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Ovid Therapeutics and University of Connecticut Enter into Strategic Research Collaboration to Accelerate the Development of Next-Generation Genetic Therapy for Angelman Syndrome
Ovid to collaborate with renowned molecular geneticist and Angelman syndrome expert Stormy J. Chamberlain, Ph.D., to advance a short hairpin RNA (shRNA)-based
About this update from Ovid Therapeutics Inc.
[{"type":"text","content":"Ovid to collaborate with renowned molecular geneticist and Angelman syndrome expert Stormy J. Chamberlain, Ph.D., to advance a short hairpin RNA (shRNA)-based therapeutic with the goal of addressing the underlying genetic cause of Angelman syndrome Ovid and UConn to collaborate on genetic therapy for potential future use alone or in combination with OV101, Ovid’s small molecule therapy for Angelman syndrome in Phase 3 development NEW YORK and FARMINGTON, Conn., July 23, 2020 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, and the University of Connecticut School of Medicine (“UConn”), today announced a research collaboration and license agreement to accelerate the development of a next-generation short hairpin RNA (shRNA)-based therapeutic for Angelman syndrome and potentially other indications. The most common cause of Angelman syndrome is the loss of function of the gene that codes for ubiquitin protein ligase E3A (UBE3A), which plays a critical role in nerve cell communication, resulting in impaired tonic inhibition. An shRNA-based therapeutic may address this underlying genetic cause of Angelman syndrome by reducing the expression of UBE3A-antisense, potentially restoring the function of UBE3A. This genetic approach may be used in combination with OV101 (gaboxadol), Ovid’s novel, small-molecule delta (δ)-selective GABAA receptor agonist, to restore tonic inhibition and address the underlying symptomology of individuals with Angelman syndrome. OV101 is currently being evaluated in the pivotal Phase 3 NEPTUNE trial in Angelman syndrome, with topline results expected in the fourth quarter of 2020.\n Under the terms of the research collaboration, Ovid will work closely with UConn’s Stormy J. Chamberlain, Ph.D., and gain exclusive 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. Dr. Chamberlain is a recognized leader in the field of Angelman syndrome and UBE3A research and currently serves as the John and Donna Krenicki Associate Professor of Genomics and Per...