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Stock Markets March 2, 2026

FDA Lifts Clinical Hold on Intellia’s Late-Stage Gene Therapy Trial, Shares Rise

Intellia stock gains after regulator removes pause on a heart-targeted gene-editing study following mitigation steps

By Priya Menon NTLA
FDA Lifts Clinical Hold on Intellia’s Late-Stage Gene Therapy Trial, Shares Rise
NTLA

Intellia Therapeutics shares climbed in premarket trading after the U.S. Food and Drug Administration lifted a clinical hold on a late-stage trial of nexiguran ziclumeran, an experimental gene-editing therapy for transthyretin amyloid cardiomyopathy (ATTR-CM). The FDA had previously paused two principal late-stage studies following a patient death attributed to severe liver complications. One trial, MAGNITUDE-2, had its hold removed earlier this year after Intellia adopted enhanced liver monitoring and stricter enrollment criteria.

Key Points

  • Intellia shares rose 4.8% to $14.44 in premarket trading after the FDA lifted a clinical hold on a late-stage trial of nexiguran ziclumeran.
  • The FDA had paused two main late-stage studies following a patient death from severe liver complications; mitigation measures were required to resume activity.
  • Sectors impacted include biotechnology and healthcare, as the development and regulatory progress of a gene-editing therapy for heart disease can affect clinical-stage biotech valuations and investor sentiment.

Shares of Intellia Therapeutics (NASDAQ:NTLA) rose 4.8% to $14.44 in premarket trading on Monday after the company said the U.S. Food and Drug Administration removed a clinical hold on one of its late-stage trials for a gene-editing therapy aimed at heart disease.

The therapy, nexiguran ziclumeran, is being evaluated in patients with transthyretin amyloid cardiomyopathy, or ATTR-CM, a progressive heart condition in which abnormal protein deposits accumulate in the heart muscle. This buildup makes the heart stiffer and reduces its ability to pump blood effectively.

The FDA had placed pauses on two main late-stage studies of the treatment after a patient died last year from severe liver complications. Regulators subsequently required additional safety measures and oversight as part of their review of the programs.

Earlier this year, the FDA lifted a clinical hold on one of the trials, MAGNITUDE-2, after Intellia implemented a set of mitigation steps. Those measures included more intensive monitoring of liver laboratory tests and a policy to exclude individuals who present with certain liver abnormalities from enrollment.

Intellia and regulators have focused on reducing the risk of serious liver-related adverse events while continuing to advance evaluation of the therapy in patients with ATTR-CM. The company’s statements indicate the lifted hold followed the introduction of safety protocols designed to detect and respond to liver issues more quickly and to limit exposure among higher-risk patients.

Context and market reaction

Investors reacted to the regulatory update with a modest premarket uptick in Intellia shares. The regulatory development affects the company’s late-stage clinical program and may influence near-term development timelines and monitoring requirements for the therapy.

What we know and what remains uncertain

Regulators removed the hold on at least one of the late-stage trials after the company added safety measures. However, the prior pause was prompted by a patient death tied to severe liver complications, highlighting ongoing safety considerations. The lifted hold applies to trials following the specific mitigation steps described by the company and acknowledged by the FDA.

Risks

  • Serious safety concerns remain relevant: a prior patient death from severe liver complications prompted the initial FDA pause, underscoring clinical risk to patients and development programs.
  • Regulatory uncertainty persists until all required mitigation measures and monitoring prove effective across affected trials.
  • Trial enrollment is now restricted by exclusion criteria for patients with certain liver abnormalities, which may affect trial recruitment and timelines and has implications for clinical-stage biotech operations and capital planning.

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