News

Spark Therapeutics Enters into Licensing Agreement with Genethon

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Audentes Therapeutics announces dosing of first patient in ASPIRO for the treatment of X-Linked Myotubular Myopathy

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The American organization Team Titin awards its first research grant funding to Genethon

Team Titin, a non-profit organization, will fund $50,000 to support the project “Generation and characterization of skeletal muscle organoids from TTN patients’ iPSC for therapeutic screening”. (read more)

Genethon evaluates a new therapeutic approach for glycogen storage disease III

In a study published in the « The Journal of Clinical Investigation » on November 28, Jérémy Rouillon, a research engineer at Genethon and Antoine Gardin, a pediatrician and PhD student at Généthon, in the Immunology and Liver Diseases team led by Giuseppe Ronzitti, have designed an innovative strategy that provides a truncated version of… (read more)

NEWSLETTER : “We have multiple examples of how these technologies can be used to develop products that more efficient and safer with improved production technologies”

CEO Frederic Revah discusses how Artificial Intelligence is creating next-generation gene therapy vectors and reducing bioproduction costs in the latest issue of Genethon’s Newsletter. (read more)

Myotubular myopathy: gene therapy trial results demonstrate major improvement of motor and respiratory functions

The Lancet Neurology published on November 15 the clinical results of a gene therapy trial using a drug candidate developed at Genethon in 24 children suffering from myotubular myopathy, a rare genetic muscle disease. (read more)

First clinical results of a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy presented at ESGCT Congress

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Genethon assesses a therapeutic potential of a Dual AAV vector approach for Duchenne muscular dystrophy

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Genethon’s Optidys project rewarded by BpiFrance as part of France 2030

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Genethon researchers involved in the MAGIC international consortium

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”A milestone in gene therapy: proof of long-term efficacy”

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Research work carried out at Genethon rewarded at the 26th edition of the ASGCT

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Chronic septic granulomatosis: biomarkers predict the efficacy of gene therapy

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“We made important achievements in 2022 but we still face key challenges in the year ahead”

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Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease

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