Genethon: patient-empowered research

Abstract

Genethon is a non-profit pharmaceutical R&D organization with the mission of developing innovative gene therapy treatments for patients affected with rare diseases. It was created by the patient organization AFM-Telethon in 1990, which has since been the source of most of its funding, thanks to the donations received during the yearly French Telethon. Genethon was a pioneer in the field of modern genetics after establishing the first maps of the human genome in the 1990s; it is now a pharmaceutical organization whose activity spans from upstream research to clinical development, encompassing innovative bioprocess development and large-scale GMP-manufacturing. It leads a diversified pipeline of products both at clinical and preclinical stages in the field of neuromuscular diseases, primary immunodeficiencies, retinal and liver diseases. The development of Genethon illustrates a unique example of patient-empowered research where a private non-profit structure, funded thanks to public generosity, favors highly innovative developments centered on the needs of patients.

Keywords::

• gene therapy
• genetic diseases
• patient organization
• rare diseases

Acknowledgements

The author wishes to acknowledge the support of Susan Cure in preparing this manuscript.

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