First iPSC-Derived CAR T-Cell Therapy Created by Kyoto University CiRA and Takeda Collaboration Enters Process Development Toward Clinical Testing

− iCART Program Advances the Potential for a Highly Scalable, Lower Cost, Off-the-Shelf
CAR T-Cell Therapy to Treat Cancer

Kyoto and Osaka, Japan, July 16,2019 –  The Center for iPS Cell Research and Application (CiRA) at Kyoto University and Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced that a novel induced pluripotent stem (iPS) cell-derived chimeric antigen receptor (CAR) T-cell therapy (iCART) has been transferred from their T-CiRA research collaboration to Takeda as the program begins process development toward clinical testing. Under the terms of the T-CiRA agreement, Takeda has the global rights to develop and commercialize the iCART product and CiRA will receive development and approval milestones. The teams will continue to collaborate as they prepare the iCART program for a clinical trial in 2021.

Autologous CAR T-cell therapy is a type of immunotherapy that uses a patient’s own genetically modified T-cells to find and kill cancer cells. Because first-generation autologous CAR-T therapies are created from the blood of each individual patient, it is a slow and expensive process.

Developed by CiRA’s Shin Kaneko, M.D., Ph.D., who will remain involved as a Takeda advisor, the iCART program uses a clonal master iPS cell bank to create off-the-shelf CAR-T therapies that can be tailored to each patient on demand. The goal is to develop a process that is highly scalable – capable of creating very large quantities of homogenous doses from a single master cell bank – and less costly than first generation CAR-Ts. In vivo pre-clinical studies demonstrated robust antitumor efficacy with a CD19-targeted iCART.

Advancing the Next Generation of CAR T-Cell Therapy
“The iCART program demonstrates the value of our T-CiRA collaboration – applying iPSC technology to develop new approaches to drug discovery and creating a bridge to transfer promising programs to Takeda to accelerate them toward clinical development and therapeutic use,” said Shinya Yamanaka, M.D., Ph.D., director of CiRA and T-CiRA, who received a Nobel Prize in 2012 for his groundbreaking iPSC research.

T-CiRA was established in 2015 as a 10-year joint research program between CiRA and Takeda. Under the direction of Dr. Yamanaka Takeda is providing 20 billion yen in collaborative funding and is jointly running multiple projects led by researchers invited from CiRA, and other research institutes. As part of the ongoing collaboration, T-CiRA conducts cutting-edge research in the clinical application of iPS cells in areas including immuno-oncology, heart failure, diabetes mellitus, neuro-psychiatric disorders, and intractable muscle diseases at Takeda’s Shonan Health Innovation Park.

“Today, we celebrate with Dr. Yamanaka, Dr. Kaneko, and our CiRA colleagues as the first T-CiRA cell therapy program is transferred to Takeda for clinical development,” said Takeda President of Research and Development Andy Plump, M.D., Ph.D. “The iCART program demonstrates our commitment to bringing transformational, next-generation CAR-T therapies to patients by applying our translational cell therapy engine. Of Takeda’s 12 CAR-T programs in development, iCART is one of five with planned first-in-human studies by 2021.”

Led by Stefan Wildt, Ph.D., Takeda’s translational cell therapy engine is designed to rapidly translate cell therapy discovery into the clinic and accelerate the development of new and differentiated therapies. The engine team provides bioengineering, chemistry, manufacturing and control (CMC), analytical, clinical and translational expertise in a single footprint, overcoming many of the process development and manufacturing challenges companies typically experience.

About the Center for iPS Cell Research and Application (CiRA) at Kyoto University 
CiRA was established on April 1, 2010 to serve as the world’s first core institute dedicated to pioneering iPS cell research. With Prof. Shinya Yamanaka serving as Director, CiRA’s 30 research groups are engaged in fundamental science of cell reprogramming and human development, clinical applications, and bioethics, with the overall goal of using iPS cells to realize regenerative medicine and drug discovery. Additional information about CiRA is available through its website at http://www.cira.kyoto-u.ac.jp/e/ 

About Takeda Pharmaceutical Company Limited
Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology (GI), Rare Diseases and Neuroscience. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions.
For more information, visit https://www.takeda.com 

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