Draupnir Bio is developing small molecule modulators of PCSK9 based on new insights into its structural biology
PCSK9 (proprotein convertase subtilisin kexin type 9) was first discovered in 2003, and its role in regulating the recycling of the hepatic LDL receptor has been thoroughly validated. Loss-of-function mutations in PCSK9 are causally linked to hypocholesterolemia and confer protection against heart disease, while gain-of-function mutations have been found to cause hypercholesterolemia and an increased risk of heart disease. Modulating the interaction of PCSK9 with the LDL receptor is a therapeutically validated strategy for lowering blood LDL cholesterol and the risk of heart disease.
