A Medical University of South Carolina startup based in Charleston, SX, Gruthan Bioscience, LLC is structured around developing a novel class of cholesterol-lowering drugs to treat familial hypercholesterolemia. Founded by an expert is liver research, principals note that those having familial hypercholesterolemia have very high levels of low-density lipoprotein cholesterol (LDL-C), colloquially known as "bad cholesterol." The receptor that should bind to the cholesterol and remove it from the blood is mutated and no longer functions. A subset of these patients inherit mutations in the LDL receptor from both parents and are at high risk of developing severe and deadly cardiovascular disease at a young age. Such patients do not respond to any of the four major classes of cholesterol-lowering drugs due tothe fact that most of those drugs act through the LDL receptor. Two new drugs approved for treating this life-threatening disease can have serious side effects. Gruthan Bioscience LLC is structured around developing a new class of drugs for the safe and effective treatment of patients with this rare form of familial hypercholesterolemia. Noting that rare diseases as a class are common but that the total number of patients with a specific disease may number a few hundred meaning that few for-profit entities can justfy the incurred cost to work in these arenas. The focus of the work at Gruthan Biosciences ha sbeen to provide familial hypercholesterolemia patients with a safe treatment with a poromisng option being a new class of cholesterol-lowering molecules that may have the potential to be used broadly to help patients with high cholesterol meet their goals. Having identified a therapeutic target using a novel procedure, the firm's principals screened small molecules in the South Carolina Compound Collection (SC3) and were able to identify the class of small molecules that Gruthan Bioscience will now develop to determine whether this new class of cholesterol-lowering drugs also works in animals. If the drugs prove effecive, application wil bemade for Phase II STTR funding.
