- Able to provide informed consent and comply with requirements of the study
- Males ≥18 y.o. with confirmed diagnosis of hemophilia B (≤2 IU/dL or ≤2% endogenous
factor IX)
- Received ≥50 exposure days to factor IX products
- A minimum average of 4 bleeding events per year requiring episodic treatment of factor
IX infusions or prophylactic factor IX infusions
- No measurable factor IX inhibitor as assessed by the central laboratory and have no
prior history of inhibitors to factor IX protein
- Agree to use reliable barrier contraception until 3 consecutive samples are negative
for vector sequences
- Evidence of active hepatitis B or C
- Currently on antiviral therapy for hepatitis B or C
- Have significant underlying liver disease
- Have serological evidence* of HIV-1 or HIV-2 with CD4 counts ?200/mm3 (* subjects who
are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible
to enroll)
- Have detectable antibodies reactive with AAV-Spark100
- Participated in a gene transfer trial within the last 52 weeks or in a clinical trial
with an investigational drug within the last 12 weeks
- Unable or unwilling to comply with study assessments