Rocket Pharmaceuticals is a fully integrated biotechnology company developing genetic therapies for rare, fatal genetic disorders with high unmet medical need, particularly inherited cardiomyopathies and blood disorders. The company employs two delivery approaches—adeno-associated virus vectors for direct patient administration and lentiviral vectors for ex vivo modification of patient cells—and maintains in-house GMP manufacturing and R&D capabilities to control production and clinical development. Rocket generates value by advancing first-in-class and best-in-class therapies through clinical trials toward regulatory approval and commercialization, targeting monogenic disorders affecting patient populations in the tens of thousands across the United States and Europe.