The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to the investigational gene therapy, AMT-060 (uniQure), for the treatment of severe hemophilia B.

The designation is supported by data from the ongoing, dose-ranging Phase 1/2 trial of AMT-060 in patients with severe hemophilia B. Results demonstrated that treatment with AMT-060 is associated with sustained increases in Factor IX (FIX), reductions in FIX replacement usage and a near cessation of spontaneous bleeding at up to 12 months follow-up. The trial also found AMT-060 to be well-tolerated, with no severe adverse events observed. Updated study data were recently presented at the 58th American Society of Hematology (ASH) Annual Meeting. 

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AMT-060 is an investigational gene therapy consisting of an AAV5 viral vector carrying a therapeutic fully-humanized Factor IX gene cassette. The AAV5 vector has shown particular affinity for reaching the liver and has a low prevalence of pre-existing anti-AAV5 antibodies.

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