A first phase I clinical trial designed to evaluate the safety and pharmacodynamics effects of NTLA-2001 in six patients with ATTRv-PN with or without cardiomyopathy showed that after 28 days, a single dose of NTLA-2001 led to a dose-dependent decrease in serum TTR ranging from 47% to 96% with only mild adverse events [141]. Here, TTR is linked to cardiomyopathy.