FDA Lifts Hold on Rocket Pharmaceuticals’ Pivotal Gene Therapy Study at Lower Dose

FDA; Rocket Pharmaceuticals; gene therapy; Danon disease; clinical hold; RP-A501; dose adjustment; C3 complement inhibitor; capillary leak syndrome; patient death; Phase II trial

Neurogene Details New Safety Measures After Young Patient’s Death in Rett Syndrome AAV Gene Therapy Trial

Neurogene; AAV gene therapy; Rett syndrome; NGN-401; ASGCT; safety measures; HLH; clinical trial; MECP2 gene; FDA; dose adjustment