Rocket制药

Rocket Pharmaceuticals, Inc. is a clinical-stage biotechnology company. The Company is focused on developing gene therapy treatment options for rare, devastating diseases. The Company’s multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. The Company’s lead clinical program is a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a genetic disease that leads to bone marrow failure and potentially cancer. Its additional pipeline programs for bone marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO). The Company is also developing an AAV-based gene therapy program for pediatric heart failure indication, Danon disease.