MyoPAXon by Myogenica for Muscle Regeneration
Duchenne muscular dystrophy (DMD), resulting from dystrophin gene mutations, is the most common form of muscular dystrophy. It leads to progressive muscle wasting, necessitating wheelchair dependence by age 12, and often results in early death around age 20. While various therapeutic approaches aim to increase dystrophin production, including MyoPAXon—an induced pluripotent stem cell (iPSC)-derived myogenic platform developed by Myogenica—many face challenges such as immune response issues and high costs. Myogenica has advanced the intramuscular delivery of MyoPAXon towards clinical trials, with an Investigational New Drug (IND) application expected in Q2 this year. Their ongoing STTR Fast Track project, in collaboration with the University of Minnesota, focuses on validating the efficacy and safety of systemically delivered MyoPAXon in preclinical animal models.