Genethon Highlights Key Developments in Gene Therapy Research

December 19, 2024 – Paris, France – Genethon, a pioneering non-profit gene therapy research and development organization founded by the French Muscular Dystrophy Association (AFM-Telethon), has unveiled significant progress in its clinical and scientific endeavors as 2024 concludes. These achievements are poised to further transform the landscape of gene therapies for rare diseases.

Phase 3-Ready Gene Therapy for Duchenne Muscular Dystrophy (DMD)

Genethon revealed positive safety and efficacy results from the Phase 1/2 dose-escalation portion of its international Phase 1/2/3 trial for GNT0004, an innovative gene therapy for Duchenne muscular dystrophy. This trial, targeting boys aged 6–10, demonstrated that higher doses of GNT0004 led to stable or improved motor functions over one to two years. Muscle biopsies showed 54% of fibers expressing microdystrophin— a functional version of the dystrophin protein—by eight weeks post-treatment, while muscle damage markers decreased by 74% within 12 weeks.

Encouraged by these findings, an independent data monitoring committee approved the pivotal Phase 3 trial, set to launch in Europe in mid-2025, followed by the U.S. The trial aims to enroll over 60 children and could pave the way for regulatory approval of GNT0004 as a best-in-class DMD therapy.

Combatting AAV Neutralizing Antibodies: Phase 2 Trial of Imlifidase

In collaboration with Hansa Biopharma, Genethon initiated a Phase 2 trial to address a key barrier in gene therapy: pre-existing antibodies against AAV (adeno-associated virus) vectors. These antibodies can render therapies ineffective in certain patients. The study, involving patients with Crigler-Najjar syndrome, tests imlifidase, an enzyme that breaks down AAV antibodies, before administering Genethon’s GNT0003 gene therapy.

This approach offers hope for extending gene therapy eligibility to more patients, overcoming one of the field’s most significant challenges.

AI-Driven Innovations: Next-Gen Capsid Design

Genethon scientists published groundbreaking research in Nature Communications on leveraging artificial intelligence to design next-generation capsids. These advanced capsids, tailored for muscle-targeted therapies, promise enhanced efficiency and safety. This innovation positions Genethon at the forefront of AI-driven advancements in gene therapy.

A Vision for Transformative Gene Therapies

With more than 200 scientists and professionals, Genethon continues to lead the charge in developing life-changing therapies for rare genetic diseases. Thirteen products stemming from its research are in clinical trials, targeting conditions affecting the liver, blood, muscles, immune system, and eyes. Seven additional candidates are expected to enter clinical trials in the next five years.
CEO Frederic Revah summarized the organization’s mission: “These milestones demonstrate our commitment to providing solutions to complex genetic diseases. Gene therapy has the power to transform lives, and we’re proud to contribute to this evolving field.”

For a detailed overview of Genethon’s achievements, visit www.genethon.com.

Source:
1. https://musculardystrophynews.com/news/dmd-gene-therapy-gnt0004-phase-3-trial-eu-us/
2. https://www.businesswire.com/news/home/20241219420964/en/A-Phase-3-Ready-DMD-Gene-Therapy-a-Clinical-Trial-to-Offset-AAV-Neutralizing-Antibodies-and-an-AI-Inspired-New-Generation-of-Capsids-Highlight-Genethon%E2%80%99s-2024-Fall-Milestones

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