GENE AND STEM CELL THERAPIES FOR AGE-RELATED MACULAR DEGENERATION: RECENT ADVANCES AND TRANSLATIONAL PERSPECTIVES

Abstract

This study aimed to critically review recent evidence on gene and cell therapies for age-related macular degeneration (AMD). An integrative review was conducted in PubMed/MEDLINE, LILACS, SciELO, BVS and ClinicalTrials.gov, between 2020 and 2025, including phase I–III clinical trials, translational studies, and systematic reviews. A total of 25 studies were analyzed. Gene therapy using adeno-associated viral vectors, such as RGX-314, Ixo-vec and 4D-150, showed up to 80% reduction in retreatment need for neovascular AMD, with sustained vision for up to two years. Cell therapy, particularly OpRegen, led to average gains of +6 to +9 letters maintained for 36 months in patients with geographic atrophy, along with OCT-documented structural improvement. Trials with induced pluripotent stem cells confirmed safety, although functional outcomes remain limited. In conclusion, gene and cell therapies represent a milestone in AMD treatment, offering complementary approaches that may constitute the second major revolution in ophthalmology, with clinical, social, and economic impact.