This clinical trial is focused on studying a condition known as Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM). This is a disease where abnormal proteins build up in the heart, leading to heart problems. The study will test a new treatment called NTLA-2001, which is a type of gene therapy. Gene therapy is a treatment that aims to fix or replace faulty genes in the body. NTLA-2001 contains two special RNA substances that work together to target and modify the gene responsible for the disease.

The purpose of the study is to evaluate how effective and safe NTLA-2001 is for people with ATTR-CM. Participants in the study will receive either NTLA-2001 or a placebo through an intravenous infusion, which means the treatment is given directly into a vein. The study will monitor participants over time to see how the treatment affects their heart health, specifically looking at the risk of heart-related deaths and events.

Throughout the study, researchers will also track changes in certain health markers, such as the level of a protein called transthyretin in the blood, and assess participants’ quality of life. The study is designed to provide valuable information about whether NTLA-2001 can help improve the condition of people with ATTR-CM and potentially offer a new treatment option for this challenging disease.