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Hemophilia B Gene Therapy Market PRIMEd In Europe

Executive Summary

UniQure’s gene therapy, AMT-060, has been accepted into the European Medicines Agency’s priority medicines scheme, PRIME, a move likely to intensify the race to market of a small group of gene therapies for hemophilia B, all currently in Phase I/II studies.

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UniQure Hemophilia B Gene Therapy AMT-061 Has Early Phase IIb Success

Early positive data from a Phase IIb dose-confirmation study showed AMT-061 achieved and sustained therapeutic levels of Factor IX (FIX) activity in all three participating patients at six weeks after a single administration of the investigational AAV5-based gene therapy.

UniQure Hemophilia B Gene Therapy Changes Ignite A Race To Market

UniQure is switching the gene therapy it plans to move into a pivotal trial in hemophilia B patients because the new version could have clinical benefits. Regulatory agencies in the US and Europe have given the change a green light, and uniQure plans to begin a pivotal trial in 2018.

Blood Disorders Overtake Cancer On EMA’s PRIME Scheme

uniQure has become the third company to get accepted on the European Medicines Agency’s PRIME (priority medicines) scheme for a gene therapy for hemophilia, making hematology-hemostaseology the most common therapeutic areas for products on the scheme.

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