Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), achieved several major clinical development and scientific milestones over the past several months as highlighted in its year-end Newsletter.

Chief among them was the release of positive safety and efficacy results of the Phase 1/2 dose escalation part of its multicenter international all-in-one Phase 1/2/3 study of GNT0004, a gene therapy for Duchene muscular dystrophy. Based on these results the study’s independent data monitoring committee has given Genethon the green light to proceed with Phase 3 pivotal trials.

For all the details, including what distinguishes GNT0004 from all other DMD gene therapies, listen to two videos featuring Genethon CEO Frederic Revah in his CEO Video Commentary. Spoiler alert: The Phase 3 trial is expected to begin in Europe in 2025, followed by the US.

A second milestone is the start of a Phase 2 trial of antibody cleaving enzyme, imlifidase, to breakdown the anti-AAV antibodies that prevent gene therapies from working in patients with pre-existing antibodies against AAV, in partnership with Hansa Biopharma. The study will enroll Crigler-Najjar syndrome patients with pre-existing AAV neutralizing antibodies who will be treated with imlifidase before receiving Genethon’s GNT0003 gene therapy.

Yet a third major achievement is an article in Nature Communications describing the groundbreaking research by Genethon scientists in perfecting an innovative methodology using artificial intelligence (AI) to design a new generation of capsids for gene therapy vectors in treating muscle diseases.

Read the full Newsletter to learn about these milestones and others.

About Genethon

As a pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a non-profit laboratory that was established by AFM-Telethon. A first gene therapy for spinal muscular atrophy to which Genethon contributed has obtained a product license. With more than 200 scientists and professional staff, Genethon is pursuing its aim to develop therapies which change the lives of patients suffering from rare genetic diseases. Thirteen products stemming from Genethon’s R&D or from collaborations are in clinical trial for diseases of the liver, blood, immune system, muscles and eyes. Seven other products could enter clinical trials over the next five years. More information at www.genethon.com.

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