What type of study is this?

This is a In Vitro Study study.

December 8, 2025Blood

GB3226, a novel, orally active, first-in-class small molecule inhibitor of ENL-yeats and FLT3 kinase for the treatment of Relapsed/Refractory Acute Myeloid Leukemia

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GB3226 effectively reduced leukemia burden in both MV4;11 and MOLM13 cell lines, indicating strong therapeutic potential.
In cell line models, GB3226 demonstrated potency with IC50 values of 18 nM and 32 nM against leukemia cells respectively.
Assessment using ligand binding assays determined GB3226's activity against both ENL-YEATS and FLT3, highlighting its dual inhibitory capacity.
Potential for clinical use of GB3226 is reinforced by favorable pharmacokinetic properties and an acceptable safety profile.

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Preclinical pharmacology shows GB3226 improves leukemia outcomes in acute myeloid leukemia, suggesting it may enhance combination therapies.

Stacia Kargman (2025) studied this question.

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Preclinical pharmacology shows GB3226 improves leukemia outcomes in acute myeloid leukemia, suggesting it may enhance combination therapies.