Hemophilia B Gene Therapy AMT-061 Prevents Bleeds After 1 Year

Hemophilia B Gene Therapy AMT-061 Prevents Bleeds After 1 Year

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Hemophilia B Gene Therapy AMT-061 Prevents Bleeds After 1 Year

After one year, the investigational gene therapy AMT-061 (etranacogene dezaparvovec) continued to safely and effectively increase factor IX (FIX) activity and prevent bleeds in men with moderate to severe hemophilia B, according to new data from the Phase 3 HOPE-B trial. The men participating in the study did not need preventive treatment, the one-year data showed, raising hope for uniQure, the therapy’s developer, that AMT-061 will have long-term benefit. The trial findings will be presented…

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