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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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