RUNX1 isoform imbalance drives ML-DS, VWF-binding RNA aptamer for hemophilia A treatment, and EZH1/2 inhibitor valemetostat in relapsed/refractory ATL
Blood Podcast
00:00
Efficacy of Run Daptovon-Pegel in Hemophilia A
A cornerstone of bleeding prevention in severe hemophilia is regular prophylactic administration of factor A. That's expensive and can lead to development of factor A inhibitors. Factor A has a limited half-life that hinges on how quickly its chaperone, Von Willabrand Factor, gets cleared. Run Daptovon-Pegel is a methylated RNA aptimer conjugated to a 40-kilodolton polyethylene glycol or PEG. The aptimer component binds to the A1 region of Von WillabRand Factor.
Transcript
Play full episode
