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UCART123 is an allogeneic (donor-derived) chimeric antigen receptor T-cell (CAR-T) therapy engineered to target CD123, the interleukin-3 receptor subunit alpha, which is highly expressed on acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN) cells. Unlike autologous CAR-T therapies that use a patient’s own T-cells, UCART123 uses healthy donor T-cells that are gene-edited using TALEN technology to knock out the TCRαβ gene, reducing the risk of graft-versus-host disease. The product also incorporates a safety switch (RQR8), allowing for elimination of the cells if needed via rituximab administration. Its mechanism involves immunologic cytotoxicity—engineered T-cells recognize and kill CD123-positive malignant cells through targeted immune response. Developed by Cellectis, it has been evaluated in phase 1 clinical trials for relapsed/refractory AML and BPDCN[1][2][3][4][5][7].
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