Drug intelligence / Profile preview

AAV2.IvtB

Development stage
Preclinical
Lead developer
UMass Chan Medical School
Modality
Gene Therapies
Administration
Intravitreal
01

Overview

AAV2.IvtB is a novel, engineered recombinant adeno-associated virus (rAAV) capsid designed for efficient intravitreal (IVT) gene delivery to the outer retina, specifically targeting photoreceptors and Müller glia. Developed by researchers at the University of Massachusetts Chan Medical School, the capsid was discovered using a receptor-guided strategy that optimizes for reduced heparan sulfate (HS) affinity to facilitate penetration through the inner limiting membrane and enhanced engagement with N-acetyl-glucosamine (N-GlcNAc) moieties expressed on photoreceptor segments. In preclinical models, including mice and pigs, AAV2.IvtB demonstrated significantly higher transduction efficiency and therapeutic efficacy compared to the benchmark AAV2.7m8 capsid. It has been evaluated for the delivery of therapeutic transgenes such as KH902 (an anti-VEGF fusion protein) for choroidal neovascularization and Pde6b for inherited retinal degeneration.

02

Targets

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

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