Globe Biotech LDLR gene therapy

Globe Biotech LDLR gene therapy is an investigational gene therapy being developed by Globe Biotech for the treatment of familial hypercholesterolemia (FH). The therapy is designed to target the low-density lipoprotein receptor (LDLR) gene, which is often mutated or defective in patients with FH, leading to dangerously high levels of LDL cholesterol. By delivering a functional copy of the LDLR gene or correcting the existing defect, the therapy aims to restore the liver's ability to clear LDL cholesterol from the bloodstream, thereby reducing the risk of premature cardiovascular disease. As of early 2024, the program is in the early stages of development, with specific details regarding the delivery vector (e.g., viral vs. non-viral) and clinical phase not yet publicly detailed.

Other names

Globe Biotech LDLR gene therapy programLDLR gene therapy (Globe Biotech)