Parent Project Muscular Dystrophy is excited to announce a $1 million programmatic investment in Satellos Bioscience Inc. (Satellos) to support the development of a new regenerative medicine for the therapeutic treatment of Duchenne.

Duchenne is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in 5,000 live male births. Duchenne is caused by a change in the dystrophin gene. In people living with Duchenne, it was discovered by Dr. Michael Rudnicki, the scientific founder of Satellos, that muscle stem cells are severely compromised in their ability to create muscle progenitor cells which repair injured muscle. As a result, people with Duchenne are unable to keep up with the continuous damage to their muscles throughout life.

Satellos’s technology is based on this ground-breaking discovery into what controls muscle stem cell differentiation, and the significant impact its dysregulation has on the progressive nature of Duchenne. The company is developing a means to correct this problem through a novel therapeutic approach involving the administration of a small molecule drug, which mobilizes the body’s own muscle stem cells, also known as satellite cells, to repair and regenerate muscles.

Such a small molecule drug could be used to treat all people living with Duchenne and furthermore, could be used throughout life to boost the continuous repair and regeneration of skeletal muscle ravaged by this disease, thereby enhancing and extending the lives of people with Duchenne.

Eric Camino, PhD, PPMD’s Vice President of Research and Clinical Innovation, explains:

“For over 25 years, PPMD has been committed to exploring and supporting every single therapeutic possibility. With this programmatic investment in Satellos, we continue our cutting-edge approach to accelerate finding treatments that have the potential to end Duchenne for every single person impacted by the disease. There is compelling proof of concept data showing that the Satellos technology can improve muscle quality and restore function in the mdx mouse model of Duchenne. This investment from PPMD will enable the Satellos team to build on their proof of concept by amplifying their efforts to invent and refine a drug development candidate suitable for testing in humans.”

As Frank Gleeson, Satellos founder and CEO, describes:

“We are delighted to be working with PPMD and proud to have the therapeutic potential of our novel science recognized by such a leading entity in the fight against Duchenne. Our profound discoveries into how muscle stem cells repair and regenerate muscle offers a new avenue for addressing a root cause of the progressive debilitation characteristic of Duchenne. Support from PPMD will enable us to accelerate the development of our new treatment approach which offers the promise of helping Duchenne patients of all ages and stages of disease.”

PPMD makes programmatic investments in early-stage pharmaceutical companies to spur development of novel therapies to treat Duchenne and Becker. Early stage investment allows researchers to complete critical studies to advance investigational products to the clinic. Success of these early stage companies broadens PPMD’s reach for supporting crucial work to understand and treat Duchenne, as returns from initial investments may be received and are then reinvested to support additional Duchenne projects and initiatives. Learn more about PPMD’s robust Research Strategy, funding initiatives and strategies for accelerating drug development.