Gene Therapy for Chinese Hemophilia B

Inclusion Criteria

• •1. Be able to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local privacy regulations;
• •2. Be male and ≥18 years of age;
• •3. Have hemophilia B with ≤2 IU/dL (≤2 %) endogenous FIX activity levels as documented by a certified clinical laboratory at the time of screening. If the screening result is \>2% due to insufficient washout from FIX protein product, then the severity of hemophilia B may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ≤2% FIX coagulant activity (FIX:C) ;
• •4. Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products based on historical data from the subject's record/history;
• •5. a. Prophylaxis subjects: have had bleeding events and/or infusions with FIX protein products during the last 12 weeks documented in the subjects' medical records; OR b. On-demand subjects: have had ≥4 bleeding events in the last 52 weeks and/or chronic hemophilic arthropathy (pain, joint destruction, and loss of range of motion) in one or more joints;
• •6. Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration;
• •7. Have no measurable FIX inhibitor as assessed by laboratory; or documented no prior history of FIX inhibitor after 50 EDs (family history of inhibitors will not exclude the subject) and no clinical signs or symptoms of decreased response to FIX administration;
• •8. Have acceptable laboratory values:
• •1. Hemoglobin ≥11 g/dL;
• •2. Platelets ≥100,000 cells/μL;
• •3. AST, ALT, alkaline phosphatase ≤2x upper limit of normal at the testing laboratory;
• •4. Bilirubin ≤3x ULN ;
• •5. Creatinine ≤2.0 mg/dL.
• •9. Agree to use reliable barrier contraception until 52 weeks and semen samples after the administration of BBM- H901 are negative for vector sequences.