What's Happening?
Quiver Bioscience has received a strategic investment from the Porta family to advance its antisense oligonucleotide (ASO) therapeutic program for Dup15q syndrome. This investment will fund preclinical safety studies essential for the development of a treatment
for this neurodevelopmental disorder. Dup15q syndrome is characterized by symptoms such as hypotonia, motor and language delays, intellectual disability, epilepsy, and autism spectrum disorder. Quiver's ASO program aims to reduce excess UBE3A gene expression, the molecular driver of the condition. The company utilizes a unique platform integrating human neuronal data with AI and machine learning to identify and optimize ASO candidates.
Why It's Important?
The investment represents a significant step towards developing a treatment for Dup15q syndrome, a condition with no current disease-modifying therapies. The Porta family's involvement highlights the personal and community-driven commitment to addressing this unmet medical need. The advancement of Quiver's ASO program could lead to breakthroughs in precision medicine for neurological disorders, potentially improving the quality of life for individuals affected by Dup15q syndrome and their families.
What's Next?
Quiver Bioscience plans to progress its lead ASO molecules through safety and tolerability studies, aiming to select a final development candidate by the second half of 2026. The company's collaboration with the Dup15q Alliance and the support from the Porta family will be crucial in achieving these milestones. Successful development of a treatment could pave the way for clinical trials and eventual regulatory approval, offering hope to those affected by Dup15q syndrome.
