Articles from Genethon

Genethon unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, May 13 – 17, 2025. Five patients, aged 6 to 10 years, were treated, 2 at the first dose level and 3 at the second dose level (3x10¹³ vg/kg). The initial part of the clinical trial aimed at selecting the optimal dose (dose escalation phase), evaluating the tolerance and preliminary efficacy of the treatment and determined the effective dose for the pivotal phase of the GNT-016-MDYF trial, which is expected to start in mid-2025 (3x10¹³ vg/kg).

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 of these life-saving medicines and make them more accessible for patients across the globe.

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), achieved several major clinical development and scientific milestones over the past several months as highlighted in its year-end Newsletter.

Hansa Biopharma, “Hansa” (Nasdaq Stockholm: HNSA) and Genethon, a pioneer and a leader in gene therapy research and development for rare genetic diseases, today announced initiation of GNT-018-IDES, a Phase 2 trial in patients with Crigler-Najjar syndrome with pre-existing antibodies against adeno-associated virus (AAV) vectors. The trial will evaluate the efficacy and safety of a single intravenous administration of Genethon’s gene therapy GNT-0003 following pre-treatment with imlifidase, Hansa’s first-in-class immunoglobulin G (IgG) antibody cleaving enzyme therapy, in patients with severe Crigler-Najjar syndrome and pre-formed antibodies to AAV serotype 8 (AAV8).

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, 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 expects to launch pivotal trial in Europe in Q2/2025 and in the US.

Genethon, the French laboratory created by AFM-Telethon, announced today that 20 scientists will present the latest advances in development of gene therapies for many different types of diseases at the European Society of Gene & Cell Therapy’s 31st Annual Congress October 22 - 25, 2024 in Rome, Italy.

Genethon, the pioneering French laboratory and leader in the research and development of gene therapies for rare diseases, announced today publication in Nature Communications of an article describing the use of artificial intelligence (AI) in designing a new generation of capsids, the structures that envelop genetic material of adeno-associated viruses (AAV), to improve gene therapies for muscle diseases.

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 solution to overcome this fatal disease.

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), provided updates in its latest Newsletter on results of its gene therapy clinical trials for Duchenne Muscular Dystrophy and advances in gene therapies for limb-girdle muscular dystrophies by its spinoff Atamyo Therapeutics.

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, 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 Duchenne muscular dystrophy.

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), provided an update on its efforts to employ Artificial Intelligence (AI) in revolutionizing the development and bioproduction of gene therapies.

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), today announced The Lancet Neurology published online preliminary results of a clinical trial conducted by Astellas Pharma using a gene therapy developed by Genethon for X-linked myotubular myopathy, a rare genetic muscle disease affecting 1 in 50,000 newborn boys.

Genethon, a pioneer and leader in gene therapy research and development for rare genetic diseases, announced today positive Phase 1/2 clinical trial results of its gene therapy, GNT-0003, for Crigler-Najjar syndrome were published in The New England Journal of Medicine. The data demonstrate the possibility of restoring expression of liver enzyme UGT1A1 by using gene therapy in cases of Crigler-Najjar syndrome.

Genethon, a pioneer in gene therapy, said today the French government’s support of the GenoTher Biocluster validates the efforts of its founding organizations in creating a global center of excellence in gene therapy and establishes their work and dedication as a national priority for advancing gene therapies to treat both rare and common diseases.

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), today announced that the European Medicines Agency (EMA) has granted PRIME (Priority Medicines) status to the gene therapy, GNT-0003, currently in clinical trials for Crigler-Najjar syndrome, a rare liver disease. This status, granted only to drug candidates with major therapeutic potential, follows the successful completion of the first phases of the clinical trials.

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), has launched a pivotal clinical trial in Europe for treatment of Crigler-Najjar syndrome, a life threatening liver disease. The trial of the gene therapy, GNT-003, will be conducted in France, Italy and the Netherlands and will enroll patients aged 10 years and older with the objective of confirming efficacy and safety seen in the previous clinical part.

Genethon, a non-profit research organization focused on developing gene therapies for rare diseases, today recognizes International Rare Disease Day by reflecting on successes and highlighting challenges that remain in bringing hope to 300 million patients worldwide who suffer from more than 7,000 rare diseases.