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Drug intelligence / Profile preview
Anti-CD33 CAR-T cells are chimeric antigen receptor T cells genetically engineered to express a synthetic receptor targeting CD33, a transmembrane receptor highly expressed on the majority of acute myeloid leukemia (AML) blasts and some myeloid cells. These therapies use a single-chain variable fragment (scFv) specific for CD33 fused to costimulatory and signaling domains (commonly CD28 or 4-1BB and CD3ζ) to redirect T cells to recognize and kill CD33-expressing malignant cells. Anti-CD33 CAR-T therapies are under active investigation for relapsed/refractory AML; they demonstrate potent cytotoxicity in preclinical models and early human trials but are associated with risks including pancytopenia and cytokine release syndrome due to CD33 expression on normal hematopoietic cells. Variants include rapamycin-regulated (DARIC33) and bicistronic constructs for enhanced safety and efficacy. No anti-CD33 CAR-T therapy is FDA-approved as of August 2025[1][3][4][5][6][9].
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