Rocket Pharmaceuticals has received FDA clearance to resume its Phase 2 gene therapy trial for Danon disease, a rare genetic disorder affecting multiple organs. The clinical hold issued in May, following a patient death linked to a complement inhibitor added to the pre-treatment regimen, has been lifted. The study will continue with a lower dose of the RP-A501 gene therapy and discontinuation of the complement inhibitor. This development restores momentum for Rocket, which was previously a high-profile gene therapy developer. Analysts remain focused on the efficacy and safety of the adjusted protocol as the trial proceeds.