AFM-Téléthon is a non-profit of patients and their relatives, committed to fighting diseases. Thanks to donations from the Téléthon (€85.8 million in 2018), it has become a major player in biomedical research into rare diseases in France and across the world. Today, it supports clinical trials testing treatments for genetic diseases of the eyes, blood, brain, immune system and muscles. It is unlike other non-profits in that its laboratories have the ability to design, produce and test their own innovative therapies.
Created by the AFM-Telethon and located in Evry (France), Genethon is a non-profit research and development centre dedicated to the development of biotherapies for rare diseases, from research to clinical validation. Genethon specialises in the discovery and development of gene therapy drugs and has several ongoing clinical, preclinical and research programmes for genetic diseases of muscle, blood, immune system and liver. A first product to which Genethon has contributed has obtained marketing authorisation in the United States in 2019 and is currently undergoing approval in Europe for spinal muscular atrophy. 7 other products resulting from Genethon’s R&D, alone or in collaboration, are currently in clinical trials and several others are in the preparation phase for clinical trials in 2020 and 2021. With 180 experts in the gene therapy, Genethon is one of the leader in this field.
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Created in 2005 through a collaboration between Inserm – National Institute of Health and Medical Research – and AFM-Telethon, The Institute for Stem cell Therapy and Exploration of Monogenic diseases (I-Stem) is a research and development centre dedicated to the development of treatments based on the potentials offered by pluripotent stem cells and applicable to rare diseases of genetic origin. Its field of activity extends from basic research to the pathological mechanisms and transfer of new therapies to clinical research. Currently, the major pathological indications studied concern diseases of the muscle, motor neurons, skin, retina and those associated with abnormalities in the development of the central nervous system. The technology platform team is exploring and implementing the large tools of cell production, high throughput screening, automated cell imaging, next generation sequencing and technological innovations in cell biology.
Yposkesi is one of the leading industrial development and contract manufacturing (CDMO) platforms for gene therapy vectors in Europe, and has 180 employees.
Yposkesi was created in November 2016 in Corbeil-Essonnes (France) by AFM Telethon as a spin-off of one of the leaders in gene therapy, Genethon with the support of BPI France. Yposkesi provides integrated services, covering the development of bioprocesses, the scaling up of production processes, analytical development as well as the manufacturing to GMP standards of clinical batches of lentiviral and AAV vectors. Its current facility consists of a 5,000 m2 building which operates several production suites for the active substance as well as aseptic filling. By 2022, it will increase its footprint with the construction of a second large-scale facility designed for commercial production and in accordance with European and American regulations. Capitalising on the expertise of more than 25 years of Généthon, Yposkesi invests significantly in the innovation of bioprocesses.