The first proof of an effective gene therapy for a metabolic disease of the liver is reported in patients with Crigler-Najjar syndrome. No serious adverse events were reported in the phase I/II study ...

PARIS--(BUSINESS WIRE)--Genethon, a pioneer and leader in gene therapy research and development for rare genetic diseases, announced today positive Phase 1/2 clinical trial results of its gene therapy ...

Gene therapy for Crigler-Najjar syndrome appeared promising in patients treated at a higher dose, a phase I/II trial showed. A single infusion of GNT0003 -- an adeno-associated virus serotype 8 vector ...

LUND, Sweden & EVRY, France--(BUSINESS WIRE)--Hansa Biopharma, “Hansa” (Nasdaq Stockholm: HNSA) and Genethon, a pioneer and a leader in gene therapy research and development for rare genetic diseases, ...

Genethon, a worldwide pioneer and leader in research and development of gene therapy for rare genetic diseases, and Hansa Biopharma, a Sweden-based leader in IgG cleaving enzyme technology announced ...

“We’re excited about the EMA’s PRIME recognition of GNT-0003,” said Genethon CEO Frederic Revah. “If successful, GNT-0003 would be the first gene therapy for Crigler-Najjar syndrome. The PRIME status ...

Inherited hyperbilirubinemia, encompassing Gilbert syndrome, Crigler-Najjar syndrome, Dubin-Johnson syndrome, and Rotor syndrome, represents a spectrum of disorders rooted in genetic defects affecting ...

A group of European researchers from a nonprofit biotech firm Genethon in France has arrived with an experimental treatment for a rare liver disease. The experts managed to restore the living function ...

Here at Généthon, a nonprofit research organization, we have a history of accepting big challenges. At our founding in 1990, we devoted ourselves to genetics research. Seven years later, we ...

One newborn in a million inherits a defective gene behind a deadly condition named Crigler-Najjar Syndrome. It is caused by a total lack of total of an enzyme needed by the liver to eliminate ...

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