Genethon announces dosing of the first patient with Crigler–Najjar Syndrome

Genethon announces dosing of the first patient with Crigler–Najjar Syndrome, a rare liver disorder, in the European phase I/II gene therapy clinical trial – CareCN.

Following patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. Genethon, the AFM-Telethon laboratory, is sponsoring this multicenter international phase I/II trial, injecting intravenously an AAV vector that is able to transfer the UGT1A1 gene (coding for the production of bilirubin GT) into liver cells. A total of 17 patients will be treated over the next few months.

The CareCN European clinical trial will assess the product’s safety, determine the optimal dose and evaluate the drug candidate’s therapeutic efficacy. It is being conducted in four clinical centres in Europe: in France (Prof. Labrune, Béclère Hospital in Clamart); in Italy (Prof. Brunetti-Pierri, Federico II Hospital in Naples and Prof. d’Antiga, Azienda Ospedaliera Papa Giovanni XXIII in Bergamo) and in the Netherlands (Prof. Beuers, Academic Medical Center in Amsterdam).

“We have dedicated many years of research at Genethon to design a gene therapy for the treatment of Crigler–Najjar syndrome, and we’ve worked in close collaboration with leading European clinical centres and with patients’ associations to prepare the clinical trial. Treating the first patient is an important milestone for patients and their families, doctors and researchers but also for our laboratory, Genethon, whose quality of R&D is once again making it possible to propose innovative treatment for a rare disease” says Genethon CEO Frederic Revah.