Press releases

First Clinical Trial Results of Gene Therapy (GNT0004) for Duchenne Muscular Dystrophy presented at International Myology 2024 Congress

Today, Professor Francesco Muntoni, principal investigator of the international multicenter gene therapy trial for Duchenne Muscular Dystrophy with GNT0004 product, sponsored by Genethon, presented the innovative trial design and its initial results at the Myology 2024 international scientific congress, currently taking place in Paris.

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Genethon will introduce its latest advances in gene therapy research in multiple presentations at the International Myology 2024 Congress

PARIS, FRANCE (April 22, 2024) – Genethon, a unique non-profit gene therapy R&D organization, announced today its research will be featured in 3 oral presentations and 18 posters of their latest research on neuromuscular diseases at the 8th International Myology 2024 Congress in Paris, April 22 to 25, 2024.

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Genethon Pursues Different Strategies for Ensuring Patient Access to Gene Therapies for Rare Diseases

PARIS, FRANCE (February 29, 2024) – Genethon, a non-profit research organization focused on developing gene therapies for rare diseases, today marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and… (read more)

Clinical results of the gene therapy trial in myotubular myopathy : efficacy on respiratory and motor functions in this extremely severe disease, challenges ahead

In a press release co-signed with the Institute of Myology, Genethon announces that The Lancet Neurology published yesterday the clinical results of a gene therapy trial conducted by Astellas Gene Therapies using a drug candidate developed at Genethon in 24 children suffering from myotubular myopathy, a very severe muscle disease.

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The Latest Research of 14 Généthon Scientists to be Featured at the European Society of Gene & Cell Therapy’s 30th Annual Congress, Oct. 24-27, 2023, in Brussels, Belgium

The gene therapy pioneering organization’s research, aimed at curing rare and ultra-rare genetic diseases, is featured in 3 oral and 11 poster presentations.

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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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