MyoPAXon

MyoPAXon is an investigational, allogeneic cell therapy product consisting of induced pluripotent stem cell (iPSC)-derived CD54+ muscle progenitor cells. Developed to regenerate skeletal muscle, it is designed for the treatment of Duchenne muscular dystrophy (DMD) and potentially other muscular dystrophies and degenerative muscle conditions. The therapy involves generating healthy muscle stem cells from human umbilical cord cells or pluripotent stem cells, which are then transplanted into patients to replace diseased or damaged muscle fibers and establish a new pool of functional muscle stem cells. Unlike gene-specific therapies, MyoPAXon is not limited by the underlying genetic mutation causing DMD, making it broadly applicable across different forms of muscular dystrophy. The product aims to restore functional dystrophin production in newly formed myofibers after transplantation[1][3][5][6].

Brand names

MyoPAXon