VEGF-A

Class

AAV8 vector-based in-vivo gene therapy encoding a secreted anti-VEGF Fab (ranibizumab-like); AbbVie-partnered/licensed version of REGENXBIO's RGX-314, delivered via a single subretinal or suprachoroidal injection for sustained intraocular anti-VEGF protein expression (INN: surabgene lomparvovec)

Pathway

VEGF-A / VEGFR angiogenic signaling

VEGF-A (vascular endothelial growth factor A) is a signaling protein that binds receptors on blood vessel lining cells, driving new vessel formation and increasing vessel permeability. In the eye, excessive VEGF-A activity underlies several blinding conditions, including wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy, where abnormal, leaky vessels grow beneath or within the retina and cause fluid accumulation, hemorrhage, and progressive vision loss. Neutralizing VEGF-A reduces this abnormal vascularization and vascular leakage, which is why anti-VEGF therapy has become a mainstay of retinal disease management. Conventional treatment requires repeated intraocular injections of antibody-based drugs because these proteins are cleared over weeks to months. Gene therapy approaches address this limitation by delivering, via a single injection, a viral vector containing genetic instructions for retinal cells to continuously produce their own anti-VEGF protein, in this case a secreted antibody fragment. The goal is durable, self-sustaining suppression of VEGF-A activity within the eye, potentially reducing the treatment burden associated with chronic dosing while achieving the same underlying biological effect as injected anti-VEGF biologics.