Ovid Therapeutics Collaborates with UCONN Health and Stormy Chamberlain, PhD to Develop Gene Therapy for AS
On July 23, 2020, Ovid Therapeutics announced a research collaboration with UCONN Health and Stormy Chamberlain, PhD. This exciting work began as an innovative idea funded by the Angelman Syndrome Foundation. Short hairpin RNA (shRNAs) work like ASOs, but can be delivered using AAV, like gene therapy. One dose would last indefinitely.
Ovid will have access to identified genetic sequences for a potential shRNA-based therapeutic. Ovid plans to validate select sequences and leverage its translational medicine capabilities and drug development expertise in Angelman syndrome to advance an shRNA-based therapeutic into clinical studies.
“We are thrilled to be working with Dr. Chamberlain and the team from UCONN to develop OV882 as a potential treatment for individuals living with Angelman syndrome. Not only does Dr. Chamberlain and her team share our heartfelt commitment to the Angelman community; they are breaking open the frontiers of neuroscience by developing a therapy that, for the first time, may overcome the underlying genetic cause of the condition.”
Jeremy Levin, CEO
“We are over-the-moon excited to work with Ovid to develop our shRNA targeting UBE3A-ATS, now called OV882. This program builds on what we know about ASOs to activate paternal UBE3A, however it is designed to be longer lasting. Instead of repeated injections, this approach will be a single injection that should last decades. It combines the best parts of ASO and gene therapy approaches. We’re particularly delighted to partner with Ovid Therapeutics, a company that cares deeply about the Angelman syndrome community and has invested so much into it already.”
Stormy Chamberlain, PhD UCONN
Thank you to donors to ASF who helped bring us yet another promising potential therapeutic!