STX-1400

STX-1400 is an investigational CRISPR-based gene editing therapy developed by Scribe Therapeutics for the treatment of hypertriglyceridemia, specifically Familial Chylomicronemia Syndrome (FCS) and severe hypertriglyceridemia (sHTG). It utilizes Scribe's proprietary XE platform, which employs a CasX-based gene editor delivered via lipid nanoparticles (LNPs) to target hepatocytes. The therapy is designed to permanently disrupt the *APOC3* (Apolipoprotein C-III) gene, thereby knocking out its hepatic expression. By permanently reducing APOC3 levels, STX-1400 aims to provide a durable, single-dose treatment for lowering serum triglycerides. Preclinical data in non-human primates and mouse models have demonstrated high editing efficiency (over 75%) and significant reductions in both APOC3 protein and triglyceride levels (over 90%) with high specificity and no detectable off-target effects at therapeutic doses.