When cells in the innermost layer of the cornea die from disease or injury, they disappear forever. Unlike the rest of the eye, the corneal endothelium does not regenerate. Corneal transplantation is a treatment option, and while the procedure is well established, patients who need it run into the old economics of supply and demand. Because the demand for corneal transplants far exceeds the supply of donated corneas, transplants are reserved for the most severe cases. Aurion Biotech aims to change this balance with a cell therapy that offers more patients the potential to restore vision.
A single cornea donated for transplantation can only treat a single patient. Aurion can convert the same corneas into cell therapy for up to 100 patients, CEO Greg Kunst said. This experimental therapy expands the range of treatments in other ways. Transplant surgery is an invasive procedure that must be performed in a hospital. Aurion Cell Therapy is administered as an eye injection and may be performed on an outpatient basis. By expanding the limited supply of corneas, Kunst said his company’s technology could bring vision-restoring treatments to people in the early stages of vision loss.
“It has dramatically increased the number of treatments and providers,” said Kunst, an ophthalmology industry veteran who has held positions at Glaukos and Alcon. “We think we can really get into early disease and reduce blindness.”
Aurion is preparing to evaluate its cell therapy in its first U.S. clinical trial, and the Seattle-based startup has Get $120 million for that study. Deerfield Management led the funding round announced Tuesday.
The cornea is one of the most common transplanted tissues, according to Centers for Disease Control and Prevention. Many of these donations came from people who checked organ donation boxes when renewing their driver’s license. But this tissue from the eye is different from other tissues. It cannot be frozen. It also has a very limited lifespan after death. These factors, combined with the high demand for corneal tissue, have put enormous pressure on the supply of donated corneas.
Aurion’s technology comes from Japan. Shigeru Kinoshita, an ophthalmologist and professor at Kyoto Prefectural University of Medicine, is studying how to regenerate corneal endothelial cells in the lab, Kunst said. For various reasons, these efforts have failed. But in trying to overcome those failures, Kinoshita developed what is now Aurion’s cell therapy, Kunst said. There are structures in the eye that prevent endothelial cells from regenerating and proliferating, Kunst said. Removing them allows them to proliferate while they are still in the eye. But it doesn’t work in the lab. Kunst said Kinoshita solved the problem. Proprietary laboratory processes start with corneal endothelial cells and end with more functional corneal endothelial cells.
Unlike other cell therapies, such as CAR T therapies made by engineering a patient’s own immune cells, Aurion’s manufacturing process is scalable, Kunst said. Corneal endothelial cells are kept in solution and injected into the eye. Due to the immune-privileged state of the eye, these cells are at little risk of rejection, meaning it is one of the less susceptible parts of the body to an immune response.
Kinoshita’s cell therapy technology was acquired in 2020 by CorneaGen, a Seattle company that processes and supplies corneal tissue for transplantation. CorneaGen’s strategic vision is to solve the global problem of corneal blindness, but the company realized it couldn’t achieve this by providing tissue alone, Kunst said. It founded Aurion to develop corneal endothelial cell therapy technology. Aurion operated as a subsidiary of CorneaGen until it became an independent company, backed by financing announced Tuesday. The company is developing its cell therapy to treat corneal edema secondary to endothelial dysfunction. The disease is essentially corneal swelling caused by endothelial dysfunction. According to Aurion, the disease affects about 16 million people in the United States, Europe and Japan.
Aurion already has some clinical data. The cell therapy was first tested in Japan, and Kunst said the open-label studies established safety and efficacy. Shortly after CorneaGen acquired the technology, Aurion began a new series of studies in El Salvador to ensure the company could replicate the results. In both groups of studies, improved vision was reported over several weeks. Those with severe vision loss recovered better than they had before treatment, Kunst said.The data for the first 11 patients treated in Japan are post In the 2018 New England Journal of Medicine. Improvements in corneal endothelial cell density, corneal thickness, and visual acuity were reported 24 weeks after injection. The effect seems to be long lasting.
“These patients are now living for about seven years, and they’re all doing well,” Kunst said.
Aurion is preparing an application seeking marketing authorization for the cell therapy in Japan based on clinical data from approximately 100 patients. Meanwhile, the company is preparing for clinical trials in the United States. Kunst said studies from Japan and El Salvador are supportive, but he expects the FDA will still require the company to move through Phases 1 to 3 for the experimental treatment. The goal is to submit an investigational new drug application later this year, he added. In the longer term, the company is working on other eye diseases. The technique could be used to address other forms of blindness caused by non-regenerative cell death, such as glaucoma and certain retinal diseases, Kunst said. Aurion will assess where it can apply its technology following cell therapy through the regulatory process of endothelial dysfunction in the cornea, Kunst said.
The latest Aurion financing includes participation from early-stage investors Petrichor Healthcare Capital Management, Flying L Partners, KKR-backed ophthalmology-focused investment platform Falcon Vision, and leading ophthalmology-focused venture capital fund Visionary Ventures, which is backed by Better Insight creates value. Eye-care giant Alcon also invested.
Photos by Flickr users Rakesh Rocky through Creative Commons license
