Kusatsu/Shiga, Japan –March 27, 2018 – Takara Bio Inc. (Takara Bio), today announced that a NY-ESO-1・siTCR™ gene therapy product (Development code: TBI-1301) for synovial sarcoma tested in ongoing clinical trial in Japan has been designated as a product under the “SAKIGAKE Designation System” (hereinafter, “SAKIGAKE”) by the Ministry of Health, Labour and Welfare.
SAKIGAKE introduced by the Ministry of Health, Labour and Welfare in 2015 aims at early launching of the most advanced products to lead the world by putting to practical application of the designated products speedily for innovative new medical products that satisfy certain criteria.
The gene of TCR which recognizes NY-ESO-1 antigen, a tumor antigen, is transduced ex-vivo to lymphocytes of patients, and the gene-modified lymphocytes called NY-ESO-1・siTCR™ gene therapy product designated lately are infused back to the patients as a therapeutic product of cancer. Takara Bio is conducting domestic Phase I/II clinical trial of NY-ESO-1・siTCR™ gene therapy product targeting synovial sarcoma since January 2017.
With this designation, the NY-ESO-1・siTCR™ gene therapy product is capable of receiving any type steps of prioritized consultation on clinical trials with the Pharmaceuticals and Medical Services Agency (PMDA), prior evaluation, review, etc. Takara Bio aims for early commercialization of NY-ESO-1・siTCR™ gene therapy product by utilizing advantage of this designation maximally.
SAKIGAKE Designation System
The system started by the Ministry of Health, Labour and Welfare since 2015. It aims at early launching of the most advanced products to lead the world for innovative new medical products that satisfy certain criteria, by designating such products during the early stages of development, providing prioritized process for consultation and review regarding pharmaceutical approval, and conducting further speedy practical application based on the sufficient efforts in order to construct the manufacture framework and provide to medical sites smoothly following approval along with schedule for approval review from applicants.
http://www.mhlw.go.jp/english/policy/health-medical/pharmaceuticals/140729-01.html
Synovial sarcoma
Synovial sarcoma is one of aggressive soft-tissue tumors associated with malignancy and with patterns of regional and distant metastasis and carries a poor prognosis. The high incidence of NY-ESO-1 antigen expression is seen in cases of synovial sarcoma, and its expression is relatively uniform throughout tumor tissue and is abundantly observed per cell.
NY-ESO-1
NY-ESO-1 is one of tumor antigen, and its expression is observed in synovial sarcoma, malignant melanoma, esophageal cancer, ovarian cancer, multiple myeloma, head and neck cancer, etc. Tumor antigen serves as a mark which immune cells distinguish cancer cells from normal cells, and is characteristic of cancer cells.
NY-ESO-1 siTCR™ gene therapy product
TCR which recognizes NY-ESO-1 antigen, a tumor antigen, is transduced ex-vivo to lymphocytes (T cells) of patients with cancer, and the gene-modified lymphocytes are subject to the efficient expansion culture and infused back to the patients as a therapeutic drug. It acquires the capability to specifically recognize and attack cancer cells and eliminate them. When transducing TCR gene to lymphocytes, the product for TCR gene therapy based on Takara Bio’s proprietary siTCR vector technique is named NY-ESO-1・siTCR™ gene therapy drug.
siTCR vector technique
The siTCR vector technique artificially minimizes the expression of endogenous TCRs from lymphocytes (T cells) and allows for obtaining more lymphocytes (T cells) that express the target TCR, leading to the reduced side effect of TCR gene therapy and the enhanced effectiveness more likely.
TCR (T Cell Receptor)
TCR is a glycoprotein expressed on lymphocytes (T cells) and acts at the time of recognizing of tumor antigen by lymphocytes.
