RESEARCH TRIANGLE PARK — There’s hope on the horizon for people struggling with the rare neurogenetic disorder Angelman syndrome, thanks to a newly formed partnership between a Triangle gene therapy company and the University of North Carolina at Chapel Hill.

Asklepios BioPharmaceutical (AskBio), a clinical stage gene therapy company based in the Research Triangle Park, has joined forces with the university, “leveraging groundbreaking research” with the company’s proprietary adeno-associated virus (AAV) technology to develop a viable therapy for the rare neurogenetic disorder.

Neither partner disclosed financial terms of the agreement.


“Individuals with Angelman syndrome face lifelong challenges, and our gene therapy approaches hold the potential to correct this disorder at its genetic roots,” said Mark Zylka, PhD, director of the UNC Neuroscience Center.

“We are incredibly excited to partner with AskBio, as they have been vanguards of clinical gene therapies for rare diseases.”

500,000 individuals worldwide

Angelman syndrome is 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, according to Angelman Syndrome Foundation.

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 Zylka and Ben Philpot, Ph.D., has generated preclinical evidence that gene therapy may help individuals with Angelman syndrome by improving seizure and motor outcomes.

Casey Childers, AskBio’s chief medical officer, said he first got the chance to meet the scientists in late 2018 after he became aware of their work through mutual connections at UNC.

“From that discussion, it was clear there might be an opportunity to support their mission for Angelman syndrome patients and families by leveraging our gene therapy experience,” he told the North Carolina Biotechnology Center.

AskBio is a privately held, clinical-stage gene therapy company dedicated to improving the lives of children and adults with genetic disorders. Bootstrapped with funding from NCBiotech, it’s now recognized as a world leader in gene therapy technologies.

AskBio’s gene therapy platform includes an extensive adeno-associated virus (AAV) capsid and promoter library, and an industry-leading proprietary cell line manufacturing process called Pro10.

The company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing, and holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids.

It’s also well funded after receiving a $225 million boost from TPG Capital and Vida Ventures in exchange for a minority stake in the company last April.

Transforming lives

Childers said AskBio is excited to work with the UNC team to progress this project.

“Right now, we’re in the early stages to determine the most effective pathway to translate preclinical work into a clinical product.”

Amanda Moore, Angelman Syndrome Foundation CEO, also welcomed the partnership between AskBio and UNC.

“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,” she said.

“The collaboration between UNC and AskBio brings us a step closer to delivering a viable gene therapy to the people and families we serve.”

(c) North Carolina Biotechnology Center