SIG-001

We are advancing our preclinical lead candidate, a liver targeted siRNA-GalNac with the potential to address lipid driven liver and cardiometabolic diseases, starting with MASH and artherogenic dyslipidemia with hepatic steatosis.

  • SIG-001 is a precision liver-targeted siRNA-GalNAc therapeutic aimed at an emerging metabolic pathway that links atherogenic dyslipidemia and hepatic steatosis. By silencing a target associated with elevated triglycerides, apoB-rich lipoproteins, reduced HDL, and fatty liver biology, SIG-001 is designed to address the dyslipidemia–steatosis phenotype at its source.

  • Silen7 uses a GalNAc-conjugated siRNA approach designed for precise delivery to hepatocytes, the key cells driving lipoprotein and triglyceride metabolism in the liver. GalNAc enables receptor-mediated uptake into hepatocytes, and the siRNA payload harnesses RNA interference to selectively silence disease-relevant gene expression. This provides a targeted, potent, and potentially durable therapeutic approach for liver-driven cardiometabolic disease.

  • Silen7 is currently in preclinical development. Preclinical and translational evidence supporting the target indicates that hepatic target silencing may reduce apoB, lower hepatic triglycerides and steatosis, improve MASH-related phenotypes, and decrease NAS and fibrosis-associated readouts, while maintaining normal liver safety markers in preclinical models. Our ongoing siRNA program is now finalizing the lead candidate for IND-enabling studies.