nexiguran ziclumeran

**Nexiguran ziclumeran** is an investigational, single-administration **in vivo CRISPR-Cas9 genome-editing therapy** being developed by Intellia Therapeutics in collaboration with Regeneron for transthyretin amyloidosis. It uses a lipid nanoparticle to deliver messenger RNA encoding *Streptococcus pyogenes* Cas9 and a transthyretin-targeting single-guide RNA to hepatocytes. The resulting CRISPR-Cas9 editing introduces disruptive insertions or deletions in the hepatic **TTR** gene, durably lowering production of circulating transthyretin protein. It is administered by intravenous infusion and is in Phase 3 development for transthyretin amyloidosis with cardiomyopathy and hereditary transthyretin amyloidosis with polyneuropathy.

Brand names

nex-z

Other names

nex-znexiguran ziclumeran