CAMBRIDGE, Mass., March 26, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) 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.”
The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including genetic therapies, that are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation includes benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product’s biologics license application (BLA).
Nex-z has been granted Regenerative Medicine Advanced Therapy designations by the U.S. FDA for both cardiomyopathy and polyneuropathy. Nex-z has also been granted Orphan Drug Designation by the U.S. FDA and European Commission.
About nexiguran ziclumeran (nex-z, also known as NTLA-2001)
Based on Nobel Prize-winning CRISPR/Cas9 technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is an investigational in vivo CRISPR-based therapy designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron.