Intellia Therapeutics paused enrollment and dosing in two late‑stage trials of its CRISPR therapy nex‑z after a trial volunteer was hospitalized with severe liver injury. The company stopped activity while it investigates the event and implements enhanced safety monitoring; the halt applies to trials in transthyretin amyloidosis (ATTR) with cardiomyopathy and polyneuropathy. Regulatory and investor attention arrived rapidly: Intellia’s shares plunged on the news and commentators noted prior liver‑toxicity signals across some in‑body gene‑editing programs. Company statements stressed a safety‑first posture while teams analyze causality and potential protocol adjustments. The pause raises broader questions about in vivo CRISPR tolerability in diseases with existing effective therapies. Industry groups and investors will watch Intellia’s safety review and any subsequent regulatory guidance for implications across gene‑editing development.
Get the Daily Brief