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Genethon Moving Gene Therapy to Pivotal Trial in Crigler-Najjar Syndrome After Positive Early Data

NEW YORK – Promising early activity of an investigational gene therapy in patients with Crigler-Najjar syndrome has given sponsor Genethon confidence to conduct a larger trial to confirm the findings.

Crigler-Najjar syndrome, a rare, inherited disease due to a deficiency in the UGT1A1 enzyme, causes bilirubin to build up and patients to develop jaundice. The excess bilirubin can cause neurological damage and be fatal to patients, who have no treatment options other than daily phototherapy for up to 12 hours or a liver transplant.

Paris-based Genethon has developed GNT-0003, which delivers a normal copy of the UGT1A1 gene that encodes the bilirubin metabolizing enzyme. The firm on Tuesday said it had published findings in the New England Journal of Medicine from a Phase I/II trial showing that GNT-0003 was safe and well tolerated by five patients, and three patients on a higher dose of the treatment saw their bilirubin levels go below the toxic level and were able to stop phototherapy for at least 18 months.

Genethon is conducting this Phase I/II trial at several European centers in partnership with the consortium CureCN. According to the firm, the published data represents the first evidence suggesting that a gene therapy can potentially treat a metabolic disease of the liver. Genethon will now confirm GNT-0003's efficacy in a larger trial involving children at least 10 years old.

"If the results of the pivotal part confirm the efficacy of our gene therapy for Crigler-Najjar syndrome, we will be able to move on to a product license application, making the treatment available to patients and providing them with significantly improved quality of life," Genethon CEO Frederic Revah said in a statement.