Intellia and Regeneron Announce Latest Phase 1 Clinical Trial Data of NTLA-2001

On March 1, Intellia Therapeutics and Regeneron Pharmaceutical announced the latest phase 1 clinical trial data of their in vivo genome editing candidate therapy NTLA-2001

The disease targeted by NTLA-2001 is transthyretin amyloidosis (ATTR)

As an innovative therapy for in vivo gene editing, NTLA-2001 delivers the CRISPR gene editing system targeting the TTR gene into the human body through lipid nanoparticles (LNP), which can specifically knock out the TTR gene in the liver, thereby reducing the The expression of TTR protein reduces the accumulation of pathogenic proteins

▲The mechanism of action of NTLA-2001 (Image source: Reference [2])

Today's news releases more data from the clinical trial to the industry -- data from 15 patients who were given intravenous infusions of NTLA-2001 in four different dose groups

After 28 days of treatment

▲The role persistence data of NTLA-2001 (Image source: Intellia's official website)

In all four dose groups, NTLA-2001 was generally well tolerated, with the most important adverse reactions including headache, infusion-related reactions, back pain, rash, and nausea

According to the plan of the clinical trial, the researchers will further expand the treatment group in the first quarter of this year

References:

[1] Intellia and Regeneron Announce Updated Phase 1 Data Demonstrating a Single Dose of NTLA-2001, Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis, Resulted in Rapid, Deep and Sustained Reduction in Disease-Causing Protein, Retrieved February 28, 2022, from https:// -for-transthyretin-attr-amyloidosis-resulted-in-rapid-deep-and-sustained-reduction-in-disease-causing-301491764.

[2] Gillmore et al.

(Original abridged)