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MiamiOH OARS on 20 May 201. Basic Science of Gene Therapy for Hemophilia * Basic science, tropism, transduction efficiency & tolerability of Adeno associated virus (AAV) * AAV antibody seroprevalence, titer- assessment, reduction & tolerance * Role of immunosuppression in managing immune response and potential retreatment * Effect of Gene Therapy on liver biology 2. Basic Science of TFPI & Anti-TFPI Monoclonal Antibodies * Basic biology of TFPI interactions with Protein C, ATIII & Protein S * Cross talk among regulators (e.g., Protein S being a co-factor for both Protein C and TFPI) * Role of different TFPI pools in regulation of coagulation * Impact of concomitant treatments (especially antifibrinolytics) added to anti-TFPI on the physiology of hemostasis * Note: Pfizer will not supply study drug 3. Burden of disease: Clinical Hemophilia A and B * Natural history of hemophilia and adherence to current standard of care * Arthropathy: presence, development, clinical burden & joint damage in hemophilia * Patient experiences with hemophilia, treatment preferences and quality of care * Quality of Life/Work analysis and cost of care in Hemophilia