Repair Biotechnologies Reports Atherosclerotic Plaque Regression with REP-0003 in Preclinical Study
Key Insights
Repair Biotechnologies (search) presented preclinical data at the American Heart Association's Scientific Sessions 2025 demonstrating that REP-0003 achieved safe, rapid regression of established atherosclerotic plaque (search) in mouse models after six weeks of treatment.
The company's proprietary Cholesterol Degrading Platform targets excess free cholesterol (search) inside cells, addressing a previously undruggable target that contributes to atherosclerotic lesion growth in homozygous familial hypercholesterolemia (search) patients.
REP-0003 received FDA Orphan Drug Designation in May 2025 for treating homozygous familial hypercholesterolemia (search), a condition where established LDL-cholesterol lowering therapies have limited effectiveness on intracellular free cholesterol (search).
Repair Biotechnologies (search) announced preclinical data demonstrating that its experimental drug REP-0003 achieved rapid and safe regression of established atherosclerotic plaque (search) in mouse models. The data was presented at the American Heart Association's Scientific Sessions 2025, held November 7-10 in New Orleans, Louisiana.
Novel Approach Targets Intracellular Cholesterol
The company's REP-0003 therapy is built on the proprietary Cholesterol Degrading Platform (CDP), which targets excess free cholesterol (search) inside cells—a previously undruggable target. This intracellular free cholesterol (search) is toxic to cells and contributes to numerous conditions with high unmet medical need, particularly the accelerated growth of atherosclerotic lesions that characterizes patients with homozygous familial hypercholesterolemia (search) (HoFH (search)).
"Our recently presented preclinical data demonstrates safe, rapid regression of established atherosclerotic plaque (search) in the Lldr-/- mouse model following six weeks of treatment with REP-0003," said Dr. Mourad Topors, Chief Scientific Officer of Repair Biotechnologies (search). "The positive results and safety profile reinforce our belief in the utility of selective clearance of intracellular free cholesterol (search) as a potential treatment for HoFH (search)."
Addressing Treatment Limitations
Current established therapies that reduce LDL-cholesterol in the bloodstream have little effect on intracellular free cholesterol (search), highlighting a significant treatment gap. The company's approach specifically targets this cellular cholesterol accumulation that drives atherosclerotic progression in HoFH (search) patients.
Regulatory Recognition and Future Development
REP-0003 received Orphan Drug Designation from the U.S. Food and Drug Administration in May 2025 for the treatment of HoFH (search). Dr. Topors noted that the company has been encouraged by growing interest in this previously undruggable target following the regulatory designation.
The research was presented in a poster titled "Selective Degradation of Excess Free Cholesterol (search) in the Liver by REP-0003 Regresses Atherosclerotic Plaque (search) in Ldlr -/- Mice: A Novel Approach for HoFH (search)" during a moderated digital poster session on November 9th. The American Heart Association's Scientific Sessions represents a leading annual gathering in cardiovascular research and medicine, attracting experts from more than 100 nations to share discoveries across 21 disciplines.