In Brief: uniQure AMT-130 Phase I/II Trial update

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Continued progress in uniQure AMT-130 Phase I/II Trial

R. David Cooper

The first HD gene therapy trial using adeno-associated virus (AAV) vector delivery is now months into Phase I/II and reporting a good safety profile with all participants to date. uniQure says that their enrollment in the first cohort, which uses a lower dose, was completed in May, with six patients treated with AMT-130, and four patients receiving the imitation surgery. Following a positive recommendation by an independent Data Safety Monitoring Board (DSMB), the second, higher dose cohort of the trial has begun, with four patients enrolled as of the end of August.

“We continue to be very pleased with the enrollment of this important study and look forward to having the trial fully enrolled by the middle of next year,” said Dr. David Cooper, VP of Clinical Development at uniQure. “We also look forward to sharing preliminary imaging and biomarker data from initial patients in the U.S. clinical trial before the end of the year and initiating a separate open-label study of AMT-130 in Europe later this year.”

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