Press releases

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

Genethon’s R&D to be Featured in Multiple Presentations at ASGCT Annual Meeting, May 16-19

Genethon will be featured in six presentations at the 25th Annual Meeting of the American Society of Cell and Gene Therapy (ASCGT), May 16-19, 2022, at the Walter E. Washington Convention Center in Washington, D.C. The international convention attracts thousands of scientists, physicians, patient advocates and government… (read more)

Genethon Joins U.S. Bespoke Gene Therapy Consortium Dedicated to Finding Treatments for Ultra-Rare Diseases

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today it has joined the U.S.-based Bespoke Gene Therapy Consortium (BGTC) launched in October 2021 by the Foundation for the National Institutes of Health (FNIH) as part of its Accelerating… (read more)

Gene Therapies Continue to Emerge as Effective Treatments for Rare Diseases, But Complex Challenges Remain

To mark International Rare Disease Day, Genethon identifies the most urgent priorities in overcoming these obstacles.

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Genethon’s Lentiviral Vector-Based Gene Therapy Demonstrates Long-Term Safety and Efficacy for Wiskott-Aldrich Syndrome

Genethon announced that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.

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Genethon Announces Publication of Results paving the way for Gene Therapy for XLH, a Skeletal Disorder

The AAV-based therapy targets the liver’s hepatocytes to express therapeutic proteins for secretion into the bloodstream and long-lasting treatment of the rare disease.

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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)