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Gene therapy
treatments for
rare diseases

Serving the public interest

Our mission: Conception, clinic and preclinic development of drugs of genic therapy for rare diseases
Our goal: Providing these innovative treatments to the sick

Our science

News

September 26, 2022 First Patient Dosed in Phase 1/2 Clinical Trial in Europe of Gene Therapy for LGMD-R9 (read more)
September 12, 2022 Genethon to Present Its Latest Gene Therapy Research Advancements in Neuromuscular Diseases at the International Myology 2022 Congress (read more)
May 16, 2022 La R&D de Généthon à l'honneur dans plusieurs présentations à la réunion annuelle de l'American Society of Cell & Gene Therapy du 16 au 19 mai 2022, à Washington, DC (read more)
May 16, 2022 Genethon’s R&D to be Featured in Multiple Presentations at ASGCT Annual Meeting, May 16-19 (read more)
April 4, 2022 Genethon Joins U.S. Bespoke Gene Therapy Consortium Dedicated to Finding Treatments for Ultra-Rare Diseases (read more)
February 28, 2022 Gene Therapies Continue to Emerge as Effective Treatments for Rare Diseases, But Complex Challenges Remain (read more)
February 10, 2022 Genethon’s Lentiviral Vector-Based Gene Therapy Demonstrates Long-Term Safety and Efficacy for Wiskott-Aldrich Syndrome (read more)
January 6, 2022 Genethon Announces Publication of Results paving the way for Gene Therapy for XLH, a Skeletal Disorder (read more)
October 20, 2021 Genethon Presents Significant Advances in Gene Therapy at European Society for Gene and Cell Therapy, Oct 19-22, 2021 (read more)
June 28, 2021 Preliminary results of clinical trial for rare liver disease Crigler-Najjar syndrome presented at ESGCT congress (read more)
April 23, 2021 Genethon announces First Patient dosed in Clinical Trial of Investigational Gene therapy GNT 0004 for Duchenne Muscular Dystrophy (read more)
February 28, 2021 Genethon, 30 years of pioneering research and innovation in treating rare diseases (read more)
February 10, 2021 Genethon and WhiteLab Genomics join forces to enhance gene therapy through artificial intelligence (read more)
February 3, 2021 Genethon is delighted about the launch of a gene therapy clinical trial for late-onset Pompe disease (read more)
December 2, 2020 Genethon gets the green light from the ANSM to start an innovative gene therapy trial for Duchenne muscular dystrophy (read more)
September 21, 2020 Genethon welcomes the submission to EMA of Marketing Authorisation Application for LUMEVOQ® Gene Therapy to Treat Vision Loss due to Leber Hereditary Optic Neuropathy (LHON) (read more)
June 10, 2020 A Genethon team succeeds in inhibiting the immune response linked to AAV, opening up the possibility of treating more patients by gene therapy (read more)
January 30, 2020 Genethon welcomes the conclusive results of a gene therapy trial in Chronic Septic Granulomatosis, a rare disease of the immune system (read more)
January 9, 2020 Genethon strengthens its collaboration with Sarepta Therapeutics for the development of the gene therapy product GNT0004 in Duchenne muscular dystrophy (read more)
September 9, 2019 A gene therapy based clinical trial for Fanconi anemia patients offers its first successful results (read more)