Gene therapy
News
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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October 21, 2024
Genethon to Showcase the Latest Advances in Gene Therapies for Multiple Diseases at the ESGCT 31st Annual Congress October 22 – 25, 2024 in Rome, Italy
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September 17, 2024
Genethon and Samabriva strengthen their partnership to develop of a cost-effective plant-based AAV manufacturing process for greater patient access to gene therapies.
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September 12, 2024
Genethon Announces Publication in Nature Communications of a Next-Generation Gene Therapy Vector for Muscle Diseases, Using AI Predictive Methodology to Improve Efficacy and Safety
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September 6, 2024
Marking World Duchenne Muscular Dystrophy Day, Genethon Reaffirms Its Commitment to Helping Patientsfor 35 years and Provides an Update on Its PromisingGene Therapy Drug Candidate for This Fatal Disease
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May 3, 2024
Genethon’s Scientists Featured in Multiple Presentations at American Society of Gene & Cell Therapy Annual Meeting May 7-11, 2024, in Baltimore, MD
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April 23, 2024
First Clinical Trial Results of Gene Therapy (GNT0004) for Duchenne Muscular Dystrophy presented at International Myology 2024 Congress
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April 22, 2024
Genethon will introduce its latest advances in gene therapy research in multiple presentations at the International Myology 2024 Congress
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March 4, 2024
Clinical results of the gene therapy trial in myotubular myopathy: efficacy on respiratory and motor functions in this extremely severe disease, challenges ahead
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February 28, 2024
Genethon Pursues Different Strategies for Ensuring Patient Access to Gene Therapies for Rare Diseases
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February 7, 2024
The American organization Team Titin awards its first research grant funding to Genethon
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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.
