I-Stem

I-Stem is a premier French research and development center focused on the therapeutic potential of human pluripotent stem cells for rare genetic diseases. Established in 2005 through a collaboration between AFM-Téléthon, Inserm, and the University of Évry, the institute specializes in monogenic disorders across neurology, dermatology, and ophthalmology. I-Stem employs two primary therapeutic strategies: cell replacement therapy using hESC-derived tissues and high-throughput drug discovery using iPSC-based disease models. The organization has successfully transitioned several programs into clinical trials, including treatments for Huntington's disease and retinitis pigmentosa. By bridging the gap between fundamental stem cell biology and clinical application, I-Stem remains at the forefront of regenerative medicine in Europe.