The Genethon – an inspiring story

I just got back last night from the World Orphan Drug Summit in Frankfurt, Germany, and thought I should tell you about an amazing presentation I saw there.

It was by Dr Frederic Revah, the CEO of Genethon. With funding from the Association Française des Myopathies (French Muscular Dystrophy Association), Genethon has a 2010 budget of approximately 26M€. It has a staff of 220 including 180 scientists and technicians. Genethon has also built multiple strategic collaborations with leading academic labs, biotech companies and clinical institutions around the world.

Dr Revah presented Genethon’s latest research into gene therapy, including successful tests of new treatments. I was blown away. To think that the Genethon is funded by the French public and rare disease patient movement, through the annual Telethon fundraising event, is just so inspiring. It shows much much patient groups can accomplish if they just put their mind and effort to it.

Located in Evry near Paris, Genethon is a non-profit organization that brings together a unique spectrum of expertise, from therapeutic research to clinical development, including preclinical development in rodents and large animal models, vector innovation, bioprocess, regulatory toxicology studies, and cGMP manufacturing. Even more impressive is that it will open the largest cGMP manufacturing site for gene therapy products later in 2011, with an ambitious programme for expanding clinical development of gene therapy treatments.

I came away convinced that we need to do the same thing in the UK. We need to build a rare disease patient movement here, inspired by the French model, that will radically change the lives of the millions of patients who are still without treatments.