August 22, 2023
Positive Phase 1/2 Clinical Trial Results of Genethon’s Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease, Published in The New England Journal of Medicine
Genethon, a pioneer and leader in gene therapy research and development for rare genetic diseases, announced today positive Phase 1/2 clinical trial results of its gene therapy, GNT-0003, for Crigler-Najjar syndrome were published in The New England Journal of Medicine.