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CLL-1 CAR T-cell therapy is an investigational autologous, second-generation chimeric antigen receptor (CAR) T-cell therapy developed by Baylor College of Medicine for the treatment of relapsed or refractory acute myeloid leukemia (AML). The therapy involves genetically modifying a patient's own T-cells using a gammaretroviral vector to express a CAR targeting C-type lectin-like molecule-1 (CLL-1, also known as CLEC12A or CD371). CLL-1 is a transmembrane glycoprotein highly expressed on AML blasts and leukemic stem cells but notably absent from normal hematopoietic stem cells, allowing for selective targeting while potentially sparing normal myelopoiesis. The CAR construct incorporates a CD28 costimulatory domain to enhance T-cell activation and persistence. Administered via a single intravenous infusion following lymphodepleting chemotherapy, the therapy has been evaluated in Phase 1 clinical trials (such as the CARMEN trial) with the primary goal of inducing morphologic remission to serve as a bridge to potentially curative allogeneic hematopoietic stem cell transplantation (HSCT).
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