In a PhI­II show­down with Spark, uniQure switch­es out its lead gene ther­a­py for he­mo­phil­ia B

Late last year, the strug­gling gene ther­a­py biotech uniQure $QURE did some painful cost cut­ting, drop­ping pro­grams, ax­ing staff and tai­lor­ing the pipeline to con­cen­trate on a he­mo­phil­ia B pro­gram for AMT-060, which they said could be hus­tled through a piv­otal pro­gram and in­to the mar­ket, rac­ing with a ri­val ther­a­py from in­dus­try leader Spark Ther­a­peu­tics.

This morn­ing, though, uniQure ex­e­cut­ed a quick switch on its lead ef­fort. AMT-060, which had been des­ig­nat­ed as a break­through drug at the FDA, has been re­placed by AMT-061. And the ex­ecs at uniQure say that both the FDA and the EMA have es­sen­tial­ly de­cid­ed to keep this new drug in their VIP drug pro­gram and on the fast track to a reg­is­tra­tion tri­al.

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