Development of recombinant Lecithin Cholesterol Acyltransferase as a therapeutic

National Heart, Lung, And Blood Institute

Investigators

Linked publications & trials

Abstract

We have completed and published the first Phase I study of recombinant LCAT showing that it was safe and raised HDL-C. In addition we published the first enzyme replacement therapy of LCAT in a patient with Familial LCAT Deficiency, showing that it normalized their lipoprotein profile. Finally, we completed and published a study showing that LpX, the abnormal lipoprotein particle that accumulates in LCAT Deficiency, causes renal disease in an animal model. This finding supports the future use of LpX as a potential biomarker for monitoring rLCAT therapy.