February 8, 2019 / 12:30 PM / 15 days ago

UniQure gene therapy shows rise in blood-clotting protein in trial

Feb 8 (Reuters) - UniQure NV said on Friday its gene therapy to treat patients with a blood disorder, hemophilia B, increased the levels of a protein that helps in blood clotting after 12 weeks.

The updated data from a mid-stage study showed that all three patients on the therapy, AMT-061, showed increasing and sustained levels of factor IX, which helps stop bleeding through blood clots.

Hemophilia B is a genetic disorder where the lack of enough factor IX (FIX) prevents blood from clotting, resulting in severe bleeding.

FIX activity at 12 weeks increased to 38 percent of normal, which exceeded the threshold levels generally considered enough to eliminate or cut down the risk of bleeding events, UniQure said. (Reporting by Manas Mishra in Bengaluru; Editing by Arun Koyyur)

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