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

Preliminary results from the European gene therapy trial for Crigler-Najjar syndrome, conducted by Généthon in collaboration with European network CureCN, were presented at the EASL (European Association for the Study of the Liver) annual International Liver Congress on June 26. Based on initial observations, the drug candidate is well tolerated and the first therapeutic effects have … [Read more]

Cholesterol Metabolism: A Potential Therapeutic Target in Duchenne Muscular Dystrophy

David Israeli, his student Ai Vu Hong PhD, and Fatima Amor, all members of the Progressive Dystrophies laboratory directed by Isabelle Richard at GENETHON, have published the results of research offering new perspectives on the treatment of Duchenne muscular dystrophy in the Journal of Cachexia, Sarcopenia and Muscle) The researchers characterized a mechanism of metabolic … [Read more]

Genethon’s teams present their progress at the ASGCT meeting

Limb girdle muscular dystrophies, rare diseases of the liver or production of vectors on a large scale: these are some of the topics presented by Genethon’s teams at the annual meeting of the American Society for Gene and Cell Therapy (ASGCT), to be held virtually from May 11 to 14.   The annual meeting of … [Read more]

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

A first participant was dosed at I-Motion, the pediatric clinical trial platform for neuromuscular diseases located at Trousseau hospital in Paris, as part of the gene therapy trial in Duchenne muscular dystrophy (DMD) conducted by Genethon. A young boy suffering from Duchenne muscular dystrophy received a first dose of the investigational gene therapy GNT 0004 … [Read more]

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]