NAV AAV9

NAV AAV9 is a proprietary gene therapy delivery vector based on adeno-associated virus serotype 9 (AAV9), developed as part of the NAV Technology Platform by REGENXBIO. It is engineered to deliver therapeutic genes to target cells, particularly for the treatment of genetic diseases affecting both systemic and central nervous system (CNS) tissues. The vector is designed to be non-replicating and not known to cause disease in humans. By delivering a functional copy of a missing or defective gene into patient cells, it enables production of therapeutic proteins that can correct underlying metabolic or genetic defects. The platform has been licensed for use in multiple investigational gene therapies targeting rare lysosomal storage disorders such as Sanfilippo syndrome type A (MPS IIIA), type B (MPS IIIB), infantile Batten disease (CLN1), juvenile Batten disease (CLN3), and other CNS-related conditions[1][4][5]. Its design allows for broad tissue tropism with reduced immunogenicity and potential long-lasting effects from single administration.

Other names

NAV AAV9NAV Vector AAV9REGENXBIO AAV9 vector