Overview

Jaya Biosciences is developing CNS-directed gene therapies for neurodegeneration, including Alzheimer’s disease. We are pursuing a novel paradigm based on the breakthrough discovery that mutations in certain lysosomal genes are highly enriched in patients with neurodegenerative phenotypes. To date, we have validated five lysosomal gene targets in vivo. Our lead program, JB111, is an experimental CNS-directed AAV9-mediated PPT1 gene therapy currently being developed for Alzheimer’s associated with PPT1 haploinsufficiency. JB111 showed very promising results in the animal model of PPT1-associated Alzheimer’s disease. A single intracerebroventricular injection of JB111 resulted in a significantly increased life span and improved cognitive performance.