Rocket Pharmaceuticals Touts Positive Early Safety Data from Revised Genetic Therapy Trial

Rocket Pharmaceuticals (RCKT) announced positive early safety data from the first three patients in its Phase 2 trial for Danon disease, a rare genetic heart condition. The trial utilized a modified protocol with a recalibrated dose of RP-A501 and a refined immunomodulatory regimen, showing no significant safety concerns. The company is in discussions with the FDA regarding the next steps for dosing the remaining trial participants.