Press Releases

RSS feed

New partnership for the treatment of Duchenne muscular disease (DMD)

Sarepta Therapeutics, a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and Genethon, have signed a gene therapy research collaboration to jointly develop treatments for Duchenne muscular dystrophy (DMD). Genethon’s micro-dystrophin gene therapy approach can target the majority of patients with DMD. … [Read more]

New step towards the treatment of myotubular myopathy: gene therapy restores strength and prolongs lives in affected dogs

Evry (France), 5 April, 2017. A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy. The researchers demonstrated the efficacy of administration of a therapeutic … [Read more]

Creation of YposKesi

AFM-Téléthon and the SPI fund, managed by Bpifrance under the Programme d’Investissement d’Avenir, are creating YposKesi, the first French industrial pharmaceutical company dedicated to producing gene and cell therapy drugs for rare  diseases.

Genethon and CRISPR Therapeutics announce Research Collaboration

EVRY, France, BASEL, Switzerland and CAMBRIDGE, Mass. – December 16th, 2015 – Généthon, a leader in the field of gene therapy treatments for rare diseases, and CRISPR Therapeutics, a biopharmaceutical company focused on developing transformative genebased medicines for patients with serious diseases, have today announced an ongoing research collaboration. Généthon is supporting the development of one CRISPR Therapeutics’ undisclosed propriety … [Read more]

Genethon:a gene therapy drug being tested in the US

Phase I/II clinical trial to enroll 10 patients with an immunodeficiency disorder at three different U.S. locations. Read the press release

ASGCT 18th Annual Meeting 2015: Ana Buj Bello receives the Outstanding New Investigator Award

Dr. Ana Buj Bello, Inserm research scientist and head of the neuromuscular disorders team at Genethon, received the Outstanding New Investigator Award from the American Society of Gene & Cell Therapy (ASGCT) at the 18th Annual Meeting (May 13-16, 2015/New Orleans). This award recognizes four researchers every year for the quality and importance of their work in gene and cell … [Read more]

Selecta and Genethon Collaborate to Create Next Generation Gene Therapies Using Selecta’s Synthetic Vaccine Particle Platform

Watertown, Mass., USA, and Evry, France – May 13, 2015 – Selecta Biosciences, Inc. and Genethon today announced an ongoing research collaboration with the goal of enabling repeat dosing for gene therapies. Based on preliminary results, the companies have identified three applications that might benefit from combining Généthon’s expertise in the development of gene therapy vectors and Selecta’s Synthetic … [Read more]

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]