When the cells of the innermost layer of the cornea die from disease or injury, they’re lost forever. Unlike some other parts of the eye, the corneal endothelium does not regenerate. Corneal transplants are a treatment option, and while this procedure is well established, the patients who need it run into the old economics problem of supply and demand. Because the need for corneal transplants far outstrips the supply of donated corneas, transplants are reserved for the most severe cases. Aurion Biotech aims to change the equation with a cell therapy that offers the potential to restore vision for many more patients.
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A single cornea donated for transplant can treat only a single patient. Aurion can take that same cornea and turn it into cell therapies for as many as 100 patients, said Greg Kunst, the company’s CEO. This experimental therapy expands the reach of treatment in other ways. Transplant surgeries are invasive procedures that must be done in hospitals. The Aurion cell therapy is administered as an injection into the eye, and could potentially be done on an outpatient basis. Kunst said that by extending the limited cornea supply, his company’s technology can bring a vision-restoring treatment to those in earlier stages of vision loss.
“It dramatically increases the number of number of treatments, the number of providers,” said Kunst, an ophthalmic industry veteran whose experience includes roles at Glaukos and Alcon. “We think we can really get into the early disease and put a dent into blindness.”
Aurion is preparing to evaluate its cell therapy in its first U.S. clinical trials and the Seattle-based startup has secured $120 million for that research. Deerfield Management led the round of financing announced Tuesday.
The cornea is one of the most commonly transplanted tissues, according to the Centers for Disease Control and Prevention. Many of those donations come from people who check the organ donor box when they renew their driver’s licenses. But this tissue from the eye is unlike other tissues. It can’t be frozen. It also has a very limited life span after the donor dies. Those factors, combined with high demand for corneal tissue, put tremendous pressure on the supply of donated corneas.
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Aurion’s technology comes from Japan. Shigeru Kinoshita, an ophthalmic surgeon and professor at Kyoto Prefecture University of Medicine, was researching ways to get corneal endothelial cells to regenerate in the lab, Kunst said. For various reasons, those efforts failed. But Kunst said that in trying to overcome those failures, Kinoshita developed the technology that is now Aurion’s cell therapy. There are structures in the eye that keep endothelial cells from regenerating and proliferating, Kunst said. Removing them enables them to proliferate while they’re still in the eye. But it doesn’t work in the lab. Kunst said Kinoshita solved that problem. The proprietary lab process starts with corneal endothelial cells and ends with many more corneal endothelial cells that are functional.
Unlike other cell therapies, such as CAR T treatments made by engineering a patient’s own immune cells, Aurion’s manufacturing process is scalable, Kunst said. The corneal endothelial cells are kept in a solution, which is injected into the eye. Those cells pose little risk of rejection due to the eye’s immuno-privileged status, meaning that it’s one of the parts of the body less susceptible 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 transplant. Kunst said CorneaGen’s strategic vision is to address corneal blindness around the world, but the company realized that it couldn’t achieve that goal solely by providing tissue. It formed Aurion to develop the corneal endothelial cell therapy technology. Aurion operated as a CorneaGen subsidiary until it became a standalone company supported by the financing that was announced Tuesday. The company is developing its cell therapy as a treatment for corneal edema secondary to endothelial dysfunction. The disease is essentially a swelling of the cornea resulting from endothelial dysfunction. According to Aurion, the disorder affects about 16 million people in the U.S., Europe, and Japan.
Aurion already has some clinical data under its belt. The cell therapy was first tested in Japan, where Kunst said open-label studies established both safety and efficacy. Shortly after CorneaGen acquired the technology, Aurion began a new set of studies in El Salvador to ensure that the company could replicate the results. In both sets of studies, vision improvement was reported within weeks. Kunst said that these patients who had significant vision loss experienced vision recovery better than what they had before treatment. Data for the first 11 patients treated in Japan were published in the New England Journal of Medicine in 2018. At 24 weeks after the injection, improvements were reported in the density of corneal endothelial cells, corneal thickness, and visual acuity. The effects appear to be durable.
“Those patients are now out to about seven years, they’re all doing very well,” Kunst said.
Aurion is readying an application seeking marketing approval for the cell therapy in Japan based on clinical data from about 100 patients. At the same time, the company is also laying the groundwork for a U.S. clinical trial. Kunst said that the studies from Japan and El Salvador are supportive, but he expects the FDA will still require the company to take its experimental treatment through Phases 1 through 3. He added that the goal is to file an investigational new drug application later this year. Longer term, the company is looking at other eye disorders. Kunst said that the technology can be used to address other forms of blindness, such as glaucoma and certain retinal diseases, that are also caused by the death of non-regenerating cells. Kunst said that after taking the cell therapy through the regulatory process in corneal endothelial dysfunction, Aurion will evaluate where else it can apply its technology.
The latest Aurion financing included participation from earlier investors Petrichor Healthcare Capital Management, Flying L Partners, Falcon Vision, an ophthalmology-focused investment platform supported by KKR, and Visionary Ventures, a leading ophthalmology-focused venture fund creating value with better insight. Eye care giant Alcon also invested.
Photo by Flickr user Rakesh Rocky via a Creative Commons license
