Cellusion Announces First Patient Transplanted iPSC-Derived Corneal Endothelial Cell Substitute (CLS001)


– Short-term safety was confirmed.
– Efficacy including visual acuity and corneal thickness is improving.

Cellusion Inc. (CEO: Shin Hatou, M.D. Ph.D., “Cellusion”) announced that our research collaborator, Dr. Shigeto Shimmura, Professors of Fujita Health University and Keio University presented at the 22nd Japanese Society for Regenerative Medicine Annual Conference (Kyoto, Japan, March 23-25, 2023) that iPS cell-derived corneal endothelial cell substitute (“CLS001”) was transplanted to the first patient (First In Human*, “FIH Study”).

The details of presentation information are as follows;
Title: iPS-derived Cornea Endothelial Substitutes for Corneal Regeneration
Lecture Number: SY-02-2
Date & Time: Thursday, March 23, 2023, 8:30-10:30 a.m.

This FIH study (Study title: Exploratory Clinical Study to Examine Safety and Efficacy of iPS Cell-Derived Corneal Endothelial Cell Substitutes for Bullous Keratopathy, jRCT study protocol number: jRCTa031210199) was conducted at the Department of Ophthalmology, Keio University Hospital to evaluate the safety and efficacy of CLS001 transplantation in patients with regrafted cases of bullous keratopathy.

In the presentation, Prof. Shimmura reported that the safety was confirmed since no adverse event has been observed for 3 months after the CLS001 transplantation at the review of the Independent Data Monitoring Committee held in January 2023. In addition, in terms of the efficacy, he also reported that the visual acuity, central corneal thickness, and minimal corneal thickness has tended to be improved so far.

Cellusion is challenging to solve the global cornea shortage issue by developing CLS001. With the progress of the FIH study, we will accelerate the preparation of the Cellusion sponsored clinical trial.

*First-in-human (FIH) studies: Clinical studies or Phase I clinical trials in which a drug is administered to humans for the first time after its safety and efficacy have been confirmed in animal studies.

[About Bullous Keratopathy]
Bullous keratopathy is a disease that causes blistering of the cornea and visual impairment due to clouding caused by a decrease in corneal endothelial cells, which regulate the proper amount of water in the cornea, due to cataracts or other eye surgery or hereditary factors. The disease is progressive and can lead to blindness if left untreated. Currently, the only treatment available for this disease is a corneal transplant that replaces normally functioning corneal endothelial cells. However, like other organ transplants, corneal transplants are facing a serious shortage of donors. It is estimated that there are more than 10,000 patients in Japan, and a global survey conducted in 2013 reported that there are some 13 million people worldwide on the waiting list for corneal transplants.

[About CLS001]
In spite of the fact that more than 13 million waiting patients for potential blinding diseases such as Bullous Keratopathy, which can only be prevented by corneal transplantation, only about 180,000 corneal transplants are performed annually worldwide. The reason for such a large medical supply-demand gap generated is because current treatments require donor corneas, skilled corneal transplant ophthalmologists, and Eye Banks.
Cellusion is developing CLS001, CECSi cells for Corneal Endothelial regeneration to cure Bullous Keratopathy which is applied to more than half of all cases of corneal transplantation. CLS001 is expected to replace the current supply limitations by combining “CECSi Cells made from iPS cells with excellent proliferative properties” and “a simple injection cell delivery procedure without needs of human expertise.”

[About Cellusion]
Cellusion is a regenerative medicine startup born from the Department of Ophthalmology, Keio University School of Medicine in 2015. Cellusion aims to contribute to the improvement of health and well-being around the world by solving unmet medical needs in medicine with its cutting-edge cell therapy technologies, including a unique differentiation induction method from iPS cells to CECSi endothelial cells. Cellusion’s mission is to give the worldwide community a clear vision of the future with the cellular technology breakthrough.

Company: Cellusion Inc.
CEO: Shin Hatou, M.D., Ph.D.
Headquarters: 8-6 Nihonbashi-Kobuna, Chuo, Tokyo 103-0024, JAPAN
Founded: January 2015

For further information: please ask us through the contact form on our website.

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