Rocket Capital

Rocket Pharmaceuticals is a leading late-stage biotechnology company dedicated to developing first-in-class gene therapies for rare and devastating genetic diseases. The company utilizes a multi-platform approach, leveraging both lentiviral vector (LVV) and adeno-associated viral (AAV) technologies to target hematologic and cardiovascular disorders. Its lead product, KRESLADI™ (marnetegene autotemcel), is FDA-approved for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I). Rocket's robust pipeline includes late-stage candidates for Fanconi Anemia, Danon Disease, and Pyruvate Kinase Deficiency, aiming to provide curative, one-time treatments that address the root cause of genetic conditions.