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UniQure Announces New Data from Phase I/II Trial of AMT-O60
Dec. 05, 2016 09:00AM PST
Biotech Investing
UniQure has announced new data from its ongoing phase I/II trial of AMT-060, an investigational therapy for severe hemophilia B.
UniQure (NASDAQ:QURE) has announced new data from its ongoing phase I/II trial of AMT-060, an investigational therapy for severe hemophilia B.
According to the press release:
New data presented from the second-dose cohort show a dose response with substantial improvement in disease state in all five patients, including the discontinuation of precautionary Factor IX (FIX) infusions in all four patients that previously required chronic replacement therapy. To date, only one spontaneous bleed was reported after discontinuation of prophylactic FIX replacement therapy.
Christian Meyer, Chief Medical Officer at uniQure, said the following:
“The proprietary elements of AMT-060, including our fully-humanized FIX gene cassette and AAV5 vector, are the only gene therapy components clinically demonstrated in hemophilia B to be safe, effective, and durable for up to six and a half years. We believe these factors, along with our commercial-scale manufacturing capabilities, differentiate AMT-060 from other hemophilia gene therapies in development, and we look forward to advancing our program into a late-stage clinical study.”