The Biotherapies Institute for rare diseases

Founded in 2011, the Biotherapies Institute for rare diseases includes the Institute of Myology, Généthon and I-Stem.

The Biotherapies Institute unites the knowledge and expertise of this three laboratories, initiated and supported by the AFM-Telethon, world leaders of biotherapies for rare diseases. Objective: to accelerate the development of treatments against rare diseases.

Over 530 researchers, engineers and experts in project management, clinical development, regulatory affairs, technology transfer and 25 000 m2 of laboratory space in Paris and Evry (Essone): the Biotherapies Institute is a unique and exceptional force for translational research, from fundamental to making treatments available for patients.


Clinical Immunodeficiency Network

One of the first international clinical trials of gene therapy as a treatment for a rare disease, Wiskott-Aldrich Syndrome, has allowed Genethon – already renowned as a unique center for innovation – to gather expertise and skills from around the world. Teams from Genethon, Hôpital Necker Enfants Malades in Paris, Great Ormond Street Hospital in London and the Children’s Hospital in Boston are working closely together to assess the efficacy in patients of a product that was initially developed by Genethon.