Our Science

Satellos was founded on decades of research in muscle biology that uncovered a critical insight: In Duchenne muscular dystrophy, muscle fails to regenerate as intended in response to damage. We discovered this occurs from a breakdown in how muscle stem cells divide.

Our Science
Breakthrough Research in
Duchenne Muscular Dystrophy

Our scientific founder, Dr. Michael Rudnicki, and his team were the first to show that the dystrophin protein plays a role outside the muscle fiber — inside the muscle stem cell.

His research, published in Nature Medicine, revealed that dystrophin helps guide stem cells to divide asymmetrically, a process essential to muscle regeneration. Without this signal, the body can’t produce enough of the progenitor cells needed to repair and rebuild muscle.

Our Approach
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Muscle stem cells rely on a process called “stem cell polarity” to guide how they divide. This process is essential for muscle repair. Dystrophin plays a signaling role in helping these cells orient correctly and generate the progenitor cells needed to build new muscle.
In Duchenne muscular dystrophy, the gene responsible for producing dystrophin is missing or impaired. As a result, the body doesn’t make the dystrophin protein at all — or makes too little for it to function properly.
When dystrophin is absent, muscle stem cells can’t polarize efficiently. This means they can’t divide properly and struggle to produce enough progenitor cells to keep repairing and regenerating muscle in response to ongoing damage over time.
These insights shed new light on why muscle degeneration progresses and is so devastating in Duchenne. They also revealed a new opportunity — to restore polarity, reset muscle stem cell division and reactivate the body’s natural repair process, an outcome that has the potential to change the course of the disease.
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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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MyoReGenX™ Platform
Generating Small Molecule Drug
Candidates for Degenerative
Muscle Diseases

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 conditions where muscle tissue has been lost or muscle regeneration is defective, and where enhanced muscle tissue repair is required. This could include additional genetic muscular dystrophies, as well as indications, such as sarcopenia (age-related muscle loss); cachexia, associated with cancer; or other disease conditions or muscle loss due to surgery or unforeseen trauma (e.g., combat, accidents, injuries).

Presentations & Publications