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**MDC-CAR-BCMA001** is a second-generation autologous CAR T-cell therapy targeting BCMA (B-cell maturation antigen), featuring a high-affinity binder and CD28 co-stimulatory domain, enabling efficacy in low antigen-expressing diseases and BCMA-pretreated patients. Developed by the German Cancer Research Center and Technische Universität Dresden, it has shown promising efficacy in compassionate use for heavily pretreated, triple-class refractory multiple myeloma and AL amyloidosis, with 5/6 overall responses (4 complete responses, all MRD-negative) and robust CAR T-cell expansion/persistence up to over a year. Safety is favorable, with mostly low-grade CRS, no neurotoxicity, and manageable cytopenias/infections; ongoing phase 1 trials (CARLOTTA01, NCT05836896) evaluate it in relapsed/refractory multiple myeloma, DLBCL, and planned CLEAR-AL for AL amyloidosis.[1][2][3][5][6][7][8]
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