FDA Grants RMAT Status to Intellia’s CRISPR Therapy for ATTR-CM

March 26, 2025 – Intellia Therapeutics, Inc. (NASDAQ: NTLA) has secured a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for nexiguran ziclumeran (NTLA-2001), a CRISPR-based treatment for transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM). The designation expedites regulatory review, providing early FDA interactions and potential priority review to accelerate patient access. John Leonard, M.D., CEO of Intellia, emphasized that nex-z has demonstrated unprecedented, durable reductions in serum TTR after a single dose, impacting key disease markers.

The RMAT designation, part of the 21st Century Cures Act, is granted to promising therapies targeting serious diseases. Nex-z has also received Orphan Drug Designations from the FDA and European Commission, reinforcing its potential as a transformative one-time treatment. With this milestone, Intellia advances closer to bringing CRISPR-based precision medicine to patients with ATTR-CM, marking a significant leap forward in gene editing therapeutics.