Press releases

First results of Crigler-Najjar clinical trial published in New England Journal of Medicine

The efficacy of gene therapy demonstrated in patients suffering from Crigler-Najjar Syndrome, a rare liver disease.

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Genethon and Thales collaborate on artificial intelligence to improve bioproduction efficiency

This collaboration between the pioneer and leader in research and development in gene therapy and the European leader in Artificial Intelligence for mission-critical systems, aims to develop a digital model that will use artificial intelligence to model bioproduction processes and optimize yields.

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Upholder of excellence in gene therapyGenother awarded Biocluster label

Yesterday, the French President announced that the Genother biocluster — of
which Genethon is a founding member — is being awarded biocluster status in
the context of the France 2030 “Biocluster” call for expression of interest.

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Genethon’s R&D to be Featured in Multiple Presentations at American Society of Gene & Cell Therapy Annual Meeting May 16-20, 2023, in Los Angeles, CA

Genethon’s research will be featured in 4 oral presentations and 10 posters at the 26^th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), May 16-20, 2023, at the Los Angeles Convention Center in Los Angeles, CA.

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ILTOO Pharma and the MIROCALS Consortium announce the signing of Licence Agreement for the development of low dose interleukin-2

MIROCALS (Modifying Immune Response and Outcomes in ALS) is a European Consortium including research laboratories in the UK, Italy, Sweden and research support organisations in Ireland, in the UK, and in France with Genethon.

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Crigler-Najjar syndrome: Hansa Biopharma and Genethon announce collaboration to develop imlifidase as pre-treatment to gene therapy for patients with anti-AAV antibodies

Lund, Sweden and Evry, France April 27, 2023. Hansa Biopharma AB, “Hansa”, (Nasdaq Stockholm: HNSA), a pioneer in enzyme technology for rare immunological conditions, and Genethon, a pioneer and a leader in gene therapy research and development for rare genetic diseases, today announced they have entered a research and development… (read more)

Crigler-Najjar Syndrome: Genethon obtains PRIME priority drug status from the EMA for its gene therapy

The European Medicines Agency (EMA) has granted PRIME (PRIority MEdicine) status to gene therapy product GNT-0003 currently being tested as part of a clinical trial in Crigler-Najjar syndrome, a rare liver disease.

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Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease

The gene therapy would represent a first-of-its kind treatment for this life threatening genetic disease.

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Genethon’s Latest Research, Including Advances in Gene Editing and AAV Delivery Vectors for Gene Therapy, to be Featured at European Society for Gene and Cell Therapy, Oct. 11-14, 2022

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First Patient Dosed in Phase 1/2 Clinical Trial in Europe of Gene Therapy for LGMD-R9

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Genethon to Present Its Latest Gene Therapy Research Advancements in Neuromuscular Diseases at the International Myology 2022 Congress

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Genethon’s R&D to be Featured in Multiple Presentations at ASGCT Annual Meeting, May 16-19

Genethon will be featured in six presentations at the 25^th Annual Meeting of the American Society of Cell and Gene Therapy (ASCGT), May 16-19, 2022, at the Walter E. Washington Convention Center in Washington, D.C. The international convention attracts thousands of scientists, physicians, patient advocates and government officials…. (read more)

Genethon Joins U.S. Bespoke Gene Therapy Consortium Dedicated to Finding Treatments for Ultra-Rare Diseases

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today it has joined the U.S.-based Bespoke Gene Therapy Consortium (BGTC) launched in October 2021 by the Foundation for the National Institutes of Health (FNIH) as part of its Accelerating Medicines… (read more)

Gene Therapies Continue to Emerge as Effective Treatments for Rare Diseases, But Complex Challenges Remain

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Genethon’s Lentiviral Vector-Based Gene Therapy Demonstrates Long-Term Safety and Efficacy for Wiskott-Aldrich Syndrome

Genethon announced that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.

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