Rocket Companies

Rocket Pharmaceuticals is a clinical-stage biotech developing gene therapies for rare genetic diseases. Its lead programs include Kresladi for severe LAD-I—currently under FDA review after a complete response letter regarding manufacturing—and RP-L102 for Fanconi anemia, which has received European approval and is under rolling BLA submission with the FDA. The company’s cardiovascular pipeline includes AAV-based therapies such as RP-A501 for Danon disease (currently on FDA hold following a patient death), RP-A601 for PKP2 ACM with promising early efficacy data in phase 1, and BAG3-targeted therapies. Early results show strong efficacy signals in cardiac indications but also highlight safety challenges typical of advanced gene therapies.