Additionally, CRISPR-Cas9 in vivo gene editing for the treatment of TTR-related polyneuropathy has proven safe and effective at reducing TTR protein concentrations through the targeted knockout of the TTR gene with a single infusion of NTLA-2001 in six patients with ATTRv.44 A trial in patients with ATTR-CM is in the planning phases. The gene discussed is TTR; the disease is polyneuropathy.