Clinical trial to test a gene therapy treatment for a rare liver disease

Généthon starts a European phase I/II clinical trial to test a treatment for Crigler-Najjar Syndrome, a rare liver disease.

This European trial will include 17 patients over 10 years old, (the age when the liver reaches maturity); it aims to assess the tolerance of the product, determine the optimal dose, and assess the therapeutic efficacy of the drug candidate. The trial takes place in four investigation centres in Europe: France (Prof Labrune – Hôpital Béclère in Clamart), Italy (Prof Brunetti-Pierri – Hôpital Federico II, Prof d’Antiga – Azienda Ospedaliera Papa Giovanni XXIII in Bergamo) and the Netherlands (Prof Beuers – Academic Medical Center in Amsterdam).
The technology used in the trial was developed by the ‘Immunology and Gene Therapy for Liver Disease’ team at Généthon, headed by Dr Federico Mingozzi. In partnership with research teams in Italy and the Netherlands, Généthon has developed an Adeno-Associated Viral (AAV) vector that is able to transfer a copy of the UGT1A1 gene (coding for the production of bilirubin-GT) to the liver cells. Tested first of all in the Gunn rat, an animal model of the disease, a single injection of this drug candidate has enabled the long-term correction of the disease in murine models (Molecular Therapy Methods and Clinical Development – July 2016).

“We are delighted to be starting a clinical trial of the gene therapy product we have designed to treat Crigler Najjar Syndrome, a very serious liver disease. The trial was made possible by Généthon’s pioneering research into gene therapy for rare diseases, over a period of almost 20 years. Today, seven clinical trials are taking place across the world, involving products that Généthon’s researchers either developed themselves or participated closely in developing ” explains Frédéric Revah, CEO of Généthon.