Therapy delivers long-term benefits in hemophilia B

The researchers estimated that overall spending on factor IX replacement therapy for study participants is down more than $2.5 million.

Twelve men have now joined this ongoing phase 1/2 trial. Half were treated at the University College London and half at St. Jude.

Discussions are underway about expanding the trial to include younger patients with hemophilia B. Meanwhile, work continues to improve and expand use of the vector for the treatment of hemophilia A.