STX-1100

STX-1100 is an investigational CRISPR-based gene editing therapy developed by Scribe Therapeutics for the treatment of cardiovascular diseases, specifically targeting the lowering of LDL cholesterol (LDL-C). It utilizes Scribe's proprietary CasX-based platform, consisting of an engineered CasX mRNA and a guide RNA (gRNA) specific to the PCSK9 gene, both encapsulated within lipid nanoparticles (LNPs) for targeted delivery to hepatocytes. By inducing a permanent knockout of the PCSK9 gene in the liver, STX-1100 aims to provide a durable, "one-and-done" therapeutic effect. Preclinical data in non-human primates have demonstrated significant and sustained reductions in LDL-C levels (up to 54%) for over 300 days following a single dose, with a favorable safety profile and no detectable off-target editing.