Drug intelligence / Profile preview

AAV2.N54

Development stage
Unknown
Lead developer
Avirmax Biopharma
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravitreal
01

Overview

AAV2.N54 is an engineered adeno-associated virus (AAV) capsid developed by Avirmax Biopharma to efficiently deliver therapeutic transgenes to the macular retina via intravitreal administration. It serves as the vector platform for gene therapies such as ABI-110, which are designed to treat retinal diseases including wet age-related macular degeneration (AMD) and polypoidal choroidal vasculopathy (PCV). The primary goal of using AAV2.N54 is to enable safe and effective gene delivery directly to the retina, potentially reducing or eliminating the need for frequent intravitreal injections that are standard in current care. The technology is currently being evaluated in Phase I/IIa clinical trials for its safety, tolerability, and preliminary efficacy[1][4][5][7].

02

Targets

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

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