Intellia Therapeutics halted dosing and enrollment in two late-stage CRISPR gene‑editing trials after a participant was hospitalized with severe liver injury. The company stopped screening and dosing while investigators and regulators assess causality and the mechanism behind the hepatic event. Intellia has previously advanced CRISPR programs with relatively few serious safety signals, making this pause notable for competitors and investors who track in‑vivo editing safety. The safety concern centers on on‑target or delivery‑related liver toxicity, a known risk for systemically delivered gene‑editing medicines. Sponsors and regulators will look for patterns across formulations, dosing, and patient comorbidities to determine whether protocol changes or wider holds are required. Market reaction was immediate, with Intellia shares plunging on the announcement, underlining investor sensitivity to clinical‑stage safety events. Companies developing systemic CRISPR and liver‑targeted genetic therapies should expect tightened scrutiny and potential protocol amendments. Clinical operations teams will be watching how Intellia and regulators balance patient safety with the need to preserve trial integrity and timelines.
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