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Signs Of Long-Term Efficacy Boost UniQure’s Hem B Gene Therapy

Will Await Main Study’s One-Year Milestone Before Submission

 
• By Andrew McConaghie

Unveiling its latest data, UniQure is tipped to avoid the knockback which affected BioMarin’s hemophilia A gene therapy, with a filing expected in the second half of 2021.

uniQure
UniQure expects to file for approval in the second half of 2021.

uniQure has presented promising pivotal data for its gene therapy for hemophilia B, AMT-061, putting it on course for a filing with regulators in the second half of 2021.

The trial’s principal investigator Stephen Pipe of the University of Michigan said patients’ hemophilia B had “thus far” been “transformed into a functionally

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