Rocket Pharmaceuticals has cleared a significant hurdle by obtaining FDA approval to resume its Phase 2 clinical trial of the RP-A501 gene therapy for Danon disease following a clinical hold issued earlier this year due to a patient death. The restart involves lowering the therapy dose and discontinuing a C3 complement inhibitor used in pre-treatment. Analysts view this as a meaningful positive development for Rocket, which now refocuses on demonstrating efficacy with the adjusted regimen. The therapy aims to treat this rare metabolic disorder affecting multiple organs, for which heart transplant remains the only current treatment option.