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AZD9829 is a first-in-class antibody-drug conjugate (ADC) developed by AstraZeneca for the treatment of hematological malignancies, particularly relapsed or refractory acute myeloid leukemia (AML) and myelodysplastic syndromes. The drug consists of a monoclonal antibody targeting the interleukin-3 receptor subunit alpha (CD123), which is overexpressed on certain cancer cells, conjugated to a topoisomerase I inhibitor payload. Upon binding to CD123-expressing cells, AZD9829 delivers the cytotoxic topoisomerase I inhibitor directly into malignant cells, leading to DNA damage and apoptosis. Preclinical studies have demonstrated its efficacy in vitro and in vivo against CD123-positive AML cell lines and patient-derived xenograft models[1][2][5][6][8].
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