New Partnership Formed to Develop Angelman Syndrome Treatment

According to a story from Globe Newswire, the biopharmaceutical company Ovid Therapeutics and the University of Connecticut School of Medicine (UConn) have announced a new license agreement and research collaboration with the goal of developing a treatment for the rare disorder Angelman syndrome, along with potentially other conditions as well. The therapy will utilize a mechanism based on short hairpin RNA (shRNA). Ovid is focused on the development of treatments for rare neurological illnesses.

About Angelman Syndrome

Angelman syndrome is a type of genetic disorder that impacts the nervous system. This disorder is not inherited from parents, but instead occurs as a spontaneous genetic alteration, typically the deletion of a segment from chromosome 15 called UBE3A. A mutation of this gene can also cause Angelman syndrome. Symptoms of this disorder can include seizures, developmental delays, a small head, an excited and happy demeanor, trouble with balance and movement, intellectual disability, and speech issues. Patients may share certain facial characteristics. Symptoms become noticeable at about one year old. Treatment is supportive and symptomatic, and there is currently no cure. Patients have a strong desire for social interaction, and despite limited language are able to understand much of what is happening around them. Life expectancy is generally close to that of an unaffected person. To learn more about Angelman syndrome, click here.

The UBE3A Gene and shRNA

With the majority of cases being linked to the UBE3A gene, the shRNA mechanism may help rectify the loss of function of this gene by reducing UBE3A antisense expression, which would restore at least some of the functionality. The partners envision using the future drug based on this mechanism as potentially part of a combination treatment with Ovid’s product candidate OV101, which is in phase 3 testing as a therapy for the syndrome; topline results are expected before year’s end.

Ovid is aiming to seek collaboration from Stormy J. Chamberlain, Ph.D, one of UConn’s dedicated scientists that has been recognized as a leading researcher of the UBE3A mutation and Angelman syndrome. Another member of the team will be UConn’s Assistant Professor of Genetics and Genome Sciences, Noelle Germain, Ph.D. With combined resources of the university and Ovid, the partners are uniquely position to develop and shRNA-based therapy for the syndrome.


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