To accelerate the development of innovative treatments for rare diseases, AFM-Telethon has created its own laboratories. Devoted to gene therapy of rare diseases (Genethon), to stem cells (I-Stem) and to myology (Institute of myology), they are now international leaders in their field. Gathered within the Biotherapies Institute for rare disease, the Association’s three laboratories play a major role in the medical revolution.
Genethon: the lynchpin of gene therapy
Created in 1990 and initially a pioneer in genetics then in gene therapy, Genethon is involved at all stages of the development of gene therapies for rare diseases (neuromuscular diseases, diseases of the immune system, the blood and the liver): design of therapeutic approaches and preclinical evaluation; development of innovative technologies for vectorization, bioproduction and control of gene therapy products; assessment and control of the immune response; clinical trials.
Boasting almost 220 experts and 19 gene therapy products in clinical trials or in development, Genethon, the AFM-Telethon laboratory, is now one of the world leaders in gene therapy for rare diseases, from research to clinical development.
Read more: the genetic revolution
Institute of myology: the muscle expert
In 1996 the AFM-Telethon created the Institute of myology, a unique center of expertise for muscles and their diseases in Europe. 250 experts (physicians, researchers and technicians) work there, covering all activities related to myology, the science and medicine of the muscle, for research, diagnosis and evaluation of muscle function, functional exploration, a top center for neuromuscular diseases certified as a leading European center, databases, teaching, tissue banks for research, I-Motion platforms for pediatric (Trousseau) and adult (Pitié-Salpétrière) clinical trials.
A Myology foundation project is underway. For the AFM-Telethon, this Foundation aims to sustain myology and become a national and international springboard in this discipline. Its goal is as follows:
- To study, repair and strengthen the muscles in all its forms (healthy, diseased, ageing).
- To bring together care, basic and applied research, clinical research, evaluation of treatments and teaching, all at the highest level, at the same location.
- To be of general interest, by discoveries on the muscle that benefit all.
Read more: the myology revolution
I-Stem: a pioneer in cellular therapy
In 2005, the AFM-Telethon, in partnership with Inserm, created I-Stem, a laboratory dedicated to stem cells and cellular therapy for monogenic diseases. The laboratory conducts research and development activities using stem cells in two areas, namely cell therapy and high-throughput pharmacological screening using cells modelling pathologies. Its work involves diseases of the muscle, the motor neurons, the skin, the retina and diseases associated with developmental anomalies of the central nervous system. Today it boasts more than 75 experts.
Comprising the three laboratories of the AFM-Telethon, the Institute of rare disease biotherapies boasts more than 500 researchers, technicians, physicians, engineers and other specialists spread throughout the 15,000 sq. m of lab space in Paris and Evry.