The CRISPR gene‑editing sector posted sharply divergent headlines: CRISPR Therapeutics presented early clinical data showing a single in vivo gene‑editing therapy produced large reductions in cholesterol and triglycerides, while Intellia reported a patient death in its MAGNITUDE Phase III program and faces further regulatory scrutiny. Companies said both signals will prompt detailed safety and mechanism reviews. CRISPR Therapeutics’ cholesterol program—presented at AHA—reported robust pharmacodynamic effects consistent with a potential once‑and‑done treatment for hyperlipidemia; those data have reignited investor interest in in vivo editing. At the same time, Intellia and regulators are assessing a serious liver injury linked temporally to dosing in a Phase III study, triggering a clinical hold review. The juxtaposition underscores the modality’s clinical promise and the acute safety vigilance required for systemic, in‑vivo editing approaches. Biotech leaders warned that progression to larger trials will hinge on careful risk‑benefit evaluation and regulatory dialogue.
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