Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A

CD34+ hematopoietic stem cells transduced with CD68-ET3 lentiviral vector (encoding human factor VIII gene) is administered by IV infusion following conditioning regimen with busulfan and anti-thymocyte globulin.

G-CSF and Plerixafor are administered by subcutaneous injection prior to apheresis.