Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Nexiguran Ziclumeran (nex-z) for the Treatment of Hereditary Transthyretin (ATTR) Amyloidosis with Polyneuropathy

Core Insights - The U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Intellia Therapeutics' investigational therapy nexiguran ziclumeran (nex-z, also known as NTLA-2001) for treating hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN) [1][2] - Nex-z is designed as a single-dose CRISPR-based therapy aimed at inactivating the TTR gene to prevent TTR protein production, potentially halting and reversing the disease [1][4] - This RMAT designation is the third special regulatory designation received by Intellia for nex-z, which also holds Orphan Drug Designation from the FDA and the European Commission [3] Company Overview - Intellia Therapeutics is a clinical-stage gene editing company focused on CRISPR-based therapies, with programs that enable precise editing of disease-causing genes in vivo and ex vivo [6] - The company collaborates with Regeneron for the development and commercialization of nex-z as part of a multi-target initiative [1][4] Disease Context - ATTR amyloidosis is a rare and progressive disease caused by mutations in the TTR gene, leading to the production of misfolded TTR proteins that accumulate and cause serious complications [5] - There are approximately 50,000 individuals globally with hereditary ATTR amyloidosis and between 200,000 to 500,000 with wild-type ATTR amyloidosis, with no known cure currently available [5]