Development of recombinant Lecithin Cholesterol Acyltransferase as a therapeutic

National Heart, Lung, And Blood Institute

Investigators

Linked publications & trials

Abstract

Just prior to 2017, we completed and published a Phase I study of recombinant LCAT showing that it was safe and raised HDL-C. In addition we did the first enzyme replacement therapy of LCAT in a patient with Familial LCAT Deficiency, showing that it normalized their lipoprotein profile. Recombinant LCAT was licensed to MedImmune where it is now undergoing Phase II testing. In the past year, we developed an mouse model for LCAT deficiency that form high levels of LpX and spontaneously develop renal disease and showed that recombinant LCAT prevents renal disease in this model. A revised version of this paper is under review. We also published a paper describing a novel and more convenient assay for measuring LCAT activity that can be used in future clinical trials. We also identified the mechanism for activation of LCAT by a small molecule first identified by a large drug company and have identified several other novel small molecule activators of LCAT that we published and have patented.