The FDA has approved Rocket Pharmaceuticals to resume its pivotal Phase 2 gene therapy trial for Danon disease, a rare genetic disorder affecting multiple organs including the heart. The trial had been on hold following a patient death linked to capillary leak syndrome. The restart includes a reduced dose of the RP-A501 gene therapy and discontinuation of an added C3 complement inhibitor medicine identified as a possible cause of the previous adverse event. Analyst commentary suggests the removal of the clinical hold is a positive for Rocket, yet efficacy at the lower dose remains a key focus. This development offers renewed momentum for Rocket in a rare cardiovascular gene therapy field with currently limited treatments.