Parent Project Muscular Dystrophy (PPMD) announced a $1 million investment in Satellos Bioscience to support the development of a technology that aims to repair and regenerate muscle cells in people with Duchenne muscular dystrophy (DMD).
“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,” Eric Camino, PhD, PPMD’s vice president of research and clinical innovation, said in a press release.
Frank Gleeson, founder and CEO at Satellos, added: “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.”
Previous work by Michael Rudnicki, PhD, the scientific founder of Satellos, found that DMD is characterized by flaws in the process by which muscle stem cells — or satellite cells — generate progenitor cells that repair injured muscle.
In the absence of these progenitor cells, new muscle tissue fails to form at sites of degeneration caused by Duchenne, which results in accumulating muscle damage and fibrosis (scarring).
Satellos is working to advance a small molecule therapy that aims to restore the capacity of muscle stem cells to produce progenitor cells that address muscle regeneration. The therapy, ideally, could be used to provide continuous repair and regeneration of affected skeletal muscle, improving and extending the lives of people with DMD, PPMD reports.
“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,” Gleeson said. “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.”
According to Satellos, preclinical studies — including published work in a mouse model of Duchenne — showed that restoring the balance between stem cells and resulting progenitor cells led to improved muscle structure and functional benefit.
“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,” Camino said.
“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,” he added.
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