Standfirst: A hospitalised patient with a Hy’s-law-like profile has forced Intellia to pause two late-stage CRISPR studies in ATTR amyloidosis. The central question for investors: product-specific toxicity or a broader platform overhang?
What happened — and why it matters
Intellia Therapeutics has temporarily paused dosing and screening in its two Phase 3 MAGNITUDE trials of nexiguran ziclumeran (nex-z; formerly NTLA-2001) after a participant in the cardiomyopathy study developed Grade 4 transaminase elevations with increased total bilirubin and was hospitalised. The event, reported on October 24 in a patient dosed September 30, triggered the protocol’s pausing criteria.
