Intellia Therapeutics, Inc. (NTLA), Wednesday announced that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy designation to nexiguran ziclumeran, also known as nex-z or NTLA-2001, for the treatment of transthyretin or TTR amyloidosis with cardiomyopathy.
The designation was based on the findings of interim Phase 1 clinical data, which demonstrated that the administration of nex-z led to consistent, deep and long-lasting TTR reduction.
Nex-z has been granted the designations by the U.S. FDA for both cardiomyopathy and polyneuropathy, where it showed unprecedented rapid, durable and consistent reductions in serum TTR after a single dose.
Nex-z has also been granted Orphan Drug Designation by the U.S. FDA and European Commission.
In the pre-market hours, Intellia's stock is trading at $8.90, up 1.95 percent on the Nasdaq.
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