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    Home > Active Ingredient News > Study of Nervous System > Intellia announces latest clinical follow-up data, in vivo CRISPR gene editing therapy has made further progress

    Intellia announces latest clinical follow-up data, in vivo CRISPR gene editing therapy has made further progress

    • Last Update: 2022-09-09
    • Source: Internet
    • Author: User
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    August 28, 2022 / eMedClub News / -- Intellia Therapeutics and Regeneron Pharma Announce Phase 1 of their In vivo Genome Editing Candidate NTLA-2001 Single Dose for the Treatment of Transthyretin (ATTR) Amyloidosis Interim data from the experimental trial




    ATTR is a hereditary, progressively debilitating and fatal disease caused by mutations in the TTR gene



    NTLA-2001, as an in vivo gene editing therapy for ATTR amyloidosis, opens up a whole new treatment modality for patients




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