Kusatsu/Shiga, Japan — January 24, 2017 — Takara Bio Inc. (Takara Bio), today announced that it has submitted the Clinical Trial Notification for regenerative medicines to PMDA (Pharmaceuticals and Medical Devices Agency), Japanese regulatory agency, to conduct phase I/II clinical trial for siTCR gene therapy (NY-ESO-1・siTCR gene therapy; Development code: TBI-1301) targeting synovial sarcoma in Japan.
In the clinical trial, TCR which recongnizes NY-ESO-1 antigen, a tumor antigen, is transferred ex-vivo to lymphocytes of patients, and the gene-modified lymphocytes are infused back to the patients. The patients will be monitored for safety and efficacy. The Takara Bio’s method of gene transduction and T-cell expansion using RetroNectinsup{®}, and Takara Bio’s original retroviral vectors for siTCR transduction will be used during the cell processing for the clinical trial.
After the clearance, the protocol will be reviewed by each IRB (Institutional Review Board) and the study will be initiated.
Takara Bio is also conducting Phase Ib clinical trial of NY-ESO-1・siTCR gene therapy targeting solid tumor in Canada. With the safety and efficacy data obtained from the clinical trials in Japan and Canada, Takara Bio attempts to achieve the early-approval utilizing the conditional and term-limited approval system for regenerative medicine under The Law on Securing Quality, Efficacy and Safety of Products including Pharmaceuticals and Medical devices. Takara Bio aims to commercialize the NY-ESO-1・siTCR gene therapy in the fiscal year 2020.

【Overview of the Clinical Trial】

Study name Multicenter Phase I/II Clinical Trial of TBI-1301 for synovial sarcoma with pretreatment of chemotherapeutic administration
Condition Patients with unresectable progressive or recurrent synovial sarcoma
Main Endpoint

Phase I Clinical trial: Safety
Phase II Clinical trial: Efficacy

Estimated Enrollment 8
Duration Mar 2017 – Jan 2020
Site National Cancer Center Hospital, Osaka National Hospital and others, total 5 sites