Rocket Pharmaceuticals announced alignment with the FDA on key elements for the global pivotal Phase 2 trial of RP-A501 for Danon disease. The FDA confirmed a recalibrated dose of 3.8 × 10¹³ genome copies per kilogram and a 12-patient pivotal population, including the first three patients already treated under the modified protocol. Nine additional patients remain to be enrolled, with dosing expected to complete by mid-2027. The trial utilizes established 12-month co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction in left ventricular mass index to support potential accelerated approval. Initial safety data from the first three patients showed no evidence of thrombotic microangiopathy or capillary leak syndrome. Rocket Pharmaceuticals is a commercial-stage biotechnology company developing genetic medicines for rare and life-threatening diseases, with a strategic focus on inherited cardiovascular disorders.

On August 21, 2026, the Board of Directors adopted a new Severance and Change in Control Program for eligible U.S. employees, superseding prior arrangements. The company also entered into executive employment agreements with CEO Gaurav Shah (annual base salary $674,856) and General Counsel Martin Wilson ($547,313), while amending COO Christopher Stevens' agreement to provide 12 months of severance benefits upon qualifying termination without cause or for good reason. These arrangements include change-in-control protections and are contingent on the execution of a release of claims. Rocket Pharmaceuticals, Inc. operates in the biotechnology industry, developing gene therapies for rare genetic diseases.

Rocket Pharmaceuticals reported second quarter 2026 financial results and operational updates. The company generated $123.2 million in net income, driven by a $178.2 million gain from the sale of its Priority Review Voucher for KRESLADI. Cash, cash equivalents, and investments totaled $283.7 million as of June 30, 2026, providing an expected runway into the second quarter of 2028. Research and development expenses decreased to $29.5 million from $42.7 million in the prior year period. Clinically, all three initial patients treated with RP-A501 for Danon disease completed the safety observation period without significant concerns, and Rocket is engaging with the FDA on completing the pivotal Phase 2 trial. Commercialization preparations for KRESLADI are underway for patient onboarding in the fourth quarter of 2026. The company operates as a commercial-stage biotechnology firm developing genetic medicines for rare disorders.

Rocket Pharmaceuticals announced a positive clinical safety update for its investigational gene therapy RP-A501 in Danon disease. The initial three patients treated under a modified Phase 2 protocol received a recalibrated dose of 3.8 × 10¹³ GC/kg with a refined immunomodulatory regimen. No thrombotic microangiopathy, capillary leak syndrome, or other significant safety concerns were observed. The company is engaging with the FDA to align on completing the pivotal trial and expects a regulatory pathway update in the second half of 2026. Rocket Pharmaceuticals operates as a commercial-stage biotechnology company advancing genetic medicines for rare inherited cardiovascular disorders.

John Militello resigned as VP of Finance, Senior Controller, Treasurer and Principal Accounting Officer, effective July 7, 2026. Joseph Manhede, currently Associate Vice President and Corporate Controller, will be appointed as the Company's Principal Accounting Officer, effective July 8, 2026. Rocket Pharmaceuticals, Inc. is a biopharmaceutical company focused on gene therapy for rare diseases.

Rocket Pharmaceuticals, Inc. completed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) to a large pharmaceutical company on June 10, 2026, receiving $180.0 million in gross proceeds. The voucher was issued following the FDA approval of KRESLADI (marnetegragene autotemcel) for treating pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I). Rocket Pharmaceuticals, Inc. is a biotechnology company developing gene therapies for rare pediatric diseases.