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.
