On November 25, 2024, Intellia Therapeutics announced that its CRISPR-based therapy, nexiguran ziclumeran (nex-z, NTLA-2001), received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN). Nex-z is an in vivo gene editing therapy designed as a single-dose treatment to inactivate…
