- Interim data from the cardiomyopathy arm of the Phase 1 study of NTLA-2001 showed deep and sustained mean serum transthyretin (TTR) reductions of 93% and 92% at 0.7 mg/kg and 1.0 mg/kg doses, respectively, at day 28
- NTLA-2001 was generally well-tolerated at both dose levels
- Intellia to discuss data at investor event today, Friday, September 16, at 8:00 a.m. ET
CAMBRIDGE, Mass. and TARRYTOWN, N.Y., Sept. 16, 2022 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) today announced positive interim results from an ongoing Phase 1 clinical trial of NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome editing therapy in development as a single-dose treatment for transthyretin (ATTR) amyloidosis. The interim data include 12 adult patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) with New York Heart Association (NYHA) Class I – III heart failure. Single doses of 0.7 mg/kg and 1.0 mg/kg of NTLA-2001 were administered via intravenous infusion, and the change from baseline in serum transthyretin (TTR) protein concentration was measured for each patient.
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