Mechanism
CAR-T cell therapy
Assets acting on this target.
CAR-T cell therapy is a form of cellular immunotherapy in which a patient's own T cells (or, in newer approaches, T cells inside the body) are engineered to express a chimeric antigen receptor, a synthetic protein that recognizes a specific marker on the surface of cancer cells and triggers the T cell to destroy them. This recognition does not depend on the antigen-presentation machinery that normally restrains T-cell activity, so engineered cells can attack tumors that evade conventional immune surveillance. One important application targets BCMA (B-cell maturation antigen), a protein expressed almost exclusively on plasma cells, the antibody-producing cells that become malignant in multiple myeloma. Because BCMA expression is largely confined to this lineage, targeting it allows selective elimination of malignant plasma cells while limiting damage to unrelated tissues. Manufacturing approach matters: conventional CAR-T requires collecting a patient's T cells, engineering them outside the body, and reinfusing them, a process with inherent lead time; in vivo-generated CAR-T instead aims to deliver the genetic instructions directly into the body so T cells are reprogrammed in situ, potentially simplifying logistics. Across formats, CAR-T represents a shift from small-molecule or antibody drugs toward living, self-amplifying therapeutics, relevant wherever a tumor-restricted surface antigen can be reliably identified.
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