Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.

ABSTRACT: Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous factor IX (FIX) activity/synthesis through a single treatment, eliminating the requirement for FIX concentrate. AMT-060 combines an adeno-associated virus-5 (AAV5) vector with a liver-specific promoter driving expression of a codon-optimized wild-type human FIX gene. This multinational, open-label study included 10 adults with hemophilia B (FIX ≤2% of normal) and severe-bleeding phenotype. No participants tested positive for AAV5-neutralizing antibodies using a green-fluorescent protein-based assay, and all 10 were enrolled. A single dose of 5 × 10¹² or 2 × 10¹³ genome copies of AMT-060/kilogram was administered to 5 participants each. In the low-dose cohort, mean endogenous FIX acti

SUBMITTER: Miesbach W 

PROVIDER: S-EPMC5833265 | biostudies-literature | 2018 Mar

REPOSITORIES: biostudies-literature