MyoPAXon focuses on efficiently generating immature, genetically correct, skeletal muscle cells from iPS (induced pluripotent stem) cells and then transplanting them into the muscles of individuals with Duchenne. Transplanted cells could help express missing proteins, such as dystrophin, and improve muscle function.

Status

This compound is in preclinical research, meaning it has not yet advanced to clinical trials.

Sponsor

This program is sponsored by Myogenica.