On a recent episode of The Genetics Podcast, Patrick spoke with Frank Gleeson, co-founder and CEO of Satellos Bioscience, a Canadian biotech pioneering a regenerative approach to Duchenne muscular dystrophy (DMD). They discussed Satellos’ focus on muscle stem cell regeneration, how this fills a critical therapeutic gap, and the promising results of their early clinical trials.
Rather than targeting the dystrophin gene directly like most therapies, Satellos is pursuing a novel path: restoring the body’s innate ability to regenerate muscle by correcting a polarity signaling defect in muscle stem cells.
A long-time partnership in stem cell innovation
Before launching Satellos, Frank and his scientific co-founder Dr. Michael Rudnick had already worked together for over two decades in the stem cell field. Their collaboration began with the Stem Cell Network of Canada, where Michael served as President and Frank as Chair of the Board. Both were fascinated by the potential of chemical biology to modulate stem cell function inside the body.
The inspiration behind Satellos came from Michael’s 2015 Nature paper, which revealed a surprising new function of the dystrophin protein, not just as structural support in muscle fibers, but as a critical signal for stem cell polarity and asymmetric division. In DMD, the absence of dystrophin prevents muscle stem cells from properly regenerating damaged tissue. This insight shifted Frank and Michael’s focus: instead of patching up the muscle scaffold, what if they could reboot the muscle’s repair system?
Rethinking DMD: regeneration as a therapeutic strategy
Frank explained that current DMD therapies largely fall into two categories: gene replacement (delivered via vectors) and exon skipping to enable partial dystrophin production. While these approaches aim to stabilize muscle fibers, they do not restore the ability to regenerate new muscle, a process that naturally declines in patients as damage outpaces repair.
Satellos’ approach targets a different failure mode in DMD: the inability of muscle stem cells to divide correctly in the absence of dystrophin. Normally, these cells undergo asymmetric division to produce both a replacement stem cell and a progenitor cell that matures into muscle tissue. Without the right polarity cues, this process breaks down.
By identifying and drugging a compensatory signaling pathway involving the protein AAK1, Satellos aims to trick muscle stem cells into polarizing and dividing properly again. This could potentially restore regenerative capacity regardless of the underlying mutation in the dystrophin gene, making it mutation-agnostic and broadly applicable.
Promising early results
Satellos recently completed a Phase 1b trial in adult DMD patients, and while it was small (five participants), the results were encouraging. Most functional metrics like pinch strength and forced vital capacity remained stable, but grip strength doubled on average across patients over just 28 days.
This suggests that even in late-stage disease, the regenerative machinery can be activated. Frank emphasized that these adults have very limited muscle mass remaining, so any measurable improvement is remarkable. In younger patients with more muscle to regenerate, the potential impact could be far greater.
The company plans to launch a Phase 2 trial later this year in ambulatory boys under 12, with three dose arms including placebo, and treatment over a three-month period. If successful, this could provide proof that targeting regeneration leads to functional improvements, especially early in the disease course.
Building a scalable, complementary solution
Importantly, Satellos’ therapy is designed to work alongside other DMD treatments. Because it doesn’t rely on delivering a specific gene or targeting a specific mutation, it could complement other therapies. Frank sees this as part of a broader shift in biotech, where durability, specificity, and patient quality of life are the priority. Satellos’ compound has shown a clean safety profile, minimal off-target effects, and pharmacokinetics well-suited to precise modulation.
Lessons in science, business, and partnership
A veteran of both biotech and venture capital, Frank shared valuable insights into building a company in today’s tough funding climate. He emphasized the importance of persistence and clarity of vision.
He also highlighted the importance of mutual respect between business and scientific co-founders. “I don’t want to be in the lab, and my scientific partners don’t want to do the business. That mutual understanding is what makes it work.”
As Satellos moves toward larger clinical trials, the hope is that this unique regenerative approach could finally shift the trajectory for individuals with DMD by giving muscles the power to heal themselves.
Listen to the full episode below.
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