AskBio, UNC-Chapel Hill Collaborate on Angelman Syndrome Gene Therapy Research

A UNC School of Medicine research team led by Mark Zylka, PhD, and Ben Philpot, PhD, has generated preclinical evidence that gene therapy may help individuals with Angelman syndrome by improving seizure and motor outcomes.

AskBio, UNC-Chapel Hill Collaborate on Angelman Syndrome Gene Therapy Research click to enlarge Mark Zylka, PhD, and Ben Philpot, PhD

March 18, 2020 

Asklepios BioPharmaceutical, Inc. (AskBio), a leading clinical-stage adeno-associated virus (AAV) gene therapy company, announced that it has entered into a research collaboration with the University of North Carolina at Chapel Hill for the development and commercialization of gene therapy for Angelman syndrome.

“This collaboration allows us to leverage groundbreaking research from UNC and apply our AAV development capabilities to find a gene therapy treatment for Angelman syndrome,” said Sheila Mikhail, JD, MBA, AskBio Chief Executive Officer and co-founder. “We look forward to advancing this program together.”

Angelman syndrome is a rare neurogenetic disorder caused by the loss of function of the UBE3A gene. The disorder occurs in approximately one in 15,000 people, or about 500,000 individuals worldwide, and there is currently no cure. In addition to life-altering symptoms such as speech and motor deficits, more than 80 percent of Angelman syndrome patients experience epilepsy, which typically does not respond well to standard anti-seizure medications.

A UNC School of Medicine team led by Mark Zylka, PhD, and Ben Philpot, PhD, has generated preclinical evidence that gene therapy may help individuals with Angelman syndrome by improving seizure and motor outcomes. 

“Individuals with Angelman syndrome face lifelong challenges, and our gene therapy approaches hold the potential to correct this disorder at its genetic roots. We are incredibly excited to partner with AskBio, as they have been vanguards of clinical gene therapies for rare diseases,” said Mark Zylka, PhD, Director of the UNC Neuroscience Center and W.R. Kenan Distinguished Professor of Cell Biology and Physiology.

Ben Philpot, PhD, Associate Director of the UNC Neuroscience Center and Kenan Distinguished Professor of Cell Biology and Physiology, added, “We look forward to advancing this transformative treatment to the clinic and potentially improving the lives of individuals with Angelman syndrome.”

“The partnership between AskBio and UNC could transform the lives of people living with Angelman syndrome by providing them with a potential therapy for this rare disease,” said Amanda Moore, Angelman Syndrome Foundation CEO. “The Angelman Syndrome Foundation has long been proud to support the work of UNC researchers, Drs. Ben Philpot and Mark Zylka, and invest in science that positively affects the Angelman syndrome community. The collaboration between UNC and AskBio brings us a step closer to delivering a viable gene therapy to the people and families we serve.”

Read more about UNC Neuroscience Center autism and Angelman research.

Media Contacts:

AskBio, 984.275.2705

Angelman Syndrome Foundation, 317.514.6918

UNC Health | UNC School of Medicine, 984.974.1915

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