CAMBRIDGE, Mass. — Quiver Bioscience has received a strategic investment from the Porta family to support development of its antisense oligonucleotide (ASO) therapy targeting Dup15q syndrome, a rare neurodevelopmental disorder with no disease-modifying treatments.
The funding will support key preclinical work, including safety studies needed to select a final development candidate as the program moves toward clinical trials. Company officials said the investment will help accelerate progress toward a potential treatment.
Dup15q syndrome is linked to increased expression of the UBE3A gene and is associated with symptoms including low muscle tone, developmental delays, intellectual disability, epilepsy, and autism spectrum disorder. Many patients experience seizures and face a heightened risk of sudden unexpected death in epilepsy. Prevalence estimates suggest the condition may affect as many as one in 4,000 live births.
Quiver’s ASO program is designed to reduce excess UBE3A expression, addressing the underlying cause of the disorder. The company said it has used a platform combining human single-cell neuronal electrophysiology data with artificial intelligence and machine learning to model the disease and identify promising therapeutic candidates. Early testing, including rodent toxicology studies, has produced a group of lead candidates.
With the new investment, Quiver plans to advance those candidates through safety and tolerability studies, aiming to select a final development candidate in the second half of 2026.
The investment is driven in part by a personal connection to the condition.
“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, which has worked with Quiver on the program, said the funding represents meaningful progress.
“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.”
