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GFH009 is a potent and highly selective small molecule inhibitor of cyclin-dependent kinase 9 (CDK9), the catalytic subunit of the RNA polymerase II elongation factor positive transcription elongation factor b (PTEF-b). By inhibiting CDK9, GFH009 disrupts transcriptional regulation critical for cancer cell survival, leading to reduced expression of anti-apoptotic proteins such as MCL-1 and c-MYC. This results in antiproliferative and proapoptotic effects in hematologic malignancy models. The drug has demonstrated significant antitumor activity in preclinical studies and early clinical trials, particularly in acute myeloid leukemia (AML) and relapsed/refractory lymphoma. GFH009 has received FDA Fast Track and Orphan Drug designations for treating AML and peripheral T-cell lymphomas[1][3][4][5][6][7].
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