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CAR123 (UHKT-CAR123-01) is an investigational autologous chimeric antigen receptor (CAR) T-cell therapy targeting the CD123 antigen (interleukin-3 receptor subunit alpha), which is highly expressed in various hematological malignancies. Developed by the Institute of Hematology and Blood Transfusion (ÚHKT) in Prague and Charles University, this therapy is distinguished by its non-viral manufacturing process. It utilizes a linear DNA-based piggyBac transposon system (incorporating Applied DNA Sciences' Linea DNA technology) for gene transfer, which is designed to be more cost-effective and rapid than traditional viral vector methods. The CAR construct includes a 4-1BB-zeta intracellular signaling domain to enhance T-cell persistence and activation. It is currently being evaluated in Phase 1 clinical trials for relapsed or refractory acute myeloid leukemia (AML) and other CD123-positive hematologic disorders.
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