News
RSS feedKlaudia 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
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]
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]
ALS: The MIROCALS Trial Paves the Way for a New Treatment
The results of the European MIROCALS trial provide new insights into a promising therapeutic approach for Amyotrophic Lateral Sclerosis (ALS). Low-dose interleukin-2 could slow disease progression in certain patients. A study supported by AFM-Téléthon and its laboratory, Généthon, through its biobank. Read more
€96,553,593 – an outstanding result for Telethon 2024!
€ 96, 553, 593 : this is the final amount raised during the Telethon held on 29 and 30 November 2024. This result reflects an extraordinary outpouring of solidarity, driven by millions of donors, volunteers and partners who have stood by families in their fight for nearly 40 years. It is also the outcome of … [Read more]
Genethon and Hansa Biopharma launch a phase 2 clinical trial for Crigler-Najjar syndrome
The trial will be conducted in patients for whom pre-existing anti-AAV antibodies are currently a limitation to gene therapy treatment. Genethon, the Telethon laboratory, and Hansa Biopharma, a pioneer in enzyme technology for rare immunological diseases, today announced the launch of a phase 2 trial in patients suffering from Crigler-Najjar disease, a rare liver disorder, … [Read more]
Genethon Presents Promising Results for Gene Therapy in DMD
Genethon, the pioneering gene therapy research laboratory created by AFM-Telethon, today presented positive results from the Phase 1/2 dose escalation part of the trial evaluating its gene therapy, GNT0004, for Duchenne muscular dystrophy (DMD) at the ASGCT Breakthroughs in Muscular Dystrophy conference, Nov. 19 – 20, 2024, in Chicago, IL. Based on these results, Genethon … [Read more]
World Duchenne Awareness Day: Genethon at the forefront of the fight against Duchenne muscular dystrophy
World Duchenne Muscular Dystrophy Day provides an opportunity to note the acceleration in the development of innovative therapies for this neuromuscular disease, which is the most common in children. Gene therapy is one of the most promising avenues. Genethon, the AFM-Telethon laboratory, is currently developing a drug candidate which has demonstrated its efficacy in the … [Read more]
