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Anti-CLL1 CAR T-cells (4-1BB) is an investigational adoptive cell therapy (CAR T-cell biologic) in which a patient's T cells are genetically engineered to express a chimeric antigen receptor (CAR) targeting CLL1 (C-type lectin-like molecule-1; CLEC12A). This specific construct incorporates the 4-1BB (CD137) co-stimulatory signaling domain along with a CD3-zeta activation domain. CLL1 is highly expressed on acute myeloid leukemia (AML) blasts and leukemic stem cells but is absent on normal hematopoietic stem cells, making it a selective therapeutic target. The 4-1BB co-stimulatory domain is included to enhance T-cell metabolic fitness, promote long-term persistence, and mitigate T-cell exhaustion. Preliminary clinical data from studies in pediatric relapsed/refractory AML have shown an overall response rate of approximately 66.7% and CAR T-cell persistence for up to 274 days, with a manageable safety profile characterized primarily by low-grade cytokine release syndrome.
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