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New research could potentially lead to relief for tens of thousands of Americans suffering from an untreatable disease.

In announcing its second funded project, Pinnacle Hill, the research and development partnership between the University of North Carolina at Chapel Hill and Deerfield Management Company, will support the development of a potential treatment for Angelman Syndrome, a neuro-genetic disorder characterized by developmental delays, abnormal brain activity, and severe seizures. There is currently no effective treatment for the disease.

The project is led by Ben Philpot, Kenan Distinguished Professor and associate director of the UNC Neuroscience Center in the UNC School of Medicine, and is supported by an interdisciplinary team of scientists from both UNC and Pinnacle Hill. Their work focuses on manipulating gene expression, which may be the most direct treatment strategy for Angelman Syndrome.

The team will explore new compounds for treatment identified through their investigative efforts, which could address a critically unmet need for more than 15,000 individuals suffering from the disease in the U.S. and over 500,000 worldwide.

“The project with Dr. Philpot is another great example of innovative research at UNC which greatly benefits from the funding and drug development expertise and capabilities offered through Pinnacle Hill,” said Jon Collins, the partnership’s chief scientific officer. “I can’t envision a more qualified team of academic and drug development scientists to work hand-in-hand on delivering a new medicine for Angelman’s Syndrome patients.”

Launched in October 2018, Pinnacle Hill was established with a funding commitment of up to $65 million from Deerfield to accelerate the development and translation of these biomedical and life-science innovations to address significant unmet medical needs.

Pinnacle Hill focuses on drug research projects that are approved and overseen by a joint steering committee comprised of members from UNC-Chapel Hill and Deerfield leadership teams. Each selected project has the potential to receive funding to support advancement to investigational new drug studies.

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