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REP-0003
- Development stage
- Unknown
- Lead developer
- Repair Biotechnologies
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
REP-0003 is a first-in-class gene therapy candidate developed by Repair Biotechnologies for the treatment of homozygous familial hypercholesterolemia (HoFH) and atherosclerosis. It consists of a lipid nanoparticle (LNP) encapsulating mRNA that encodes a proprietary fusion protein known as the Cholesterol Degrading Platform (CDP). Upon delivery to the liver, the CDP protein selectively degrades excess intracellular free cholesterol into non-toxic, excretable catabolites. This mechanism aims to restore liver health and promote the systemic regression of cholesterol-rich atherosclerotic plaques, addressing a significant unmet need in patients where traditional lipid-lowering therapies like statins and PCSK9 inhibitors are insufficient. Preclinical studies in Ldlr-/- mice have demonstrated significant reductions in serum ApoB levels, improved liver health markers, and regression of aortic root plaque cross-sectional area.
- Other names
- CDP therapyCholesterol Degrading Platform therapyLNP-CDP mRNA
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