Rocket Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on its pivotal Phase 2 trial of RP-A501, a gene therapy for Danon disease. The hold, imposed in May 2025 following a patient's death due to serious complications including capillary leak syndrome and infection, was lifted in under three months after the company revised its trial protocol. The study will resume with a reduced dose of 3.8x10¹³ GC/kg administered sequentially to three patients. RP-A501 is a one-time AAV9 gene therapy targeting the LAMP2B gene. Following the announcement, Rocket Pharmaceuticals' shares surged by approximately 23-25% in premarket trading, reflecting investor optimism about the trial's resumption.