TECHNOLOGY

Currently, the only treatment for corneal endothelial decompensation is corneal transplantation, in which the cornea is donated as an organ from a deceased person. Donor corneas are in such a short supply around the world that only one in 70 patients worldwide who need a corneal transplant is able to receive one. The number of corneal transplantations needed for the treatment of corneal endothelial decompensation has been increasing for two decades.

Fuchs endothelial corneal dystrophy affects 4% of persons over the age of 40. The early stages of the disease have no subjective symptoms, but as the disease progresses, patients experience glare and vision loss. As the disease progresses further, eye pain and tearing may occur, and the cornea may become cloudy.

This disease is often diagnosed at a relatively early stage when vision is good, but because no cure is available, corneal transplants are performed only after vision has decreased severely. Fuchs endothelial corneal dystrophy is the leading cause of corneal transplantation worldwide, and the development of alternative treatments for corneal transplantation is highly anticipated.

Professors Noriko Koizumi and Naoki Okumura at Doshisha University, both co-founders of ActualEyes, Inc., have been working to elucidate the mechanism of Fuchs endothelial corneal dystrophy and to develop pharmaceutical therapies. Corneal endothelial cells were obtained from the patients with Fuchs endothelial corneal dystrophy during corneal transplantation. The corneal endothelial cells were first cultured as a disease model of Fuchs endothelial corneal dystrophy. Using this cell model, Drs. Koizumi and Okumura investigated potential drugs for the treatment of Fuchs endothelial corneal dystrophy. Their drug screening identified sirolimus as a drug that dramatically stops the death of corneal endothelial cells. ActualEyes, Inc. has obtained an exclusive license from Doshisha University to use sirolimus as a treatment for Fuchs endothelial corneal dystrophy and is developing it as a therapeutic agent.

ActualEyes, Inc. is also developing AE002 (development code: AE002), which is expected to be an effective treatment for Fuchs endothelial corneal dystrophy through a different mechanism of action. Once these eye drops are launched, the future need for corneal transplants due to Fuchs endothelial corneal dystrophy may be drastically reduced. We expect that these drops will change the treatment of corneal endothelial disease.

Noriko Koizumi and Naoki Okumura, founding members of ActualEyes, Inc., have devoted their efforts to developing regenerative therapy for corneal endothelial diseases. They have succeeded in the extremely difficult task of culturing corneal endothelial cells for clinical use. Furthermore, they discovered that injection of cultured corneal endothelial cells, together with a drug called ROCK inhibitor, into the eye can be an alternative to conventional corneal transplantation. These technologies were transferred to the Kyoto Prefectural University of Medicine, and the world's first-in-human study of cell injection therapy was initiated in December 2013. Since then, more than 50 patients have been successfully treated, and the efficacy and safety of this treatment have been confirmed. The fact that patients who needed corneal transplants have regained their sight through regenerative medicine has been a remarkable achievement. This regenerative medicine involves injecting cells into the eye using an injection needle, and the treatment is less burdensome to the patient than a corneal transplant, as it is completed in less than 10 minutes. Furthermore, the resulting reconstructed cornea is nearly anatomically normal, so the restoration of vision is considered ideal.

The success of the clinical research conducted at the Kyoto Prefectural University of Medicine Hospital has increased the expectations for successful regenerative medicine for corneal endothelial disorders worldwide. ActualEyes, Inc. has developed a formulation of cultured cells that can be frozen and then simply thawed and injected when needed. Once commercialized, the product will be delivered worldwide for the treatment of patients on an as-needed basis. ActualEyes, Inc. is now preparing clinical trials in Japan and China (in collaboration with Arctic Vision, Inc.).