TOKYO--(BUSINESS WIRE)--Cellusion Inc. (CEO: Shin Hatou, M.D. Ph.D., “Cellusion”), a deep tech start-up developing a novel cell therapy, announced that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation (“ODD”) to our regenerative medicine product “iPS cell-derived corneal endothelial cell substitute” (“CLS001”) for the treatment of bullous keratopathy..

东京——（商业新闻短讯）——Cellusion Inc.（首席执行官：Shin Hatou，医学博士，“Cellusion”），一家开发新型细胞疗法的深度科技初创公司，宣布美国食品和药物管理局（FDA）已授予我们的再生医学产品“iPS细胞衍生的角膜内皮细胞替代品”（“CLS001”）孤儿药名称（“ODD”），用于治疗大疱性角膜病。。

FDA grants ODD if the drug meets specific criteria, the treatment, diagnosis, or prevention of rare diseases that affect less than 200,000 people in the United States. The ODD qualifies sponsors for incentives, including tax credits for qualified clinical (in humans) testing, waiver of the prescription drug user fee and potential 7 years of market exclusivity after FDA approval..

如果ODD药物符合特定标准、治疗、诊断或预防影响美国不到20万人的罕见疾病，FDA将授予ODD。ODD使赞助商有资格获得奖励，包括合格临床（人体）测试的税收抵免，处方药使用费的豁免以及FDA批准后潜在的7年市场排他性。。

Cellusion has developed CLS001 for bullous keratopathy, which accounts for more than half of all corneal transplants and is currently preparing for corporate clinical trials in Japan and global clinical trials. With this designation, we will further accelerate the global development of CLS001 and aim to contribute to patients around the world who are waiting for the treatment..

Cellusion已开发出用于大疱性角膜病的CLS001，占所有角膜移植的一半以上，目前正在为日本的公司临床试验和全球临床试验做准备。通过这一指定，我们将进一步加速CLS001的全球发展，并旨在为世界各地等待治疗的患者做出贡献。。

The ODD information for CLS001 is as follows:

CLS001的奇数信息如下：

FDA (Search Orphan Drug Designations and Approvals)

FDA（搜索孤儿药名称和批准）

https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=975623

https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedindex.cfm?cfgridkey=975623

[About CLS001]

[关于CLS001]

CLS001 is alternative cell to corneal endothelial cells produced by efficiently differentiating iPS cells, which can be differentiated into various types of cells, based on Cellusion’s proprietary technique. Cellusion is promoting the development of CLS001 as a therapeutic regenerative medicine product for bullous keratopathy, which accounts for more than half of the corneal transplant cases, by combining “CECSi Cells* made from iPS cells with excellent proliferative properties” and “a simple injection cell delivery procedure without needs of human expertise.” Cellusion is also advancing preparations for company-sponsored clinical trials in Japan, as well as preparations for global clinical trials..

CLS001是通过有效分化iPS细胞产生的角膜内皮细胞的替代细胞，基于Cellusion的专有技术，iPS细胞可以分化成各种类型的细胞。Cellusion通过将“由具有优异增殖特性的iPS细胞制成的CECSi细胞”和“一种简单的注射细胞递送程序”相结合，正在促进CLS001作为大疱性角膜病的治疗性再生医学产品的发展，该产品占角膜移植病例的一半以上。“Cellusion还正在推进日本公司赞助的临床试验的准备工作，以及全球临床试验的准备工作。。

*CECSi Cells: Corneal Endothelial Cell Substitute from iPS Cells

*CECSi细胞：iPS细胞的角膜内皮细胞替代物

[About Bullous Keratopathy]

[关于大疱性角膜病]

Bullous keratopathy is a rare disease that occurs when corneal endothelial cells, which control the amount of fluid at the inner-most part of the cornea, decrease due to eye surgery of cataract or genetic factors, resulting in problems with their function, whereby the cornea becomes swollen and causes visual impairment due to opacity.

大疱性角膜病是一种罕见的疾病，当控制角膜最内层液体量的角膜内皮细胞由于白内障或遗传因素的眼科手术而减少时，会导致其功能出现问题，从而导致角膜肿胀并由于不透明而导致视力障碍。

This disease is progressive and may lead to blindness if left untreated. Today, this disease is mainly treated by transplanting normally functioning corneal endothelial cells via corneal transplant. Nevertheless, as with other organ transplants, corneal transplants are also facing a serious problem of donor shortage.

这种疾病是进行性的，如果不及时治疗，可能会导致失明。今天，这种疾病主要通过角膜移植移植正常功能的角膜内皮细胞来治疗。然而，与其他器官移植一样，角膜移植也面临着供体短缺的严重问题。

In Japan alone, it is estimated that there are about 10,000 patients, and in the global survey conducted in 2013 it was reported that approximately 13 million patients are waiting for corneal transplants..

仅在日本，估计就有大约10000名患者，2013年进行的全球调查报告显示，大约1300万患者正在等待角膜移植。。

[About Cellusion]

[关于Cellusion]

Cellusion is a regenerative medicine start-up originating from the Department of Ophthalmology, Keio University School of Medicine, with “Regenerating Human Potential” as its mission and “More Freedom and More Smiles to the World” as its vision. Based on patented technology such as its proprietary technique of efficiently producing corneal endothelial substitute cell from iPS cells, Cellusion is promoting R&D aimed at meeting medical needs from the perspective of “patient centricity” by leveraging cutting-edge regenerative medicine technology, including the lead program CLS001 that aims to meet the global corneal transplant demands..

Cellusion是一家再生医学初创公司，起源于庆应义塾大学医学院眼科，其使命是“再生人类潜力”，愿景是“给世界带来更多自由和微笑”。Cellusion基于专利技术，例如其从iPS细胞高效生产角膜内皮替代细胞的专有技术，通过利用尖端的再生医学技术，包括旨在满足全球角膜移植需求的领先项目CLS001，从“以患者为中心”的角度促进旨在满足医疗需求的研发。。

Company: Cellusion Inc.

公司：Cellusion Inc。

CEO: Shin Hatou, M.D., Ph.D.

首席执行官：Shin Hatou，医学博士、博士。

Headquarters: 8-6 Nihonbashi-Kobuna, Chuo, Tokyo 103-0024, JAPAN

总部：日本东京中央区Nihonbashi Kobuna 8-6 103-0024

Founded: January 2015

成立日期：2015年1月

URL: https://cellusion.jp/en/

网址：https://cellusion.jp/en/