Drug intelligence / Profile preview

NAV AAV8

Development stage
Unknown
Lead developer
REGENXBIO
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravenous, Subretinal
01

Overview

NAV AAV8 is a proprietary adeno-associated virus serotype 8 (AAV8) gene delivery vector developed by REGENXBIO. It is part of the broader NAV Technology Platform, which includes a suite of novel recombinant adeno-associated virus vectors designed for efficient and targeted gene transfer. The primary mechanism involves using the engineered viral capsid to deliver therapeutic genes to specific tissues—most notably muscle and retinal tissue—enabling long-term expression of transgenes for the treatment of genetic diseases. The platform has been used in investigational therapies such as RGX-202 (for Duchenne muscular dystrophy) and RGX-314 (for wet age-related macular degeneration), where it delivers genes encoding functional proteins or antibody fragments directly into patient cells[2][4][5][6]. The versatility and efficiency of the NAV AAV8 vector have made it a key component in multiple clinical-stage gene therapy programs targeting rare diseases.

Other names
NAV AAV8 vectorNAV® AAV8REGENXBIO's proprietary AAV8 vector
02

Targets

AAVR (AAV receptor)

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