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RSS feedASGCT 2026: Genethon presents results from three gene therapy programs in clinical development
At ASGCT 2026 in Boston, Genethon presented new clinical data from three of its gene therapy programs in Duchenne muscular dystrophy, gamma-sarcoglycanopathy (LGMD-R5), and Crigler-Najjar syndrome. In total, Genethon teams presented 8 oral presentations and 13 posters. Genethon, the French laboratory dedicated to the research and development of gene therapies for rare diseases created by … [Read more]
ASGCT 2026: Atamyo Therapeutics presents encouraging ATA-200 results in LGMD-R5
Atamyo Therapeutics presented the first safety, pharmacodynamics, and efficacy data from its ATA-200 gene therapy in LGMD-R5 at the ASGCT 2026 Annual Meeting. Results observed in the first treated patients showed high SGCG protein expression together with improvements in several biological and functional parameters First results presented at ASGCT 2026 Atamyo Therapeutics presented the first … [Read more]
Téléthon 2025: 100,548,314 euros raised
More than ever, the French people said “Yessss” to the Téléthon! The exceptional Téléthon 2025 fundraising total reflects the remarkable trust and loyalty of volunteers, donors and partners committed alongside us. A huge thank you to each and every one of you! The final Téléthon 2025 total stands at €100,548,314. An exceptional result reflecting a … [Read more]
Klaudia Kuranda joins Genethon and the Institute of Myology to create a center of expertise dedicated to immunology
An internationally recognized expert in the field of immunology and the development of gene therapies, Klaudia Kuranda is joining the AFM-Téléthon laboratories to establish and lead a new Immunology Center that will bring together the complementary expertise of Genethon, a leading gene therapy non-profit biotech organization, and the Institute of Myology, a center of expertise … [Read more]
Progress in Treating Patients Immunised Against AAV Vectors Used in Gene Therapy
A patient with Crigler–Najjar syndrome, immunised against the AAV vector used in gene therapy, was able to receive treatment as part of a Genethon-led trial, thanks to the prior administration of imlifidase, an enzyme developed by Hansa Biopharma. Read more
Mitogether.com: the new reference website on genetic mitochondrial diseases
To celebrate World Mitochondrial Disease Week, the “MitoGether” consortium, uniting 12 patient and family associations, is launching Mitogether.com. This new portal aims to become the leading online resource for everyone affected by a genetic mitochondrial disease: patients, families, healthcare professionals, researchers, and pharmaceutical industry stakeholders. Read more
Duchenne Muscular Dystrophy: Start of the pivotal phase of Genethon’s gene therapy trial
A new milestone has been reached in the clinical trial of gene therapy conducted by the Généthon laboratory for Duchenne muscular dystrophy. Building on the positive results of the dose escalation phase, which demonstrated the safety and efficacy of the second dose tested, Genethon has just obtained authorization from the European Medicines Agency (EMA) (with … [Read more]
Cure Through Innovation 2025: AFM-Téléthon, Driving Force Behind a New Medicine
Discover Cure Through Innovation 2025, the reference brochure presenting at a glance the core missions, commitments, and achievements of AFM-Téléthon. An essential document to grasp the diversity of its actions and the emergence of an innovative medicine born from 65 years of fighting. Read more
GenoTher Summit 2025: An International Summit to Sharpen the Future of Gene Therapy
On June 11, 2025, Généthon, the AFM-Téléthon laboratory, is co-organizing the GenoTher Summit 2025 in Évry, a major international scientific event bringing together the gene therapy ecosystem with a shared ambition: sharpening the strategic trajectory and industrial readiness of innovative treatments for rare diseases and beyond. Read more
Gene Therapy: Two-Year Consolidated Results from Genethon’s Clinical Trial for Duchenne Muscular Dystrophy
Presented on May 17 at the 2025 Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), the results of Genethon’s trial for Duchenne Muscular Dystrophy show stabilization of motor functions and a significant, sustained reduction in CPK levels in patients treated at the effective dose. The pivotal phase is scheduled to begin … [Read more]
