(US) Rocket Pharmaceuticals, Inc. is a clinical-stage biotechnology company, which engages in the development of gene therapy treatment options for rare and devastating pediatric diseases. Its multi-platform development approach applies the lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. The firm's clinical program is a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. The company was founded in 1999 and is headquartered in New York, NY.