Healios KK: Joint research with the Division of Regenerative Medicine, the Institute of Medical Sciences for the development of a method for the mass production of liver buds UDC

April 1st, 2022

Company name: Representative:


Hardy TS Kagimoto, Chairman and CEO

(EST parent code: 4593)

Joint research with the Division of Regenerative Medicine, Institute of Medical Sciences for the development of a mass production method of liver buds UDC

HEALIOS KK (“Healios”) is currently developing a regenerative medicine treatment whereby liver organ sprouts created from iPS cells are injected into the liver and grown into functional liver tissue, with the aim of improving or restoring the function of a damaged liver (development code: HLCL041). This treatment could potentially replace the need for an organ transplant for some patients. Liver sprouts are created by co-cultivating liver progenitor cells, which can differentiate into hepatocytes; MSCs, which have the ability to develop into various types of connective tissue; and vascular endothelial cells, which form blood vessels. Healios has continued research and generated data on the functional evaluations and quality standards of these constituent cells and the liver buds created from them, and also continues the development of cultivation methods and mass manufacturing.

Besides, as announced on October 20and2020, Healios has created Universal Donor Cells (“UDC”)*, which are next-generation iPS cells created with gene-editing technology that exhibit a reduced risk of immune rejection regardless of a patient’s HLA type, and its proprietary clinical-grade UDC line. We are currently conducting research both internally and in joint collaborations with several institutions on new treatments for diseases for which there is no cure.

As part of these efforts, Healios is pleased to announce that it has entered into a joint research agreement with the Division of Regenerative Medicine (Prof. Hideki Taniguchi) of the Institute of Medical Sciences, University of Tokyo, to advance HLCL041 using UDCs. In this joint research, we plan to establish a new method to induce liver bud differentiation using CDUs and develop a highly efficient and scalable cell culture and mass manufacturing system.

For many diseases where the only effective treatment is an organ transplant, Healios believes that organ buds created from iPSCs, which have the potential to restore organ function, hold great promise as an alternative to organ transplants. organs and as a means of overcoming the continuing shortage of organ donors.

This agreement does not have a material impact on our consolidated financial results for the current fiscal year. We will promptly make an announcement on any matters requiring disclosure in the future.

■ Presentation of the collaboration partner

Name of Contributor: Division of Regenerative Medicine, The Institute of Medical Science Address:4-6-1 Shirokanedai Minato-ku, Tokyo, 108-8639, Japan

Representative: Professor Taniguchi Hideki


UDCs are iPS cells created using gene-editing technology that allows them to ward off and/or reduce the body’s immune rejection response. The production of Healios UDCs involves the deletion of certain HLA genes that cause a rejection response, the introduction of an immunosuppression gene to enhance immune evasion, and the addition of a suicide gene to serve as a safety mechanism , each in an allogeneic iPS cell. This next-generation technology platform enables the creation of regenerative medicine products with increased safety and lower risk of immune rejection, while preserving the inherent ability of iPS cells to continuously replicate and their pluripotency to differentiate into various other types. of cells.

About the Division of Regenerative Medicine, The Institute of Medical Science:

Regenerative medicine is a challenging scientific field that will convert pioneering knowledge of developmental biology and stem cell biology into clinical applications. For patients with end-stage organ failure, organ transplantation is the only effective treatment; however, the scarcity of transplantable organs hinders the application of this treatment for most patients. Recently, regenerative medicine with transplantable organs has attracted attention. Our laboratory is developing a new therapeutic strategy to replace organ transplantation. We have implemented novel organoid culture technologies to reconstruct human organs from stem cells, including human induced pluripotent stem cells (iPSC), and we will perform the transplantation of human liver primordia ( hepatic buds [LBs]) generated from iPSC for the treatment of liver disease. https://stemcell-imsut.org/laboratory/?id=fr#labo1

About Healios:

Healios is Japan’s leading clinical-stage biotechnology company harnessing the potential of stem cells for regenerative medicine. It aims to bring new therapies to patients suffering from diseases without effective treatment options. Healios is a pioneer in regenerative drug development in Japan, where it established a proprietary line of genetically engineered “universal donor” (iPSC) induced pluripotent stem cells to develop next-generation regenerative treatments in immuno-oncology, ophthalmology , liver disease, and other areas with serious unmet medical needs. Healios’ lead candidate for iPSC-derived cell therapy, HLCN061, is a next-generation NK cell treatment for solid tumors that has been functionally enhanced through gene editing. Its near-term pipeline includes the somatic stem cell product HLCM051, which is currently being evaluated in Japan in phase 2/3 and phase 2 trials in ischemic stroke and acute respiratory distress syndrome (ARDS), respectively. Healios was established in 2011 and has been listed on the Tokyo Stock Exchange since 2015 (TSE Mothers: 4593). https://www.healios.co.jp/en .


Corporate Communications Department, HEALIOS KK

E-mail:[email protected]

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