The results of a European gene therapy clinical trial sponsored by Généthon, involving clinicians from France, Italy and the Netherlands, were published today in The New England Journal of Medicine. It demonstrates the possibility of restoring expression of liver enzyme UGT1A1 by using gene therapy in cases of Crigler-Najjar syndrome. The candidate drug designed by Généthon lowered bilirubin levels below the toxic threshold with a single intravenous injection to such an extent that 3 patients treated with the highest dose have been able to stop using phototherapy for the last 18 months or more. The clinical trial is currently in the pivotal phase.
