September 21, 2017
Audentes Therapeutics announces dosing of first patient in ASPIRO for the treatment of X-Linked Myotubular Myopathy
Preliminary data from ASPIRO expected to be available in the fourth quarter of 2017
(read more)September 20, 2017
Spark Therapeutics Enters into Licensing Agreement with Genethon
Spark Therapeutics, a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, today announced it has entered into a licensing agreement with… (read more)
July 25, 2017
Restore muscle strength in Duchenne muscular dystrophy through microdystrophin
Gene therapy: Microdystrophin restores muscle strength in Duchenne muscular dystrophy. Researchers from Genethon, the AFM-Téléthon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy. Indeed, after… (read more)
June 21, 2017
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… (read more)
April 5, 2017
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… (read more)
November 3, 2016
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.
(read more)December 18, 2015
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… (read more)
July 28, 2015
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 more)May 15, 2015
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… (read more)
May 13, 2015
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… (read more)
May 7, 2015
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… (read more)
April 28, 2015
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… (read more)
April 21, 2015
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… (read more)
November 10, 2014
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… (read more)
March 11, 2014
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… (read more)
