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Gandotinib is an orally bioavailable small molecule inhibitor developed by Eli Lilly for the treatment of cancer, particularly myeloproliferative neoplasms. It selectively and competitively inhibits Janus kinase 2 (JAK2), with increased potency for the JAK2 V617F mutation—a key driver in diseases such as primary myelofibrosis, polycythemia vera, and essential thrombocythemia. By inhibiting JAK2V617F activation, gandotinib disrupts the JAK-STAT signaling pathway and induces apoptosis in affected tumor cells. Clinical trials have shown its potential antineoplastic activity and acceptable safety profile at a maximum-tolerated dose of 120 mg daily[1][3][5].
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