Intellia announces FDA granted RMAT designation to nexiguran ziclumeran

Intellia Therapeutics (NTLA) announced that the U.S. Food and Drug Administration, FDA, has granted Regenerative Medicine Advanced Therapy, RMAT, designation to nexiguran ziclumeran for the treatment of hereditary transthyretin amyloidosis with polyneuropathy, ATTRv-PN. Development and commercialization of nex-z is led by Intellia as part of a multi-target collaboration with Regeneron (REGN). “This RMAT designation underscores the transformative potential of nex-z, our investigational in vivo CRISPR-based gene editing therapy for those living with hereditary ATTR amyloidosis with polyneuropathy,” said Intellia President and Chief Executive Officer John Leonard, M.D. “It was granted following the FDA’s review of our compelling interim Phase 1 data that indicated our one-time treatment led to rapid, deep and durable TTR reduction, which is expected to halt and potentially reverse the disease. We look forward to working closely with the FDA to bring this potential paradigm-shifting therapy to patients as quickly as possible.”