Rocket Pharmaceuticals is a late-stage biotechnology company developing one-time gene therapies for rare, often fatal genetic diseases. Rather than treating symptoms chronically, Rocket's therapies aim to correct the underlying genetic defect with a single treatment. Rocket uses two delivery platforms: AAV vectors administered directly to patients (in vivo), and lentiviral vectors used to modify a patient's own stem cells outside the body before reinfusion (ex vivo). Rocket has no approved products and generates no revenue today. Its most near-term potential approval is KRESLADI, a gene therapy for LAD-I — an extremely rare disorder affecting only hundreds of patients globally — with a PDUFA date of March 28, 2026. Rocket's primary strategic focus is its cardiovascular pipeline, led by RP-A501 for Danon disease, a fatal cardiomyopathy, which is in a Phase 2 pivotal trial following a resolved FDA clinical hold. Two earlier-stage cardiovascular programs, RP-A601 and RP-A701, are in Phase 1 and entering Phase 1, respectively. Its hematology programs outside of KRESLADI are being de-prioritized and out-licensed. If products are approved, Rocket would charge a one-time, high per-patient price consistent with other approved gene therapies, targeting a small number of patients through a focused specialty sales effort. Rocket operates its own AAV manufacturing facility in Cranbury, NJ, giving it in-house control over its cardiovascular programs. The company funds operations through equity and cash, and a July 2025 restructuring cut headcount by roughly 30%.