In a groundbreaking stride within the realm of ophthalmological medicine, Aurion Biotech, a clinical-stage biotech company with a visionary aim to restore vision through regenerative therapies, has recently championed a significant achievement by administering the first Canadian subject in a Phase 1 / 2 clinical trial. Known as ABA-1, or CLARA, this trial is exploring the efficacy of AURN001, a pioneering cell therapy devised for the treatment of corneal edema triggered by corneal endothelial dysfunction—a condition that detrimentally impacts the vision of millions globally.

Michael Goldstein, MD, MBA, President and Chief Medical Officer of Aurion Biotech, expressed enthusiasm over this development, emphasising the trial’s critical role in their expansive clinical program. By extending the trial to include subjects from both the United States and Canada, Aurion Biotech marks a pivotal point in its journey towards establishing a new paradigm in corneal patient care globally.

At the heart of AURN001 lies a unique blend of biologic/drug—a combination cell therapy product comprising neltependocel (allogeneic human corneal endothelial cells [CECs]) and Y-27632 (an inhibitor of RHO-associated, coiled-coil containing protein kinase [ROCK]). This innovative concoction is tailored for a one-time, intracameral injection, showcasing a promising alternative to current treatments for corneal endothelial dysfunction. The ABA-1, CLARA trial aims to decipher the optimal dosage by assessing three different doses of neltependocel in tandem with Y-27632 across a cohort of approximately 100 subjects in various locations in the U.S. and Canada.

This trial is organized to meticulously evaluate the safety, tolerability, and ultimately the efficacy of AURN001, with a keen eye on the percentage of subjects who experience a significant improvement in vision—defined as a gain of 3 lines of vision at the 6-month mark.

The significance of this research cannot be overstated, given the profound impact of corneal endothelial dysfunction worldwide. This condition, which involves the deterioration and non-regeneration of corneal endothelial cells, leads to corneal edema and, if unchecked, permanent vision loss. The traditional route to remedy this has been corneal transplants, yet this method faces daunting challenges, not least the scarcity of donor corneas—a critical bottleneck given the demand ratio of one healthy donor cornea for every 70 diseased eyes.

Moreover, the recovery process post-corneal transplant is fraught with inconvenience, necessitating patients to remain supine for extended periods to ensure successful grafting—a factor that adds layers of complexity to the treatment.

Aurion Biotech, headquartered across Seattle, Cambridge, and Tokyo, has been at the forefront of pioneering cell therapy for corneal care, boasting regulatory approval in Japan for its first candidate. The company’s venture into initiating clinical trials in the U.S. and Canada echoes its commitment to harnessing the potential of regenerative therapies in reimagining vision care.

Recipient of the prestigious Prix Galien award for the best startup in biotech, Aurion Biotech continues to leverage support from leading investors in its quest to address one of the most pressing challenges in ophthalmology today. As the clinical trial progresses, the global community watches on with bated breath, hopeful for a breakthrough that could dramatically alter the landscape of corneal care and restore the gift of sight to millions.