Repair Biotechnologies is developing REP-0004, an mRNA-based drug delivered via lipid nanoparticles directly to liver cells. It works by breaking down excess intracellular free cholesterol — a toxic buildup that worsens with age and obesity. By making the liver act as though it is cholesterol-deficient, the drug triggers the body to pull excess cholesterol from tissues throughout the body, creating a systemic clearing effect. Early results show rapid and dramatic regression of atherosclerotic plaque and reversal of liver disease including fibrosis. The FDA has granted orphan drug designation, and clinical trials are targeted for mid-2027. This approach could represent a meaningful advance in treating cardiovascular and metabolic disease driven by cholesterol accumulation.
