Aptamers in Ocular Disease Therapy and Drug Delivery: Current Progress and Future Opportunities

Aptamers are single-stranded oligonucleotides with the ability to bind specific target molecules with high affinity and selectivity, positioning them as valuable tools for precision therapeutics and targeted drug delivery. In ophthalmology, aptamer-based platforms have emerged as effective solutions for managing retinal diseases, glaucoma, and ocular surface disorders. This review provides a comprehensive overview of aptamers that have received approval or are in experimental, preclinical, or clinical development stages for ocular diseases.

A narrative literature search was conducted using PubMed, Scopus, Cochrane Library, Embase, and Web of Science databases, concentrating on aptamers that have reached experimental, preclinical, clinical, or FDA-approved stages. Studies published up to March 2026 were screened according to predefined inclusion and exclusion criteria, based on their reported efficacy, molecular targets, and relevance to ocular diseases.

Twenty-four records were included, comprising 24 aptamer-based platforms. Across ocular diseases, aptamers were identified in three principal application domains: therapeutic intervention, diagnostic, and targeted drug delivery. In age-related macular degeneration, aptamers development was predominantly therapeutic, with agents targeting vascular endothelial growth factor, platelet-derived growth factor, fibroblast growth factor 2, complement components, nucleolin, CBF1, and CD44. In glaucoma, aptamers were mainly directed toward neuroprotection, antifibrotic modulation, aqueous humor outflow regulation, biomarker detection, and topical drug delivery. In ocular surface diseases, aptamers were primarily developed for antimicrobial and antiviral therapy, antiangiogenic treatment, anti-allergic modulation, corneal surface targeting, and controlled topical drug delivery. Overall, aptamers demonstrated broad translational versatility across both posterior and anterior segment diseases.

The available evidence demonstrates a diverse and rapidly expanding repertoire of aptamers developed for ocular diseases, underscoring their versatile potential as next-generation platforms for therapeutic, biomarker-guided diagnosis, and targeted drug delivery in ophthalmology.