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BioWorld - Saturday, July 11, 2026
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Home » Uniqure gene therapy still looking good in hemophilia B at second interim peek
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Uniqure gene therapy still looking good in hemophilia B at second interim peek

Feb. 11, 2019
By Cormac Sheridan
DUBLIN – The mood music coming out of Uniqure NV's phase IIb trial for its hemophilia B gene therapy, AMT-061, remains positive. A second interim readout last week, after an initial peek at the data sparked a share-buying frenzy last November, showed continued improvement in levels of factor IX (FIX) expression in two of the three patients enrolled in the study. The third patient has plateaued but has maintained his response to the therapy, which comprises an adeno-associated virus 5 (AAV5) vector encoding the highly active Padua variant of FIX.
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