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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. Généthon: a unique firepower against rare diseases Today Genethon has 220 researchers and experts. A unique firepower against rare diseases: … [Read more]

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. As part of this partnership, Genethon teams will use … [Read more]

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. Pompe disease is a hereditary genetic disorder caused by a deficiency of acid alpha-glucosidase leading to build-up of glycogen in the lyosomes, which then causes cell damage in … [Read more]

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

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

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 natural … [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 complications generated … [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. 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]

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