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Repair Biotechnologies is developing a gene therapy based on its proprietary Cholesterol Degrading Platform (CDP) for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). The therapy utilizes lipid nanoparticle (LNP) delivery of messenger RNA (mRNA) to provide cells with the ability to safely degrade excess intracellular free cholesterol. This intracellular cholesterol is toxic to cells and is a primary driver of the pathology and liver fibrosis associated with MASH. In preclinical studies using high-fat diet mouse models, the CDP LNP-mRNA treatment demonstrated up to a 44% reduction in liver fibrosis measures over eight weeks. As of March 2024, the company has received favorable pre-IND feedback from the FDA for the treatment of liver disease.
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