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Genethon Works Toward Gene Therapy For Crigler-Najjar Syndrome

 
• By Joseph Haas

The French non-profit, whose IP is incorporated in Zolgensma, is taking its Crigler-Najjar gene therapy into a pivotal study on its own for now. It hopes to be ready for an EU filing in two years.

Gene therapy
In addition to R&D, Genethon is focused on gene therapy affordability and immunity issues • Source: Shutterstock

Genethon may not be a traditional biotech, but as a non-profit it had research and intellectual property incorporated into Novartis AG’s gene therapy Zolgensma (onasemnogene abeparvovec), and now hopes to advance a candidate on its own that can address the underlying cause of a rare liver disorder, Crigler-Najjar Syndrome. Genethon believes its ongoing pivotal trial could position the candidate GNT-0003 for a European regulatory submission in approximately two years.

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