Press releases

Media contact: Stéphanie Bardon – communication@genethon.fr – 01.69.47.12.78

Genethon Presents Significant Advances in Gene Therapy at European Society for Gene and Cell Therapy, Oct 19-22, 2021

Genethon’s R&D will be featured in 6 oral presentations and 10 e-posters at the virtual conference.

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

After a first presentation at the International Liver Congress last June, new preliminary results have just been presented at the European Society of Geneand Cell Therapy (ESGCT) congress by Dr d’Antiga, one of the investigators of the trial (Bergamo, Italy). Based on initial observations, the drug candidate is well tolerated and the first… (read more)

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

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

On February 28, International Rare Disease Day will put the spotlight on the rare diseases community throughout the world. In France, Genethon, a unique not-for-profit laboratory, innovates to develop gene therapy treatments for rare diseases.

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

WhiteLab Genomics, a specialist in artificial intelligence applied to gene and cell therapies, has signed a partnership agreement with Genethon, a pioneering research center in the field of gene therapy. The alliance will harness the power of artificial intelligence to accelerate development of innovative gene therapies.

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

Genethon is delighted about the launch of a gene therapy clinical trial for late-onset Pompe disease, led by the company Spark Therapeutics, using technologies developed at Genethon.

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

Genethon, dedicated to designing and developing gene therapy products for rare diseases, received this Monday 30th of November the authorisation from the ANSM, the French National Agency for Medicines and Health Products Safety, to start in France a multicentre international clinical trial for the treatment of Duchenne muscular… (read more)

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

A Genethon team, working as part of an international collaboration, published the results of their work in Nature Medicine on June 1, 2020, opening up new perspectives for gene therapy. Using the IdeS enzyme, these researchers succeeded in inhibiting the immune response to AAV, which is caused by antibodies present as a result of… (read more)

Genethon welcomes the conclusive results of a gene therapy trial in Chronic Septic Granulomatosis, a rare disease of the immune system

Genethon, which developed the lentiviral vector used and sponsored initial clinical studies, is pleased with the conclusive results of the gene therapy trial underway in the United States and England in X-linked Chronic Septic Granulomatosis (X-CGD), a rare and severe immune dysfunction. Six of the nine patients are free of treatments related to… (read more)

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

Following an initial research and development collaboration on an innovative gene therapy product using micro-dystrophin, Genethon and Sarepta Therapeutics are now started a co-development clinical program for Duchenne muscular dystrophy.

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

The prestigious journal Nature Medicine publishes the first results of a gene therapy based clinical trial in patients with Fanconi anemia. In this study the authors demonstrate for the first time the production of blood cells derived… (read more)

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

Ana Buj Bello’s team, a researcher at Genethon, has made the proof-of-concept of a CRISPR-Cas9 approach in a mouse model of Steinert’s myotonic dystrophy, the most common neuromuscular disease in adults. (read more)

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

Isabelle Richard’s team, a CNRS researcher in an Inserm unit at Genethon, the AFM-Telethon laboratory, has demonstrated the efficacy of gene therapy and determined the effective dose for treating a rare muscle disease, gamma-sarcoglycanopathy, in mouse models of the disease. Based on these encouraging results, published in Molecular Therapy -… (read more)

Clinical Proof-of-Concept Data for OTL-102 for the Treatment of X-CGD

Orchard Therapeutics Presents Clinical Proof-of-Concept Data for OTL-102 for the Treatment of X-CGD (read more)