Gene therapy
News
October 10, 2025
Data from GNT-018-IDES trial supports feasibility of imlifidase as pretreatment in gene therapy treatment for patients with Crigler–Najjar syndrome who are immune to AAV
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October 9, 2025
Genethon Confirms 2-year Efficacy in Duchenne Muscular Dystrophy Patients Treated with Its Low Dose Micro-Dystrophin Gene Therapy (GNT0004) at the ESGCT 32nd Annual Congress
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October 1, 2025
Genethon to Present Long-Term Follow-Up of Duchenne Muscular Dystrophy Patients Treated in Its Phase 1/2/3 Trial at the European Society of Gene and Cell Therapy 32nd Annual Congress October 7 – 10, 2025
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July 28, 2025
Genethon to Launch Pivotal Trial in Europe of GNT0004 a Low-Dose Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
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June 18, 2025
Clinical Milestones, Vector Innovation Key to Gene Therapy, GenoTher Biocluster’s First International Summit, Atamyo Therapeutics’ Progress for LGMDs Highlight Genethon’s Most Recent Newsletter
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May 17, 2025
Duchenne Muscular Dystrophy: Genethon presents Two-Year consolidated results of the GNT0004 Gene Therapy
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May 17, 2025
Genethon Presents Two Year Consolidated Results of Its Gene Therapy Trial for Duchenne Muscular Dystrophy: Maintenance of Motor Functions and Significant, Sustained Reduction in CPK Levels in Patients Treated at the Effective Dose at ASGCT 2025
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May 16, 2025
New advances in the treatment of ALS: Genethon's DNA bank involved in the trial
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May 13, 2025
Genethon arrives in force at ASGCT 2025 with 12 communications, including 5 oral presentations
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March 20, 2025
High-throughput screening identification of two molecules as potential approaches for treating a form of limb-girdle muscular dystrophy (LGMD-R2)
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March 7, 2025
Gene therapy for sickle cell anemia: Genethon has contributed its expertise to the design of an innovative vector
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February 27, 2025
Genethon Celebrates Rare Disease Day by Highlighting New Technologies to Advance Gene Therapies and Bring Hope to Millions of Patients Worldwide
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January 23, 2025
Genethon and Eukarÿs announce a strategic partnership to develop a breakthrough technology to reduce the biomanufacturing cost of gene therapies
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December 19, 2024
Ana Buj Bello, winner of the Léon Baratz Prize, Docteur Darolles for her research on a gene therapy to cure myotubular myopathy
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December 3, 2024
Genethon and Hansa Biopharma announce initiation of a Phase 2 trial of imlifidase as a pre-treatment to GNT-0003 in severe Crigler-Najjar syndrome
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November 19, 2024
Genethon Presents Positive Initial Results from a Phase 1/2/3 Trial of its Gene Therapy (GNT0004) for Duchenne Muscular Dystrophy at ASGCT Breakthroughs in Muscular Dystrophy in Chicago
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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
Act for the general interest
Our mission: the design and preclinical and clinical development of gene therapy drugs for rare diseases.
Our objective: to make these innovative treatments available to patients.
