NTLA Genome-Editing Therapy Gets FDA's RMAT Tag for New Indication

Intellia Therapeutics (NTLA) announced that the FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational in vivo genome-editing candidate, Nexiguran ziclumeran (nex-z, also known as NTLA-2001), for a new indication.The regulatory body has now granted the RMAT designation to nex-z for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM).The RMAT designation provides the candidate with increased opportunities to meet FDA officials, as well ...