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Corneal Endothelial
Cell Therapy

Two lab workers on either edge of the frame, turned looking at each other and smiling while holding lab equipment in front of a lab bay.

Our initial candidate aims to treat dystrophies that cause corneal blindness.

Healthy cells from a donor cornea are propagated in a novel, multistep, proprietary, and patented process. From a single donor, we believe we have the potential to manufacture corneal endothelial cells to treat up to 1,000 recipient eyes. We believe our cell therapy procedure is designed to be considerably less complex, easier to learn and more efficient to perform than corneal transplantation. Post-operative recovery for cell therapy typically involves patients lying face down for three hours, as compared to patients typically lying on their backs for up to three days for corneal endothelial transplant procedures.

Pipeline

In Japan, early clinical research1 followed by dose-ranging and confirmatory clinical trials have successfully demonstrated the safety, efficacy, and tolerability of our first-generation cell therapy product, Vyznova. We have received regulatory approval from Japan’s PMDA.

In the United States and Canada, we are currently conducting a Phase 1/2 trial for our lead product candidate, AURN001. Enrollment and dosing for this trial have been completed and we announced topline data in the fourth quarter of 2024.

Product/
Product Candidate		 Indication Preclinical Phase 1 Phase 2 Phase 3 Approved Next Anticipated Milestone
Vyznova Bullous keratopathy of the cornea Approved (Japan) Approval of Supplemental New Drug Application submitted to the MHLW in September 2024
AURN001 Corneal edema secondary to corneal endothelial dysfunction Phase 1/2 (U.S.) * Twelve-month topline results from U.S. Phase 1/2 trial in 2H 2025
Cryopreserved formulation** Corneal edema secondary to corneal endothelial dysfunction Twelve-month topline results from pilot clinical trial in Q1 2026
* Currently in Phase 1/2 development in the United States and Canada and clinical development planning stage in the EU
** Trial is taking place in El Salvador
Product/Product Candidate
Vyznova
Indication
Bullous keratopathy of the cornea
Phase
Approved (Japan)
Next Anticipated Milestone
Approval of Supplemental New Drug Application submitted to the MHLW in September 2024
Product/Product Candidate
AURN001
Indication
Corneal edema secondary to corneal endothelial dysfunction
Phase
Phase 1/2 (U.S.) *
Next Anticipated Milestone
Twelve-month topline results from U.S. Phase 1/2 trial in 2H 2025
Product/Product Candidate
Cryopreserved formulation**
Indication
Corneal edema secondary to corneal endothelial dysfunction
Phase
N/A
Next Anticipated Milestone
Twelve-month topline results from pilot clinical trial in Q1 2026

* Currently in Phase 1/2 development in the United States and Canada and clinical development planning stage in the EU

** Trial is taking place in El Salvador

Reference

  1. Kinoshita S, Koizumi N, Ueno M, et al. Injection of cultured cells with a ROCK inhibitor for bullous keratopathy. N Engl J Med. 2018;378(11):995-1003.

How it works