Aug 10, 2026 —
Rocket Pharmaceuticals reported second-quarter 2026 financial results and provided updates across its genetic medicine pipeline, led by new safety findings from RP-A501, its investigational AAV gene therapy for Danon disease.
All three initial patients treated under the modified pivotal Phase 2 protocol for RP-A501 completed the protocol-defined safety observation period. Rocket reported no thrombotic microangiopathy, or TMA, no capillary leak syndrome, and no other significant safety concerns at the recalibrated dose with the refined immunomodulatory regimen.
RP-A501 is being evaluated as a gene therapy for Danon disease, a rare inherited cardiomyopathy caused by mutations in LAMP2. The modified Phase 2 protocol uses a recalibrated dose of 3.8 × 10¹³ GC/kg, selected based on the characteristics of the current drug product, Danon disease biology, and prior Phase 1 experience.
Rocket said it is actively engaging with the FDA to align on the path to dosing additional patients and completing the pivotal Phase 2 trial. The company expects to provide an update on the regulatory pathway in the second half of 2026 and also plans to host an investor webinar with a broader Danon disease program update.
In parallel with the pivotal Phase 2 trial, Rocket’s global natural history study has enrolled more than 50 patients, including both males and females, to build a longitudinal dataset characterizing Danon disease progression.
Rocket also provided updates on two additional cardiovascular AAV gene therapy programs. RP-A601 is being evaluated for PKP2-associated arrhythmogenic cardiomyopathy, or PKP2-ACM. Rocket continues to engage with the FDA on the design of a potential pivotal study, with a regulatory update expected in the second half of 2026.
Previously reported Phase 1 findings for RP-A601 showed increased PKP2 protein expression and improved desmosomal localization, along with directional improvements in arrhythmia measures and right ventricular function. The Phase 1 study remains open and actively enrolling.
For RP-A701, Rocket has begun patient screening and enrollment in BAG3-associated dilated cardiomyopathy, or BAG3-DCM, with initial patient dosing expected in the second half of 2026. The multicenter, dose-escalation Phase 1 study is designed to evaluate safety, biological activity, and preliminary efficacy in adults with BAG3-DCM.
Rocket also highlighted commercial progress for KRESLADI™, also known as marnetegragene autotemcel, following FDA accelerated approval for severe leukocyte adhesion deficiency-I. The company is preparing for commercial availability and patient onboarding beginning in the fourth quarter of 2026 through a focused network of specialized Qualified Treatment Centers.
The sale of Rocket’s Rare Pediatric Disease Priority Review Voucher generated $180 million in gross proceeds, providing non-dilutive capital to support the company’s prioritized cardiovascular pipeline.
As of June 30, 2026, Rocket reported $283.7 million in cash, cash equivalents, and investments. The company expects its current cash position to fund operations into the second quarter of 2028.
The update reinforces Rocket’s strategic focus on inherited cardiovascular disorders, with near-term clinical and regulatory milestones expected across Danon disease, PKP2-ACM, and BAG3-DCM in the second half of 2026.
