In the context of cardiac amyloidosis, NTLA-2001 (Nexiguran ziclumeran or nex-z), a CRISPR-Cas9-based gene-editing therapy, demonstrated an 89% reduction in serum transthyretin levels at 28 days and a 90% reduction at 12 months during Phase 1 trials. The gene discussed is TTR; the disease is cardiac amyloidosis.