Gene therapy in retinal neovascular disease: a recent update

Authors: Li Yuntong, Yan Wenjia, Ding Xiaoyan

Corresponding author: Ding Xiaoyan, Email: dingxiaoyan@gzzoc.com

Published:2026-09-10

DOI: 10.3760/cma.j.cn115989-20230324-00101

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ABSTRACT 

Retinal diseases characterized by neovascularization or vascular leakage, such as neovascular age-related macular degeneration, diabetic retinopathy, and secondary choroidal neovascularization, are common retinal vascular disorders that can result in irreversible vision loss. Currently, the standard treatment for these retinal vascular disorders is intravitreal injection of anti-vascular endothelial growth factor agents. However, this treatment is costly, requires frequent injections, and has a high medical and financial burden for patients. Hence, there is an urgent need to develop more effective and longer-lasting therapeutic strategies. The success of gene therapy in inherited retinal diseases has provided both a theoretical foundation and a practical basis for its application to retinal vascular pathologies. As of August 2026, several anti-angiogenic gene therapy candidates, including RGX-314, 4D-150, and LX102, have entered phase Ⅲ clinical trials. Nevertheless, their translation into routine clinical practice still faces significant challenges, including optimization of administration routes, refinement of delivery vectors, exploration of novel therapeutic targets, and rigorous assessment of long-term safety. This review outlines the fundamental components of gene therapy and summarizes the current state of research on anti-angiogenic gene therapy in the treatment of retinal neovascular diseases.

KEYWORDS：

Gene therapy;Neovascularization;Vascular endothelial growth factor;Age-related macular degeneration;Diabetic retinopathy

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Authors Info & Affiliations 

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References click to collapse

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