Created in 2005 through a collaboration between Inserm – Institut National Health and Medical Research – and AFM-Telethon – French Association against Myopathies – I-Stem is the largest French laboratory research and development dedicated to human pluripotent stem cells, embryonic origin or obtained by reprogramming gene. I-Stem is part of the Biotherapy Institute for Rare Diseases, which includes so far the four centers of research and development funded directly by the AFM Telethon. The specific vocation of I-Stem is to explore all the therapeutic potential of human pluripotent stem cells for applications in patients affected by rare diseases of genetic origin. In this context, our teams are developing two major areas of research. The first one is cell therapy, which aims to replace lost or diseased cells to other cells with the same characteristics, produced in the laboratory from pluripotent stem cells. The second area is pharmacology based on automated screening of large libraries of compounds with therapeutic potential, following modeling of molecular mechanisms associated with diseases, as revealed by the study of pluripotent stem cells from affected donors. I-Stem teams are currently working on a dozen genetic diseases that affect different organs. The Institute also hosts every year many researchers interested in other diseases and provides training and technology support.