Intellia Therapeutics Clears Key FDA Hurdle for Gene-Editing Trial

Investors in Intellia Therapeutics have received a significant, though partial, reprieve. The U.S. Food and Drug Administration (FDA) has lifted a clinical hold on a crucial Phase 3 study, allowing the biotech firm to proceed with a key component of its development program. However, regulatory concerns persist as a separate trial remains suspended.

The focus is on the gene-editing therapeutic candidate, Nexiguran Ziclumeran. The FDA has authorized the resumption of the MAGNITUDE-2 study, which is investigating the treatment for hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). This decision followed Intellia's agreement to implement revised trial protocols designed to address the regulator's safety concerns.

A central modification involves enhanced patient monitoring. Participants will now undergo more frequent liver function testing. Furthermore, Intellia intends to expand the trial's enrollment, increasing the number of participants from 50 to 60. The company aims to commence recruiting and treating new patients at the earliest opportunity.

A Fatal Setback and Lingering Uncertainty

The initial clinical hold, imposed in October 2025, was triggered by a serious adverse event. A patient enrolled in the parallel MAGNITUDE study suffered severe liver toxicity and subsequently died. This tragic incident raised fundamental questions about the safety profile of the CRISPR-based therapy.

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While the path is now clear for the polyneuropathy variant of the disease, the cardiology application (ATTR-CM) faces continued obstacles. The clinical hold on that specific study remains firmly in place. Intellia management is engaged in ongoing discussions with the FDA to chart a potential path forward for this portion of the program.

Institutional Backing and Forward Path

Despite these challenges, major institutional investors have maintained their positions. A regulatory filing dated February 3, 2026, disclosed that ARK Investment Management continued to hold a 12.27% stake in Intellia Therapeutics at the close of 2025.

The latest FDA decision removes a major overhang for the company's shares, but it does not eliminate all risk. Management remains under pressure to reach a similar agreement with regulators concerning the second stalled trial. The market now awaits a promised update on the MAGNITUDE study, expected once discussions with the agency have concluded.