YTHDC1 Inhibitor

Impairs leukemia stem cells

A first-in-class selective small-molecule inhibitor of YTHDC1 that induces AML apoptosis by blocking m⁶A binding while sparing normal hematopoietic cells.

Zhang, Hailin · Li, Yueshan · Zhao, Yin · Niu, Ting · He, Li · Liu, Lina · Yi, Yuyao · He, Yanmin · Zhou, Pei · You, Jing · Liao, Yi · Yang, Chenlu · Zhao, Ailin · Chen, Chong · Li, Linli · Yang, Shengyong

Science Translational Medicine 2026

Advantages

First-in-class

First selective small-molecule inhibitor of YTHDC1, offering a novel target for AML therapy.

Eliminates leukemia

Blocks YTHDC1 binding to m6A substrates, reduces mRNA stability, and induces apoptosis and myeloid differentiation in AML cells.

Spares normal cells

Selectively impairs leukemia stem cells while sparing normal hematopoietic counterparts.

Applications

• AML therapy：Eliminates leukemia cells and extends survival
• Epigenetic drugs：Targets the RNA m6A modification pathway
• Small-molecule inhibitors：Blocks YTHDC1 function with high selectivity
• Hematopoietic stem cell protection：Preserves normal hematopoietic cell function