En­cour­ag­ing gene ther­a­py da­ta give uniQure an in­jec­tion of con­fi­dence as it preps PhI­II show­down with Spark

Gene ther­a­py com­pa­ny uniQure start­ed the week in style with the news that it’s dosed the first pa­tient in its Phase III tri­al — a cru­cial step to­ward its quest for the best-in-class, first-in-mar­ket ti­tle in he­mo­phil­ia B gene ther­a­py. And to­day it’s of­fer­ing an up­date on the kind of mid-stage da­ta it’s armed with.

Mean Fac­tor IX lev­els hit 38% of nor­mal lev­els 12 weeks af­ter the three pa­tients tracked in the Phase IIb study re­ceived the one-time in­jec­tion, reach­ing what the com­pa­ny calls “the nor­mal range” and well above the 12% com­mon­ly re­gard­ed as suf­fi­cient to sub­stan­tial­ly re­duce spon­ta­neous bleeds.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER