Quiver Bioscience Announces Collaboration with the Dup15q Alliance to Advance Antisense Oligonucleotide Therapeutic Program

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Quiver Bioscience (“Quiver”), a discovery technology and therapeutics company advancing programs for the treatment of serious central nervous system (CNS) disorders, announces today a collaboration with the Dup15q Alliance to advance an antisense oligonucleotide (ASO) therapeutic program for chromosome 15q duplication (Dup15q) syndrome using Quiver’s innovative “Genomic Positioning System” (GPS). There are currently no disease-modifying treatments for Dup15q, which remains a substantial unmet medical need.

Dup15q is a neurodevelopmental disorder characterized by moderate-to-severe hypotonia, motor and language delays, intellectual disability, epilepsy, and autism spectrum disorder (ASD). Most Dup15q individuals experience seizures and are at an increased risk for sudden unexpected death in epilepsy (SUDEP). Dup15q is associated with genomic copy number variations that result in increased levels of the UBE3A gene product. Dup15q syndrome is clinically related to Angelman syndrome (AS), a distinct neurodevelopmental disorder caused by UBE3A loss of function. ASO therapeutics for AS are currently in clinical trials and have shown early potential benefits in disease modification. The current prevalence estimates for Dup15q syndrome are as high as 1 in 4,000 live births.

Genetic medicine approaches, such as ASOs, to reduce UBE3A levels are a viable precision therapeutic path for the treatment of Dup15q and have disease-modifying potential. Quiver has leveraged its unique GPS platform, which integrates unique-in-world, scalable, human single-cell neuronal electrophysiology data with artificial intelligence and machine learning (AI/ML) analytics, and extensive experience in ASO design to robustly model Dup15q syndrome and identify, optimize, and advance effective ASO candidates capable of reducing UBE3A and rescuing associated human Dup15q patient-specific cellular phenotypes.

This initial grant formalizes a growing partnership between Quiver and the Dup15q Alliance that has been built over several years. The grant will support requisite preclinical studies to characterize Quiver’s lead therapeutic candidate ASOs and advance towards a transformative treatment for the Dup15q community. “We’re thrilled to continue working shoulder to shoulder with the excellent team at the Dup15q Alliance in advancing this important therapeutic for the community of patients and their families,” says Graham Dempsey, Ph.D., Founder and Chief Scientific Officer at Quiver Bioscience. “This is a devastating disease,” says Mike Porath, Executive Director of the Dup15q Alliance. “We are committed to developing a therapeutic treatment so people with Dup15q syndrome can thrive. We're excited to partner with Quiver to reach our ambitious goals as quickly as possible.”

About Quiver Bioscience

Quiver Bioscience is a technology-driven company established to create transformational medicines for the brain while simultaneously uncovering new biology and novel, effective drug targets. Using advanced single-cell imaging and multi-omics, we are building the world's most information-rich neuronal insight map via our "Genomic Positioning System". Our approach integrates cutting-edge scalable human models, state-of-the-art technology and proprietary engineering, and learning and surrogate AI/ML models to identify novel therapeutic targets and the best candidate molecules to deliver new and meaningful therapeutics to patients. For information, including additional publications describing the application of Quiver’s GPS to drug discovery, visit www.quiverbioscience.com.

About Dup15q Alliance

The Dup15q Alliance is a nonprofit organization devoted to bringing awareness to Dup15q syndrome and empowering individuals living with the condition by supporting scientific and clinical research towards effective therapeutics.