Gene Therapy Business

Detail Information

The business focuses on commercilization of gene therapies for diseases such as cancer. These therapies represent the oncolytic virus therapy utilizing Canerpaturev (C-REV) and the engineered T cell therapy utilizing the original Takara Bio technologies such as the Retronectin® method for a high efficiency gene transduction; the RetroNectin® expansion-culture system for a high efficient expansion for lymphocytes; as well as siTCRTM.

Oncolytic Virus

HF10 project: Canerpaturev (Abbreviated to C-REV)

Canerpaturev (C-REV) is an attenuated strain of the herpes simplex virus 1 (HSV-1) that exhibits antitumor activity when inserted into a cancerous region. The administration of Canerpaturev (C-REV) also strengthens immunity to cancer cells, giving it promise as a means to prevent tumors from forming, even in tumor regions where Canerpaturev (C-REV) was not administered. This type of virus is called an oncolytic virus. Oncolytic viruses selectively replicate within tumorous tissue and break it down without doing excessive damage to normal tissue.

In Japan, we are currently conducting clinical trials targeting melanoma at the National Cancer Center Hospital and other facilities, and are aiming for approval application. In the U.S., investigator initiated clinical trials targeting melanoma are underway at the Huntsman Cancer Institute. In December 2016, we entered into a joint development and exclusive sales agreement for Canerpaturev (C-REV) in Japan with Otsuka Pharmaceutical Co,. Ltd., and will pursue ongoing clinical development of Canerpaturev (C-REV).

Engineered T Cell Therapy

Cell culture

1. siTCRTM Gene Therapy

TCR gene therapies taking autologous lymphocytes from a patient, transducing TCR genes capable of recognizing cancer antigens into the lymphocytes, multiplying the lymphocytes, and introducing the lymphocytes back into the patient. The therapy makes use of the advantage of the gene transduction into lymphocytes, which identify, attack, and eliminate cancer cells.

We are developing siTCRTM gene therapy that involves the use of our proprietary siTCRTM vector technique, which is thought to reduce the risk of side effects and improve effectiveness.

In clinical development, we are engaged in Phase I/II clinical trials of NY-ESO-1 siTCRTM gene therapy targeting synovial sarcoma in Japan.

In April 2018, we conducted a joint development and exclusive sales agreement in Japan with Otsuka Pharmaceutical Co., Ltd. We are cooperatively undertaking development aimed at obtaining early manufacturing and sales approval for pharmaceuticals.

2. CAR Gene Therapy

Chimeric Antigen Receptors (CARs) are receptors that are made by artificially combining parts derived from antibodies that specifically recognize certain cancer antigens with parts with cytotoxic functions derived from T-cell receptors. CAR gene therapies involve putting autologous lymphocytes transduced with CAR genes back into the patient, allowing these lymphocytes to identify and attack cancer cells, thereby eliminating them.

In Japan, we are also conducting clinical trials of CD19 CAR gene therapy targeting adult acute lymphocytic leukemia. As in the NY-ESO-1 siTCRTM gene therapy project, we are addressing unmet medical needs in collaboration with our partner Otsuka Pharmaceutical Co., Ltd.


In engineered T cell therapy, one type of gene therapy, a therapeutic gene is transduced into cells such as lymphocytes taken from a patient and infused back into the patient. With this therapy, the RetroNectin® method for highly-efficient gene transduction and the RetroNectin® expansion culture method for highly-efficient expansion culture of lymphocytes, developed by our company are used. These technologies have become the standard in gene therapy and have been licensed to many companies and organizations seeking to commercialize gene therapies. Takara Bio will continue to provide RetroNectin® and promote the licensing out of technology to companies and organizations engaged in the clinical development of Engineered T cell Therapy, and will work to expand sales.

Business Outline

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