Our Science
Satellos was founded on a simple but powerful idea: the greatest challenge in degenerative muscle diseases isn't just muscle damage. It’s that the body can’t repair it.
to Degenerative Muscle Diseases
Our scientific founder, Dr. Michael Rudnicki, and his team made pioneering discoveries that changed how we understand muscle regeneration.
Their research, published in Nature Medicine, showed that dystrophin does more than protect muscle fibers — it also helps guide muscle stem cells through asymmetric division, a process essential for muscle repair. This breakthrough not only reshaped our understanding of Duchenne muscular dystrophy but also laid the scientific foundation for Satellos’s regenerative approach across multiple muscle diseases.
Following injury, muscle stems cells produce new muscle cells while preserving a pool of stem cells for future regeneration. This process depends on a complex network of signals that coordinate muscle repair.
In degenerative muscle diseases different disease processes can reduce the muscle’s ability to repair and maintain healthy tissue, contributing to progressive muscle loss.
Our approach is to enhance regeneration. We’re developing oral small molecule therapies designed to modulate key signaling pathways that influence muscle stem cell behavior, with the goal of supporting the body’s natural regenerative response. Our lead small molecule drug candidate is forazapadin which we discovered and patented to inhibit AAK1.
Pathway for Muscle Regeneration
Further research by Dr. Rudnicki and team showed Duchenne patients have abundant muscle stem cells, but lack muscle progenitor cells, which leads to a defect in muscle tissue regeneration. This research also led to the identification of AAK1, a member of the Notch pathway, that when inhibited is capable of re-establishing stem cell polarity to restore asymmetric muscle stem cell division and thus regenerate muscle, despite the absence of dystrophin. Satellos has identified a series of small molecule AAK1 inhibitors and is advancing a lead drug candidate to clinical trials for Duchenne muscular dystrophy.
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