Intellia Therapeutics paused enrollment and dosing in two late‑stage clinical trials of its in vivo CRISPR candidate for transthyretin amyloidosis after a study volunteer was hospitalized with severe liver injury. The company said it is evaluating the event and will implement additional safety measures before resuming dosing. Intellia’s pause affects pivotal readouts and prompted sharp market reactions; the company is reassessing risk mitigation steps and working with investigators and regulators. Company statements and investor calls framed the move as temporary while safety signals are reviewed, but analysts noted the action raises existential questions for in‑body gene‑editing programs given available alternative therapies. This development underscores liver safety as a recurring challenge for systemic gene‑editing approaches and will likely trigger renewed scrutiny from regulators, sponsors and investors about dosing strategies, delivery platforms and monitoring protocols for in vivo editing.
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