PTC’s GEMS technology to be used to search for potential drugs for DMD patients.

PTC Therapeutics a received a $15.4-million, five-year grant to further research into Duchenne muscular dystrophy (DMD).

The funding from the National Institute of Neurological Disorders and Stroke and the National Institute of Arthritis and Musculoskeletal and Skin Diseases will support a research collaboration between PTC and the University of Pennsylvania School of Medicine. This partnership is based on previous research and discovery work supported by Parent Project Muscular Dystrophy (PPMD) under an initiative known as Project Catalyst.

“This grant supports promising preclinical research on a number of targets believed to be medically relevant for DMD,” says H. Lee Sweeney, Ph.D., professor and chairman of the department of physiology at the University of Pennsylvania School of Medicine, scientific advisor to PPMD, and principal investigator on this NIH grant. “Over the course of this grant, our goal is to have Project Catalyst compounds ready for advancement into clinical trials for patients with DMD.”

In 2003, PTC and PPMD initiated Project Catalyst to identify new treatments for patients with DMD. Project Catalyst leveraged PTC’s drug discovery technology called GEMS (gene expression modulation by small molecules). The GEMS technology allows PTC to identify small molecules that up- or down-regulate the production of proteins. Utilizing this tool, promising lead compounds were identified for several targets believed to be medically relevant for DMD, according to the partners.

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