Initially a pioneer in the field of genetics and then gene therapy, Genethon is involved in all stages of the development of gene therapies for rare diseases (neuromuscular diseases, immune deficiencies, blood, liver and vision disorders): design of therapeutic approaches and preclinical evaluation; development of innovative technologies for vectorization, bioproduction, and control of gene therapy products; evaluation and control of immune response; clinical trials.
The first gene therapy drug resulting from Genethon’s research is now available for spinal muscular atrophy. Thousands of young children around the world have benefited from it.
Around 20 drug candidates are currently in development or undergoing clinical trials in France and around the world.
To accelerate the research and development of its products, Genethon partners with academic institutions, industrial companies, biotech firms, and pharmaceutical companies in Europe and the United States.
A mission: to cure rare diseases
Genethon has a clear and ambitious mission: to treat rare genetic diseases. Every scientific breakthrough, every clinical trial, every therapeutic victory is a concrete response to the expectations of families. It is also a promise kept, made possible by the mobilization of citizens through the Telethon.
