Ocular Gene Therapy in China: Technological Framework, Clinical Translation, and Future Perspectives

Abstract

The eye is well suited to gene therapy because of its compartmentalized anatomy, accessibility for local delivery, and capacity for longitudinal assessment. In China, ocular gene therapy is undergoing rapid clinical translation, with expanding innovation in vector engineering, delivery technologies, genome editing, and therapeutic modalities. Clinical development has progressed from inherited optic neuropathies and retinal diseases to complex acquired ocular disorders, anterior-segment genome editing, and optogenetic vision restoration. This review summarizes the technological landscape and representative clinical advances of ocular gene therapy in China, highlighting the evolution from established gene-replacement approaches toward increasingly differentiated therapeutic strategies. We further discuss key challenges in interpreting clinical benefit and translating early clinical signals into durable, reproducible outcomes. Future progress will depend on clinically driven innovation, rigorous evidence of meaningful patient benefit, and standardized pathways for sustainable clinical translation.