MB-111

MB-111 is an investigational in vivo gene therapy developed by Mammoth Biosciences. It utilizes CasPhi, an ultracompact CRISPR gene editing system encapsulated in a lipid nanoparticle for intravenous delivery to the liver. The therapy is designed to permanently disrupt the expression of the APOC3 gene in hepatocytes, thereby reducing production of apolipoprotein C-III (ApoC-III), a key regulator of lipid metabolism. By lowering ApoC-III levels, MB-111 aims to reduce plasma triglycerides and provide a one-time curative treatment for patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS). As of May 2024, it is in preclinical development[3][4].