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AAV2.XG
- Development stage
- Preclinical
- Lead developer
- Akron Children's Hospital
- Modality
- Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, scFv Fragments → Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics
- Administration
- Intravitreal
01
Overview
AAV2.XG is an engineered adeno-associated virus (AAV) serotype 2 capsid variant developed for ocular gene therapy. It was identified through rational design and directed evolution in non-human primates to achieve high retinal transduction efficiency via intravitreal delivery. In the context of neovascular age-related macular degeneration (nAMD), the AAV2.XG vector is used to deliver dual-expression cassettes encoding single-chain variable fragments (scFvs) that target both vascular endothelial growth factor (VEGF) and angiopoietin-2 (ANG-2). By simultaneously inhibiting these two key pathways of angiogenesis, the therapy aims to provide a long-term alternative to frequent anti-VEGF injections.
02
Targets
VEGFA (Vascular endothelial growth factor A)Vascular niche (Vascular biology niche)HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))
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