In a pivotal pilot study by Gillmore et al., NTLA-2001 (also known as nexiguran ziclumeran), a CRISPR-Cas9 therapy targeting the TTR gene, was administered via a lipid nanoparticle (LNP) delivery system designed to edit TTR genes in hepatocytes, in six patients with hereditary TTR amyloidosis with polyneuropathy. The gene discussed is TTR; the disease is familial amyloid neuropathy.