Gene Therapy for Hemophilia: Learnings from Phase 1/2 studies

Time: 3:30 pm
day: Day One


• AAV mediated gene delivery of FIX and FVIII to treat HemB and HemB results in sustained (up to 4 year) therapeutic correction of bleeding with a good safety profile
• Assays to monitor factor activity following treatment give variable results and the choice of relevant assay is a topic of interest
• While all patients show a significant cessation of bleeding at doses chosen for phase 3 development, there is patient to patient variability in level of expression. Determinants of this variability are currently unknown