Bolstered by promising PhIIb snapshot, uniQure plays catch-up with Spark in the race to a hemophilia B gene therapy
The pivotal lead switch that uniQure executed late last year appears to have paid off, as the gene therapy pioneer offers some positive, if early, numbers for its tweaked hemophilia B treatment.
Among three patients treated with AMT-061 in the dose-confirmation study, researchers reported a mean factor IX level that represented 31% of normal levels at six weeks, exceeding the 12% commonly regarded as sufficient to substantially reduce spontaneous bleeds. A quick breakdown by individual showed FIX activity in the first patient was 37% of normal 10 weeks after administration; in the second patient, 23% at week 8; and the third, 30% at week 6.
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