The Telethon has created its own laboratories grouped together within the Institute of Biotherapies for Rare Diseases. Its mission is to accelerate the availability of innovative treatments.
With nearly 30 hours of live on francetélévisions and a reinforced digital device, the Telethon 2015 will be one of the biggest audiovisual solidarity events in the world. Between Friday 4 and Saturday 5 December, the French Association against Myopathies (AFM), which funds research projects on genetic neuromuscular diseases (myopathies, Steinert myotonia, etc.), will again appeal to the generosity of French. In 2014, for example, it raised nearly 93 million euros in donations, a record! And since 1987, date of 1er Telethon, it also allowed these activists to acquire a new status.
Build your own laboratories
Contacted by Why actor, Laurence Tiennot-Herment, president of the AFM, explains that thanks to the money collected, these families of sick children were able to organize themselves from A to Z. “We have built our laboratories, recruited our own research teams , create the “rare diseases” platform at the Broussais Hospital (Paris), etc. In parallel with its actions, the association continued to meet with political decision-makers to obtain public funding ”. “A long-term job that ended up paying off,” admits Laurence Tiennot-Herment.
Laurence Tiennot-Herment : ” We went to the public authorities and told them that we had shown that our actions were working. They were asked to take over… ” (interview carried out on 11/25/15)
As a result of this success, the Généthon which, in 2013, obtained the status of a pharmaceutical establishment. Its production center, Généthon Bioprod, now has the largest production capacity for gene therapy drugs in the world. It makes it possible to produce molecules intended for testing in humans.
An isolated example? Not really, because in addition to supporting 230 research programs, the Telethon has several laboratories. They are brought together within the Institute of Biotherapies for Rare Diseases which brings together 4 research centers initiated and mainly funded by the Association: Généthon and Atlantic Gene Therapies therefore for gene therapy for rare diseases, I-Stem for cell therapy. of monogenic diseases, Institute of Myology for Muscle Diseases.
With an annual budget of 62.9 million euros (M €) in 2014, funded to the tune of 37.9 M € (60%) by AFM-Telethon, the Institute of Biotherapies is a unique strike force. in the world, admit all researchers.
Laurence Tiennot-Herment : ” When we are in the research or drug testing stages on mice, we do not need to do it in a pharmaceutical setting with good practices ” (interview carried out on 11/25/15)
Medicines: soon to be produced on an industrial scale
Despite this success, Généthon-Bioprod is now reaching saturation point of its production capacity. But the AFM-Telethon, which always knows how to mobilize, and accompanied by the investment fund “SPI – Industrial Project Companies”, it announced last month its objective to create the largest European center for the development and production of gene therapies. and cellular (13,000 m2 from 2019).
This project aims, on the one hand, to create an “industrial-scale” production platform and, on the other hand, to complete the development of gene and cell therapies. “It will make it possible to multiply the efforts of the AFM-Telethon to provide patients with curative therapeutic solutions, while being able to bring them to the point of commercialization”, concludes Laurence Tiennot-Herment.
.
