uniQure maps plan for hemophilia B gene therapy

uniQure N.V. (NASDAQ:QURE) rose $5.69 (60%) to $15.16 after it said it will begin a Phase III trial next year of gene therapy AMT-061 to treat hemophilia B. The adeno-associated viral serotype 5 (AAV5) vector's structure is identical to uniQure's AMT-060, which is in an ongoing Phase I/II trial, but AMT-061 carries a Factor IX (FIX) gene with the FIX-Padua (R338L) mutation. The enhanced version of the candidate will remain under AMT-060's IND.

uniQure said that in preclinical studies, the Padua variant has shown up to a ninefold increase in FIX activity compared to wild-type FIX protein. Next quarter, the company plans to submit data to FDA and EMA from its comparability analysis of AMT-061 and AMT-060...