Longevity & AgingRepair Bio Gets FDA Nod for mRNA Therapy That Removes Arterial Plaque
Repair Biotechnologies has received FDA Rare Disease Evidence Principles eligibility for REP-0003, an mRNA therapy designed to remove excess intracellular free cholesterol from cells — a toxic driver of atherosclerotic plaque. The initial target is homozygous familial hypercholesterolemia, a severe inherited condition where dangerously high LDL levels cause heart disease from birth. Unlike standard cholesterol-lowering drugs, REP-0003 aims to eliminate existing cellular damage rather than just slow its progression. The company holds Orphan Drug Designation and sees HoFH as a regulatory stepping stone toward broader cardiovascular and age-related disease applications, with the same pathological mechanism implicated in liver, retinal, and brain aging.