Our Science

Satellos was founded on decades of research in muscle biology that has uncovered new pathways to rethink how Duchenne muscular dystrophy is treated.

Our Science
Breakthrough Research in Duchenne Muscular Dystrophy

Our scientific founder, Dr. Michael Rudnicki, and his research team were the first to discover that dystrophin played a role outside of the muscle fiber and within the muscle stem cell. For decades it has been known that muscle regeneration is impaired in Duchenne muscular dystrophy, but the how and why were not understood until Dr. Rudnicki’s landmark publication in Nature Medicine, describing the role of dystrophin in regulating muscle stem cell polarity and muscle tissue regeneration.

Our Approach
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Independent Pathway
A Dystrophin-Independent
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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