Intellia finds genetic marker linked to liver enzyme spikes in CRISPR ATTR amyloidosis trial

Intellia Therapeutics announced that analysis of its ongoing CRISPR gene-editing trial for transthyretin (ATTR) amyloidosis has identified a common genetic variant among participants who experienced the greatest elevations in liver enzymes.

The finding suggests that the variant may predispose patients to hepatic stress when exposed to the gene-editing platform, providing a potential explanation for the safety signals observed.

Researchers said the discovery will guide patient selection and monitoring strategies in future study cohorts, aiming to mitigate the risk while preserving therapeutic benefit.

The company did not disclose the specific gene or frequency of the variant, and the trial continues under close regulatory oversight.