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Evidence of efficacy of gene therapy in rodents affected by a rare genetic liver disease, Crigler-Najjar syndrome

Federico Mingozzi, head of the Immunology and Liver Gene Therapy team at Généthon, the laboratory created by the AFM Téléthon, presented at the 48th Annual Meeting of the European Society for Pediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN, May 6-9, Amsterdam), work done in collaboration with an Italian and Dutch teams showing long term correction of a genetic defect causing toxic … [Read more]

Généthon, Winner of the World Innovation Competition 2030

Tuesday April 28, during a ceremony at the Elysee Palace, Généthon, the laboratory of the AFM-Telethon, was named recipient of the Global Innovation Competition 2030 in the category “Risk lift.” A prize that rewards the expert laboratory and world leader in the field of gene therapy for the development of an industrial production process of gene therapy vectors. Read … [Read more]

New gene therapy success in a rare disease of the immune system: Wiskott-Aldrich syndrome

French teams from CIC Biothérapie (AP-HP/Inserm), from pediatric hematology department of Necker Hospital for Children (AP-HP), led by Marina Cavazzana, Salima Hacein Bey Albina and Alain Fischer and from Genethon led by Anne Galy (Genethon/Inserm UMR-S951), and English teams from UCL Institute of Child Health and Great Ormond Street Hospital in London led by Adrian Thrasher and Bobby Gaspar demonstrated … [Read more]

Effectiveness of innovative gene therapy treatment demonstrated in canine model of Duchenne muscular dystrophy

A collaboration involving three laboratories supported by the AFM-Telethon, Atlantic Gene Therapies (AFM-Telethon, Inserm UMR 1089, Université de Nantes, Nantes University Hospital), Généthon (Evry) and the Institute of Myology (Paris), demonstrated the effectiveness of an innovative gene therapy treatment in the canine model of Duchenne muscular dystrophy. This work, published in the journal Molecular Therapy in November, was mainly financed … [Read more]

GENETHON and ESTEVE announce agreement to manufacture the gene therapy for the treatment of Sanfilippo Syndrome

France – March 11, 2014 –ESTEVE, a Spanish pharmaceutical company devoted to the research, development, manufacturing and commercialization of novel medicines and Genethon, a non‐profit organization dedicated to the research and development of gene therapies for orphan genetic diseases, announce that they have entered into an agreement to manufacture Esteve’s investigational gene therapy for the … [Read more]

Audentes Therapeutics and Genethon Announce Agreement to Develop Treatment for Severe Genetic Disease X‐Linked Myotubular Myopathy

SAN FRANCISCO, CA – February 5, 2014 –Audentes Therapeutics, Inc., a biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, and Genethon, a non‐profit organization dedicated to the research and development of biotherapies for orphan genetic diseases, announce that they have entered into an agreement to develop AT001 for the treatment of X‐Linked Myotubular Myopathy … [Read more]

Efficacy of gene therapy demonstrated in canine and murine models of myotubular myopathy

Evry (France), 22 January, 2014. A team of French researchers, led by Dr. Anna Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, have demonstrated the efficacy of gene therapy in models of myotubular myopathy, an extremely severe neuromuscular disease in children. Transfer of the MTM1 gene, … [Read more]

Généthon, the French AFM-Telethon laboratory, becomes the first not-for-profit to obtain authorization from ANSM to be a pharmaceutical manufacturer

Evry, June 27, 2013. Généthon, the AFM-Telethon laboratory, has received the authorization delivered by the National Agency for Drug Safety (ANSM) to become a pharmaceutical manufacturer. Its production center, Généthon BioProd, is now authorized to produce drugs for innovative treatments. This is a first for a laboratory created by an association of patients and financed … [Read more]

The European charity Myotubular Trust is supporting Généthon in developing gene therapy for myotubular myopathy

Evry (France), June 24, 2013. The European charity Myotubular Trust announces its support to the development of a pre-clinical gene therapy trial for myotubular myopathy, a project co-ordinated by Dr. Anna Buj-Bello at Généthon (Evry, France). Read the press release – 24/06/2013 (2 pages – 46,2 ko)

Genethon receives approval from the UK medicine regulatory agency to start a new clinical trial for an inherited immune deficiency: chronic granulomatous disease

On January 10th, the British Medicine and Healthcare Regulatory Agency (MHRA) approved Genethon’s Phase I / II gene therapy clinical trial application in X-linked chronic granulomatous disease (XCGD) to start at Great Ormond Street Hospital in London. Genethon is also seeking approval for this multicenter trial in Germany, Switzerland and France. In total the trial … [Read more]