Intellia pauses two late-stage CRISPR gene-editing trials after emergence of safety concern

Intellia Therapeutics said Monday it is pausing two late-stage CRISPR gene-editing trials after a patient in one study was hospitalized with liver damage. 

The company’s stock plummeted nearly 50% in pre-market trading. 

Liver damage has been a known risk for many CRISPR-based medicines in development, in part because they often involve trying to knock out or modify a gene in that organ. In 2024, Verve Therapeutics shelved its lead program for heart disease and moved to a backup molecule, in part over liver safety concerns.

Intellia, though, had encountered few serious safety concerns, despite dosing hundreds of patients across its clinical trials — more than any other company attempting to edit genes directly in patients. 

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