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Intellia Therapeutics Begins In Vivo Gene-Editing Therapy Dosing in Pivotal ATTR Amyloidosis Trial

NEW YORK – The first patient with transthyretin (ATTR) amyloidosis-associated cardiomyopathy has received Intellia Therapeutics' investigational in vivo CRISPR gene-editing therapy in a pivotal Phase III trial, the firm said Monday.

In the global, randomized-controlled Phase III MAGNITUDE study, investigators are assessing the efficacy and safety of NTLA-2001 as a treatment for ATTR amyloidosis cardiomyopathy. This rare and progressive disease is caused by abnormal and misfolded TTR proteins that build up as amyloid in the body and lead to complications in the heart, nerves, and digestive systems, and eventually heart failure.

NTLA-2001, which was developed as part of a multitarget discovery, development, and commercialization collaboration between Intellia and Regeneron, is designed as a single-dose treatment that inactivates the TTR gene encoding the eponymous protein.

Cambridge, Massachusetts-based Intellia, which leads development and commercialization activities for NTLA-2001, plans to enroll about 765 patients with ATTR amyloidosis-associated cardiomyopathy into the MAGNITUDE trial. Researchers will evaluate the treatment's efficacy based on a composite endpoint comprising cardiovascular-related mortality and events. 

Intellia previously said NTLA-2001 was the first investigational in vivo CRISPR-based gene-editing therapy cleared by the FDA for late-stage clinical development.

"We look forward to evaluating the efficacy and safety of NTLA-2001 in patients with cardiomyopathy in the pivotal Phase III trial," Intellia President and CEO John Leonard said in a statement. "Assuming a positive outcome, it will pave the way for future global marketing applications, ultimately supporting our goal to bring forth a groundbreaking therapy for the ATTR amyloidosis community."