Rocket Pharmaceuticals, Inc. is a multi-platform biotechnology company focused on the development of first or best-in-class gene therapies for rare and devastating diseases. Rocket's lead lentiviral vector (LVV) program is currently being evaluated in a clinical trial for Fanconi Anemia (FA), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells. The Company has three additional LVV programs targeting other rare genetic diseases. On November 26, 2018, the company announced that the RP-A501 AAV program is initially being developed for the treatment of Danon disease. Rocket's two leading LVV and AAV technology platforms are each being designed in collaboration with leading academic and industry partners. Through Rocket's gene therapy platforms, the Company aims to restore normal cellular function by modifying the defective genes that cause each of the targeted disorders