EPI-001T

EPI-001T is an investigational CRISPR-based epigenetic modulation therapy developed by Epigenic Therapeutics for the treatment of atherosclerotic cardiovascular disease (ASCVD), heterozygous familial hypercholesterolemia (HeFH), and other forms of hypercholesterolemia. The drug utilizes a proprietary "EPI-Regulator" platform, which consists of a deactivated Cas9 (dCas9) protein fused to a transcriptional repressor, delivered alongside a guide RNA (gRNA) via lipid nanoparticles (LNPs). Unlike traditional CRISPR therapies that cut DNA, EPI-001T targets the promoter region of the **PCSK9** gene to induce stable epigenetic silencing, thereby reducing the production of the PCSK9 protein. This reduction leads to increased low-density lipoprotein receptor (LDLR) expression on the surface of hepatocytes and a subsequent decrease in circulating LDL cholesterol levels. EPI-001T is currently being evaluated in a Phase 1 dose-escalation and expansion study (ChiCTR2500114949) to assess its safety, tolerability, and lipid-lowering efficacy in patients on background statin therapy.