Purpose Regenerative ophthalmology is an emerging field aiming to restore vision by repairing or replacing damaged ocular tissues through stem-cell, bioengineered, and cell-free therapies. Despite increasing preclinical success, the clinical translation of these approaches remains limited and fragmented. This study provides a systematic landscape analysis of interventional clinical trials investigating regenerative therapies for eye diseases registered on ClinicalTrials.gov. Methods Clinical trial data were retrieved from ClinicalTrials.gov on February 09, 2026, using a modality-oriented search strategy targeting structural ocular regeneration. Interventional studies in Phases 1–3 evaluating regenerative modalities were included. Trials were analyzed by condition, intervention type, phase, enrollment, funding source, geographic distribution. Results A total of 46 trials were identified, of which 19 met eligibility criteria. The majority were early-phase studies (Phase 1, 74%), with small enrollment sizes (< 50 participants in most trials). Limbal stem cell deficiency represented the largest category (32%), followed by corneal endothelial disorders (26%), corneal surface disorders (21%), retinal and macular disorders (11%), and optic-nerve disorders (11%). Therapeutic strategies included cultivated epithelial grafts, endothelial cell injection, retinal pigment epithelium transplantation, recombinant biologics, and encapsulated neurotrophic implants. Visual function outcomes were the most common primary endpoint, although structural and safety endpoints predominated in early-phase trials. Academic and hospital institutions conducted the majority of studies, while industry sponsorship was concentrated in biologic and protein-based therapeutics. Trials were conducted across multiple countries, with the United States leading in activity. Conclusions Clinical research in ocular regeneration is rapidly expanding but remains primarily exploratory. Corneal reconstruction and endothelial repair represent the most advanced areas of clinical translation, whereas retinal and optic-nerve therapies remain early in development. The heterogeneity of endpoints and predominance of early-phase studies indicate that current trials characterize feasibility and biological repair rather than definitive functional restoration.

Clinical landscape of eye regeneration

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2026年3月18日

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Liudmila Iamukova

摘要

Purpose

Regenerative ophthalmology is an emerging field aiming to restore vision by repairing or replacing damaged ocular tissues through stem-cell, bioengineered, and cell-free therapies. Despite increasing preclinical success, the clinical translation of these approaches remains limited and fragmented. This study provides a systematic landscape analysis of interventional clinical trials investigating regenerative therapies for eye diseases registered on ClinicalTrials.gov.

Methods

Clinical trial data were retrieved from ClinicalTrials.gov on February 09, 2026, using a modality-oriented search strategy targeting structural ocular regeneration. Interventional studies in Phases 1–3 evaluating regenerative modalities were included. Trials were analyzed by condition, intervention type, phase, enrollment, funding source, geographic distribution.

Results

A total of 46 trials were identified, of which 19 met eligibility criteria. The majority were early-phase studies (Phase 1, 74%), with small enrollment sizes (< 50 participants in most trials). Limbal stem cell deficiency represented the largest category (32%), followed by corneal endothelial disorders (26%), corneal surface disorders (21%), retinal and macular disorders (11%), and optic-nerve disorders (11%). Therapeutic strategies included cultivated epithelial grafts, endothelial cell injection, retinal pigment epithelium transplantation, recombinant biologics, and encapsulated neurotrophic implants. Visual function outcomes were the most common primary endpoint, although structural and safety endpoints predominated in early-phase trials. Academic and hospital institutions conducted the majority of studies, while industry sponsorship was concentrated in biologic and protein-based therapeutics. Trials were conducted across multiple countries, with the United States leading in activity.

Conclusions

Clinical research in ocular regeneration is rapidly expanding but remains primarily exploratory. Corneal reconstruction and endothelial repair represent the most advanced areas of clinical translation, whereas retinal and optic-nerve therapies remain early in development. The heterogeneity of endpoints and predominance of early-phase studies indicate that current trials characterize feasibility and biological repair rather than definitive functional restoration.