We use adeno-associated virus (AAV) as a vector to deliver functional copies of the ASPA gene throughout the body and into the brain, with the goal of addressing the underlying cause of Canavan disease. Unlike many other AAV gene therapy approaches, our investigational treatment is delivered intravenously—providing a less invasive option for families.

Our gene therapy was developed by Guangping Gao, Ph.D. and Dominic J. Gessler, M.D. at UMass Chan Medical School. Dr. Gao, a pioneer in AAV gene therapy, was the first to clone the ASPA gene in 1993 and has dedicated more than 25 years to research aimed at treating Canavan disease.