Dr. Steven Pipe is the Principle Investigator on the clinical trial "A Phase 1/2 Open-Label, Two Part, Dose-Finding, Safety, Tolerability, and Efficacy Study of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B.” Presently, patients with severe hemophilia B are reliant on lifelong prophylactic infusions for factor IX (FIX) concentrates beginning as infants, in order to prevent life threatening bleeds and recurrent joint bleeding that can lead to debilitating joint disease as adults. Gene therapy for hemophilia B offers the potential to effectively prevent bleeding episodes, and subsequent complications, for many years following a single treatment.
This is the first gene therapy trial for hemophilia to open in the Midwest. The University of Michigan is the lead center and has already infused the first subject and the second subject is in queue for infusion in the month of February. This study will examine three doses of the vector as part of a multi-center international study group. Based on the success of this trial, Dr. Pipe expects to take part in opening a similar trial for hemophilia A (factor VIII) deficiency later this year. Very strong work, Dr. Pipe!