Platform
Incorporating three decades of research in muscle stem cell biology, the MyoReGenX™ platform powers the discovery of small molecules with the potential to treat degenerative muscle diseases.
We are using our MyoReGenX™ platform to generate small molecule drug candidates for diseases and disorders where muscle tissue is lost or where regeneration is impaired, and enhanced repair is needed. The MyoReGenX™ platform recreates the muscle stem cell niche — the microenvironment where muscle progenitor cells divide to grow, maintain, and repair muscle tissue. By exploring both intrinsic signals and cell-to-niche interactions, we can identify defects in muscle regeneration and discover small molecules to help restore it.
This platform was used to reveal the regeneration defect in Duchenne muscular dystrophy and guided the discovery of our lead drug candidate. We’re now applying the same platform to additional degenerative muscle diseases, including facioscapulohumeral muscular dystrophy.
