AVROBIO is a clinical stage gene therapy company focused on developing potentially curative lentiviral-based gene therapies to treat rare diseases following a single dose. The Company’s gene therapies employ hematopoietic stem cells that are extracted from the patient and then modified with lentiviral vectors to insert a functional copy of the gene that is defective in the target disease. The Company’s pipeline is comprised of four lentiviral-based gene therapies, including lead candidate AVR-RD-01 for the treatment of Fabry disease, AVR-RD-02 for the treatment of Gaucher disease, AVR-RD-03 for the treatment of Pompe disease and AVR-RD-04 for the treatment of cystinosis.