UniQure’s (QURE) AAV5-enabled AMT-060 looks good in its first human testing to date, with four of five Hemophilia B patients in the low-dose cohort fully discontinuing prophylactic recombinant Factor IX therapy within 12 weeks of treatment. Two patients with more than 12 weeks of follow-up (12 and 20) are generating Factor IX expression of 4.5% and 5.5% of normal respectively, within the range expected to be therapeutically relevant. Prophylactic FIX discontinuation is the ultimate measure of success, and 4 of 5 patients (80%) achieving this milestone is a great outcome for AMT-060. It appears that AMT-060 is effectively transducing hepatocytes.
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