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    Home > Active Ingredient News > Drugs Articles > Express Hemophilia B Gene Therapy Receives FDA Priority Review, Reduces Annual Bleeding Rate by 64%

    Express Hemophilia B Gene Therapy Receives FDA Priority Review, Reduces Annual Bleeding Rate by 64%

    • Last Update: 2022-08-15
    • Source: Internet
    • Author: User
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    Today, CSL Behring announced that the.


    If approved, this would be the first gene therapy to treat hemophili.


    People with hemophilia B have mutations in the gene encoding coagulation factor IX (FIX), resulting in a loss or deficiency of F.


    Transgenes expressing FIX Padua variants were delivered using AAV5 vecto.


    The patient's annual bleeding rate (ABR) was 64% lower than in the run-in period before receiving treatment (p=000


    References:

    References:

    [1] FDA Accepts CSL Behring's Biologics License Application for Etranacogene Dezaparvovec for Priority Revi.


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