Aurion Biotech Completes Enrollment in U.S. Phase 3 ASTRA Study of Regenerative Cell Therapy AURN001
Aurion Biotech, Inc., a commercial-stage regenerative cell therapy company focused on restoring vision, has announced the completion of patient enrollment in its U.S. ASTRA Phase 3 pivotal study evaluating AURN001, an investigational, single-administration regenerative cell therapy for corneal edema associated with corneal endothelial dysfunction.
The milestone marks a significant step in the company’s U.S. clinical development program as Aurion Biotech advances AURN001 toward potential regulatory submission. The company is developing the therapy as a potential first cell therapy approved in the United States for the treatment of corneal edema secondary to corneal endothelial dysfunction.
Corneal endothelial disease can impair the ability of the cornea to maintain its normal clarity, ultimately affecting vision. Diseases affecting the corneal endothelium include Fuchs endothelial corneal dystrophy, a condition in which corneal endothelial cells are progressively lost or become dysfunctional. As the number and function of these cells decline, fluid can accumulate in the cornea, resulting in corneal edema and reduced visual acuity.
Aurion Biotech is developing AURN001 as a regenerative approach intended to address the underlying loss or dysfunction of corneal endothelial cells rather than relying solely on conventional surgical intervention.
ASTRA Phase 3 Study Reaches Full Enrollment
The completion of enrollment in ASTRA represents an important clinical milestone for Aurion Biotech. As a pivotal Phase 3 study, ASTRA is designed to provide clinical evidence that could support the company’s planned Biologics License Application (BLA) submission to the U.S. Food and Drug Administration.
The study is evaluating AURN001 in patients with corneal edema caused by corneal endothelial dysfunction. The therapy is administered as a single intracameral injection, with the objective of restoring corneal clarity and improving vision through regenerative cell therapy.
Eris P. Jordan, OD, Chief Development Officer at Aurion Biotech, said completing enrollment was the result of collaboration among clinical investigators, study-site teams, patients and the company’s internal teams.
“Completing enrollment in ASTRA is a shared achievement,” Jordan said. She noted that reaching the final patient dosing milestone reflected the work of investigators and site teams, as well as patients who participated in the study.
The company also incorporated patient input into the design of the Phase 3 program. Through a dedicated patient advisory board, Aurion Biotech sought feedback from individuals affected by corneal endothelial disease regarding aspects of the clinical trial, including feasibility and outcomes that are important to patients.
According to Jordan, this patient-centered approach was intended to ensure that the pivotal trial addressed both regulatory requirements and the practical experiences of people living with the disease.
Regenerative Approach to Corneal Endothelial Disease
The corneal endothelium is a specialized layer of cells located on the inner surface of the cornea. These cells help regulate fluid balance within the cornea, allowing it to remain sufficiently clear for light to pass through.
Unlike many other cell types in the human body, corneal endothelial cells have limited ability to naturally regenerate. Significant loss or dysfunction of these cells can therefore result in corneal swelling and impaired vision.
Current treatment approaches for advanced corneal endothelial disease can involve surgical procedures that replace dysfunctional endothelial tissue with donor tissue. While corneal transplantation and related procedures can restore vision for appropriate patients, they involve surgical complexity and depend on the availability of suitable donor tissue.
The global shortage of donor corneal tissue represents an additional challenge. As demand for corneal procedures continues, access to donor material can vary significantly between regions.
Aurion Biotech believes a regenerative cell therapy that can be administered without conventional tissue transplantation could potentially address some of these limitations.
AURN001 is being developed as a combination therapy consisting of unmodified human corneal endothelial cells, known as neltependocel, together with the rho-kinase (ROCK) inhibitor Y-27632.
The therapy is delivered through an intracameral injection directly into the eye. The company’s approach is designed to support the restoration of corneal endothelial function and improve corneal clarity.
Potential Application in Fuchs Dystrophy
One of the important diseases associated with corneal endothelial dysfunction is Fuchs endothelial corneal dystrophy. The condition can cause progressive deterioration of corneal endothelial cells and, in more advanced stages, can result in corneal edema and vision impairment.
Because the disease is associated with the progressive loss of functional endothelial cells, regenerative strategies are being investigated as an alternative to procedures that depend on donor tissue.
Aurion Biotech describes AURN001 as a potential first-in-class investigational combination therapy for corneal endothelial disease, including Fuchs dystrophy.
The company’s approach is based on the concept that delivering healthy human corneal endothelial cells could help restore the cell population responsible for maintaining corneal fluid balance.
The inclusion of Y-27632, a ROCK inhibitor, is intended to support the behavior and survival of the transplanted cells. Together, the cell component and ROCK inhibitor form the investigational AURN001 therapy.
The Phase 3 ASTRA study is intended to provide further clinical evidence regarding the safety and effectiveness of this approach in patients with corneal edema associated with endothelial dysfunction.
Addressing Donor Tissue and Surgical Limitations
The potential importance of AURN001 extends beyond its mechanism of action. Aurion Biotech is also seeking to address some of the logistical limitations associated with existing approaches to corneal endothelial disease.
Donor tissue availability remains a challenge in many parts of the world. Conventional transplantation procedures can also require specialized surgical expertise and operating-room resources.
A cell therapy administered through an intracameral injection could potentially offer a different treatment model. If clinical development and regulatory review are successful, such an approach could provide a more scalable and transportable option for patients who have limited access to donor tissue or specialized corneal transplantation services.
The company emphasizes that these potential benefits remain dependent on the successful completion of clinical development and regulatory evaluation.
For patients, however, the underlying need is substantial. Progressive corneal endothelial disease can have a significant impact on vision and quality of life, particularly as the disease advances and corneal swelling becomes more pronounced.
Preparing for a Potential U.S. BLA Submission
The completion of ASTRA enrollment also brings Aurion Biotech closer to its planned U.S. regulatory strategy.
Arnaud Lacoste, PhD, MBA, Chief Executive Officer and Chief Scientific Officer of Aurion Biotech, described the completion of the pivotal study’s enrollment as a major milestone for the company and an important step toward a planned BLA submission.
“Completing enrollment in our U.S. pivotal study marks a major milestone for Aurion and an important step toward our planned BLA submission,” Lacoste said.
He credited the company’s regulatory, quality, manufacturing and clinical teams with establishing the infrastructure needed to advance the program.
The successful completion of enrollment means the company can now progress the study toward subsequent clinical milestones while continuing to prepare the regulatory and manufacturing components required for a potential U.S. filing.
For cell therapy developers, manufacturing consistency and quality control are particularly important components of regulatory development. Aurion Biotech is therefore building its clinical program alongside its manufacturing and quality infrastructure.
The company believes that its progress in these areas will support its objective of developing a regenerative cell therapy that can be manufactured and transported at scale.
Building on Development in Japan
Aurion Biotech’s U.S. program builds on the company’s previous clinical and commercial experience in Japan.
The company has previously advanced its regenerative cell therapy program in Japan, providing experience in clinical development and commercialization within a market that has established regulatory pathways for regenerative medicine products.
Lacoste said Aurion Biotech intends to build on its progress in Japan while developing a broader global solution for corneal endothelial disease.
The company is positioning AURN001 as a potentially scalable and transportable therapy that could address the needs of patients in multiple healthcare systems.
The U.S. Phase 3 program represents an important component of that strategy because regulatory approval in the United States could potentially expand access to regenerative treatment for patients with corneal endothelial disease.
Next Steps for AURN001
With enrollment now complete, the ASTRA study enters the next phase of its clinical development. The company will continue to follow patients and collect the data required to assess the investigational therapy according to the study protocol.
The resulting data will be important for determining the next regulatory steps for AURN001 and supporting the company’s planned BLA submission.
For Aurion Biotech, the enrollment milestone also demonstrates the company’s progress from developing regenerative cell therapy technology toward late-stage clinical development.
The company is pursuing an approach intended to address several interconnected challenges in corneal endothelial disease: loss of functional endothelial cells, limited donor tissue availability and the complexity associated with current surgical treatment.
AURN001 combines unmodified human corneal endothelial cells with a ROCK inhibitor and is administered through a single intracameral injection. The objective is to restore corneal endothelial function, improve corneal clarity and ultimately improve vision.
As the ASTRA study progresses toward its next milestones, Aurion Biotech will focus on completing the pivotal clinical program and preparing for potential regulatory review in the United States.
The completion of enrollment is therefore an important step in the company’s broader effort to advance regenerative cell therapy for vision restoration. If the Phase 3 program demonstrates the required clinical benefits and the therapy receives regulatory approval, AURN001 could introduce a new cell-based treatment option for patients with corneal endothelial dysfunction and associated corneal edema, including those affected by Fuchs dystrophy.
About ASTRA Phase 3 Study
Patients were enrolled at 12 sites across the U.S. The primary endpoint is the percentage of participants who achieve a ≥ 15-letter improvement (3-line gain) from baseline in Best Corrected Visual Acuity (BCVA) at Month 6. Secondary endpoints are changes from baseline in central corneal thickness (CCT) and best corrected visual acuity (BCVA) at Month 6. For more information on the ASTRA study, please visit: https://aurionclinicaltrials.com/ and NCT07368959 on clinicaltrials.gov.
About Corneal Endothelial Cell Therapy (CECT)
Aurion Biotech is advancing Corneal Endothelial Cell Therapy (CECT), a regenerative treatment that aims to replace lost or dysfunctional endothelial cells. Because endothelial cells cannot naturally regenerate, patients experience progressive vision loss. The cells delivered to patients are human corneal endothelial cells obtained from donated corneas. They do not contain any synthetic materials or artificial scaffold. The cells do not undergo genetic reprogramming. Aurion’s cells are intended to repopulate the endothelium and help re-establish a corneal structure that is virtually indistinguishable from a healthy cornea with healthy fluid balance.
About Aurion Biotech
Aurion’s mission is to restore vision to millions of patients with life-changing regenerative therapy. Aurion is a commercial stage company in Japan and a clinical stage company in Phase 3 development in the U.S. The company is developing AURN001, an investigational, single-administration, allogeneic cell therapy for corneal endothelial disease, a condition that causes progressive vision loss in millions of patients worldwide.
In 2024, Aurion launched Vyznova™, the first cell therapy for corneal endothelial disease commercially available in Japan. Aurion received the prestigious Prix Galien award for Best Start-Up in Biotech in 2022 and was named to Fast Company’s annual list of Most Innovative Companies in 2025. In 2025, Alcon acquired majority ownership of Aurion Biotech.