The mission of Genethon is to promote the development of innovative therapies based on gene therapy, for rare diseases.

In order to achieve successful translational development, Genethon has the full range of skills and tools necessary to convert proofs of concept into treatments validated in patients. Genethon resources are used for projects arising from internal research or projects performed in the framework of international collaborations.

The teams at Genethon build upon discoveries from academic teams to develop them into gene therapy treatments for patients.