13 Oct 2015

A new step forward to gene-medicine for AFM-Telethon : to produce and cure

Illustration for article: A new step forward to gene-medicine for AFM-Telethon : to produce and cure
AFM-Telethon has decided to take up a new challenge: to produce gene-medicines from innovative biotherapies developed in the laboratories part of its Biotherapies Institute for Rare Diseases at an industrial scale, and to give patients suffering from rare genetic diseases access to them at a fair and contained price. With this in view, the association will co-fund with Bpifrance the creation of the largest European center for the development and manufacturing of gene and cell therapies. This ambitious project opens up a new key stage on the way to the gene-medicine for the organization.

After the first successes of gene therapies in 2000 in  the "boy in the bubble" syndrome, proofs of their efficacy have increased in rare genetic disorders affecting the eye, blood, brain, immune system, muscles... Clinical trials have multiplied worldwide and production of these innovative treatments at an industrial scale is now crucial to be addressed. This project fits into the struggle and actions undertaken by AFM-Telethon for the last 30 years. It comes from a strategic vision about breakthrough medical innovation and from the pioneering and unique expertise developed by the Biotherapies Institute for Rare Diseases. The association is thus positioned as the leading player that builds up a new cutting edge economic sector creating value and many jobs in France, as well as the inventor of a new “non-financial profit” pharmaceutical business model. The launch will make it possible to create value to give back to the biomedical research activity, so that it proceeds further and quicker to the exclusive benefit of the patients.
Laurence Tiennot-Herment, chairman of AFM-Téléthon underlines « Since we created the association, our goal has never changed: to cure. Federating patients and their parents, we are committed to defeating rare diseases long considered incurable. Thanks to the public involvement in the annual French Telethon, we have funded many studies and created translational research institutes able to develop innovative therapies and conduct clinical trials up to proof-of-efficacy. Thanks to this unrivaled firepower, clinical trials have multiplied and the first investigational drugs have arisen. We must continue our struggle until the end and guarantee to the greatest number of patients an access to these innovative medicines, at a fair and contained price. Therefore, it’s with real pride, and by leveraging our unique expertise, that we are committed to proceed further with this next step to gene-medicine, along with BpiFrance.”
These facilities aim at making available to patients the gene-medicine developed by the AFM-Telethon labs but also by some academic ones or biotech firms. This should be at a fair and contained price based on the patient needs as it the historic goal of the association. This production hub is co-funded by AFM-Telethon  and the “Société de Projets Industriels” (SPI) fund managed by the public investment bank Bpifrance as part of the Investments Program for the Future.
The new company will have as its majority shareholder AFM-Telethon, holding 54% of its shares at inception. AFM-Telethon will contribute know-how and intellectual property crucial to develop this activity and additionally will invest 36 million euros in cash. Bpifrance will invest 84 million euros as part of the Investment Program for the Future.
Magali Joessel, Head of the SPI fund at Bpifrance says that “Right from the start, the SPI fund has worked on this project, which is most important to us, along with AFM-Telethon teams in order to have it emerge. Indeed, it’s both about helping AFM-Telethon to achieve its mission and enabling the developement of a leading player that builds up a new economic sector and creates many jobs. Cell and gene therapies are very promising technologies, and a final response to several diseases incurable up to now.”