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KCC07 (also known as KCC-07) is an experimental, blood-brain barrier (BBB) permeable small molecule inhibitor of Methyl-CpG-binding domain protein 2 (MBD2). As an epigenetic modulator, KCC07 prevents MBD2 from binding to methylated DNA, thereby derepressing genes typically silenced by the MBD2-NuRD (Nucleosome Remodeling and Deacetylase) complex. In glioblastoma models, KCC07 reactivates the expression of the tumor suppressor BAI1 (ADGRB1), which in turn inhibits TGFβ1 maturation and suppresses the mesenchymal switch, invasion, and metastasis. In the context of acute myeloid leukemia (AML), particularly MLL-rearranged and NPM1-mutant subtypes, KCC07 has been shown to synergize with Menin inhibitors (such as revumenib) to lock in terminal myeloid differentiation and overcome the reversibility of Menin inhibitor monotherapy.
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