uniQure Announces Hemophilia B Gene Therapy Program To Enter Pivotal Study

Note: The followed is excerpted from a press release from uniQure. Read uniQure’s full press release HERE. 

uniQure announced today plans to advance their development program for hemophilia B gene therapy into a pivotal study using an enhanced version of their product candidate called AMT-061. AMT-061 is identical to AMT-060 in every way apart from a change from the wild-type Factor IX gene to what is commonly known as the “Padua” gene – a variant of the Factor IX gene that has been shown to provide substantial increases in Factor IX activity compared to the wild-type gene used in AMT-060.