health
AAV gene therapy raises factor IX in 11 adolescents with hemophilia B
A multicentre phase 1 trial in China tested the AAV gene therapy BBM-H901 in 11 adolescents with severe or moderately severe hemophilia B. At 52 weeks, mean factor IX activity was 41.8 IU/dl and the mean annualized bleeding rate had fallen from 13.9 to 0.5.