Quiver Bioscience Receives Strategic Investment From the Porta Family to Advance UBE3A ASO Therapeutic Program for Dup15q Syndrome

Mission-driven investment will fund critical safety studies accelerating the path to a treatment for Dup15q syndrome.

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, today announced a targeted investment from the Porta family (Argentina) office to advance its antisense oligonucleotide (ASO) therapeutic program for chromosome 15q duplication (Dup15q) syndrome. The investment will fund preclinical activities essential to the program, including safety studies for development candidate selection on the path toward clinical development.

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. The current prevalence estimates for Dup15q syndrome are as high as 1 in 4,000 live births. There are currently no disease-modifying treatments for Dup15q, which remains a substantial unmet medical need.

Quiver's ASO program takes a precision medicine approach to target reduction of excess UBE3A gene expression, which is the underlying molecular driver of the condition. The company has leveraged its unique 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 in patient-derived models and identify and optimize ASO candidates capable of reducing UBE3A and rescuing associated human Dup15q patient-specific cellular phenotypes. Quiver's ASO program has already undergone extensive rodent toxicology assessment to produce a promising set of lead candidates. With the support of the Porta family investment, Quiver will progress lead ASO molecules through safety and tolerability studies towards selection of a final development candidate in 2H 2026.

The Porta family's commitment is driven by deep personal connection to the Dup15q community. "We see in Quiver an extraordinary team with the tools, the talent, and the vision to make a real treatment for Dup15q possible. As the father of a daughter living with this condition, this is not an ordinary investment — it is a deeply personal commitment to every family navigating this journey every single day. We are also grateful to the Dup15q Alliance for their guidance and for connecting families like ours with the right people and science to make a difference. We are here to support them and to help ensure that all those living with Dup15q have access to a therapy as soon as possible," said José A. Porta, President of Porta Hnos S.A.

“There are currently no disease-modifying treatments for Dup15q syndrome, a reality that affects thousands of families worldwide. This investment will be a catalyst towards achieving a critical milestone for Quiver’s ASO program, bringing us one step closer to our goal of delivering meaningful treatments for the Dup15q community. The Quiver team is grateful to the Porta family and Dup15q Alliance for their trust and support of our mission,” said Co-Founder and CEO, Graham T. Dempsey.

The Dup15q Alliance, a longstanding partner to Quiver in this program, welcomes news of the investment. “This investment is a powerful signal of what’s possible when families, science, and community come together around a shared goal,” said Mike Porath, executive director of the Dup15q Alliance. “We see this as more than funding. It’s real momentum toward a future where Dup15q is treatable. The leadership of both Quiver and the Porta family brings us meaningfully closer to that reality.”

About Quiver Bioscience

Quiver Bioscience is building the world's leading human functional intelligence platform for CNS drug discovery and advancing an internal pipeline of precision therapeutics for neurological and pain disorders. The company's proprietary platform is anchored by the largest functional neuronal dataset comprised of over 1.5 billion action potentials across over 30 million individual neurons, 18,000 gene perturbations, and 3,500 drug fingerprints enabling precise target identification, functional validation, and safety assessment at unmatched scale. Quiver's integrated wetware, hardware, and AI software architecture operates as a self-reinforcing lab-in-the-loop, where every experiment improves the underlying models and every partnership deepens the platform's competitive advantage. The platform has been validated across several CNS programs and partnerships and has yielded multiple therapeutic candidates now advancing through IND-enabling development. For more information, visit www.quiverbioscience.com.

About Porta Hnos S.A.

Porta is an Argentine family-owned company with 144 years of history dedicated to transforming grains into food products, sanitation solutions, and human and animal nutrition products. Driven by innovation and sustainability, Porta also develops industrial and technological solutions for other companies. The company is a certified B Corp, reflecting its long-standing commitment to people, communities, and the environment.

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. https://dup15q.org/