Drug intelligence / Profile preview

AAV-PHP.S

Development stage
Preclinical
Lead developer
Broad Institute
Modality
Gene Therapies
Administration
Intravenous, Intracranial, Intrathecal
01

Overview

AAV-PHP.S is an engineered adeno-associated virus (AAV) capsid variant derived from AAV9, specifically designed for enhanced gene delivery to the peripheral nervous system (PNS). Developed through directed evolution techniques (such as CREATE), this vector exhibits a significantly improved ability to transduce peripheral sensory neurons, including those in the dorsal root ganglia and enteric nervous system, compared to its parent serotype when administered systemically. While its primary characterization involved bypassing the blood-nerve barrier in murine models, recent research has explored its utility in human tissues. In the context of neuro-oncology, AAV-PHP.S has demonstrated the ability to transduce GFAP-positive cells in human glioblastoma tissue, suggesting potential applications as a delivery vehicle for gene therapies targeting malignant brain tumors and other complex neurological conditions.

Other names
AAV9-PHP.SAAV-9-PHP.SAAV 9-PHP.SPHP.s
02

Targets

HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))

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