DTX101: Preliminary Ph I/II data

Preliminary data from 6 patients with moderate/severe to severe hemophilia B in an open-label, international Phase I/II trial showed that single doses of 1.6x10¹² and 5x10¹² GC/kg DTX101 led to peak Factor IX levels of 10-11% and 12-20%, respectively. The 3 patients receiving high-dose DTX101 had peak Factor IX expression of 13%, 20% and 12% at weeks 4, 8 and 8, respectively. All 6 patients’ Factor IX levels improved from either moderate/severe or severe to either the moderate or mild range based on World Federation of Hemophilia (WFH) criteria. No patient in the high-dose DTX101 cohort required prophylactic or on-demand recombinant Factor IX transfusion for spontaneous bleeds post-treatment...