Gene therapy
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
6 January 2022
Genethon Announces Publication of Results paving the way for Gene Therapy for XLH, a Skeletal Disorder
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20 October 2021
Genethon Presents Significant Advances in Gene Therapy at European Society for Gene and Cell Therapy, Oct 19-22, 2021
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28 June 2021
Preliminary results of clinical trial for rare liver disease Crigler-Najjar syndrome presented at ESGCT congress
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23 April 2021
Genethon announces First Patient dosed in Clinical Trial of Investigational Gene therapy GNT 0004 for Duchenne Muscular Dystrophy
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28 February 2021
Genethon, 30 years of pioneering research and innovation in treating rare diseases
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10 February 2021
Genethon and WhiteLab Genomics join forces to enhance gene therapy through artificial intelligence
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3 February 2021
Genethon is delighted about the launch of a gene therapy clinical trial for late-onset Pompe disease
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2 December 2020
Genethon gets the green light from the ANSM to start an innovative gene therapy trial for Duchenne muscular dystrophy
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21 September 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)
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10 June 2020
A Genethon team succeeds in inhibiting the immune response linked to AAV, opening up the possibility of treating more patients by gene therapy
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30 January 2020
Genethon welcomes the conclusive results of a gene therapy trial in Chronic Septic Granulomatosis, a rare disease of the immune system
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9 January 2020
Genethon strengthens its collaboration with Sarepta Therapeutics for the development of the gene therapy product GNT0004 in Duchenne muscular dystrophy
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9 September 2019
A gene therapy based clinical trial for Fanconi anemia patients offers its first successful results
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26 June 2019
First in vivo proof-of-concept in Steinert’s myotonic dystrophy, a neuromuscular disease
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24 May 2019
Towards a clinical trial for gamma-sarcoglycanopathy, limb-girdle muscular dystrophy
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10 December 2018
Genethon announces dosing of the first patient with Crigler–Najjar Syndrome
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5 October 2018
Co-administration of AAV Vectors with SVP-Rapamycin Enables Vector Re-administration in Pre-clinical Gene Therapy Study Published in Nature Communications by Généthon and Selecta Biosciences
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12 February 2018
Genethon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler-Najjar Syndrome
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