Drug intelligence / Profile preview

AAV.PHP.eB-CAG-GFP

Development stage
Preclinical
Lead developer
California Institute of Technology
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intracisternal, Intrathecal, Intravenous
01

Overview

AAV.PHP.eB-CAG-GFP is an engineered adeno-associated virus (AAV) gene therapy vector designed for high-efficiency, widespread transduction of the central nervous system (CNS). It utilizes the PHP.eB capsid, a variant of AAV9 engineered through Cre-recombination-based AAV learner evolution (CREATE) to significantly enhance blood-brain barrier (BBB) penetration. The vector carries a Green Fluorescent Protein (GFP) reporter gene under the control of the strong, constitutive CAG promoter (comprising the CMV early enhancer, chicken beta-actin promoter, and rabbit beta-globin splice acceptor). In preclinical research, particularly in rodent models, AAV.PHP.eB-CAG-GFP demonstrates superior biodistribution throughout the brain and spinal cord compared to its parent serotype, AAV9, when delivered via intravenous or intrathecal routes. It is primarily used as a research tool to evaluate CNS gene delivery efficiency and as a prototype for developing neuroprotective gene therapies for disorders such as Parkinson's disease.

Other names
AAV-PHP.eB-CAG-GFPAAV.PHP.eB-GFP
02

Targets

Galactose-terminated glycansLy6A (Lymphocyte antigen 6 complex locus A)Galactose (Terminal N-linked galactose-containing glycans)

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