Gene Therapy for Chinese Hemophilia A

"Patients will be enrolled sequentially every 3 weeks or more between cohorts. Dose escalation may occur after a single patient has been safely dosed if the resulting FVIII activity at Week 3 is \< 5 IU/dL.The dose levels are as follows:~1. 2×10\^12 vg/kg~2. 6×10\^12vg/kg or other recommended doses~3. 2×10\^13 vg/kg or other recommended doses"