uniQure gene therapy leads to sustained Factor IX activity in Phase IIb study

uniQure N.V. (NASDAQ:QURE) said Feb. 8 new data showed sustained Factor IX activity in a Phase IIb study of gene therapy AMT-061 to treat hemophilia B. Separately, the company said Feb. 4 it had dosed the first patient in the Phase III HOPE-B study of the therapy.

The updated data showed that among three patients with severe disease, mean Factor IX levels rose to 38% of normal 12 weeks after a single IV administration, from 31% six weeks after the therapy was given. uniQure had reported the six-week data in an interim update in November (see "uniQure Gains on Factor IX Activity Data for Hemophilia B Gene Therapy")...