Advancing a pipeline of genetic therapies for rare disorders
Rocket is developing first-in-class gene therapy treatment options for rare, devastating diseases. Rocket’s multi-platform development approach leverages both lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapies. Rocket's lead clinical program is an 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. Preclinical and clinical studies of additional bone marrow-derived disorders are ongoing in Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO). Rocket is also developing an AAV-based gene therapy program for Danon Disease, a rare pediatric disease that primarily affects the heart.