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.
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.
Improving transduction efficiency and expansion of T cells for therapies
In engineered T-cell therapies, such as CAR and siTCR gene therapies, a therapeutic gene is transduced into cells taken from a patient or a donor and infused back into the patient. Often, transduction efficiency and T-cell expansion rates are low. To address this problem, Takara Bio Inc. and Indiana University codeveloped RetroNectin reagent, a cell culture coating reagent backed by validated protocols that promotes highly efficient gene transduction and expansion culture of lymphocytes. The RetroNectin method is widely used in the development of gene therapies, and the technology has been licensed to many companies seeking to commercialize gene therapies.