Cambridge-based AVROBIO, Inc. is making waves in the biotech industry as a clinical-stage gene therapy company focused on ex vivo lentiviral-based gene therapies to treat rare diseases with only a single dose. AVROBIO's cutting-edge gene therapies utilize hematopoietic stem cells sourced from patients and are modified with a lentiviral vector to insert a functional copy of the mutated gene that causes the disease. The company's pipeline is comprised of four key programs: AVR-RD-02 is currently in a phase 1/2 clinical trial for the treatment of Gaucher disease type 1 and type 3; AVR-RD-03 is in preclinical stage for Pompe disease; AVR-RD-04 is in a phase 1/2 clinical trial for cystinosis treatment; and AVR-RD-05 is in preclinical stage for the treatment of Hunter syndrome. Founded in 2015, AVROBIO is dedicated to revolutionizing the field of gene therapy and providing hope to those suffering from rare diseases worldwide.