Table 2.

Typical inclusion criteria in AAV gene therapy trials, current and proposed

Severe hemophilia B: FIX ≤ 1%

Age ≥ 18 y

HCV RNA viral load-negative*

HIV-negative†

No previous history of FIX inhibitor

At least 20 exposure days to FIX concentrate

Anti–AAV-neutralizing antibody titer ≤ 1:5

^*Earlier trial included subjects who were HCV RNA viral load-positive, but they are now excluded because of potential need for course of steroids.

^†Some trials may include HIV-positive persons who are stable with adequate CD4 counts on highly active antiretroviral therapy.