Corneal Endothelial Cell Therapy

Corneal endothelial failure afflicts millions worldwide, yet only ~1 in 70 people who need a donor cornea ever receive one, leaving a huge, sight-robbing treatment gap. Traditional keratoplasty is invasive, technically demanding, and constrained by limited tissue supply, slow visual recovery in some cases, and the constant threat of immunologic graft rejection. 

Aurion Biotech is creating a solution: an off-the-shelf, allogeneic suspension of cultured human corneal endothelial cells (HCECs) combined with a ROCK inhibitor (Y27632). Each donor line can yield roughly 1,000 therapeutic doses, transforming a single transplant into population-scale medicine (Figure). The therapy removes the “carrier problem” that plagues partial-thickness grafts. Cultured HCECs settle onto the polished posterior cornea, where the ROCK inhibitor increases focal adhesion complexes and promotes rapid regeneration of a fully functional corneal endothelium.

Published Data

In a study, 11 eyes with undetectable endothelial cells received 1 × 10^6 cultured cells in combination with a ROCK inhibitor.¹ By 24 weeks, every cornea regained clarity, central CEC density averaged 1,924 cells/mm², and nine eyes gained ≥ 2 lines of vision. No serious ocular adverse events occurred.

Long-term follow-up of the same cohort showed 10 of 11 corneas remained clear; mean central endothelial density was still 1,257 ±467 cells/mm², and BCVA improved from 0.88 to 0.046 logMAR.² Specular microscopy revealed orderly, hexagonal mosaics reminiscent of healthy endothelium. Importantly, no late immune rejections, uveitis, or steroid-induced glaucoma threatened the grafts.

The El Salvador-based ESCALÓN trial extends these findings.³ Twenty-two pseudophakic eyes with bullous keratopathy or Fuchs dystrophy received the same cell dose plus three ROCK inhibitor concentrations. At 12 months, mean central corneal thickness fell from 697 µm to 571 µm, and 89% of patients achieved ≥ 3-line visual gains. No treatment-related serious adverse events emerged; transient increases in intraocular pressure in 23% of eyes resolved with short-term drops.

Regulatory Horizon and ScaleUp

MHLW and PMDA in Japan granted the therapy (Vyznova™) full approval in 2023, validating both its safety profile and manufacturing strategy. The FDA granted Aurion’s AURN001 program Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy designation on June 14, 2024, and Aurion plans on conducting a pivotal trial in the U.S. by 2026.

A New Corneal Era

By converting a scarce tissue transplant into a reproducible therapy, HCEC therapy offers patients rapid visual recovery, surgeons a shorter learning curve, and payers a scalable, value-based alternative. In addition, Aurion’s ability to create 1,000 therapeutic doses from tissue obtained from a single donor ensures more efficient use of precious donated corneas and increases the impact that eye banks have on patients in need.

1. Kinoshita S, et al. N Engl J Med. 2018;378(11):995-1003.

2. Numa K, et al. Ophthalmology. 2021;128(4):504-514.

3. Holland EJ, et al. Escalón: Cornea. Published online: March 3, 2025.

For more information: https://aurionbiotech.com/

For inquiries, contact Michele Gray at michele@mgraycommunications.com