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Neurology
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Ovid, UConn collaborating on genetic therapy for Angelman syndrome

Posted on August 1, 2020

Ovid Therapeutics Inc and the University of Connecticut School of Medicine (UConn), announced a research collaboration and license agreement to accelerate the development of a next-generation short hairpin RNA (shRNA)-based therapeutic for Angelman syndrome, according to a press release.

One of the most common causes of Angelman syndrome is the loss of function of the gene that codes for ubiquitin-protein ligase E3A (UBE3A). Researchers believe an shRNA-based therapeutic may reduce the expression of UBE3A-antisense, potentially restoring the function of UBE3A. This potential treatment may be used in combination with OV101 (gaboxadol), a novel, small-molecule delta (δ)-selective GABAA receptor agonist. OV101 is currently being evaluated in the pivotal Phase 3 NEPTUNE trial in Angelman syndrome.

“Ovid is deeply committed to the Angelman syndrome community. We have made great progress and are excited to see the topline data from our Phase 3 NEPTUNE trial with OV101 expected in Q4 2020,” said Amit Rakhit, MD, MBA, President and Chief Medical Officer of Ovid Therapeutics in the release. “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. If successful, OV101 may be used in combination with genetic approaches in the future to address the needs of Angelman syndrome. This collaboration with Drs. Chamberlain and Germain, both accomplished scientific leaders in the field of Angelman syndrome, will enable us to accelerate and share in their mission to identify and develop next-generation genetic therapies. 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.”

Read the full press release here.

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