A Step Forward for Danon Disease as Rocket’s Gene Therapy Trial Resumes

August 20, 2025

DENVER, Colo., Aug 20, 2025 (247marketnews.com)- Rocket Pharmaceuticals’ (NASDAQ:RCKT) reported that the FDA has lifted the clinical hold on its pivotal Phase 2 trial of RP-A501 offers renewed hope for patients with Danon disease, a rare and often fatal genetic condition. The pause, which lasted less than three months, reflects both the efficiency of the FDA’s review and Rocket’s swift action to address safety concerns.

The resumption comes with a recalibrated dosing strategy, lowering the dose to 3.8 x 10¹³ GC/kg, aligned with levels that showed promising efficacy and safety in earlier trials. The trial will restart with three patients treated sequentially, spaced four weeks apart, allowing careful monitoring before expanding to the full 12-patient cohort. Importantly, the immunosuppressive protocol has also been refined to reflect the successful approach used in pediatric patients from Phase 1, further enhancing safety measures.

To date, six patients have already received treatment under the Phase 2 protocol. As Rocket prepares to treat the next three, the data will be critical in shaping the path toward accelerated approval, especially as the trial evaluates both biomarker improvements and functional heart outcomes.

Danon disease is a progressive, life-threatening condition with no approved therapies. RP-A501, the first gene therapy for a cardiovascular disease to demonstrate safety and efficacy in clinical studies, holds the potential to transform how we treat this devastating disorder. With the clinical hold lifted, Rocket can now continue its mission, advancing not only a single trial, but the broader promise of gene therapy for cardiac genetic diseases.

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