Intellia Therapeutics announces FDA RMAT designation to nex-z

Intellia Therapeutics (NTLA) announced that the U.S. Food and Drug Administration, FDA, has granted Regenerative Medicine Advanced Therapy, RMAT, designation to nexiguran ziclumeran, nex-z, for the treatment of transthyretin, ATTR, amyloidosis with cardiomyopathy, ATTR-CM. “This is a meaningful step forward in our efforts to accelerate the development of nex-z to bring this potentially transformative treatment to patients as quickly as possible,” said Intellia President and Chief Executive Officer John Leonard, M.D. “In both cardiomyopathy and polyneuropathy, nex-z has been shown, after a single dose, to lead to unprecedented rapid, durable and consistent reductions in serum TTR, which positively impacts markers of disease progression.”