NTLA-2001 (Nexiguran Ziclumeran): A Comprehensive Clinical and Scientific Review of a CRISPR-Based Therapy for Transthyretin Amyloidosis

I. Executive Summary

NTLA-2001, also known by the non-proprietary name nexiguran ziclumeran (nex-z), is a first-in-class, investigational gene-editing therapeutic agent designed to address the underlying cause of transthyretin amyloidosis (ATTR). Developed through a collaboration between Intellia Therapeutics and Regeneron Pharmaceuticals, NTLA-2001 leverages the Nobel Prize-winning CRISPR/Cas9 technology to achieve permanent inactivation of the transthyretin (TTR) gene within hepatocytes.[1] The therapy is administered as a single intravenous infusion, with the objective of providing a "one-and-done," potentially curative treatment for this rare, progressive, and fatal disease.[3] By permanently halting the production of the pathogenic TTR protein, NTLA-2001 offers the possibility of stopping and even reversing the debilitating complications of ATTR amyloidosis.[5]