FDA Eyes Reversal on Peptide Compounding Ban
The FDA signals a reversal of its 2023 peptide compounding ban, raising patient safety concerns, while gene therapy researchers face manufacturing hurdles.
By James Liu
Gene Therapy
3 articles tagged "Gene Therapy"
Sarepta Therapeutics Shares Surge on Rare Disease Drug Data
Sarepta Therapeutics shares rose 20% after releasing early clinical data for two rare muscle-wasting disease candidates, SRP-1001 and SRP-1003.
FDA Approves Kresladi Gene Therapy for Rare LAD-1 Disease
The FDA has approved Rocket Pharma's Kresladi for LAD-1, a rare immunodeficiency affecting roughly 25 US patients annually, marking a gene therapy milestone.
By Robert Kim