Regenerating Muscle From Within™

At Satellos, our research began with studying Duchenne muscular dystrophy (“DMD” or “Duchenne”). Our research revealed that the dystrophin protein does more than protect muscle fibers from damage. Critically, it also plays a key role in regeneration by guiding muscle stem cells through a process called asymmetric division. When a stem cell enters mitosis in response to muscle damage, it divides asymmetrically to produce one stem cell to preserve the pool for future use and one muscle precursor cell, which then goes on to regenerate the muscle fiber itself. In Duchenne, the lack of dystrophin disrupts this process, leading to insufficient muscle repair and ongoing degeneration. Duchenne is the first clinical indication we are pursuing with our lead drug candidate, forazapadin.

That discovery pointed us in the direction of something bigger: how might this process be regulated in other situations where muscle mass is lost? While the underlying biology differs across degenerative muscle diseases, we found that many ultimately share a common challenge: the body’s ability to repair and regenerate damaged muscle declines over time because their muscle stem cells are not able to fully respond to the damage. Consequently, Satellos is expanding its research to study other diseases characterized by a loss of muscle mass, including facioscapulohumeral muscular dystrophy (FSHD), with the goal of developing therapies that help improve this natural repair process.