Intellia Therapeutics presented updated interim data from an ongoing phase 1 clinical trial of NTLA-2001 showing a reduction of greater than 90% in mean serum transthyretin (TTR) of patients with transthyretin amyloidosis (ATTR) with cardiomyopathy after a single-dose treatment.

The trial evaluates NTLA-2001 in adults with ATTR with cardiomyopathy or hereditary ATTR (hATTR) with polyneuropathy.

Interim results were presented for the first time in a late-breaking oral presentation at the American Heart Association (AHA) Scientific Sessions 2022. “This presentation at the AHA Scientific Sessions marks the first time Intellia has had the opportunity to share interim data from our landmark clinical trial of NTLA-2001 for the treatment of ATTR amyloidosis with the cardiology community,” said John Leonard, MD, president and chief executive officer of Intellia. “We believe a single dose of NTLA-2001 has the potential to halt and potentially reverse this life-threatening disease. With the depth and consistency of TTR protein reduction seen with NTLA-2001 thus far, we envision a future where physicians and patients can make treatment decisions based on a therapy’s ability to achieve specific threshold protein levels. We look forward to progressing this first-ever systemically administered in vivo CRISPR investigational therapy toward completion of the phase 1 study and subsequent pivotal studies in collaboration with our partners at Regeneron.”

The results showed deep reductions in serum TTR at day 28, which were sustained through the observation period (follow up, 4-6 months). Moreover, NTLA-2001 was generally well-tolerated. The only observed treatment-related adverse events were transient infusion reactions, which were reported by 2 patients (of 12).

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Participants received single 0.7 and 1.0 mg/kg doses of intravenous NTLA-2001 during the dose-escalation portion of the phase 1 study.

Intellia plans to enroll both arms of the trial by the end of 2022.


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. News release. Intellia Therapeutics, Inc.; November 5, 2022.