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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 Genethon, a non-profit research and development organization dedicated to the development of gene therapies for orphan genetic diseases from research to clinical validation, for the development and commercialization of … [Read more]

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 injecting microdystrophin (a “shortened” version of the dystrophin gene) via a drug … [Read more]

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]