Development of recombinant Lecithin Cholesterol Acyltransferase as a therapeutic

National Heart, Lung, And Blood Institute

Investigators

Linked publications & trials

Abstract

In the past year, we completed a Phase I clinical trial of LCAT. The study was done at the NIH and was a single dose escalation study in 18 subjects. There was no significant adverse events and the the recombinant LCAT behaved like expected based on animal studies in regard to its ability to modulate HDL metabolism. We also commenced a long-term animal toxicity study in collaboration with AlphaCore Pharma, our CRADA partner and plan to file IND early next year to begin a clinical trial treating patients with a genetic deficiency of LCAT. We are also planning on doing a clinical study on monitoring the flux of cholesterol after recombinant LCAT treatment.