NEW YORK – Intellia Therapeutics on Wednesday said it has received permission from the US Food and Drug Administration to begin a pivotal Phase III trial of NTLA-2001, its investigational gene-editing treatment for transthyretin (ATTR) amyloidosis patients with cardiomyopathy.
Cambridge, Massachusetts-based Intellia plans to kick off a global Phase III study by year-end to evaluate NTLA-2001 as a treatment for the rare, progressive disorder.
NTLA-2001 is an in vivo CRISPR-based gene-editing therapy candidate designed to correct the TTR gene. Mutations in TTR cause the liver to produce abnormal proteins that misfold and build up as amyloid in the body, causing ATTR amyloidosis. NTLA-2001 is administered through a patient's vein, and lipid nanoparticles deliver it to the liver.
The treatment is the first investigational in vivo CRISPR-based gene-editing therapy cleared by the FDA to enter late-stage clinical development, according to Intellia, which is in charge of developing and commercializing NTLA-2001 under a deal with Regeneron Pharmaceuticals.