- Data presented in the latest oral presentation showed a deep and consistent TTR reduction after a single dose of NTLA-2001 in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM)
- Greater than 90% of mean serum TTR reductions after a single dose of NTLA-2001 were maintained at both doses tested, with follow-up now reaching four to six months
- NTLA-2001 was generally well tolerated
CAMBRIDGE, Mass., Nov. 5, 2022 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. NTLAa leading clinical-stage genome editing company focused on developing potentially curative therapeutics utilizing CRISPR-based technologies, today presented additional interim results from an ongoing Phase 1 clinical trial of NTLA-2001, an investigational drug in vivo CRISPR/Cas9 genome editing therapy in development in collaboration with Regeneron Pharmaceuticals as a single-dose treatment for transthyretin (ATTR) amyloidosis. The findings were presented in an oral presentation by Late-Breaking Science at the American Heart Association (AHA) Scientific Sessions 2022, held November 5-7 in Chicago, Illinois.
Preliminary 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. Data presented is as of the data cut-off 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…
