Rocket Pharmaceuticals has received approval from the FDA to resume its Phase 2 gene therapy trial testing RP-A501 for Danon disease following a clinical hold. The hold was issued due to a patient death linked to a capillary leak syndrome, prompting Rocket to adjust the protocol by lowering the dose and discontinuing an added C3 complement inhibitor pre-treatment. The FDA lifted the hold under these new conditions, allowing the pivotal study to continue. Analysts highlight the significance of this development for the gene therapy's approval prospects, noting ongoing attention to dosing efficacy and safety as the trial progresses. This trial aims to provide a novel treatment for Danon disease, currently managed only by heart transplantation.