Drug intelligence / Profile preview

AAV.CAP-B10-GFP

Development stage
Preclinical
Lead developer
California Institute of Technology
Modality
Gene Therapies
Administration
Intravenous
01

Overview

AAV.CAP-B10-GFP is a research-stage adeno-associated virus (AAV) gene delivery vector designed for high-efficiency transduction of the central nervous system (CNS). It utilizes the AAV.CAP-B10 capsid, an engineered variant of AAV9 developed by the Gradinaru Lab at Caltech, which exhibits a significantly enhanced ability to cross the blood-brain barrier (BBB) and a four-fold increase in neuronal transduction compared to parental AAV9, while also showing reduced liver tropism. The vector carries a Green Fluorescent Protein (GFP) reporter transgene, making it a valuable tool for mapping CNS transduction patterns and evaluating the delivery efficiency of the CAP-B10 capsid in vivo. While the GFP version is primarily a research reagent, the CAP-B10 platform is being explored for therapeutic applications in neurological disorders such as KCNQ2-associated genetic epilepsy and traumatic brain injury.

02

Targets

AAVR (AAV receptor)Cell-surface glycans with terminal galactose

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