FDA Lifts Clinical Hold on Intellia’s Phase 3 CRISPR Trial for Hereditary Nerve Disease

CAMBRIDGE, MA —January 27, 2026— Intellia Therapeutics (NASDAQ: NTLA) announced today that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on its MAGNITUDE-2 Phase 3 trial, clearing the way for the company to resume dosing and enrollment for its CRISPR-based candidate, nexiguran ziclumeran (nex-z). The trial focuses on treating hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a progressive and debilitating condition caused by the accumulation of misfolded proteins in the nerves.

The FDA originally suspended the program in late 2025 following reports of severe liver toxicity in a patient participating in a parallel study. To address these safety concerns, Intellia has implemented protocol modifications that include enhanced monitoring of liver laboratory tests. Additionally, the target enrollment for the MAGNITUDE-2 study has been increased from 50 to approximately 60 patients to further strengthen the safety and efficacy data set. The trial utilizes a randomized, double-blind, placebo-controlled design to measure changes in neuropathy impairment and serum TTR levels.

While the polyneuropathy trial moves forward, the FDA’s clinical hold remains in effect for the MAGNITUDE Phase 3 trial involving patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). Intellia stated that engagement with the agency is ongoing to determine the path forward for the cardiomyopathy program. Nex-z is designed as a one-time treatment that uses CRISPR/Cas9 delivered via lipid nanoparticles to permanently inactivate the TTR gene in the liver, potentially halting the production of the toxic proteins that drive both nerve and heart damage.

Intellia President and CEO John Leonard, M.D., expressed appreciation for the FDA’s review, noting that the team is focused on resuming activities as quickly as possible. As the first in vivo CRISPR therapy to reach this stage of development, the progress of nex-z is closely watched by the medical community as a potential landmark in genetic medicine for rare diseases.