News

Preliminary results of clinical trial for rare liver disease Crigler-Najjar syndrome presented at ESGCT congress

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Cholesterol Metabolism: A Potential Therapeutic Target in Duchenne Muscular Dystrophy

David Israeli, his student Ai Vu Hong PhD, and Fatima Amor, all members of the Progressive Dystrophies laboratory directed by Isabelle Richard at Genethon, have published the results of research offering new perspectives on the treatment of Duchenne muscular dystrophy in the (read more)

Genethon’s teams present their progress at the ASGCT meeting

Limb girdle muscular dystrophies, rare diseases of the liver or production of vectors on a large scale: these are some of the topics presented by Genethon’s teams at the annual meeting of the American Society for Gene and Cell Therapy (ASGCT), to be held virtually from May 11 to 14.

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Genethon announces First Patient dosed in Clinical Trial of Investigational Gene therapy GNT 0004 for Duchenne Muscular Dystrophy

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Genethon, 30 years of pioneering research and innovation in treating rare diseases

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Genethon and WhiteLab Genomics join forces to enhance gene therapy through artificial intelligence

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Genethon is delighted about the launch of a gene therapy clinical trial for late-onset Pompe disease

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Genethon gets the green light from the ANSM to start an innovative gene therapy trial for Duchenne muscular dystrophy

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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)

GenSight Biologics, a french biopharma company, just submitted to EMAMarketing Autorisation Application for LUMEVOQ® Gene Therapy to Treat Vision Loss due to Leber Hereditary Optic Neuropathy (LHON), a rare, mitochondrial genetic disease, caused by mutation in the ND4 mitochondrial gene.

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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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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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Genethon strengthens its collaboration with Sarepta Therapeutics for the development of the gene therapy product GNT0004 in Duchenne muscular dystrophy

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A gene therapy based clinical trial for Fanconi anemia patients offers its first successful results

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First in vivo proof-of-concept in Steinert’s myotonic dystrophy, a neuromuscular disease

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Towards a clinical trial for gamma-sarcoglycanopathy, limb-girdle muscular dystrophy

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