RP-A601

About Rocket Pharmaceuticals

Rocket Pharmaceuticals is a clinical-stage biotech focused on developing curative gene therapies for rare genetic disorders in cardiac and hematologic systems. The company has achieved a significant milestone with the FDA approval of KRESLADI™ for Severe Leukocyte Adhesion Deficiency-I (LAD-I) and is advancing a pivotal Phase 2 program for Danon Disease. Its strategy is built on a flexible, dual-platform technology approach (AAV and lentiviral) matched to disease biology, targeting high-value, niche markets with profound patient impact. Rocket operates as a public company, trading on NASDAQ under the ticker RCKT, and is navigating the critical transition from clinical development to commercialization.

Therapeutic Areas