CAR gene therapy

The use of genetically modified T cells (T lymphocytes) to target cancer is a promising approach, especially for cancers that are difficult to treat using traditional methods. Chimeric antigen receptors (CARs) are artificial T-cell receptors that are made by combining 1) parts derived from antibodies that specifically recognize certain cancer antigens and 2) parts with cytotoxic functions derived from T-cell receptors. CAR gene therapies involve transducing autologous lymphocytes with the TCR genes capable of recognizing cancer antigens; putting the lymphocytes back into the patient; and allowing these lymphocytes to identify, attach, and eliminate cancer cells.

TBI has entered into a license agreement with Otsuka Pharmaceutical Co., Ltd. for codevelopment/exclusive sales of CD19 and NY-ESO-1 siTCR in Japan.

Get more information on CAR T-cell therapy, including how Takara Bio supports basic and translational research »

siTCR gene therapy

TBI has developed another approach to engineering T cells. siTCR gene therapy involves the use of their proprietary siTCR vector technique, which is thought to reduce the risk of side effects from and improve effectiveness of the reinfused engineered T cells. In this technique, the vector technology is introduced into the patient's ex vivo lymphocytes containing endogenous T-cell receptors (TCRs), leading to efficient expression of the introduced TCRs while blocking the expression of endogenous and mispaired TCRs.

Learn more about this promising siTCR vector approach for TCR gene therapy »

Oncolytic viruses selectively replicate within tumorous tissue and break it down without doing excessive damage to normal tissue, making them tremendously promising avenues in cancer treatment. C-REV is an attenuated strain of the herpes simplex virus 1 (HSV-1) that exhibits antitumor activity when injected into a cancerous region. The administration of C-REV also strengthens immunity against cancer cells and prevents tumors from forming—even in tumor regions where C-REV was not administered.

TBI is working with partners to conduct clinical trials using C-REV to treat malignant melanoma. A U.S. Phase II clinical trial administered by the Huntsman Cancer Institute at the University of Utah was completed in March 2017.

The trial examined the efficacy and safety of C-REV/Ipilimumab combination therapy. Ipilimumab is a type of anticancer drug known as an immune checkpoint inhibitor. With regards to efficacy, the combination therapy of Ipilimumab with C-REV was more effective than Ipilimumab monotherapy, suggesting the potential for this combination therapy as a new treatment for malignant melanoma. With regards to safety, the adverse events caused by C-REV were, for the most part, moderate to mild (grade 2 and below), and dose-limiting toxicity was not observed.

In Japan, Phase II clinical trials for malignant melanoma and Phase I clinical trials for pancreatic cancer are being carried out. The Takara Bio Group entered into exclusive licensing agreements with Otsuka Pharmaceutical Co., Ltd. and with Dong-A ST to develop and sell C-REV in Japan and in Korea, respectively, and will actively promote further clinical development.

Support for the development of regenerative medical products

Takara Bio Inc. offers comprehensive and seamless manufacturing and quality-testing services for cellular and gene therapy products. At the core of these efforts is the award-winning Center for Gene and Cell Processing in West Japan. The facility conducts contracted vector production for gene transduction and contracted cell processing based on GCTP/GMP*. It also produces, conducts quality testing for, and stores RetroNectin reagent, which is used in gene therapies and other products developed by Takara Bio Inc.

April 2017 saw the start of operations at the Life Innovation Center (LIC) Annex of the Center for Gene and Cell Processing in East Japan, increasing total production capacity by 50%. In addition to providing contracted cell processing services for stem cells, gene-transduced cells, and other cells used in clinical applications, the facility will serve as a cell processing center that provides investigational drugs from gene therapy projects conducted by Takara Bio Inc. to clinical testing facilities in the greater Tokyo region.

Visit our cell and gene and cell therapy manufacturing services section for a full list of services »

*GCTP: Good Gene, Cellular, and Tissue-based Products Manufacturing Practice—a standard used in manufacturing and quality control for regenerative medical products; GMP: Good Manufacturing Practice—a standard used in manufacturing and quality control for products including pharmaceuticals.