Clinical hold on uniQure's hemophilia B gene therapy program

Netherlands-based gene therapy company uniQure (Nasdaq: QURE) saw its shares down 15.7% at $38.75 around midday in New York trading today, after it announced that its hemophilia B gene therapy program, including the pivotal, Phase III HOPE-B study, has been placed on clinical hold by the US Food and Drug Administration.

Patient dosing is complete in each of uniQure’s three hemophilia B gene therapy studies, and there is no plan to enroll or treat additional patients. Earlier this month, uniQure presented promising pivotal data on its hemophilia B gene therapy etranacogene dezaparvovec at the ASH meeting.

Hold related to possibly related serious adverse event