Key Takeaways Aurion Biotech has begun dosing patients in its US phase 3 ASTRA trial of AURN001, a regenerative cell therapy that could become the first approved treatment of its kind for corneal endothelial disease The therapy uses cultured human corneal endothelial cells combined with a rho-kinase inhibitor, delivered via a single injection The trial is a critical step toward US regulatory approval Aurion Biotech announced that the first patients have been dosed in the US phase 3 ASTRA clinical trial evaluating AURN001, an investigational therapy for corneal edema caused by corneal endothelial dysfunction. The milestone marks a step toward what could become the first approved cell therapy in the United States for this condition, a leading cause of vision impairment globally. “Dosing the first ASTRA study participants marks an important milestone for patients, physicians, and Aurion as we work together to advance new solutions for vision loss from corneal endothelial disease,” said Edward J. Holland, MD, Chief Medical Officer at Aurion Biotech. He noted that while endothelial keratoplasty procedures have improved outcomes, challenges remain. “AURN001 represents the potential next evolution by moving from donor tissue-based graft surgery toward a cultured cell therapy designed to restore corneal clarity and expand access to care for patients worldwide.” The ASTRA study is a pivotal phase 3 trial that will enroll patients across 12 sites in the United States. Participants will receive AURN001 in combination with Y-27632, a rho-kinase inhibitor. The primary endpoint is the proportion of patients achieving a clinically meaningful improvement in vision—defined as a gain of at least 15 letters in Best Corrected Visual Acuity (BCVA)—at 6 months. Secondary measures include changes in corneal thickness and additional visual acuity improvements. AURN001 is a first-in-class combination therapy composed of cultured human corneal endothelial cells (neltependocel) and Y-27632. Delivered via a single intracameral injection, the therapy is designed to regenerate the corneal endothelium, restore clarity, and potentially reduce complications associated with traditional transplant surgery while enabling faster recovery. “This pivotal study is the foundation for our U.S. biologics license application and a critical step in our mission to address a very large global unmet patient need,” said Arnaud Lacoste, PhD, MBA, the company’s CEO and Chief Scientific Officer. “By using donor corneas to create scalable and transportable cell therapy, we are overcoming the limitations of traditional surgery.” The company pointed to prior success in Japan as a foundation for its global ambitions, with the ASTRA trial representing a key step toward expanding access to regenerative treatments for blindness worldwide. More information about the ASTRA trial is available at aurionclinicaltrials.com and on ClinicalTrials.gov under identifier NCT07368959.