NTLA-2001 is an investigational in vivo CRISPR-based gene editing therapy designed to be a single-dose treatment for transthyretin (ATTR) amyloidosis.
Intellia Therapeutics Announced The Acceptance Of An Abstract Featuring Redosing Data From The Phase 1 Study Of NTLA-2001 Has Been Selected For An Oral Presentation At The Peripheral Nerve Society Annual Meeting
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