Rocket Pharmaceuticals Inc

Rocket Pharmaceuticals is a fully integrated, commercial-stage biotechnology company developing genetic medicines for rare and devastating diseases, with a strategic focus on inherited cardiovascular disorders and additional programs in hematology and immunology. The company's cardiovascular portfolio includes three clinical-stage gene therapy programs: RP-A501 for Danon disease (hypertrophic cardiomyopathy), RP-A601 for PKP2 arrhythmogenic cardiomyopathy, and RP-A701 for BAG3-associated dilated cardiomyopathy, representing one of the broadest pipelines focused on inherited heart disease. Rocket's first FDA-approved product is KRESLADI™ (marnetegragene autotemcel), an autologous hematopoietic stem cell-based gene therapy for severe leukocyte adhesion deficiency-I (LAD-I), an ultra-rare pediatric immunodeficiency. The company's integrated platform combines proprietary AAV manufacturing capabilities, extensive clinical experience in cardiac gene therapy, and long-term safety and efficacy data. Rocket trades on NASDAQ under the ticker RCKT. Updated Aug 10, 2026