Intellia Therapeutics Receives Key FDA Clearance for Gene Editing Trial

The U.S. Food and Drug Administration (FDA) has removed a clinical hold on a pivotal late-stage study conducted by Intellia Therapeutics. This clearance, granted on January 27, allows the company to resume its MAGNITUDE-2 trial for the gene-editing candidate Nexiguran Ziclumeran (nex-z). The development marks a significant step forward, though investors are now focused on the pace at which the program can regain momentum.

This regulatory green light follows an agreement between Intellia and the FDA on amended study protocols. Key modifications include enhanced safety monitoring of liver function. Furthermore, the biotech firm intends to expand its target enrollment from approximately 50 to 60 participants.

The therapy, which utilizes CRISPR-based technology, is being evaluated for the treatment of patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). Company officials stated that patient recruitment will recommence at the earliest possible opportunity.

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One Hold Remains in Place

The agency had initially placed partial clinical holds on two of Intellia's Phase 3 studies in October 2025. This action was prompted by a serious liver-related adverse event occurring in a patient enrolled in the parallel MAGNITUDE trial. That separate study is investigating the same therapeutic candidate for a different indication: transthyretin amyloidosis with cardiomyopathy (ATTR-CM).

While the hold on the polyneuropathy study has now been lifted, discussions with regulators concerning the cardiomyopathy trial are ongoing. Intellia has indicated it will provide a further update only after the path forward for that study is determined.

Pipeline and Upcoming Milestones

Attention within Intellia's development portfolio extends beyond the nex-z program. Results from the Phase 3 HAELO study for a separate drug candidate are anticipated around mid-2026. The company's next quarterly financial report is expected to be released on or around February 26, 2026.