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.

Genethon: a unique firepower against rare diseases

Today Genethon has 220 researchers and experts. A unique firepower against rare diseases: 10 products from its research or to which it has contributed are in clinical trial throughout the world for rare diseases in muscle, liver, blood, the immune system and vision; 8 others should enter clinical trials in the coming 5 years. A first gene therapy medication using technologies derived from its research received marketing authorization in the United States, Europe and Japan for type 1 spinal muscular atrophy. A second medication to which Genethon contributed in the initial R&D phases is currently being evaluated by the health authorities for Leber Hereditary Optic Neuropathy and a third is reaching the end of clinical studies and marketing approval will be requested for myotubular myopathy. Providing hope for patients and their families!

“Today we are faced with new challenges as we seek to overcome the scientific and technological hurdles still linked to gene therapy, Frederic Revah, Genethon’s CEO, comments. We have to design more specific vectors, develop approaches for injecting these treatments and continue to innovate to treat rare diseases, as well as those said to be ultra-rare, and that should not be neglected. These are challenges for which Genethon is once again on the front lines.”

Find out more

Press release – International Rare Disease Day