Industry-Sponsored Research Week

UConn to collaborate with Ovid Therapeutics on gene therapy for Angelman Syndrome


By David Schwartz
Published: July 28th, 2020

The University of Connecticut School of Medicine has entered a research collaboration and license agreement with Ovid Therapeutics Inc., a developer of medicines for rare neurological diseases, to accelerate development of a next-generation short hairpin RNA (shRNA)-based therapeutic for Angelman syndrome.

Characteristic features of this condition include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. It is most commonly caused by 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. Though they have normal lifespans, individuals with Angelman syndrome are unable to live independently. The syndrome affects approximately 1 in 12,000 to 1 in 20,000 people globally, and there are no currently approved therapies beyond

behavioral interventions and pharmacologic management of symptoms.

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 agent designed to restore tonic inhibition and address the underlying symptoms of Angelman syndrome. OV101 is currently being evaluated in a Phase 3 trial, with topline results expected in the fourth quarter of 2020.

Under the terms of the research collaboration, Ovid will work closely with UConn’s Stormy J. Chamberlain, PhD, and gets exclusive access to identified genetic sequences for a potential shRNA-based therapeutic. Chamberlain is a recognized leader in the field of Angelman syndrome and UBE3A research. Along with his UConn research he chairs the Angelman Syndrome Foundation (ASF) Scientific Advisory Committee. Ovid will also work closely with UConn’s Noelle Germain, PhD, assistant professor of genetics and genome sciences.

 “We believe OV101 has the potential to serve as a core therapy for this disorder and are now focused on building a comprehensive and strategic Angelman syndrome longer term pipeline,” said Amit Rakhit, MD, MBA, Ovid’s president and chief medical officer. “Together with our early-stage microRNA approach, this research collaboration now provides us with additional targets against this disorder, greater strategic optionality, and underpins our broad capability to bring new therapies to individuals living with Angelman syndrome both near-term and into the future.”

“Our lab shares in Ovid’s demonstrated commitment to advance innovative therapeutic options for Angelman syndrome,” added Chamberlain. “An shRNA therapeutic can target the genetic cause of Angelman syndrome at its source and may offer potential advantages to other next-generation approaches, including antisense oligonucleotide therapy, via a lower rate of degradation and turnover and plasmid delivery allowing for a less-frequent dosing profile.”

Source: Ovid Therapeutics

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