV-based vectors in our own facilities with our proprietarymanufacturing process, which uses insect cells and baculoviruses, a common family of viruses found in invertebrates. We believe thatour manufacturing facility in Amsterdam, which the European Medicines Agency, or EMA, has approved for clinical and35Table of Contentscommercial grade production, and our facility near Boston, Massachusetts, which we are currently building out and equipping, willenable us to produce Glybera and other gene therapies cost-effectively at commercial scale.Glybera Our first product, Glybera, was approved by the European Commission in October 2012 under exceptional circumstances for the treatment of asubset of patients with lipoprotein lipase deficiency, or LPLD. LPLD is a serious, debilitating disease caused by mutations in the lipoprotein lipase, or LPL, gene, resulting in significantly diminished or absentactivity of the LPL protein and, as a consequence, severe hypertriglyceridemia. Severe hypertriglyceridemia results in hyper-chylomicronemia, ordramatic and potentially life-threatening increases in the level of fat-carrying particles, called chylomicrons, in the blood after eating. In manycases, LPLD and the associated elevated levels of chylomicrons can cause acute and potentially life-threatening inflammation of the pancreas, known aspancreatitis, thus leading to frequent hospitalizations. Recurrent pancreatitis can lead to chronic abdominal pain, pancreatic insufficiency, which is aninability to properly digest food due to a lack of digestive enzymes made by the pancreas, and diabetes. Prior to Glybera, there was no approved therapyfor LPLD. Patients are required to adhere to a strict low-fat diet and to abstain from alcohol. These restrictions, as well as the need for frequenthospitalizations and the constant fear of pancreatitis attacks, have a significant negative impact on the daily activity level of LPLD patients and on theirquality of life. Glybera is designed to restore the LPL enzyme activity required by tissues of the body to clear, or process, the fat-carrying chylomicron particlesthat are formed in the intestine and transported via the blood to the muscle after a fat-containing meal. The product consists of an engineered copy of thehuman LPL gene packaged in a non-replicating AAV1 vector together with promoters that allow tissue-specific gene expression. AAV1 has a particularaffinity, or tropism, for muscle cells. In the European Union, we have been granted orphan drug exclusivity for Glybera for treatment of LPLD until October 2022, subject to theconditions applicable to orphan drug exclusivity. The FDA has also granted orphan drug designation to Glybera for treatment of LPLD.Post-EU Approval Program for Glybera To fulfill the key conditions of the approval of Glybera by the European Medicines Agency, or EMA, we are required to implement a patientregistry prior to commercial launch and to complete an additional, post-approval clinical trial of Glybera, which we intend to commence in the secondhalf of 2014. The principal goal of these programs will be to obtain additional data regarding the safety, efficacy and clinical benefit of Glybera. We alsobelieve that these programs will help us to better define and target the LPLD patient population, as well as to raise awareness of LPLD and of Glyberain the clinician community.Planned U.S. Program for Glybera We met with the FDA in August and December 2013 to discuss the regulatory pathway in the United States for Glybera. In contrast with theEuropean Union, the United States does not have a process to approve marketing of a drug under exceptional circumstances. In our meetings, the FDAadvised that it would require data in addition to what we had submitted to obtain marketing approval for Glybera in the European Union. The FDAadvised that severe hypertriglyceridemia is currently considered a hallmark of LPLD, and agreed that changes in chylomicron metabolism following ameal may provide data to support the bioactivity of Glybera. However, the FDA also advised that changes in chylomicron metabolism following a mealalone would not be adequate for obtaining marketing approval in the United States at this stage, since it is not yet sufficiently understood how thisbiological36Table of Contentseffect translates into clinical meaningfulness. The FDA recommended that we identify the clinical manifestations of LPLD for which Glybera mighthave the best prospects for demonstrating a meaningful impact in designing an adequate and appropriately controlled trial. We plan to discuss the details of the EU post-approval trial and patient registry with the FDA, and if applicable to seek to amend the protocols forthe post-approval trial and patient registry so that they could also serve as a clinical program with a design that addresses the FDA's requirements. Wealso plan to file an IND with the FDA for Glybera in the first half of 2014 so that we can include U.S. LPLD patients in the post-approval trial andregistry. We believe the patient registry will provide valuable data for the FDA to consider as part of the totality of our U.S. regulatory submissions.Our current expectation, subject to satisfactory completion of regulatory discussions with the FDA, is to have sufficient data from a further clinical trialof Glybera and the patient registry to file a BLA for Glybera with the FDA in 2017.Glybera Commercialization Plan We expect to launch Glybera commercially through our collaboration with Chiesi in selected countries in the European Union in mid 2014. We andChiesi are working together through a joint commercialization committee to, among other things, plan a market roll-out strategy in the territory coveredby the agreement, including developing a business model for the commercialization of a therapy administered in a one-time intervention. We and Chiesiare also building new models for product pricing and reimbursement, expanding key opinion leader relationships, identifying centers of excellence, anddeveloping physician and patient education and patient access programs. Pricing and Reimbursement in the European Union. To obtain payment coverage for Glybera from the relevant pricing and reimbursementagencies in countries in the European Union, Chiesi must generally submit price and reimbursement dossiers to the relevant bodies in each country.Chiesi is in discussions with these bodies in several countries, and expects to begin commercial sales in mid 2014. We expect that reference prices in thelarger countries in the European Union will provide a basis for pricing discussions in other countries in the European Union. Pricing and reimbursementdecisions are made on a country-by-country basis in the European Union and no country is under the obligation to follow another's pricing; however,prices in one country can influence the price level in other countries. Commercial Preparation and Roll-Out. Chiesi plans to identify centers of excellence in each of the five largest EU markets—France, Germany,Italy, Spain and the United Kingdom—where Glybera will be administered. Chiesi is developing a strategy to facilitate patient referrals to these centers,in part through broader educational efforts and outreach to relevant medical practitioners throughout the European Union. As part of this effort, we haveestablished a publications library of clinical and non-clinical materials regarding Glybera and materials for key opinion leaders, as well as materialsregarding LPLD and gene therapy generally. If we obtain marketing approval for Glybera in the United States, we currently plan to commercialize Glybera ourselves. We have begunpreliminary preparations for a potential launch in the United States, including commissioning a third party pricing and reimbursement study, and haveconducted two market research studies directed at key opinion leaders. We have also initiated the development of a diagnostic referral program, engagedin key opinion leader and patient identification efforts and begun networking with key patient organizations in the United States.Product and Development Pipeline In addition to Glybera, our development pipeline includes our internal program for hemophilia B, two collaborator-sponsored programs formonogenic diseases, one collaborator-sponsored program for a chronic degenerative disease and several programs in early preclinical development.37Table of Contents Our most advanced pipeline programs include the following: Internal program: AMT-060 for hemophilia B. In collaboration with Chiesi, we are developing AMT-060, a gene therapy for the treatment ofhemophilia B, which is a severe blood clotting disorder that can lead to repeated and sometimes life-threatening episodes of external and internalbleeding. The current standard of care for the treatment of hemophilia B is prophylactic protein replacement therapy, requiring frequent intravenousadministrations of human Factor IX, or hFIX, often costing approximately $220,000 to $340,000 per patient per year in the United States. We believethat the approximately 60% to 70% of the hemophilia B patient population who have either severe or moderately severe hemophilia would be eligible fortreatment with gene therapy. AMT-060 consists of an AAV5 vector carrying an hFIX transgene that we have exclusively licensed from St. Jude. We are currently conductingpre-IND toxicology animal studies of this product candidate. We plan to file an IND with the FDA and an Investigational Medicinal Product Dossier,or IMPD, with the EMA and then to initiate a Phase I/II, open label, dose escalation clinical trial of this product candidate in the second half of 2014 in13 to 16 patients in Europe. We expect data from our clinical trial to be available in the second half of 2015. St. Jude is currently conducting a Phase I/II, open label, dose escalation clinical trial in this indication with a gene therapy consisting of an AAV8vector carrying the same therapeutic hFIX gene that we are using in AMT-060. In an article published in the New England Journal of Medicine inDecember 2011 reviewing interim data from six patients in the St. Jude clinical trial, the principal investigators reported that the vector used in the trialconsistently led to long-term expression of the hFIX transgene at therapeutic levels in patients with severe hemophilia B, without acute or long-lastingtoxicity. We understand from public presentations by the principal investigators for this trial that two additional patients at the highest dose level in thisclinical trial have now also demonstrated such long-term expression. We believe that the interim results from this clinical trial constitute proof of conceptof the use of this therapeutic gene in treating hemophilia B and may reduce the risks involved in our development of AMT-060. Collaborator-sponsored programs. We are also collaborating with third parties that are sponsoring early-stage clinical trials of gene therapyproduct candidates to which we hold certain rights. We believe that this approach enables us to cost-effectively obtain access to preclinical and early-stage clinical results without expending significant resources of our own. These programs utilize either clinical materials that we have manufactured aspart of our collaborations or gene cassettes that we have licensed. We generally have the rights to the data generated in these collaborator-sponsoredclinical development programs, but do not control their design or timing. If we decide to progress any of these programs internally, we may need todevelop or in-license additional technology. The most advanced of these programs are the following:•AMT-021 for Acute Intermittent Porphyria. We and our collaborator Digna Biotech are developing AMT-021 as a gene therapy foracute intermittent porphyria, or AIP, a severe liver disorder. AMT-021 consists of an AAV5 vector carrying a therapeuticporphobilinogen deaminase, or PBGD, gene that we exclusively license from Applied Medical Research Center of the University ofNavarra in Spain. Our collaborator Digna Biotech is currently conducting a Phase I clinical trial of AMT-021 in eight patients in Spain.We have manufactured the gene therapy being used in this clinical trial. We understand that, to date, Digna has not observed a reductionin the urinary levels of toxic metabolites in trial participants that might have served as a surrogate marker for efficacy. We understandfrom Digna Biotech that clinical outcomes data are expected in the second half of 2014. Under an agreement with Digna Biotech, wehave an exclusive right to use all preclinical and Phase I clinical trial data from this program. •AMT-110 for Sanfilippo B Syndrome. We and our collaborator Institut Pasteur are developing AMT-110 as a gene therapy forSanfilippo B syndrome, a potentially fatal lysosomal storage38Table of Contentsdisease that results in serious brain degeneration in children. This gene therapy consists of an AAV5 vector carrying a therapeutic a-N-acetylglucosaminidase, or NaGLU, gene. Our collaborator Institut Pasteur is currently conducting a Phase I/II clinical trial of AMT-110in four patients in France. We have manufactured the gene therapy being used in this clinical trial. We have an agreement in principlewith Institut Pasteur to acquire the clinical results and commercial rights under this program following completion of this Phase I/IIclinical trial, and are currently in negotiations with Institut Pasteur regarding the terms of a definitive agreement in this regard. Weunderstand from Institut Pasteur that data are expected in the first half of 2015. We believe that if the results of this clinical trial arepositive, it will constitute proof of concept of the administration to the brain of a gene therapy for lysosomal storage diseases.•AAV2/GDNF for Parkinson's Disease. We and our collaborator the University of California at San Francisco, or UCSF, aredeveloping a gene therapy for Parkinson's disease, a progressive neurodegenerative disorder. UCSF is collaborating with the NIH toconduct a Phase I clinical trial of a gene therapy in this indication consisting of an AAV2 vector carrying a therapeutic gene we haveexclusively licensed in the gene therapy field from Amgen, Inc., or Amgen, that expresses a protein called glial cell line-derivedneurotrophic factor, or GDNF. This clinical trial is being funded and sponsored by the NIH and will involve 24 patients. UCSF'sproduct candidate has been manufactured by a third party using a mammalian cell-based process. In this clinical trial, the NIH isadministering the gene therapy using convection enhanced delivery, which is a process developed by UCSF with the goal of achievingmore precisely targeted administration than the methods used in earlier approaches, which may result in improved efficacy. We have alicense under UCSF's rights to use all preclinical and clinical data from the UCSF program for any future development program. Basedon the results of the UCSF program, we may decide to develop an AAV2-based gene therapy containing the GDNF gene manufacturedwith our insect cell-based manufacturing process. Potential Additional Pipeline Programs. We are also conducting early-stage preclinical research into a number of other potential applications ofour technologies. Currently these programs focus on utilizing AAV5 in liver and CNS indications. Based on defined criteria for indications that webelieve most likely to be well suited to our gene therapy approach, we have prioritized approximately ten additional target diseases. We may seek todevelop these programs either independently or with collaborators who are already working in the relevant disease area, including collaborators that mayhave already conducted pre-clinical or clinical studies.Our Strategy Our strategic goal is to transform the paradigm of care for many severe and chronic diseases by moving from the short-term management ofsymptoms to the potentially curative resolution of the disease through sustained therapeutic gene expression in target tissues. We are building on thecapabilities that have enabled us to obtain the first regulatory approval of a gene therapy in the European Union to address a range of diseases for whichwe believe we can reach the market with a gene therapy ahead of our competitors. We seek to achieve our goal by pursuing the following keyobjectives:•Maximize the value of Glybera. •Exploit the potential of our gene therapy platform to develop AAV-based gene therapies for additional orphan monogenic diseases andselected chronic degenerative diseases. •Leverage our competitive strengths to retain our position as a leading gene therapy company and establish additional collaborations. •Continue to invest in our technology platform and expand our modular capabilities.39Table of ContentsIntellectual PropertyIntroduction We strive to protect the proprietary technologies that we believe are important to our business, including seeking and maintaining patent protectionin the United States, Europe and other countries for novel components of gene therapies, the chemistries and processes for manufacturing these genetherapies, the use of these components in gene therapies, and other inventions and related technology that are important to our business, such as thoserelating to our technology platform. We also rely on trade secrets and careful monitoring of our proprietary information to protect aspects of ourbusiness that are not amenable to, or that we do not consider appropriate for, patent protection. Our success will depend significantly on our ability to obtain and maintain patent and other proprietary protection for commercially importanttechnology, inventions and know-how related to our business, defend and enforce our patents, maintain our licenses to use intellectual property ownedby third parties, preserve the confidentiality of our trade secrets and operate without infringing the valid and enforceable patents and other proprietaryrights of third parties. We also rely on know-how, continuing technological innovation and in-licensing opportunities to develop, strengthen, andmaintain our proprietary position in the field of AAV-based gene therapies. We also are heavily dependent on the patented or proprietary technology of third parties to develop and commercialize our products. We mustobtain licenses from such third parties on commercially reasonable terms, or our business could be harmed, possibly materially. For example, we licensefrom third parties essential parts of the therapeutic gene cassette used in Glybera and our other gene therapies, as well as the principal AAV vectors weuse and key elements of our manufacturing process. We anticipate that we will require additional licenses in the future. Our intellectual property portfolio consists of owned and in-licensed patents, licenses, trademarks, trade secrets and other intellectual propertyrights.Patent Portfolio Our gene therapy programs are protected by patents and patent applications directed to various aspects of our technology. For example, our genetherapy programs are protected by patents and patent applications with composition- of-matter or method of use claims that cover the therapeutic gene,the promoter, the viral vector capsid or other specific parts of these technologies. We also seek protection of core aspects of our manufacturing process,particularly regarding our baculovirus expression system for AAV vectors in insect cells. In addition, we have filed manufacturing patent applicationswith claims directed to alternative compositions-of-matter and manufacturing processes to seek better protection from competitors. We file the initial patent applications for our commercially important technologies in both Europe and the United States. For the same technologies,we typically file international patent applications under the Patent Cooperation Treaty, or PCT, within a year. We also may seek, usually on a case-by-case basis, local patent protection in Canada, Australia, Japan, China, India, Israel, South Africa, New Zealand, South Korea and Eurasia, as well asSouth American jurisdictions such as Brazil and Mexico. Our patent portfolio includes the following patent families:•13 patent families that we own; •8 patent families that we exclusively in-license; and •6 patent families that we non-exclusively in-license.40Table of Contents The geographic breakdown of our owned patent portfolio is as follows:•2 issued U.S. patents; •2 granted European Patent Office patents; •1 pending PCT patent application; •7 pending U.S. patent applications; •8 pending European Patent Office patent applications; and •57 pending patent applications in other jurisdictions. The patent portfolios for our manufacturing platform and most advanced programs are summarized below.NIH Patents Our manufacturing patent families contain issued patents in the United States, Europe and other territories, as well as numerous pending patentapplications. We have non-exclusively in-licensed from the NIH a patent family relating to the insect cell-based manufacturing of AAV-based vectors. Thepatents in this family include two issued patents in the United States and one issued patent in Europe, as well as issued patents in other jurisdictions.The standard 20-year term for patents in this family will expire in 2022. This patent family relates to technology used in Glybera and all of ourdevelopment programs. We also in-license from the NIH two patent families relating to AAV5-based vectors. These patents are licensed exclusively for AAV5-basedtherapeutic products to be delivered to the brain or liver for the treatment of human diseases originating in the brain or liver, excluding arthritis-relateddiseases, and non-exclusively for AAV5-based therapeutic products to treat any human disease in any manner not covered by the exclusive license. Thepatents in the first family include two issued patents in the United States, one issued patent in Europe and two issued patents in Japan, as well as issuedpatents and a pending application in other jurisdictions. The standard 20-year term for patents in this family will expire in 2019. This patent familyrelates to technology used in our AIP, hemophilia B and Sanfilippo B programs. The second family includes one issued U.S. patent with a standard 20-year term that will expire in 2020. This patent family relates to technology used in our Sanfilippo B program. See "Risk Factors—Risks Related to OurIntellectual Property—Our intellectual property licenses with third parties may be subject to disagreements over contract interpretation, which couldnarrow the scope of our rights to the relevant intellectual property or technology or increase our financial or other obligations to our licensors."Other Manufacturing Patents We own a patent family directed to improved AAV vectors that are stably expressed in insect cells. The family includes an issued patent in theUnited States and pending applications in the United States, Europe, Japan and other jurisdictions. The standard 20-year term for patents in this familywill expire in 2027. This patent family relates to technology used in Glybera and all of our development programs. We also in-license a patent family related to aspects of the AAV insect cell production technology from Protein Sciences Corporation. This familyincludes issued patents in the United States, Europe and elsewhere. This license is exclusive in respect of the products we develop with the use of thispatent family for LPLD, hemophilia B and AIP, and we may add additional products to the license on an exclusive basis except in certain specifiedcircumstances. The standard 20-year term for patents in41Table of Contentsthis family will expire in 2019. This patent family relates to technology used in Glybera and all of our development programs. We non-exclusively in-license a family of patents relating to methods for intramuscular administration of AAV vectors from AsklêpiosBiopharmaceutical, Inc., or AskBio. This family includes issued patents in Europe, Japan and other jurisdictions, and a pending application in theUnited States. The standard 20-year term for patents in this family will expire in 2016. This patent family relates to technology used in Glybera. We own a method of manufacturing patent family relating to a second- generation manufacturing method used in our AIP, hemophilia B andParkinson's disease programs. This patent family contains pending applications in the United States, Europe, Japan and other jurisdictions, and issuedpatents in several jurisdictions. The standard 20-year term for patents in this family will expire in 2028. We also own a PCT application that relates to a proprietary baculovirus filtration process. The standard 20-year term for patents in this family, ifissued, will expire in 2032. This patent family relates to technology used in Glybera and all of our development programs.Glybera We co-own with University of British Columbia, or UBC, a patent family relating to the lipoprotein lipase variant LPL-S447X transgene used inGlybera, including issued patents in Europe and Japan. The standard 20-year term for patents in this family will expire in 2020. UBC exclusivelylicensed its patent rights to Xenon, which has granted us the sublicense described below. We exclusively in-license from Aventis Pharma S.A., or Aventis, a patent family co-owned by UBC and Aventis that relates to the use of AAV-LPL vectors for LPL-deficiency, including issued patents in Europe and other jurisdictions and two pending U.S. patent applications. The standard 20-year term for patents in this family will expire in 2015. We own a family of patents relating to a VP1 vector capsid modification, which relates to the production of AAV vectors in insect cells and toAAV vectors with an altered ratio of viral capsid proteins that provides improved infectivity of the viral particles. This patent family includes issuedpatents in the United States, Europe and elsewhere, as well as pending applications in Europe, Japan and other jurisdictions. The standard 20-year termfor patents in this family will expire in 2026. We non-exclusively in-license a patent family from the Salk Institute that relates to a genetic promoter that enhances the expression of LPL- S447Xdelivered to the target tissues. This family includes four issued patents in the United States that have standard 20-year terms that will expire in 2017, andissued patents in Europe and other jurisdictions that have standard 20-year terms that will expire in 2018. We non-exclusively in-license a patent family relating to the AAV1 capsid from AmpliPhi Biosciences, Inc. (formerly Targeted GeneticsCorporation), or AmpliPhi. This family includes three issued patents in the United States, and one each in Europe and Japan, as well as issued patentselsewhere and a pending application in the United States. The standard 20-year term for patents in this family will expire in 2019. The University ofPennsylvania exclusively licensed its patent rights to AmpliPhi, which has granted us the sublicense described below.Other Programs Hemophilia B. Our patent portfolio covering our hemophilia B program includes an exclusively in-licensed patent family from St. Jude relatingto a specific promoter and a codon optimized hFIX transgene. This patent family includes two issued patents in the United States and one in Europe.The U.S. patent rights will expire in 2028 and the European patents will expire in 2025.42Table of Contents AIP. Our patent portfolio covering our AIP program includes a patent family co-owned with Proyecto de Biomedicina Cima S.L. andexclusively licensed to us. This family relates to the codon optimized PBGD transgene and its use for the treatment of AIP. This family includespending applications in the United States, Europe, Japan and elsewhere. The standard 20-year term for patents in this family will expire in 2029. Parkinson's disease. For our Parkinson's disease program, we have in- licensed a patent family and corresponding know-how relating to theGDNF transgene from Amgen for the field of gene therapy. The license is exclusive and expires on a country-by-country basis on the later of 10 yearsfollowing launch of the relevant product or of expiration of the last- to-expire licensed patent in the applicable country, after which the license willbecome non-exclusive for that given country. This patent family includes two issued patents in the United States, one of which will expire in 2015 andone in 2017.Licenses We have obtained exclusive or non-exclusive rights from third parties under a range of patents and other technology that we use in our product anddevelopment programs, as described below. Our agreements with these third parties generally grant us a license to make, use, sell, offer to sell andimport products covered by the licensed patent rights in exchange for our payment of some combination of an upfront amount, annual fees, royalties, aportion of amounts we receive from our licensees and payments upon the achievement of specified development, regulatory or commercial milestones.Some of the agreements specify the extent of the efforts we must use to develop and commercialize licensed products. The agreements generally expireupon expiration of the last-to-expire valid claim of the licensed patents. Each licensor may terminate the applicable agreement if we materially breach ourobligations and fail to cure the breach within a specified cure period, in addition to other termination rights in some cases.Technology Used for Multiple Programs We are exploiting technology from the third party sources described below in more than one of our programs. National Institutes of Health—AAV production. In 2007, we entered into a license agreement with the NIH, which we amended in 2009 and2013. Under the license agreement, the NIH has granted us a non-exclusive license to patents relating to production of AAV vectors, to make, use, sell,offer to sell and import specified plasmids, which are small DNA molecules that are physically separate from, and can replicate independently of,chromosomal DNA within a cell, or other materials, which we refer to as AAV products. We may only grant sublicenses under this agreement with theNIH's consent, which may not be unreasonably withheld. We are exploiting this technology for our Glybera program and our programs for hemophiliaB, AIP, and Sanfilippo B syndrome, and Parkinson's disease. Payment obligations to the NIH under this license agreement include a one-time upfront payment of $12,000, which we have paid; a low single-digit percentage royalty on the sale of AAV products by us or on our behalf; a maximum sub-teen double-digit percentage of sublicensing income;potential additional development milestone fees for the initiation of each clinical trial, which would total in the aggregate $255,000 for one Phase I,Phase II and Phase III trial; potential regulatory milestone fees totaling $750,000 for the first marketing approvals in specified countries or jurisdictions;and an annual maintenance fee of $15,000 creditable against royalties. We do not have to pay royalties or milestone fees under this agreement if we haveto pay royalties or milestone fees under our 2011 agreement with the NIH, described below, for the same product. In connection with entering into ourrelationship with Chiesi and obtaining the NIH's consent to sublicense our rights under this agreement to Chiesi, we also paid the NIH a total of$328,684 in amendment and sublicense payments. Under the license agreement,43Table of Contentswe have agreed to meet benchmarks in our development efforts, including as to development events, clinical trials and marketing approval, withinspecified timeframes. The NIH may terminate this agreement in specified circumstances relating to our insolvency or bankruptcy. We may terminate this agreement forany reason, in any territory, subject to a specified notice period. National Institutes of Health—AAV5. In 2011, we entered into another license agreement with the NIH, which superseded a prior 2007agreement and which we amended in 2013. Under this agreement, the NIH granted us an exclusive, worldwide license to patents relating to AAV5 foruse in therapeutic products to be delivered to the brain or liver for treatment of human diseases originating in the brain or liver, but excluding arthritis-related diseases, and a non-exclusive, worldwide license to patents relating to AAV5 for all other diseases, in each case to make, use, sell, offer to selland import products within the scope of the specified patent claims. We refer to the products licensed under this agreement as AAV5 products. We maygrant sublicenses under this agreement only with the NIH's consent, which may not be unreasonably withheld. We are currently exploiting thistechnology for our programs on hemophilia B, AIP, and Sanfilippo B syndrome. See "Risk Factors—Our intellectual property licenses with thirdparties may be subject to disagreements over contract interpretation, which could narrow the scope of our rights to the relevant intellectual property ortechnology or increase our financial or other obligations to our licensors." We have agreed to pay the NIH an initial payment of $140,000, which we have paid, an amendment royalty fee of $500,000, of which $250,000would be payable upon a sublicense of the corresponding rights, which we have paid in full, royalties equal to a low single-digit percentage of net salesof AAV5 products, if any, by or on behalf of us or our sublicensees; a single to sub-teen double-digit percentage of sublicensing income; potentialadditional development milestone fees for the initiation of each clinical trial, which would total in the aggregate $267,500 for one Phase I, Phase II andPhase III trial; total potential regulatory milestone fees of $1,731,000 for the first marketing approvals in specified countries or jurisdictions; and anannual maintenance fee of $15,000 creditable against royalties. In connection with entering into our relationship with Chiesi and obtaining the NIH'sconsent to sublicense our rights under this agreement to Chiesi, we paid the NIH a total of $716,567 in amendment and sublicense payments. If anAAV5 product is also covered by our 2007 agreement with the NIH, our obligation to pay royalties on net sales and our obligation to pay milestonefees only apply under this 2011 agreement and not the 2007 agreement. We have agreed to meet benchmarks in our development efforts, including as todevelopment events, clinical trials and marketing approval, within specified timeframes. The NIH may terminate this agreement in specified circumstances relating to our insolvency or bankruptcy. We may terminate this agreement forany reason, in any country or territory, subject to a specified notice period. Protein Sciences. In 2007, we entered into a license agreement with Protein Sciences Corporation, or PSC, which we amended in 2012. Underthe license agreement, PSC granted us a worldwide license, with a right to sublicense, to specified claims of a patent relating to an insect cell line, toresearch, develop, manufacture, import, market, and to offer for sale and sell certain products using a recombinant AAV vector developed using PSC'stechnology. The license is exclusive with respect to LPLD, hemophilia B and AIP, and we are exploiting this technology for those programs. We arelicensed to use this technology for products listed in the agreement and we may add additional products to the agreement on an exclusive basis except incertain specified circumstances. Payments obligations under the PSC agreement include a one-time upfront payment of $50,000, which we have paid, payments of $50,000 foreach additional product added to the license agreement, and an annual maintenance fee of $50,000 for each product up to an annual maximum of$150,000 and limited by an overall specified life- time maximum of $500,000 for each product. We are not required44Table of Contentsto pay maintenance fees on products we no longer wish to develop. In addition, we must pay PSC an annual fee of $50,000 while any product is beingsold or is subject to a license, partnership or funding relationship with another party, but for no more than 10 years after the first commercial sale of theproduct. We have no royalty payment obligations under the agreement. The agreement will remain in effect as long as we remain current with our payments or until we or PSC exercise our rights to terminate it. PSCmay terminate the agreement in circumstances relating to our insolvency or bankruptcy. We may terminate the agreement for convenience subject to aspecified notice period.Technology Used for Specific ProgramsGlybera We are exploiting technology from the following third party sources in our Glybera program. Academic Medical Center at the University of Amsterdam. In 2006, we entered into an agreement with the Academic Medical Center at theUniversity of Amsterdam, or AMC, and certain other parties, through which AMC invested in our predecessor company. Under this agreement, AMCassigned patent rights to us relating to LPLD and certain other indications. We have agreed to pay AMC royalties equal to a low single-digit percentage of net sales, if any, of gene therapies to treat LPLD or certain otherindications sold by us or our sublicensees that are covered by the assigned patent. We have agreed to use commercially reasonable and diligent efforts to obtain marketing approvals for, and to commercialize, gene therapies totreat LPLD and certain other indications. If we decide to cease developing and commercializing a product to treat LPLD or certain other indications ineach of Europe, the United States and Canada, we must re-assign to AMC the patent rights related to that product upon AMC's request. Xenon Genetics, Inc. In 2001, we entered into a sublicense agreement with Xenon Genetics, Inc., or Xenon, which we subsequently amended.Under the sublicense agreement, Xenon has granted us an exclusive, worldwide sublicense to patents and related technology relating to a truncated formof the LPL protein, to use, manufacture, distribute and sell products using the licensed patents or technology. We may only grant sublicenses under thisagreement with consent of Xenon and its licensor UBC. Payment obligations under the agreement include an initial sublicense fee of Canadian dollars C$75,000 and a one-time upfront payment to Xenonin the total amount of C$600,000, both of which we have paid, payment of certain past and future patent costs, a mid-single-digit percentage royalty onnet sales, if any, of licensed products sold by us or our affiliates while covered by a valid patent claim, a low single-digit percentage royalty in countrieswhere no patent protection covers the products, a low-twenties double-digit percentage share of the royalties paid to us by Chiesi and an equal orslightly higher share of royalties paid to us by other sublicensees in other specified circumstances. The share of the royalty we receive from Chiesi andany other sublicensee that we have agreed to pay to Xenon decreases to a mid-single digit percentage after patent coverage expires, and the obligationterminates 10 years after the first commercial sale of the product. We have also agreed to pay Xenon development milestone fees totaling a maximum ofC$350,000, plus an additional maximum of C$200,000 per additional product for a different indication upon our achievement of specified developmentmilestones, as well as fees upon our achievement of specified regulatory milestones totaling a maximum of C$400,000 plus an additional maximum ofC$400,000 per additional product for a different indication; or, if higher, a low-twenties double-digit percentage share of any non-royalty fees wereceive from a sublicensee.45Table of Contents The agreement will remain in effect until we or Xenon exercise our rights to terminate it. Either party may terminate the agreement in circumstancesrelating to the other party's insolvency or bankruptcy. Aventis. In 2006, we entered into a license agreement with Aventis Pharma, S.A., or Aventis, which we amended in 2013. Under the licenseagreement, Aventis has granted us an exclusive license, with a right to sublicense, to patents owned by Aventis and co-owned by Aventis and UBC, todevelop, use, make, sell and offer to sell gene therapies containing a recombinant virus with an LPL gene. Under the agreement, we made a one-time upfront payment to Aventis of €10,000 and agreed to pay Aventis a high single-digit to sub-teendouble-digit royalty as a percentage of our net sales of licensed products, or if sales are made by a commercialization partner, a low single-digit as apercentage of net sales royalty, or, if higher, a high single-digit to sub- teen double-digit royalty as a percentage of royalties we receive from suchcommercialization partner plus an equivalent percentage of the price we invoice the commercialization partner for the licensed products less our cost ofgoods sold, subject to a floor of a low single-digit percentage of net sales by Chiesi or another commercialization partner. We have also agreed to payAventis a one-time milestone fee of €50,000 upon our achievement of a specified regulatory milestone and €75,000 upon our achievement of a specifiedcommercial milestone. In conjunction with amending the agreement in 2013, we have agreed to provide Aventis with a right of first negotiation regarding a specifiedproduct candidate to treat AIP if, at the time we complete Phase I/II clinical trials of the product candidate or within a specified period thereafter, wecontemplate entering into a partnership for the co-development and commercialization of the product candidate. The agreement will remain in effect until the expiration of the protection provided by the licensed patents, or until we or Aventis exercise our rightsto terminate it. Aventis may terminate the agreement in circumstances relating to our bankruptcy. Asklêpios Biopharmaceutical. In 2010, we entered into a license agreement with AskBio under which AskBio granted us a non-exclusive,worldwide license, with a right to sublicense, to patents relating to administration of an AAV vector to muscle tissue for use in treatment of LPLD withGlybera or other products that contain an AAV vector having an AAV genetic construct encoding an LPL gene variant, to research, develop, make, use,sell, offer for sale, and import the products to treat LPLD. We made a one-time upfront payment to AskBio of $50,000 and have agreed to pay AskBio annual maintenance fees of $50,000 during the termof the license. The agreement will remain in effect on a country-by-country basis until the earlier of June 5, 2016 or the expiration of the last to expire of the validclaims in the licensed patents. We may terminate the agreement for convenience at any time subject to a specified notice period. Salk Institute for Biological Studies. In 2008, we entered into a license agreement with the Salk Institute for Biological Studies, or Salk, whichwe amended in 2013. Under the license agreement, Salk has granted us a non- exclusive license to specified biological materials and patents relating to aDNA promoter, to research, develop, make, use, import, offer for sale, and sell products using their technology for gene therapy. We have a right toenter into sublicenses under this agreement, subject to prior written consent by Salk, which may not be unreasonably withheld, and to other conditions. Payment obligations under the agreements include an upfront payment of $35,000 in 2008 and $5,000 in 2013 in connection with an amendmentand consent to sublicense to Chiesi, both of which we have paid, as well as annual maintenance fees of $30,000, a royalty equal to a low single-digitpercentage of net sales, if any, of licensed products sold by us, or, if higher, by Chiesi, and payments of46Table of Contentsa low single-digit percentage of all execution fees, maintenance fees, milestone fees and other non-royalty payments received by us from Chiesi or anyother sublicensee. The agreement will remain in effect on a country-by-country basis until the latest of 15 years from the effective date, the date of expiration of thelast to expire licensed patent or the abandonment of the last remaining licensed patent application. AmpliPhi Biosciences. In 2006, we entered into a license agreement with AmpliPhi (formerly Targeted Genetics Corporation), which weamended in 2013. Under the license agreement, AmpliPhi has granted us a non-exclusive, worldwide sublicense to patents exclusively licensed byAmpliPhi from the University of Pennsylvania, or Penn, relating to AAV1, to make, develop, use, sell, offer to sell and import products using thepatent rights to treat LPLD type 1, which includes the Glybera patient population, and LPLD type 5 by in vivo gene therapy. We may only grantsublicenses under this agreement with the consent of AmpliPhi and Penn, which may not be unreasonably withheld. We have to date paid to AmpliPhi a one-time up-front payment of $1,750,000. We have agreed to pay AmpliPhi annual fees of $100,000, a total of$4,950,000 in development and regulatory milestone payments, and a royalty equal to a low single-digit percentage of net sales, if any, of licensedproducts sold by us or Chiesi. Either party may terminate the agreement in circumstances relating to the other party's insolvency or bankruptcy. We may terminate the agreementfor convenience at any time subject to a specified notice period. If the agreement is terminated by us due to AmpliPhi's insolvency, bankruptcy or material uncured breach, or if AmpliPhi's license agreement withPenn is terminated, our license from AmpliPhi may be assigned to Penn. The assignment must be made on our request but is at Penn's discretion, whichPenn may not unreasonably withhold, provided that the agreement specifies that Penn's obligations are consistent with its current obligations andprovided that we assume all AmpliPhi's obligations.Hemophilia B St. Jude Children's Research Hospital. In 2008, we entered into a license agreement with St. Jude, which we amended in 2012. Under thelicense agreement, St. Jude has granted us an exclusive license, with a right to sublicense, to patent rights relating to expression of hFIX in gene therapyvectors, to make, import, distribute, use and commercialize products containing hFIX covered by a valid patent claim in the field of gene therapy fortreatment or prophylaxis of hemophilia B. In addition, we have a first right of negotiation regarding any patent applications that are filed by St. Jude forany improvements to the patent rights licensed to us. We have agreed to pay St. Jude a royalty equal to a low single-digit percentage of net sales, if any, by us or our sublicensees of products coveredby the licensed patent rights, and a portion of certain amounts we receive from sublicensees ranging from a mid-single digit to a mid-teen double-digitpercentage of such amounts. We have also agreed to pay St. Jude one-time milestone fees totaling $6,500,000 upon the achievement of specifieddevelopment and regulatory milestones, and an annual maintenance fee of $10,000 creditable against royalties and milestones in the same year. We haveagreed to use commercially reasonable efforts to diligently develop and commercialize products licensed under this agreement. The agreement will remain in effect until no further payment is due relating to any licensed product under this agreement or either we or St. Judeexercise our rights to terminate it. St. Jude may terminate the agreement in specified circumstances relating to our insolvency. We may terminate theagreement for convenience at any time subject to a specified notice period.47Table of ContentsAIP Digna Biotech. In 2010, we entered into a license agreement with Digna Biotech, S.L, or Digna Biotech, Fundación para la InvestigaciónMédica Applicada, or FIMA, the members of a collaborative research consortium known as UTE CIMA, and Proyecto de Biomedicina CIMA S.L., orProyecto, which superseded several prior agreements amongst such parties. We refer to Digna Biotech, FIMA, UTE CIMA and Proyecto collectivelyas the CIMA Parties. Under the license agreement, Proyecto granted us an exclusive, worldwide license, with a right to sublicense, under its interest inpatent rights we jointly own with Proyecto relating to PBGD gene therapy to use, develop, make, have made and commercialize products using thelicensed patent rights. In addition, UTE CIMA granted us a non-exclusive, worldwide license, with the right to grant sublicenses, under certain patentrights, know-how and materials required for the use, development, manufacture or commercialization of products covered by our exclusive license fromProyecto in the gene therapy field. We have agreed to pay Digna Biotech royalties equal to a mid-single digit percentage of net sales, if any, by us or our affiliates of licensed productscovered by our exclusive license and a sub-teen double-digit percentage share of net revenues we receive from our sublicensees. Digna Biotech isresponsible for apportioning the amounts we pay Digna Biotech amongst the CIMA Parties. Under the agreement we have to use commercially reasonable efforts to further develop, manufacture and commercialize licensed products as soonas reasonably practicable. The agreement will remain in effect until our payment obligations expire or we or another party exercise our rights to terminate it. A party mayterminate the agreement in circumstances relating to another party's insolvency or bankruptcy or if our agreement under which Digna Biotech isconducting a Phase I clinical trial of AMT-021 terminates. We may terminate this agreement for convenience, subject to a specified notice period. IfDigna Biotech terminates the license agreement for breach or insolvency, we or Digna Biotech terminate the license agreement because our otheragreement with Digna Biotech terminates other than for breach or insolvency of Digna Biotech or if we terminate the license agreement for convenience,the CIMA Parties will have the exclusive right to use the patent rights we jointly own with Proyecto that were exclusively licensed to us to furtherdevelop and commercialize licensed products for the treatment or prevention of AIP without financial obligations to us.Parkinson's disease Amgen. In 2010, we entered into a license agreement with Amgen, Inc. which superseded a prior 2008 agreement. Under the license agreement,Amgen granted us an exclusive, worldwide license, with a right to sublicense, to patents and know-how relating to GDNF to research, develop, make,use, offer for sale, sell, import, export and otherwise exploit gene therapies capable of delivering GDNF, the gene encoding GDNF, or any fragment ofGDNF that has specified functional activity, which we refer to as GDNF products. The license exclusivity, and our obligation to make the revenuesharing payments described below, with respect to a given GDNF product in a given country expires on the later of expiration of the last-to-expirelicensed patent in such country that covers such GDNF product and the tenth anniversary of the first commercial sale of such GDNF product in suchcountry. Thereafter the license would become non-exclusive with respect to that GDNF product in that country. We have agreed to pay Amgen revenue sharing payments equal to a sub-teen double-digit percentage of net revenues, if any, that we receive fromour sales of GDNF products, from granting sublicenses under the intellectual property licensed from Amgen or from granting licenses under certain ofour intellectual property rights. Upon receipt of the first marketing approval anywhere in the world for the first GDNF product we have also agreed topay Amgen a one-time milestone fee of the greater of $10 million or a sub-teen double digit percentage of any milestone payments we receive from thirdparties with respect to receiving such approval.48Table of Contents We agreed to use reasonably diligent efforts to develop at least one GDNF product and seek to obtain regulatory approvals for this GDNF productin the United States and the European Union, and to commercialize it. We granted Amgen an option to negotiate an exclusive license from us to research, develop, make, use, offer for sale, sell and otherwise exploitGDNF products in the United States, Mexico and Canada. Amgen may exercise the option within a specified period following completion of the firstPhase II clinical trial of the first GDNF product we develop. If Amgen exercises the option but we and Amgen do not execute a definitive agreement togrant these rights to Amgen within a specified period of time, we retain these rights but may not grant development or commercialization rights to a thirdparty in these North American countries on financial terms less favorable to us than those last offered by Amgen. The agreement will remain in effect until either we or Amgen exercise our rights to terminate it. We may terminate the agreement for convenience atany time subject to a specified notice period. If we terminate the agreement for convenience, or if Amgen terminates the agreement due to our uncuredmaterial breach, rights to GDNF products will revert to Amgen. As part of such reversion, if Amgen requests, we have agreed to grant Amgen anexclusive, worldwide license under our relevant intellectual property rights so that Amgen can research, develop, make, use, offer for sale, sell andotherwise exploit GDNF products, subject to a specified revenue sharing and a one-time regulatory milestone payment from Amgen to us. UCSF. In 2012, we entered into a data license agreement with the University of California in San Francisco, or UCSF, related to UCSF's rightsto the clinical trial data from a Phase I/II clinical trial, sponsored by the NIH, and that UCSF is conducting, of a product candidate consisting of anAAV2 vector carrying the GDNF gene, and to certain related preclinical data and know-how. Under the data license agreement, UCSF granted us anon- exclusive license, with a right to sublicense, to research, develop, make, use, offer for sale, sell and otherwise exploit pharmaceutical productscontaining or consisting of an AAV2 genetic construct encoding GDNF, or any fragment of GDNF that has specified functional activity, for thetherapeutic, palliative and prophylactic treatment of Parkinson's disease in humans. During the term of the data license agreement, UCSF has agreed notto grant to any other for-profit entity any of the rights granted to us thereunder, except under specified circumstances involving a breach of our diligenceobligations described below. Payment obligations under the agreement include a one-time, up-front payment of $300,000, which we have paid, a royalty equal to a low single-digit percentage of our net sales, if any, of products that are identified or developed through material use of the data licensed from UCSF, or identifiedproducts, as well as third party license fees with the percentage due to UCSF ranging from a low double-digit percentage for earlier-granted sublicensesto a low single-digit percentage for later-granted sublicenses. Our obligation to pay UCSF earned royalties with respect to a given country begins on thefirst commercial sale of an identified product in such country, and our obligation to pay earned royalties and third-party license fees expires on the tenthanniversary of such first commercial sale, after which the data license will become perpetual, non-exclusive, fully paid-up, and royalty-free in suchcountry. The UCSF agreement also contains certain other obligations we have agreed to complete by specified dates, including obligations to deliver toUCSF by June 12, 2014 specified materials for UCSF to complete a non-clinical study of an AAV2 vector carrying the GDNF gene, to demonstrateequivalent product release specifications of our vector to the vector used in the ongoing NIH-sponsored Phase I clinical trial, to pursue a bridging studyusing our AAV2 vector carrying a GDNF gene, and to use commercially reasonable efforts to proceed, either directly or through a third party licensee,to develop, seek to obtain regulatory approval for and market at least one identified product in the United States and the European Union.49Table of Contents If we materially fail to comply with any of the diligence obligations described above and do not cure such failure within specified cure periods,UCSF may at its option either terminate the data license agreement or be freed from its covenant not to grant to any other for-profit entity any of therights granted to us thereunder. The data license agreement will remain in effect until all of our payment obligations to UCSF have ended in all countries, unless either we orUCSF exercise our rights to terminate it earlier. UCSF may terminate the agreement in specified circumstances relating to our bankruptcy. We mayterminate the agreement for convenience at any time subject to a specified notice period.Trade Secrets In addition to patents and licenses, we rely on trade secrets and know-how to develop and maintain our competitive position. For example,significant aspects of the process by which we manufacture Glybera and our gene therapies are based on unpatented trade secrets and know-how. Weseek to protect our proprietary technology and processes and obtain and maintain ownership of certain technologies, in part, through confidentialityagreements and invention assignment agreements with our employees, consultants, scientific advisors, contractors and commercial partners. We alsoseek to preserve the integrity and confidentiality of our data, trade secrets and know-how by maintaining physical security of our premises and physicaland electronic security of our information technology systems.Trademarks uniQure and Glybera are registered trademarks in various jurisdictions including the United States and the European Union. We intend to seektrade mark protection for other product candidates as and when appropriate.Strategic Collaboration: Chiesi We have entered into two agreements with Chiesi, a family-owned Italian pharmaceutical company with 2012 worldwide revenues ofapproximately € 1.1 billion. One is an agreement for the commercialization of Glybera for LPLD and the second is an agreement for the co-developmentand commercialization of our hemophilia B program. We have retained full rights in the United States, Canada and Japan under both agreements. Wehave received €17.0 million in aggregate upfront payments as well as a €14.0 million investment in our ordinary shares. In addition, these agreementsprovide us with research funding for further development of our hemophilia B product candidate, as well as the potential for commercial milestonepayments of up to €42.0 million for Glybera for LPLD. Our collaboration with Chiesi is guided by a joint steering committee and a jointcommercialization committee. Under our Glybera commercialization agreement, we will receive payments from Chiesi for the quantities of Glybera we manufacture and supplyto them. We are required to pay the cost of goods sold, including royalty and other payments to third parties in connection with the sale of Glybera.Based on our estimates, we anticipate we will retain in the range of 20% to 30% of the net sales of Glybera by Chiesi in the European Union and othercountries under our agreement, net of the cost of goods sold, including the royalties and other obligations we owe to third parties. In addition, we arerequired to repay 20% of the gross amount received from Chiesi related to Glybera sales in repayment of a technical development loan from the Dutchgovernment, which has a current outstanding balance of €5.5 million. Under our hemophilia B co-development agreement, we will also receive payments from Chiesi for any commercial quantities of our hemophilia Bproduct candidate we manufacture and supply to them, if we receive regulatory approval for such product candidate. We estimate that the amount wewould50Table of Contentsretain, net of cost of goods sold, including third party royalties and related amounts, will be between 25% and 35% of the revenues from sales of suchproduct by Chiesi, varying by country of sale.Strategic Collaboration: 4D Molecular Therapeutics In January 2014, we entered into a collaboration and license agreement with 4D for the discovery and optimization of next-generation AAVvectors. Under this agreement, 4D has granted us an exclusive, worldwide license, with the right to grant sublicenses, to 4D's existing and certain futureknow-how and other intellectual property, including certain patent rights 4D has exclusively licensed from the Regents of the University of California,to develop, make, use and sell certain AAV vectors and products containing such AAV vectors and gene constructs, for delivery of such geneconstructs to CNS or liver cells for the diagnosis, treatment, palliation or prevention of any disease or medical condition. Under this collaboration, the4D team, including Dr. David Schaffer, 4D's co-founder and Professor of Chemical and Biomolecular Engineering at the University of California,Berkeley, has agreed to establish a laboratory to identify next generation AAV vectors. In addition, in connection with our entry into this collaboration,Dr. Schaffer will join our Supervisory Board. We have agreed to fund a three-year research collaboration, which can be extended at our option for an additional year, to be conducted under amutually agreed research plan. We are entitled to select a specified number of AAV variants from the research collaboration. We will have exclusiverights to further research, develop, manufacture and commercialize the selected AAV variants, as well as AAV vectors and products containing suchAAV variant and gene constructs, or licensed products, and, during the research collaboration and for the term of the agreement, 4D retains no rights tothe selected AAV variants for any use. During the research collaboration and throughout the term of the agreement, 4D has agreed to work exclusivelywith us to research, develop, manufacture and commercialize AAV variants, AAV vectors and products containing AAV vectors and gene constructs,for delivery of gene constructs to CNS or liver cells for the diagnosis, treatment, palliation or prevention of any disease or medical condition. Our research collaboration with 4D is guided by a joint research steering committee. Under the agreement, we have agreed to make a one-timeupfront payment of $100,000 and another one-time payment of $100,000 upon the joint research steering committee's approval of the research plan,including an associated budget. Our payment obligations under the agreement include the research collaboration funding described above as well aspayments for the achievement of specified pre-clinical, clinical and regulatory milestones of up to $5,000,000 for each licensed product that we developunder the collaboration, and, for each licensed product, each indication. We have also agreed to pay 4D royalties equal to a single-digit percentage of netsales, if any, of licensed products by us or our affiliates. We will also pay 4D a low to upper-low double-digit percentage of any sublicensing incomewe receive, subject to a floor of a low single-digit percentage of net sales, if any, by sublicensees of certain licensed products.Competition The biotechnology and pharmaceutical industries, including in the gene therapy field, are characterized by rapidly advancing technologies, intensecompetition and a strong emphasis on intellectual property. We face substantial competition from many different sources, including large and specialtypharmaceutical and biotechnology companies, academic research institutions and governmental agencies and public and private research institutions. We are aware of several companies focused on developing gene therapies in various indications, including bluebird bio, Sangamo BioScience,AGTC, Oxford Biosciences, Spark Therapeutics, Audentes Therapeutics, RegenX and Baxter, as well as several companies addressing other methodsfor modifying genes and regulating gene expression. Although companies and research institutions in the gene51Table of Contentstherapy field tend to focus on particular target indications, any advances in gene therapy technology made by a competitor may be used to developtherapies competing against Glybera or one of our product candidates. We may also face competition with respect to the treatment of some of thediseases that we are seeking to target with our gene therapies from protein pharmaceuticals under development at pharmaceutical and biotechnologycompanies such as Pfizer, Baxter, Bayer, Novo Nordisk, Genzyme, Shire, BioMarin, Biogen Idec and numerous other pharmaceutical andbiotechnology firms. We must also compete with existing standards of care, therapies and symptomatic treatments, as well as any new therapies that may becomeavailable in the future for the indications we are targeting. Many of our current or potential competitors, either alone or with their collaboration partners, have significantly greater financial resources andexpertise in research and development, manufacturing, preclinical testing, conducting clinical trials, and marketing approved products than we do.Mergers and acquisitions in the pharmaceutical, biotechnology and gene therapy industries may result in even more resources being concentrated amonga smaller number of our competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborativearrangements with large and established companies. These competitors also compete with us in recruiting and retaining qualified scientific andmanagement personnel and establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies complementaryto, or necessary for, our programs. The key competitive factors affecting the success of all of our programs are likely to be their efficacy, safety, convenience, price and the availabilityof reimbursement from government and other third party payors. We also believe that, due to the small size of the patient populations in the orphanindications we target, being first to market will be a significant competitive advantage. We believe that our advantages in vector and manufacturingtechnology will allow us to reach market in a number of indications ahead of our competitors, and to capture the markets in these indications.Government Regulation and Reimbursement Government authorities in the United States, European Union and other countries extensively regulate, among other things, the approval, research,development, pre-clinical and clinical testing, manufacture (including any manufacturing changes), packaging, storage, recordkeeping, labeling,advertising, promotion, distribution, marketing, post-approval monitoring and reporting, and import and export of pharmaceutical products, biologicalproducts and medical devices. We believe that all of our product candidates will be regulated as biological products, or biologics, and in particular, asgene therapies, and will be subject to such requirements and regulations under U.S. and foreign laws.Regulation in the United States In the United States, the Food and Drug Administration, or FDA, regulates biologics under the Public Health Service Act, or PHSA, and theFederal Food, Drug, and Cosmetic Act, or FDCA, and regulations and guidance implementing these laws. Obtaining regulatory approvals and ensuringcompliance with applicable statutes and regulatory requirements entails the expenditure of substantial time and financial resources. The failure to complywith applicable requirements may subject an applicant and/or sponsor to a variety of administrative or judicial sanctions, including refusal by the FDAto approve pending applications, withdrawal of a license, imposition of a clinical hold, issuance of warning letters and other types of letters, productrecalls, fines, and civil or criminal investigations and penalties brought by the Department of Justice and other federal and state government agencies.52Table of Contents All of our current product candidates are subject to regulation by the FDA as biologics. An applicant seeking approval to market and distribute anew biologic in the United States must typically undertake the following:•completion of preclinical laboratory tests, animal studies and formulation studies in compliance with the FDA's current Good LaboratoryPractice, or cGLP, regulations; •submission to the FDA of an Investigational New Drug, or IND Application, which allows human clinical trials to begin unless theFDA objects within 30 days; •approval by an independent institutional review board, or IRB, representing each clinical site before each clinical trial may be initiated; •performance of adequate and well-controlled human clinical trials in accordance with the FDA's or EMA's good clinical practices, orGCP, to establish the safety, potency, purity and efficacy of the proposed biological product for each indication; •preparation and submission to the FDA of a Biologics License Application, or BLA; •satisfactory review of the BLA by an FDA advisory committee, when appropriate or if applicable; •satisfactory completion of one or more FDA inspections of the manufacturing facility or facilities at which the product, or componentsthereof, are produced to assess compliance with cGMP requirements and to assure that the facilities, methods and controls are adequateto preserve the product's identity, strength, quality and purity; •payment of user fees and securing FDA approval of the BLA; and •compliance with any post-approval requirements, including Risk Evaluation and Mitigation Strategies, or REMS, and post-approvalstudies required by FDA.Human Clinical Studies Under an IND Clinical trials involve the administration of the investigational biologic to human subjects under the supervision of qualified investigators inaccordance with cGCP requirements. A protocol for each clinical trial and any subsequent protocol amendments must be submitted to the FDA as partof an IND. A clinical trial may not proceed unless and until an IND becomes effective, which is 30 days after its receipt by the FDA unless before thattime the FDA raises concerns or questions related to one or more proposed clinical trials and places the trial on clinical hold. In addition, an IRB at each institution participating in the clinical trial must review and approve the plan for any clinical trial before it commences atthat institution, and the IRB must conduct continuing review and reapprove the study at least annually. An IRB must operate in compliance with FDAregulations, and information about certain clinical trials must be submitted within specific timeframes to the NIH for public dissemination on theirClinicalTrials.gov website. Human clinical trials are typically conducted in three sequential phases, which may overlap or be combined:•Phase I: The biological product is initially introduced into healthy human subjects or patients with the target disease or condition andtested for safety, dosage tolerance, absorption, metabolism, distribution, excretion and, if possible, to gain an early understanding of itseffectiveness. •Phase II: The biological product is administered to a limited patient population to identify possible adverse effects and safety risks, topreliminarily evaluate the efficacy of the product for specific targeted diseases and to determine dosage tolerance and optimal dosage.53Table of Contents•Phase III: The biological product is administered to an expanded patient population in adequate and well-controlled clinical trials togenerate sufficient data to statistically confirm the potency and safety of the product for approval, to establish the overall risk-benefitprofile of the product and to provide adequate information for the labelling of the product. Progress reports detailing the results of the clinical trials must be submitted at least annually to the FDA and more frequently if serious adverseevents occur. The FDA or the sponsor may suspend or terminate a clinical trial at any time on various grounds, including a finding that the researchsubjects are being exposed to an unacceptable health risk. Similarly, an IRB can suspend or terminate approval of a clinical trial at its institution if theclinical trial is not being conducted in accordance with the IRB's requirements or if the biologic has been associated with unexpected serious harm topatients.FDA Guidance Governing Gene Therapy Products The FDA has issued various guidance documents regarding gene therapies, which outline additional factors that the FDA will consider at each ofthe above stages of development and relate to, among other things, the proper preclinical assessment of gene therapies; the chemistry, manufacturing,and control information that should be included in an IND application; the proper design of tests to measure product potency in support of an IND orBLA application; and measures to observe delayed adverse effects in subjects who have been exposed to investigational gene therapies when the risk ofsuch effects is high. If a gene therapy trial is conducted at, or sponsored by, institutions receiving the NIH funding for recombinant DNA research, a protocol andrelated documentation must be submitted to, and the study registered with, the NIH Office of Biotechnology Activities, or OBA, pursuant to the NIHGuidelines for Research Involving Recombinant DNA Molecules prior to the submission of an IND to the FDA. In addition, many companies andother institutions not otherwise subject to the NIH Guidelines voluntarily follow them. The NIH will convene the Recombinant DNA AdvisoryCommittee (RAC), a federal advisory committee, to discuss protocols that raise novel or particularly important scientific, safety or ethical considerationsat one of its quarterly public meetings. The OBA will notify the FDA of the RAC's decision regarding the necessity for full public review of a genetherapy protocol. RAC proceedings and reports are posted to the OBA web site and may be accessed by the public.Compliance with cGMP Requirements Manufacturers of biologics must comply with applicable cGMP regulations, including quality control and quality assurance and maintenance ofrecords and documentation. Manufacturers and others involved in the manufacture and distribution of such products must also register theirestablishments with the FDA and certain state agencies. Both domestic and foreign manufacturing establishments must register and provide additionalinformation to the FDA upon their initial participation in the manufacturing process. Establishments may be subject to periodic unannounced inspectionsby government authorities to ensure compliance with cGMPs and other laws. Discovery of problems may result in a government entity placingrestrictions on a product, manufacturer, or holder of an approved BLA, and may extend to requiring withdrawal of the product from the market. TheFDA will not approve an application unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements andadequate to assure consistent production of the product within required specification.Submission of a BLA The results of the preclinical and clinical studies, together with detailed information relating to the product's chemistry, manufacture, controls andproposed labeling, among other things, are submitted to the FDA as part of a BLA requesting a license to market the product for one or moreindications.54Table of ContentsUnder federal law, the submission of most BLAs is subject to an application user fee, currently exceeding $2.1 million, and the sponsor of an approvedBLA is also subject to annual product and establishment user fees, currently exceeding $104,000 per product and $554,600 per establishment. Thesefees are typically increased annually. The FDA has agreed to specified performance goals in the review of BLAs. Most such applications are meant tobe reviewed within ten months from the date of filing, and most applications for "priority review" products are meant to be reviewed within six monthsof filing. The FDA may also refer applications to an advisory committee for review and a vote on approval. Typically, an advisory committee includesclinicians and other experts who review, evaluate and vote on a recommendation as to whether the application should be approved. The FDA is notbound by the recommendations of an advisory committee, but it considers such recommendations carefully when making decisions.Expedited Review The FDA is authorized to expedite the review of BLAs in several ways. Under the fast track program, the sponsor of a biologic candidate mayrequest the FDA to designate the product for a specific indication as a fast track product concurrent with or after the filing of the IND for the productcandidate. In addition to other benefits, such as the ability to use surrogate endpoints and have greater interactions with the FDA, the FDA may initiatereview of sections of a fast track product's BLA before the application is complete. FDA may also take certain actions with respect to productsdesignated as breakthrough therapies, including holding meetings with the sponsor and the review team throughout the development process; providingtimely advice to and communication with the product sponsor regarding development and approval; involving more senior staff in the review process;assigning a cross-disciplinary project lead for the review team; and taking certain steps to design the clinical trials in an efficient manner.FDA's Decision on a BLA and Post-Approval Requirements On the basis of the BLA and accompanying information, including the results of the inspection of the manufacturing facilities, the FDA may issuean approval letter or a complete response letter. An approval letter authorizes commercial marketing of the biological product with specific prescribinginformation for specific indications. A complete response letter generally outlines the deficiencies in the submission and may require substantialadditional testing or information in order for the FDA to reconsider the application. If and when those deficiencies have been addressed to the FDA'ssatisfaction in a resubmission of the BLA, the FDA will issue an approval letter. If the FDA approves a product, it may limit the approved indications for use of the product, require that contraindications, warnings or precautionsbe included in the product labeling, require that post-approval studies, including Phase IV clinical trials, be conducted to further assess a biologic'ssafety after approval, require testing and surveillance programs to monitor the product after commercialization, or impose other conditions, includingdistribution restrictions or other risk management mechanisms, including Risk Evaluation and Mitigation Strategies (REMS). The FDA may prevent orlimit further marketing of a product based on the results of post-market studies or surveillance programs. Following approval, some types of changes to the approved product, such as adding new indications, manufacturing changes and additionallabeling claims, are subject to further testing requirements and the FDA review and approval. The product may also be subject to official lot release,meaning that the manufacturer is required to perform certain tests on each lot of the product before it is released for distribution. Other post-approvalrequirements include reporting of cGMP deviations that could affect the identity, potency, purity and overall safety of a distributed product, reporting of55Table of Contentsadverse effects, reporting new information regarding safety and efficacy, maintaining adequate record-keeping, and complying with electronic recordand signature requirements.Biosimilars and Exclusivity The 2010 Patient Protection and Affordable Care Act authorized the FDA to approve biosimilars. Under the Act, a manufacturer may submit anapplication for licensure of a biologic product that is "biosimilar to" or "interchangeable with" a previously approved biological product or "referenceproduct." In order for the FDA to approve a biosimilar product, it must find that there are no clinically meaningful differences between the referenceproduct and proposed biosimilar product. A finding of "interchangeability" requires that a product is determined to be biosimilar to the referenceproduct, and that the product can be expected to produce the same clinical results as the reference product. An application for a biosimilar product maynot be submitted to the FDA until four years following approval of the reference product, and it may not be approved until 12 years thereafter. Theseexclusivity provisions only apply to biosimilar companies and not companies that rely on their own data and file a full BLA.Orphan Drug Exclusivity Under the Orphan Drug Act, the FDA may designate a biological product as an "orphan drug" if it is intended to treat a rare disease or condition(generally meaning that it affects fewer than 200,000 individuals in the United States, or more in cases in which there is no reasonable expectation thatthe cost of developing and making a biological product available in the United States for treatment of the disease or condition will be recovered fromsales of the product). If a product with orphan status receives the first FDA approval, it will be granted 7 years of market exclusivity (meaning that theFDA may not approve any other applications for the same product for the same indication for seven years, except in certain limited circumstances).Competitors may receive approval of different products for the indication for which the orphan product has exclusivity and may obtain approval for thesame product but for a different indication. Orphan product designation does not convey any advantage in or shorten the duration of the regulatoryreview and approval process. In the European Union, we have been granted orphan drug exclusivity for Glybera for treatment of LPLD until October2022, subject to the conditions applicable to orphan drug exclusivity. The FDA has also granted orphan drug designation to Glybera for treatmentof LPLD, meaning that it will receive orphan drug exclusivity if it is the first product approved for that indication.Pediatric Exclusivity Pediatric exclusivity is another type of regulatory exclusivity in the United States and, if granted, provides for the attachment of an additional sixmonths of marketing protection to the term of any existing regulatory exclusivity, including orphan exclusivity and exclusivity against biosimilars. Thissix-month exclusivity may be granted if a BLA sponsor submits pediatric data that fairly respond to a written request from the FDA for such data. Thedata do not need to show the product to be effective in the pediatric population studied; rather, if the clinical trial is deemed to fairly respond to theFDA's request, the additional protection is granted. If reports of requested pediatric studies are submitted to and accepted by the FDA within thestatutory time limits, whatever statutory or regulatory periods of exclusivity cover the product are extended by six months.FDA Regulation of Companion Diagnostics We may seek to develop in vitro and in vivo companion diagnostics for use in selecting the patients that we believe will respond to our genetherapies. FDA officials have issued draft guidance that, when finalized, would address issues critical to developing in vitro companion diagnostics,such as biomarker qualification, establishing clinical validity, the use of retrospective data, the appropriate patient56Table of Contentspopulation and when the FDA will require that the device and the drug be approved simultaneously. The draft guidance issued in July 2011 states that ifsafe and effective use of a therapeutic product depends on an in vitro diagnostic, then the FDA generally will require approval or clearance of thediagnostic at the same time that the FDA approves the therapeutic product. The FDA has yet to issue further guidance, and it is unclear whether it willdo so, or what the scope would be.Anti-Kickback Provisions and Requirements The federal anti-kickback statute prohibits, among other things, knowingly and willfully offering, paying, soliciting or receiving remuneration toinduce or in return for purchasing, leasing, ordering or arranging for the purchase, lease or order of any healthcare item or service reimbursable underMedicare, Medicaid or other federally financed healthcare programs. This statute has been interpreted to apply to arrangements between pharmaceuticalmanufacturers on the one hand and prescribers, purchasers and formulary managers on the other. Violations of the anti-kickback statute are punishableby imprisonment, criminal fines, civil monetary penalties and exclusion from participation in federal healthcare programs. Although there are a numberof statutory exemptions and regulatory safe harbors protecting certain common activities from prosecution or other regulatory sanctions, the exemptionsand safe harbors are drawn narrowly, and practices that involve remuneration intended to induce prescribing, purchases or recommendations may besubject to scrutiny if they do not qualify for an exemption or safe harbor. Federal false claims laws prohibit any person from knowingly presenting, or causing to be presented, a false claim for payment to the federalgovernment, or knowingly making, or causing to be made, a false statement to have a false claim paid. Pharmaceutical and other healthcare companieshave been prosecuted under these laws for allegedly inflating drug prices they report to pricing services, which in turn were used by the government toset Medicare and Medicaid reimbursement rates, and for allegedly providing free product to customers with the expectation that the customers wouldbill federal programs for the product. In addition, certain marketing practices, including off-label promotion, have also been alleged by governmentagencies to violate false claims laws. The majority of states also have statutes or regulations similar to the federal anti-kickback law and false claimslaws, which apply to items and services reimbursed under Medicaid and other state programs, or, in several states, apply regardless of the payor.Coverage, Pricing and Reimbursement The containment of healthcare costs has become a priority of federal, state and foreign governments, and the prices of drugs have been a focus inthis effort. Third party payors are also increasingly challenging the prices charged for medical products and services and examining the medicalnecessity and cost- effectiveness of medical products and services, in addition to their safety and efficacy. If these third party payors do not consider aproduct to be cost- effective compared to other available therapies, they may not cover the product after approval as a benefit under their plans or, if theydo, the level of payment may not be sufficient to allow a company to sell its products at a profit. The U.S. government, state legislatures and foreigngovernments have shown significant interest in implementing cost containment programs to limit the growth of government-paid health care costs,including price controls, restrictions on reimbursement and requirements for substitution of generic products for branded prescription drugs. Adoptionof such controls and measures, and tightening of restrictive policies in jurisdictions with existing controls and measures, could limit payments forpharmaceuticals. As a result, the marketability of any product which receives regulatory approval for commercial sale may suffer if the government and third partypayors fail to provide adequate coverage and reimbursement. In addition, an increasing emphasis on managed care in the United States has increasedand will continue to increase the pressure on drug pricing. Coverage policies, third party57Table of Contentsreimbursement rates and drug pricing regulation may change at any time. In particular, the Patient Protection and Affordable Care Act containsprovisions that may reduce the profitability of drug products, including, for example, increased rebates for drugs sold to Medicaid programs, extensionof Medicaid rebates to Medicaid managed care plans, mandatory discounts for certain Medicare Part D beneficiaries and annual fees based onpharmaceutical companies' share of sales to federal health care programs. Even if favorable coverage and reimbursement status is attained for one ormore products that receive regulatory approval, less favorable coverage policies and reimbursement rates may be implemented in the future.Regulation in the European Union Product development, the regulatory approval process, and safety monitoring of medicinal products and their manufacturers in the European Unionproceed in much the same manner as they do in the United States. Therefore, many of the issues discussed above apply similarly in the context of theEuropean Union. In addition, drugs are subject to the extensive price and reimbursement regulations of the various EU member states.Clinical trials As is the case in the United States, the various phases of preclinical and clinical research in the European Union are subject to significant regulatorycontrols. The Clinical Trials Directive 2001/20/EC, as amended, provides a system for the approval of clinical trials in the European Union viaimplementation through national legislation of the member states. Under this system, approval must be obtained from the competent national authority ofan EU member state in which the clinical trial is to be conducted. Furthermore, a clinical trial may only be started after a competent ethics committee hasissued a favorable opinion on the clinical trial application, which must be supported by an investigational medicinal product dossier with supportinginformation prescribed by the Clinical Trials Directive and corresponding national laws of the member states and further detailed in applicable guidancedocuments. The sponsor of a clinical trial, or its legal representative, must be based in the European Economic Area. European regulators and ethicscommittees also require the submission of adverse event reports during a study and a copy of the final study report.Marketing approval Marketing approvals under the European Union regulatory system may be obtained through a centralized or decentralized procedure. Thecentralized procedure results in the grant of a single marketing authorization that is valid for all—currently 28—EU member states. Pursuant to Regulation (EC) No 726/2004, as amended, the centralized procedure is mandatory for drugs developed by means of specifiedbiotechnological processes, advanced therapy medicinal products as defined in Regulation (EC) No 1394/2007, as amended, drugs for human usecontaining a new active substance for which the therapeutic indication is the treatment of specified diseases, including but not limited to acquiredimmune deficiency syndrome, neurodegenerative disorders, auto-immune diseases and other immune dysfunctions, as well as drugs designated asorphan drugs pursuant to Regulation (EC) No 141/2000, as amended. The CHMP also has the discretion to permit other products to use the centralizedprocedure if it considers them sufficiently innovative or they contain a new active substance. Given our focus on gene therapies, which fall within thecategory of advanced therapy medicinal products, or ATMPs, and orphan indications, our products and product candidates should typically qualify forthe centralized procedure. In the marketing authorization application, or MAA, the applicant has to properly and sufficiently demonstrate the quality, safety and efficacy ofthe drug. Under the centralized approval procedure, the CHMP, is responsible for drawing up the opinion of the EMA on any matter concerning the58Table of Contentsadmissibility of the files submitted in accordance with the centralized procedure, such as an opinion on the granting, variation, suspension or revocationof a marketing authorization, and pharmacovigilance. For ATMPs, the CAT is responsible in conjunction with the CHMP for the evaluation ofATMPs. The CAT is primarily responsible for the scientific evaluation of ATMPs and prepares a draft opinion on the quality, safety and efficacy ofeach ATMP for which a MAA 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 also responsible for providing guidelines on ATMPs and have published numerous guidelines, including specificguidelines on gene therapies. These guidelines provide additional guidance on the factors that the EMA will consider in relation to the development andevaluation of ATMPs and include, among other things, the preclinical studies required to characterize ATMPs; the manufacturing and controlinformation that should be submitted in a MAA; and post-approval measures required to monitor patients and evaluate the long term efficacy andpotential adverse reactions of ATMPs. Although these guidelines are not legally binding, we believe that our compliance with them is likely necessaryto gain and maintain approval for any of our product candidates. The maximum timeframe for the evaluation of an MAA by the CHMP under the centralized procedure is 210 days after receipt of a validapplication. This period will be suspended until such time as the supplementary information requested by the CHMP, or in the case of ATMPsinformation also requested by the CAT, has been provided by the applicant. Likewise, this time-limit will be suspended for the time allowed for theapplicant to prepare oral or written explanations. When an application is submitted for a marketing authorization in respect of a drug which is of majorinterest from the point of view of public health and in particular from the viewpoint of therapeutic innovation, the applicant may request an acceleratedassessment procedure. If the CHMP accepts such request, the time-limit of 210 days will be reduced to 150 days but it is possible that the CHMP canrevert to the standard time-limit for the centralized procedure if it considers that it is no longer appropriate to conduct an accelerated assessment. If the CHMP concludes that the quality, safety and efficacy of the product is sufficiently proven, it adopts a positive opinion. This is sent to theEuropean Commission which drafts a decision. After consulting with the member states, the European Commission adopts a decision and grants amarketing authorization, which is valid for the whole of the European Union. The European Commission may grant a so-called "marketing authorization under exceptional circumstances". Such authorization is intended forproducts for which the applicant can demonstrate that it is unable to provide comprehensive data on the efficacy and safety under normal conditions ofuse, because the indications for which the product in question is intended are encountered so rarely that the applicant cannot reasonably be expected toprovide comprehensive evidence, or in the present state of scientific knowledge, comprehensive information cannot be provided, or it would be contraryto generally accepted principles of medical ethics to collect such information. Consequently, marketing authorization under exceptional circumstancesmay be granted subject to certain specific obligations, which may include the following:•the applicant must complete an identified programme of studies within a time period specified by the competent authority, the results ofwhich 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 only understrict medical supervision, possibly in a hospital and in the case of a radio- pharmaceutical, by an authorised person; and59Table of Contents•the package leaflet and any medical information must draw the attention of the medical practitioner to the fact that the particulars availableconcerning 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 annual re-assessment procedure. Continuation of the authorization is linked to the annual reassessment and a negative assessment could potentially result in themarketing authorization being suspended or revoked. The renewal of a marketing authorization of a medicinal product under exceptional circumstances,however, follows the same rules as a "normal" marketing authorization. Thus, a marketing authorization under exceptional circumstances is granted foran initial 5 years, after which the authorization will become valid indefinitely, unless the EMA decides that safety grounds merit one additional five-yearrenewal. The European Union also provides for a system of regulatory data and market exclusivity. According to Article 14(11) of Regulation (EC) No726/2004, as amended, and Article 10(1) of Directive 2001/83/EC, as amended, upon receiving marketing authorization, new chemical entities approvedon the basis of complete independent data package benefit from eight years of data exclusivity and an additional two years of market exclusivity. Dataexclusivity prevents regulatory authorities in the European Union from referencing the innovator's data to assess a generic (abbreviated) application.During the additional two-year period of market exclusivity, a generic marketing authorization can be submitted, and the innovator's data may bereferenced, but no generic medicinal product can be marketed until the expiration of the market exclusivity. The overall ten-year period will be extendedto a maximum of eleven years if, during the first eight years of those ten years, the marketing authorization holder obtains an authorization for one ormore new therapeutic indications which, during the scientific evaluation prior to their authorization, are held to bring a significant clinical benefit incomparison with existing therapies. Even if a compound is considered to be a new chemical entity and the innovator is able to gain the period of dataexclusivity, another company nevertheless could also market another version of the drug if such company obtained marketing authorization based on anMAA with a complete independent data package of pharmaceutical test, pre-clinical tests and clinical trials. Additional rules apply to medicinal products for pediatric use under Regulation (EC) No 1901/2006, as amended. Potential incentives include asix-month extension of any supplementary protection certificate granted pursuant to Regulation (EC) No 469/2009, however not in cases in which therelevant product is designated as an orphan medicinal product pursuant to Regulation (EC) No 141/2000, as amended. Instead, medicinal productsdesignated as orphan medicinal product may enjoy an extension of the ten-year market exclusivity period granted under Regulation (EC) No 141/2000,as amended, to twelve years subject to the conditions applicable to orphan drugs.Manufacturing and manufacturers' license Pursuant to Directive 2003/94/EC as transposed into the national laws of the member states, the manufacturing of investigational medicinalproducts and approved drugs is subject to a separate manufacturer's license and must be conducted in strict compliance with cGMP requirements, whichmandate the methods, facilities, and controls used in manufacturing, processing, and packing of drugs to assure their safety and identity. Manufacturersmust have at least one qualified person permanently and continuously at their disposal. The qualified person is ultimately responsible for certifying thateach batch of finished product released onto the market has been manufactured in accordance with cGMP and the specifications set out in the marketingauthorization or investigational medicinal product dossier. cGMP requirements are enforced through mandatory registration of facilities and inspectionsof those facilities. Failure to comply with these requirements could interrupt supply and result in delays, unanticipated costs and lost revenues, andsubject the applicant to potential legal or regulatory action,60Table of Contentsincluding but not limited to warning letters, suspension of manufacturing, seizure of product, injunctive action or possible civil and criminal penalties.Advertising In the European Union, the promotion of prescription medicines is subject to intense regulation and control, including a prohibition on direct-to-consumer advertising. All medicines advertising must be consistent with the product's approved summary of products characteristics, factual, accurate,balanced and not misleading. Advertising of medicines pre-approval or off-label is prohibited. Some jurisdictions require that all promotional materialsfor prescription medicines be subjected to either prior internal or regulatory review and approval.Other Regulatory Requirements A holder of a marketing authorization for a medicinal product is legally obliged to fulfill a number of obligations by virtue of its status as amarketing authorization holder, or MAH. The MAH can delegate the performance of related tasks to third parties, such as distributors or marketingpartners, provided that this delegation is appropriately documented and the MAH maintains legal responsibility and liability. The obligations of an MAH include:•Manufacturing and Batch Release. MAHs should guarantee that all manufacturing operations comply with relevant laws andregulations, applicable good manufacturing practices, with the product specifications and manufacturing conditions set out in themarketing authorization and that each batch of product is subject to appropriate release formalities. •Pharmacovigilance. MAHs are obliged to establish and maintain a pharmacovigilance system, including a qualified person responsiblefor oversight, to submit safety reports to the regulators and comply with the good pharmacovigilance practice guidelines adopted by theEMA. •Advertising and Promotion. MAH holders remain responsible for all advertising and promotion of their products, includingpromotional activities by other companies or individuals on their behalf and in some cases must conduct internal or regulatory pre-approval of promotional materials. •Medical Affairs/Scientific Service. MAHs are required to disseminate scientific and medical information on their medicinal products tohealthcare professionals, regulators and patients. •Legal Representation and Distributor Issues. MAHs are responsible for regulatory actions or inactions of their distributors and agents. •Preparation, Filing and Maintenance of the Application and Subsequent Marketing Authorization. MAHs must maintain appropriaterecords, comply with the marketing authorization's terms and conditions, fulfill reporting obligations to regulators, submit renewalapplications and pay all appropriate fees to the authorities. We hold the marketing authorization under exceptional circumstances granted for Glybera in the European Union and we may hold any futuremarketing authorizations granted for our product candidates in our own name, or appoint an affiliate or a collaboration partner to hold marketingauthorizations on our behalf. Any failure by an MAH to comply with these obligations may result in regulatory action against an MAH and ultimatelythreaten our ability to commercialize our products.61Table of ContentsReimbursement In the European Union, the pricing and reimbursement mechanisms by private and public health insurers vary largely by country and even withincountries. In respect of the public systems reimbursement for standard drugs is determined by guidelines established by the legislator or responsiblenational authority. The approach taken varies from member state to member state. Some jurisdictions operate positive and negative list systems underwhich products may only be marketed once a reimbursement price has been agreed. Other member states allow companies to fix their own prices formedicines, but monitor and control company profits and may limit or restrict reimbursement. The downward pressure on healthcare costs in general,particularly prescription drugs, has become very intense. As a result, increasingly high barriers are being erected to the entry of new products and someof EU countries require the completion of studies that compare the cost-effectiveness of a particular product candidate to currently available therapies inorder to obtain reimbursement or pricing approval. Special pricing and reimbursement rules may apply to orphan drugs. Inclusion of orphan drugs inreimbursement systems tend to focus on the medical usefulness, need, quality and economic benefits to patients and the healthcare system as for anydrug. Acceptance of any medicinal product for reimbursement may come with cost, use and often volume restrictions, which again can vary by country.In addition, results-based rules of reimbursement may apply.Orphan Drug Regulation In the European Union, Regulation (EC) No 141/2000, as amended, states that a drug will be designated as an orphan drug if its sponsor canestablish:•that it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not morethan five in ten thousand persons in the Community when the application is made, or that it is intended for the diagnosis, prevention ortreatment of a life- threatening, seriously debilitating or serious and chronic condition in the European Union and that without incentivesit is unlikely that the marketing of the drug in the European Union would generate sufficient return to justify the necessary investment;and •that there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorized in theEuropean Union or, if such method exists, that the drug will be of significant benefit to those affected by that condition. Regulation (EC) No 847/2000 sets out further provisions for implementation of the criteria for designation of a drug as an orphan drug. Anapplication for the designation of a drug as an orphan drug must be submitted at any stage of development of the drug before filing of a marketingauthorization application. If an EU-wide community marketing authorization in respect of an orphan drug is granted pursuant to Regulation (EC) No 726/2004, as amended,or if all the European Union member states have granted marketing authorizations in accordance with the procedures for mutual recognition, theEuropean Union and the member states will not, for a period of 10 years, accept another application for a marketing authorization, or grant a marketingauthorization or accept an application to extend an existing marketing authorization, for the same therapeutic indication, in respect of a similar drug. Thisperiod may however be reduced to six years if, at the end of the fifth year, it is established, in respect of the drug concerned, that the criteria for orphandrug designation are no longer met, in other words, when it is shown on the basis of available evidence that the product is sufficiently profitable not tojustify maintenance of market exclusivity. Notwithstanding the foregoing, a marketing authorization may be granted, for the same therapeutic indication,to a similar drug if:•the holder of the marketing authorization for the original orphan drug has given its consent to the second applicant;62Table of Contents•the holder of the marketing authorization for the original orphan drug is unable to supply sufficient quantities of the drug; or •the second applicant can establish in the application that the second drug, although similar to the orphan drug already authorized, is safer,more effective or otherwise clinically superior. Regulation (EC) No 847/2000 lays down definitions of the concepts 'similar drug' and 'clinical superiority'. Other incentives available to orphandrugs in the European Union include financial incentives such as a reduction of fees or fee waivers and protocol assistance. Orphan drug designationdoes not shorten the duration of the regulatory review and approval process.Regulation in Other Countries For other countries outside of the United States and the European Union the requirements governing the development and approval process as wellas post- approval and pricing and reimbursement requirements vary from country to country. In general, clinical studies are to be conducted inaccordance with cGCP and the applicable regulatory requirements and the ethical principles originating from the Declaration of Helsinki. If we fail to comply with applicable foreign regulatory requirements, we may be subject to, among other things, fines, suspension or withdrawal ofregulatory approvals, product recalls, seizure of products, operating restrictions and criminal prosecution.C. Organizational Structure uniQure N.V. has ten direct and indirect wholly owned subsidiaries each of which is listed in Note 1 to the financial statements which form part ofthis annual report and are also set forth in Exhibit 8.1 to this annual report. Our principal operating companies are uniQure biopharma B.V., aNetherlands company and uniQure, Inc., a Delaware corporation.D. Property, Plant and Equipment We lease a facility of approximately 26,000 square feet from the AMC, located at Meibergdreef in Amsterdam, the Netherlands, which forms ourheadquarters and principal laboratories, and also houses our manufacturing facility which the EMA has approved for clinical and commercial gradeproduction. The lease terminates in 2016. We have also leased a facility in Lexington, Massachusetts, where we have begun the build out of a 53,000square foot manufacturing facility. The lease for this facility terminates in 2024, and subject to the provisions of the lease, may be renewed for twosubsequent five year terms. We believe that our existing facilities are adequate to meet current needs and that suitable alternative spaces will be availablein the future on commercially reasonable terms. See "Operating and Financial Review and Prospects—Capital Expenditures" and "—ContractualObligations and Commitments."Item 4A Unresolved Staff Comments Not applicable.Item 5 Operating and Financial Review and Prospects You should read the following discussion and analysis of our financial condition and results of operations in conjunction with the "SelectedConsolidated Financial Information" section of this annual report and our consolidated financial statements and related notes appearing elsewhere inthis annual report. In addition to historical information, this discussion contains forward-looking statements based on our current expectations thatinvolve risks, uncertainties and assumptions. Our actual results may differ materially from those anticipated in these forward-looking statements as aresult of various factors, including those set forth in the "Risk Factors" and "Forward-Looking Statements" sections and elsewhere in this annualreport.63Table of ContentsOverview We are a leader in the field of gene therapy and have developed the first and currently the only gene therapy product to receive regulatory approvalin the European Union. Our first product, Glybera, was approved by the European Commission in October 2012 under exceptional circumstances forthe treatment of a subset of patients with lipoprotein lipase deficiency, or LPLD, a potentially life-threatening, orphan metabolic disease. We expect tolaunch Glybera commercially in selected European countries in mid 2014 through our collaboration with Chiesi, which we entered into in April 2013.We retain full commercial rights to Glybera in the United States. In August and December 2013, we met with the FDA to discuss the regulatorypathway for Glybera in the United States and we plan to file an IND with the FDA for Glybera in the first half of 2014. We are developing a pipeline ofadditional AAV-based gene therapies through multiple collaborations designed to accelerate the development and commercialization of these programs.We develop our gene therapies using our innovative, modular technology platform, including our proprietary, cost-effective manufacturing process. Our business was founded in 1998 by scientists who were investigating LPLD at the Academic Medical Center of the University of Amsterdam,or the AMC. In our early years we received funding and subsidized rent from the AMC, government grants, income for cGMP contract manufacturingof biologics for third parties, and small amounts of equity financing. From our first institutional venture capital financing in 2006 until our initial publicon February 10, 2014, we funded our operations primarily through private and public placements of equity securities, and other convertible debtsecurities, in the aggregate amount of €134.8 million ($181.9 million). During this period, we also received total other income, consisting principally ofgovernment grants and subsidies, of €5.9 million, and total nonrefundable collaboration funding of €17.0 million. Our predecessor entity, AmsterdamMolecular Therapeutics (AMT) N.V., or AMT, completed an initial public offering of its ordinary shares on Euronext Amsterdam in 2007 andsubsequently delisted from that exchange in 2012. We acquired the business of AMT in the first half of 2012. The total amounts described above include the following funds received in 2013:•€12.0 million in convertible loan financing, which we received in the first quarter of 2013, and which was converted into equity in July2013; •$10.0 million (€7.5 million) in venture debt financing, which we received in the second quarter of 2013; •€17.0 million in upfront payments from Chiesi under our collaboration agreements for Glybera and hemophilia B, which we received inJuly 2013; and •€14.0 million in equity funding from Chiesi, which we received in July 2013. As of December 31, 2013, we had cash and cash equivalents of €23.8 million. To date, we have not generated any revenues from royalties orproduct sales. We do not expect to generate royalty or revenues from product sales prior to the commercial launch of Glybera by Chiesi. We had a net loss of €26.8 million in fiscal year 2013, €14.7 million in 2012 and €17.3 million in 2011. As of December 31, 2013, we had anaccumulated deficit of €144.0 million. We anticipate that our expenses will increase substantially in the future as we:•complete our EMA-mandated post-approval clinical trial of Glybera and implement an LPLD patient registry; •conduct a clinical trial of Glybera, either as part of the EMA-mandated post-approval clinical trial or separately, to obtain data needed tofile a BLA for Glybera with the FDA; •seek marketing approval for Glybera in the United States and other countries;64Table of Contents•initiate a Phase I/II clinical trial of AMT-060 for hemophilia B in collaboration with Chiesi; •advance the preclinical and clinical development of our other product candidates, most of which are at relatively early stages ofdevelopment, and seek to discover and develop additional product candidates; •seek marketing approval for any product candidates that successfully complete clinical trials; •establish a sales, marketing and medical affairs infrastructure in the United States; •complete the building out and equipping of our manufacturing facility in Lexington, Massachusetts to expand our manufacturingcapabilities for Glybera and our pipeline of product candidates; •fund the ongoing operations of our Lexington facility; •fund expenses in connection with our new collaboration with 4D Molecular Therapeutics; •maintain, expand and protect our intellectual property portfolio, including in-licensing additional intellectual property rights from thirdparties; •hire additional personnel, particularly in our manufacturing, research, clinical development, medical affairs, commercial and qualitycontrol groups; •add operational, financial and management information systems and related finance and compliance personnel; and •operate as a public company. On February 5, 2014 we successfully completed our initial public offering, placing 5,400,000 shares at $17 per share, raising total gross proceedsof $91.8 million (€67.3 million) and net proceeds of $85.4 million (€62.6 million) after commissions but before expenses.Accounting for our Corporate Reorganization and Strategic Restructuring At the end of 2011, following the initial rejection of the application for marketing approval for Glybera in the European Union, AMT initiated astrategic restructuring in order to reduce its cost base, conserve resources and improve its financial position. As part of this effort, AMT significantlyreduced personnel, programs and expenditures. As a result, we lost a number of employees, including employees with an extensive understanding ofour clinical programs as well as our regulatory and financial affairs. AMT implemented a strategic restructuring in the fourth quarter of 2011, as a resultof which total staff was reduced from 92 to 49. AMT incurred significant restructuring expenses in connection with this reduction in staff, which wererecorded in full during the fourth quarter of 2011. Since that time, we have hired a number of new staff. As of December 31, 2013, we had a total of87 employees and engaged 33 consultants and contract workers. In the first half of 2012, we completed a corporate reorganization pursuant to which uniQure acquired the entire business of the AMT group.Pursuant to IFRS, this reorganization was treated as a reverse acquisition of AMT and accordingly, for accounting purposes, AMT was treated as theacquirer. As a result, the historical financial statements of AMT are treated as the financial statements of uniQure. See Note 1 to the audited consolidatedfinancial statements included elsewhere in this annual report for further details. At the time AMT originally prepared its audited financial statements for 2011, the business of AMT was in liquidation and therefore the relatedfinancial statements were prepared on a liquidation basis rather than a going concern basis. As of December 31, 2011, it was regarded as probable thatthe business and assets of AMT would be disposed of, and therefore AMT's assets and liabilities were recorded as assets and liabilities held for saleand its operating results were recorded as discontinued65Table of Contentsoperations. Following the corporate reorganization described above, we restated the financial information of AMT as of and for the year endedDecember 31, 2011 on a going concern basis.Collaboration and License AgreementsChiesi Agreements In April 2013, we entered into two collaboration agreements with Chiesi. In July 2013, we received an aggregate of €17.0 million in upfrontpayments from Chiesi under these agreements, as well as a €14.0 million investment in our ordinary shares.Glybera agreement Under the Glybera agreement, we granted Chiesi the exclusive right to commercialize Glybera for LPLD in the European Union and otherspecified countries, excluding the United States. In July 2013, we received a €2.0 million upfront payment in recognition of our past expendituresincurred in developing the product. In addition, we are eligible to earn up to €42.0 million in commercial milestone payments based on annual sales ofGlybera. We will receive payments for the quantities of Glybera we manufacture and supply to Chiesi, payable in part upon order and in part upon deliveryof such product quantities. We will bear the cost of goods sold for the Glybera we deliver, including the royalties and related payments to third partieswe must make under the license agreements covering various aspects of the technology underlying the composition and manufacture of Glybera. Weestimate that the amount we will retain, net of cost of goods sold, including such third party royalties and related amounts, will be between 20% and30% of the revenues from sales of Glybera by Chiesi, varying by country of sale. We believe that the amount that we will retain from net sales ofGlybera in the European Union will initially be at the lower end of this range and will increase toward the higher end of that range beginning in 2015,upon the expiration of an in-licensed patent on which we pay royalties. In addition, we are required to pay 20% of the gross amount we receive fromChiesi in respect of Glybera product sales to the Dutch government, in repayment of a technical development loan in the outstanding amount of€5.5 million as of December 31, 2013, until the earlier of repayment in full of such amount and 2017, as described below.Hemophilia B agreement Under the Hemophilia B agreement, we granted to Chiesi an exclusive license, for the European Union and specified countries other than theUnited States, to co-develop and exclusively commercialize AMT-060, a gene therapy product for the treatment of hemophilia B. We received a€15.0 million upfront payment under this agreement. Of this amount, €5.0 million related to the future development of our hemophilia B productcandidate and €10.0 million related to the use of our manufacturing capacity for our hemophilia B product candidate. In addition, we will share equallywith Chiesi specified development expenses attributable to the hemophilia B program according to a defined development plan and budget, includingexpenses associated with preclinical and clinical studies as well as development and regulatory milestone payments associated with existing in-licenseagreements. We will receive payments from Chiesi for commercial quantities of our hemophilia B product candidate we manufacture and supply tothem, if we receive regulatory approval for such product candidate. We estimate that the amount we would retain, net of cost of goods sold, includingthird party royalties and related amounts, will be between 25% and 35% of the revenues from sales of such product by Chiesi, varying by country ofsale. We and Chiesi have agreed to negotiate a separate supply and distribution agreement in respect of the potential commercialization of our hemophiliaB product candidate prior to dosing the first patient in any pivotal study. We are not entitled to any milestone payments under this co-developmentagreement.66Table of Contents4D Molecular Therapeutics In January 2014, we entered into a collaboration and license agreement with 4D for the discovery and optimization of next-generation AAVvectors. Under this agreement, we have an exclusive license to 4D's existing and certain future know-how and other intellectual property for the deliveryof AAV vectors to CNS or liver cells for the diagnosis, treatment, palliation or prevention of all diseases or medical conditions. Under thiscollaboration, the 4D team, including Dr. David Schaffer, 4D's co-founder and Professor of Chemical and Biomolecular Engineering at the Universityof California, Berkeley, will establish a laboratory, which we will fund, at a cost of approximately $3.0 million in aggregate over the next three years, toidentify next generation AAV vectors. We are also required to make payments for pre-clinical, clinical and regulatory milestones under the collaborationas well as to pay single-digit royalties. In addition, we have granted options to purchase an aggregate of 609,744 ordinary shares in connection with thiscollaboration, and will recognize resulting share-based payment expense over the next three years. To the extent that the collaboration is successful, wemay also incur additional third party costs in developing any product candidates and also in preparing, filing and prosecuting additional patentapplications.Other License Agreements We have obtained exclusive or non-exclusive rights from third parties under a range of patents and other technology that we are exploiting inGlybera and our development programs. Our agreements with these third parties generally grant us a license to make, use, sell, offer to sell and importproducts covered by the licensed patent rights in exchange for our payment of some combination of an upfront amount, annual fees, royalties, a portionof amounts we receive from our sub-licensees and payments upon the achievement of specified development, regulatory or commercial milestones. Ourpotential aggregate financial obligations under these agreements are material. Some of the agreements may also specify the extent of the efforts we mustuse to develop and commercialize licensed products. See "Information on the Company—Intellectual Property—Licenses."Financial Operations OverviewRevenues To date, we have not generated any revenues from royalties or product sales. We do not expect to generate royalty or product revenues prior to thecommercial launch of Glybera by Chiesi. When and if Chiesi generates commercial sales of Glybera, we will record the gross amounts we receive fromChiesi as product revenues. We will record the related expenses, including third party royalties and related payments, as cost of goods sold. During the year ended December 31, 2013, we recognized collaboration revenues of €2.5 million in respect of development activities that werereimbursable by Chiesi under our co-development agreement for hemophilia B. We expect to continue to recognize such collaboration revenues goingforward, in accordance with our contractual agreements. During the year ended December 31, 2013, we also recognized license revenues of €0.4 million. This amount reflects the amortization during theperiod of the non-refundable upfront payments we received from Chiesi under our collaboration agreements. The balance of €16.6 million of theselicense revenues will be recognized on a straight-line basis through the remaining period of the intellectual property protection of our manufacturingtechnologies, which is currently expected to be until September 2032. The timing of our operating cash flows may vary from the recognition of the related amounts, as we defer the recognition of some upfrontpayments, including the upfront payments under our Chiesi agreements, and recognize these as revenue when earned or over a defined period, while wetreat other revenue, such as milestone payments or service fees, as earned when received. We expect our revenues67Table of Contentsto vary from quarter to quarter and year to year, depending upon, among other things, the commercial success of Glybera, our success in obtainingmarketing approval for Glybera in the United States and additional countries, the structure and timing of milestone events, the number of milestonesachieved, the level of revenues earned for ongoing development efforts, any new collaboration arrangements we may enter into and the terms we areable to negotiate with our collaborators. We currently intend to sell Glybera in the United States, if approved, ourselves, in which case we wouldrecognize revenues in the full amount of the sales price. In addition, because LPLD is an orphan disease and we expect that the number of patients thatwill be treated with Glybera is relatively small, and because we currently expect that we will receive a one-time payment for a single patient treatment,we anticipate that revenues from Glybera may vary significantly from period to period. Further, because we currently anticipate that LPLD patients willrequire only a single administration with Glybera, we do not expect to earn recurring revenue from treated patients. We therefore believe that period toperiod comparisons should not be relied upon as indicative of our future revenues.Other Income Our other income consists principally of government grants, subsidies and investment credits that support our research efforts in defined researchand development projects, which we refer to as grants. These grants generally provide for reimbursement of our approved expenses incurred as definedin various grants. We recognize grants when expenses are incurred in accordance with the terms and conditions of the grant and the collectability of thereceivable is reasonably assured. Because we have limited or no control over the timing of receipt of grants, the amount of other income varies fromperiod to period and is not indicative of underlying trends in our operations. We have received grants from the Dutch government and from the European Union. We have also participated in collaborations and consortia inwhich our collaborators and fellow consortium members have received grants from governmental authorities, which have enabled us to accesspreclinical and clinical data while minimizing the expenses we incur. We have received a research and development subsidy from the Dutch government in the form of reimbursement of payroll taxes related to relevantemployees. The amount we receive is tied directly to the number of employees and number of hours devoted to specified research and developmentprograms, and therefore varies directly with the size of our workforce and direction of our research and development programs. We have no obligationto repay these amounts. Some of the grants we have received are repayable under specified circumstances. In particular, we would be required to repay some grants if wesuccessfully commercialize a supported program within a specified timeframe. None of the grants we have received to date relate to programs that wecurrently anticipate commercializing, other than the technical development loan in respect of Glybera, described under "Costs of Goods Sold" below.Accordingly, we do not currently expect that we will be required to repay any of these grants. Other income also includes amounts we receive as payment or reimbursement for expenses of manufacturing and development of AMT-110 underour collaboration agreement with Institut Pasteur.Cost of Goods Sold Cost of goods sold includes the purchase price of raw materials, directly attributable labor costs and directly related charges by third party serviceproviders, and the royalties and other related payments to third parties we must make under the license agreements covering various aspects of thetechnology underlying the composition and manufacture of Glybera. We also include in cost of goods sold amounts that we are required to repay to the Dutch government in respect of a technical development loanthat we received in the period from 2000 to 2005 to support the early development of Glybera. As of December 31, 2013, the total amount of68Table of Contentsprincipal and interest outstanding was €5.5 million. Under the terms of this contingent commitment, we are required to make repayments based on thetiming and amount of revenues we receive from product sales of Glybera. In connection with our receipt of upfront payments from Chiesi for thecommercialization of Glybera, we repaid €0.8 million of this loan in September 2013, which we recorded as cost of goods sold although no productsales occurred. No further payments will be made until sales of Glybera commence. We expect to pay to the Dutch government 20% of any grossamounts we receive from Chiesi in connection with sales of Glybera, as and when received, until the earlier of such time as the loan is repaid in full orDecember 31, 2017. Amounts that remain outstanding as of December 31, 2017, if any, would be forgiven. We have not recorded any liability for theseamounts. To the extent we generate revenues from the sale of Glybera, we will recognize a liability and a corresponding charge to cost of goods sold infuture periods. Should we obtain marketing approval in the United States for Glybera, we expect that our costs of goods sold for sales of Glybera in the UnitedStates would be significantly lower than our costs of goods sold for sales of Glybera in the European Union due principally to the existence of lowerroyalty obligations on U.S. sales.Research and Development Expenses Research and development expenses consist principally of expenses associated with employees, manufacturing facilities, clinical development,collaboration with third parties, license fees, laboratory consumables and depreciation. During the period from 2006, when we received our first significant venture capital equity investment, to December 31, 2013, we incurred anaggregate of €96.7 million in research and development expenses. Our total research and development expenses in 2013 were €13.2 million. In addition,we began to capitalize our development expenses related to Glybera from March 21, 2013. We capitalized €3.1 million of such expenses in fiscal year2013, which we expect to begin amortizing once sales of Glybera commence, over the period through September 2032. We allocate our direct researchand development expenses to our various programs on the basis of actual external expenses incurred in respect of each program and our allocation oftime spent by our research and development team on each program. We do not allocate our overhead expenses to specific development programs. Ourresearch and development expenses mainly relate to the following key programs:•Glybera. We are undertaking preparations for the EMA-mandated post-approval clinical trial and patient registry. In addition, we areundertaking preparations for the submission of an IND with the FDA in the first half of 2014. We bear all of the costs of this programoutside of the territories covered by the Chiesi agreement. Certain costs, including the patient registry for territories covered by the Chiesiagreement, will be shared equally with Chiesi. •Hemophilia B. We plan to initiate a Phase I/II clinical trial of AMT-060 for the treatment of hemophilia B in the second half of 2014 incollaboration with Chiesi. Under our co-development agreement, we and Chiesi will each bear half of the development costs of thisprogram. •Acute intermittent porphyria (AIP). We have incurred costs related to the development and manufacture of clinical supplies of AMT-021for the treatment of AIP provided to our collaboration partner, Digna Biotech, for its ongoing Phase I clinical trial in this indication. •CNS programs. We have incurred costs related to the development and manufacture of clinical supplies of AMT-110 for the treatmentof Sanfilippo B provided to our collaboration partner, Institut Pasteur, for its ongoing Phase I/II clinical trial. We also incur expensesrelated to the research and preclinical activities related to our other CNS programs. •Technology platform development and other research. We incur significant research and development costs related to our gene deliveryand manufacturing technology platform that are69Table of Contentsapplicable across all of our programs, as well as our other research programs, including intellectual property expenses, depreciationexpenses and facility costs. These costs are not allocated to specific projects. The table below sets forth our direct research and development expenses by program for the years ended December 31, 2011, 2012 and 2013. Our research and development expenses may vary substantially from period to period based on the timing of our research and developmentactivities, including regulatory approvals and enrollment of patients in clinical trials. We expect that our research and development expenses will increasesignificantly as we increase our staff, conduct further clinical development of Glybera, advance the research and development of our other productcandidates and commence manufacturing at our manufacturing facility in Lexington, Massachusetts. The successful development of our productcandidates is highly uncertain. At this time, we cannot reasonably estimate the nature, timing or estimated costs of, or any cash inflows resulting from,the development of any of our product candidates. This is due to numerous risks and uncertainties associated with developing gene therapies, includingthe uncertainty of:•the scope, rate of progress and expense of our research and development activities; •clinical trial and early-stage results; •the terms and timing of regulatory approvals; •the expense of filing, prosecuting, defending and enforcing patent claims and other intellectual property rights; and •our and our collaborators' ability to market, commercialize and achieve market acceptance for Glybera or any other product candidate thatwe may develop in the future. A change in the outcome of any of these variables with respect to the development of Glybera or any other product candidate that we may developcould mean a significant change in the expenses and timing associated with the development of Glybera or such product candidate. For example, if theFDA or another regulatory authority were to require us to conduct preclinical and clinical studies for Glybera or any other product candidate beyondthose which we currently anticipate will be required for the completion of clinical development, or if we experience significant delays in enrollment inany clinical trials, we could be required to expend significant additional financial resources and time on the completion of our clinical development. See"Risk Factors—Risks Related to the Development of Our Product Candidates" and "—Risks Related to the Regulatory Approval of Our ProductCandidates". We have incurred significant expenses in the development of Glybera. Under applicable accounting principles, we capitalize development expensesupon receipt of marketing approval for a product70 YEAR ENDED DECEMBER 31, (€ in thousands, except percentages) 2011 2012 CHANGE 2013 CHANGE % % Glybera program* 4,381 1,055 (76)% 2,727 158%Hemophilia B program 671 1,131 69% 3,034 168%AIP program 1,383 1,055 (24)% 241 (77)%CNS programs 363 922 154% 822 (11)%Technology platform development andresearch programs 8,702 6,068 (30)% 6,358 5% Total 15,500 10,231 (34)% 13,182 29% *Excludes capitalized development expenses of €3.1 million in 2013 (2011 and 2012: nil).Table of Contentscandidate, provided that we have the technical, scientific and financial resources to complete the development and commercialization of the program. Wereceived marketing approval from the European Commission for Glybera for a subset of LPLD patients in October 2012. Because we did not havesufficient financial resources at that time to complete the development of Glybera, including the post-approval activities required by the EMA prior tocommercial launch, we did not capitalize the development expenses related to Glybera during the year ended December 31, 2012. Following our receiptof an additional €10.0 million in convertible debt financing in the first quarter of 2013, we determined that we had sufficient financial resources tocomplete these post-approval activities, and accordingly began to capitalize the related development expenses in the first quarter of 2013. Over the period through 2016, we anticipate that we will incur external expenses related to the further development of Glybera, includingimplementation of the patient registry, initiation and conduct of the post-approval clinical trial and additional development work to seek FDA approval,of approximately €7.0 million; in addition, we will incur significant related employee expenses. See "Risk Factors—Risks Related to the Developmentof Our Product Candidates" and "—Risks Related to the Regulatory Approval of Our Product Candidates." In addition, in connection with the collaboration and license agreement we entered into with 4D Molecular Therapeutics during January 2014, wewill incur additional expenses as we fund a joint research effort with 4D. Further, we granted options to purchase an aggregate of 609,744 of ourordinary shares to two consultants who will be providing services to us in connection with that agreement. The fair value of these options will vest overa future service period, and will have a significant impact on our expenses recognized. Finally, to the extent certain pre-clinical, clinical and regulatorymilestones are met, we will make milestone payments to 4D. See "Information on the Company—Strategic Collaboration: 4D Molecular Therapeutics."Selling, General and Administrative Expenses Our selling, general and administrative expenses have consisted to date principally of employee, office, consultancy and other administrativeexpenses. We expect that our selling, general and administrative expenses will increase significantly in the future as our business expands and we addpersonnel, particularly in our medical affairs, commercial, quality control, finance and compliance groups, and as we commence manufacturingoperations in our facility in Lexington, Massachusetts. We also expect to incur additional expenses associated with operating as a public company,including expenses for additional personnel, additional legal, accounting and audit fees, directors' and officers' liability insurance premiums andexpenses related to investor relations. In future periods, we will include in selling, general and administrative expenses our sales expenses related to thecommercialization of Glybera in the European Union, including our market access and medical affairs efforts, as well as the costs related to the sales andmarketing efforts we intend to undertake in the United States in advance of potential marketing approval for Glybera from the FDA.Other Losses—Net Other losses—net consists of foreign exchange losses that do not relate to borrowings. We are exposed to foreign exchange risk arising fromvarious currency exposures, primarily with respect to the U.S. dollar and, to a lesser extent, the British pound, as we acquire certain materials and payfor certain licenses and other services in these two currencies. We have not established any formal practice to manage the foreign exchange risk againstour functional currency.Finance Income Our finance income consists of interest income earned on our cash and cash equivalents and gains on our derivative instruments, described below.We deposit our cash and cash equivalents primarily in71Table of Contentssavings and deposit accounts with original maturities of three months or less. Savings and deposit accounts have historically generated only minimalinterest income. We have entered into various financing arrangements with our investors, including convertible notes issued in 2009 and converted into ordinaryshares in April 2012, and further convertible notes issued in 2012 and 2013, which were converted into ordinary shares in July 2013. See "RelatedParty Transactions" for further detail. Each of the convertible notes consists of a debt element and an embedded financial derivative element. Derivativesare initially recognized at fair value on the date a derivative contract is entered into and are subsequently measured at fair value through profit and loss.The resulting gain is recognized in the consolidated income statement and accounted for as finance income.Finance Expense Finance expense consists primarily of interest due on our convertible notes, losses on the fair value measurements of our derivative instruments,and, to a lesser extent, the interest component of finance leases.A. Operating ResultsOverview Our results of operations in the periods under review were significantly affected by the corporate reorganization and strategic restructuring, andrelated contraction of our research and development and other activities, that we initiated at the end of 2011 in order to conserve resources and improveour financial position following the initial rejection of the application for marketing approval for Glybera in the European Union. Following the approvalof Glybera in the European Union in October 2012 and additional investment received in the first quarter of 2012, we began to significantly expand ouroperations.Comparison of the year ended December 31, 2011, 2012 and 201372 YEAR ENDED DECEMBER 31, (€ in thousands) 2011 2012 2013 Revenues: License revenues — — 440 Collaboration revenues — — 2,503 Total revenues — — 2,943 Cost of goods sold — — (800)Other income 2,192 649 585 Expenses: Research and development expenses (15,500) (10,231) (13,182)Selling, general and administrative expenses (3,807) (4,564) (11,628)Other losses, net (26) (45) (453) Operating result (17,141) (14,191) (22,535)Finance income 277 22 102 Finance expense (436) (547) (4,387) Net loss (17,300) (14,716) (26,820) Table of ContentsRevenues License revenues of €0.4 million in the year ended December 31, 2013 related to the amortization of the upfront payment received from Chiesi inJuly 2013. Collaboration revenues of €2.5 million in the year ended December 31, 2013 consisted mainly of reimbursements of covered expenses by Chiesiunder our agreements (€2.2 million), together with revenue from Institut Pasteur relating to our Sanfilippo B collaboration (€0.3 million). We had norevenues in the years ended December 31, 2012 or 2011.Cost of Goods Sold Cost of goods sold of €0.8 million in the twelve months ended December 31, 2013 consisted of the recognition of a repayment obligation to theDutch government with respect to a portion of a technical development loan. This repayment obligation was triggered by our entitlement to receiveduring the second quarter of 2013 a €2.0 million upfront payment from Chiesi in relation to our Glybera program. We had no cost of goods sold in theyears ended the year ended December 31, 2012 or 2011.Other Income Other income for the year ended December 31, 2013 was €0.6 million, was in line with the €0.6 million recognized for the year endedDecember 31, 2012. This income represented reimbursement of payroll taxes received from the Dutch government and the receipt of grants to supportresearch projects. Other income for the year ended December 31, 2012 was €0.6 million, a 70% decrease from the €2.2 million recognized for the year endedDecember 31, 2011. The higher amounts in 2011 reflected a grant in the amount of €1.0 million accounted for in that period from the European Unionthrough our collaborator in connection with our AIP program, as well as €0.8 million from our collaborator Institut Pasteur related to the supply by usof material for use in our Sanfilippo B program. The reduction in the amount of Other income in 2012 reflects the variable nature of payments receivableunder these arrangements.Research and Development Expenses Research and development expenses for the year ended December 31, 2013 were €13.2 million, a 29% increase from the €10.2 million incurred forthe twelve months ended December 31, 2012. This increase reflected the expansion of our research and development activities to support the furtherdevelopment of our pipeline product candidates as well as expenditure during the first quarter of 2013 on development to support the plannedcommercial launch of Glybera in the European Union (from March 21, 2013 onwards Glybera development expenditure in the European Union hasbeen capitalized). Following our receipt of additional convertible loan and debt funding in the first nine months of 2013, we increased the level ofresearch and development expenditures compared with the relatively low level of expenditure during 2012 attributable to our strategic restructuring atthe end of 2011. Glybera-related raw materials that cannot be used for commercial purposes since March 2013 are capitalized as development costs (prior to March2013 they were expensed); Glybera-related materials, including raw materials, work-in- progress and finished goods, that are expected to be used forcommercial purposes are recorded as inventory on the balance sheet and are not accounted for within research and development expenses. Research and development expenses for the year ended December 31, 2012 were €10.2 million, a 34% decrease from the €15.5 million incurredfor the year ended December 31, 2011. The decrease reflected the strategic restructuring and related reduction in our workforce we undertook at the endof73Table of Contents2011. Following the reduction in staff, we also reduced our overall level of activity. Furthermore, during the first half of 2012, we focused on our early-stage programs, which generally require less investment than more advanced programs.Selling, General and Administrative Expenses Selling, general and administrative expenses for the year ended December 31, 2013 were €11.6 million, a 155% increase from the €4.6 millionincurred for the year ended December 31, 2012. This increase resulted principally from our increased headcount in 2013 as we continued to ramp upour operations following our strategic restructuring at the end of 2011, share-based expenses relating to the costs of warrants and options grantedduring the period, and increased commercial, legal and other advisory fees. Selling, general and administrative expenses for the year ended December 31, 2012 were €4.6 million, a 20% increase from the €3.8 millionincurred for the year ended December 31, 2011. This increase reflected principally increased legal and other advisory costs incurred in 2012 inconnection with our corporate reorganization, described above, and to a lesser extent expanded business development activities in 2012.Other losses—Net Other losses—net for the year ended December 31, 2013 were a loss of €0.5 million; a 907% increase from the loss of €0.05 million for the yearended December 31, 2012, and related to foreign exchange impacts. This increase reflects changes in the exchange rate between the euro and theU.S. dollar. Other losses—net were not material in 2011.Finance Income Finance income for the year ended December 31, 2013 was €0.1 million, a 364% increase from the €0.02 million for the year ended December 31,2012, and a 92% decrease from the €0.3 million for the year ended December 31, 2011. This reflects our average cash balances and low interest rates inboth periods.Finance Expense Finance expense for the year ended December 31, 2013 was €4.4 million, compared with €0.5 million for the year ended December 31, 2012. Thisincrease primarily related to the revaluation and/or early conversion of the embedded derivatives related to our convertible loans and the venture loan,which totaled €3.5 million during the year ended December 31, 2013. Finance expense remained relatively stable at €0.5 million for the year ended December 31, 2012 compared with €0.4 million for the year endedDecember 31, 2011, principally representing interest due on convertible loans in 2011, and the charge on the movement in the value of the derivativeelement of our convertible loans, which were converted on our restructuring in April 2012.B. Liquidity and Capital Resources In our early years we received funding and subsidized rent from the AMC, government grants, income for cGMP contract manufacturing ofbiologics for third parties, and small amounts of equity financing. From our first institutional venture capital financing in 2006 until our initial public onFebruary 10, 2014, we funded our operations primarily through private and public placements of equity securities, and convertible and other debtsecurities, in the aggregate amount of €134.8 million ($181.9 million). During this period, we also received total other income, consisting principally of74Table of Contentsgovernment grants and subsidies, of €5.9 million, and total nonrefundable collaboration funding of €17.0 million, and $10.0 million (€7.5 million) inventure debt financing. We had a net loss of €26.8 million in the year ended December 31, 2013, €14.7 million in full year 2012 and €17.3 million in full year 2011. As ofDecember 31, 2013, we had an accumulated deficit of €144.0 million. On February 5, 2014 we successfully completed our initial public offering,placing 5,400,000 shares at $17 per share, raising total gross proceeds of $91.8 million (€67.3 million) and net proceeds of $85.4 million(€62.6 million) after commissions but before expenses.Cash flows Our cash and cash equivalents as of December 31, 2013 were €23.8 million. The table below summarizes our consolidated cash flow data for theyears ended December 31, 2011, 2012 and 2013:Net Cash Used in Operating Activities Net cash used in operating activities was €4.1 million in the year ended December 31, 2013, a 63% decrease from net cash used in operatingactivities of €11.3 million in the year ended December 31, 2012. The change reflected the receipt of the upfront payment under our collaborationagreements with Chiesi, for a total of €17.0 million. Net cash used in operating activities was €11.3 million in 2012, a 33% decrease from €16.7 million in 2011. The decrease reflected the reduction innet loss before corporate income tax for 2012 compared to 2011, which in turn was due to the strategic restructuring and related reduction in ourworkforce we undertook at the end of 2011. Following the reduction in staff we also reduced our overall activity. In 2012 our net loss before corporateincome tax was €14.7 million, a decrease of €2.6 million compared to 2011. In addition, changes in overall composition of our working capital balancealso resulted in an overall reduction in the cash used in operations.Net Cash Used in Investing Activities Net cash used in investing activities was €6.0 million in the year ended December 31, 2013, compared with net cash used in investing activities of€0.8 million in the year ended December 31, 2012. The increase reflected the capitalization of €3.1 million of Glybera development expenses beginningin March 2013, as well as the capitalization of payments due to licensors following the upfront payment under our collaboration agreements with Chiesi. Net cash used in investing activities was €0.8 million in 2012, an increase of 414% from €0.2 million in 2011. This increase was due to purchasesof intangible assets and, to a lesser extent, purchases of property, plant and equipment.Net Cash Generated from Financing Activities Net cash generated from financing activities was €33.6 million in the year ended December 31, 2013, compared with net cash generated fromfinancing activities of €11.3 million in the year ended December 31, 2012. The increase reflected the receipt of €12.0 million in funding from theissuances of convertible notes (all of which were fully converted in the period), $10.0 million in funding from a75 YEAR ENDEDDECEMBER 31, (€ in thousands) 2011 2012 2013 Net cash used in operating activities (16,705) (11,277) (4,136)Net cash used in investing activities (162) (832) (5,971)Net cash generated from financing activities 108 11,272 33,642 Table of Contentsventure loan and the receipt of the €14.0 million equity investment from Chiesi during year ended December 31, 2013. Net cash generated from financing activities was €11.3 million in year ended December 31, 2012, compared with €0.1 million in year endedDecember 31, 2011. The increase reflected our private placements of convertible notes and equity securities in year ended December 31, 2012 inconnection with and following our corporate reorganization.Cash and Funding Sources The table below summarizes our sources of financing for the years ended December 31, 2011, 2012 and 2013: Our sources of financing in the year ended December 31, 2013 were:•the issuance and sale of 90,747 ordinary shares to our employees for gross proceeds of €0.3 million; •the issuance and sale of €12.0 million of our convertible notes; •a venture loan in the principal amount of $10.0 million from Hercules Technology Growth Capital, or Hercules, pursuant to a loan andsecurity agreement dated June 14, 2013, or the Hercules Agreement; and •the acquisition of 1,109,214 ordinary shares by Chiesi for €14.0 million. As of December 31, 2013, we had debt of $10 million, equivalent to €7.5 million, which consisted solely of amounts outstanding under theHercules Agreement.Funding Requirements We believe our cash and cash equivalents, including proceeds of our initial public offering in February 2014, will enable us to fund our operatingexpenses, including our debt repayment obligations as they become due, and capital expenditure requirements, including the build-out of our Lexington,Massachusetts facility, for at least the next 12 months. We have based this estimate on assumptions that may prove to be incorrect, and we could use ourcapital resources earlier than we currently expect. Our future capital requirements will depend on many factors, including:•the commercial success of Glybera, including the timing and amount of revenues generated, as well as our cost of goods sold; •our collaboration agreements remaining in effect and our ability to obtain research and development funding and achieve milestonesunder these agreements; •the progress and results of our current and planned clinical trials, including for Glybera, and those of our collaborators;76(€ in thousands) EQUITYCAPITAL(1) CONVERTIBLENOTES OTHERDEBT TOTAL Year ended December 31, 2013 14,294 11,999 7,492 33,785 Year ended December 31, 2012 9,774 1,498 — 11,272 Year ended December 31, 2011 108 — — 108 Total 24,176 13,497 7,492 45,165 (1)Excludes shares issued upon conversion of convertible notes.Table of Contents•the scope, progress, results and costs of preclinical development, laboratory testing and clinical trials for our additional productcandidates; •the number and development requirements of other product candidates that we pursue; •the cost, timing and outcome of regulatory review of our product candidates, particularly for approval of Glybera in the United States; •the cost and timing of future commercialization activities by us or our collaborators, including product manufacturing, marketing, salesand distribution, for Glybera and any of our product candidates for which we receive marketing approval in the future; •the amount and timing of revenue, if any, we receive from commercial sales of any product candidates for which we receive marketingapproval in the future; •expenses in connection with our collaboration with 4D Molecular Therapeutics; •the costs and timing of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rightsand defending any intellectual property-related claims; •the extent to which we acquire or in-license other products or technologies; and •the cost and progress of the build-out of our Lexington, Massachusetts manufacturing facility. We have no committed sources of additional financing, other than our collaboration agreements with Chiesi. Until such time, if ever, as we cangenerate substantial product revenues from sales of Glybera by Chiesi or otherwise, we expect to finance our cash needs through a combination ofequity offerings, debt financings, collaborations, strategic alliances and marketing, distribution and licensing arrangements. We are subject to covenantsunder the Hercules Agreement, and may become subject to covenants under any future indebtedness, that could limit our ability to take specific actions,such as incurring additional debt, making capital expenditures or declaring dividends, which could adversely impact our ability to conduct our business.In addition, our pledge of assets as collateral to secure our obligations under the Hercules Agreement may limit our ability to obtain debt financing. Ifwe raise additional funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may haveto relinquish valuable rights to our technologies, future revenue streams or product candidates or grant licenses on terms that may not be 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 ourproduct development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer todevelop and market ourselves. For more information as to the risks associated with our future funding needs, see "Risk Factors—Risks Related to Our Financial Position andNeed for Additional Capital".Capital Expenditures The following table sets forth our capital expenditures for the years ended December 31, 2011, 2012, and 2013.77 YEAR ENDEDDECEMBER 31, (€ in thousands) 2011 2012 2013 Investments in property, plant and equipment 200 392 1,336 Investments in intangible assets 109 553 4,652 Total 309 945 5,988 Table of Contents We are currently building out a 53,000 square foot leased manufacturing facility in Lexington, Massachusetts. We anticipate that the totalconstruction costs will amount to approximately $16.4 million (€ 11.9 million), of which the landlord is obligated to pay $7.3 million (€5.3 million) inlandlord improvements. In addition, we anticipate the total investment in property, plant and equipment to be approximately $6.4 million (€4.7 million).As of December 31, 2013, we had capitalized $1.8 million (€1.3 million) and had contractual commitments of a further $14.9 million (€10.8 million). Inaddition, we provided a landlord deposit of $1.2 million (€0.9 million). We anticipate that we will have paid the full amount of these build-out costs bythe end of the second quarter of 2014. We also anticipate that we will incur additional capital expenditures related to our planned expansion of our facility in Amsterdam.Hercules Loan and Security Agreement We are party to a Loan and Security Agreement entered into with Hercules on June 13, 2013. Under the Loan and Security Agreement, weborrowed $10.0 million (€7.5 million) from Hercules, bearing interest at a variable rate of the greater of 11.85% or an amount equal to 11.85% plus theprime rate of interest minus 3.25%. We are required to pay only interest in monthly payments until October 2014. From October 2014, we will berequired to make monthly payments of interest and principal in the amount of $387,000 (€281,000). The loan matures on October 1, 2016, when wewill be required to make a final payment of $2.6 million (€1.9 million). The loan and security agreement also provides for payment of a maturity charge,the amount of which was reduced in exchange for the issuance to Hercules, on September 24, 2013, of 37,174 warrants, at an exercise price of $13.45per share. We have pledged substantially all of our assets as collateral to the Hercules loan, by means of a first ranking right of pledge. The Loan and SecurityAgreement contains covenants that restrict our ability to, among other things, incur future indebtedness and obtain additional financing, to makeinvestments in securities or in other companies, to transfer our assets, to perform certain corporate changes, to make loans to employees, officers anddirectors, and to make dividend payments and other distributions. Further, we are required to keep a minimum cash balance deposited in bank accountsin the United States, equivalent to the lesser of the outstanding balance of principal due and 50% of our worldwide cash reserves. The Loan andSecurity Agreement contains default provisions that include the occurrence of a material adverse effect, as defined therein, which would entitle Herculesto declare all principal, interest and other amounts owed by us immediately due and payable.Quantitative and Qualitative Disclosures about Market Risk We are exposed to a variety of financial risks, including market risk (including currency risk, price risk and cash flow and fair value interest raterisk), credit risk and liquidity risk. Our overall risk management program focuses on preservation of capital and the unpredictability of financial marketsand has sought to minimize potential adverse effects on our financial performance and position.Market Risk We are exposed to foreign exchange risk arising from various currency exposures, primarily with respect to the U.S. dollar, particularly as weexpand our operations in the United States and build-out our manufacturing facility in Lexington, Massachusetts. We have not established any formalpractice to manage the foreign exchange risk against our functional currency. Our loan from Hercules, was received and is repayable in U.S. dollars,and in the fourth quarter of 2013, we incurred obligations in U.S. dollars in respect of our manufacturing facility in Lexington, Massachusetts, asdescribed above.78Table of Contents Our interest rate risk arises from short and long-term borrowings. As of December 31, 2012, we had no borrowings with variable rates and wewere not exposed to cash flow interest rate risk. In June 2013, we entered into the Hercules Agreement under which our borrowings bear interest at avariable rate. Borrowings issued at fixed rates expose us to fair value interest rate risk. As of December 31, 2012, we had neither significant long-term interest-bearing assets nor significant long-term interest bearing liabilities otherthan our convertible notes, which were subsequently converted into ordinary shares on July 26, 2013. As of December 31, 2013, the loans issued underthe Hercules Agreement bore interest at the rate of the greater of 11.85% and an amount equal to 11.85% plus the prime rate of interest minus 3.25%.Credit Risk We have a limited group of material external counterparties, of which the most significant is Chiesi. Over the coming years, funding under ourcollaboration and co-development agreements with Chiesi, including milestone payments, collaboration revenues and reimbursable research expenses,remains critical for our product development programs and represents our principal credit risk. Our cash and cash equivalents are invested primarily in savings and deposit accounts with original maturities of three months or less. Savings anddeposit accounts generate a small amount of interest income. For banks and financial institutions, we accept only independently rated parties with aminimum rating of 'A-'.Liquidity Risk We believe that our existing cash and cash equivalents, including proceeds of our initial public offering in February 2014, and anticipated paymentsunder our agreements with Chiesi will enable us to fund our operating expenses and capital expenditure requirements for at least the next 12 months.Internal Control Over Financial Reporting In connection with the preparation and external audit of our consolidated financial statements as of and for the year ended December 31, 2012 and2013, we and our auditors, an independent registered public accounting firm, noted three material weaknesses in our internal control over financialreporting. The material weaknesses identified were:•a lack of accounting resources required to fulfill IFRS and SEC reporting requirements, •a lack of comprehensive IFRS accounting policies and financial reporting procedures; and •a lack of segregation of duties given the size of our finance and accounting team. We have implemented and are continuing to implement various measures to address the material weaknesses identified; these measures are outlinedbelow. Neither we nor our independent registered public accounting firm undertook a comprehensive assessment of our internal control for purposes ofidentifying and reporting material weaknesses, significant deficiencies and control deficiencies in our internal control over financial reporting as we willbe required to do now that, effective February 10, 2014, we are a public company. We believe it is possible that, had we performed a formal assessmentof our internal control over financial reporting or had our independent registered public accounting firm performed an audit of our internal control overfinancial reporting, additional control deficiencies may have been identified. We have progressed the evaluation of our internal control over financial reporting. We have also taken several remedial actions to address thematerial weaknesses that have been identified. To this end, we have hired additional staff for the finance department who have external reporting andIFRS79Table of Contentsexperience, and experience with establishing appropriate financial reporting policies. Moreover, we have engaged a team of external consultants to assistus to improve our corporate governance and internal control procedures and help us design and implement a structured control environment forcomplying with the Sarbanes-Oxley Act of 2002, and we have devoted significant efforts to remedy any deficiencies or control gaps identified in theprocess. We expect to complete the measures above as soon as practicable and we will continue to implement measures to remedy our internal controldeficiencies in order to meet the deadline imposed under Section 404 of the Sarbanes-Oxley Act. However, the implementation of these measures maynot fully address the existing material weaknesses in our internal control over financial reporting, and we cannot yet conclude that they have been fullyremedied. The process of designing and implementing an effective financial reporting system is a continuous effort that requires us to anticipate and react tochanges in our business and the economic and regulatory environments and to expend significant resources to maintain a financial reporting system thatis adequate to satisfy our reporting obligations. See "Information on the Company—Risk Factors—Risks Related to our Ordinary Shares—If we fail toimplement and maintain an effective system of internal control, we may be unable to accurately report our results of operations or prevent fraud or fail tomeet our reporting obligations, and investor confidence and the market price of our ordinary shares may be materially and adversely affected."Critical Accounting Policies and Significant Judgments and Estimates Our operating and financial review and prospects is based on our consolidated financial statements, which we have prepared in accordance withIFRS as issued by the IASB. The preparation of these financial statements requires us to make estimates and assumptions that affect the reportedamounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reportedrevenues and expenses during the reporting periods. Actual results may differ from these estimates under different assumptions or conditions. Our significant accounting policies are more fully described in the notes to our consolidated financial statements appearing elsewhere in this annualreport. We believe that the following accounting policies involve the most significant judgments and estimates by management and are the most criticalto fully understanding and evaluating our financial condition and results of operations.Revenue Recognition We did not generate any revenues from royalties or product sales for 2011 or 2012. During 2013, we received upfront payments in connection with our Glybera commercialization agreement and hemophilia B co-developmentagreement, each with Chiesi. Revenues from such non-refundable, up-front payments are initially reported as deferred revenues on the consolidatedbalance sheet and are recognized in revenues on the income statement as earned over the period of the development, commercialization, collaboration ormanufacturing obligation. We also generate revenues from collaborative research and development arrangements. Such agreements may consist of multiple elements andprovide for varying consideration terms, such as up-front, milestone and similar payments, which require significant analysis by management in order todetermine the appropriate method of revenue recognition. Where such arrangements can be divided into separate units of accounting (each unit constituting a separate earnings process), the arrangementconsideration is allocated to the different units based on their relative fair values and recognized over the respective performance period. Where thearrangement cannot be divided into separate units, the individual deliverables are combined as a single unit of accounting and the total arrangementconsideration is recognized over the estimated80Table of Contentscollaboration period. This analysis requires considerable estimates and judgments to be made by us, including estimates of the relative fair values of thevarious elements included in such agreements and the estimated length of the respective performance periods. Non-refundable upfront payments received from Chiesi related to licenses and reimbursement of past development costs for Glybera and ourhemophilia B program. We have concluded that the elements of the payments are linked in such a way that the commercial effect cannot be understoodwithout reference to the series of transactions as a whole. Therefore the individual performance obligations have been treated as a single unit ofaccounting and the total arrangement consideration is recognized over the estimated life of the agreements under which the continuing performanceobligations exist.Research and Development Expenses We recognize research expenses as incurred. We recognize expenses incurred on development projects as intangible assets as of the date that it canbe established that it is probable that future economic benefits that are attributable to the asset will flow to us, considering the development projects'commercial and technological feasibility, generally when we receive regulatory approval for commercial sale, and when expenses can be measuredreliably. Given the stage of the development of our products and product candidates, we did not capitalize any development expenditures prior to 2013.As noted above, we incurred significant expenses in the development of Glybera. We received marketing approval from the European Commission forGlybera for a subset of LPLD patients in October 2012. Because we did not have sufficient financial resources at that time to complete the developmentof Glybera, including the post-approval activities required by the EMA prior to commercial launch, however, we did not capitalize the developmentexpenses related to Glybera during the year ended December 31, 2012. Following our receipt of an additional €10.0 million in convertible debtfinancing in the first quarter of 2013, we determined that we had sufficient financial resources to complete these post-approval activities, andaccordingly began to capitalize the related development expenses from March 21, 2013. Glybera-related raw materials that cannot be used forcommercial purposes are expensed; Glybera-related materials, including raw materials, work-in-progress and finished goods, that are expected to beused for commercial purposes are recorded as inventory on the balance sheet and are not accounted for within research and development expenses. As of each balance sheet date, we estimate the level of service performed by our vendors or other counterparties and the associated costs incurredfor the services performed. As part of the process of preparing our financial statements we are required to estimate our accrued expenses. This processinvolves reviewing quotations and contracts, identifying services that have been performed on our behalf, estimating the level of service performed andthe associated costs incurred for the service when it has not yet been invoiced or we have not otherwise been notified of the actual costs. The majority ofour service providers invoice us monthly in arrears for services performed or when contractual milestones are met. We make estimates of our accruedexpenses as of each balance sheet date in our financial statements based on facts and circumstances known to us at that time. We periodically confirmthe accuracy of our estimates with the service providers and make adjustments if necessary. The significant estimates in our accrued research anddevelopment expenses are related to fees paid to clinical research organizations, or CROs, in connection with research and development activities forwhich we have not yet been invoiced. We base our expenses related to CROs on our estimates of the services received and efforts expended pursuant toquotes and contracts with CROs that conduct research and development on our behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. Theremay be instances in which payments made to vendors and other counterparties will exceed the level of services provided and result in a prepayment ofthe research and development expenses. In accruing service fees, we estimate the time period over which81Table of Contentsservices will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effortvaries from our estimate, we adjust the accrual or prepayment expense accordingly. Although we do not expect our estimates to be materially differentfrom amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of servicesperformed may vary and could result in reporting amounts that are too high or too low in any particular period.Corporate and Other Taxes We are subject to corporate taxes in the Netherlands and the United States. Significant judgment is required in determining the use of net operatingloss carry forwards and taxation of upfront and milestone payments for corporate tax purposes. There are many transactions and calculations for whichthe ultimate tax determination is uncertain. Where the final tax outcome of these matters is different from the amounts that were initially recorded, suchdifferences will impact the current and deferred corporate tax assets and liabilities in the period in which such determination is made. We did not recognize any taxes or income during the periods covered by financial statements contained in this annual report, since we are in a lossmaking position and have a history of losses. As of December 31, 2013, the total amount of tax losses carried forward was €130.9 million. We have a history of tax losses, and therefore recognize deferred tax assets arising from unused tax losses or tax credits only to the extent that therelevant consolidated Dutch entity has sufficient taxable temporary differences or there is convincing other evidence that sufficient taxable profit will beavailable against which the unused tax losses or unused tax credits can be utilized by the consolidated Dutch entities. Management believes thatsufficient convincing other evidence is not currently available and therefore we have not recorded a deferred tax asset in the financial statementscontained in this annual report. Tax losses in the Netherlands may be carried forward for nine years.Impairments of Assets Assets that are subject to amortization are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amountmay not be recoverable. In the years ended December 31, 2012 and 2013 we have reviewed the carrying amount of these assets and determined that noadjustments to carrying values were required. In the year ended December 31, 2011, we recorded an impairment charge of €0.3 million in respect of the termination of a research license underwhich we had made an initial payment of €0.3 million. We test assets that are not subject to amortization annually for impairment. For the purpose of assessing impairment, we group assets at the lowestlevels for which there are separately identifiable cash flows (cash-generating units). We currently use all material assets in the development of our genetherapies. Therefore, our management regularly reviews all activities of our group as a single component and one cash-generating unit. Although we arenot currently selling any products, our collaborator, Chiesi, is preparing the commercial launch of Glybera in the European Union. Our future revenuesfrom product sales, will depend on the success of Chiesi's commercialization efforts in the European Union and our success in obtaining marketingauthorization for Glybera and any other product candidates in additional countries. We have determined that no impairment was required to be recorded during the years ended December 31, 2012 or 2013. Performing a furthersensitivity analysis on the fair value calculation (by for example, reducing the fair value per ordinary share by 20%, as used in the calculation of theenterprise value), did not change management's conclusion that no impairment charge was required.82Table of ContentsThis conclusion was further supported by the IPO proceeds realized in February 2014 and the current market capitalization. Based on our expectations of revenues and gross margin from anticipated sales of Glybera by Chiesi, we have determined that no impairmentcharge in respect of intangible assets relating to Glybera is necessary. These expectations are based principally on our estimate of the market size forGlybera and the gross margin that we expect to realize.Compound Financial Instruments We classify a financial instrument or its component parts on initial recognition as a financial liability, a financial asset or an equity instrument inaccordance with the substance of the contractual arrangement and the definitions of a financial liability, a financial asset and an equity instrument. Wehave analyzed the convertible loans we issued in 2012 and 2013 and the venture debt financing received from Hercules in 2013, and concluded that bothinstruments were composed of a loan component and an embedded financial derivative component, which qualified as financing liabilities. We estimatedthe fair value of these liabilities using option pricing models and assumptions that are based on the individual characteristics of the instruments on thevaluation date.Share-Based Compensation We issue share-based compensation awards, in the form of options to purchase ordinary shares, to certain of our employees, supervisory boardmembers and consultants. We measure share-based compensation expense related to these awards by reference to the estimated fair value of the awardat the date of grant. The total amount of the awards is expensed over the estimated vesting period. We have used the Black-Scholes option pricing modelto determine the fair value of option awards, which requires the input of various assumptions that require management to apply judgment and makeassumptions and estimates, including:•the expected life of the option award, which we have estimated based on a weighted average expected option life for the entire participantgroup; •the expected volatility of the underlying ordinary shares, which we estimate based on the historical volatility of a peer group ofcomparable publicly traded companies with product candidates in similar stages of development; and •historically, the fair value of our ordinary shares determined on the date of grant. At each balance sheet date, we revise our estimates of the number of options that are expected to become exercisable. We recognize the impact ofthe revision of original estimates, if any, in the statement of comprehensive income and a corresponding adjustment to equity. We expect all vestedoptions to be exercised over the remainder of their contractual life. We consider the expected life of the options to be in line with the average remainingterm of the options post vesting. Prior to our acquisition of the AMT business on April 5, 2012, AMT was listed on Euronext Amsterdam from June 2007 through April 2012.This period provided company-specific historical and implied volatility information. Since the de-listing of AMT in April 2012, we have not had thesame level of company-specific historical and implied volatility information; therefore, we estimate the expected volatility based on the historicalvolatility of publicly traded peer companies with a similar focus on gene therapies, biological products or orphan diseases, including OxfordBiomedica plc, MolMed S.p.A., Transgene SA, Sarepta Therapeutics, Inc., Sangamo Biosciences Inc. and Synageva BioPharma Corp. We account for share options as an expense in the statement of comprehensive income over the estimated vesting period, with a correspondingcontribution to equity. See Note 12 to our audited consolidated financial statements included elsewhere in this annual report for a discussion of the total83Table of Contentsexpense recognized in the statement of comprehensive income for share options granted to employees, supervisory board members and consultants. The following table summarizes, by grant date, the number of ordinary shares underlying share options granted from January 1, 2012 throughApril 11, 2014, as well as the associated per share exercise price, the estimated fair value per ordinary share on the grant date, the retrospective estimatedfair value per share on the grant date, and the estimated fair value per option as of the grant date: Of the 2,301,588 options which have been granted under our equity incentive plans and in connection with our agreement with 4D MolecularTherapeutics, and that remained outstanding as of April 11, 2014, an aggregate of 478,217 options were granted to members of the management board.1,507,443 options which have been granted vested in full on or before the closing of our initial public offering on February 10, 2014, which will resultin the acceleration of any unrecognized expense related to these options. As of December 31, 2013, the unrecognized expense related to the optionswhich have been granted and remained outstanding was €1.7 million. The intrinsic value of all outstanding vested and unvested options as of April 11, 2014 was $30.1 million, based on the our initial public offeringprice of $17.00 per ordinary share (€12.60 per ordinary share) on February 10, 2014, and was based on 2,301,588 ordinary shares issuable upon theexercise of options outstanding as of the date of this annual report with a weighted average exercise price of €2.89 per share.Recent Accounting Pronouncements There are no IFRS standards as issued by the IASB or interpretations issued by the IFRS interpretations committee (e.g. IFRS 10, 11, 12, 13 andIAS 19R) that are effective for the first time for the financial year beginning on or after January 1, 2013 that had or are expected to have a materialimpact on our financial position. A number of new standards and amendments to standards and interpretations (e.g IFRS9, IAS36, IAS39) are effective for annual periodsbeginning after January 1, 2014 and have not been applied in preparing these consolidated financial statements. None of these are expected to have amaterial effect on the consolidated financial statements of the Company.84GRANT DATE NUMBER OFORDINARYSHARESUNDERLYINGOPTIONSGRANTED EXERCISEPRICE PERORDINARYSHARE ESTIMATEDFAIRVALUE PERORDINARYSHAREAT GRANTDATE RETROSPECTIVEFAIR VALUEPER ORDINARYSHARE AS OFGRANT DATE(1) ESTIMATEDFAIRVALUEPEROPTIONAS OFGRANTDATE April 5, 2012 1,366,304 €3.07 €3.07 €3.07 €2.05 June 12, 2012 15,000 3.07 3.07 3.07 2.05 December 1,2012 140,652 3.07 3.07 4.85 3.35 December 22,2012 84,391 3.07 3.07 5.10 3.60 January 1, 2013 112,000 5.00 5.00 5.45 3.40 March 26, 2013 14,065 5.00 5.00 7.65 5.30 June 5/6, 2013 28,000 10.10 10.10 12.60 8.15 September 1,2013 140,652 10.10 13.30 N/A 8.85 October 1, 2013 6,751 3.07 13.40 N/A 12.35 January 17,2014 609,744 0.05 12.60(2) N/A 12.55 (1)The fair value of our ordinary shares at the grant date was adjusted in connection with our retrospective fair value assessment forfinancial reporting purposes, as described below. (2)The Euro equivalent of the initial public offering price on February 10, 2014.Table of ContentsC. Research and Development Expenses, Patents and Licences, etc See "Information on the Company—Business Overview—Intellectual Property" and "Operating and Financial Review and Prospects."D. Trend Information See "Operating and Financial Review and Prospects."E. Off-Balance Sheet Arrangements Over the period from October 1, 2000 through May 31, 2005, we received a grant called a "Technisch ontwikkelingskrediet," or technicaldevelopment loan, from the Dutch government. We received grants totaling €3.6 million during the grant period. The grant amount bears interest of5.7% per year and includes a repayment clause in the event we generate revenues from Glybera, during the period from January 1, 2008 throughDecember 31, 2017, based upon a percentage of revenues which are derived from the sale of Glybera, if any. If future amounts received are notsufficient to repay the grant on or prior to December 31, 2017, or if there are no revenues generated from Glybera, the remaining balance will beforgiven. The amount of this contingent commitment as of December 31, 2013 totaled €5.5 million, comprising the original grant together with accruedinterest, less an initial repayment made in the third quarter of 2013. We have not recorded any liability to repay amounts in respect of this contingentcommitment. Further amounts may be recognized once revenues related to produce sales at Glybera commence. During the periods presented in this annual report, we did not have any other off-balance sheet arrangements.F. Tabular Disclosure of Contractual ObligationsContractual Obligations and Commitments The table below sets forth our contractual obligations and commercial commitments as of December 31, 2013 that are expected to have an impacton liquidity and cash flows in future periods. The table above does not include:85 PAYMENTS DUE BY PERIOD (€ in thousands) LESSTHAN1 YEAR BETWEEN1 AND 2YEARS BETWEEN2 AND 5YEARS MORETHAN5 YEARS TOTAL License maintenanceobligations(1)(2) 327 300 687 856 2,170 Debt obligations 1,498 3,372 4,442 — 9,312 Operating lease obligations 1,243 1,766 4,287 7,927 15,223 Finance lease obligations 156 168 134 — 458 Construction commitment USFacility 10,824 — — — 10,824 Total 14,048 5,606 9,550 8,783 37,987 (1)Annual license maintenance payments will be no longer payable following the expiration of the license payment obligations.Thereafter, we have a fully paid-up license. (2)Amounts are paid annually in advance; to the extent that we could terminate the agreement prior to the date of the nextmaintenance payment, these maintenance fees are not recognized within research commitments in the notes to the financialstatements.Table of Contents•Payments we may be obligated to make under our license or collaboration agreements, other than fixed periodic maintenance costs. Suchadditional payment obligations may be material. See "—Collaboration and License Agreements" and "Information on the Company—Intellectual Property—Licenses". •Our obligations to repay the Dutch technical development loan described above. •Our obligations under the collaboration and license agreement with 4D Molecular Therapeutics, entered into in January 2014, to fundresearch and development activities at a cost of approximately $3.0 million in aggregate over the next three years and approximately$200,000 of licenses fees during the first year.G. Safe harbor See "Forward Looking Statements".Item 6 Directors, Senior Management and EmployeesA. Directors and Senior Management We have a two-tier board structure consisting of our management board (raad van bestuur) and a separate supervisory board (raad vancommissarissen). Below is a summary of relevant information concerning our supervisory board, management board and senior management.Members of Our Supervisory Board, Management Board and Senior ManagementSupervisory board The following table sets forth information with respect to each of our supervisory board members and their respective ages as of the date of thisannual report. The terms of office of all our supervisory board members expire according to a rotation plan drawn up by our supervisory board. Thebusiness address of our supervisory board members is our registered office address at Meibergdreef 61, Amsterdam 1105 BA, the Netherlands. Our supervisory board is currently composed of the following members, all of whom are independent under applicable NASDAQ standards: Ferdinand Verdonck has served as our chairman since July 2012 and served as chairman of the AMT supervisory board from April 2007 untilJuly 2012. He is a director on the boards of J.P. Morgan European Investment Trust, Groupe SNEF, Laco Information Services and Virtus Funds.From 1992 to 2003, he was the managing director of Almanij NV, a financial services company which has since merged with KBC, and hisresponsibilities included company strategy, financial control, supervision of executive management and corporate governance, including boardparticipation in publicly-traded and86NAME AGE POSITION MEMBERSINCE(1) TERMEXPIRES FerdinandVerdonck 71 Member of the SupervisoryBoard (Chairman) 2012 2017 SanderSlootweg 45 Member of the SupervisoryBoard 2012 2015 Sander vanDeventer 59 Member of the SupervisoryBoard 2012 2016 Joseph M.Feczko 65 Member of the SupervisoryBoard 2012 2016 François Meyer 65 Member of the SupervisoryBoard 2012 2015 David Schaffer 44 Member of the SupervisoryBoard 2014 2016 PaulaSoteropoulos 46 Member of the SupervisoryBoard 2013 2017 (1)For periods prior to 2012, certain of our directors served as directors of AMT, our predecessor entity.Table of Contentsprivately-held companies in many countries. He served as a member of the board of directors and chairman of the audit committee of two biotechnologycompanies in Belgium, Movetis and Galapagos. He has previously served as chairman of Banco Urquijo, a director of Dictaphone Corporation and adirector of the Dutch Chamber of Commerce for Belgium and Luxembourg, member of the General Council and chairman of the audit committee of theVlerick Leuven Ghent Management School. Mr. Verdonck holds a law degree from KU Leuven and degrees in economics from KU Leuven and theUniversity of Chicago. We believe that Mr. Verdonck is qualified to serve on our supervisory board due to his expertise in the financial services andmanufacturing industries and his service on the boards of directors of other companies. Sander Slootweg has served as a member of our supervisory board since April 2012 and served as member of the AMT supervisory board fromSeptember 2006 to April 2008, including as Chairman from 2006 to 2007. Mr. Slootweg is a managing partner at Forbion Capital Partners, theNetherlands, a venture capital firm he co-founded in 2006. He currently serves on the boards of Forbion's portfolio companies Xention, Ltd, PulmagenTherapeutics, Ltd, Dezima Pharma, B.V. (Chairman), Ario Pharma Ltd. and Oxyrane, Ltd. In addition, in recent years Mr. Slootweg has served on theboards of Argenta Discovery Ltd (sold to Galapagos in 2010), Alantos Pharmaceuticals, Inc. (sold to Amgen in 2007), BioVex Group, Inc. (sold toAmgen in 2011), Impella Cardiosystems AG (sold to Abiomed, Inc. in 2005), Glycart AG (sold to Roche in 2005), Cambridge Drug Discovery Ltd(sold to Biofocus Plc in 2001), Fovea Pharmaceuticals S.A. (sold to Sanofi-Aventis in 2009) and Pieris AG. Mr. Slootweg holds degrees in Businessand Financial Economics from the Free University of Amsterdam and in Business Administration from Nijenrode University, The Netherlands. Webelieve that Mr. Slootweg is qualified to serve on our supervisory board due to his expertise in the healthcare technology industry and his service on theboards of directors of other companies. Sander van Deventer has served as a member of our supervisory board since April 2012 and served as member of the AMT supervisory boardfrom April 2010 to April 2012. Dr. van Deventer was one of our co-founders and currently chairs uniQure's Scientific Advisory Board. He served asour interim Chief Executive Officer from February to October 2009. He has been Professor of Translational Gastroenterology at the Leiden UniversityMedical Center since 2008 and is a partner of Forbion Capital Partners, which he joined in 2006. He serves on the boards of Cardoz AS, ArgosBiotherapeutics, gICare Pharma Inc and Hookipa Biotech. He was previously a professor, head of the department of experimental medicine andchairman of the department of gastroenterology of the Academic Medical Center at the University of Amsterdam from 2002 to 2004, and subsequentlyprofessor of experimental medicine at the University of Amsterdam Medical School until 2008. He has more than 15 years of experience inbiotechnology product development. He is the author of more than 350 scientific articles in peer-reviewed journals, and he serves as an advisor toregulatory authorities including the EMA and FDA. Dr. van Deventer holds a degree in medicine as well as a Ph.D. from the University of Amsterdam.We believe that Dr. van Deventer is qualified to serve on our supervisory board due to his expertise in the biotechnology industry and his service on theboards of directors of other biotechnology companies. Joseph M. Feczko has served as a member of our supervisory board since April 2012 and served as a member of the AMT supervisory boardfrom August 2010 to April 2012. Dr. Feczko worked for Pfizer Inc. from 1982 to 1992 and from 1996 to 2009, where he held positions of increasingresponsibility in clinical research, regulatory affairs and safety culminating in the role of Senior Vice President and Chief Medical Officer. From 1992 to1996, Dr. Feczko was Medical Director for GlaxoSmithKline R&D in the United Kingdom. Dr. Feczko is chairman of the board of directors at CardozPharmaceuticals AB, and a director of Keryx Biopharmaceuticals, Inc. and ChemoCentryx Inc., as well as a member of the supervisory board ofCytheris. He is also a member of the board of directors of Accordia Global Health Foundation Research!America, and the Foundation of NationalInstitute of Health, and a trustee of the New York Academy of Medicine. Dr. Feczko is a member of87Table of Contentsthe Technical Expert Committee for the International Trachoma Initiative of the Task Force for Global Health. Between 2006-2011 he was a member ofthe Governing Board of the Technology Strategy Board of the United Kingdom. Dr. Feczko is Board Certified in Internal Medicine and InfectiousDiseases. Dr. Feczko holds a bachelor of science degree from Loyola University and an M.D. from the University of Illinois College of Medicine. Webelieve that Dr. Feczko is qualified to serve on our supervisory board due to his expertise in the pharmaceutical and biotechnology industries. François Meyer has served as a member of our supervisory board since April 2012 and served as a member of the AMT supervisory board fromJuly 2010 to April 2012. Dr. Meyer was until recently CEO and Chairman of the board of TxCell SA, a cell therapy company located in France, and ofwhich he is currently Executive Chairman. Prior to this, he was CEO of Gencell, a fully owned gene therapy subsidiary of Aventis until 2006. He wassenior vice president R&D at Aventis Pharma until 2002 and prior to that he led global research at Rhone Poulenc Rorer. In the earlier part of his careerhe held senior management positions at Sandoz and led the gene and cell therapy business. He was a member of the board of directors or the scientificadvisory board of a number of biotech companies in the gene and cell therapy area including Introgen Therapeutics, Inc., Gene Therapy Inc.,Systemix, Inc. and Biotransplant, Inc. We believe that Dr. Meyer is qualified to serve on our supervisory board due to his expertise and insight in thebiotechnology industry. David Schaffer has served as a member of our supervisory board since January 2014. Dr. Schaffer is Professor of Chemical and BiomolecularEngineering, Bioengineering, and Neuroscience at University of California Berkeley, a position he has held since 2007, as well as Director of theBerkeley Stem Cell Center since 2011. Dr. Schaffer is also co-founder of 4D Molecular Therapeutics, a company specializing proprietary technologyfor gene therapy products. We entered into a collaboration and license agreement with 4D Molecular Therapeutics in January 2014. Previously,Dr. Schaffer was Assistant Professor from 1999 to 2005 and Associate Professor from 2005 to 2007 at the University of California, BerkeleyDepartment of Chemical Engineering & Helen Wills Neuroscience Institute. He serves on the boards of the American Society for Gene and CellTherapy and the Society for Biological Engineering. He has more than 20 years of experience in chemical and molecular engineering, and stem cell andgene therapy research, has over 130 scientific publications, and serves on 5 journal editorial boards and 5 industrial scientific advisory boards.Dr. Schaffer holds a bachelor of science degree in chemical engineering from Stanford University and a Ph.D. in Chemical Engineering from theMassachusetts Institute of Technology. We believe Dr. Schaffer is qualified to serve on our supervisory board due to his extensive relevant scientificexpertise and experience in the biotechnology industry. Paula Soteropoulos has served as a member of our supervisory board since July 2013. Ms. Soteropoulos is Senior Vice President and GeneralManager, Cardiometabolic Business and Strategic Alliances at Moderna Therapeutics, Inc., a position she has held since July 2013. Previously,Ms. Soteropoulos has worked at Genzyme Corporation, a biotechnology company, from 1992 to 2013, most recently as Vice President and GeneralManager, Cardiovascular, Rare Diseases. Ms. Soteropoulos holds a bachelor of science degree in chemical engineering and a master of science degreein chemical and biochemical engineering, both from Tufts University, and holds an executive management certificate from the University of Virginia,Darden Graduate School of Business Administration. We believe Ms. Soteropoulos is qualified to serve on our supervisory board due to her extensiveexperience in the biotechnology industry.Management board The following table sets out information with respect to each of our management board members, their respective ages and their positions atuniQure as of the date of this annual report. The business88Table of Contentsaddress of our management board members is our registered office address at Meibergdreef 61, Amsterdam 1105 BA, the Netherlands. Jörn Aldag has served as our chief executive officer since he joined AMT, now uniQure, in October 2009. He has led our corporate developmentincluding the expansion of our gene therapy pipeline, the marketing authorization process with the EMA for Glybera and the recapitalization of AMT toform uniQure. Before joining our company he was instrumental in building Evotec AG, a drug discovery company listed on the Frankfurt StockExchange, serving as chief financial officer from 1997 to 2000 and as president and chief executive officer from 2001 to 2009. Prior to Evotec,Mr. Aldag served in various financial management positions at MAN AG, and as Business Director at Treuhandanstalt, the agency responsible forprivatizing the East German economy after the German reunification. Mr. Aldag is Chairman of Molecular Partners AG, Zurich, Switzerland, and holdsbusiness degrees from the Harvard Business School (Advanced Management Program) and the European Business School. We believe that Mr. Aldagis qualified to serve on our management board due to his broad expertise in the biotechnology industry and his deep general management experience. Piers Morgan has served as our chief financial officer since he joined AMT in December 2009. Mr. Morgan is currently chairman of the boardand a member of the audit committee of Trino Therapeutics Ltd, a biotechnology company. He has more than 13 years of experience as chief financialofficer of several biotechnology companies, including Phytopharm plc, BioAlliance Pharma SA, and Arrow Therapeutics Ltd. Prior to this period, hespent ten years in investment banking, working in mergers & acquisitions and equity capital markets with Close Brothers and Ernst & Young CorporateFinance. He qualified as a chartered accountant in London with PricewaterhouseCoopers. Mr. Morgan holds a degree in law and management studiesfrom Cambridge University. We believe that Mr. Morgan is qualified to serve on our management board due to his expertise in the biotechnologyindustry and his accounting background. Mr. Morgan has tendered his resignation as our chief financial officer and member of the management board,effective May 20, 2014.Senior management Our management board is supported by our senior management team. The following table sets forth information with respect to each of themembers of our senior management team, their respective ages and their positions as of the date of this annual report. The business address of themembers of our senior management is our registered office address at Meibergdreef 61, Amsterdam 1105 BA, the Netherlands.89NAME AGE POSITION DATE OF APPOINTMENTJörn Aldag 54 Chief Executive Officer October 4, 2009Piers Morgan(1) 47 Chief Financial Officer December 1, 2009(1)Mr. Morgan has tendered his resignation, which will be effective May 20, 2014.NAME AGE POSITIONPhilip Astley-Sparke 42 President, U.S. OperationsChristian Meyer, M.D. 46 Chief Medical OfficerHarald Petry 54 Chief Science OfficerHans Preusting 51 Chief Business OfficerHans Christian Rohde 56 Chief Commercial OfficerTable of Contents Philip Astley-Sparke has served as the president of our U.S. operations since January 2013. Mr. Astley-Sparke has been a venture partner atForbion Capital Partners, a venture capital fund, since May 2012. He served as vice president and general manager at Amgen, Inc., a biopharmaceuticalcompany, until December 2011, following Amgen's acquisition of BioVex Group, Inc., a biotechnology company, in March 2011. Mr. Astley-Sparkehad been president and chief executive officer of BioVex Group since 2007, which he joined in 2000, and previously served in the roles of President &COO and CFO. He oversaw the company's relocation to the United States where he grew operations from scratch, including overseeing theconstruction of a commercial-grade manufacturing facility. Prior to Biovex Group, Mr. Astley-Sparke was a healthcare investment banker with ChaseH&Q/Robert Fleming. He qualified as a chartered accountant with Arthur Andersen in London and holds a bachelor's degree in cellular pathology andmolecular pathology from Bristol University in the United Kingdom. He also serves as chairman of the board of Oxyrane, a biotechnology company. Christian Meyer, M.D. has served as our chief medical officer since October 2013. Dr. Meyer has more than 13 years of clinical researchexperience with both biotechnology companies and large pharma, with particular expertise in the development of treatments for rare diseases, includingacute intermittent porphyria and lysosomal storage disorders. From 2010-2013 he was the chief medical officer at Cardoz AB, a pharmaceuticalcompany. Prior to that, from 2006 to 2010, Dr. Meyer held leadership positions in clinical development at Symphogen A/S, a biopharmaceuticalcompany, where he was senior vice president for medical affairs and vice president of clinical development. Prior to Symphogen A/S, he played animportant role in clinical development at Zymenex A/S and spent five years in clinical development at Novo Nordisk A/S, both biopharmaceuticalcompanies. Dr. Meyer received both his M.D. and Ph.D. degrees from the University of Copenhagen, Denmark. Harald Petry has served as our chief science officer since January 2012. Dr. Petry joined AMT in May 2007 as director of research anddevelopment. He has worked in the area of gene therapy for more than 15 years and has extensive experience in pharmaceutical research. Prior tojoining us, he worked at Jenapharm GmbH (Germany), a pharmaceutical company, from 2001 to 2002 and Berlex Biosciences (US), a biotechnologycompany, from 2002 to 2007 in different functions with increasing managerial and leadership responsibility. Dr. Petry holds his doctoral degree inbiology from Justus-Liebig-Universität Giessen. Hans Preusting has served as our chief business officer since July 2011, including at AMT where he first joined us as a Director of ProcessDevelopment and Manufacturing in August 2006. He holds a PhD in biochemistry and an MBA from Rotterdam School of Management. He has morethan 20 years of experience in product development and manufacturing using fermentation and cell culture techniques. Prior to joining us, he was atSolvay Pharmaceuticals, DSM and Gist-brocades. Dr. Preusting holds two patents and has published more than 20 scientific articles. Hans Christian Rohde has served as our chief commercial officer since December 2012. Mr. Rohde has almost 25 years of experience incommercial roles at leading biotechnology and pharmaceutical companies. From 2007 until 2012 he was chief commercial officer at BasileaPharmaceutica, a pharmaceutical company, and a member of its executive management committee with responsibility for global commercial operations,marketing, supply chain, medical affairs, pricing and market access. Prior to Basilea Pharmaceutica, Mr. Rohde was corporate vice president, head ofglobal therapeutic areas reproductive health and endocrinology at Merck-Serono, a pharmaceutical company, from 2003 until 2007. Prior to this, he wasresponsible for international marketing and global market development at Biogen Idec, a biotechnology company. Mr Rohde holds a master of sciencefrom the University of Copenhagen and a master of business administration from the Birmingham Business School, the University of Birmingham inthe United Kingdom.90Table of ContentsB. Compensation The below table sets out a breakdown of the compensation, in aggregate, for members of our supervisory board, management board and seniormanagement: For further detail on compensation of members of our supervision board, management board and senior management, see Note 30 to the auditedconsolidated financial statements included elsewhere in this annual report.C. Board PracticesCommittees of the Supervisory Board We have an audit committee, a remuneration committee and a nominating and corporate governance committee. We have adopted a charter for eachof these committees.Audit Committee Our audit committee consists of Mr. Feczko (Chairman), Ms. Soteropoulos and Mr. Verdonck. Each member satisfies the independencerequirements of the NASDAQ listing standards, and Mr. Verdonck qualifies as an "audit committee financial expert," as defined in Item 16A ofForm 20-F and as determined by our supervisory board. The audit committee oversees our accounting and financial reporting processes and the auditsof our consolidated financial statements. The audit committee is responsible for, among other things:•making recommendations to our supervisory board regarding the appointment by the general meeting of shareholders of our independentauditors; •overseeing the work of the independent auditors, including resolving disagreements between management and the independent auditorsrelating to financial reporting; •pre-approving all audit and non-audit services permitted to be performed by the independent auditors; •reviewing the independence and quality control procedures of the independent auditors; •discussing material off-balance sheet transactions, arrangements and obligations with management and the independent auditors; •reviewing and approving all proposed related-party transactions; •discussing the annual audited consolidated and statutory financial statements with management; •annually reviewing and reassessing the adequacy of our audit committee charter; SHORTTERMEMPLOYEEBENEFITS SHARE-BASEDPAYMENTS(1) POST-EMPLOYMENTBENEFITS ADVISORSFEES TERMINATIONBENEFITS TOTAL (€ in thousands) Year endedDecember 31,2013 SupervisoryBoard — 296 — 104 — 400 ManagementBoard 747 377 60 — — 1,184 SeniorManagement 1,101 873 109 — — 2,083 1,848 1,546 169 104 — 3,667 (1)For information on share ownership and options held by our supervisory directors, managing directors and senior management, please see "Major Shareholdersand Related Party Transactions—Major Shareholders."91Table of Contents•meeting separately with the independent auditors to discuss critical accounting policies, recommendations on internal controls, theauditor's engagement letter and independence letter and other material written communications between the independent auditors and themanagement; and •attending to such other matters as are specifically delegated to our audit committee by our supervisory board from time to time.Remuneration Committee Our remuneration committee consists of Messrs. van Deventer (Chairman), Meyer and Verdonck. Each member satisfies the independencerequirements of the NASDAQ listing standards. The remuneration committee assists the supervisory board in reviewing and approving orrecommending our compensation structure, including all forms of compensation relating to our supervisory directors and management. Members of ourmanagement may not be present at any committee meeting while the compensation of our chief executive officer is deliberated. Subject to the terms ofthe remuneration policy approved by our general meeting of shareholders from time to time, as required by Dutch law, the remuneration committee isresponsible for, among other things:•reviewing and making recommendations to the supervisory board with respect to compensation of our management board andsupervisory board members; •reviewing and approving the compensation, including equity compensation, change-of-control benefits and severance arrangements, ofour chief executive officer, chief financial officer and such other members of our management as it deems appropriate; •overseeing the evaluation of our management; •reviewing periodically and making recommendations to our supervisory board with respect to any incentive compensation and equityplans, programs or similar arrangements; •exercising the rights of our supervisory board under any equity plans, except for the right to amend any such plans unless otherwiseexpressly authorized to do so; and •attending to such other matters as are specifically delegated to our compensation committee by our supervisory board from time to time.Nominating and Corporate Governance Committee Our nominating and corporate governance committee consists of Messrs. van Deventer (Chairman), Meyer and Verdonck. Each member satisfiesthe independence requirements of the NASDAQ listing standards. The nominating and corporate governance committee assists the supervisory board inselecting individuals qualified to become our supervisory directors and in determining the composition of the supervisory board and its committees. Thenominating and corporate governance committee is responsible for, among other things:•recommending to the supervisory board persons to be nominated for election or re-election to the supervisory board at any meeting ofthe shareholders; •overseeing the supervisory board's annual review of its own performance and the performance of its committees; and •considering, preparing and recommending to the supervisory board a set of corporate governance guidelines.92Table of Contents For information on the current term of office and the period during which the members of our supervisory board, management board and oursenior management have served in office see "—Directors and Senior Management."D. Employees As of December 31, 2013, we had a total of 87 employees, of whom 29 had an M.D. or Ph.D. degree, or the foreign equivalent. Of theseemployees, 20 were engaged in research and development, seven in clinical development, and two in business development functions. We also engaged33 consultants and contract workers. We do not currently have in place a works council.E. Share Ownership See "Major Shareholders and Related Party Transactions."Item 7 Major Shareholders and Related Party TransactionsA. Major Shareholders The following table sets forth information with respect to the beneficial ownership of our ordinary shares as of April 11, 2014 by:•each of the members of our management board and supervisory board; •each of our other members of senior management; and •each person, or group of affiliated persons, who is known by us to beneficially own more than 5% of our ordinary shares. The column entitled "Total Percentage" is based on a total of 17,594,906 ordinary shares outstanding as of April 11, 2014, determined on thefollowing basis: beneficial ownership is determined in accordance with the rules and regulations of the SEC and includes voting or investment powerwith respect to our ordinary shares. Ordinary shares subject to options that are currently exercisable or exercisable within 60 days of April 11, 2014 areconsidered outstanding and beneficially owned by the person holding the options for the purpose of calculating the percentage ownership of that personbut not for the purpose of calculating the percentage ownership of any other person. Except as otherwise noted, the persons and entities in this tablehave sole voting and investing power with respect to all of our ordinary shares beneficially owned by them, subject to community property laws, whereapplicable.93Table of ContentsExcept as otherwise set forth below, the address of the beneficial owner is c/o uniQure B.V., Meibergdreef 61, 1105 BA, Amsterdam, the Netherlands.94NAME AND ADDRESS OF BENEFICIAL OWNER NUMBER OFSHARESBENEFICIALLYOWNED TOTALPERCENTAGE Major Shareholders: Entities affiliated with Forbion(1) 4,393,523 25.0%Cooperatieve Gilde Healthcare II U.A.(2) 1,730,415 9.8%Coller International Partners V-A, L.P.(3) 6,512,043 37.0%Chiesi Farmaceutici S.p.A.(4) 1,109,214 6.3%Management Board Members, Supervisory Board Members and SeniorManagement Ferdinand Verdonck(5) 159,826 * Sander Slootweg(6) 4,393,523 25.0%Sander van Deventer(7) 4,393,523 25.0%Joseph M. Feczko(8) 65,275 * François Meyer(9) 55,279 * David Schaffer(10) — — Paula Soteropoulos(11) 3,406 — Jörn Aldag(12) 376,954 2.1%Piers Morgan(13) 168,444 * Philip Astley-Sparke(14) 4,505,523 25.6%Christian Meyer — — Harald Petry(15) 141,279 * Hans Preusting(16) 143,179 * Hans Christian Rohde(17) 153,752 * All current management board members, supervisory board members, andsenior management as a group 5,772,917 30.2%Total shares held by management board members, supervisory boardmembers, senior management and majorshareholders 10,731,066 56.2%*Represents beneficial ownership of less than one percent of our outstanding ordinary shares. (1)Consists of (i) 987,673 ordinary shares held by Coöperatieve AAC LS U.A., or Coöperatieve; (ii) 1,520,598 ordinary sharesheld by Forbion Co-Investment Coöperatief U.A., or FCI; (iii) 1,865,493 ordinary shares held by Forbion Co-Investment IICoöperatief U.A., or FCI II; (iv) warrants held by FCI to purchase 9,900 ordinary shares that are exercisable as of April 11,2014 or will become exercisable within 60 days after such date; and (v) 9,859 ordinary shares held by SJH van Deventer CV, orSJH. Forbion 1 Management B.V., the director of Coöperatieve and FCI, and Forbion 1 Co II Management B.V., the director ofFCI II, and Forbion Capital Partners Management Services B.V., or Forbion Capital Partners, the general partner of SJH, may bedeemed to have voting and dispositive power over the ordinary shares held by Coöperatieve, FCI, FCI II and SJH. Investmentdecisions with respect to the ordinary shares held by Coöperatieve, FCI, FCI II and SJH can be made by any two of the dulyauthorized representatives of Coöperatieve, FCI, FCI II and SJH. Mr. Slootweg and Dr. van Deventer are partners of ForbionCapital Partners, which acts as the investment advisor to the directors of Coöperatieve, FCI, FCI II and as General Partner toSJH. Mr. Astley-Sparke, among others, as a venture partner acts as an independent contractor in an advisory function to ForbionCapital Partners. Each of Mr. Slootweg, Dr. van Deventer and Mr. Astley-Sparke disclaim beneficial ownership of such ordinaryshares, except to the extent of his pecuniary interest therein. The address of ForbionTable of Contents95Capital Partners, Coöperatieve, FCI, FCI II and SJH is Gooimeer 2-35, 1411 DC Naarden, The Netherlands.(2)Consists of (i) 1,720,515 ordinary shares held by Coöperatieve Gilde Healthcare II U.A. and (ii) warrants held by CoöperatieveGilde Healthcare II U.A. to purchase 9,900 ordinary shares that are exercisable as of April 11, 2014 or will become exercisablewithin 60 days after such date. The manager of Coöperatieve Gilde Healthcare II U.A. is Gilde Healthcare II Management B.V.,or Gilde Management, and Gilde Management is owned by Gilde Healthcare Holding B.V., or Gilde Holding. Three managingpartners, Edwin de Graaf, Marc Olivier Perret and Martenmanshurk B.V. (of which Pieter van der Meer is the owner andmanager) each own 28.66% of Gilde Holding and Stichting Administratiekantoor Gilde Healthcare Holding, or Stichting, owns14% of Gilde Holding. Stichting is controlled by Mr. de Graaf, Mr. Perret and Martenmanshurk B.V. and issued depositoryreceipts for shares in Gilde Holding to two partners, Arthur Franken and Dirk Kersten. Each of Mr. de Graaf, Mr. Perret andMr. van der Meer share voting and dispositive power of the shares, and disclaim beneficial ownership of the shares except to theextent of their respective pecuniary interest therein. The address of Coöperatieve Gilde Healthcare II U.A. is Newtonlaan 91,3584 BP, Utrecht, The Netherlands. (3)Consists of (i) 2,019,511 ordinary shares held by Coller International Partners V-A, L.P., or Coller; (ii) warrants held by Collerto purchase 99,009 ordinary shares that are exercisable as of April 11, 2014 or will become exercisable within 60 days after suchdate; (iii) 987,673 ordinary shares held by Coöperatieve; (iv) 1,520,598 ordinary shares held by FCI; (v) 1,865,493 ordinaryshares held by FCI II; (vi) warrants held by FCI to purchase 9,900 ordinary shares that are exercisable as of April 11, 2014 orwill become exercisable within 60 days after such date and (vii) 9,859 ordinary shares held by SJH. Coller is a limited partner ofthe Forbion funds. Coller has no dispositive or voting power over ordinary shares held by the Forbion funds and disclaimsbeneficial ownership of such ordinary shares except to the extent of its pecuniary interest therein. See footnote 1 above. Thegeneral partner of Coller is Coller International General Partner V, L.P. of which Coller Investment Management Limited, orCIML, is the general partner. The directors of CIML are Jeremy Joseph Coller, Cyril Joseph Mahon, Roger Alan Le Tissier,Paul McDonald, Peter Michael Hutton, John Charlton Loveless and Andrew Thane Maden Hitchon and may be deemed to sharevoting and dispositive power with respect to the ordinary shares held by Coller. The CIML directors disclaim beneficialownership of such ordinary shares except to the extent of their pecuniary interest therein. The address of Coller is c/o CollerInvestment Management Limited, PO Box 255, Trafalgar Court, Les Banques, St Peter Port, Guernsey, Channel Islands. (4)The registered office of Chiesi Farmaceutici S.p.A is Via Palermo, 26, 43122 Parma, Italy. (5)Consists of 75,435 ordinary shares and options to purchase 84,391 ordinary shares that are exercisable as of April 11, 2014 orwill become exercisable within 60 days after such date. (6)Consists of securities held by funds affiliated with Forbion. See footnote 1 above. (7)Consists of securities held by funds affiliated with Forbion. See footnote 1 above. (8)Consists of 27,768 ordinary shares and options to purchase 37,507 ordinary shares that are exercisable as of April 11, 2014 orwill become exercisable within 60 days after such date. (9)Consists of 17,772 ordinary shares and options to purchase 37,507 ordinary shares that are exercisable as of April 11, 2014 orwill become exercisable within 60 days after such date. (10)Dr. Schaffer joined our supervisory board in January 2014. (11)Consists of options to purchase 3,406 ordinary shares that are exercisable as of April 11, 2014 or will become exercisable within60 days after such date.Table of Contents(12)Consists of 39,389 ordinary shares and options to purchase 337,565 ordinary shares that are exercisable as of April 11, 2014 or will becomeexercisable within 60 days after such date. (13)Consists of 27,805 ordinary shares and options to purchase 140,652 ordinary shares that are exercisable as of April 11, 2014 or will becomeexercisable within 60 days after such date. (14)Consists of options to purchase 112,000 ordinary shares that are exercisable as of April 11, 2014 or will become exercisable within 60 daysafter such date, together with securities held by entities affiliated with Forbion. See footnote 1 above. (15)Consists of 627 ordinary shares and options to purchase 140,652 ordinary shares that are exercisable as of April 11, 2014 or will becomeexercisable within 60 days after such date. (16)Consists of 2,527 ordinary shares and options to purchase 140,652 ordinary shares that are exercisable as of April 11, 2014 or will becomeexercisable within 60 days after such date. (17)Consists of 13,100 ordinary shares and options to purchase 140,652 ordinary shares that are exercisable as of April 11, 2014 or will becomeexercisable within 60 days after such date.Holdings by U.S. Shareholders As of April 11, 2014, there was one holder of record of ordinary shares (Cede & Co., as nominee for DTC), holding approximately 30.7% of ourordinary shares.B. Related Party Transactions Since January 1, 2010, we have engaged in the following transactions with the members of our supervisory board, management board, seniormanagement, holders of ordinary shares, and their affiliates, which we refer to as our related parties.2014 Initial Public Offering In February 2014 we completed our initial public offering, raising $91.8 million before expenses and underwriting commissions, through an issueof our ordinary shares at a price of $17 per share. The following table sets forth the number of ordinary shares purchased by our related parties.2013 Chiesi Collaboration In June 2013 we completed three inter-conditional agreements with Chiesi; two of these agreements form the basis of our strategic collaborationwith Chiesi, as described in "Item 4B—Business Overview". Under the third agreement Chiesi subscribed for 1,109,214 of our ordinary shares at aprice of €12.60 per share, raising a total of €14.0 million for uniQure. For the year ended December 31, 2013 uniQure has received an aggregate amount of €1.2 million from Chiesi under the terms of the strategiccollaboration; at December 31, 2013 an amount of €1.4 million was receivable from Chiesi under the strategic collaboration.96SHAREHOLDER NUMBER OFORDINARYSHARES Forbion Co-Investment Coöperatief U.A. 58,823 Coller International Partners V-A, L.P. 1,029,412 Cooperatieve Gilde Healthcare II U.A. 79,412 Table of Contents2012 and 2013 Convertible Notes In December 2012, January 2013 and March 2013, we sold convertible promissory notes in the aggregate principal amount of €13.5 million in aprivate placement to certain of our existing investors, which we refer to as the convertible notes. The convertible notes accrued interest at a rate equal to8% per year, and had a maturity date of December 31, 2014, unless previously converted. No payments of principal or interest were made under thesenotes. In addition, in connection with the issuance of the convertible notes we issued the holders of such convertible notes warrants to purchase anaggregate of 133,628 of our ordinary shares. In July 2013, the convertible notes were converted into an aggregate of 1,336,331 of our ordinary shares. The following table sets forth the participation in this financing by our related parties:2012 Share Purchase Incentive Plan In November 2012, we raised an aggregate of €552,202 through the issue of ordinary shares at a price of €3.07 per share in part to members ofour supervisory board and senior management, including Joseph Feczko, Francois Meyer, Ferdinand Verdonck, Piers Morgan and Hans ChristianRohde. These funds were received in 2012 and 2013.Grants of Options to Related Parties The Company granted options to members of the supervisory board, management board and senior management. Details of options granted areincluded within the beneficial ownership table above.Shareholders Agreements Our shareholders' agreements related to our class A, B and C shares, were terminated upon the conversion of our class A, B and C shares intoordinary shares on a 1-for-1 basis, effective February 10, 2014.4D Molecular Therapeutics Collaboration On January 17, 2014, we entered into a collaboration and license agreement with 4D Molecular Therapeutics, as described in "Information on theCompany—Business Overview" above. 4D Molecular Therapeutics is a company co-founded by Dr. David Schaffer, who was appointed to oursupervisory board on January 27, 2014 pursuant to the terms of that collaboration. In connection with this transaction, we have agreed to providespecified research and development financing, are obligated to make certain upfront, royalty and milestone payments, and have granted an option topurchase up to97PURCHASER AGGREGATEPRINCIPALAMOUNT OFCONVERTIBLENOTES ORDINARYSHARESISSUED UPONCONVERSION OFCONVERTIBLENOTES ORDINARYSHARESISSUABLEUPONEXERCISE OFWARRANTS Forbion Co-Investment Cooperatief U.A.(1) €1,000,000 99,009 9,900 Cooperatieve Gilde Healthcare II U.A. €1,000,000 99,009 9,900 Coller International Partners V-A, L.P. €10,000,000 990,099 99,009 (1)Sander Slootweg, a member of our supervisory board, is a Managing Partner at Forbion Capital Partners. Sander van Deventer, amember of our supervisory board, is a Managing Partner at Forbion Capital Partners.Table of Contents304,872 ordinary shares at an exercise price of €0.05 per share to Dr. Schaffer. See "Business—Strategic Collaboration: 4D Molecular Therapeutics."C. Interests of Experts and Counsel Not applicable.Item 8 Financial Information A. Consolidated Statements and Other Financial Information See the financial statements beginning on page F-1.Legal Proceedings Except as described below, we are not involved in any material legal proceedings. On December 11, 2013, we received a formal request for arbitration from Extera Partners, a consulting firm based in Cambridge, Massachusetts,alleging a fee to be due in respect of consulting services provided to us in connection with a partnering transaction. The request for arbitration wasreceived by the International Court of Arbitration at the International Chamber of Commerce on December 12, 2013, which represents the start date ofthe arbitration. The amount claimed is $100,000 plus 2.5% of all proceeds, including equity investments, we receive from Chiesi pursuant to ourcollaboration agreements entered into in the second quarter of 2013. Our engagement letter with Extera Partners contains a cap limiting the maximumpayment to €5.0 million. We have reviewed this claim with counsel and believe that the claim is without merit. We intend to vigorously defend against it.Dividends We do not at present plan to pay cash dividends on our ordinary shares. Under Dutch law, we may only pay dividends if our shareholders' equityexceeds the sum of the paid-up and called-up share capital plus the reserves required to be maintained by Dutch law or by our articles of association. Inaddition, our loan agreement with Hercules contains, and any other loan facilities that we may enter into may contain, restrictions on our ability, or thatof our subsidiaries, to pay dividends. Subject to such restrictions, a proposal for the payment of cash dividends in the future, if any, will be at thediscretion of our management board, subject to the approval of our supervisory board, and will depend upon such factors as earnings levels, capitalrequirements, contractual restrictions, our overall financial condition and any other factors deemed relevant by our management board.B. Significant Changes See Note 32 to the audited consolidated financial statements included elsewhere in this annual report.Item 9 The Offer and Listing A. Offering and Listing Details Not applicable.B. Plan of Distribution Not applicable.98Table of ContentsC. Markets Our ordinary shares are currently listed on The NASDAQ Global Select Market under the symbol "QURE". The following table sets forth the high and low sale prices on The NASDAQ Global Select Market for our ordinary shares from February 4, 2014,the initial trading date for our ordinary shares on NASDAQ through April 11, 2014. On April 23, 2014, the closing sale price per share on The NASDAQ Global Select Market was $9.62.D. Selling Shareholders Not applicable.E. Dilution Not applicable.F. Expenses of the Issue Not applicable.Item 10 Additional Information A. Share Capital Not applicable.B. Memorandum and Articles of Association We incorporate by reference into this Annual Report the description of our amended articles of association contained in our F-1 registrationstatement (File No. 333-193158) originally filed with the SEC on January 2, 2014, as amended. Our articles of association were amended and weconverted our company into a public company with limited liability (naamloze venootschap) effective February 10, 2014.C. Material Contracts We have not entered into any material contracts other than in the ordinary course of business, as described under "Business—IntellectualProperty", "—Strategic Collaboration: Chiesi," and "—Strategic Collaboration: 4D Molecular Therapeutics."99 High Low Annual Highs and Lows $ $ 2014 (from February 4, 2014 through April 11, 2014) 18.75 13.00 Annual and Quarterly Highs and Lows First Quarter 2014 (from February 4, 2014) 18.75 13.10 Second Quarter 2014 (through April 11, 2014) 16.50 13.00 Monthly Highs and Lows February 2014 (from February 4, 2014) 18.75 13.10 March 2014 17.10 14.70 April 2014 (through April 11, 2014) 16.50 13.00 Table of ContentsD. Exchange Controls Under existing laws of the Netherlands, there are no exchange controls applicable to the transfer to persons outside of the Netherlands of dividendsor other distributions with respect to, or of the proceeds from the sale of, shares of a Dutch company.E. TaxationTaxation in the Netherlands This taxation summary solely addresses the principal Dutch tax consequences of the acquisition, ownership and disposal of ordinary shares. Itdoes not purport to describe all the tax considerations that may be relevant to a particular holder of our ordinary shares, or a Shareholder. Shareholdersare advised to consult their tax counsel with respect to the tax consequences of acquiring, holding and/or disposing of ordinary shares. Where in thissummary English terms and expressions are used to refer to Dutch concepts, the meaning to be attributed to such terms and expressions shall be themeaning to be attributed to the equivalent Dutch concepts under Dutch tax law. This summary does not address the tax consequences of:•A Shareholder who is an individual, either resident or non-resident in the Netherlands, and who has a substantial interest (aanmerkelijkbelang) in us within the meaning of the Dutch Income Tax Act 2001 (Wet inkomstenbelasting 2001). Generally, if a person holds aninterest in us, such interest forms part of a substantial interest, or a deemed substantial interest, in us, if any or more of the followingcircumstances is present: •If a Shareholder, either alone or, in the case of an individual, together with his partner owns or is deemed to own, directly orindirectly, either a number of shares in us representing five percent or more of our total issued and outstanding capital (or theissued and outstanding capital of any class of our shares), or rights to acquire, directly or indirectly, shares, whether or notalready issued, representing five percent or more of our total issued and outstanding capital (or the issued and outstanding capitalof any class of our shares), or profit participating certificates (winstbewijzen), relating to five percent or more of our annual profitor to five percent of our liquidation proceeds. •If the shares, profit participating certificates or rights to acquire shares in us are held or deemed to be held following theapplication of a non-recognition provision. •If the partner of a Shareholder, or one of certain relatives of the Shareholder or of this partner has a substantial interest (asdescribed under 1. and 2. above) in us. •A Shareholder receiving income or realizing capital gains in their capacity as future, present or past employee (werknemer) ormember of a management board (bestuurder), or supervisory director (commissaris). •Pension funds, investment institutions (fiscale beleggingsinstellingen), exempt investment institutions (vrijgesteldebeleggingsinstellingen) and other entities that are exempt from corporate income tax in the Netherlands, as well as entities that areexempt from corporate income tax in their country of residence, such country of residence being another state of the EuropeanUnion, Norway, Liechtenstein, Iceland or any other state with which the Netherlands have agreed to exchange information in linewith international standards. For purposes of Dutch personal income tax and Dutch corporate income tax, ordinary shares legally owned by a third party, such as a trustee,foundation or similar entity or arrangement, may under certain circumstances have to be allocated to the (deemed) settler, grantor or similar organiser100Table of Contentsor, upon the death of the Settlor, his/her beneficiaries in proportion to their entitlement to the estate of the Settlor of such trust or similar arrangement. This summary is based on the tax laws and principles (unpublished case law not included) in the Netherlands as in effect on the date of this annualreport, which are subject to changes that could prospectively or retroactively affect the stated tax consequences. Where in this summary the terms "theNetherlands" and "Dutch" are used, these refer solely to the European part of the Kingdom of the Netherlands.Dividend Withholding TaxGeneral We are generally required to withhold Dutch dividend withholding tax at a rate of 15% from dividends distributed by us. The concept dividends"distributed by us" as used in this section includes, but is not limited to:•distributions of profits in cash or in kind, whatever they may be named or in whatever form; •liquidation proceeds, or proceeds from the repurchase of ordinary shares by us in excess of the average paid-in capital recognized forDutch dividend withholding tax purposes; •the par value of ordinary shares issued to a Shareholder in us or an increase of the par value of ordinary shares, to the extent that it doesnot appear that a contribution, recognized for Dutch dividend withholding tax purposes, has been made or will be made; and •partial repayment of share capital, if and to the extent that there are net profits (zuivere winst), unless (a) the general meeting ofshareholders has resolved in advance to make such repayment and (b) the par value of the shares concerned has been reduced by anequal amount by way of an amendment to our articles of association. In general, we will be required to remit all amounts withheld as Dutch dividend withholding tax to the Dutch tax authorities. However, undercertain circumstances, we are allowed to reduce the amount to be remitted to the Dutch tax authorities by the lesser of:•3% of the portion of the distribution paid by us that is subject to Dutch dividend withholding tax; and •3% of the dividends and profit distributions, before deduction of foreign withholding taxes, received by us from qualifying foreignsubsidiaries in the current calendar year (up to the date of the distribution by us) and the two preceding calendar years, as far as suchdividends and profit distributions have not yet been taken into account for purposes of establishing the above mentioned reduction. Although this reduction reduces the amount of Dutch dividend withholding tax that we are required to remit to the Dutch tax authorities, it does notreduce the amount of tax that we are required to withhold on dividends distributed.Residents of the Netherlands A Shareholder which is resident or deemed resident in the Netherlands is generally entitled to a full credit of any Dutch dividend withholding taxagainst the Dutch (corporate) income tax liability of such Shareholder, and is generally entitled to a refund in the form of a negative assessment of Dutch(corporate) income tax, insofar such Dutch dividend withholding tax, together with any other creditable domestic and/or foreign taxes, exceeds suchShareholder's aggregate Dutch income tax or Dutch corporate income tax liability.101Table of Contents If and to the extent that such a corporate Shareholder is eligible for the application of the participation exemption with respect to the ordinaryshares, dividends distributed by us are in principle exempt from Dutch dividend withholding tax. Pursuant to domestic anti-dividend stripping rules, no exemption from Dutch dividend withholding tax, credit against Dutch (corporate) incometax, refund or reduction of Dutch dividend withholding tax shall apply if the recipient of the dividend paid by us is not considered to be the beneficialowner (uiteindelijk gerechtigde) as meant in these rules, of such dividends.Non-residents of the Netherlands (including but not limited to U.S. Shareholders) A non-resident Shareholder, which is resident in the non-European part of the Kingdom of the Netherlands or in a country that has concluded a taxtreaty with the Netherlands, may be eligible for a full or partial relief from Dutch dividend withholding tax, provided such relief is timely and dulyclaimed. In addition, a non-resident Shareholder that is not an individual, is entitled to an exemption from Dutch dividend withholding tax, provided thateach of the following tests are satisfied:•the non-resident Shareholder is, according to the tax law of a Member State of the European Union or a state designated by a ministerialdecree, that is a party to the Agreement regarding the European Economic Area, resident there and it is not transparent for tax purposesaccording to the tax law of such state; •anyone or more of the following threshold conditions are satisfied: •at the time the dividend is distributed by us, the non-resident Shareholder holds shares representing at least five percent of ournominal paid-up capital; or •the non-resident Shareholder has held shares representing at least five percent of our nominal paid-up capital for a continuousperiod of more than one year at any time during four years preceding the time the dividend is distributed by us; or •the non-resident Shareholder is connected with us within the meaning of article 10a, paragraph 4 of the Dutch Corporate IncomeTax Act 1969 (Wet op de vennootschapsbelasting 1969, or CITA); or •an entity connected with the non-resident Shareholder within the meaning of article 10a, paragraph 4 of CITA holds at the time ofthe dividends distributed by us, shares representing at least five per cent of our nominal paid-up capital; and •the non-resident Shareholder is not considered to be resident outside the Member States of the European Union or the states designatedby ministerial decree, that are party to the Agreement regarding the European Economic Area, under the terms of a tax treaty concludedwith a third state. A non-resident Shareholder which is resident in a Member State of the European Union with which the Netherlands has concluded a tax treaty thatprovides for a reduction of Dutch tax on dividends based on the ownership of the number of voting rights, the test under 2.a. above is also satisfied ifthe non-resident Shareholder owns at least five percent of the voting rights in us. The exemption from Dutch dividend withholding tax is not available to a non-resident Shareholder if pursuant to a provision for the prevention offraud or abuse included in a tax treaty between the Netherlands and the country of residence of the non-resident Shareholder, the non-residentShareholder is not entitled to the reduction of Dutch tax on dividends provided for by such treaty.102Table of Contents Furthermore, pursuant to domestic anti-dividend stripping rules, no exemption from Dutch dividend withholding tax, refund or reduction of Dutchdividend withholding tax shall apply if the recipient of the dividend paid by us is not considered to be the beneficial owner (uiteindelijk gerechtigde) asmeant in these rules, of such dividends. The Dutch tax authorities have taken the position that this beneficial ownership test can also be applied to denyrelief from Dutch dividend withholding tax under tax treaties and the Tax Arrangement for the Kingdom (Belastingregeling voor het Koninkrijk). A non-resident Shareholder which is subject to Dutch income tax or Dutch corporate income tax in respect of any benefits derived or deemed to bederived from ordinary shares, including any capital gain realized on the disposal thereof, can generally credit Dutch dividend withholding tax against itsDutch income tax or its Dutch corporate income tax liability, as applicable, and is generally entitled to a refund pursuant to a negative tax assessment ifand to the extent the Dutch dividend withholding tax, together with any other creditable domestic and/or foreign taxes, exceeds its aggregate Dutchincome tax or its aggregate Dutch corporate income tax liability, respectively.Taxes on Income and Capital GainsResidents of the NetherlandsIndividuals A Shareholder, who is an individual resident or deemed to be resident in the Netherlands, or who has elected to be taxed as a resident of theNetherlands for Dutch personal income tax purposes, will be subject to regular Dutch personal income tax at progressive rates (up to a maximum rate of52%) under the Dutch Income Tax Act 2001 on the income derived from the ordinary shares and gains realized on the disposal thereof if:•such Shareholder derives any benefits from the ordinary shares, which are attributable to an enterprise of such Shareholder, whether asan entrepreneur or pursuant to a co-entitlement to the net worth of an enterprise, other than as a shareholder or an entrepreneur; or •such income or gain is taxable in the hands of such Shareholder as benefits from miscellaneous activities (resultaat uit overigewerkzaamheden), including but not limited to activities with respect to the ordinary shares that are beyond the scope of regular activeportfolio management activities. If neither of the two abovementioned conditions apply, such Shareholder must determine his or her taxable income with regard to the ordinaryshares on the basis of a deemed return on income from savings and investments (sparen en beleggen), rather than on the basis of income actuallyreceived or gains actually realized. This deemed return on income from savings and investments has been fixed at a rate of 4% of the individual's yieldbasis at the beginning of the calendar year, insofar as the individual's yield basis exceeds a certain threshold. The individual's yield basis is determinedas the fair market value of certain qualifying assets held by the individual less the fair market value of certain qualifying liabilities at the beginning of thecalendar year.Corporate entities Generally, corporate Shareholders that are resident or deemed to be resident in the Netherlands for Dutch corporate income tax purposes will besubject to regular Dutch corporate income tax, levied at a rate of 25% (20% over profits up to €200,000) over income derived from the ordinary sharesand gains realized upon acquisition, redemption and disposal of ordinary shares. If and to the extent that such Shareholder is eligible for the application of the participation exemption with respect to the ordinary shares, incomederived from the ordinary shares and gains and103Table of Contentslosses (with the exception of liquidation losses under strict conditions) realized on the ordinary shares may be exempt from Dutch corporate income tax.Non-residents of the Netherlands (including but not limited to U.S. Shareholders)Individuals A Shareholder, who is an individual not resident or deemed to be resident in the Netherlands, and who has not elected to be taxed as a resident ofthe Netherlands for Dutch income tax purposes, will not be subject to any Dutch taxes on income or capital gains in respect of dividends distributed byus or in respect of any gain realized on the disposal of ordinary shares (other than dividend withholding tax as described above), except if:•such holder has an enterprise or an interest in an enterprise that is, in whole or in part, carried on through a permanent establishment or apermanent representative in the Netherlands and to which enterprise or part of an enterprise, as the case may be, the ordinary shares areattributable; or •such income or gain such income or gain is taxable in the hands of such Shareholder as benefits from miscellaneous activities" (resultaatuit overige werkzaamheden), including but not limited to activities with respect to the ordinary shares that are beyond the scope ofregular active portfolio management. If one of the two abovementioned conditions apply, the income or gains in respect of dividends distributed by us or in respect of any capital gainrealized on the disposal of ordinary shares will in general be subject to Dutch personal income tax at the progressive rates up to 52%.Corporate entities A corporate Shareholder, which is not resident or deemed to be resident in the Netherlands for Dutch corporate income tax purposes, will not besubject to any Dutch taxes on income or capital gains in respect of dividends distributed by us, or in respect of any gain realized, on the disposal ofordinary shares (other than dividend withholding tax as described above), except if:•such Shareholder has an enterprise or an interest in an enterprise that is, in whole or in part, carried on through a permanentestablishment or a permanent representative in the Netherlands, to which the ordinary shares are attributable; or •such holder has a substantial interest or a deemed substantial interest in us (as described above), that (i) is held with the evasion ofincome tax or dividend withholding tax as (one of) the main purpose(s) and (ii) is not attributable to the assets of an enterprise of suchShareholder; or •such holder is an entity resident of Aruba, Curaçao or Saint Martin with a permanent establishment or permanent representative inBonaire, Saint Eustatius or Saba to which such income or gain is attributable, and the permanent establishment or permanentrepresentative would be deemed to be resident of the Netherlands for Dutch corporate income tax purposes (i) had the permanentestablishment been a corporate entity (lichaam), or (ii) had the activities of the permanent representative been conducted by a corporateentity, respectively. If one of the abovementioned conditions applies, income derived from the ordinary shares and gains realized on ordinary shares will, in general, besubject to regular Dutch corporate income tax levied at a rate of 25% (20% over profits up to €200,000), except that a holder referred to under (2) abovewill generally be subject to an effective corporate income tax rate of 15% if it holds the substantial interest in us only with the purpose of avoidingdividend withholding tax and not with (one of) the main purposes to avoid income tax.104Table of ContentsGift or Inheritance Taxes No Dutch gift or Dutch inheritance tax is due in respect of any gift, in form or in substance, of the ordinary shares by, or inheritance of the shareson the death of, a Shareholder except if:•at the time of the gift or death of the Shareholder, the Shareholder is resident, or deemed to be resident, in the Netherlands for purposesof Dutch gift tax or Dutch inheritance tax, as applicable; or •in the case of a gift of ordinary shares by an individual who at the date of the gift was neither resident nor deemed to be resident in theNetherlands (i) such individual dies within 180 days after the date of the gift, while being resident or deemed to be resident in theNetherlands; or (ii) the gift of ordinary shares is made under a condition precedent and the Shareholder is resident, or is deemed to beresident in the Netherlands at the time the condition is fulfilled. For purposes of the above, a gift of ordinary shares made under a condition precedent (opschortende voorwaarde) is deemed to be made at thetime the condition precedent is satisfied. For purposes of Dutch gift or Dutch inheritance taxes, an individual not holding the Dutch nationality will be deemed to be resident in theNetherlands, inter alia, if he or she has been resident in the Netherlands at any time during the ten years preceding the date of the gift or his or her death.Additionally, for purposes of Dutch gift tax, an individual not holding the Dutch nationality will be deemed to be resident in the Netherlands if he or shehas been resident in the Netherlands at any time during the twelve months preceding the date of the gift. Applicable tax treaties may override deemedresidency in the Netherlands.Value Added Tax No Dutch value added tax will arise in respect of payments in consideration for the issue, acquisition, ownership and disposal of ordinary shares,other than value added taxes on fees payable in respect of services not exempt from Dutch value added tax.Other Taxes and Duties No Dutch registration tax, capital tax, custom duty, transfer tax, stamp duty or any other similar tax or duty, other than court fees, will be payablein the Netherlands in respect of or in connection with the subscription, issue, placement, allotment, delivery or transfer of the ordinary shares.Residence A Shareholder will not become resident, or deemed resident in the Netherlands for tax purposes by reason only of holding the ordinary shares.Taxation in the United States The following summary of the material U.S. federal income tax consequences of the acquisition, ownership and disposition of our ordinary sharesis based upon current law and does not purport to be a comprehensive discussion of all the tax considerations that may be relevant to our ordinaryshares. This summary is based on current provisions of the Code, existing, final, temporary and proposed U.S. Treasury Regulations, administrativerulings and judicial decisions, in each case as available on the date of this annual report. All of the foregoing are subject to change, which change couldapply retroactively and could affect the tax consequences described below. This section summarizes the material U.S. federal income tax consequences to U.S. holders, as defined below, of ordinary shares. This summaryaddresses only the U.S. federal income tax considerations for U.S. holders that acquire the ordinary shares at their original issuance and hold the105Table of Contentsordinary shares as capital assets. This summary does not address all U.S. federal income tax matters that may be relevant to a particular U.S. holder.Each prospective investor should consult a professional tax advisor with respect to the tax consequences of the acquisition, ownership ordisposition of the ordinary shares. This summary does not address tax considerations applicable to a holder of ordinary shares that may be subject tospecial tax rules including, without limitation, the following:•certain financial institutions; •insurance companies; •dealers or traders in securities, currencies, or notional principal contracts; •tax-exempt entities; •regulated investment companies; •persons that hold the ordinary shares as part of a hedge, straddle, conversion, constructive sale or similar transaction involving more thanone position; •persons that hold the ordinary shares through partnerships or certain other pass-through entities; •holders (whether individuals, corporations or partnerships) that are treated as expatriates for some or all U.S. federal income taxpurposes; •holders that own (or are deemed to own) 10% or more of our voting shares; and •holders that have a "functional currency" other than the U.S. dollar. Further, this summary does not address alternative minimum tax consequences or the indirect effects on the holders of equity interests in entitiesthat own our ordinary shares. In addition, this discussion does not consider the U.S. tax consequences to holders of ordinary shares that are not "U.S.holders" (as defined below). For the purposes of this summary, a "U.S. holder" is a beneficial owner of ordinary shares that is (or is treated as), for U.S. federal income taxpurposes:•an individual who is either a citizen or resident of the United States; •a corporation, or other entity that is treated as a corporation for U.S. federal income tax purposes, created or organized in or under thelaws of the United States or any state of the United States or the District of Columbia; •an estate, the income of which is subject to U.S. federal income taxation regardless of its source; or •a trust, if a court within the United States is able to exercise primary supervision over its administration and one or more U.S. personshave the authority to control all of the substantial decisions of such trust or has a valid election in effect under applicable U.S. TreasuryRegulations to be treated as a U.S. person. If a partnership holds ordinary shares, the tax treatment of a partner will generally depend upon the status of the partner and upon the activities ofthe partnership. We will not seek a ruling from the U.S. Internal Revenue Service, or IRS, with regard to the U.S. federal income tax treatment of an investment inour ordinary shares, and we cannot provide assurance that that the IRS will agree with the conclusions set forth below. Distributions. Subject to the discussion under "Passive Foreign Investment Company Considerations" below, the gross amount of anydistribution (including any amounts withheld in respect of Dutch106Table of Contentswithholding tax) actually or constructively received by a U.S. holder with respect to ordinary shares will be taxable to the U.S. holder as a dividend tothe extent of our current and accumulated earnings and profits as determined under U.S. federal income tax principles. Distributions in excess ofearnings and profits will be non-taxable to the U.S. holder to the extent of, and will be applied against and reduce, the U.S. holder's adjusted tax basis inthe ordinary shares. Distributions in excess of earnings and profits and such adjusted tax basis will generally be taxable to the U.S. holder as capitalgain from the sale or exchange of property. However, since we do not calculate our earnings and profits under U.S. federal income tax principles, it isexpected that any distribution will be reported as a dividend, even if that distribution would otherwise be treated as a non-taxable return of capital or ascapital gain under the rules described above. The amount of any distribution of property other than cash will be the fair market value of that property onthe date of distribution. The U.S. holder will not be eligible for any dividends-received deduction in respect of the dividend otherwise allowable tocorporations. Under the Code and subject to the discussion below regarding the "Medicare tax," qualified dividends received by non-corporate U.S. holders (i.e.,individuals and certain trusts and estates) are subject to a maximum income tax rate of 20%. This reduced income tax rate is applicable to dividends paidby "qualified foreign corporations" to such non-corporate U.S. holders that meet the applicable requirements, including a minimum holding period(generally, at least 61 days during the 121-day period beginning 60 days before the ex-dividend date). We expect to be considered a qualified foreigncorporation under the Code. Accordingly, dividends paid by us to non-corporate U.S. holders with respect to shares that meet the minimum holdingperiod and other requirements are expected to be treated as "qualified dividend income." However, dividends paid by us will not qualify for the 20%maximum U.S. federal income tax rate if we are treated, for the tax year in which the dividends are paid or the preceding tax year, as a "passive foreigninvestment company" for U.S. federal income tax purposes, as discussed below. Dividends received by a U.S. holder with respect to ordinary shares generally will be treated as foreign source income for the purposes ofcalculating that holder's foreign tax credit limitation. Subject to applicable conditions and limitations, and subject to the discussion in the next paragraph,any Dutch income tax withheld on dividends may be deducted from taxable income or credited against a U.S. holder's U.S. federal income tax liability.The limitation on foreign taxes eligible for the U.S. foreign tax credit is calculated separately with respect to specific classes of income. For this purpose,dividends distributed by us generally will constitute "passive category income" (but, in the case of some U.S. holders, may constitute "general categoryincome"). Upon making a distribution to shareholders, we may be permitted to retain a portion of the amounts withheld as Dutch dividend withholding tax.See "—Taxation in the Netherlands—Dividend Withholding Tax—General." The amount of Dutch withholding tax that we may retain reduces theamount of dividend withholding tax that we are required to pay to the Dutch tax authorities but does not reduce the amount of tax we are required towithhold from dividends paid to U.S. holders. In these circumstances, it is likely that the portion of dividend withholding tax that we are not required topay to the Dutch tax authorities with respect to dividends distributed to U.S. holders would not qualify as a creditable tax for U.S. foreign tax creditpurposes. Sale or other disposition of ordinary shares. A U.S. holder will generally recognize gain or loss for U.S. federal income tax purposes upon thesale or exchange of ordinary shares in an amount equal to the difference between the U.S. dollar value of the amount realized from such sale orexchange and the U.S. holder's tax basis for those ordinary shares. Subject to the discussion under "Passive Foreign Investment CompanyConsiderations" below, this gain or loss will generally be a capital gain or loss and will generally be treated as from sources within the United States.Such capital gain or loss will be treated as long-term capital gain or loss if the U.S. holder has held the ordinary shares for more than one year at thetime of the sale or exchange. Long-term capital gains of non-corporate holders may be eligible for a preferential tax rate; the deductibility of capitallosses is subject to limitations.107Table of Contents Medicare Tax. A "United States person," within the meaning of the Code, that is an individual, an estate or a nonexempt trust is generally subjectto a 3.8% surtax on the lesser of (i) the United States person's "net investment income" for the year and (ii) the excess of the United States person's"modified adjusted gross income" for that year over a threshold (which, in the case of an individual, will be between $125,000 and $250,000,depending on the individual's U.S. tax filing status). A U.S. holder's net investment income generally will include, among other things, dividends on,and gains from the sale or other taxable disposition of, our ordinary shares, unless (with certain exceptions) those dividends or gains are derived in theordinary course of a trade or business. Net investment income may be reduced by deductions properly allocable thereto; however, the U.S. foreign taxcredit may not be available to reduce the surtax. An additional 3.8% tax is imposed on the net investment income (which includes taxable dividends and net capital gains) received by certain U.S.holders that are individuals, trusts or estates.Passive foreign investment company considerations. A corporation organized outside the United Statesgenerally will be classified as a passive foreign investment company, or PFIC, for U.S. federal income tax purposes in any taxable year in which either:(i) at least 75% of its gross income is passive income, or (ii) on average at least 50% of the gross value of its assets is attributable to assets that producepassive income or are held for the production of passive income. In arriving at this calculation, a pro rata portion of the income and assets of eachcorporation in which we own, directly or indirectly, at least a 25% interest, as determined by the value of such corporation, must be taken into account.Passive income for this purpose generally includes dividends, interest, royalties, rents and gains from commodities and securities transactions. We believe that we were not a PFIC for the 2013 taxable year. Based on our estimated gross income, the average value of our gross assets, and thenature of the active businesses conducted by our "25% or greater" owned subsidiaries, we do not believe that we will be classified as a PFIC in thecurrent taxable year and do not expect to become one in the foreseeable future. However, our status for any taxable year will depend on our assets andactivities in each year, and because this is a factual determination made annually after the end of each taxable year, there can be no assurance that we willnot be considered a PFIC for the current taxable year or any future taxable year. The market value of our assets may be determined in large part byreference to the market price of our ordinary shares, which is likely to fluctuate and may fluctuate considerably given that market prices of technologycompanies have been especially volatile. In addition, the composition of our income and assets will be affected by how, and how quickly, we spend theour cash. If we were a PFIC for any taxable year during which a U.S. holder held ordinary shares, under the "default PFIC regime" (i.e., in the absence ofone of the elections described below) gain recognized by the U.S. holder on a sale or other disposition (including a pledge) of the ordinary shares wouldbe allocated ratably over the U.S. holder's holding period for the ordinary shares. The amounts allocated to the taxable year of the sale or otherdisposition and to any year before we became a PFIC would be taxed as ordinary income. The amount allocated to each other taxable year would besubject to tax at the highest rate in effect for individuals or corporations, as appropriate, for that taxable year, and an interest charge would be imposedon the resulting tax liability for that taxable year. Similar rules would apply to the extent any distribution in respect of ordinary shares exceeds 125% ofthe average of the annual distributions on ordinary shares received by a U.S. holder during the preceding three years or the holder's holding period,whichever is shorter. In the event we were treated as a PFIC, the tax consequences under the default PFIC regime described above could be avoided by either a "mark-to-market" or "qualified electing fund" election. As long as our ordinary shares are regularly traded on the NASDAQ Global Select Market or another"qualified exchange," a U.S. holder making a mark-to-market election generally would not be subject to the PFIC rules discussed above, except withrespect to any portion of the holder's holding period that precedes the effective date of the election. Instead, the electing holder would include in ordinary108Table of Contentsincome, for each taxable year in which we were a PFIC, an amount equal to any excess of (a) the fair market value of the ordinary shares as of the closeof such taxable year over (b) the electing holder's adjusted tax basis in such ordinary shares. In addition, an electing holder would be allowed adeduction in an amount equal to the lesser of (a) the excess, if any, of (i) the electing holder's adjusted tax basis in the ordinary shares over (ii) the fairmarket value of such ordinary shares as of the close of such taxable year or (b) the excess, if any, of (i) the amount included in ordinary income becauseof the election for prior taxable years over (ii) the amount allowed as a deduction because of the election for prior taxable years. The election wouldcause adjustments in the electing holder's tax basis in the ordinary shares to reflect the amount included in gross income or allowed as a deductionbecause of the election. In addition, upon a sale or other taxable disposition of ordinary shares, an electing holder would recognize ordinary income orloss (not to exceed the excess, if any, of (a) the amount included in ordinary income because of the election for prior taxable years over (b) the amountallowed as a deduction because of the election for prior taxable years). Alternatively, a U.S. holder making a valid and timely "QEF election" generally would not be subject to the default PFIC regime discussed above.Instead, for each PFIC year to which such an election applied, the electing holder would be subject to U.S. federal income tax on the electing holder'spro rata share of our net capital gain and ordinary earnings, regardless of whether such amounts were actually distributed to the electing holder.However, because we do not intend to prepare or provide the information that would permit the making of a valid QEF election, that election will not beavailable to U.S. holders. If we were considered a PFIC for the current taxable year or any future taxable year, a U.S. holder would be required to file annual informationreturns for such year, whether or not the U.S. holder disposed of any ordinary shares or received any distributions in respect of ordinary shares duringsuch year. Backup Withholding and Information Reporting. U.S. holders generally will be subject to information reporting requirements with respect todividends on ordinary shares and on the proceeds from the sale, exchange or disposition of ordinary shares that are paid within the United States orthrough U.S.-related financial intermediaries, unless the U.S. holder is an "exempt recipient." In addition, U.S. holders may be subject to backupwithholding (at a 28% rate) on such payments, unless the U.S. holder provides a taxpayer identification number and a duly executed IRS Form W-9 orotherwise establishes an exemption. Backup withholding is not an additional tax, and the amount of any backup withholding will be allowed as a creditagainst a U.S. holder's U.S. federal income tax liability and may entitle such holder to a refund, provided that the required information is timelyfurnished to the IRS.F. Dividends and Paying Agents Not applicable.G. Statements by Experts Not applicable.H. Documents on Display We are subject to the periodic reporting and other informational requirements of the Securities Exchange Act of 1934, as amended, or the ExchangeAct. Under the Exchange Act, we are required to file reports and other information with the SEC. Specifically, we are required to file annually aForm 20-F no later than four months after the close of each fiscal year, which is December 31. Copies of reports and other information, when so filed,may be inspected without charge and may be obtained at prescribed rates at the public reference facilities maintained by the Securities and Exchange109Table of ContentsCommission at Judiciary Plaza, 100 F Street, N.E., Washington, D.C. 20549, and at the regional office of the Securities and Exchange Commissionlocated at Citicorp Center, 500 West Madison Street, Suite 1400, Chicago, Illinois 60661. The public may obtain information regarding the Washington,D.C. Public Reference Room by calling the Commission at 1-800-SEC-0330. The SEC also maintains a web site at www.sec.gov that contains reports,proxy and information statements, and other information regarding registrants that make electronic filings with the SEC using its EDGAR system. As aforeign private issuer, we are exempt from the rules under the Exchange Act prescribing the furnishing and content of quarterly reports and proxystatements, and officers, directors and major shareholders are exempt from the reporting and short-swing profit recovery provisions contained inSection 16 of the Exchange Act.I. Subsidiary Information Not applicable.Item 11 Quantitative and Qualitative Disclosures About Market Risk See "Operating and Financial Review and Prospects—Quantitative and Qualitative Disclosures about Market Risk."Item 12 Description of Securities Other than Equity Securities Not applicable.PART II Item 13 Defaults, Dividend Arrearages and Delinquencies Not applicable.Item 14 Material Modification to the Rights of Security Holders and Use of Proceeds A. Material Modifications to the Rights of Securities Holders Not applicable.B. Use of Proceeds The following "Use of Proceeds" information relates to our initial public offering, at $17.00 per ordinary share, of 5,400,000 ordinary shares. Theaggregate offering price was $91,800,000, before underwriting discounts and commissions and offering expenses. The registration statement onForm F-1 (File No. 333-193158) for our initial public offering was declared effective by the SEC on February 4, 2013. Jefferies LLC, LeerinkPartners LLC and Piper Jaffray & Co. were the underwriters for our initial public offering. We received proceeds of $85.4 million (€62.6 million) from our initial public offering, net of underwriting discounts and commissions but beforeexpenses. To date, we have used €9.3 million of the net proceeds of the offering, including €4.3 million to complete the building out and equipping ofour manufacturing facility in Lexington. We intend to use the remaining net proceeds to complete the building out and equipping of our manufacturingfacility in Lexington, Massachusetts; to support our further clinical development of Glybera, and our application for marketing approval of Glybera andpreparation for potential commercial launch in the United States; to fund our share of the costs of our planned Phase I/II clinical trial of AMT 060 inhemophilia B; to advance the development of our other product candidates and research activities, including our collaboration with 4D MolecularTherapeutics; and for working capital and for general corporate purposes, including the costs of operating our110Table of Contentsfacilities in Amsterdam and in Lexington, Massachusetts, service on our indebtedness and potentially acquisitions or investments in other businesses,technologies or product candidates. Our management retains broad discretion in the allocation and use of the remaining net proceeds of our initial publicoffering. Pending such decisions, we have invested such proceeds in investment grade, interest bearing securities or bank deposits.Item 15 Control and Procedures The company's management, with the participation of the company's chief executive officer and chief financial officer, evaluated the effectivenessof the company's disclosure controls and procedures as of December 31, 2013. The term "disclosure controls and procedures," as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act, means controls and other procedures of a company that are designed to ensure that information required tobe disclosed by a company in the reports that it files or submits under the Exchange Act is recorded, processed, summarized and reported, within thetime periods specified in the SEC's rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed toensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is accumulated andcommunicated to the company's management, including its principal executive and principal financial officers, as appropriate to allow timely decisionsregarding required disclosure. Based on the evaluation of the company's disclosure controls and procedures as of December 31, 2013, the company'schief executive officer and chief financial officer concluded that, as of such date, the company's disclosure controls and procedures were not effective asa result of the material weaknesses in internal control described below. This annual report does not include a report of management's assessment regarding internal control over financial reporting or an attestation reportof the company's independent registered public accounting firm due to a transition period established by rules of the Securities and ExchangeCommission for newly public companies. Neither we nor our independent registered public accounting firm undertook a comprehensive assessment of our internal control for purposes ofidentifying and reporting material weaknesses, significant deficiencies and control deficiencies in our internal control over financial reporting. In itsreview of internal control over financial reporting in connection with the company's initial public offering in February 2014, management identified threematerial weaknesses:•a lack of accounting resources required to fulfill IFRS and SEC reporting requirements; •a lack of comprehensive IFRS accounting policies and financial reporting procedures; and •a lack of segregation of duties given the size of our finance and accounting team. We have implemented and are continuing to implement various measures to address the material weaknesses identified; these measures are outlinedin "Operating and Financial Review and Prospects—Internal Control Over Financial Reporting". We believe it is possible that, had we performed a formal assessment of our internal control over financial reporting or had our independentregistered public accounting firm performed an audit of our internal control over financial reporting, additional control deficiencies may have beenidentified. There has been no change in our internal control over financial reporting (as defined in Rule 13a-15(f) under the Exchange Act) during the periodcovered by this annual report that has materially affected, or is reasonably likely to materially affect, internal control over financial reporting, other thanas described in "Operating and Financial Review and Prospects-Internal Control Over Financial Reporting."111Table of ContentsItem 16A Audit Committee Financial Expert Mr. Ferdinand Verdonck, an independent director and a member of the Audit Committee, qualifies as an "audit committee financial expert," asdefined in Item 16A of Form 20-F and as determined by our supervisory board and management board.Item 16B Code of Ethics We have adopted a written code of ethics applicable to supervisory and managing directors, members of senior management and employees of thecompany and any of the company's direct and indirect subsidiaries. Our code of ethics is posted on our company website at:http://www.uniqure.com/uploads/Exhibit%20I%20_%20uniQure%20Code%20of%20Business%20Conduct%20and%20Ethics_(115245793)_(5).pdf Any amendments to our code of ethics will be disclosed on our website within five business days of the amendment.Item 16C Principal Accountant Fees and Services The following table sets forth, for each of the years indicated, the fees billed by our independent public accountants and the percentage of each ofthe fees out of the total amount billed by the accountants. Audit Fees are defined as the standard audit work that needs to be performed each year in order to issue opinions on our consolidated financialstatements and to issue reports on our local statutory financial statements. Also included are services that can only be provided by our auditor, such asreviews of quarterly financial results, consents and comfort letters and any other audit services required for SEC or other regulatory filings. Audit Related Fees include those other assurance services provided by the independent auditor but not restricted to those that can only be providedby the auditor signing the audit report. Tax Fees relate to the aggregated fees for services rendered on tax compliance.Pre-Approval Policies and Procedures for Non-Audit Services Our Audit Committee has adopted a policy pursuant to which we will not engage our auditors to perform any non-audit services unless the auditcommittee pre-approves the service, effective for the period following the completion of the IPO. The policy was not in place during 2013.Item 16D Exemptions From the Listing Requirements and Standards for Audit Committees Not applicable.Item 16E Purchases of Equity Securities by the Issuer and Affiliated Purchasers None.112 Year endedDecember 31,2011 Year endedDecember 31,2012 Year endedDecember 31,2013 EUR'000 % EUR'000 % EUR'000 % Audit Fees 167 66% 65 93% 1,021 98%Audit-related Fees 46 18% — —% — 0%Tax Fees 39 16% 5 7% 20 2% Total 252 100% 70 100% 1,041 100% Table of ContentsItem 16F Change in Registrants Certifying Accountant None.Item 16G Corporate Governance The Sarbanes-Oxley Act of 2002, as well as related rules subsequently implemented by the SEC, requires foreign private issuers, including ourcompany, to comply with various corporate governance practices. In addition, NASDAQ rules provide that foreign private issuers may follow homecountry practice in lieu of the NASDAQ corporate governance standards, subject to certain exceptions and except to the extent that such exemptionswould be contrary to U.S. federal securities laws. The home country practices followed by our company in lieu of NASDAQ rules are described below:•We do not follow NASDAQ's quorum requirements applicable to meetings of shareholders. In accordance with Dutch law and generallyaccepted business practice, our articles of association do not provide quorum requirements generally applicable to general meetings ofshareholders. •We do not follow NASDAQ's requirements regarding the provision of proxy statements for general meetings of shareholders. Dutchlaw does not have a regulatory regime for the solicitation of proxies and the solicitation of proxies is not a generally accepted businesspractice in the Netherlands. We do intend to provide shareholders with an agenda and other relevant documents for the general meetingof shareholders. We intend to take all actions necessary for us to maintain compliance as a foreign private issuer under the applicable corporate governancerequirements of the Sarbanes-Oxley Act of 2002, the rules adopted by the SEC and NASDAQ's listing standards. As a Dutch company listed on agovernment-recognized stock exchange, we are required to apply the provisions of the DCGC, or explain any deviation from the provisions of suchcode in our Dutch annual report required by Dutch law. Because we are a foreign private issuer, our supervisory board members, management board members and senior management are not subject toshort-swing profit and insider trading reporting obligations under section 16 of the U.S. Securities Exchange Act of 1934, as amended (the "ExchangeAct"). They will, however, be subject to the obligations to report changes in share ownership under section 13 of the Exchange Act and related SECrules.Item 16H Mine Safety Disclosure Not applicable.PART III Item 17 Financial Statements See "Item 18 Financial Statements."Item 18 Financial Statements See the Financial Statements beginning on page F-1.113Table of ContentsItem 19 Exhibits 114ExhibitNo. Description 1.1* Amended Articles of Association of the Company 4.1† Patent License Agreement (L-107-2007), effective as of May 2, 2007, by and between the Company and theNational Institutes of Health, as amended on December 31, 2009, May 31, 2013 and November 11, 2013(incorporated by reference to Exhibit 10.1 of the Company's registration statement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.2† Patent License Agreement (L-116-2011), effective as of August 10, 2011, by and between the Company andNational Institutes of Health, as amended on May 31, 2013 and November 11, 2013 (incorporated by referenceto Exhibit 10.2 of the Company's registration statement on form F-1 (file no. 333–193158) filed with theSecurities and Exchange Commission). 4.3† License Agreement, effective as of March 22, 2007, by and between the Company and Protein SciencesCorporation, as amended on June 13, 2012 (Incorporated by reference to Exhibit 10.3 of the Company'sregistration statement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission.) 4.4† Agreement, dated June 16, 2006, by and among the Company, Academish Medisch Centrum andBeheersmaatschappij Dienstverlening En Deelneming Azua (incorporated by reference to Exhibit 10.4 of theCompany's registration statement on form F-1 (file no. 333–193158) filed with the Securities and ExchangeCommission). 4.5† Sublicense and Research Agreement, effective June 18, 2001, by and between the Company and XenonGenetics Inc., as amended (incorporated by reference to Exhibit 10.5 of the Company's registration statementon form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission.). 4.6† License Agreement, effective as of December 20, 2006, between the Company and Aventis Pharma S.A., asamended on June 28, 2013 (incorporated by reference to Exhibit 10.6 of the Company's registration statementon form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.7† Non-Exclusive License Agreement, effective as of September 3, 2010, by and between the Company andAsklêpios Biopharmaceutical, Inc. (incorporated by reference to Exhibit 10.7 of the Company's registrationstatement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.8† License Agreement, dated February 8, 2008, by and between the Company and Salk Institute for BiologicalStudies (incorporated by reference to Exhibit 10.8 of the Company's registration statement on form F-1 (fileno. 333–193158) filed with the Securities and Exchange Commission). 4.9† License Agreement, dated December 5, 2006, by and between the Company and AmpliPhi Biosciences, Inc.,as amended on June 28, 2013 (incorporated by reference to Exhibit 10.9 of the Company's registrationstatement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.10† Exclusive License Agreement, effective as of July 7, 2008, by and between the Company and St. JudeChildren's Research Hospital, Inc., as amended on July 12, 2012 (incorporated by reference to Exhibit 10.10 ofthe Company's registration statement on form F-1 (file no. 333–193158) filed with the Securities and ExchangeCommission).Table of Contents115ExhibitNo. Description 4.11† Co-Development and License Agreement, entered into as of April 29, 2013, by and between the Company andChiesi Farmaceutici S.p.A. (incorporated by reference to Exhibit 10.11 of the Company's registration statementon form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.12† Commercialization Agreement, entered into as of April 29, 2013, by and between the Company and ChiesiFarmaceutici S.p.A. (incorporated by reference to Exhibit 10.12 of the Company's registration statement onform F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.13† License Agreement, dated as of May 21, 2010, by and among the Company, Fundacion para la InvestigacionMedica Applicada, Proyecto de Biomedicina CIMA S.L. and Digna Biotech, S.L. (incorporated by reference toExhibit 10.13 of the Company's registration statement on form F-1 (file no. 333–193158) filed with theSecurities and Exchange Commission). 4.14† Development and Manufacturing Agreement, effective as of January 7, 2011, by and between the Companyand Institut Pasteur, as amended on January 7, 2011 (incorporated by reference to Exhibit 10.14 of theCompany's registration statement on form F-1 (file no. 333–193158) filed with the Securities and ExchangeCommission). 4.15† License Agreement, effective as of November 30, 2010, by and between the Company and Amgen Inc.(incorporated by reference to Exhibit 10.15 of the Company's registration statement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.16† Data License Agreement, effective June 12, 2012, by and between the Company and The Regents of theUniversity of California, acting through its Office of Technology management, University of California, SanFrancisco (incorporated by reference to Exhibit 10.16 of the Company's registration statement on form F-1 (fileno. 333–193158) filed with the Securities and Exchange Commission). 4.17 Loan and Security Agreement, dated as of June 13, 2013, by and among the Company, uniQure IP B.V., theCompany's subsidiaries listed therein, and Hercules Technology Growth Capital, Inc. (incorporated byreference to Exhibit 10.17 of the Company's registration statement on form F-1 (file no. 333–193158) filedwith the Securities and Exchange Commission). 4.18 Warrant Agreement, dated as of September 20, 2013, by and among the Company, uniQure Biopharma B.V.and Hercules Technology Growth Capital, Inc. (incorporated by reference to Exhibit 10.18 of the Company'sregistration statement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.19 Subscription Agreement, dated as of April 29, 2013, by and among Chiesi Farmaceutici S.p.A and theCompany (incorporated by reference to Exhibit 10.19 of the Company's registration statement on form F-1 (fileno. 333–193158) filed with the Securities and Exchange Commission). 4.20 Lease relating to Meibergdreef 45, 57 and 61, dated as of July 1, 2012, by and among Academisch MedischCentrum and uniQure biopharma B.V. (incorporated by reference to Exhibit 10.26 of the Company'sregistration statement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission).Table of Contents116ExhibitNo. Description 4.21 Lease relating to 113 Hartwell Avenue, Lexington, Massachusetts, dated as of July 24, 2013, by and betweenthe Company and King113 Hartwell LLC (incorporated by reference to Exhibit 10.28 of the Company'sregistration statement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.22 Business Acquisition Agreement, dated as of February 16, 2012, by and among Amsterdam MolecularTherapeutics (AMT) Holding N.V., the Company and the other Parties listed therein (incorporated by referenceto Exhibit 10.29 of the Company's registration statement on form F-1 (file no. 333–193158) filed with theSecurities and Exchange Commission). 4.23 Deed of Assignment of Certain Assets and Liabilities of Amsterdam Molecular Therapeutics (AMT)Holding N.V., dated as of April 5, 2012, by and among Amsterdam Molecular Therapeutics (AMT)Holding B.V., Amsterdam Molecular Therapeutics (AMT) Holding IP B.V. and Amsterdam MolecularTherapeutics (AMT) Holding N.V. (incorporated by reference to Exhibit 10.30 of the Company's registrationstatement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.24 Agreement for Transfer of Certain Assets and Liabilities of Amsterdam Molecular Therapeutics (AMT)Holding N.V., dated as of February 16, 2012, by and among Amsterdam Molecular Therapeutics (AMT)Holding B.V., Amsterdam Molecular Therapeutics (AMT) Holding IP B.V. and Amsterdam MolecularTherapeutics (AMT) Holding N.V. (incorporated by reference to Exhibit 10.31 of the Company's registrationstatement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.25† Collaboration and License Agreement, dated January 17, 2014, by and between uniQure biopharma B.V. and4D Molecular Therapeutics, LLC (incorporated by reference to Exhibit 10.32 of the Company's registrationstatement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.26 Option Agreement, dated January 17, 2014, by and between the Company and Dr. David Kirn (incorporatedby reference to Exhibit 10.33 of the Company's registration statement on form F-1 (file no. 333–193158) filedwith the Securities and Exchange Commission). 4.27 Option Agreement, dated January 17, 2014, by and between the Company and Dr. David Schaffer(incorporated by reference to Exhibit 10.34 of the Company's registration statement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 4.28 Commitment Letter pursuant to Collaboration Agreement, dated January 17, 2014, by the Company andacknowledged and agreed by 4D Molecular Therapeutics, LLC, Dr. David Schaffer and Dr. David Kirn(incorporated by reference to Exhibit 10.35 of the Company's registration statement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 8.1* Subsidiaries of the Company (incorporated by reference to Exhibit 10.1 of the Company's registrationstatement on form F-1 (file no. 333–193158) filed with the Securities and Exchange Commission). 12.1* Certification by Principal Executive Officer Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 12.2* Certification by Principal Financial Officer Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002Table of Contents117ExhibitNo. Description 13.1* Certification by Principal Executive Officer and Principal Financial Officer Pursuant to Section 906 of theSarbanes-Oxley Act of 2002†Confidential treatment requested as to certain portions, which portions have been omitted and filed separately with the Securitiesand Exchange Commission *Filed herewithTable of ContentsSignatures The Registrant hereby certifies that it meets all of the requirements for filing on Form 20-F and that it has duly caused and authorized theundersigned to sign this annual report on its behalf. uniQure N.V. By: /s/ JÖRN ALDAGMr Jörn AldagManaging Director/Chief Executive Officer Date: April 25, 2014 By: /s/ PIERS MORGANMr Piers MorganManaging Director/Chief Financial Officer Date: April 25, 2014Table of ContentsUNIQURE N.V.INDEX TO CONSOLIDATED FINANCIAL STATEMENTS F-1 PageReport of Independent Registered Public Accounting Firm F-2Consolidated Balance Sheets as of December 31, 2012 and 2013 F-3Consolidated Statements of Comprehensive Loss for the Years Ended December 31, 2011, 2012 and 2013 F-4Consolidated Statements of Changes in (Deficit)/Equity for the Years Ended December 31, 2011, 2012 and 2013 F-5Consolidated Statements of Cash Flows for the Years Ended December 31, 2011, 2012 and 2013 F-6Notes to Consolidated Financial Statements F-7Table of ContentsREPORT OF INDEPENDENT REGISTERED PUBLIC ACCOUNTING FIRMTo Management Board and shareholders of uniQure N.V.: In our opinion, the accompanying consolidated balance sheets and the related consolidated statements of comprehensive loss, of changes in(deficit)/equity and of cash flows present fairly, in all material respects, the financial position of uniQure N.V. and its subsidiaries at December 31, 2013and December 31, 2012, and the results of their operations and their cash flows for each of the three years in the period ended December 31, 2013 inconformity with International Financial Reporting Standards as issued by the International Accounting Standards Board. These financial statements arethe responsibility of the Company's management. Our responsibility is to express an opinion on these financial statements based on our audits. Weconducted our audits of these statements in accordance with the standards of the Public Company Accounting Oversight Board (United States). Thosestandards require that we plan and perform the audit to obtain reasonable assurance about whether the financial statements are free of materialmisstatement. An audit includes examining, on a test basis, evidence supporting the amounts and disclosures in the financial statements, assessing theaccounting principles used and significant estimates made by management, and evaluating the overall financial statement presentation. We believe thatour audits provide a reasonable basis for our opinion/s/ PricewaterhouseCoopers Accountants N.V.Utrecht, The NetherlandsApril 25, 2014drs. A.C.M. van der Linden RAF-2Table of ContentsUNIQURE N.V.Consolidated Balance Sheets(€ in thousands) As of December 31, NOTE 2012 2013 Assets Non-current assets Intangible assets 5 3,278 7,775 Property, plant and equipment 6 1,185 2,614 Other non-current assets 7 — 923 Total non-current assets 4,463 11,312 Current assets Receivables from related parties 8,29 26 1,425 Trade and other receivables 8 815 1,557 Inventories 9 — 865 Cash and cash equivalents 10 263 23,810 Total current assets 1,104 27,657 Total assets 5,567 38,969 (Deficit)/Equity Share capital 483 610 Share premium 114,795 142,459 Other reserves 1,508 6,536 Accumulated deficit (117,234) (144,041) Total (Deficit)/equity 11 (448) 5,564 Liabilities Non-current liabilities Borrowings 16 — 6,292 Financial lease liabilities 13 450 302 Deferred rent 8,28 — 680 Deferred revenue 17 — 15,679 Total non-current liabilities 450 22,953 Current liabilities Trade and other payables 15 4,067 7,601 Debt to related party—financial liability 14 1,366 — Debt to related party—embedded derivative 14 132 722 Borrowings 16 — 633 Borrowings—embedded derivative 16 — 217 Deferred revenue 17 — 1,279 Total current liabilities 5,565 10,452 Total liabilities 6,015 33,405 Total equity and liabilities 5,567 38,969 The notes are an integral part of these consolidated financial statements.F-3Table of ContentsUNIQURE N.V.Consolidated Statements of Comprehensive Loss(€ in thousands, except share and per share data) The notes are an integral part of these consolidated financial statements.F-4 YEARS ENDEDDECEMBER 31, NOTE 2011 2012 2013 License revenues 17 — — 440 Collaboration revenues 17 — — 2,503 Total revenues — — 2,943 Cost of goods sold 28 — — (800)Other income 18 2,192 649 585 Research and development expenses 19,20,23 (15,500) (10,231) (13,182)Selling, general and administrative expenses 19,21,23 (3,807) (4,564) (11,628)Other losses, net 18 (26) (45) (453) Total Operating Costs (19,333) (14,840) (25,263) Operating result (17,141) (14,191) (22,535) Finance income 24 277 22 102 Finance expense 24 (436) (547) (4,387) Finance income/(expense)—net (159) (525) (4,285) Result before corporate income taxes (17,300) (14,716) (26,820)Corporate income taxes — — — Net Loss (17,300) (14,716) (26,820) Items that may be subsequently reclassified to profit or loss 22 — — 12 Other comprehensive income — — — Total comprehensive loss* (17,300) (14,716) (26,808) Loss per share attributable to the equity holders of the Company duringthe year Basic and diluted loss per share (in euro) 26 (3.65) (1.70) (2.48)*Total comprehensive loss is fully attributable to equity holders of the groupTable of ContentsUNIQURE N.V.Consolidated Statements of Changes in (Deficit)/Equity(€ in thousands) ATTRIBUTABLE TO EQUITY HOLDERS OF THE COMPANY NOTE SHARECAPITAL SHAREPREMIUMRESERVE OTHERRESERVES ACCUMULATEDDEFICIT TOTAL(DEFICIT)/EQUITY Balance atJanuary 1,2011 235 99,841 1,788 (88,205) 13,659 Result for theyear — — — (17,300) (17,300)Capitalcontributions 11 2 106 — — 108 Share-basedpaymentexpenses 11 — — 940 — 940 Balance atDecember 31,2011 237 99,947 2,728 (105,505) (2,593) Result for theyear (14,716) (14,716)Capitalcontributions 11 246 14,848 — — 15,094 Share-basedpaymentexpensesrelating to theAMT shareoption scheme 12 — — 259 — 259 Adjustment toreserves onexpiration ofthe AMToption scheme 11 — — (2,987) 2,987 — Share-basedpaymentexpensesrelating to theuniQure shareoption scheme 12 — — 1,508 — 1,508 Balance atDecember 31,2012 483 114,795 1,508 (117,234) (448) Result for theyear — — — (26,820) (26,820)OtherComprehensiveIncome 22 — — — 12 12  The notes are an integral part of these consolidated financial statementsF-5Income 22 — — — 12 12 Capitalcontributions 11 127 27,664 — — 27,791 Result onconversion ofloan 14 — — 3,005 — 3,005 Share-basedpaymentexpenses 12 — — 2,023 — 2,023 Balance atDecember 31,2013 610 142,459 6,536 (144,041) 5,564 Table of ContentsUNIQURE N.V.Consolidated Statement of Cash Flows(€ in thousands) The notes are an integral part of these consolidated financial statements. YEARS ENDED DECEMBER 31, NOTE 2011 2012 2013 Cash flow from operating activities Result before corporate income tax (17,300) (14,716) (26,820)Adjustments for: —Depreciation 6 590 548 535 —Impairment of assets 5 300 — — —Lease Incentive 28 — — 134 —Derivative result 14 (207) (22) 2,113 —Derivative result arising on early conversion of a loan 14 — 464 1,333 —Exchange result 26 45 49 —Share-based payment expenses 12 940 1,767 2,023 —Changes in other non-current assets 7 — — (923)—Changes in trade and other receivables (442) 243 (1,439)—Movement in inventories 9 — — (865)—Changes in trade and other payables (1,039) 180 359 —Changes in deferred revenue and provisions 17 — — 16,958 —Movement in other liabilities 64 161 2,052 —Interest (income)/expense 365 61 1,244 Cash used in operations (16,703) (11,269) (3,247)Interest paid (2) (8) (889) Net cash used in operating activities (16,705) (11,277) (4,136) Cash flow from investing activities Purchases of property, plant and equipment 6 (200) (392) (1,336)Purchases of intangible assets 5 (109) (553) (4,652)Interest received 147 113 17 Net cash used in investing activities (162) (832) (5,971) Cash flow from financing activities Capital contribution from shareholders 11 108 9,774 14,294 Convertible loans drawn down 11,14 — 1,498 11,999 Proceeds from borrowings 16 — — 7,492 Redemption of financial lease 13 — — (143) Net cash generated from financing activities 108 11,272 33,642 Net increase in cash, cash equivalents, and other bank overdrafts (16,759) (837) 23,535 Currency effect cash and cash equivalents 22 — — 12 Cash, cash equivalents, and other bank overdrafts at beginning of theperiod 17,859 1,100 263 Cash, cash equivalents, and other bank overdrafts cash at end of theperiod 10 1,100 263 23,810 The notes are an integral part of these consolidated financial statements.F-6Table of ContentsUNIQURE N.V.NOTES TO CONSOLIDATED FINANCIAL STATEMENTSFOR THE YEARS ENDED DECEMBER 31, 2011, 2012 AND 20131. General informationuniQure N.V. uniQure N.V. ("uniQure" or the "Company") is a biopharmaceutical company, incorporated and domiciled in the Netherlands, with itsheadquarters at Meibergdreef 61, 1105 BA, Amsterdam. The Company is a leader in the field of gene therapy, with the first product to receiveregulatory approval in the European Union and with multiple collaborations designed to accelerate the development of a pipeline of additional productcandidates. The Company was incorporated in January 2012 to acquire and continue the gene therapy business ("AMT Business") of AmsterdamMolecular Therapeutics (AMT) Holding N.V. ("AMT") and its subsidiaries (collectively, the "AMT Group") and to facilitate additional financing, asdescribed further below. As used in these financial statements, unless context indicates otherwise, all references to "uniQure" or the "Company" refer touniQure and its consolidated subsidiaries.Formation of uniQure and combination with the AMT Business on April 5, 2012 On February 17, 2012, AMT announced that it had entered into a conditional agreement with the newly created entity, uniQure, under which AMTagreed to transfer its entire interest in the AMT Business. uniQure was a newly formed company that issued equity shares to the existing shareholdersof AMT in exchange for the transfer of the AMT Business, such that there was no change in the substance of the reporting entity. The proposed transaction between uniQure and AMT was approved at a meeting of AMT shareholders on March 30, 2012 and completed onApril 5, 2012. On April 5, 2012, uniQure raised €6.0 million through an issue to Forbion of 1,954,395 newly-issued class A ordinary shares at a price of €3.07per share.uniQure capital structure following the transactions on April 5, 2012 Following the transaction with AMT and the financing by Forbion, uniQure had a single class of shares. All shares were ordinary shares with thesame economic rights in respect of dividends and upon a winding up or sale of the business. The ordinary shares were sub-divided into class Aordinary shares and class B ordinary shares. An additional classification of uniQure class C ordinary shares with a nominal value of five euro cent("class C ordinary shares") was created on July 22, 2013. While the A, B and class C ordinary shares all had the same economic rights, the principaldifference was that class A ordinary shares and class C ordinary shares were held directly by shareholders, whereas the class B ordinary shares wereheld by a trust foundation (stichting administratiekantoor (the "STAK")) on behalf of the uniQure DR holders; the STAK Trustees attend uniQureshareholder meetings on behalf of the uniQure DR holders and will follow voting instructions from the uniQure DR holders in respect of anyresolutions at shareholder meetings. These consolidated financial statements of the Company are prepared on a going concern basis taking into account the successful completion of itsinitial public offering on February 5, 2014 generating net proceeds of €62.6 million after commissions but before expenses. On February 10, 2014, the Company converted from a private company with limited liability (besloten vennootschap met beperkteaansprakelijkheid) incorporated under the laws of the Netherlands into a public company with limited liability (naamloze vennootschap), and changed itslegalF-7Table of Contentsname from uniQure B.V. to uniQure N.V., and reclassified its class A, B and C ordinary shares as ordinary shares.Significant shareholders The Company's significant shareholders* as at December 31, 2013 were:Advent Venture PartnersColler CapitalChiesi Farmaceutici S.p.AForbion Capital PartnersGilde Healthcare PartnersGrupo Netco and affiliatesLupus Alpha PE ChampionsOmnes Capital (formerly Credit Agricole Private Equity)*Following the IPO which took place on February 5, 2014, uniQure no longer regards these investors as significant shareholders holding morethan 5% of the Company's shares.Organizational structure of the uniQure Group uniQure N.V. is the ultimate parent of the following entities which were transferred to uniQure's ownership as part of the transaction with AMT(as described above) and which were renamed following the transaction, as follows:Other matters The Company's business is not subject seasonal influences.Company name Formerly known asuniQure biopharma B.V. Amsterdam Molecular Therapeutics (AMT) B.V.uniQure IP B.V. Amsterdam Molecular Therapeutics (AMT) IP B.V.uniQure Manufacturing B.V. AMT manufacturing B.V.uniQure Assay Development B.V. AMT Assay Development B.V.uniQure Research B.V. AMT Research B.V.uniQure non clinical B.V. AMT non clinical B.V.uniQure QA B.V. AMT QA B.V.uniQure Process Development B.V. AMT Process Development B.V.uniQure clinical B.V. AMT clinical B.V.Stichting participatie AMT(1) Stichting participatie AMT(1)uniQure Inc.(2) (1)Stichting participatie AMT is a Trust, not a company, but met the conditions for consolidation within uniQure's consolidated financialstatements. Stichting participatie AMT was established to facilitate AMT's employee incentive schemes for the period up to 2010. (2)In May 2013 the Company incorporated uniQure Inc., a Delaware corporation and wholly owned subsidiary of uniQure biopharma B.V.F-8Table of Contents On January 20, 2014, the shareholders of the Company approved, and on January 21, 2014 the supervisory board of the Company confirmed, a 5-for-1 consolidation of shares, which had the effect of a reverse share split, that became effective on January 31, 2014. All share, per-share and relatedinformation, for 2011, 2012 and 2013, presented in these consolidated financial statements and accompanying footnotes has been retroactively adjusted,where applicable, to reflect the impact of the reverse share split. At the time of the initial public offering all existing shareholders agreed to a 180 day lock-up that will expire on August 4, 2014. The consolidated financial statements were authorized for issue by the supervisory board on April 14, 2014.2. Summary of Significant Accounting PoliciesIntroductory notes on the basis of preparation and presentation of the financial statements As described in Note 1 above, the combination of uniQure and the AMT Business in 2012 was accounted for as a reverse acquisition underIFRS 3. Accordingly, uniQure's consolidated financial statements consolidate the financial results of the uniQure Group for the 12 months endedDecember 31, 2012 (including the results of AMT prior to its acquisition by uniQure) and for the 12 months ended December 31, 2013. The principal accounting policies applied in the preparation of these consolidated financial statements are set out below. These policies have beenconsistently applied to all the years presented, unless otherwise stated.2.1Basis of Preparation The consolidated financial statements of uniQure have been prepared in accordance with International Financial Reporting Standards ("IFRS") asissued by the International Accounting Standards Board. The consolidated financial statements have been prepared under the historical cost convention, except for any derivative instruments, which arerecorded at fair value through profit or loss. The consolidated financial statements are presented in the company's functional currency Euro, except where otherwise indicated. The preparation of financial statements in conformity with IFRS requires the use of certain critical accounting estimates. It also requiresmanagement to exercise its judgment in the process of applying uniQure's accounting policies. The areas involving a higher degree of judgment orcomplexity or areas where assumptions and estimates are significant to the financial statements are disclosed in Note 4. Under IFRS 3, the acquisition of the AMT Business by uniQure from AMT, which was completed on April 5, 2012, is accounted for as a reverseacquisition; therefore, the financial information is presented on a continuing basis for the AMT Business and uniQure. Under IFRS 3 uniQure is thelegal parent of the AMT Business but is regarded as the accounting acquiree; conversely the AMT Group is the legal subsidiary but the accountingacquirer in the consolidated financial statements.F-9Table of Contents2.2Changes in accounting policy and disclosures (a)New and amended standards adopted by the Company The following standards and amendments to standards became effective for annual periods on January 1, 2013 and have been adopted by theCompany in the preparation of the consolidated financial statements:•Amendment to IFRS 7 Financial instruments—disclosures •IFRS 10 Consolidated financial statements •IFRS 11 Joint arrangements •IFRS 12 Disclosures of interest in other entities •IFRS 13 Fair value measurement •Amendment to IAS 1 Presentation of financial statements •Improvements to IAS 16 Property plant and equipment •Amendment to IAS 19 Employee benefits •IAS 27 (revised 2011) Separate financial statements •IAS 28 (revised 2011) Investments in associates and joint ventures •Improvements to IAS 32 Financial statements—presentation •Improvements to IAS 34 Interim financial reporting •IFRIC 21 Levies(1)The adoption of these new standards and amendments did not materially impact the Company's financial position or results of operations.(b)New and amended standards not yet adopted by the Company A number of new standards and amendments to standards and interpretations are effective for annual periods beginning after January 1, 2014 andhave not been applied in preparing these consolidated financial statements. None of these are expected to have a material effect on the consolidatedfinancial statements of the Company. The standards which could have a significant effect on the consolidated financial statements of the Company are IFRS 9 "Financial Instruments"and Amendments to IAS 36 "Impairment of Assets". IFRS 9 is the first step in the process of replacing IAS 39 "Financial Instruments: Recognitionand Measurement". The Company has yet to assess IFRS 9's full impact. Amendments to IAS 36 removes certain disclosures of the recoverableamount of Cash Generating Units which had been included in IAS 36 by the issue of IFRS 13. The Company has yet to assess Amendments toIAS 36's full impact. The IASB has also issued Exposure Drafts in which significant changes on accounting and disclosures are proposed on topics such as leaseaccounting and revenue recognition. If the current proposals lead to new or amended standards, the changes could have a substantial impact onuniQure's financial statements in the coming years. The effective date of the revised standards is still under discussion. (1)Applicable for accounting periods beginning on or after January 1, 2014, however uniQure has adopted this standard early.F-10Table of Contents2.3Consolidation Subsidiaries are all entities over which the Company has control. The Company controls an entity when the Company is exposed to, or has rightsto, variable returns from its involvement with the entity and has the ability to affect those returns through its power over the entity. Subsidiaries are fullyconsolidated from the date on which control is transferred to the Company. They are de-consolidated from the date that control ceases. Inter-company transactions, balances, income and expenses on transactions between uniQure companies are eliminated. Profits and losses resultingfrom inter-company transactions that are recognized in assets are also eliminated. Accounting policies of subsidiaries have been changed wherenecessary to ensure consistency with the policies adopted by the Company.2.4Segment Reporting Operating segments are identified on the basis of whether the allocation of resources and/or the assessment of performance of a particularcomponent of uniQure's activities are regularly reviewed by uniQure's chief operating decision maker as a separate operating segment. By these criteria,the activities of uniQure are considered to be one segment, which comprises the discovery, development and commercialization of innovative genetherapies and the segmental analysis is the same as the analysis for uniQure as a whole. The Management Board is identified as the chief operatingdecision maker, and reviews the consolidated operating results regularly to make decisions about the resources and to assess overall performance. The Company currently, and in the near future, is expected to derive the substantial majority of its revenues from a single party, Chiesi, based inItaly. The Company and Chiesi have entered into an exclusive collaboration for the development and commercialization of the Company's Glybera andHemophilia B programs in Europe and certain additional territories, pursuant to agreements which were entered into in April 2013, and which becameeffective in June 2013.2.5Foreign Currency Translation (a)Functional and Presentation Currency Items included in the financial statements of each of uniQure's entities are measured using the currency of the primary economic environment inwhich the entity operates ("the functional currency") which historically in all cases has been Euro. The consolidated financial statements are presented inEuro, which is the Company's functional and presentation currency.(b)Transactions and Balances Foreign currency transactions are translated into the functional currency using the exchange rates prevailing at the dates of the transactions. Foreignexchange gains and losses resulting from the settlement of such transactions and from the translation at year-end exchange rates of monetary assets andliabilities denominated in foreign currencies are recognized in the income statement. Foreign exchange gains and losses that relate to borrowings and cash and cash equivalents are presented within 'Finance income' or 'Financeexpenses' while all other foreign exchange gains and losses are presented within 'Other losses—net' on the Consolidated Statement of ComprehensiveIncome.2.6Notes to the cash flow statement The cash flow statement has been prepared using the indirect method. The cash disclosed in the cash flow statement is comprised of cash and cashequivalents. Cash comprises cash on hand and demand deposits. Cash equivalents are short- term, highly liquid investments that are readily convertibleto known amounts of cash and are subject to an insignificant risk of changes in value. CashF-11Table of Contentsflows denominated in foreign currencies have been translated at the average exchange rates. Exchange differences, if any, affecting cash items areshown separately in the cash flow statement. Interest paid and received, dividends received and income tax are included in the cash from operatingactivities. Further details are set out in Note 10 below.2.7Intangible Assets (a)Licenses Acquired patents have a definite useful life and are carried at cost less accumulated amortization and impairment losses. Amortization is calculatedusing the straight-line method to allocate the cost of licenses over their estimated useful lives (generally 20 years unless a license expires prior to thatdate). Amortization begins when an asset is available for use.(b)Research and Development Research expenditures are recognized as expenses as incurred. Costs incurred on development projects are recognized as intangible assets as of thedate that it can be established that it is probable that future economic benefits that are attributable to the asset will flow to the Company considering itscommercial and technological feasibility, generally when a filing is made for regulatory approval for commercial production, and when costs can bemeasured reliably.2.8Property, Plant and Equipment Property, plant and equipment comprise mainly laboratory equipment, leasehold improvements, furniture and computer hardware/software. Allproperty, plant and equipment are stated at historical cost less depreciation. Historical cost includes expenditures that are directly attributable to theacquisition of the items. Subsequent costs are included in the asset's carrying amount or recognized as a separate asset, as appropriate, only when it is probable that futureeconomic benefits associated with the item will flow to uniQure and the cost of the item can be measured reliably. All other repairs and maintenancecharges are expensed in the period in which such charges are incurred. Depreciation is calculated using the straight-line method to allocate the cost of the assets to their residual values over their estimated useful lives.Property, plant and equipment are depreciated as follows:•Leasehold improvements periods between 5 - 15 years •Laboratory equipment periods between 5 - 10 years •Computer hardware/software 3 years The assets' residual values and useful lives are reviewed, and adjusted if appropriate, at each balance sheet date. An asset's carrying amount iswritten down immediately to its recoverable amount if the asset's carrying amount is greater than its estimated recoverable amount. Gains and losses on disposals are determined by comparing proceeds with the carrying amount and are recognized in the income statement. Operating leases and financial leases are described further in Note 2.23 below.2.9Impairment of Non-Financial Assets Assets that are not subject to amortization (whether or not they are ready for use) are tested annually for impairment. Assets that are subject toamortization are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount may not be recoverable. Forthe purpose of assessing impairment, assets are grouped at the lowest levels for which there are separatelyF-12Table of Contentsidentifiable cash flows (i.e. cash-generating units). For the purpose of the impairment review the Company determined the entire uniQure group isconsidered one cash generating unit, as we currently use all material assets in the development of our gene therapies and our management regularlyreviews all activities of our group as a single component. The impairment review methodology applied is based on the fair value less cost of disposal concept. In this concept we compare the enterprisevalue (calculated by multiplying the outstanding shares as per the valuation date by the fair value of a ordinary share) with the book value of the cash-generating unit. An impairment loss is recognized for the amount by which the asset's carrying amount exceeds its recoverable amount. The recoverableamount is the higher of an asset's fair value less costs to sell and value in use. Non-financial assets that have been previously impaired are reviewed forpossible reversal of the impairment at each subsequent reporting date.2.10Recognition and measurement Financial assets and financial liabilities are included in uniQure's balance sheet when uniQure becomes a party to the contractual provisions of theinstrument. Financial assets are derecognized when the rights to receive cash flows from the investments have expired or have been transferred and thecompany has transferred substantially all risks and rewards of ownership.Non-derivative financial instrumentsCash and cash equivalents Cash and cash equivalents includes bank balances, demand deposits and other short-term, highly liquid investments (with less than three months tomaturity) that are readily convertible into a known amount of cash and are subject to an insignificant risk of fluctuations in value.Trade Receivables Trade receivables are amounts due from customers for license fee payments or services performed in the ordinary course of business. If collectionis expected in one year or less (or in the normal operating cycle of the business if longer), they are classified as current assets. Trade receivables arerecognized initially at fair value and subsequently measured at amortized cost using the effective interest method, less provision for impairment, if any.Financial liabilities and equity Financial liabilities and equity instruments issued by uniQure are classified according to the substance of the contractual arrangements entered intoand the definitions of a financial liability and an equity instrument. An equity instrument is any contract that evidences a residual interest in the assets ofuniQure after deducting all its liabilities. The accounting policies adopted for specific financial liabilities and equity instruments are set out below.Trade payables Trade payables are obligations to pay for goods or services that have been acquired in the ordinary course of business from suppliers. Tradepayables are classified as current liabilities if payment is due within one year or less (or in the normal operating cycle of the business if longer). If not,they are presented as non-current liabilities. Trade payables are initially measured at fair value, and are subsequently measured at amortized cost, using the effective interest rate method.F-13Table of ContentsEquity instruments Equity instruments issued by uniQure are recorded at the proceeds received. Direct issuance costs are processed as a deduction on equity.Derivative financial instruments uniQure does not have a policy of engaging in speculative transactions, nor does it issue or hold financial instruments for trading purposes. uniQure has entered into various financing arrangements with its investors, including convertible loans. These convertible loans each includeembedded financial derivative elements (being the right to acquire equity in the Company at a future date for a pre-determined price). Therefore, whileuniQure does not engage in speculative trading of derivative financial instruments, it may hold such instruments from time to time as part of itsfinancing arrangements. Derivatives are initially recognized at fair value on the date a derivative contract is entered into and are subsequently re-measured at their fair value.The resulting gain or loss is recognized in the consolidated income statement, as the Company currently does not apply hedge accounting.2.11Offsetting financial instruments Financial assets and liabilities are offset and the net amount is reported in the balance sheet when there is a legally enforceable right to offset therecognized amounts and there is an intention to settle on a net basis, or realize the asset and settle the liability simultaneously.2.12Inventories Inventories are stated at the lower of cost and net realizable value. Cost is determined using the first-in, first-out (FIFO) method. The cost offinished goods and work in progress comprises design costs, raw materials, direct labor, other direct costs and related production overheads (based onnormal operating capacity). It excludes borrowing costs. Net realizable value is the estimated selling price in the ordinary course of business, lessapplicable variable selling expenses.2.13Equity The Company classifies an instrument, or its component parts, on initial recognition as a financial liability or an equity instrument in accordancewith the substance of the contractual arrangement and the definitions of a financial liability and an equity instrument. An instrument is classified as a financial liability when it is either (i) a contractual obligation to deliver cash or another financial asset to anotherentity; or (ii) a contract that will or may be settled in the Company's own equity instruments and is a non-derivative for which the Company is or may beobliged to deliver a variable number of the Company's own equity instruments or a derivative that will or may be settled other than by the exchange of afixed amount of cash or another financial asset for a fixed number of the Company's own equity instruments. An equity instrument is defined as any contract that evidences a residual interest in the assets of an entity after deducting all of its liabilities. Aninstrument is an equity instrument only if the issuer has an unconditional right to avoid settlement in cash or another financial asset.Ordinary Shares Ordinary shares are classified as equity. Incremental costs directly attributable to the issue of new shares or options are shown in equity as adeduction from the proceeds, net of tax.F-14Table of ContentsConvertible Loan Where the Company issues convertible loans that do not have the unconditional right to avoid delivering cash or a variable number of shares tosettle obligations towards loan note holders, the Company accounts for such loan notes as containing an element that qualifies as a financial liability.Convertible loans are split into a debt component and a separate conversion option component. The conversion option is recognized initially at fairvalue, based on a probability-weighted scenario analysis. The debt component is the residual amount after deducting from the fair value of the loan as awhole (i.e. the issuance proceeds) the amount separately determined for the conversion option component. The debt component is subsequently carriedat amortized cost using the effective interest rate method. When estimates regarding the amount or timing of payments required to settle the obligationchange, the carrying amount of the financial liability is adjusted to reflect actual and revised estimated cash flows. The carrying amount is recalculated bycomputing the present value of estimated future cash flows at the financial instrument's original effective interest rate. Such adjustments are recognizedas income or expense in the income statement. Any incremental costs of the loan are deducted from the carrying amount and are amortized over the termof the convertible loan under the effective interest rate method. The conversion option is classified as a liability if it may be settled by either party other than by the exchange of a fixed amount of cash for a fixednumber of the entity's own equity instruments. In that case, the conversion option is carried at fair value with changes in fair value recorded in theincome statement. If the conversion option qualifies as an equity instrument, it is recognized in equity on issue date and not re-measured.2.14Borrowings Borrowings are recognized initially at fair value, net of transaction costs incurred. Borrowings are subsequently carried at amortized cost. Anydifference between the proceeds (net of transaction costs) and the redemption value is recognized in the income statement over the period of theborrowings using the effective interest rate method.2.15Deferred Corporate Income Taxes There is no tax charge in the Company's Consolidated Statements of Comprehensive Income, nor any deferred tax recognized in the balance sheetfor the periods covered by these financial statements. To the extent that any tax expense would arise, it would comprise current and deferred tax. Tax effects are recognized in the income statement,except to the extent that they relate to items recognized in other comprehensive income or directly in equity. In this case, the tax is also recognized inother comprehensive income or directly in equity, respectively. The Company's management periodically evaluates positions taken in tax returns with respect to situations in which applicable tax regulation issubject to interpretation. It establishes provisions where appropriate on the basis of amounts expected to be paid to the tax authorities. Deferred income tax is recognized, using the liability method, on temporary differences arising between the tax basis of assets and liabilities andtheir carrying amounts in the consolidated financial statements. However, the deferred income tax is not accounted for if it arises from initial recognitionof an asset or liability in a transaction other than a reorganization that at the time of the transaction affects neither accounting nor taxable profit and loss.Deferred income tax is determined using tax rates (and laws) that have been enacted or substantially enacted by the balance sheet date and are expectedto apply when the related deferred income tax asset is realized or the deferred income tax liability is settled. Deferred income tax assets are recognized only to the extent that it is probable that future taxable profit will be available against which thetemporary differences can be utilized.F-15Table of Contents Deferred income tax is provided on temporary differences arising on investments in subsidiaries and associates, except where the timing of thereversal of the temporary difference is controlled by the Company and it is probable that the temporary difference will not reverse in the foreseeablefuture. Deferred income tax assets and liabilities are offset when there is a legally enforceable right to offset current tax assets against current tax liabilitiesand when the deferred income taxes assets and liabilities relate to income taxes levied by the same taxation authority on either the taxable entity ordifferent taxable entities where there is an intention to settle the balances on a net basis.2.16Employee Benefits (a)Pension Obligations uniQure operates a defined contribution pension plan for all employees at its Amsterdam facility in the Netherlands, which is funded by theCompany through payments to an insurance company. uniQure has no legal or constructive obligation to pay further contributions if the plan does nothold sufficient assets to pay all employees the benefits relating to employee service in the current and prior periods. The contributions are recognized asemployee benefit expense when they are due. Prepaid contributions are recognized as an asset to the extent that a cash refund or a reduction in the futurepayments is available.(b)Termination benefits Termination benefits are payable when employment is terminated by the Company before the normal retirement date, or whenever an employeeaccepts voluntary redundancy in exchange for these benefits. The Company recognizes termination benefits when it is demonstrably committed to either:terminating the employment of current employees according to a detailed formal plan without possibility of withdrawal; or providing terminationbenefits as a result of an offer made to encourage voluntary redundancy. Benefits falling due more than 12 months after the end of the reporting periodare discounted to their present value.(c)Bonus plans The Company recognizes a liability and an expense for bonus plans if contractually obligated or if there is a past practice that has created aconstructive obligation.2.17Share-Based CompensationuniQure 2012 share option plan The Company operates a share-based payment plan, which is an equity settled share option plan under which options have been granted in 2012and 2013. The fair value of the options in exchange for the services received is recognized as an expense. The total amount to be expensed over the vestingperiod, if any, is determined by reference to the fair value of the options granted. For the equity-settled option plan, the fair value is determined at thegrant date. For share-based payments that do not vest until the employees have completed a specified period of service, uniQure recognizes the servicesreceived as the employees render service during that period. For the allocation of the expenses to be recognized, the Company treats each installment ofa graded vesting award as a separate share option grant. The share options' vesting periods are as follows: 33.33% vests after one year from the initialvesting date and the remaining 66.66% vest daily on a straight-line pro rata basis over years two and three. At each balance sheet date, the Company revises its estimates of the number of options that are expected to become exercisable. It recognizes theimpact of the revision of original estimates, if any, in the income statement and a corresponding adjustment to equity.F-16Table of Contents2.18Provisions Provisions are recognized when uniQure has a present legal or constructive obligation as a result of past events, it is probable that an outflow ofresources will be required to settle the obligation, and the amount can been reliably estimated. Provisions are measured at the present value of the expenditures expected to be required to settle the obligation using a pre-tax rate that reflectscurrent market assessments of the time value of money and the risks specific to the obligation. The increase in the provision due to passage of time isrecognised as interest expense.2.19Revenues Revenues comprise the fair value of the consideration received or receivable for the sale of licenses and services in the ordinary course of theCompany's activities. Revenues are shown net of value-added tax, returns, rebates and discounts and after eliminating sales within the group.License revenues License revenues consist of upfront payments and milestone payments.(a)Upfront payments Revenues from non-refundable, up-front payments are initially reported as deferred revenue on the consolidated balance sheet and are recognizedin the income statement as revenue over the period of the development, commercialization, collaboration or the manufacturing obligation.(b)Milestone payments Sales related milestone payments will be recognized in full in the period in which the relevant milestone is achieved.Collaboration revenues Collaboration revenues consist of revenues generated from collaborative research and development arrangements. Such agreements may consist ofmultiple elements and provide for varying consideration terms, such as up-front, milestone and similar payments which require significant analysis inorder to determine the appropriate method of revenue recognition. Where such arrangements can be divided into separate units of accounting (each unit constituting a separate earnings process), the arrangementconsideration is allocated to the different units based on their relative fair values and recognized over the respective performance period. Where thearrangement cannot be divided into separate units, the individual deliverables are combined as a single unit of accounting and the total arrangementconsideration is recognized over the estimated collaboration period.2.20Other income uniQure's other income comprises certain subsidies that support uniQure's research efforts in defined research and development projects. Thesesubsidies generally provide for reimbursement of approved costs incurred as defined in various grants.2.21Government grants The Company receives certain government and regional grants that support its research effort in defined projects. These grants generally providefor reimbursement of approved costs incurred as defined in the respective grants. Income in respect of grants includes contributions towards the costs ofresearch and development. Income is recognized when costs under each grant are incurred inF-17Table of Contentsaccordance with the terms and conditions of the grant and the collectability of the receivable is reasonably assured. Government and regional grants relating to costs are deferred and recognized in the income statement over the period necessary to match them withthe costs they are intended to compensate. When the cash in relation to recognized government or regional grants is not yet received the amount isincluded as a receivable on the balance sheet. Where the grant income is directly related to the specific items of expenditure incurred, the income will be netted against such expenditure. Wherethe grant income is not a specific reimbursement of expenditure incurred, the Company includes such income under 'Other income' in the incomestatement. Grants or investment credits may be repayable if uniQure successfully commercializes a relevant program that was funded in whole or in part bythe grant or investment credit within a particular timeframe. Prior to successful commercialization, uniQure does not make any provision for repayment.2.22Recognition of research and development expenses Research expenditures are recognized as expenses when incurred except when certain criteria for capitalization as intangible assets are met(Note 2.7). At each balance sheet date, the Company estimates the level of service performed by the vendors and the associated cost incurred for theservices performed.2.23LeasesOperating leases Leases in which a significant portion of the risks and rewards of ownership are retained by the lessor are accounted for as operating leases.Payments made under operating leases (net of any incentives received from the lessor) are charged to the income statement on a straight-line basis overthe period of the lease.Finance leases The Company leases certain laboratory equipment and office equipment. Leases for leasehold improvements and equipment where the Companybears substantially all the risks and rewards of ownership are accounted for as finance leases. Finance leases are capitalized at the lease's commencementat the lower of the fair value of the leased property or the present value of the minimum lease payments. Each finance lease payment is allocated between the liability and finance charges in order to achieve a constant rate on the finance balanceoutstanding. The finance balances, net of finance charges, are included in other long- term payables. The interest element of the finance cost is chargedto the income statement over the lease period to produce a constant periodic rate of interest on the remaining balance of the liability for each period. Thelaboratory and office equipment acquired under finance leases are depreciated over the shorter of the useful life of the asset or the lease term.2.24Dividend Distributions Dividend distributions to the Company's shareholders are recognized as a liability in uniQure's financial statements in the period in which thedividends are approved by the Company's shareholders. To date uniQure has not, and AMT did not, pay dividends.F-18Table of Contents3. Financial Risk Management3.1Financial Risk Factors uniQure's activities have exposed it to a variety of financial risks: market risk (including currency risk, price risk, and cash flow and fair valueinterest rate risk), credit risk, and liquidity risk. uniQure's overall risk management program is focused on preservation of capital and the unpredictabilityof financial markets and has sought to minimize potential adverse effects on uniQure's financial performance and position. Risk management is carried out by the finance department. The finance department identifies and evaluates financial risks and hedges these risks ifdeemed appropriate. Since December 31, 2012 the Company has continued to strengthen the finance department which is responsible for financial riskmanagement, through the appointment of additional senior personnel. There have been no changes in the Company's financial risk management policies,since December 31, 2012.(a)Market Risk (i)Currency risk uniQure operates within the Euro area and also internationally and is exposed to foreign exchange risk arising from various currency exposures,primarily with respect to the U.S. dollar and, to a lesser extent, the British pound as the Company acquires certain materials and pays for certain licensesand other services in these two currencies. At December 31, 2013 there was a net amount of trade payables in U.S. Dollars of €0.4 million (2012: €0.0 million) and a net trade payable inBritish Pounds of €0.1 million (2012: €0.2 million). Foreign currency denominated trade receivables and trade payables are short term in nature(generally 30 to 45 days). As a result foreign exchange rate movements on trade receivables and trade payables, during the years presented had animmaterial effect on the financial statements. In the absence of significant foreign exchange exposure, management has not set up a policy to manage the foreign exchange risk against thefunctional currency. As of December 31, 2013 and December 31, 2012, there would not have been a significant effect on the Company's loss due to strengthening orweakening of the functional currency against any foreign currency.(ii)Price risk The market prices for the provision of preclinical and clinical materials and services, as well as external contracted research may vary over time.The commercial prices of any of the Company's products or product candidates are currently uncertain. The Company is not exposed to commodityprice risk. uniQure does not hold investments classified as available-for-sale or at fair value through profit or loss; therefore uniQure is not exposed to equitysecurities price risk.(iii)Cash flow and fair value interest rate risk The Company's interest rate risk arises from short and long-term borrowings. The Company has no borrowings with variable rates and is notexposed to cash flow interest rate risk. Borrowings issued at fixed rates expose the Company to fair value interest rate risk. During 2012 the Company'sborrowings were wholly denominated in Euro; in July 2013 the Company entered into an agreement with Hercules Technology Growth Capital for a$10 million denominated loan. At December 31, 2013 if interest rates on borrowings had been 1.0% higher/lower with all other variables held constant, post-tax results for theyear would have been €42,000 (2012: nil) lower/higher as a result of changes in the fair value of the borrowings. The effect of a change in interest ratesofF-19Table of Contents1.0% on borrowings would have had an insignificant effect on post-tax results for the year as a result of changes in the fair value of the venture debtfacility. During 2013 uniQure had long-term interest bearing liabilities under the 2012 Convertible loan which was subsequently converted into 1,336,331Class A ordinary shares on July 26, 2013. uniQure does not enter into any interest rate swaps.(b)Credit Risk Credit risk is managed on group basis. Credit risk arises from cash and cash equivalents, derivative financial instruments and deposits with banksand financial institutions, as well as credit exposures to wholesale and retail customers, including outstanding receivables and committed transactions. The Company has currently no wholesale debtors other than Chiesi. Please refer to Note 17 for further information on the Company's relationshipwith Chiesi. The security deposit under other non-current assets represents the amount the Company paid to the landlord in September 2013 in relation to thefacility in Lexington, Massachusetts. The deposit is neither impaired nor past due. For banks and financial institutions, only independently rated parties with a minimum rating of 'A' are accepted. As of December 31, 2013 and December 31, 2012, the majority of uniQure's cash and cash equivalents were placed at the following banks: There are no financial assets past due date or impaired. No credit limits were exceeded during the reporting period.(c)Liquidity Risk Management considers uniQure's cash and cash equivalents as of December 31, 2013, when taken together with additional funds raised since thatdate, are sufficient to carry out the business plans going forward, at least until 12 months from the date of these financial statements. Prudent liquidityrisk management implies maintaining sufficient cash, and planning to raise cash if and when needed, either through issue of shares or through creditfacilities. Management monitors rolling forecasts of uniQure's liquidity reserve on the basis of expected cash flow. The table below analyzes the Company's financial liabilities in relevant maturity groupings based on the length of time until the contractual maturitydate, as at the balance sheet date. The amountsF-20 AS OF DECEMBER 31, 2012 2013(€ in thousands) AMOUNT CREDIT RATING AMOUNT CREDIT RATINGBank Rabo Bank(1) 258 AA2 23,810 AA2Van Lanschot(2) 5 A- — A- Total 263 23,810 (1)Rating is by Moody's (2)Rating is by Fitch RatingsTable of Contentsdisclosed in the table are the contractual undiscounted cash flows. Balances due within 12 months equal their carrying value balances as the impact ofdiscounting is not significant.3.2Capital Risk Management The Company's objectives when managing capital are to safeguard the Company's ability to continue as a going concern in order to provide returnsfor shareholders, benefits for other stakeholders and to maintain an optimal capital structure to reduce the cost of capital. In order to maintain or adjust the capital structure, the Company may adjust the amount of dividends paid to shareholders (although at this time theCompany does not have retained earnings and is therefore currently unable to pay dividends), return capital to shareholders, issue new shares or sellassets to reduce debt. The total amount of equity as recorded on the balance sheet is managed as capital by the Company.3.3Fair value estimation For financial instruments that are measured on the balance sheet at fair value, IFRS 7 requires disclosure of fair value measurements by level of thefollowing fair value measurement hierarchy:•Quoted prices (unadjusted) in active markets for identical assets or liabilities (level 1); •Inputs other than quoted prices included within level 1 that are observable for the asset or liability, either directly (that is, as prices) orindirectly (that is, derived from prices) (level 2); and •Inputs for the asset or liability that are not based on observable market data (that is, unobservable inputs) (level 3).As of December 31, 2013, 2012 and 2011 financial instruments at fair value through profit and loss amounted to €3,446,000, €464,000, and nil,respectively, and comprised of movements on the fair value of the derivative elements of convertible loans, as described further in Note 14 below.The fair value of financial instruments that are not traded in an active market (for example, over-the-counter derivatives) is determined by usingvaluation techniques. These valuation techniques maximize the use of observable market data where it is available and rely as little as possible on entityspecific estimates. If all significant inputs required to ascertain the fair value of an instrument are observable, the instrument is included in level 2.F-21 LESS THAN1 YEAR BETWEEN 1AND 2 YEARS BETWEEN 2AND 5 YEARS OVER 5YEARS (€ in thousands) At December 31, 2012 Borrowings (excl. finance lease liabilities) — — — — Financial lease liabilities 151 450 — — Debt to related party 1,618 — — — Trade and other payables 3,916 — — — Total 5,685 450 — — At December 31, 2013 Borrowings (excl. finance lease liabilities) 633 2,722 3,911 — Financial lease liabilities 156 168 134 — Debt to related party — — — — Trade and other payables 7,445 — — — Total 8,234 2,890 4,045 Table of Contents If one or more of the significant inputs is not based on observable market data, the instrument is included in level 3. The carrying amount of a financial asset or financial liability is a reasonable approximation of the fair value and therefore information about the fairvalues of each class has not been disclosed. Group valuation processes The fair value of the level 3 liabilities as of December 31, 2013 have been determined using a Black-Scholes option pricing model. Key inputsinclude the risk-free rate, volatility, term, exercise price, and fair value of ordinary shares. The values are included within the tables presented above.Changes in the fair values are analyzed at each reporting date during the quarterly review process.4. Critical Accounting Estimates and Judgments The preparation of financial statements in conformity with IFRS requires the Company to make estimates and assumptions that affect the reportedamounts and classifications of assets and liabilities, revenues and expenses in the consolidated financial statements. The estimates that have a significantrisk of causing a material adjustment to the financial statements are utilized for share-based compensation, income taxes, research and developmentexpenditures and borrowings. Actual results could differ materially from those estimates and assumptions. The preparation of financial statements in conformity with IFRS also requires the Company to exercise judgment in applying the accountingpolicies. Critical judgments in the application of the Company's accounting policies relate to research and development expenditures, revenues and thecost of license revenues. Estimates and judgments are continually evaluated and are based on historical experience and other factors, including expectations of future eventsthat are believed to be reasonable under the circumstances.F-22 LEVEL 1 LEVEL 2 LEVEL 3 TOTAL At December 31, 2012 Debt to related party—embedded derivative (warrants) — — 132 132 Borrowings—embedded derivative (warrants) — — — — — — 132 132 LEVEL 1 LEVEL 2 LEVEL 3 TOTAL At December 31, 2013 Debt to related party—embedded derivative (warrants) — — 722 722 Borrowings—embedded derivative (warrants) — — 217 217 — — 939 939 LEVEL 3 Opening Balance at January 1, 2013 132 Transfers to level 3 366 Movement in Equity on early conversion of the convertible loan (3,005)Losses recognized in Profit and Loss during the 12 months ended December 31 2013 3,446 Closing balance at December 31, 2013 939 Total losses for the period included in P&L for assets held at the end of the reporting period, under Financeexpenses 3,446 Table of Contents4.1Critical accounting estimates and assumptions The Company makes estimates and assumptions concerning the future. The resulting accounting estimates will, by definition, seldom equal therelated actual results. The estimates and assumptions that have a significant risk of causing a material adjustment to the carrying amounts of assets andliabilities within the next financial year are addressed below.Revenue recognition The Company has not generated any revenues from royalties or product sales through December 31, 2013. In July 2013, the Company received upfront payments in connection with the Glybera commercialization agreement and hemophilia B co-development agreements. Revenues from such non-refundable, up-front payments are initially reported as deferred revenues on the consolidated balancesheet and are recognized in revenues as earned over the period of the development, commercialization, collaboration or manufacturing obligation. The Company also generates revenues from collaborative research and development arrangements. Such agreements may consist of multipleelements and provide for varying consideration terms, such as up-front, milestone and similar payments, which require significant analysis bymanagement in order to determine the appropriate method of revenue recognition. Where such arrangements can be divided into separate units of accounting (each unit constituting a separate earnings process), the arrangementconsideration is allocated to the different units based on their relative fair values and recognized over the respective performance period. Where thearrangement cannot be divided into separate units, the individual deliverables are combined as a single unit of accounting and the total arrangementconsideration is recognized over the estimated collaboration period. Such analysis requires considerable estimates and judgments to be made by us,including the relative fair values of the various elements included in such agreements and the estimated length of the respective performance periods. Management has concluded that the up-front payments constitute a single unit of accounting, and accordingly, the up-front payments will berecognized over the estimated remaining period of the related manufacturing technologies.Valuation of Warrants With the venture debt loan facility and after the conversion of the convertible loan in 2013 the Company is accounting for the valuation of warrants(total warrants as per December 31, 2013: 170,802, with a corresponding carrying value of €939,000). The fair value of the warrants is based on theBlack-Scholes model. This model applies a number of parameters that range from observable inputs (Level 1) to un-observable inputs (Level 3). Assumptions are madeon inputs such as time to maturity, the fair value per ordinary share, volatility and risk free rate, in order to determine the fair value per warrant. Inaddition there is an assumption on foreign exchange to calculate the euro value of the Hercules warrants.F-23Table of Contents The effect, when some of these underlying parameters would deviate by 10% up or down is presented in the below table.Share-based payments In 2012 the Company introduced an equity settled share option plan. At the balance sheet date of December 31, 2013 a total of 1,691,844 optionswere granted and outstanding (2012: 1,606,347). This plan is accounted for in accordance with the policy as stated in Note 2.17. The option pricingmodel used and the inputs to that model are described in Note 12 below. For the periods ending December 31, 2011, 2012 and 2013 the recorded expenses for share based expenses were €940,000, €1,767,000 and€2,023,000 respectively. The Company assumes all granted options held at December 31, 2013 will be held until full vesting. At the date of the IPO atotal of 1,507,443 options vested in full.Corporate taxes The Company is subject to corporate taxes in the Netherlands. Significant judgment is required in determining the use of net operating loss carryforwards and taxation of upfront and milestone payments for corporate tax purposes. There are many transactions and calculations for which theultimate tax determination is uncertain. Where the final tax outcome of these matters is different from the amounts that were initially recorded, suchdifferences will impact the current and deferred corporate tax assets and liabilities in the period in which such determination is made.4.2Critical judgments in applying the entity's accounting policies (a)Corporate Income Taxes The Dutch corporate income tax act permits reporting pursuant to a consolidated tax regime, referred to as a fiscal unity. A fiscal unity is acombination of a parent and subsidiaries whereby formally the parent, in our case uniQure B.V., is the entity that is taxed for the consolidated profits ofthe fiscal unity. uniQure, which has a history of tax losses, recognizes deferred tax assets arising from unused tax losses or tax credits only to the extent that therelevant fiscal unity has sufficient taxable temporary differences or there is convincing other evidence that sufficient taxable profit will be availableagainst which the unused tax losses or unused tax credits can be utilized by the fiscal unity. Management's judgment is that sufficient convincing otherevidence is not available and a deferred tax asset is therefore not recognized.(b)Research and Development Expenditures The stage of a particular project generally forms the basis for the decision whether costs incurred for the Company's research and developmentprojects can be capitalized or not. In general, the Company's position is that clinical development expenditures are not capitalized until the Company filesfor regulatory approval in respect of the program, as this is considered to be the first point in time when it becomes probable that future revenues can begenerated. However, although the EMA has now granted marketing authorization under exceptional circumstances in the European Union for Glybera,such authorization is subject to further conditions before first sales may be made in the European Union.F-24 Fair value perOrdinary Share Volatility Time toMaturity -10% 792.000 827.000 901.000 Base Case 939.000 939.000 939.000 +10% 1.091.000 1.046.000 975.000 Table of Contents IAS38 describes the conditions under which development expenditure should be capitalized. These conditions include the availability of adequatetechnical, financial and other resources to complete the development of the intangible asset. On March 21, 2013, the Company secured a €10.0 millionconvertible loan, which provided resources to complete development of Glybera. Accordingly, from March 21, 2013, the Company has capitalizeddevelopment costs relating to Glybera. Following commercial launch of the product by uniQure's commercial partner, Chiesi, which is expected to occurmid 2014, the intangible asset will be carried at its cost less any accumulated amortization and any accumulated impairment losses (cost model). Theestimated useful life over which the intangible will be amortized is estimated at approximately 19 years. As of each balance sheet date, the Company estimates the level of service performed by its vendors or other counterparties and the associated costsincurred for the services performed. As part of the process of preparing the Company's financial statements the Company is required to estimate itsaccrued expenses. This process involves reviewing quotations and contracts, identifying services that have been performed on the Company's behalf,estimating the level of service performed and the associated cost incurred for the service when it has not yet been invoiced or otherwise notified of theactual cost. The majority of the Company's service providers invoice the Company monthly in arrears for services performed or when contractualmilestones are met. The Company makes estimates of its accrued expenses as of each balance sheet date in its financial statements based on facts andcircumstances known to it at that time. The Company periodically confirms the accuracy of its estimates with the service providers and makesadjustments if necessary. The significant estimates in its accrued research and development costs are related to fees paid to clinical researchorganizations, or CROs, in connection with research and development activities for which the Company has not yet been invoiced. The Company basesits expenses related to CROs on its estimates of the services received and efforts expended pursuant to quotes and contracts with CROs that conductresearch and development on its behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. Theremay be instances in which payments made to vendors and other counterparties will exceed the level of services provided and result in a prepayment ofthe research and development costs. In accruing service fees, the Company estimates the time period over which services will be performed and the levelof effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from the Company's estimate, itadjusts the accrual or prepayment expense accordingly. Although the Company does not expect its estimates to be materially different from amountsactually incurred, its understanding of the status and timing of services performed relative to the actual status and timing of services performed may varyand could result in reporting amounts that are too high or too low in any particular period.(c)Impairment of Assets Assets that are subject to amortization are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amountmay not be recoverable. In the year ended December 31, 2013, management reviewed the carrying amount of these assets and determined that noadjustments to carrying values were required. On assets that are not subject to amortization, the Company annually performs an impairment review based on the fair value less cost of disposalmethod. For the purpose of assessing impairment, the Company groups assets at the lowest levels for which there are separately identifiable cash flows(cash-generating units). The Company currently uses all material assets in the development of certain gene therapy products. Therefore, the managementregularly reviews all activities of the Company as a single component and one cash-generating unit. Although we are not currently selling any products,our collaborator, Chiesi, is preparing the commercial launch of Glybera in the European Union. The Company's future revenues from product sales, willdepend on the success of Chiesi'sF-25Table of Contentscommercialization efforts and the Company's success in obtaining marketing authorization for Glybera and any other product candidates in additionalcountries. The Company has determined that no impairment charge is required for the year ended December 31, 2013. Performing a further sensitivityanalysis on the fair value calculation (by for example, reducing the fair value per ordinary share by 20%, as used in the calculation of the enterprisevalue), did not change management's conclusion that no impairment charge was required. This conclusion was further supported by the IPO proceedsrealized in February 2014 and the current market capitalization. Based on management's expectations of revenues and gross margin from anticipated sales of Glybera by Chiesi, the management has determinedthat no impairment charge in respect of intangible assets relating to Glybera is necessary. These expectations are principally based on management'sestimate of the market size for Glybera and the gross margin that management expects to realize.(d)Compound Financial Instruments Management classifies a financial instrument or its component parts on initial recognition as a financial liability, a financial asset or an equityinstrument in accordance with the substance of the contractual arrangement and the definitions of a financial liability, a financial asset and an equityinstrument. As described under Notes 14, we have analyzed the convertible loan issued in 2012 and concluded that both the loan and the convertibleelements qualified as financial liabilities. Note 14 contains further details relating to the valuation of the convertible element.F-26Table of Contents5. Intangible Assets In the years presented in these financial statements, no amortization expense was recorded because the related products for which licenses havebeen granted have either not yet been approved for commercial sale by regulatory authorities, or uniQure lacked the financial and technical resources tobe confident of completing the remaining development (and therefore such approved products are not yet available for use) and or approved productswere not yet available for commercial sale. For the amount associated with Glybera amortization will start the month the first commercial sales of theapproved product will be recorded.F-27 INTANGIBLEASSETS (€ in thousands) At January 1, 2012 Cost 2,725 Accumulated amortization and impairment — Net book amount 2,725 Year ended December 31, 2012 Opening net book amount 2,725 Additions 553 Amortization expense — Closing net book amount 3,278 At December 31, 2012 Cost 3,728 Accumulated amortization and impairment — Net book amount 3,278 Year ended December 31, 2013 Opening net book amount 3,278 Additions 4,652 Reductions (155)Amortization expense — Closing net book amount 7,775 At December 31, 2013 Cost 7,775 Accumulated amortization and impairment — Net book amount 7,775 Table of Contents The net book amount of uniQure's intangible assets by licensor or product, is set out below: The amounts set out above arose as follows: In June 2001, the Group obtained a sub-license from Xenon Genetics, Inc. ("Xenon"), which was approved by Xenon's licensor, The Universityof British Columbia. The sub-licence was initially capitalized in the amount of €140,000. Xenon granted the Group the exclusive worldwide rights touse the Xenon licensed technology and to use, manufacture, distribute and sell licensed products (as defined in the sub-license agreement). The contractprovides for payment of license fees, milestone payments, and a portion of the royalties received from Chiesi, which will be payable to Xenon instead.Dependent upon the progress and success of the research and development activities and sales by the Company, future milestones are capitalized whenpayment is probable. In 2006, the Company paid a milestone of €70,000 that was capitalized. In December 2006, the Group acquired a sub-license from Targeted Genetics Corporation (now renamed AmpliPhi Biosciences, Inc.("AmpliPhi"). The sub-licence was approved by AmpliPhi's licensor, The University of Pennsylvania. It is related to "AAV1 Vector" technology, andthe recognized acquisition amount is €1,330,000, which was capitalized. In 2007, the Group acquired a license from the National Institutes of Health ("NIH") in the amount of €208,000 for the production of adeno-associated virus vectors. In 2008, the Company paid and capitalized a milestone payment of €357,000 to AmpliPhi under the above license. In 2008, the Group capitalized licensing fees totaling €600,000 related to a license from the La Sapienza University of Rome ("La Sapienza") fortechnology for treatment for Duchenne Muscular Dystrophy and a license from the San Rafaelle University of Milano for technology to be used in thetreatment of Factor IX Hemophilia. In 2009, the Group accrued for and capitalized a licensing milestone of $750,000 (€511,000) to AmpliPhi which became payable on thesubmission of the MAA of Glybera to EMA. The payment to AmpliPhi was made in 2010. In 2010, the Group terminated its research and license agreement with San Rafaelle University of Milano. This expense had been capitalized as anintangible asset, and accordingly this amount has been written off (€300,000). In 2011, the Group made and capitalized a payment to the NIH in the amount of €109,000 for a license to use adeno-associated virus serotype 5. During 2011, the Group stopped further development of its Duchenne Muscular Dystrophy program. At that time, the program had not met itsscientific goals. Accordingly, the amount capitalizedF-28 December, 31 2012 2013 Xenon 365 765 AmpliPhi 2,352 2,197 NIH 317 1,130 UCSF 244 244 St. Jude — 250 Salk Institute — 4 Protein Sciences Corporation — 77 Glybera Development Costs — 3,108 Total 3,278 7,775 Table of Contents(€300,000) as an intangible asset in respect of the license from La Sapienza described above has been written off. In 2012, the Group made and capitalized a payment to AmpliPhi Biosciences Corporation of $200,000 (€154,000) in accordance with its financialobligations relating to Glybera. In 2012 the Group also made and capitalized a payment to Xenon Pharmaceuticals Inc. of CAN$ 200,000 (€155,000) in respect of Glybera'sapproval by EMA. In 2012, the Group made and capitalized a payment to the University of California at San Francisco ("UCSF") of $300,000 (€244,000) in respectof the license to certain data, know-how, and other rights relating to the program for Parkinson's disease. On March 21, 2013, the Company secured a €10.0 million convertible loan, which provided resources to complete development of Glybera.Accordingly, from March 21, 2013, the Company has capitalized development costs relating to Glybera. Following commercial launch of the productby uniQure's commercial partner, Chiesi, which is expected to occur mid 2014, the intangible asset will be carried at its cost less any accumulatedamortization and any accumulated impairment losses (cost model). The estimated useful life over which the intangible will be amortized is estimated atapproximately 19 years. As at the December 31, 2013 Balance sheet date the company recorded a total of €3,108,000 related capitalized Developmentcosts for Glybera. In June 2013, when the agreements with Chiesi became unconditional, the Company booked amounts related to amendment fees in relation tolicenses granted to subcontractors for a total amount of €1,544,000, broken out as follows: Xenon €400,000, NIH €813,000, St. Jude €250,000, SalkInstitute €4,000 and Protein Sciences Corporation €77,000. For the last three parties mentioned the Company incurred annual maintenance fees only inprior years. On July 1, 2013, the Company altered the terms of the previous Glybera-related license agreement, entered into in 2012, withAmpliPhiBiosciences Corporation, reducing the capitalized amount by €155,000 (CAN$200,000).F-29Table of Contents6. Property, Plant and Equipment LEASEHOLDIMPROVEMENT CONSTRUCTIONIN PROCESS LABORATORYEQUIPMENT OFFICEEQUIPMENT TOTAL (€ in thousands) As ofJanuary 1,2012 Cost 770 — 2,939 555 4,264 Accumulateddepreciation (508) — (2,377) (484) (3,369) Net bookamount 262 — 562 71 895 Year endedDecember 31,2012 Opening netbook amount 262 — 562 71 895 Additions 494 — 20 324 838 Depreciationcharge (158) — (312) (78) (548) Closing netbook amount 598 — 270 317 1,185 As ofDecember 31,2012 Cost 1,264 — 2,959 879 5,102 Accumulateddepreciation (666) — (2,689) (562) (3,917) Net bookamount 598 — 270 317 1,185 Year endedDecember 31,2013 Opening netbook amount 598 — 270 317 1,185 Additions — 1,285 175 504 1,964 Depreciationcharge (185) — (124) (226) (535) Closing netbook amount 413 1,285 321 595 2,614 As ofDecember 31,2013 Cost 1,264 1,285 3,134 1,383 7,066 Accumulateddepreciation (851) — (2,813) (788) (4,452) Net book Construction in Process ("CIP") at December 31, 2013 relates to the build-out of the manufacturing facility in Lexington, Massachusetts, thatstarted at the end of the second quarter of 2013. Depreciation expense of €535,000 for the twelve months ended December 31, 2013 (twelve months ended December 31, 2012: €548,000, 2011:€591,000) has been charged in research and development expense. Leasehold improvements include a net book value as of December 31, 2013 of €383,000 (2012: €520,000) where uniQure is lessee under afinance lease. A further description of financial lease contracts is set out in Note 13 below. Following the reorganization in 2011, uniQure entered into revised rental agreements with AMC and its representatives, as a consequence ofwhich certain parts of the premises, with a cost of €446,000 at December 31, 2012, are now accounted for under a finance lease instead of an operatinglease; the assets covered by this change in contractual arrangements are included within the amount of €494,000 shown as additions to leaseholdimprovements for the year ended December 31, 2012.7. Other Non-Current Assets As of December 31, 2013, the amount represents a refundable security deposit for the Lexington, Massachusetts facility, paid in September 2013.F-30amount 413 1,285 321 595 2,614 Table of Contents8. Trade and Other Receivables The fair value of trade and other receivables approximates their carrying value. As of December 31, 2013 and December 31, 2012, all trade andother receivables were assessed as fully recoverable. The carrying amount of the Company's trade receivables are fully denominated in Euro. The receivables from related parties as of December 31, 2013 relate to invoiced amounts to Chiesi based on revenue recognized and expensesreimbursed of €1,402,000; (2012: nil). The remaining element of receivables from related parties relate to certain wage tax liabilities settled by AMT onbehalf of senior management in connection with purchases of AMT depositary receipts in 2007; these amounts are repayable to uniQure on sale of therelated depositary receipts or on the respective employee ceasing to be employed by the Company of €23,000; (2012: €26,000). The Other Receivables balance at December 31, 2013 consists largely of amounts of tenant improvements due to the Company from the landlord inrelation to our facility in Lexington, Massachusetts (€546,000), as well as prepayments related to rent, insurance and certain annual licence fees insoftware and intellectual property. The other classes within trade and other receivables do not contain impaired assets. The maximum exposure to credit risk at the reporting date is thecarrying value of each class of receivable mentioned above.9. Inventories Inventories as of December 31, 2013 were €865,000 (2012: € nil). The amount includes the raw materials that are to be capitalized in connectionwith the manufacturing of Glybera for commercial sale, which is expected to commence mid 2014. Also included in inventories are amounts assigned towork in progress and intermediate products following the initial production batches of Glybera.10. Cash and Cash EquivalentsF-31 DECEMBER 31,2012 DECEMBER 31,2013 (€ in thousands) Receivables from related parties 26 1,425 Other receivables 397 764 Prepaid Expenses — 391 Social security and other taxes 418 402 Trade and other receivables 841 2,982 DECEMBER 31,2012 DECEMBER 31,2013 (€ in thousands) Raw materials — 103 Work in Process/Intermediate Products — 762 Inventories — 865 DECEMBER 31, 2012 2013 Cash at bank and in hand 263 23,810 263 23,810 Table of Contents The cash balance as of December 31, 2013 reflects the receipt of €17,000,000 in up-front payments from Chiesi (July 2013), the €14,000,000investment in equity from Chiesi (July 2013), €10,000,000 in convertible debt financing from Coller Capital (March 2013), $10,000,000 in venture debtfinancing from Hercules Technology Growth Capital (June 2013) and the drawdown of the remaining advance relating to the December 2012convertible loan agreement, amounting to €1,999,000.Supplemental information relating to the Cash Flow Statement The conversion of the €5,000,000 convertible loan, together with accrued interest of €320,000, amounting to €5,320,000 in aggregate represented anon-cash item as of December 31, 2012. The conversion of the €13,497,000 convertible loan, comprising an amount of €1,498,000 drawn down inDecember 2012 and the balance of €11,999,000 drawn down during 2013, represented a non-cash item as of December 31, 2013. Refer to Note 14below. The derivative result arising on early conversion of the loan, amounting to €1,333,000 and the derivative result relating to embedded derivatives,amounting to €2,113,000, represented non-cash items as of December 31, 2013. Purchases of fixed assets and changes in trade and other payables exclude a non-cash item of €628,000 largely related to the purchase of fixedassets, which have not yet been paid as of December 31, 2013. (2012 and 2011: nil) All non-cash items described above are excluded from the Consolidated Statement of Cash Flows on page F-6.11. Shareholders' (Deficit)/Equity uniQure was incorporated on January 10, 2012; therefore, the year ending December 31, 2012 is the first accounting period for the Company. Asdescribed in Note 1 above, the business combination between uniQure and the AMT Group is accounted for as a reverse acquisition and theconsolidated financial statements of the AMT Business are presented as the consolidated financial statements of uniQure, with an adjustment required toreflect the capital of uniQure. The amount recognized as issued equity interests in the consolidated financial statements is determined by the issued equity interest in AMToutstanding immediately prior to the business combination, but the equity structure (the number and type of equity interests issued) reflects the equitystructure of uniQure. Accordingly the share capital and share premium accounts of AMT disclosed in its audited consolidated financial statements forprior years are restated as if uniQure ordinary shares had been issued. The exchange ratio of uniQure shares issued for AMT shares was 1-for-1, butbecause AMT shares had a nominal value of €0.20 and uniQure shares have a nominalF-32Table of Contentsvalue of €0.05, the impact of this approach is to reduce the balance of the share capital reported within the previous AMT accounts and correspondinglyincrease the balance on the share premium account. NUMBER OFSHARES AMOUNT OFAMT CAPITAL(BASED ONSHARES OF €0.20NOMINAL VALUE) AMOUNT OFUNIQURE CAPITAL(BASED ONSHARES OF €0.05NOMINAL VALUE) (€ in thousands) Share capital (ordinary shares) As of January 1, 2011 4,702,445 Share capital 940 235 Share premium 99,136 99,841 Total 100,076 100,076 New shares issued 47,180 Share capital 10 2 Share premium 98 106 Total 108 108 As of January 1, 2012 4,749,625 Share capital 950 237 Share premium 99,234 99,947 Total 100,184 100,184 New shares issued prior to April 5, 2012 1,470,588 Share capital 294 74 Share premium 2,206 2,426 Total 2,500 2,500 Shares in issue at April 5, 2012 6,220,213 Share capital 1,244 311 Share premium 101,440 102,373 Total 102,684 102,684 New shares issued after April 5, 2012 3,433,282 Share capital n/a 172 Share premium n/a 12,422 Total n/a 12,594 As of December 31, 2012 9,653,495 Share capital n/a 483 Share premium n/a 114,795 Total n/a 115,278 New shares issued after December 31, 2012 2,541,411 Share capital n/a 127 Share premium n/a 27,664 Total n/a 27,791 As of December 31, 2013 12,194,906 Share capital n/a 610 Share premium n/a 142,459 F-33Total n/a 143,069 Table of Contents During the period covered by these financial statements, uniQure had a single class of shares, which are denominated as ordinary shares. Withinthis class of ordinary shares, there were further sub-denominations between Class A ordinary shares, class B ordinary shares and class C ordinaryshares. Other than the fact that certain corporate resolutions required the approval of the general meeting of the class A ordinary shares, class A, B andC ordinary shares carried equal economic rights and ranked equally. Following the general meeting of shareholders of uniQure on July 22, 2013, the Company's authorized share capital was increased from€ 1,900,000 or 38,000,000 shares, to €2,000,000 or 40,000,000 shares through the creation of an additional €100,000 or 2,000,000 class C ordinaryshares, in connection with the intended equity investment by Chiesi which took place on July 24, 2013. The authorized share capital of uniQure was asfollows as of December 31, 2013: As of December 31, 2013, a total of 12,194,906 shares were issued and paid up in full at a nominal value of €0.05 per share (2012: 9,653,495shares at €0.05 per share and 2011: 4,749,625 AMT shares at €0.20 per share prior to adjustment in accordance with IFRS 3 and restated as if theywere uniQure shares at €0.05 per share). Of these, 2,541,411 are presented as being issued during the year (2012: 4,902,473 shares, 2011: 47,180shares). The total gross payment with respect to these shares issued during the period is presented as €27,791,000 (2012: €15,094,000, 2011:€108,000). A B C TOTAL Number of Ordinary Shares 34,281,263 3,718,737 2,000,000 40,000,000 Value (€) 1,714,063 185,937 100,000 2,000,000 Date Description Sub-classofordinaryshares Number ofshares SharecapitalAmounts SharepremiumAmounts TotalequityAmounts (€ in thousands) January 1,2012 Broughtforward 4,749,625 237 99,947 100,184 January 4,2012 Investmentin AMTordinaryshares 1,470,588 74 2,426 2,500 April 5, 2012 Forbionconversionof existingconvertibleloan plusinterest A 1,064,000 53 5,267 5,320 April 5, 2012 Forbionnew equityinvestment A 1,954,395 98 5,902 6,000 April 18, 2012 Gilde newequityinvestment A 325,732 16 984 1,000 November–December,2012 Employeesand otherpersonsnew equityinvestment B 89,155 5 269 274 December 31,2012 9,653,495 483 114,795 115,278 January–May,2013 Employeesand otherpersonsnew equityinvestment B 90,747 4 274 278 This note describes the shares issued during the period since January 1, 2012. In summary these were as follows:•On January 4, 2012, AMT raised €2,500,000 through the issuance of 1,470,588 new shares at a price of €1.70 per share. On April 5,2012, uniQure acquired the AMT Business, issuing 6,220,213 class B ordinary shares, represented by uniQure DRs to the AMTShareholders as consideration. Since this transaction is accounted for as a reverse acquisition, this issue of uniQure DRs is not disclosedseparately within the consolidated financial record of the business;F-34July 24, 2013 Chiesi newequityinvestment C 1,109,214 55 13,945 14,000 July 26, 2013 Conversionof 2012 &2013convertibleloans A 1,336,331 67 13,430 13,497 November,2013 Exercisingof options B 5,118 1 15 16 December 31,2013 12,194,906 610 142,459 143,069 Table of Contents•On April 5, 2012, uniQure raised €6,000,000 through the issue of 1,954,395 class A ordinary shares to Forbion, at a price of €3.07 pershare. On April 5, 2012, the Company issued 1,064,000 class A ordinary shares to Forbion, at a price of €5.00 per share inconsideration of the conversion of the outstanding €5,000,000 in convertible loan notes, together with accrued interest of €320,000; •On May 17, 2012, uniQure raised €1,000,000 through the issue of 325,732 class A ordinary shares to Gilde, at a price of €3.07 pershare; •In November and December 2012, pursuant to an agreement entered into in April 2012, the Company raised a total amount of €274,000through the issuance of an aggregate of 89,155 class B ordinary shares, represented by uniQure DRs shares to employees and relatedparties at a price of € 3.07 per share; •In January 2013 pursuant to an agreement entered into in April 2012, the Company raised a further amount of €278,000 through theissuance of an aggregate of 90,747 class B ordinary shares, represented by uniQure DRs shares to employees and related parties at aprice of € 3.07 per share; •On July 24, 2013 pursuant to various agreements with Chiesi Pharmaceutici S.p.A the Company raised a total amount of €14,000,000through the issuance of 1,109,214 Class C ordinary shares at a price of €12.60 per share; •On July 26, 2013 the Company converted the 2012 Convertible loan through the issuance of 1,336,333 Class A shares at a price of€10.10 per share; and •In November 2013 through conversion of share options the Company issued 5,118 Class B ordinary shares at a price of €3.07 pershare. In 2012 and 2011 no new shares were issued upon the exercise of share options. In November 2013 a total of 5,118 shares were issued uponexercise of share options.As of December 31, 2013, 7,258 shares were held by the stichting participatie AMT as treasury shares (2012 and 2011: 7,258). (Further details ofstichting participatie AMT are set out in Note 1 above.) These treasury shares arose under the terms of an employee incentive plan operated by AMT,underF-35 NARRATIVE CASHITEMS NONCASHITEMS TOTAL (€ in thousands) Jan 4, 2012 Investment in AMT ordinary shares 2,500 — 2,500 Apr 5, 2012 Forbion new equity investment 6,000 — 6,000 Apr 5, 2012 Forbion conversion of existing convertible loan plusinterest — 5,320 5,320 Apr 19, 2012 Gilde new equity investment 1,000 — 1,000 Nov-Dec, 2012 Employees and other persons new equity investment 274 — 274 As at Dec 31, 2012 9,774 5,320 15,094 Jan, 2013 Employees and other persons new equity investment 278 — 278 July 24, 2013 Chiesi new equity investment 14,000 — 14,000 July 26, 2013 Conversion of existing 2012 convertible loan — 13,497 13,497 Nov 2013 Exercising of options 16 — 16 As at Dec 31, 2013 14,294 13,497 27,791 Table of Contentswhich employees were permitted to subscribe for new shares at a discount to the market price, but were then required to remain with AMT for a periodof three years following the effective date of such purchase. Employees who left AMT within such three year period and who did not meet certain otherexceptional conditions were obliged to return their shares.Share Premium The presentation of the share premium account is on a consistent basis with the share capital account, including similar adjustments to reflect theimpact of the treatment under IFRS 3, as set out in the table above. The total additions to share premium in the year ended December 31, 2013 amount to €27,664,000 net of costs. This increase in share premiumwas due to the issue of shares as described above.Other Reserves The costs of equity-settled share-based payments to employees are recognized in the income statement, together with a corresponding increase inequity during the vesting period, taking into account (deferral of) corporate income taxes. The accumulated expense of the share incentive planrecognized in the income statement is shown separately in the equity category Other Reserves in the Consolidated Statement of Changes in Equity. In2011 the Company recorded an expense related to the AMT share option plan of €940,000. The accumulated expense related to the AMT share optionplan (described further below) for the period up to April 5, 2012, amounting to €2,987,000, is offset against the retained losses at April 5, 2012following the extinguishing of AMT and the AMT share option scheme, as set out in the Consolidated Statement of Changes in Equity. The Company presented in other reserves the result of the conversion of the convertible loan to the amount of €3,005,000 (see Note 14). In the years presented in these financial statements, the Company did not have any legal or other types of restricted reserves.12. Share Based Payments2012 Share Option Plan At the general meeting of shareholders on February 15, 2012, uniQure shareholders approved the adoption of the 2012 Plan. Under the 2012 Plan,share options were granted on the date of grant and vest over a period of three years on the basis set out in Note 2.17 above. Any options that vest must be exercised by the tenth anniversary of the effective date of grant. In 2012, 1,606,347 options were granted under the 2012 Plan to management and certain other employees and consultants. The expenserecognized amounted to €1,767,000 during the year ended December 31, 2012. In the year 2013 the company granted another 301,468 options, a totalof 210,853 were forfeited and a total of 5,118 options were exercised, to result in an ending balance as of December 31, 2013 of a total of 1,691,844outstanding options recognizing a share based expense of €2,023,000. On October 25, 2011, AMT announced a reorganization resulting in a reduction of the AMT Group's workforce of approximately 50% andsubsequent transfer of its assets and liabilities to uniQure pursuant to the transaction entered into on April 5, 2012. Consequently, AMT's 2010 Planwas deemed to have been closed and the outstanding options thereunder cancelled. Accordingly, AMT recognized the remaining option expense forAMT 2010 Plan participants that remained with the Company following the reorganization on the basis of a reduced vesting period, and recognized thepro rata element of this charge in 2011. The consequence of this was a total option expense recognized andF-36Table of Contentsaccounted for within retained earnings of €259,000 for the period January 1 to April 5, 2012 (for the year ended December 31, 2011 the recognizedcharge amounted to: €940,000). On April 5, 2012, the AMT 2010 Plan and the outstanding options granted under it were cancelled. Accordingly, theaccumulated reserve was transferred to retained earnings, as described in the Consolidated Statement of Changes in Equity above. Both the 2012 Plan and AMT 2010 Plan qualify as equity-settled plans. Movements in the number of outstanding share options granted in 2012and 2013, under the 2012 Plan, were as follows: Of the 1,691,844 options outstanding (2012: 1,606,347; 2011: 379,640), 773,442 options (2012 and 2011: nil) were exercisable. Optionsoutstanding at the end of the year have the following weighted average remaining contractual life and ranges of exercise prices:F-37 2011 2012 2013 NUMBER EXERCISEPRICE NUMBER EXERCISEPRICE NUMBER EXERCISEPRICE Number ofoptionsoutstandingas ofJanuary 1 270,830 9.75–14.6 379,640 9.75–14.6 1,606,347 3.07 Number ofoptionsgranted 150,241 10.3 1,606,347 3.07 301,468 3.07–10.10 Number ofoptionsforfeited (41,431) 10.3–14.6 (379,640) 9.75–14.6 (210,853) 3.07 Number ofoptionsexercised — — — — (5,118) 3.07 Number ofoptionsoutstandingas ofDecember 31 379,640 9.75–14.6 1,606,347 3.07 1,691,844 3.07–10.10 YEAR ENDED DECEMBER 31, 2013WEIGHTED AVERAGE REMAINING CONTRACTUAL LIFE RANGE EXERCISEPRICE INEUR PER SHARE OPTIONS 1–5 years — — 6 years — — 7 years — — 8 years 3.07 1,397,127 9 years 3.07-10.10 294,717 At December 31, 2013 3.07-10.10 1,691,844 Table of ContentsF-38YEAR ENDED DECEMBER 31, 2012WEIGHTED AVERAGE REMAINING CONTRACTUAL LIFE RANGE EXERCISEPRICE INEUR PER SHARE OPTIONS 1–5 years — — 6 years — — 7 years — — 8 years — — 9 years 3.07 1,606,347 At December 31, 2012 3.07 1,606,347 YEAR ENDED DECEMBER 31, 2011WEIGHTED AVERAGE REMAINING CONTRACTUAL LIFE RANGEEXERCISEPRICE INEUR PER SHARE OPTIONS 1 - 5 years — — 6 years — — 7 years — — 8 years 9.75–14.60 222,650 9 years 10.30–14.60 156,990 At December 31, 2011 9.75–14.60 379,640 Table of Contents The Black-Scholes option pricing model has been used to value these awards, based on the following key variables: Of the 1,606,347 options granted in 2012, 478,217 options were granted to members of the Management Board and 196,912 options were grantedto members of the Supervisory Board. In 2013 in total another 301,468 options were granted (of which 252,652 options to members of theManagement Board and 10,000 options to a member of the Supervisory Board) 210,853 options (of which 140,652 options from a member of theManagement Board and 37,507 options from a member of the Supervisory Board) were forfeited in 2013. In November 2013 a total of 5,118 optionswere exercised. The 1,691,844 options outstanding at December 31, 2013, in total represent a further share based expense of €1.7 million to be recognized from2014 through to 2016. In addition, in January 2014 the Company granted another 609,744 options to the management of 4D Molecular Therapeutics.Expected option term uniQure has considered various approaches to take into account the effects of expected early exercise whereby the length of the vesting period, theexpected share price development, the expected share price volatility and the participants' employee level within the organization have been analyzed. Based on the outcome of this analysis, uniQure management has determined to take the effects of expected early exercise into account by using anestimate of an option's expected life as an input into the Black-Scholes option pricing model. As historical data about employees' exercise behavior is notavailable, management's estimate is based on a weighted average expected option life for the entire participant group. The resulting expected weightedaverage life of the options granted is the midway between the vesting date and the contractual term of the options.F-39 2011 2012 2013 Options with change of control and service based vestingconditions 379,640 — — Options with an IPO, change of control and service basedvesting conditions — 1,606,347 1,691,844 Share Price: the closing share price on the grant dates €9.75–14.85 €3.07–5.10 €5.00–13.40 Estimated fair value per option as of grant date €1.95–2.97 €2.05–3.60 €3.40–12.35 Expected Volatility: uniQure used an estimated volatilityfigure which was determined based on volatility analysisof companies in the same sector and of a similar size 50% 70–80% 70%Expected Term: is the period from grant until the expectedexercise date. 6–7 years 5.5–6.3 years 5.5–6.3 years Exercise price (in €): €9.75–14.85 €3.07 €3.07–10.10 Expected Dividend Yield: the Company currently does notpay dividends and has no plans to do so 0% 0% 0%Risk-free Rate: based on Government bonds with a term that iscommensurate with the expected term of each optiontranche. Also considered is the risk-free rate over theperformance period for each option tranche 2.3% 0.5–1.1% 0.4–1.2%Table of ContentsValuation of ordinary shares AMT shares were previously listed on Euronext Amsterdam. The initial valuation of €3.07 per uniQure share derived from the average closingprice of AMT shares on each of the 5 business days immediately prior to February 17, 2012, the date of the announcement of the transaction betweenuniQure and AMT, which was also €3.07 per AMT share. Given that uniQure had no other business of its own, and that the consideration for purchaseof the business and assets of AMT was a one-for-one share issue to AMT in respect of each AMT share then in issue, the Company believed this valuewas reasonable and reflected the market valuation of the business. At the date of each grant of options subsequent to the transaction between uniQure and AMT, the fair value of the ordinary shares is determined bythe Management Board and Supervisory Board, and takes into account the most recently available valuation of ordinary shares and the assessment ofadditional objective and subjective factors the Company believes are relevant.Expected volatility Prior to the transaction between uniQure and AMT on April 5, 2012, AMT was listed on the Euronext Amsterdam exchange from June 2007through April 2012. This period has provided company-specific historical and implied volatility information. In April 2012, the weighting assigned tothe company-specific historic volatility was 50%, and uniQure has also estimated the expected volatility based on the historical volatility of the publiclytraded peer companies for the remaining 50% weighting. For option grants post April 2012, the volatility has been estimated solely by reference to thehistorical volatility of the publicly traded peer companies. This has resulted in a volatility in the range 70 - 80% in respect of the options granted in theyear ended December 31, 2012, and an applied volatility of 70% in respect of the options granted in the year ended December 31, 2013. Further details regarding the total expense recognized in the income statement for share options granted to managing directors, supervisorydirectors and selected employees are set out in Note 30. The corresponding increase in equity is separately accounted for as other reserves.13. Financial Lease Liabilities uniQure leases certain leasehold improvements by means of finance leases including the following:•Agreement between Beheersmaatschappij Dienstverlening en Deelneming AZUA BV ("BDDA"), a wholly-owned subsidiary of theAMC, and uniQure, regarding leasehold improvements at Meibergdreef, Amsterdam, ending at September 30, 2016. The rent of theleasehold improvements amounts to €156,000 per year. The Company has the right to cancel the lease earlier on a one-year term;however, the Company will then need to repay the remaining amount of leased leasehold improvements.Finance lease liabilities are effectively secured as the rights to the leased asset revert to the lessor in the event of default. The carrying amountcorresponds to the fair value as terms of the contracts wereF-40Table of Contentsagreed at arm's length and market conditions for such contracts have not subsequently changed. The interest rate imposed by the lessor for all financelease liabilities is 5.5% per annum. The present value of finance lease liabilities is as follows:14. Debt to related partyDecember 2009 Convertible loan On December 16, 2009, AMT entered into a convertible loan agreement with Forbion, one of its major shareholders, in respect of five-yearunsecured and unsubordinated loan notes ("2009 Notes"), which had an issue price of 100% and paid an annual coupon of 5%. This loan was drawndown on December 23, 2009. During the conversion period, which started six months after the funding date (or at the earlier occurrence of a limitednumber of events, such as a public offer to acquire AMT) and which ended on the final maturity date, the 2009 Notes were convertible into ordinaryshares of AMT at an initial conversion price of €19.55, representing a conversion premium compared to AMT's share price at the date of issue ofapproximately 30%. The conversion price could be adjusted in the case of certain dilutive events, including an issue of shares at a discount to theaverage share price over the preceding five day period. As a consequence, the private placement by AMT on October 6, 2010, resulted in such anadjustment to the conversion price of the bonds from €19.55 per share to €18.45 per share, representing a conversion premium compared to AMT'sshare price at this date of 54%. On April 5, 2012 the obligations under the loan were transferred from AMT to uniQure, and were then converted into new uniQure shares at aconversion price of €5.00/share. Further details on the accounting policy applied to the convertible loan agreement are described in paragraph 2.10 (convertible loan) above. At December 31, 2011 the conversion price of the convertible loan was above the market price of AMT ordinary shares. In such a situation theconvertible loan was not regarded as being dilutive at December 31, 2011. The valuation methodology used for the option part employed a Black-Scholes approach on the assumption that the loan would not be convertedbefore its maturity date.F-41 December 31, 2012 2013 Gross finance lease liabilities—minimum lease payments No later than 1 year 184 184 Later than 1 year and no later than 5 years 505 322 Later than 5 years — — Future finance charges on finance leases (88) (48) Total 601 458 December 31, 2012 2013 No later than 1 year 151 156 Later than 1 year and no later than 5 years 450 302 Later than 5 years — — Future finance charges on finance leases — — Total 601 458 Table of Contents Under IFRS 7.27, the relevant factors considered within the valuation model for the compound of the instrument are as follows:•AMT share price of €1.83 at December 31, 2011; •Conversion price of €18.45 at December 31, 2011; •Expected life of the instrument of 3 years; •Annualized volatility of AMT share price of 50%; •Implied call price of €27.68 (being 150% of the €18.45 exercise price); •Annual rate of quarterly dividends of 0%; and •Discount rate—Bond yield equivalent of 0.779%.The rate used in 2011 for discounting the financial liability represented by the loan element of the convertible in 2011 was 8.5% per annum. On February 17, 2012, AMT announced the sale and transfer of the AMT Business to uniQure. Under the terms of the transaction, the convertibleloan was transferred to uniQure and then converted at a subscription price of €5.00 per share.December 2012 Convertible loan and amendment in March 2013 On December 17, 2012, uniQure entered into a convertible loan agreement with four of its major shareholders (Forbion, Gilde, Grupo Netco andLupus Alpha), in respect of unsecured and unsubordinated loan notes, which have an issue price of 100% and pay an annual coupon of 8%. Of the totalloan €1,498,000 was drawn down in the period to December 31, 2012 and the balance of €1,999,000 was drawn down in the period from January 1,2013 to January 31, 2013, amounting to a total convertible loan amount of €3,497,000. In March 2013, uniQure increased the loan by an additional €10,000,000 investment by Coller Capital. As part of the increase, the loan note termsfor all loan note holders described in the annual consolidated financial statements were amended such that the final maturity date of the loan notes wasextended to December 31, 2014. Additionally, the warrant entitlement was reduced to 10% of the principal amount of the loan provided to uniQure. Following the subscription for new equity by Chiesi, on July 21, 2013 the full convertible loan of €13,497,000.was converted on July 26, 2013into new Class A Ordinary Shares, at a conversion price of €10.10 per share. This conversion marked the extinction of the convertible derivativeinstrument. The remaining derivative element arises from the warrants issued to the holders of the convertible loan as part of the convertible loanarrangements. The warrants associated with the convertible loan, and which survive the conversion of the loan, are presented in the consolidated Balance Sheet asat December 31, 2013 within liabilities as an embedded derivative with a fair value of €722,000 (December 31, 2012: €132,000). During the period ending December 31, 2013, an amount of €4,387,000 (compared with €547,000 for period ending December 31, 2012) wasrecorded as finance expense. This amount relates to €3,491,000 of derivative results (compared with €464,000 for the period ending December 31,2012) and the remainder consists of interest expense in relation to the convertible note, Hercules borrowing and interest expense on the financial lease. The elimination of the embedded derivative (convertible element) by the early conversion of the loan created €3,005,000 of Other Reserves withinthe Equity presentation.F-42Table of Contents15. Trade and Other Payables The carrying values of trade and other payables are assumed to approximate their fair values.Other current liabilities As of December 31, 2013 and December 31, 2012, other current liabilities consisted principally of accruals for services provided by vendors butnot yet billed, reimbursements received from research and development partners for expenses which have yet to be incurred and miscellaneousliabilities.16. BorrowingsHercules Borrowing The presented non-current borrowings relate to the Hercules Technology Growth Capital venture debt loan facility, entered into on June 14, 2013for a book value of €6,925,000 as of December 31, 2013, presented net of expenses for facility charges of 1.25% plus expenses related to legal counsel.The loan commitment is $10 million with an interest rate of 11.85% and a back-end fee of 3.45%, which matures over a period of 39 months from theloan closing date. The interest-only period was initially set at 9 months and was extended to 15 months on completion of the transaction with Chiesi. Inaddition, the loan is secured by a lien on all of the Company's assets. The venture debt loan facility is governed by certain covenants and as perDecember 31, 2013 the Company is in compliance with these covenants. On the balance sheet for the period ending December 31, 2013 the book value of €6,925,000 (fair value €7,490,000) is represented by a non-current element of €6,292,000 and a current element of €633,000. The fair value of the current borrowings does not equal the carrying amount of theloan as of December 31, 2013. The fair value is based on scheduled cash flows (future interest and principalF-43 DECEMBER 31,2012 DECEMBER 31,2013 (€ in thousands) Trade payables 2,099 3,507 Social security and other tax 152 802 Other current liabilities 1,816 3,292 Total trade and other payables 4,067 7,601 DECEMBER 31,2012 DECEMBER 31,2013 (€ in thousands) Non-current Borrowings — 6,292 Total non-current — 6,292 Current Debt to related party—Financial liability (see Note 14) 1,366 — Debt to related party—Embedded derivative (see Note 14) 132 722 Borrowings — 633 Borrowings—Embedded derivative — 217 Total current 1,498 1,572 Total 1,498 7,864 Table of Contentspayments) discounted using a rate of 13.5% (2012: not applicable as the loan initiated in 2013) and are within level 2 of the fair value hierarchy. The warrant included in this loan agreement is not closely related to the host contract and therefore has been split and accounted for separately as afinancial derivative measured at fair value though profit or loss. The fair value of this embedded derivative is €217,000 and is included within theCurrent liabilities: Borrowings—embedded derivative on the consolidated balance sheet as of December 31, 2013. There is no exposure for the Company's borrowings to interest rate changes and contractual repricing. Interest rates are fixed until maturity.17. Revenues and Deferred Revenues During the period ending December 31, 2013, an amount of €440,000 (period endings December 31, 2012 and December 31, 2011: €nil) wasrecognized as license revenues. This amount relates to the recognition of the up-front payments received from Chiesi. During the period endingDecember 31, 2013, an amount of €2,503,000 (periods ending December 31, 2012 and December 31, 2011: €nil) was recognized as collaborationrevenues. This amount related to certain approved activities the Company was able to recharge and reimbursements of expenses under its Co-Development Agreement with Chiesi in respect of its Hemophilia B program. Upon signing of the Commercialization Agreement and the Co-Development and Commercialization Agreement with Chiesi on April 29, 2013, theCompany received €17,000,000 as a non-refundable upfront payment. Based on an assessment performed to the Company, the €17,000,000 will beamortized on a straight-line basis, and presented as license revenues, over a period from July 2013 through September 2032: the date of expiration ofthe last intellectual property protection related to the manufacturing process. The Company determined that the €17,000,000 of up-front paymentsreceived from Chiesi constituted a single unit of accounting. The up-front payments related to licenses and reimbursement of past development costs forGlybera and hemophilia B as follows:1)€2,000,000—Reimbursement of past development costs related to Glybera. Continuing performance obligation: maintaining the marketauthorization for Glybera (including the post-approval commitment to conduct the Phase IV study); 2)€5,000,000—for past development costs related to hemophilia B. Continuing performance obligation: complete the Co-Development programand file for Marketing Authorization in the European Union;F-44 For the years ended DECEMBER 31,2011 DECEMBER 31,2012 DECEMBER 31,2013 (€ in thousands) License Revenues — — 440 Collaboration Revenues — — 2,503 — — 2,943 DECEMBER 31,2012 DECEMBER 31,2013 (€ in thousands) Deferred License Revenues Current Portion — 1,279 Deferred License Revenues — 15,679 — 16,958 Table of Contents3)€10,000,000—for having set up an EMA approved manufacturing/production facility. Continuing performance obligation: supply ofcommercial product to Chiesi.Although the Company believes that the different elements have different cost levels, the Company is not able to properly estimate the respective fairvalues of the various elements. Therefore, the Company has concluded that the three deliverables within the arrangement are linked in such a way thatthe commercial effect cannot be understood without reference to the series of transactions as a whole. Therefore, the individual performance obligationswere combined as a single unit of accounting and the total arrangement consideration will be recognized over the estimated life of the agreements underwhich the continuing performance obligations exist. The elements described above are based on the current assumption that hemophilia B is anticipated to receive regulatory approval in late 2018, andthat the commercial launch is within 3 months following approval. Based on the above, best estimate of the anticipated duration of the agreements is inline with the expiration term of the patent for manufacturing of commercial product which is 19 years. Based on the aforementioned facts, the Companyhas deferred the revenue and will recognize the €17,000,000 of up-front payments as license revenue on a straight-line basis over 19 years. For the period ending December 31, 2013, the Company recognized an expense, under Costs of goods sold, in relation to its obligation to repay tothe Dutch Government a portion of a grant received between 2001 and 2005 in connection with the development of Glybera; the amount was calculatedas an agreed 40% of the upfront payment received in relation to Glybera. See a further description under Note 28, Contingent Liabilities. Collaboration revenues from contracts, typically from delivering research and development services, relate to the agreements, and is recognized onthe basis of labor hours delivered at the Agreements' full time employee rate. Cost reimbursements to which the Company is entitled to under agreements are also recognized as collaboration revenues in the income statementin the same quarter of the recorded cost they intend to compensate, except for reimbursement of certain expenses incurred in the periods prior to thecompletion of the Chiesi agreements (on June 30, 2013); such revenues are recognized at the moment that Chiesi incurred the obligation to reimbursethem, i.e. on June 30, 2013. When the reimbursable costs are not yet invoiced these amounts are included as a component of trade and other receivableson the balance sheet.18. Other Income/Other Losses uniQure's other income consists of government subsidies and grants that support uniQure's research efforts in defined research and developmentprojects. Other income was €585,000 in 2013 (2012 €649,000; 2011: €2,192,000) and relates to grants received and rebates on payroll taxes. Grant income was reduced in 2013 as this income includes an element of rebate on payroll taxes and in 2013 the levels of rebate were reducedfurther. The Other Losses line represents the currency effect from regular operations whereas the currency risk associated with borrowings is presentedunder Finance Income or Expense.19. Expenses by Category Research and development costs amounted to €13,182,000, € 10,231,000 and €15,500,00 in 2013, 2012 and 2011, respectively, and consist ofallocated employee costs, Good Manufacturing Practices ("GMP") facility costs, clinical development costs, collaboration costs, license costs, the costsof laboratory consumables and allocated depreciation costs. General and administrative costs amounted to €F-45Table of Contents11,628,000, €4,564,000 and €3,807,000 in 2013, 2012 and 2011, respectively, and consist of allocated employee costs, office costs, consultancy costsand administrative costs. Research and development costs and general administrative costs included the following costs by function:20. Research and development expenses Research and development expenses increased from €10,231,000 in the period ending December 31, 2012 to €13,182,000 in the period endingDecember 2013, due to the additional development and clinical activities required to support the planned commercial launch of Glybera, as well as theprogression of uniQure's other programs through late stage research and clinical development, (for the period ending December 31, 2011:€15,500,000).21. General and administrative expenses General and administrative expenses increased from €4,564,000 for the period ending December 31, 2012 to € 11,628,000 for the period endingDecember 31, 2013. The increase is primarily due to expenses related to consultants (commercial, operations and administrative) and professional fees,(for the period ending December 31, 2011: €3,807,000).22. Other Comprehensive Income For the period ending December 31, 2013 the amount shown as €12,000 represents the foreign currency translation arising from the U.S.subsidiary, which was established in 2013 (for the period ending December 31, 2012 and 2011: €nil).F-46 For the years endedDecember 31, 2011 2012 2013 Employee benefit expenses 8,493 8,350 11,904 Laboratory and development expenses 4,854 2,065 3,404 Legal and advisory expenses 2,416 1,622 5,001 Office and housing expenses 1,420 1,197 1,592 Patents and licenses 853 619 835 Other operating expenses 683 394 1,539 Depreciation expenses (See Note 6) 590 548 535 Other losses—net (exchange differences) 26 45 453 19,334 14,840 25,263 Table of Contents23. Employee Benefit Expense Wages and salaries in 2011 included termination expenses amounting to €228,000 incurred in respect of the redundancies of certain staff pursuantto the Company's restructuring. In 2012 the company recorded no termination expense and in 2013 a total of €157,000 was recorded.24. Finance Income and Expense25. Income Tax ExpenseF-47 For the years endedDecember 31, 2011 2012 2013 Wages and salaries 5,499 4,553 5,012 Social security costs 502 361 377 Share options and depository receipts granted to directors and employees (See Note 12) 940 1,767 2,023 Pension costs—defined contribution plans 400 303 415 Other employee expenses 1,152 1,366 4,077 8,492 8,350 11,904 Number of employees at the end of the period 85 67 87 For the years endedDecember 31, 2011 2012 2013 Finance income: Interest income current accounts 70 22 58 Derivative result 207 — 44 277 22 102 Finance expense: Bank borrowings—overdrafts and other debt (42) — — Derivative result arising on early conversion of the loan — (464) (1,333)Derivative result — — (2,158)Loan from related party (379) (63) (691)Venture Debt Facility — — (165)Finance leases (14) (20) (40) (435) (547) (4,387) Finance costs—net (158) (525) (4,285) For the years endedDecember 31, 2011 2012 2013 Current tax — — — Deferred tax — — — Profit/(loss) before tax (17,300) (14,716) (26,820)Expenses not deductible for tax purposes 741 2,268 2,161 Tax losses for which no deferred income tax asset was recognized (16,559) (12,448) (24,659)Tax charge — — — Table of Contents No tax charges or liabilities were incurred in the years 2013, 2012 and 2011 since the Company was in a loss-making position. No deferred taxasset has been recognized in respect of carry-forward losses. Under Dutch income tax law a tax loss carry-forward is subject to a time limitation of nine years. Losses incurred in the years up to 2004 can stillbe offset against profits up to and including 2013. In connection with the transfer of the AMT Business from AMT to uniQure, uniQure has discussedwith Belastingdienst, the Dutch tax authorities, the transfer of all accumulated tax losses that relate to the AMT Business, excluding tax losses relatingspecifically to the activities of the AMT legal entity. In order to promote innovative technology development activities and investments in new technologies, a corporate income tax incentive has beenintroduced in Dutch tax law called the Innovations Box. For the qualifying profits, the company effectively owes only 5% income tax (should availabletax losses carried forward be utilized) instead of the general tax rate of 25.0%. Because uniQure is loss-making it has not currently made any applicationto the tax authorities for such an agreement, but intends to do so when it reaches profitability. uniQure has recognized the full amount of its losses in the year in which they were incurred. As noted above, these losses are available for usewithin nine years of being incurred. The total amount of tax losses carried forward was €130,877,000 as of December 31, 2013 (2012: €106,274,000). The expiration dates of these losses, is summarized in the following table. In the year ended December 31, 2013, the amount of unused tax lossesthat expired was €56,000 (2012: €nil and 2011: €644,000).26. Loss per ShareBasic Loss per Share Basic loss per share is calculated by dividing the profit attributable to equity holders of the Company by the weighted average number of sharesoutstanding during the periodDiluted Loss per Share Diluted loss per share is calculated by adjusting the weighted average number of ordinary shares outstanding to assume conversion of all dilutivepotential ordinary shares. Due to the fact that the Company is loss making, all potential ordinary shares had an antidilutive effect, if converted, and thushave been excluded from the computation of loss per share.27. Dividends per Share The Company did not declare dividends for the years ended December 31, 2013, 2012 and 2011.F-48(€ in thousands) 2014 2015 2016 2017 2018 2019 2020 2021 2022 Loss expiring 1,336 1,838 3,310 35,633 16,735 18,359 16,559 12,448 24,659 For the years endedDecember 31, 2011 2012 2013 (€ in thousands, except for per sharedata) Loss attributable to equity holders of the Company (17,300) (14,716) (26,820)Weighted average number of ordinary shares outstanding ('000) 4,709 8,637 10,796 Basic loss per share (3.65) (1.70) (2.48)Table of Contents28. Commitments and Contingent LiabilitiesRoyalties and Milestones In the course of its business uniQure enters as a licensee into contracts with other parties with regard to the development and marketing of itspipeline products. Among other payment obligations, is obligated to pay royalties to the licensors based on future sales levels and milestone paymentswhenever defined milestones are met. As both future sales levels and the timing and achievement of milestones are uncertain, the financial effect of theseagreements cannot be estimated reliably.Operating Lease Commitments uniQure leases various office space and laboratory space under operating lease agreements. The Company leases its headquarters facilities under anagreement between uniQure and AMC, represented by BDDA and Amsterdam Vector Productions B.V. ("AVP"), both subsidiaries of AMC (SecondRental Agreement) in respect of facilities located at Meibergdreef 61 Amsterdam, from October 1, 2005 until September 30, 2016, and an agreement forthe lease of facilities at Meibergdreef 57, Amsterdam, from July 1, 2006 until September 30, 2016. The aggregate annual lease payments amount to€542,000. The lease expenditure charged to the income statement for operating leases amounts to €542,000 in the year ended December 31, 2013 (2012:€542,000). The future aggregate minimum lease payments under non-cancellable operating leases are as follows: On July 24, 2013 uniQure entered into an agreement for the lease of facilities at 113 Hartwell Avenue, Lexington, Massachusetts, United Statesfrom November 5, 2013 until November 5, 2023. uniQure has an option to extend the lease for up to an additional 10 years. The aggregate annual leasepayments for the period to November 5, 2023 amount to $18,937,000 (€13,756,000), including an initial rent-free period of seven months from thecommencement of the lease which was effective at November 5, 2013. The lease payments under an operating lease will be recognized as an expense on a straight line basis over the full duration of the lease, (for a totalof $7,259,000 (€5,273,000)) taking into account the Lease Incentives as received from the landlord; This resulted in a monthly expense of $91,950(€66,795); for the period ending December 31, 2013 the company accounted for an related expense of $183,900 (€134,416). As of December 31, 2013the Company recorded a deferred rent of €680,000 ($936,000).F-49 For the years ended December31, 2011 2012 2013 No later than 1 year 435 542 1,243 Later than 1 year and no later than 5 years 1,632 1,627 6,053 Later than 5 years — 7,927 2,067 2,169 15,223 Table of ContentsResearch and Development Commitments uniQure has entered into research and development commitments in relation to uniQure's product pipeline. The future aggregate minimumpayments under these commitments are as follows:Grant Commitments From October 1, 2000 until May 31, 2005, AMT received a grant called a "Technisch ontwikkelingskrediet" (TOK) (or technical developmentloan) from the Dutch government. This TOK grant includes a repayment clause in the event the Company generates revenues from the related project.AMT received total grants of €3,605,000 relating to eligible project costs in the grant period. The grant amount received bears interest of 5.7% perannum and must be repaid in the period January 1, 2008 through December 31, 2017 as a percentage of revenues which are derived from the sale ofGlybera. If future royalty payments are not sufficient to repay the grant on or prior to December 3, 2017, or if there are no revenues generated, theremaining balance will be forgiven. Repayment obligations continue to apply if the product is not commercialized or transferred to others. The totalamount of the liability at December 31, 2013 was €5,508,000 (2012: € 5,979,000), comprising the original total amount of the grant together withaccrued interest. The Company has not recorded any liability to repay amounts in respect of this grant within these financial statements. The Companyhas commenced repayments of the TOK and associated interest from the commercialization proceeds of Glybera arising from the agreement with Chiesi.During the period ending December 31, 2013 the Company recognized an amount of €800,000 as a charge in the consolidated statement ofcomprehensive income within Costs of goods sold. This amount was paid to the Dutch Government in September 2013 and was calculated as 40% ofthe upfront amount received specifically related to Glybera. Historically, the Company also received a "Technisch ontwikkelingsproject" (TOP) (or technical development project) grant from the Dutchgovernment amounting to €130,000 on a project that was terminated. If the Company realizes income from the sale of assets developed under that grant,repayment clauses will apply. The Company has not recorded any liability to repay amounts in respect of this grant within these financial statements. On January 5, 2010, the Company was awarded an investment credit (innovatiekrediet) from the Dutch government (Ministry of Economic Affairs—Agentschap.nl) in respect of the Company's program for Duchenne Muscular Dystrophy. The credit covers 35% of the costs incurred in respect ofthe program up to a maximum of €4,000,000. The credit includes a repayment clause dependent on the technical success of this program (which isexpected to be demonstrated if the product can be successfully commercialized). The credit is interest-bearing at a rate of 11.4% per annum. To date, theCompany has received €729,000 under this investment credit, and as of December 31, 2013, the total amount of the liability was € 1,063,000,representing the amount of the original advance together with accrued interest (2012: €956,000). The credit was to be repaid after the funded part of theprogram was completed in 2013, out of a percentage of revenues derived from the sales resulting from the Company's Duchenne Muscular Dystrophyprogram. The assets which are financed by means of the investment credit are subject to a right of pledge for the benefit of the Dutch Ministry ofEconomicF-50 For the years endedDecember 31, 2011 2012 2013 No later than 1 year 343 277 327 Later than 1 year and no later than 5 years — — — Later than 5 years — — — 343 277 327 Table of ContentsAffairs. The project has been terminated following failure to achieve the scientific goals and the Company does not anticipate that any amounts will berealized from this project and consequently there will not be any obligation to repay any of these amounts.Other contingent liabilities On October 22, 2013, the Company received a demand letter from Extera Partners, a consulting firm based in Boston, Massachusetts, regardingcertain fees alleged to be owed by the Company in respect of consulting services provided in connection with the Company's collaboration agreementswith Chiesi, under an engagement which expired on December 31, 2012. The total amount claimed by Extera Partners for present and future feesallegedly due as a result of the Company's collaboration agreements with Chiesi, which were entered into in the second quarter of 2013, is said to be inthe order to €7,000,000 to €8,000,000; the engagement letter with Extera contained a cap limiting the maximum payment to €5,000,000. The Company has reviewed the demand with counsel and believes that the claim is without merit, and consequently it is not expected to havefinancial consequences for the Company. (see also Note 31)29. Related-Party Transactions In the period ending December 31, 2013 and 2012, the Management Board received regular salaries and contributions to post-employmentschemes. Additionally, selected members of the Supervisory Board received compensation for their services in the form of cash compensation. Funds affiliated with Forbion Capital partners have a material interest in the Company. In addition, Professor Sander van Deventer and Mr. SanderSlootweg, who were appointed as members of the Supervisory Board of uniQure on April 5, 2012, are each partners of Forbion. Based on theinformation above, Forbion is a related party of uniQure. Funds affiliated with Gilde Healthcare have a material interest in the Company. In addition, Mr. Edwin de Graaf, who was appointed as a memberof the Supervisory Board of uniQure on April 5, 2012, and resigned on November 8, 2013, is a partner of Gilde Healthcare Partners. Based on theinformation above, Gilde Healthcare is a related party of uniQure. Funds affiliated with Grupo Netco and Lupus Alpha also have material interests in the company. Chiesi became a related party following the thecommercial and investment agreements concluded with the Company on June 30, 2013, and Coller Capital became a related party following theconversion of the convertible loan in July 2013.Transactions The related parties identified above participated in the following transactions during the periods ended December 31, 2013 and December 31, 2012. The 2009 convertible loan from Forbion accrued interest of 5% (a finance charge of €70,000), during the period from January 1, 2012 until itsconversion on April 5, 2012 The 2012 convertible loan from Forbion, Gilde, Lupus Alpha, Grupo Netco and affiliates, and Coller Capital, as amendedin March 2013, generated in the period ending December 31, 2013 a combined funding of €11,998,000. This loan accrued interest of 8% up until thedate of conversion in July 2013 (plus an amount up to the interest payment date), amounting to a total interest amount payable of €434,000. In the period ending December 31, 2013, the Company received various payments from Chiesi comprising a subscription for ordinary C shares of€14,000,000 and up-front commercial payments of €16,875,000 (after deduction of Italian withholding tax). In addition, the Company received fundsfrom Chiesi for issued invoices totaling €1,222,000.F-51Table of Contents As of December 31, 2013 the Company had a receivable outstanding with Chiesi for €1,402,00030. Key Management Compensation The aggregate remuneration of the Supervisory Directors amounted to €400,000 in 2013 (2012: €255,000; 2011: €174,000) as follows: The table below sets out a breakdown in the remuneration for the year ended December 31, 2013 of the members of the Management Board andSenior Management:YEAR ENDEDDECEMBER 31, 2013 SALARY BONUS SHARE-BASEDPAYMENTS(1) PENSIONS ADVISOR'SFEE 2013TOTAL 2012TOTAL 2011TOTAL (€ in thousands) FerdinandVerdonck — — 244 — 37 281 43 37 Sander vanDeventer(2) — — — — — — 8 56 Joseph Feczko — — 30 — 28 58 69 27 Edwin de Graaf(3) — — — — — — — Francois Meyer — — 30 — 28 58 69 27 SanderSlootweg(3) — — — — — — — — Philippe VanHolle(4) — — (40) — — (40) 66 27 PaulaSoteropoulos(5) — — 32 — 11 43 — — Robert Coffin(6) — — — — — — — — Total — — 296 — 104 400 255 174 (1)The share-based payment reflects the value of equity-settled share options expensed during the year, as required by IFRS 2. (2)Following the combination of uniQure and AMT on April 5, 2012, Professor van Deventer has received no remuneration. (3)Appointed April 5, 2012; Messrs de Graaf and Slootweg receive no remuneration. Mr de Graaf resigned on November 8, 2013 (4)Resigned January 1, 2013 (5)Appointed June 5, 2013 (6)Appointed November 18, 2013 and resigned December 10, 2013YEAR ENDEDDECEMBER 31, 2013 SHORTTERMEMPLOYEEBENEFITS(1) SHARE-BASEDPAYMENTS(2) POST-EMPLOYMENTBENEFITS OTHERLONGTERMBENEFITS TERMINATIONBENEFITS TOTAL (€ in thousands) Jörn Aldag 480 266 41 — — 787 Piers Morgan 267 111 19 — — 397 Total forManagementDirectors 747 377 60 — — 1,184 SeniorManagement 1,101 873 109 — — 2,083 F-52Management 1,101 873 109 — — 2,083 Total 1,848 1,250 169 — — 3,267 (1)In addition the Company recognized in 2013 an expense of €55,000 (2012: €20,000, 2011: nil) in respect to the Dutch Crisis Taxlevy.Table of Contents The total remuneration (excluding share-based payments) paid to or for the benefit of members of the Management Board and Senior Managementin 2013 amounted to approximately €2,017,000 (2012: €1,517,000).The table below sets out a breakdown in the remuneration for the year ended December 31, 2012 of the members of the Management Board and SeniorManagement: The table below sets out a breakdown in the remuneration in 2011 of the members of the Management Board and Senior Management:F-53(2)The share-based payment reflects the value of options expensed during the year together with a charge for the period to April 5,2012 in respect of options granted by AMT.YEAR ENDEDDECEMBER 31, 2012 SHORTTERMEMPLOYEEBENEFITS SHARE-BASEDPAYMENTS(1) POST-EMPLOYMENTBENEFITS OTHERLONGTERMBENEFITS TERMINATIONBENEFITS TOTAL (€ in thousands) Jörn Aldag 437 359 64 — — 860 Piers Morgan 258 150 28 — — 436 Total forManagementDirectors 695 509 92 — — 1,296 SeniorManagement 689 452 41 — — 1,182 Total 1,384 961 133 — — 2,478 (1)The share-based payment reflects the value of options expensed during the year.DECEMBER 31, 2011 SHORTTERMEMPLOYEEBENEFITS SHARE-BASEDPAYMENTS(1) POST-EMPLOYMENTBENEFITS OTHERLONGTERMBENEFITS TERMINATIONBENEFITS TOTAL (€ in thousands) Jörn Aldag 390 267 57 — — 714 Piers Morgan 227 186 17 — — 430 Total forManagementDirectors 617 453 74 — — 1,144 SeniorManagement 403 271 41 — — 715 Total 1,020 724 115 — — 1,859 (1)The share-based payment reflects the value of options expensed during the year.Table of ContentsShares and Share Options Held by Key ManagementOptionsDepositary receiptsReceivables and Payables Key Management These receivables relate to certain wage tax liabilities settled by AMT on behalf of senior management in connection with purchases of AMTdepositary receipts in 2007; these amounts are repayable to uniQure on sale of the related depositary receipts or on the respective employee ceasing to beemployed by the Company.31. Litigation and Arbitration On December 11, 2013, the Company received a formal request for arbitration from Extera Partners, a consulting firm based in Cambridge,Massachusetts, alleging a fee to be due in respect of consulting services provided to the Company in connection with a partnering transaction. Therequest for arbitration was received by the International Court of Arbitration at the International Chamber of Commerce on December 12, 2013, whichrepresents the start date of the arbitration. The amount claimed is $100,000 plus 2.5% of all proceeds, including equity investments, the Companyreceives from Chiesi pursuant to its collaboration agreements entered into in the second quarter of 2013. The Company's engagement letter with ExteraPartners contains a cap limiting the maximum payment to €5.0 million. On December 23, 2013 proceedings under the International Court of ArbitrationformallyF-54 NUMBER OFOPTIONSATJANUARY 1,2013 OPTIONSGRANTEDDURINGTHE YEAR OPTIONSLAPSED/EXPIREDDURINGTHE YEAR NUMBER OFOPTIONS ATDECEMBER 31,2013 Jörn Aldag 337,565 — — 337,565 Piers Morgan 140,652 — — 140,652 Senior Management 562,608 252,652 (140,652) 674,608 Total 1,040,825 252,652 (140,652) 1,115,825 NUMBER OFDEPOSITARYRECEIPTS FORSHARES(1) Jörn Aldag 39,389 Piers Morgan 27,805 Senior Management 16,254 Total 83,448 (1)These Depositary Receipts represent ordinary shares. December 31, 2012 2013 Receivables from Senior Management 26 23 Total 26 23 Table of Contentscommenced. The Company has reviewed this claim with counsel and believes that the claim is without merit. The Company intends to vigorouslydefend against it.32. Events after the balance sheet date Since December 31, 2013 uniQure has entered into certain material agreements. These agreements do not have a material impact on the results orfinancial position of uniQure for the period covered by these consolidated financial statements, but are expected to have a material impact in futurefinancial periods. In January 2014, the Company entered into a collaboration and license agreement with 4D for the discovery and optimization of next-generationAAV vectors. Under this agreement, the Company has an exclusive license to 4D's existing and certain future know-how and other intellectual propertyfor the delivery of AAV vectors to CNS or liver cells for the diagnosis, treatment, palliation or prevention of all diseases or medical conditions. Underthis collaboration, the 4D team, including Dr. David Schaffer, 4D's co-founder and Professor of Chemical and Biomolecular Engineering at theUniversity of California, Berkeley, will establish a laboratory, which the Company will fund, at a cost of approximately $3.0 million in aggregate overthe next three years, to identify next generation AAV vectors. The Company is also required to make payments for pre-clinical, clinical and regulatorymilestones under the collaboration as well as to pay single-digit royalties. In addition, the Company has granted options to purchase an aggregate of609,744 ordinary shares in connection with this collaboration, and will recognize resulting share-based payment expense over the next three years. Tothe extent that the collaboration is successful, the Company may also incur additional third party costs in developing any product candidates and also inpreparing, filing and prosecuting additional patent applications On January 20, 2014, the shareholders of the Company approved, and on January 21, 2014 the supervisory board of the Company confirmed, a 5-for-1 consolidation of shares, which had the effect of a reverse share split, that became effective on January 31, 2014. All share, per-share and related information presented in these consolidated financial statements and accompanying footnotes has been retroactivelyadjusted, where applicable, to reflect the impact of the reverse share split. On February 5, 2014 the Company successfully completed its initial public offering, placing 5,400,000 shares at $17 per share, raising total grossproceeds of $91.8 million (€67.3 million) and net proceeds of $85.4 million (€62.6 million) after commissions but before expenses. On such date, theCompany also reclassified its class A, B and C ordinary shares as ordinary shares. No other events occurred after the balance sheet date that would have a material impact on the result or financial position uniQure.F-55Exhibit 1.1 True copy of deed executed on 10 February 2014 NOTE ABOUT TRANSLATION: This document is an English translation of a document prepared in Dutch. In preparing this document, an attempt has been made to translate as literally aspossible without jeopardizing the overall continuity of the text. Inevitably, however, differences may occur in translation and if they do, the Dutch text willgovern by law. In this translation, Dutch legal concepts are expressed in English terms and not in their original Dutch terms. The concepts concerned may not be identical toconcepts described by the English terms as such terms may be understood under the laws of other jurisdictions. CONVERSION AND AMENDMENT OF THE ARTICLES OF ASSOCIATION UNIQURE Today, the tenth day of February two thousand fourteen appeared before me, Cornelia Holdinga, civil law notary in Amsterdam, the Netherlands: mr. Hajo Bart Hendrik Kraak, office address Holdinga Matthijssen Kraak, Diepenbrockstraat 54, 1077 WB Amsterdam, born in TandjungPandan, Indonesia on the twenty-fifth day of March nineteen hundred fifty. The person appearing has declared that: · the extraordinary general meeting of uniQure B.V., a private company with limited liability, having its official seat at Amsterdam, the Netherlands,with address at Meibergdreef 61, 1105 BA Amsterdam Zuidoost, the Netherlands, registered at the Dutch Trade Register with number 54385229,hereinafter referred to as: the “Company”, held on the twentieth day of January two thousand fourteen, in conjunction with the extraordinary generalmeeting of the Company held on the twenty-seventh day of January two thousand fourteen, resolved to convert the Company into a public companywith limited liability (“naamloze vennootschap”) and to amend the articles of association of the Company in its entirety as stated hereinafter as wellas to authorize the person appearing to execute this deed of conversion and amendment of the articles of association, as appears from a copy of anextract from the minutes of the general meetings which will be attached to this deed as Annex I and II; · the articles of association of the company were for the last time amended by deed executed on the thirty-first day of January two thousand fourteenthe before me, civil law notary. Subsequently the appearer declared to convert the Company into a public company with limited liability (“naamloze vennootschap”) and to amend thearticles of association of the Company in its entirety to read as follows: 1 1. DEFINITIONS. In the articles of association the following terms shall have the meaning as defined below: · Annual Accounts: the annual accounts referred to in section 2:361 DCC; · Annual Report: the annual report referred to in section 2:391 DCC; · Annual Statement of Accounts: the Annual Accounts and, if applicable, the Annual Report as well as the additional informationreferred to in section 2:392 DCC; · Company: the public limited company which organisation is laid down in these articles of association; · DCC: the Dutch Civil Code; · General Meeting: the corporate body that consists of Shareholders entitled to vote and all other persons entitled to vote / the meeting inwhich Shareholders and all other persons entitled to attend general meetings assemble; · Management Board: the corporate body entrusted with the management of the Company; · Managing Director: a member of the Management Board; · Meeting Rights: the right to, either in person or by proxy authorised in writing, attend the General Meeting and to address such meeting; · Persons entitled to attend General Meetings: Shareholders as well as holders of a right of use and enjoyment (vruchtgebruik) andholders of a right of pledge with Meeting Rights; · Persons entitled to vote: Shareholders with voting rights as well as holders of a right of use and enjoyment (vruchtgebruik) and holdersof a right of pledge with voting rights; · Share: a share in the share capital of the Company; · Shareholder: a holder of a Share; · Subsidiary: a subsidiary as referred to in section 2:24a DCC; · Supervisory Board: the corporate body entrusted with the statutory supervision of the policies of the Management Board and the otherresponsibilities imposed on the supervisory board by the law and these articles of association; · Supervisory Director: a member of the Supervisory Board. 2 2. NAME. CORPORATE SEAT. 2.1. The name of the Company is: uniQure N.V. Its corporate seat is in Amsterdam, the Netherlands, and it may establish branch offices elsewhere. 2.2. Objects. The objects of the Company are: (a) to research, develop, produce and commercialise products, services and technology in the (bio-)pharmaceutical sphere; (b) to incorporate, participate in, conduct the management of and take any other financial interest in other companies and enterprises; (c) to render administrative, technical, financial, economic or managerial services to other companies, persons or enterprises; (d) to acquire, dispose of manage and exploit real and personal property, including patents, marks, licenses, permits and other intellectualproperty rights; (e) to borrow and/or lend moneys, act as surety or guarantor in any other manner, and bind itself jointly and severally or otherwise inaddition to or on behalf of others, the foregoing, whether or not in collaboration with third parties, and inclusive of the performance and promotion of all activities which directly andindirectly relate to those objects, all this in the broadest sense. 3. SHARE STRUCTURE. 3.1. Authorised share capital 3.1.1. The authorised share capital of the Company amounts to three million euro (EUR 3.000.000,00) and is divided into sixty million(60,000,000) shares, each with a nominal value of five cent (€ 0.05). 3.1.2. The Shares shall be in registered form and shall be consecutively numbered from 1 onwards. 3.1.3. No share certificates shall be issued. 3.2. Issue of Shares. 3.2.1. Shares shall be issued pursuant to a resolution of the Management Board, subject to the approval of the Supervisory Board, if byresolution of the General Meeting the Management Board has been authorised for a specific period not exceeding five (5) years to issueShares. The resolution granting the aforesaid authorisation must determine the number and class of the Shares that may be issued. Theauthorisation may from time to time be extended for a period not exceeding five (5) 3 years. Unless otherwise stipulated at its grant, the authorisation cannot be withdrawn. 3.2.2. If and insofar as an authorisation as referred to in article 3.2.1 is not in force, the General Meeting shall have the power, upon theproposal of the Management Board - which proposal must be approved by the Supervisory Board - to resolve to issue Shares. 3.2.3. Article 3.2.1 and 3.2.2 shall equally apply to a grant of rights to subscribe for Shares, but shall not apply to an issue of Shares to aperson who exercises a previously acquired right to subscribe for Shares. 3.2.4. Save for the provisions of section 2:80 DCC, the issue price may not be below nominal value of the Shares. 3.2.5. Shares shall be issued by deed in accordance with the provisions of sections 2:86c and 2:96 DCC. 3.3. Payment for Shares. 3.3.1. Shares may only be issued against payment in full of the amount at which such Shares are issued and with due observance of theprovisions of sections 2:80a and 2:80b DCC. 3.3.2. Payment must be made in cash, unless an alternative contribution has been agreed. Payment other than in cash is made with dueobservance of the provisions of section 2:94b DCC. 3.3.3. Payment in cash may be made in a foreign currency if the Company agrees to this. In that case, the payment obligation shall be fulfilledfor the amount up to which the amount paid up can be freely exchanged into euro. This rate of exchange shall be determined by the rate ofexchange prevailing on the day of payment or, after application of the provisions of the next sentence, on the day referred to there. TheCompany may demand payment at the rate of exchange prevailing on a specific day within two (2) months prior to the last day on whichpayment must have been made, provided that the Shares shall be included on the official list of any stock exchange immediately followingthe issue. 3.3.4. The Company may grant loans for the purpose of a subscription for or an acquisition of Shares in its share capital subject to anyapplicable statutory provisions. 3.3.5. The Management Board may perform legal acts as referred to in section 2:94 DCC without the prior approval of the General Meeting. 3.4. Pre-emptive rights. 3.4.1. Upon the issue of Shares, each Shareholder shall have a pre-emptive right to acquire such newly issued Shares in proportion to theaggregate 4 amount of his Shares, it being understood that this pre-emptive right shall not apply to: (a) any issue of Shares to employees of the Company or employees of a group Company; (b) Shares which are issued against payment in kind. 3.4.2. Pre-emptive rights may be limited or excluded by resolution of the General Meeting upon proposal of the Management Board. TheManagement Board, subject to approval of the Supervisory Board, shall have the power to resolve upon the limitation or exclusion of thepre-emptive right, if and to the extent the Management Board has been designated by the General Meeting. Such designation shall only bevalid for a specific period of not more than five (5) years and may from time to time be extended with a period of not more than five(5) years. Unless provided otherwise in the designation, the designation cannot be cancelled. A resolution of the General Meeting to limit or exclude the pre-emptive rights as well as a resolution to designate the Management Board asreferred to in this article 3.4.2 requires a two thirds majority of the votes cast if less than half the issued share capital is represented at ameeting. 3.4.3. Without prejudice to section 2:96a DCC, the General Meeting or the Management Board, as the case may be, shall, when adopting aresolution to issue Shares, determine the manner in which and the period within which such pre-emptive rights may be exercised. 3.4.4. The Company shall announce the issue with pre-emptive rights and the period within which such rights can be exercised in such manneras shall be prescribed by applicable law and applicable stock exchange regulations, including, but not limited to, an announcementpublished by electronic means of communication. 3.4.5. This article 3.4 shall equally apply to a grant of rights to subscribe for Shares, but shall not apply to an issue of Shares to a person whoexercises a previously acquired right to subscribe for Shares. 3.5. Depositary receipts for shares The Company is not authorised to cooperate in the issue of depositary receipts for Shares. 4. OWN SHARES. CAPITAL REDUCTION. 4.1. Acquisition of Shares. 4.1.1. Subject to authorisation by the General Meeting, the Management Board, subject to the approval of the Supervisory Board and with dueobservance of the applicable relevant statutory provisions, may resolve on the acquisition by the Company of fully paid-up Shares. Such 5 authorisation shall only be valid for a specific period of not more than eighteen (18) months and may from time to time be extended with aperiod of not more than eighteen (18) months. Acquisition by the Company of non-paid up Shares is null and void. 4.1.2. The authorisation of the General Meeting as referred to in article 4.1.1 shall not be required if the Company acquires fully paid-up Sharesfor the purpose of transferring such Shares, by virtue of an applicable employee stock purchase plan, to persons employed by theCompany or by a group Company, provided such Shares are quoted on the official list of any stock exchange. 4.2. Capital reduction. 4.2.1. With due observance of the statutory requirements the General Meeting may resolve to reduce the issued share capital by (i) reducing thenominal value of Shares by amending the articles of association, or (ii) cancelling: (a) Shares in its own share capital which the Company holds itself in the Company’s share capital, or (b) all issued Shares against repayment of the amount paid-up on those Shares; 4.2.2. Partial repayment on Shares pursuant to a resolution to reduce their nominal value will be made proportionally. 5. TRANSFER. 5.1. Form of transfer of Shares. 5.1.1. The transfer of a Share shall require a deed executed for that purpose and, save in the event that the Company itself is a party to thetransaction, written acknowledgement by the Company of the transfer. The acknowledgement is to be made either in the transfer deed, orby a dated statement endorsed upon the transfer deed or upon a copy of or extract from that deed certified by a notary (notaris) or bailiff(deurwaarder), or in the manner as referred to in article 5.1.2. Service of notice of the transfer deed or of the aforesaid copy or extractupon the Company shall be the equivalent of acknowledgement as stated in this paragraph. 5.1.2. The preceding paragraph shall apply mutatis mutandis to the transfer of any limited right to a Share, provided that a pledge may also becreated without acknowledgement by or service of notice upon the Company and that section 3:239 DCC applies, in which caseacknowledgement by or service of notice upon the Company shall replace the announcement referred to section 3:239, subsection 3 DCC. 6 6. REGISTERS. PLEDGE. USE AND ENJOYMENT (VRUCHTGEBRUIK). 6.1. Shareholders register. 6.1.1. With due observance of the applicable statutory provisions in respect of registered shares, a shareholders register shall be kept by or onbehalf of the Company, which register shall be regularly updated and, at the discretion of the Management Board, may, in whole or inpart, be kept in more than one copy and at more than one address. Part of the shareholders register may be kept abroad in order to complywith applicable foreign statutory provisions or applicable listing rules. 6.1.2. Each Shareholder’s name, his address and such further information as required by law or considered appropriate by the ManagementBoard, shall be recorded in the shareholders register. 6.1.3. The form and the contents of the shareholders register shall be determined by the Management Board with due observance of the articles6.1.1 and 6.1.2. 6.1.4. Upon his request a Shareholder shall be provided free of charge with written evidence of the contents of the shareholders register withregard to the Shares registered in his name, and the statement so issued may be validly signed on behalf of the Company by a person to bedesignated for that purpose by the Management Board. 6.1.5. The provisions of the articles 6.1.3 and article 6.1.4 shall equally apply to persons who hold a right of use and enjoyment(vruchtgebruik) or a right of pledge on one or more Shares. 6.2. Joint holding. If through any cause whatsoever one or more Shares are jointly held by two or more persons, such persons may jointly exercise the rights arisingfrom those Shares, provided that these persons be represented for that purpose by one from their midst or by a third party authorised by them forthat purpose by a written power of attorney. The Management Board may, whether or not subject to certain conditions, grant an exemption for the provision of the previous sentence. 6.3. Right of pledge. 6.3.1. Shares may be encumbered with a pledge as security for a debt. 6.3.2. If a Share is encumbered with a pledge, the voting right attached to that Share shall vest in the Shareholder, unless at the creation of thepledge the voting right has been granted to the pledgee. 6.3.3. Shareholders who as a result of a right of pledge do not have voting rights, have Meeting Rights. 6.4. Right of use and enjoyment (vruchtgebruik). 6.4.1. Shares may be encumbered with a right of use and enjoyment. 7 6.4.2. If a Share is encumbered with a right of use and enjoyment, the voting right attached to that Share shall vest in the Shareholder, unless atthe creation of the right of use and enjoyment the voting right has been granted to the holder of the right of use and enjoyment. 6.4.3. Shareholders who as a result of a right of use and enjoyment do not have voting rights, have Meeting Rights. 7. MANAGEMENT. SUPERVISION. 7.1. Management. Supervision of management. 7.1.1. The Company shall be managed by a Management Board under the supervision of a Supervisory Board. The Supervisory Board shalldetermine the number of Managing Directors and the number of Supervisory Directors. 7.1.2. Each Managing Director is obliged vis-a-vis the Company to perform his duties in a proper manner. These duties include all managingduties that have not been allocated to one or more other Managing Directors by law or by these articles of association. In fulfilling theirtasks, the Managing Directors must be guided by the interests of the Company and its business. Each Managing Director is responsiblefor the Company’s general course of affairs. 7.1.3. Supervision of the policies of the Management Board and of the general course of the Company’s affairs and its business enterprise shallbe carried out by the Supervisory Board. It shall support the Management Board with advice. In fulfilling their duties the SupervisoryDirectors shall serve the interests of the Company and its business enterprise. The Management Board shall in due time provide theSupervisory Board with the information it needs to carry out its duties. 7.2. Management Board: appointment, suspension and dismissal. 7.2.1. Managing Directors shall be appointed by the General Meeting. 7.2.2. If a Managing Director is to be appointed, the Supervisory Board shall make a binding nomination of at least the number of personsprescribed by law. The General Meeting may at all times overrule the binding nomination by a resolution adopted by at least a two thirds majority of the votescast, provided such majority represents more than half the issued share capital. If the General Meeting overruled the binding nomination,the Supervisory Board shall make a new nomination. The nomination shall be included in the notice of the General Meeting at which the appointment shall be considered. If a nomination has not been made or has not been made in due time, this shall be stated in the notice and the General Meeting shall be freeto appoint a Managing Director at its discretion. 8 7.2.3. A resolution to appoint a Managing Director that was not nominated by the Supervisory Board may only be adopted by at least a twothirds majority of the votes cast, provided such majority represents more than half the issued share capital. 7.2.4. Managing Directors are appointed for a maximum term of four (4) years, provided that, unless a Managing Director resigns earlier, histerm of appointment shall end at the close of the annual General Meeting to be held in the fourth year after the year of his appointment. A Managing Director may be reappointed with due observance of the preceding sentence. The Supervisory Board shall draw up aretirement schedule for the Managing Directors. 7.2.5. The General Meeting shall at all times be entitled to suspend or dismiss a Managing Director. The General Meeting may only adopt aresolution to suspend or dismiss a Managing Director by at least a two thirds majority of the votes cast, provided such majorityrepresents more than half the issued share capital, unless the proposal was made by the Supervisory Board in which case a simplemajority of the votes cast is sufficient. A second General Meeting as referred to in section 2:120, subsection 3 DCC may not be convened. The Supervisory Board shall also at all times be entitled to suspend (but not to dismiss) a Managing Director. Within three (3) monthsafter a suspension of a Managing Director has taken effect, a General Meeting shall be held, in which meeting a resolution must beadopted to either terminate or extend the suspension for a maximum period of another three (3) months. The suspended Managing Directorshall be given the opportunity to account for his actions at that meeting. 7.2.6. If neither such resolution is adopted nor the General Meeting has resolved to dismiss the Managing Director, the suspension shallterminate after the period of suspension has expired. In the event that one or more Managing Directors are absent or prevented from acting, the remaining Managing Director(s) shalltemporarily be in charge of the management, without prejudice to the right of the Supervisory Board to replace such Managing Director fora temporary Managing Director. In the event that all Managing Directors are, or the sole Managing Director is, absent or prevented from acting, the Supervisory Boardshall temporarily be in charge of the management; the Supervisory Board shall be authorised to designate one or more temporaryManaging Directors. 7.2.7. In the event that all Managing Directors are, or the sole Managing Director is, absent or prevented from acting, the Supervisory Boardshall as soon as possible take the necessary measures to make a definitive 9 arrangement. The term prevented from acting means: (i) suspension; (ii) illness; (iii) inaccessibility, in the events referred to under sub (ii) and (iii) without the possibility of contact between the Managing Director concerned and theCompany for a period of five (5) days, unless the Supervisory Board sets a different term in the case at hand. 7.3. Management Board: remuneration. 7.3.1. The Company must establish a policy in respect of the remuneration of the Management Board. The remuneration policy is adopted bythe General Meeting upon the proposal of the Supervisory Board. 7.3.2. The remuneration of the Management Board shall be determined by the Supervisory Board with due observance of the remunerationpolicy adopted by the General Meeting. 7.3.3. A proposal with respect to remuneration schemes in the form of Shares or rights to Shares is submitted by the Supervisory Board to theGeneral Meeting for its approval. This proposal must set out at least the maximum number of Shares or rights to Shares to be granted to members of the ManagementBoard and the criteria for granting or amendment. 7.4. Management Board: adoption of resolutions. 7.4.1. If there is more than one Managing Director, the Supervisory Board can appoint one (1) of the Managing Directors as chairman of theManagement Board and grant such chairman a title. 7.4.2. With due observance of these articles of association, the Management Board may adopt written rules governing its internal proceedingsand providing for the division of their duties among themselves. The adoption and amendment of the rules governing the Management Board shall be subject to the approval of the Supervisory Boardwithout prejudice of the rights of initiative of the Supervisory Board provided for therein. 7.4.3. The Management Board shall meet whenever a Managing Director so requires. The Management Board shall adopt its resolutions by asimple majority of the votes cast. In a tie vote the chairman of the Management Board shall have a casting vote. 10 7.4.4. At a meeting of the Management Board, a Managing Director may only be represented by another Managing Director holding a writtenproxy. 7.4.5. If a Managing Director has a direct or indirect personal conflict of interest with the Company, he shall not participate in the deliberationsand the decision-making process concerned in the Management Board. If as a result thereof no resolution of the Management Board can beadopted, the resolution may be adopted by the Supervisory Board. 7.4.6. The Management Board may also adopt resolutions without holding a meeting, provided such resolutions are adopted in writing or in areproducible manner by electronic means of communication and all the Managing Directors entitled to vote have consented to adopting theresolution outside a meeting. 7.4.7. Articles 7.4.3 and 7.4.5 shall equally apply to adoption by the Management Board of resolutions without holding a meeting. 7.4.8. Without prejudice to any other applicable provisions of these articles of association, the Management Board shall require the approval ofthe General Meeting for resolutions of the Management Board regarding a significant change in the identity or nature of the Company orthe enterprise, including in any event: (a) the transfer of the enterprise or practically the entire enterprise to a third party; (b) the entry into or termination of any long-lasting cooperation by the Company or a Subsidiary with any other legal person orcompany or as a fully liable general partner of a limited partnership or a general partnership, provided that such cooperation or thetermination thereof is of significant importance to the Company; and (c) the acquisition or disposal of a participating interest in the capital of a Company with a value of at least one-third of the sum ofthe assets according to the consolidated balance sheet with explanatory notes thereto according to the last adopted Annual Accountsof the Company, by the Company or a Subsidiary. 7.4.9. Without prejudice to any other applicable provisions of these articles of association, the Management Board resolutions relating to any ofthe following matters shall be subject to the approval of the Supervisory Board: (a) the sale or disposition of all, or an essential part of, the assets of the Company; (b) the issuance and acquisition of Shares and of debentures chargeable against the Company or chargeable against a limitedpartnership (commanditaire vennootschap), or a general partnership (vennootschap onder firma) of which the Company is thefully liable partner; 11 (c) the application for quotation, or withdrawal of quotation, of Shares or debt of the Company on any stock exchange; (d) the entry into or termination of any long-term, material cooperation by the Company or a Subsidiary of the Company with anotherlegal entity or partnership or as a fully liable general partner in a limited partnership or general partnership, if such cooperation ortermination is of significant importance to the Company; (e) the participation by the Company or a Subsidiary of the Company in the capital of another company in an amount equal to atleast one fourth of the issued capital plus the reserves of the Company, as reflected in the balance sheet with explanatory notes ofthe Company, as well as a material change to such participation; (f) investments requiring an amount equal to at least one fourth of the issued capital plus the reserves of the Company, as reflected inthe balance sheet with explanatory notes; (g) filing a petition for bankruptcy (faillissement) or for suspension of payments (surseance van betaling) by the Company; (h) the termination of a significant number of the employees of the Company or a Subsidiary simultaneously or within a short periodof time; (i) a significant change in the employment conditions of the employees of the Company or of a Subsidiary; and (j) a decrease in the issued capital of the Company. 7.4.10. The Supervisory Board may determine that a resolution that would be subjected to its approval pursuant to article 7.4.9 will not requiresuch approval if the amount involved does not exceed a value fixed by the Supervisory Board and notified to the Management Board inwriting. 7.4.11. The Supervisory Board may also require that additional actions than required under article 7.4.9 by the Management Board be subjectedto the approval of the Supervisory Board. Such actions must be clearly specified to the Management Board in writing. 7.4.12. The absence of approval of the Supervisory Board does not affect the authority of the Management Board or its members to represent theCompany in dealings with third parties. 7.5. Representation. 7.5.1. The Management Board, as well as two (2) Managing Directors acting jointly are authorised to represent the Company. 7.5.2. The Management Board may grant one or more persons, whether or not employed by the Company, the power to represent the Company 12 (procuratie) or grant the power to represent the Company on a continuing basis in a different manner. 7.6. Supervisory Board: appointment, suspension and dismissal. 7.6.1. Supervisory Directors shall be appointed by the General Meeting. 7.6.2. If a Supervisory Director is to be appointed, the Supervisory Board shall make a binding nomination. The General Meeting may at all times overrule the binding nomination by a resolution adopted by at least a two thirds majority of the votescast, provided such majority represents more than half of the issued share capital. If the General Meeting overruled the bindingnomination, the Supervisory Board shall make a new nomination. The nomination shall be included in the notice of the General Meeting at which the appointment shall be considered. 7.6.3. If a nomination has not been made or has not been made in due time, this shall be stated in the notice and the General Meeting shall befree to appoint a Supervisory Director at its discretion. 7.6.4. A resolution to appoint a Supervisory Director that was not nominated by the Supervisory Board, may only be adopted by at least a twothirds majority of the votes cast, provided such majority represents more than half of the issued share capital. 7.6.5. Supervisory Directors are appointed for a maximum term of three (3) years, provided that, unless a Supervisory Director resigns earlier,his term of appointment shall end at the close of the annual General Meeting that will be held in the third year after his appointment. A Supervisory Director may be reappointed for a term of not more than three (3) years at a time, with due observance of the previoussentence. A Supervisory Director may be a Supervisory Director for a period not longer than twelve (12) years, which period may or maynot be interrupted, unless the General Meeting resolves otherwise. The Supervisory Board shall draw up a resignation retirement schedulefor the members of the Supervisory Board. 7.6.6. The General Meeting shall at all times be entitled to suspend or dismiss a Supervisory Director. The General Meeting may only adopt aresolution to suspend or dismiss a Supervisory Director by at least a two thirds majority of the votes cast, provided such majorityrepresents more than half the issued share capital, unless the proposal was made by the Supervisory Board in which case a simplemajority of the votes cast is sufficient. A second General Meeting as referred to in section 2:120, subsection 3 DCC may not be convened. 13 7.6.7. In the event that one or more Supervisory Directors are absent or prevented from acting the remaining Supervisory Director(s) shalltemporarily be in charge of the supervision, without prejudice to the right of the General Meeting to replace such Supervisory Director for atemporary Supervisory Director. In the event that one or more Supervisory Directors are absent or prevented from acting, the remaining Supervisory Directors shall as soonas possible take the necessary measures to make a definitive arrangement. In the event that all Supervisory Directors are absent orprevented from acting, the Management Board shall as soon as possible take the necessary measures to make a definitive arrangement. The term prevented from acting means: (i) suspension; (ii) illness; (iii) inaccessibility, in the events referred to under sub (ii) and (iii) without the possibility of contact between the Supervisory Director concerned and theCompany for a period of five (5) days. 7.7. Supervisory Board: remuneration. The General Meeting shall determine the remuneration of Supervisory Directors. Supervisory Directors shall be reimbursed for their expenses. 7.8. Supervisory Board: adoption of resolutions. 7.8.1. If there is more than one (1) Supervisory Director, the Supervisory Board shall appoint one of its members as chairman. TheSupervisory Board may also appoint a secretary, whether or not from among its members. Furthermore, the Supervisory Board may appoint one or more of its members as delegate Supervisory Director to be in charge ofcommunicating with the Management Board on a regular basis. They shall report their findings to the Supervisory Board. The offices ofchairman of the Supervisory Board and delegate Supervisory Director are compatible. 7.8.2. With due observance of these articles of association, the Supervisory Board may adopt written rules governing its internal proceedings. 7.8.3. The Supervisory Board shall meet whenever a Supervisory Director so requires. The Supervisory Board shall adopt its resolutions by asimple majority of the votes cast. In a tie vote the chairman shall have a casting vote. 14 7.8.4. At a meeting of the Supervisory Board, a Supervisory Director may only be represented by another Supervisory Director holding awritten proxy. 7.8.5. If a Supervisory Director has a direct or indirect personal conflict of interest with the Company, he shall not participate in thedeliberations and the decision-making process concerned in the Supervisory Board. If as a result thereof no resolution of the SupervisoryBoard can be adopted the resolution can nonetheless be adopted by the Supervisory Board. In that case each Supervisory Director shall beentitled to participate in the deliberations and the decision-making process concerned in the Supervisory Board. 7.8.6. The Supervisory Board may also adopt resolutions without holding a meeting, provided such resolutions are adopted in writing or in areproducible manner by electronic means of communication and all Supervisory Directors entitled to vote have consented to adopting theresolution outside a meeting. 7.8.7. Articles 7.8.3 and 7.8.5 shall equally apply to adoption by the Supervisory Board of resolutions without holding a meeting. 7.8.8. The Managing Directors must, if invited to do so, attend the meetings of the Supervisory Board and they shall provide in such meetingsall information required by the Supervisory Board. 7.8.9. The Supervisory Board may decide that one or more of its members shall have access to all premises of the Company and shall beauthorised to examine all books, correspondence and other records and to be fully informed of all actions which have taken place, or maydecide that one or more of its members shall be authorised to exercise a portion of such powers. 7.8.10. At the expense of the Company, the Supervisory Board may obtain such advice from experts as the Supervisory Board deems desirablefor the proper fulfilment of its duties. 7.9. Indemnification Managing Directors and Supervisory Directors. 7.9.1. Unless Dutch law provides otherwise, the following shall be reimbursed to current and former members of the Management Board orSupervisory Board: (a) the reasonable costs of conducting a defence against claims based on acts or failures to act in the exercise of their duties or anyother duties currently or previously performed by them at the Company’s request; (b) any damages or fines payable by them as a result of an act or failure to act as referred to under a; (c) the reasonable costs of appearing in other legal proceedings in which they are involved as current or former members of theManagement Board or Supervisory Board, with the exception of 15 proceedings primarily aimed at pursuing a claim on their own behalf. There shall be no entitlement to reimbursement as referred to above if and to the extent that: (d) a Dutch court or, in the event of arbitration, an arbitrator has established in a final and conclusive decision that the act or failureto act of the person concerned can be characterised as wilful (opzettelijk), intentionally reckless (bewust roekeloos) or seriouslyculpable (ernstig verwijtbaar) conduct, unless Dutch law provides otherwise or this would, in view of the circumstances of thecase, be unacceptable according to standards of reasonableness and fairness; or (e) the costs or financial loss of the person concerned are covered by an insurance and the insurer has paid out the costs or financialloss. If and to the extent that it has been established by a Dutch court or, in the event of arbitration, an arbitrator in a final and conclusivedecision that the person concerned is not entitled to reimbursement as referred to above, he shall immediately repay the amount reimbursedby the Company. 7.9.2. The Company may take out liability insurance for the benefit of the persons concerned. 7.9.3. The Management Board may by agreement give further implementation to the above. 8. MEETINGS. 8.1. General Meetings. 8.1.1. General Meetings shall be held in Amsterdam or in the municipality of Haarlemmermeer (Schiphol Airport). 8.1.2. A General Meeting shall be held once a year, no later than six (6) months after the end of the financial year of the Company. 8.1.3. The Management Board and the Supervisory Board shall provide the General Meeting with all requested information, unless this wouldbe contrary to an overriding interest of the Company. If the Management Board or Supervisory Board invokes an overriding interest, itmust give reasons. 8.2. Extraordinary General Meetings. Extraordinary General Meetings shall be convened by the Management Board or Supervisory Board. 16 8.3. General Meetings: notice and agenda. 8.3.1. Notice of the General Meeting shall be given by the Management Board or Supervisory Board upon a term of at least such number ofdays prior to the day of the meeting as required by law, in accordance with law and the regulations of the stock exchange where the Sharesin the share capital of the Company at the Company’s request are officially listed. 8.3.2. The Management Board or Supervisory Board may decide that the convocation letter in respect of a person authorised to attend a GeneralMeeting who agrees thereto, is replaced by a legible and reproducible message sent by electronic mail to the address indicated by him to theCompany for such purpose. 8.3.3. The notice shall state the subjects on the agenda or shall inform the persons authorised to attend a General Meeting that they may inspectthe agenda at the office of the Company and that copies thereof are obtainable at such places as are specified in the notice. 8.3.4. The agenda for the annual General Meeting shall in any case include the following items: (a) the consideration of Annual Statement of Accounts; (b) the adoption of the Annual Accounts; (c) the appropriation of profits; (d) proposals relating to the composition of the Management Board or Supervisory Board, including the filling of any vacancies inthe Management Board or Supervisory Board; (e) the proposals placed on the agenda by the Management Board or Supervisory Board together with proposals made byShareholders in accordance with provisions of the law and the provisions of the articles of association. 8.3.5. A matter, the consideration of which has been requested in writing by one or more Shareholders, representing solely or jointly at least thepercentage prescribed by law of the issued share capital, will be placed on the notice or will be announced in the same manner if theCompany has received the request not later than on the date as prescribed by law. 8.3.6. The Management Board shall inform the General Meeting by means of a shareholders’ circular or explanatory notes to the agenda of allfacts and circumstances relevant to the proposals on the agenda. 8.4. General Meetings: attendance of meetings. 8.4.1. The persons who are entitled to attend the General Meeting are persons who: (i) are a Shareholder or a person who is otherwise entitled to attend the General Meeting as per a certain date, determined by the 17 Management Board, such date hereinafter referred to as: the “record date”; (ii) are as such registered in a register (or one or more parts thereof) designated thereto by the Management Board, hereinafter referredto as: the “register”; and (iii) have given notice in writing to the Company prior to a date set in the notice that they will attend a General Meeting, regardless of who will be Shareholder at the time of the meeting. The notice will contain the name and the number of Shares the person willrepresent in the meeting. The provision above under (iii) concerning the notice to the Company also applies to the proxy holder of a personauthorised to attend a General Meeting. 8.4.2. The Management Board may decide that Persons entitled to attend General Meetings and vote thereat may, within a period prior to theGeneral Meeting to be set by the Management Board, which period cannot begin prior to the record date as meant in article 8.4.1, cast theirvotes electronically in a manner to be decided by the Management Board. Votes cast in accordance with the previous sentence are equal tovotes cast at the meeting. 8.4.3. The Management Board may decide that the business transacted at a General Meeting can be taken note of by electronic means ofcommunication. 8.4.4. The Management Board may decide that each person entitled to attend General Meetings and vote thereat may, either in person or bywritten proxy, vote at that meeting by electronic means of communication, provided that such person can be identified via the electronicmeans of communication and furthermore provided that such person can directly take note of the business transacted at the GeneralMeeting concerned. The Management Board may attach conditions to the use of the electronic means of communication, which conditionsshall be announced at the convocation of the General Meeting and shall be posted on the Company’s website. 8.4.5. Managing Directors and Supervisory Directors shall have admission to the General Meetings. They shall have an advisory vote at theGeneral Meetings. 8.4.6. Furthermore, admission shall be given to the persons whose attendance at the General Meeting is approved by the chairman of themeeting. 8.4.7. All issues concerning the admittance to the General Meeting shall be decided by the chairman of the meeting. 8.5. General Meetings: order of the meeting, minutes. 8.5.1. The General Meeting shall be chaired over by the chairman of the Supervisory Board. However, the chairman may charge another person 18 to chair the General Meeting in his place even if he himself is present at the meeting. If the chairman of the Supervisory Board is absentand he has not charged another person to chair the meeting in his place, the Supervisory Directors present at the meeting shall appoint oneof them to be chairman. If no members of the Supervisory Board are present at the General Meeting, the General Meeting shall be chairedby the chairman of the Management Board, or, if the chairman of the Management Board is absent, by one of the other members of theManagement Board designated for that purpose by the Management Board. The chairman shall designate the secretary. 8.5.2. The chairman of the meeting shall determine the order of proceedings at the meeting with due observance of the agenda and he mayrestrict the allotted speaking time or take other measures to ensure orderly progress of the meeting. 8.5.3. All issues concerning the proceedings at the meeting, shall be decided by the chairman of the meeting. 8.5.4. Minutes shall be kept of the business transacted at the meeting unless a notarial record is prepared thereof. Minutes shall be adopted andin evidence of such adoption be signed by the chairman and the secretary of the meeting concerned. 8.5.5. A certificate signed by the chairman and the secretary of the meeting confirming that the General Meeting has adopted a particularresolution, shall constitute evidence of such resolution vis-à-vis third parties. 8.6. General Meetings: adoption of resolutions. 8.6.1. Resolutions proposed to the General Meeting by the Management Board or Supervisory Board shall be adopted by a simple majority ofthe votes cast unless the law or these articles of association provide otherwise. Unless another majority of votes or quorum is required byvirtue of the law, all other resolutions shall be adopted by at least a simple majority of the votes cast, provided such majority representsmore than one-third of the issued share capital. A second meeting referred to in article 2:120, subsection 3 DCC cannot be convened. 8.6.2. Each Share confers the right to cast one (1) vote at the General Meeting. Blank votes and invalid votes shall be regarded as not having been cast. 8.6.3. No votes may be cast at the General Meeting in respect of Shares which are held by the Company or any of its Subsidiaries. Holders of a right of use and enjoyment (vruchtgebruik) and pledgees of Shares which belong to the Company or its Subsidiaries shallnot be excluded from the right to vote if the right of use and enjoyment or pledge was created before the Shares concerned were held by theCompany or a Subsidiary of the Company and at the creation of the 19 right of pledge or the right of use and enjoyment, the voting rights were granted to the pledgee or holder of the right of use and enjoyment. 8.6.4. The chairman of the General Meeting determines the method of voting. 8.6.5. The ruling pronounced by the chairman of the General Meeting in respect of the outcome of any vote taken at a General Meeting shall bedecisive. The same shall apply to the contents of any resolution passed. 8.6.6. Any and all disputes with regard to voting for which neither the law nor the articles of association provide shall be decided by thechairman of the General Meeting. 9. FINANCIAL YEAR. AUDITOR. 9.1. Financial year; Annual Statement of Accounts. 9.1.1. The financial year of the Company shall be the calendar year. 9.1.2. Annually, within the term set by law, the Management Board shall prepare Annual Accounts. The Annual Accounts shall be accompanied by the auditor’s statement referred to in article 9.2.1, if the instruction referred to in thatarticle has been given, by the Annual Report, unless section 2:391 DCC does not apply to the Company, as well as by the otherparticulars to be added to those documents by virtue of applicable statutory provisions. The Annual Accounts shall be signed by all Managing Directors and by all Supervisory Directors; if the signature of one or more of themis lacking, this shall be disclosed, stating the reasons therefor. 9.1.3. The Company shall ensure that the Annual Accounts as prepared, the Annual Report (if applicable) and the other particulars referred toin article 9.1.2 shall be made available at the office of the Company as of the date of the notice of the General Meeting at which they are tobe discussed. The Shareholders and other Persons entitled to attend General Meetings may inspect the above documents at the office of the Companyand obtain a copy thereof free of charge. 9.2. Auditor. 9.2.1. The General Meeting shall instruct a registered accountant or another expert, as referred to in section 2:393, subsection 1 DCC, bothhereinafter called: the “auditor”, to audit the Annual Accounts prepared by the Management Board, in accordance with the provisions ofsection 2:393, subsection 3 DCC. The auditor shall report on his audit to the Management Board and shall present the results of hisexamination regarding the accuracy of the Annual Accounts in an auditor’s statement. 20 9.2.2. If the General Meeting fails to give such instructions, then the Supervisory Board, or if the Supervisory Board also fails to give suchinstruction, the Management Board shall be so authorised. 9.2.3. The instruction given to the auditor may be revoked by the General Meeting and by the corporate body which has given such instruction;furthermore, the instruction given by the Management Board may be revoked by the Supervisory Board. The instruction may only be revoked for good reasons with due observance of section 2:393, subsection 2 DCC. 9.2.4. The Management Board as well as the Supervisory Board may give instructions to the auditor or any other auditor at the expense of theCompany. 10. PROFITS. 10.1. Profit and loss. Distributions on Shares. 10.1.1. The Management Board will keep a share premium reserve and profit reserve for the Shares. 10.1.2. The Company may make distributions on Shares only to the extent that its shareholders’ equity exceeds the sum of the paid-up andcalled-up part of the capital and the reserves which must be maintained by law. 10.1.3. Distributions of profit, meaning the net earnings after taxes shown by the adopted Annual Accounts, shall be made after the adoption ofthe Annual Accounts from which it appears that they are permitted, without prejudice to any of the other provisions of these articles ofassociation. 10.1.4. The Management Board may determine, subject to the approval of the Supervisory Board, that any amount out of the profit shall beadded to the reserves. 10.1.5. The profit remaining after application of article 10.1.4 shall be at the disposal of the General Meeting, which may resolve to carry it to thereserves or to distribute it among the Shareholders. 10.1.6. On a proposal of the Management Board — which proposal must be approved by the Supervisory Board — the General Meeting mayresolve to distribute to the Shareholders a dividend in the form of Shares in the share capital of the Company. 10.1.7. Subject to the other provisions of this article 10.1 the General Meeting may, on a proposal made by the Management Board — whichproposal must be approved by the Supervisory Board — resolve to make distributions to the Shareholders to the debit of one (1) orseveral reserves which the Company is not prohibited from distributing by virtue of the law. 21 10.1.8. No dividends shall be paid on Shares held by the Company in its own share capital, unless such Shares are encumbered with a right ofuse and enjoyment (vruchtgebruik) or pledge. 10.2. Interim distributions. 10.2.1. The Management Board may resolve, subject to the approval of the Supervisory Board, to make interim distributions to the Shareholdersif an interim statement of assets and liabilities shows that the requirement of article 10.1.2 has been met. 10.2.2. The interim statement of assets and liabilities shall relate to the condition of the assets and liabilities on a date no earlier than the first dayof the third month preceding the month in which the resolution to distribute is published. It shall be prepared on the basis of generallyacceptable valuation methods. The amounts to be reserved under the law and these articles of association shall be included in the statementof assets and liabilities. It shall be signed by the Managing Directors and Supervisory Directors. If the signature of one or more of them islacking, this shall be disclosed, stating the reasons therefor. 10.2.3. Any proposal for distribution of dividend on Shares and any resolution to distribute an interim dividend on Shares shall immediately bepublished by the Management Board in accordance with the regulations of the stock exchange where the Shares at the Company’s requestare officially listed. The notification shall specify the date when and the place where the dividend shall be payable or - in the case of aproposal for distribution of dividend - is expected to be made payable. 10.2.4. Dividends shall be payable no later than thirty (30) days after the date they were declared, unless the body declaring the dividenddetermines a different date. 10.2.5. Dividends which have not been claimed upon the expiry of five (5) years and one (1) day after the date when they became payable shallbe forfeited to the Company and shall be carried to the reserves. 10.2.6. The Management Board may determine that distributions on Shares shall be made payable either in euro or in another currency. 11. AMENDMENT OF THE ARTICLES OF ASSOCIATION; DISSOLUTION OF THE COMPANY. 11.1. A resolution to amend the articles of association or to dissolve the Company may only be adopted at the proposal of the Management Board with theprior approval of the Supervisory Board. 11.2. Liquidation. 11.2.1. On the dissolution of the Company, the liquidation shall be carried out by the Management Board, unless otherwise resolved by theGeneral Meeting. 22 11.2.2. Pending the liquidation the provisions of these articles of association shall remain in force to the fullest extent possible. 11.2.3. The surplus assets of the Company remaining after satisfaction of its debts shall, in accordance with the provisions of section 2:23bDCC, be for the benefit of the Shareholders in proportion to the nominal value amount of the Shares held by each of them. 12. TRANSITIONAL PROVISION. As per the date of the present conversion and amendment to the articles all the issued shares class A, class B and class C have been redesignated asordinary shares numbered from 1 to 12,194,906 and thirty nine (39) fractions of each one cent (€ 0.01). Consequently, the issued capital as pertoday amounts to EUR 609,745,69. 13. ACCOUNTANTS CERTIFICATE Pursuant to the law, the accountant certificate as required by Article 2: 18 in connection with 72 DCC is attached to the present deed, certifying thatthe Company’s equity as per 30 September 2013, (being a date not earlier than 5 months prior to today’s date) at least amounts to the issued and paidup capital of the Company. END This deed was executed in Amsterdam on the date first above written. The contents of this instrument were given and explained to the person appearing. He then declared that she had timely noted and approved the contents and did not want a full reading thereof. Thereupon, after limited reading, this instrument was signed by the person appearing and by me, civil law notary. 23Exhibit 8.1 SUBSIDIARIES OF UNIQURE N.V. Name of SubsidiaryJurisdiction of OrganizationuniQure biopharma B.V.The NetherlandsuniQure IP B.V.The NetherlandsuniQure Inc.DelawareuniQure manufacturing B.V.The NetherlandsuniQure Assay Development B.V.The NetherlandsuniQure Research B.V.The NetherlandsuniQure non clinical B.V.The NetherlandsuniQure QA B.V.The NetherlandsuniQure process development B.V.The NetherlandsuniQure clinical B.V.The Netherlands Exhibit 12.1 Certification by the Chief Executive OfficerPursuant to Section 302 of the Sarbanes-Oxley Act of 2002 I, Jörn Aldag, certify that: 1. I have reviewed this annual report on Form 20-F of uniQure N.V. (the “Company”); 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 the statementsmade, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report; 3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financialcondition, results of operations and cash flows of the Company as of, and for, the periods presented in this report; 4. The Company’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in ExchangeAct Rules 13a-15(e) and 15d-15(e)) for the Company and have: (a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure thatmaterial information relating to the Company, including its consolidated subsidiaries, is made known to us by others within those entities, particularly duringthe period in which this report is being prepared; (b) [Deliberately omitted] (c) Evaluated the effectiveness of the Company’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of thedisclosure 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 Company’s internal control over financial reporting that occurred during the period covered by the annual reportthat has materially affected, or is reasonably likely to materially affect, the Company’s internal control over financial reporting; and 5. The Company’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to theCompany’s auditors and the audit committee of the Company’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 are reasonably likely toadversely affect the Company’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 Company’s internal control overfinancial reporting. Date: April 25, 2014 By:/S/ JÖRN ALDAGName:Jörn AldagTitle:Chief Executive Officer Exhibit 12.2 Certification by the Chief Financial OfficerPursuant to Section 302 of the Sarbanes-Oxley Act of 2002 I, Piers Morgan, certify that: 1. I have reviewed this annual report on Form 20-F of uniQure N.V. (the “Company”); 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 the statementsmade, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report; 3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financialcondition, results of operations and cash flows of the Company as of, and for, the periods presented in this report; 4. The Company’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in ExchangeAct Rules 13a-15(e) and 15d-15(e)) for the Company and have: (a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure thatmaterial information relating to the Company, including its consolidated subsidiaries, is made known to us by others within those entities, particularly duringthe period in which this report is being prepared; (b) [Deliberately omitted] (c) Evaluated the effectiveness of the Company’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of thedisclosure 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 Company’s internal control over financial reporting that occurred during the period covered by the annual reportthat has materially affected, or is reasonably likely to materially affect, the Company’s internal control over financial reporting; and 5. The Company’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to theCompany’s auditors and the audit committee of the Company’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 are reasonably likely toadversely affect the Company’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 Company’s internal control overfinancial reporting. Date: April 25, 2014 By:/S/ PIERS MORGANName:Piers MorganTitle:Chief Financial Officer Exhibit 13.1 Certification by the Chief Executive Officer and Chief Financial Officer pursuant to 18 U.S.C.Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002 In connection with the Annual Report on Form 20-F of uniQure N.V. (the “Company”) for the year ended December 31, 2013, as filed with the U.S.Securities and Exchange Commission on the date hereof (the “Report”), the undersigned Jörn Aldag, as Chief Executive Officer of the Company, and PiersMorgan, as Chief Financial Officer of the Company, each hereby certifies, pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of theSarbanes-Oxley Act of 2002, that to the best of his 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: April 25, 2014 By:/S/ JÖRN ALDAGName:Jörn AldagTitle:Chief Executive Officer By:/S/ PIERS MORGANName:Piers MorganTitle:Chief Financial Officer 

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