Intellia gets first FDA OK for late-stage trial of in vivo CRISPR-based therapy

Oct. 19, 2023

Intellia Therapeutics announced this week that the FDA has cleared its Investigational New Drug (IND) application for its CRISPR-based gene therapy, NTLA-2001.

NTLA-2001 is the first-ever investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. The treatment is designed to target transthyretin (ATTR) amyloidosis with cardiomyopathy, a rare and severe condition. ATTR amyloidosis is a progressive and fatal disease characterized by the buildup of abnormal protein in the body. With the recent regulatory clearance, Intellia expects to kick off a global phase 3 study for NTLA-2001 by the end of 2023.

NTLA-2001 represents the first investigational CRISPR therapy to be administered systemically, allowing gene editing directly within the human body via intravenous delivery. Intellia deploys a unique non-viral platform employing lipid nanoparticles to transport a two-part genome editing system to the liver: guide RNA targeting the disease-associated gene and messenger RNA encoding the Cas9 enzyme for precise gene modification. Preclinical and clinical data has demonstrated long-lasting reductions in TTR, highlighting the hope of NTLA-2001 as a one-time treatment. 

The drug was developed with Regeneron, as a part of a multi-year CRISPR/CAS collaboration. The partners first began working together in 2016 when Intellia received a $75 million upfront payment and in exchange Regeneron gained exclusive rights to discover and develop CRISPR-based products against up to 10 targets.