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UniQure Hemophilia B Gene Therapy AMT-061 Has Early Phase IIb Success

 
• By Sten Stovall

Early positive data from a Phase IIb dose-confirmation study showed AMT-061 achieved and sustained therapeutic levels of Factor IX (FIX) activity in all three participating patients at six weeks after a single administration of the investigational AAV5-based gene therapy.

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AMT-061 Is modified version of earlier hemophilia B gene therapy AMT-60 • Source: Shutterstock

Good early data from a Phase IIb trial showed its three patients with severe or moderately severe hemophilia B responded well to uniQure NV's new gene therapy AMT-061, reinforcing its competitive position in the race to bring the first gene therapy for hemophilia B to market.

The Phase IIb study’s primary goal is to assess Factor IX (FIX) activity levels following administration of AMT-061 and to confirm the dose that will be used in a Phase III study, HOPE-B, expected to begin in the

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Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.