Otsu/Shiga, Japan – February 10, 2014 – Takara Bio Inc., announced today that an application for a Phase I clinical trial on MAGE-A4 antigen-specific TCR gene therapy was submitted to the PMDA (Pharmaceuticals and Medical Devices Agency) on February 5th, 2014 by Dr. Hiroshi SHIKU, professor of Mie University Hospital, who has been developing TCR (T-cell receptor) gene therapy in collaboration with Takara Bio. The clinical trial is scheduled to start after the PMDA’s 30-day review period.


The main mechanism of action for TCR gene therapy is via gene-transduced lymphocytes, which acquire the capability to specifically recognize and attack cancer cells and eliminate them. TCR genes that are capable of recognizing cancer antigens are transduced into the patient’s own lymphocytes, which are then re-infused into the patient. The RetroNectin method that Takara Bio established and the next-generation retroviral vectors that Takara Bio and Mie University developed (patent issued) play an essential role in transducing the TCR genes into the lymphocytes. This clinical trial will be the first ever genetic immunotherapy for cancer in Japan. For this clinical trial, Takara Bio will produce an investigational drug named TBI-1201 which is composed of MAGE-A4 TCR modified T lymphocytes.


The Japanese government is instituting a wide range of measures to support development in the regenerative medicine and cell therapy fields. For example, the Regenerative Medicine Promotion Act was put into force on May 10, 2013. In addition, revisions to the Pharmaceutical Affairs Act and the Act on Ensuring the Safety of Regenerative Medicine were approved on November 20, 2013. According to the Revision to the Pharmaceutical Affairs Act, an accelerated approval system for new regenerative medicines will be introduced in 2014.


We aim to commercialize the MAGE-A4 TCR gene therapy by fiscal 2022, but will continue to proceed and try to accelerate the TCR gene therapy project by shortening the development period before commercialization with careful consideration of this new accelerated approval system.

[ Outline of planned clinical trial ]


Multi-center, Investigator Initiated Phase I Study for Patients with Solid Tumors Using Lymphodepleting Conditioning Followed by Infusion of MAGE-A4 Specific TCR Gene Transduced T Lymphocyte


HLA-A*24:02 positive patients with solid tumors which are 
1) unresectable, refractory to standard therapy (chemotherapy, radiotherapy, etc), metastatic or recurrent, and 
2) MAGE-A4- expressing

Primary outcomes To evaluate the safety and in vivo kinetics of TCR gene transduced T lymphocytes
Secondary outcomes

To evaluate clinical effect (clinical tumor regression)

Number of subjects 12 subjects
Trial period 2 years (from March 2014 to March 2016)


[ About Takara Bio Inc. ]

Takara Bio Inc. is an innovative biotechnology company based in Shiga, Japan. As a world leader in biotechnology research and development, Takara Bio was the first company to market PCR technology in Japan and is also the developer of the RetroNectin® reagent, which is a world-standard in gene therapy protocols. In addition to providing research reagents and equipment to the life science research market, Takara Bio has active research and product development activities in the fields of gene and cell-based therapy, and agricultural biotechnology including functional food and mushroom production businesses; and is committed preventing disease and improving the quality of life for all people through the use of biotechnology. Through strategic alliances with other industry leaders, the Company aims to extend its reach around the world. More information is available at http://www.takara-bio.com.