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

The gene therapy would represent a first-of-its kind treatment for this life threatening genetic disease.

Genethon has launched a pivotal clinical trial in Europe for treatment of Crigler-Najjar syndrome, a life threatening liver disease. The trial of the gene therapy, GNT-0003, will be conducted in France, Italy and the Netherlands and will enroll patients aged 10 years and older with the objective of confirming efficacy and safety seen in the previous clinical part…

“We are very pleased with this new step because if the results of the pivotal part confirm the efficacy of GNT-0003, we will be able to move forward towards a registration application and the availability of the treatment for patients,” said Frederic Revah, CEO of Genethon. “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.”

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