The Institute for Stem cell Therapy and Exploration of Monogenic diseases (I-Stem), established on 1st January 2005, is a research and development center dedicated to the development of treatments based on the potentials offered by pluripotent stem cells and applicable to rare diseases of genetic origin. I-Stem is composed of two separate administrative entities of roughly equal size, one associated with public institutions (Inserm / UEVE 861, whose scientific director is Cécile Martinat) and the other directly linked to the AFM-Téléthon (CECS, Center for the Study of Stem Cells, whose scientific director is Marc Peschanski). However, there is no separation between the two entities at the functional level and the Institute I-Stem is a set of teams in which staff work related to either one or the other administrative structures. The Institute has an executive management consists of a Scientific Director (Marc Peschanski), a CEO (Raymond Zakhia), and a board of directors that includes all team leaders and programs, scientific and administrative.
Defined by three keywords, “therapeutics”, “monogenic diseases” and “pluripotent stem cells”, the field of activity of I-Stem extends from basic research to the pathological mechanisms and transfer of new therapies to clinical research. The programs of each of the research teams are devoted either to a set of genetic diseases or to the development of new technologies around the major instruments implemented in the Institute. 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. Research teams permanently interact with each other around common projects and with the technological platforms that are all shared. I-Stem teams are involved in collaborative networks with many research teams both academic and industrial, in programs concerning various aspects of the therapeutic use of pluripotent stem cells. In particular, they participate in two major national infrastructures (IngeStem and Neuratris), European networks and research and development consortia funded by the National Agency for Research. I-Stem developed since its inception a significant activity in formation and training, from introductory tours for the general public to multi-months internships for colleagues interested in technology transfer (in the past, reprogramming of iPS lines for example, today the use of CRISPR/Cas for the generation of isogenic cellular models of pathologies), through the support of student internships (Masters 1 and 2), supervision of PhD theses and post-docs.