The mission of Genethon is to develop gene therapies for rare genetic diseases.

To successfully translate research into gene therapy product candidates to be made available to patients, Genethon has established an integrated R&D organisation, promoting excellence and high level international collaborations.

This organisation includes:

  • upstream translational research programs focusing on design and preclinical characterization of high added-value treatments,
  • vectorology and technology innovation programs aiming at innovation for production technologies,
  • a development platform with capacity for state-of the art technology development and preclinical evaluation,
  • a clinical research program involving highly specialized internal clinical and regulatory experts working with leading clinical centers worldwide.