Skip to main content

Intellia Presents Updated Interim Data from the Cardiomyopathy Arm of Ongoing Phase 1 Study of NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis at the American Heart Association Scientific Sessions 2022

Intellia Therapeutics Logo

November 5, 2022

  • Data presented in late-breaking oral presentation demonstrated deep and consistent TTR reduction following a single dose of NTLA-2001 in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM)
  • Greater than 90% mean serum TTR reductions after a single dose of NTLA-2001 were sustained at both doses tested, with follow-up now reaching four to six months
  • NTLA-2001 was generally well-tolerated

CAMBRIDGE, Mass., Nov. 05, 2022 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today presented additional 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 in collaboration with Regeneron Pharmaceuticals. Results were presented in a Late-Breaking Science oral presentation at the American Heart Association (AHA) Scientific Sessions 2022, held November 5 – 7 in Chicago, Illinois.

The interim data from the dose-escalation portion of the Phase 1 study include 12 adult patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) with New York Heart Association (NYHA) Class I – III heart failure. The data presented were as of a data cutoff date of August 25, 2022. Single doses of 0.7 mg/kg and 1.0 mg/kg of NTLA-2001 were administered via a single intravenous infusion, and the change from baseline in serum transthyretin (TTR) protein concentration was measured for each patient.

Read the full article here