uniQure presents positive trial results for gene therapy AMT-061

8 February 2019

Researchers today presented updated clinical data from Netherlands headquartered biotech uniQure’s (Nasdaq: QURE) ongoing Phase IIb study of AMT-061, an investigational gene therapy for patients with severe and moderately severe hemophilia B.

Results showed increases in FIX activity sustained at up to 51% of normal and mean FIX activity of 38% of normal at 12 weeks after a single administration of AMT-061, which exceeds threshold FIX levels generally considered sufficient to eliminate or significantly reduce the risk of bleeding events. The data were announced in an oral presentation at the Annual Congress of the European Association of Hemophilia and Allied Disorders (EAHAD) in Prague, Czech Republic.

The positive news, which comes after disappointing results at the same meeting from Solid Biosciences (Nasdaq: SLDB) for SGT-001 in Duchenne muscular dystrophy, saw uniQure’s shares leap 8.95% to $36.15 by mid-morning trading today.

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