Aspa Therapeutics: A Study of AAV9 Gene Therapy in Children With Canavan Disease
(CANaspire Clinical Trial) [https://treatcanavan.com/canaspire-trial/]
Aspa’s investigational gene therapy trial uses an AAV9 vector (adeno-associated virus serotype 9), which is designed to deliver functional copies of the ASPA gene throughout the body, including into the brain, to address the underlying cause of Canavan disease. The AAV9 vector Aspa is using for its investigational gene therapy has been studied in other rare disease gene therapy clinical trials, and is the vector used in the recently approved first gene therapy for spinal muscular atrophy, a pediatric neurodegenerative disease. Evidence from human studies shows that the AAV9 vector when given intravenously (IV) gets into the central nervous system as well as tissues throughout the body.
