MGX-006

MGX-006 is an investigational *in vivo* CRISPR-based gene editing therapeutic candidate developed for the treatment of transthyretin amyloidosis (ATTR). Developed by Metagenomi, the therapy utilizes a proprietary, metagenomics-derived CRISPR-associated (Cas) nuclease delivered via lipid nanoparticles (LNPs) to the liver. The mechanism of action involves the targeted disruption of the *TTR* gene in hepatocytes, which significantly reduces the production of both wild-type and mutant transthyretin proteins. By lowering circulating TTR levels, MGX-006 aims to prevent the formation and deposition of amyloid fibrils that cause progressive organ damage in patients with ATTR polyneuropathy or cardiomyopathy. This approach offers the potential for a one-time curative treatment by permanently silencing the source of the toxic protein.