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UniQure Has High HOPE For Hemophilia B Gene Therapy

 
• By Kevin Grogan

The Dutch company's update on AMT-061 and the initiation of a pivotal trial called HOPE-B of around 50 patients could put the product in pole position to be the first gene therapy on the market for the severe orphan blood clotting disorder.

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Uniqure in pole position for hemophilia gene therapy? • Source: Shutterstock

uniQure NV has presented promising updated results from a Phase IIb trial of its gene therapy AMT-061 for hemophilia B which the Netherlands-headquartered firm believes could be best in class and first to market ahead of Spark Therapeutics Inc. and Pfizer Inc.'s rival candidate.

Initial data released in November demonstrated that after six weeks, three patients receiving AMT-061, an AAV5-based gene therapy which contains a FIX-Padua variant, had mean factor IX (FIX) activity of 31% of normal

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