CAMBRIDGE, Mass. — Quiver Bioscience and QurAlis Corporation have announced a research collaboration aimed at developing a novel gene-targeted therapy for Fragile X syndrome (FXS), a leading inherited cause of intellectual disability and autism spectrum disorder.
The partnership will combine Quiver’s proprietary “Genomic Positioning System” (GPS) drug discovery platform with QurAlis’ expertise in precision medicines for neurological and neurodegenerative diseases. The goal is to generate foundational research data that could support the development of a potentially transformative treatment for FXS, which currently lacks any approved disease-modifying therapies.
Quiver’s GPS platform uses scalable human neuronal electrophysiology data—what the company calls the “language of the brain”—alongside artificial intelligence and machine learning to uncover new insights into disease biology and accelerate drug discovery. The company has already applied the platform to various central nervous system disorders and recently reported progress in FXS modeling.
“Our technology is uniquely suited to unlocking the cellular and molecular mechanisms of neurogenetic disorders like Fragile X,” said Dr. Graham Dempsey, co-founder and CEO of Quiver Bioscience. “This collaboration with QurAlis is an important step toward delivering breakthrough treatments to the Fragile X community.”
Fragile X syndrome is caused by the loss of the FMRP protein, encoded by the FMR1 gene. It affects roughly 87,000 individuals in the United States, with symptoms ranging from intellectual disability and developmental delays to anxiety, seizures, speech challenges, and attention deficit issues. The disorder affects about 1 in 4,000 males and 1 in 6,000 females.
Dr. Kasper Roet, CEO and co-founder of QurAlis, emphasized the urgency of developing new therapies for FXS. “This is a devastating neurodevelopmental disease, and the need for disease-modifying treatments is critical,” he said. “By partnering with Quiver, we are combining cutting-edge technologies and deep drug development experience to pursue promising new therapeutics.”
Quiver was advised on the transaction by Destum Partners.