Pioneer in the development of innovative therapies for rare diseases, in 2013 the AFM-Telethon should also become the first patient association to produce gene therapy medicines through Genethon Bioprod. This establishment created by Genethon, the Telethon laboratory, is going to allow the accelerated production of treatments, in sufficient quantity and quality, for clinical trials in humans. The launch of Genethon Bioprod will be effective upon certification by The French National Agency for Medicines and Health Products Safety (Agence nationale de sécurité du médicament et des produits de santé - ANSM).
Three new trials
In 2013, three new trials should start. In January, Genethon launched a gene therapy trial for X-linked chronic granulomatous disease, a rare genetic disease. Result of an international cooperation and supported by Europe, this trial conducted in 20 patients will take place over two years and in four countries (Germany, France, UK, Switzerland).
After having supported the first French gene therapy trial in humans for an eye disease, the AFM-Telethon will initiate a new trial with Genethon and the Vision Institute. It will focus on Leber's hereditary optic neuropathy, a rare genetic disease that primarily affects individuals between 15 and 30 years of age. Finally, patients affected by Hurler syndrome, a degenerative brain disease, will benefit from a hope of treatment with the launch of a gene therapy trial in collaboration with the Pasteur Institute.
Help on a daily basis
In addition, more than 200 research projects will be supported in the context of the two yearly calls for proposals. Open to external researchers, they provide the opportunity to identify therapies and the most promising procedures in France and abroad.
The development of respite care centres for patients and caregivers will accelerate. After Salamandre, the AFM-Telethon will launch in April the construction of a second Respite Care Family Village in the Jura.