FDA Lifts Hold on Rocket Pharmaceuticals' Pivotal Gene Therapy Study at Lower Dose on August 20, 2025 C3 complement inhibitor Capillary Leak Syndrome Dosage gene therapy Glycogen Storage Disease Type IIb Individual Adjustment Rocket RP-A501 Study on Hold United States Food and Drug Administration +
Patient Death in Rocket Pharmaceuticals Gene Therapy Trial Prompts FDA Clinical Hold on May 27, 2025 acute systemic infection Adverse event Capillary Leak Syndrome gene therapy Glycogen Storage Disease Type IIb Patient Death Rocket RP-A501 Study on Hold United States Food and Drug Administration +