Orizuru Therapeutics Launches Operations as Research and Development-driven Company for Products Including induced Pluripotent Stem Cell (iPSC) Regenerative Medicine

Orizuru Therapeutics Inc. announced the launch of its operations on June 1, 2021. OZTx is a research and development-driven company focusing on the development of iPSC―based regenerative medicine and the application of iPSC technology, and is based at Shonan Health Innovation Park.

In 2015, the Center for iPS Cell Research and Application ("CiRA") at Kyoto University and Takeda Pharmaceutical Company Limited ("Takeda Pharmaceutical") launched a joint research program, called T-CiRA, to accelerate research for the practical application of iPSC technology in drug development and regenerative medicine. The 10-year T-CiRA program has been led by Professor Shinya Yamanaka, Director of CiRA and Representative Director of the CiRA Foundation, with total funding of 20 billion yen in R&D from Takeda Pharmaceutical and human resources and facility-related support provided by Shonan iPark. Following the recent validation of pre-clinical efficacy of two cell therapy projects, CiRA and Takeda Pharmaceutical agreed to transfer the research assets from the projects to OZTx.

OZTx has two primary business areas of focus. First is the working toward the clinical application of the two cell therapy projects for which pre-clinical efficacy was validated in the T-CiRA program: the iPSC-derived cardiomyocyte project led by Project Leader Yoshinori Yoshida (Associate Professor at CiRA) and the iPSC-derived pancreatic islet cell project led by Project Leader Taro Toyoda (Junior Associate Professor at CiRA). In addition, OZTx has entered into a patent transfer and research asset license agreement with Kyoto University and Takeda Pharmaceutical, which own the intellectual property of iPSC-derived cardiomyocytes and iPSC-derived pancreatic islet cells, to research, develop, and provide regenerative medicine products for both projects.

The second area of focus encourages the application of iPSC technology through the sharing of distinguished technologies in iPSC differentiation, culture, and purification, to rigorously support drug discovery research and develop the infrastructure for research in regenerative medicine. OZTx seeks to share its iPSC-related business activities broadly with communities and society, and also contribute to the delivery of iPSC-based regenerative medicine to patients and society in appropriate manners.

"We are very pleased that the innovative research outcomes from the T-CiRA collaboration where academic scientists and Takeda researchers work as one team are now transferred to OZTx toward their quick implementation," says Professor Yamanaka, Head of the T-CiRA program. To ensure continuity of research and development from T-CiRA, Professor Yamanaka has also been invited to chair the Scientific Advisory Board at OZTx. Dr. Yasushi Kajii, who oversees the T-CiRA collaboration on the Takeda side as Head of T-CiRA Discovery, also demonstrates his enthusiasm to collaborate with OZTx for implementation of the innovative iPSC technology, stating that, "I count on the establishment of OZTx so that pathways to bring outcomes from the T-CiRA collaboration to the bedside of patients suffering from intractable diseases can be strengthened."

OZTx is comprised of approximately 60 employees who have in-depth expertise in the latest iPSC technology as well as extensive experience in pharmaceutical R&D. Each of the 60 employees brings a strong commitment to the fore, demonstrating their passion and a sense of urgency to deliver iPSC technology to patients and society at large. The company is led by President, Representative Director, and Chief Executive Officer Kenji Nonaka, M.D., Ph.D. OZTx has raised a total of 6 billion yen through private placement with numerous partners listed below that stand in solidarity with OZTx's strong mission to put this Japan-born iPSC technology to practical use in regenerative medicine:

Seed round: Kyoto University Innovation Capital Co., Ltd. ("KYOTO-iCAP"), Takeda Pharmaceutical Company Limited, SMBC Venture Capital Co., Ltd. 

Series A round: MUFG Bank, Ltd., SMBC Venture Capital Co., Ltd, MEDIPAL HOLDINGS CORPORATION, Sumitomo Mitsui Finance and Leasing Company Co. Ltd., Sumitomo Mitsui Trust Bank, Limited, Nippon Venture Capital Co., Ltd.

OZTx is committed to developing high quality products and services using the finest processes and seeks to contribute to society and patients by delivering regenerative medicine using iPSCs, which was first developed in Japan.

About iPSC-derived cardiomyocytes (iCM)

iCM was developed through a project led by Project Leader Yoshinori Yoshida, Associate Professor at CiRA. The cell is currently being refined for future clinical use, based on the cardiomyocyte differentiation method researched at CiRA and the myocardial purification method using small-molecule compounds developed in the T-CiRA program. iCM is a highly purified cardiomyocyte that demonstrated a high level of engraftment and safety in pre-clinical studies. When transplanted to the patient's heart, it is expected to supplement cardiomyocytes that are lost as diseases progress and accelerate the regeneration of cardiac muscles. iCM can potentially recover cardiac function in severe heart failure, which has been difficult to treat, as well as improve patients' quality of life and life prognosis.

About iPSC-derived pancreatic islet cells (iPIC)

The cell was developed after five years of research in the T-CiRA program, based on the method of inducing pancreatic cell differentiation discovered by Taro Toyoda, Junior Associate Professor at CiRA. iPIC are highly purified pancreatic endocrine cell aggregates that contain pancreatic beta cell-like cells, to the same extent as pancreatic islets in vivo. Once transplanted, it forms within the body pancreatic islet structures that include glucagon-positive cells and demonstrates physiological insulin secretion functions that respond to blood glucose levels. Thus, iPIC can potentially contribute to controlling the condition of patients with diabetes.

About Orizuru Therapeutics Inc. (OZTx)

OZTx, founded in April 2021 by KYOTO-iCAP, is steadfast in its dedication to bringing hope for better health through the infinite power of science. To deliver cell therapies to patients, the company promotes the wide use of cell therapy products and innovative iPSC-related technology through the following activities:

Development of regenerative medical products through cell transplantation

Support for drug discovery research and development of regenerative medicine research infrastructure using iPSC-related technology

About the Takeda-CiRA Joint Program for iPS Cell Applications (T-CiRA)

The agreement to form T-CiRA was reached in 2015 as a ten-year joint research program between CiRA and Takeda Pharmaceutical. Research activities commenced in 2016. With Takeda Pharmaceutical's funding of over 20 billion yen and the leadership of Professor Yamanaka, and researchers at CiRA, Tokyo Medical and Dental University, and RIKEN, multiple projects are being driven, including advanced research on the clinical application of iPSC technology for cancer, heart failure, diabetes, neuro-psychiatric disorders, and intractable muscle diseases.

About the Center for iPS Cell Research and Application (CiRA), Kyoto University

CiRA was founded on April 1, 2010, at Kyoto University, as the world's first central research institute specializing in iPSCs. The institute is led by Professor Yamanaka, who serves as Director. With the aim of the medial application of iPSC, about 30 research groups engage in a range of research projects, including basic research, application research and bioethics research.

About Kyoto University Innovation Capital Limited (KYOTO-iCAP)

KYOTO-iCAP, as a wholly-owned subsidiary of Kyoto University, provides investment and other business support to companies that utilize the results of research generated by Kyoto University and other national universities. KYOTO-iCAP currently manages the Innovation Kyoto 2016 Investment Limited Partnership ("KYOTO-iCAP No.1 Fund"), established in January 2016, with a total value of 16 billion yen, and the KYOTO-iCAP No.2 Fund, established in January 2021, with a total value of 18 billion yen. The KYOTO-iCAP No.1 Fund has a maturity of up to 20 years and the KYOTO-iCAP No.2 Fund has a maturity of up to 17 years, enabling the provision of long-term support for the practical application of the results of Kyoto University's strong fundamental research. In addition, the KYOTO-iCAP No.2 Fund will invest part of its funds in ventures originating from national universities other than Kyoto University. 

About Takeda Pharmaceutical Company Limited (Takeda Pharmaceutical)

Takeda Pharmaceutical is a global, R&D-driven pharmaceutical company that is committed to enhancing its R&D pipeline by strengthening its R&D activities, life cycle management, and partnerships and to fulfill its mission of contributing to better health for people and a brighter future for healthcare through high-quality pharmaceutical products.

About Shonan Health Innovation Park (Shonan iPark)

Shonan iPark is a pharmaceutical company-driven science park founded by Takeda Pharmaceutical in April 2018. Housing organizations across the public and private sectors and academia that are diverse in business type and scale, the science park aims to accelerate innovation in healthcare. More than 120 companies and organizations including pharmaceutical companies as well as next-generation medicine, AI, venture capital, and government-related bodies, and more than 2,100 workers (as of August 2021) together form an ecosystem at Shonan iPark.