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Repair-A
Syracuse, New York, United States
- Ownership
- Private
- Development stage
- Other
01
Overview
Repair Biotechnologies' lead mRNA program (REP-0003) has demonstrated dramatic, rapid regression of atherosclerotic plaques in preclinical models and received FDA Orphan Drug Designation for homozygous familial hypercholesterolemia[1]. The company received positive feedback from a Pre-IND meeting with the FDA for conducting a Phase 1b study[3]. In mouse models of atherosclerosis, a single gene therapy treatment resulted in a rapid 50% reduction in atherosclerotic plaque lipids within one month with no identified side effects[3].
- Therapeutic areas
- CardiovascularGastroenterology
- Modalities
- mRNA-based drugsCell therapyLipid nanoparticle delivery therapy
- Industry
- Biotech
02
Partnerships
Genevant Sciences
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