Généthon

First a pioneer of human genome research, Genethon now explores the new therapeutic hypotheses stemming from the extraordinary progresses of contemporary biology.

Today, Genethon is a charity-funded biotherapy organization, financed at 88.7% by the AFM, Under the management of Dr Frédéric Revah, CEO, Genethon is a unique structure where researchers, technicians, chemists and physicians elaborate technologies to develop innovative therapeutics, like gene therapy or cell therapy, to treat rare or orphan diseases. Scientists of Genethon focus on therapeutic tools for in vivo or ex vivo gene transfer, on rare diseases and specifically on neuromuscular diseases as well as on immune system or skin diseases. These pathologies are real models to help accelerate the research towards new therapeutic concepts which can subsequently be adapted to more well-known pathologies.

For gene therapy and cell culture, a large engineering team then develops vector manufacturing processes that are transferred to our GMP production service, accredited as Gene and Cell Therapy Establishment (Etablissement de thérapie génique et cellulaire -ETGC). The latter provides GMP quality controlled vectors, not only for Généthon’s own projects but for numerous collaborations or external requests. Today we can manufacture vectors for use in gene therapy clinical trials. In view of increasing the production capacity, Généthon has inaugurated the novel bioproduction center (Généthon BioProd) on the 30^th of November 2010, allowing the production of clinical grade viral vectors for phase I and II clinical studies at production scales going up to 200 L per reactor run.
Involved in WP: 7, 8, and TACT.