TBI-1301

TBI-1301 is an autologous T-cell receptor (TCR) engineered T-cell therapy developed by Takara Bio in collaboration with Mie University for the treatment of hematologic malignancies, including acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The therapy involves the retroviral transduction of a patient's own T-cells with the MS3-WT1-siTCR vector, which expresses a transgenic TCR specific for the WT1 235-243 peptide (CYTWNQMNL) presented by the HLA-A*24:02 allele. A key innovation in TBI-1301 is the use of "siTCR" technology, which incorporates small interfering RNAs (siRNAs) into the vector to knock down the expression of endogenous TCR alpha and beta chains. This process minimizes TCR mispairing, thereby enhancing the surface expression and antigen-specific reactivity of the therapeutic TCR while potentially reducing the risk of off-target toxicities. Following intravenous administration of the transduced T-cells, patients typically receive sequential WT1 peptide vaccinations to promote cell expansion and persistence. While early trials showed safety and evidence of antileukemic activity, Takara Bio announced the termination of the TBI-1301 program in 2023.

Other names

WT1-specific TCR-gene transduced T-cellsWT-1-specific TCR-gene transduced T-cellsWT 1-specific TCR-gene transduced T-cellsWT1-siTCR T-cellsWT-1-siTCR T-cellsWT 1-siTCR T-cellsWT1 TCR-gene transduced T-cellsWT-1 TCR-gene transduced T-cellsWT 1 TCR-gene transduced T-cells