Satellos scientists have discovered the major underlying cause of the progressive nature of Duchenne muscular dystrophy (DMD): impaired muscle stem cell driven repair. Muscle stem cells, or satellite cells, reside in the muscle tissue and are responsible for producing new muscle tissue in response to injury or exercise. However, in the case of DMD, this repair process is flawed, whereby the muscle stem cell fails to properly divide and produce tissue regenerating cells called progenitors. In the absence of these progenitor cells, new muscle tissue fails to form at sites of degeneration caused by DMD, and instead is replaced by fibrosis.
Satellos is developing a small molecule drug that restores the capacity of muscle stem cells to properly divide and produce progenitor cells. Our initial preclinical proof of concept studies have demonstrated that restoration of the balance between stem cells and the functional progenitor cells they produce results in enhanced muscle architecture and functional benefit.