Press releases

Genethon Celebrates Rare Disease Day by Highlighting New Technologies to Advance Gene Therapies and Bring Hope to Millions of Patients Worldwide

PARIS, FRANCE (February 27, 2025) – Genethon, a pioneering gene therapy research organization created by the AFM-Telethon, marks Rare Disease Day February 28, 2025, by highlighting recent technological breakthroughs signaling a next generation of safer, more effective and lower cost gene therapies to expand applications… (read more)

Genethon and Eukarÿs announce a strategic partnership to develop a breakthrough technology to reduce the biomanufacturing cost of gene therapies

Evry, January 23, 2025- Genethon, a pioneer and leader in the research and development of gene therapies for rare diseases, and Eukarÿs, a biotechnology company developing a disruptive technology that significantly increases biomanufacturing yields, today announced the signing of a strategic partnership. The collaboration aims to reduce the… (read more)

Genethon and Hansa Biopharma announce initiation of a Phase 2 trial of imlifidase as a pre-treatment to GNT-0003 in severe Crigler-Najjar syndrome

The trial will be conducted in patients with pre-existing anti-AAV antibodies which limit use of gene therapy treatment

Genethon Presents Positive Initial Results from a Phase 1/2/3 Trial of its Gene Therapy (GNT0004) for Duchenne Muscular Dystrophy at ASGCT Breakthroughs in Muscular Dystrophy in Chicago

PARIS, FRANCE (November 19, 2024) – Genethon, a pioneering gene therapy research organization created by AFM-Telethon, today presented positive results from the Phase 1/2 dose escalation part of an international multicenter all-in-one Phase 1/2/3 trial evaluating its gene therapy, GNT-0004, for Duchenne muscular dystrophy (DMD) at the (read more)

Genethon to Showcase the Latest Advances in Gene Therapies for Multiple Diseases at the ESGCT 31st Annual Congress October 22 – 25, 2024 in Rome, Italy

Twenty Genethon researchers and collaborators will be featured in four oral and 18 poster presentations

Genethon and Samabriva strengthen their partnership to develop of a cost-effective plant-based AAV manufacturing process for greater patient access to gene therapies.

Amiens, France – 17th of September 2024 – Genethon, a pioneer and leader in gene therapy research and development for rare genetic diseases and Samabriva, a plant-based biotechnology company are pleased to announce their continued collaboration through on a project that will complete the development of a new way of producing AAVs using… (read more)

Genethon Announces Publication in Nature Communications of a Next-Generation Gene Therapy Vector for Muscle Diseases, Using AI Predictive Methodology to Improve Efficacy and Safety

Isabelle Richard, Ph.D., and her Progressive Muscular Dystrophies Team at Genethon have perfected an innovative methodology based on artificial intelligence (AI) to design a new generation of capsids for developing more effective gene therapy vector for muscle diseases.

Marking World Duchenne Muscular Dystrophy Day, Genethon Reaffirms Its Commitment to Helping Patientsfor 35 years and Provides an Update on Its PromisingGene Therapy Drug Candidate for This Fatal Disease

PARIS, FRANCE (September 6, 2024) – Genethon, a non-profit research organization and world leader in gene therapies for rare diseases, is marking World Duchenne Muscular Dystrophy Day on September 7, 2024, by reiterating its 35-year commitment to the families affected by Duchenne muscular dystrophy and its determination to offer patients a… (read more)

Genethon’s Scientists Featured in Multiple Presentations at American Society of Gene & Cell Therapy Annual Meeting May 7-11, 2024, in Baltimore, MD

GenoTher, biocluster in the field of gene therapy is officially launched

On March 25th, founders and partners of GenoTher, the award-winning biocluster of the French Innovation Santé 2030 plan, gathered for the association’s first constituent assembly and board meeting. This key milestone marks the launch of GenoTher, and the creation of an ecosystem unique in France and Europe to meet the scientific,… (read more)

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.

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.

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.

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.