Pierre, suffering from limb girdle muscular dystrophy: “For me, a cure would mean no more losing”
… (read more)Spinal muscular atrophy: for Victoire’s mom, treatment was a magical moment
… (read more)June 28, 2021
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)
May 27, 2021
Cholesterol Metabolism: A Potential Therapeutic Target in Duchenne Muscular Dystrophy
… (read more)April 23, 2021
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
(read more)February 28, 2021
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.
(read more)February 10, 2021
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.
(read more)February 3, 2021
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.
(read more)December 2, 2020
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)
September 21, 2020
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)
… (read more)June 10, 2020
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)
January 30, 2020
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… (read more)
