Press Releases

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 dystrophy with product GNT 004. This trial’s … [Read more]

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. LHON mainly affects young males, between 15 and 30 years old. The ND4 mutation results … [Read more]

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 natural … [Read more]

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 complications generated … [Read more]

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. Gene therapy combining an AAV-type viral vector and a shortened version of the dystrophin gene (microdystrophin) is an innovative technology developed by Genethon researchers in … [Read more]

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 from the patients’ genetically corrected stem cells. This clinical trial has been carried out by members of a … [Read more]

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 the press release

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 – Methods and Clinical Development, the … [Read more]

Orchard Therapeutics Presents Clinical Proof-of-Concept Data for OTL-102 for the Treatment of X-CGD Six Patients Continue to Show Sustained Levels of Functioning Neutrophils After 12 Months and No Longer Receive Treatment with CGD-related Prophylactic Antibiotics. Regulatory Discussions on Registrational Trial Design Planned for 2019. Read the press release More Information

Genethon announces dosing of the first patient with Crigler–Najjar Syndrome, a rare liver disorder, in the European phase I/II gene therapy clinical trial – CareCN. Following patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. Read the press release