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BRN-002

Development stage: Preclinical
Lead developer: Beren Therapeutics
Modality: Small Molecules
Administration: Intravenous, Oral

Overview

BRN-002 is a 2-hydroxypropyl-β-cyclodextrin (HPBCD) derivative being developed by Beren Therapeutics as a small molecule cholesterol-depleting agent. It is designed to remove excess cholesterol from cell membranes and arterial plaques, specifically targeting the reversal of atherosclerosis in patients with Homozygous Familial Hypercholesterolemia (HoFH). The drug has been awarded an Innovation Passport under the United Kingdom's Innovative Licensing and Access Pathway (ILAP). Research also suggests its potential in treating sickle cell anemia by improving red blood cell deformability and reducing hemolysis through the removal of membrane cholesterol.

Other names: hydroxypropyl betadex2-hydroxypropyl-beta-cyclodextrin derivative

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