Shiga, Japan – October 5, 2023 - Takara Bio Inc., an innovative biotechnology company, announced that the results from the clinical trial of NY-ESO-1・siTCRTM gene therapy (Development Code: TBI-1301, INN: mipetresgene autoleucel) for patients with advanced or recurrent synovial sarcoma were published in Clinical Cancer Research online, a journal of the American Association for Cancer Research (AACR).


Safety and Efficacy of NY-ESO-1 Antigen-specific T-cell Receptor Gene-Transduced T Lymphocytes in Patients with Synovial Sarcoma: A Phase I/II Clinical Trial


  • Synovial sarcoma is one of malignant soft tissue tumors with a poor prognosis. 50 to 80% of synovial sarcoma patients express cancer-testis antigen NY-ESO-1 in tumors. The purpose of this study was to evaluate the safety and efficacy of gene engineered autologous T cell product (TBI-1301) transduced with NY-ESO-1 siTCR retroviral vector.
  • Subjects in this study were HLA-A*02:01 or HLA-A*02:06 positive patients with advanced or recurrent synovial sarcoma expressing NY-ESO-1, which were surgically unresectable and refractory to anthracycline therapy. Eight subjects were enrolled and treated with TBI-1301.
  • Objective response rate (ORR) was 50.0% with best overall partial response in 4 of 8 subjects. Median overall survival (OS) was 650.0 days.
  • Cytokine release syndrome (CRS) occurred in 50.0% of patients, but well managed with pre-specified treatment.
  • Adoptive immunotherapy with TBI-1301 to NY-ESO-1 positive tumors will become a promising treatment for advanced or recurrent synovial sarcoma with acceptable toxicity.

Takara Bio is proceeding with the preparation for the New Drug Application of TBI-1301 in Japan along with the establishment of its post marketing supply chain system. We are also open to discuss possible partnering or licensing arrangements of TBI-1301.

Takra Bio provides Contract Development Manufacturing Organization (CDMO) services using our siTCR~{TM} and JAK/STAT technologies to pharmaceutical industries and academic laboratories pursuing gene therapy research. We are dedicated to contribute to good health of humankind through our innovative gene and cell processing technologies.