RUNX1 isoform imbalance drives ML-DS, VWF-binding RNA aptamer for hemophilia A treatment, and EZH1/2 inhibitor valemetostat in relapsed/refractory ATL

In this week’s episode we will review an imbalance among RUNX1 isoforms is key to the pathogenesis of trisomy 21-associated myeloid leukemia, raising the possibility that equilibrium could be restored genetically or pharmacologically. Next, an RNA aptamer demonstrating promising results in patients with hemophilia A. Lastly, phase 2 data on valemetostat, a selective inhibitor of EZH1 and 2, in relapsed or refractory adult T-cell leukemia/lymphoma.