Intellia Therapeutics Clears Key Regulatory Hurdle for Gene Therapy Trial

The U.S. Food and Drug Administration (FDA) has lifted a clinical hold on a pivotal Phase 3 study conducted by biotechnology firm Intellia Therapeutics. This clearance allows the company to resume patient enrollment for its investigational gene therapy, Nexiguran Ziclumeran. The decision pertains specifically to the MAGNITUDE-2 trial, which is evaluating the treatment for hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).

In late January 2026, Intellia announced the FDA's decision to remove the partial clinical hold. The company is now initiating preparations to restart trial activities. As part of the resumption agreement, Intellia and the regulatory agency have agreed on specific amendments to the study protocol. These modifications include enhanced liver safety monitoring procedures and an increase in the target enrollment from approximately 50 to 60 participants. The removal of this obstacle puts a crucial late-stage development program back on track.

Origin of the Clinical Hold and Current Status

The regulatory agency initially imposed the clinical hold in October 2025. The action affected both the MAGNITUDE-2 study and a related trial named MAGNITUDE, which is testing the same therapeutic candidate for a different condition—transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The hold was triggered by a report of a serious adverse liver event involving a patient enrolled in the MAGNITUDE study.

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While the path is now clear for the ATTRv-PN study (MAGNITUDE-2) to proceed, the clinical hold remains in effect for the ATTR-CM study (MAGNITUDE). According to company statements, Intellia continues to engage in discussions with the FDA regarding the future pathway for that particular trial.

Pipeline Developments and Forward Focus

The restart of the MAGNITUDE-2 trial shifts investor attention toward the collection of additional safety data. Market observers are also monitoring another advanced program in Intellia's pipeline. Topline results from a Phase 3 study for Lonvo-z, a candidate for treating hereditary angioedema (HAE), are anticipated around mid-2026.