The CRISPR field recorded sharply divergent clinical outcomes this week: Intellia disclosed a patient death tied to acute liver injury in its MAGNITUDE Phase III trial and reported it is awaiting FDA action on the study. Intellia said the case is under further evaluation and that the company is cooperating with regulators. Days earlier, CRISPR Therapeutics presented positive early clinical data for an in vivo gene-editing therapy that produced substantial cholesterol and triglyceride reductions, renewing optimism about one-time editing treatments for lipid disorders. Presentations and company statements highlighted both the promise and the safety complexities inherent to in vivo genome editing. Investors and regulators reacted to the juxtaposition: the field’s science is advancing rapidly, but the Intellia fatality underscores the need for robust safety monitoring and caution in scaling in vivo editing programs.
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