Intellia begins phase 3 trial for CRISPR therapy NTLA-2001

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Written By Pinang Driod
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© Reuters.

CAMBRIDGE, Mass. – Intellia Therapeutics, Inc. (NASDAQ:), a pioneer in gene editing treatments, has started its Phase 3 MAGNITUDE trial for NTLA-2001, marking a significant step in developing a potential one-time therapy for transthyretin (ATTR) amyloidosis patients. The first patient received a dose today, signaling the commencement of a study that could reshape the treatment landscape for this fatal condition.

NTLA-2001, crafted using the CRISPR/Cas9 technology, aims to inactivate the TTR gene to halt the production of the disease-causing TTR protein. ATTR amyloidosis leads to heart failure and other severe complications, with a growing number of diagnoses each year. The MAGNITUDE trial, which is randomized, double-blind, and placebo-controlled, will assess the efficacy and safety of NTLA-2001 in roughly 765 patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM).

During the trial, adult patients with either hereditary or wild type ATTR-CM will be randomized in a 2:1 ratio to receive a single 55 mg infusion of NTLA-2001 or a placebo. The primary endpoint is a composite of cardiovascular-related mortality and events. Interim Phase 1 results have already shown promising reductions in the TTR protein after a single dose of NTLA-2001.

John Leonard, M.D., President and CEO of Intellia, expressed optimism about the trial’s potential impact, emphasizing the rapid patient enrollment across multiple sites, including the U.S. Julian Gillmore, M.D., Ph.D., the study’s national coordinating investigator, echoed this sentiment, highlighting the patient community’s significant interest in a single-dose treatment option.

Intellia Therapeutics is spearheading the development and commercialization of NTLA-2001 in collaboration with Regeneron (NASDAQ:). The company’s mission is to revolutionize medicine through in vivo and ex vivo CRISPR-based therapies, targeting diseases directly inside the human body or by engineering human cells outside the body.

The information for this article is based on a press release statement from Intellia Therapeutics, Inc.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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