rAAV9.ASPA

rAAV9.ASPA is an investigational gene therapy candidate designed for the treatment of Canavan disease, a progressive and fatal neurodegenerative condition caused by mutations in the aspartoacylase (ASPA) gene. The therapy utilizes a recombinant adeno-associated virus serotype 9 (rAAV9) vector to deliver a functional copy of the human ASPA gene to the central nervous system. Canavan disease is characterized by the accumulation of N-acetylaspartate (NAA) in the brain, which leads to spongiform degeneration and severe neurological symptoms. rAAV9.ASPA aims to restore ASPA enzyme activity, thereby reducing NAA levels and mitigating neuroinflammation. Preclinical research conducted at the University of Massachusetts Chan Medical School has demonstrated that systemic administration of rAAV9.ASPA can normalize levels of Galectin-3, a biomarker of microglial activation, and improve neuropathological outcomes in mouse models of the disease.

Other names

rAAV9-hASPArAAV-9-hASPArAAV 9-hASPAAAV9-ASPAAAV-9-ASPAAAV 9-ASPArAAV9 expressing human ASPArAAV-9 expressing human ASPArAAV 9 expressing human ASPA