Chapel Hill Review

A research union between the University of North Carolina and Deerfield Management Company might lead to relief for people with a neurogenetic syndrome that has no cure or effective treatment. 

The research partnership, known as Pinnacle Hill, will focus on finding treatment for Angelman Syndrome, a genetic disorder that can cause severe seizures and developmental delays, the University of North Carolina at Chapel Hill reported on June 24. It can also result in severe speech impairment and abnormal brain activity. 

Though there is no effective treatment for the more than 15,000 people in the United States living with Angelman, the UNC-Chapel Hill report said that some patients can find symptom relief from anti-seizure medications and physical, communication and behavior therapy. Worldwide, there are more than 500,000 people with the syndrome. 

Ben Philpot, the associate director of the UNC Neuroscience Center, will lead the research project with a team of interdisciplinary scientists at his side, UNC-Chapel Hill reported. The team is exploring new compounds for treatment, focusing on manipulating gene expression to help treat the disorder. 

“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,” Jon Collins, the partnership’s chief scientific officer, said in the UNC-Chapel Hill report. “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.”