Aurion Biotech has dosed the first patients in its pivotal phase 3 ASTRA trial, marking a key step toward bringing regenerative cell therapy to patients with corneal endothelial disease. The US-based study will evaluate AURN001, an investigational, single-administration therapy for corneal edema secondary to endothelial dysfunction, including Fuchs dystrophy.
The trial will enrol patients across 12 sites in the United States and assess both efficacy and safety against placebo. Its primary endpoint is a ≥15-letter improvement in best corrected visual acuity (BCVA) at six months, alongside secondary measures including central corneal thickness and additional visual acuity outcomes.
AURN001 combines cultured human corneal endothelial cells (neltependocel) with a rho-kinase inhibitor (Y-27632), delivered via a single intracameral injection. The approach is designed to regenerate the corneal endothelium – an area of the eye with limited natural regenerative capacity – potentially restoring corneal clarity and vision without the need for donor tissue graft surgery.
Current standard-of-care treatments, such as endothelial keratoplasty, rely on donor corneas and are associated with surgical complexity and potential complications. Aurion positions AURN001 as a next-generation alternative, aiming to reduce procedural burden while expanding access to treatment globally.
“Dosing the first ASTRA study participants marks an important milestone for patients, physicians, and Aurion,” said Edward J. Holland, Chief Medical Officer at Aurion Biotech, in the company’s announcement. The company’s leadership emphasized the broader ambition to move beyond tissue-based transplantation toward scalable, cell-based therapies.
The program builds on Aurion’s earlier progress in Japan, where its corneal endothelial cell therapy has already reached commercialization. With the ASTRA study now underway, the company is targeting a future U.S. biologics license application, positioning AURN001 as a potential first-in-class therapy in this space.
Corneal endothelial dysfunction remains a major cause of visual impairment worldwide, driven in part by the inability of endothelial cells to regenerate. By using donor-derived cells expanded into a scalable therapy, Aurion aims to address longstanding supply constraints associated with corneal transplantation.
While clinical outcomes will determine its ultimate impact, the initiation of phase 3 signals growing momentum behind regenerative approaches in ophthalmology – particularly those seeking to replace, rather than transplant, diseased tissue.
Source: BusinessWire.
