STX1150

STX1150 is an investigational gene therapy developed by Scribe Therapeutics for the treatment of hyper LDL cholesterolaemia and atherosclerotic cardiovascular disease. It utilizes a highly engineered CRISPR-CasX-based epigenetic silencer delivered via lipid nanoparticles to target and repress the Proprotein convertase subtilisin kexin type 9 (PCSK9) gene in hepatocytes, resulting in significant and durable reductions in serum PCSK9 protein and LDL cholesterol levels. Unlike traditional genome editing, this approach introduces reversible epigenetic marks without altering the underlying DNA sequence, offering high specificity with minimal off-target effects. Preclinical studies have demonstrated that a single dose can reduce LDL-C by up to 67% for at least six months in non-human primates, with favorable tolerability profiles[1][3][4][5].