Toward Corneal Regenerative Medicine – Part 1: Epithelial Regeneration

Shigeru KinoshitaJapan Speaker Toward Corneal Regenerative MedicineSevere ocular surface and corneal disorders—including Stevens-Johnson syndrome, chemical injury, ocular cicatricial pemphigoid, Fuchs endothelial corneal dystrophy (FECD), and advanced corneal endothelial failure—remain difficult to manage. Recent advances in ocular surface biology and regenerative medicine have enabled the development of transplantable epithelial sheets, such as allogeneic/autologous corneal epithelial stem-cell sheets, autologous cultivated oral mucosal epithelial sheets (COMET), and iPSC-derived corneal epithelial sheets, some of which have received regulatory approval from the EMA and PMDA. Corneal endothelial dysfunction is also being addressed through regenerative medicine approaches. The transplantation (via injection) of mature, differentiated cultured human corneal endothelial cells (CHCEC) combined with a ROCK inhibitor into the anterior chamber has demonstrated favorable efficacy and safety, leading to PMDA approval in 2023 and subsequent clinical application in Japan beginning in September 2024. In addition, ROCK-inhibitor eye drops have demonstrated potential in treating early-stage endothelial dysfunction in FECD. Continued progress in corneal regenerative therapies promises to advance global regulatory acceptance and contribute meaningfully to the prevention of blindness worldwide.