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Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease

Retrieved on: 
Tuesday, January 10, 2023

Genethon , a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), has launched a pivotal clinical trial in Europe for treatment of Crigler-Najjar syndrome, a life threatening liver disease.

Key Points: 
  • Genethon , a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), has launched a pivotal clinical trial in Europe for treatment of Crigler-Najjar syndrome, a life threatening liver disease.
  • “This new step demonstrates once again the excellence of the research conducted at Genethon, which is today one of the major players in gene therapy at the international level."
  • Crigler-Najjar syndrome is a rare genetic liver disease characterized by abnormally high levels of bilirubin in the blood (hyperbilirubinemia).
  • The gene therapy, administered intravenously, was designed by Genethon's Immunology and Gene Therapy of Liver Diseases team, led by Dr. Giuseppe Ronzitti.