Homozygous familial hypercholesterolemia (HoFH) is a genetic condition caused by mutations in the LDLR gene and characterized by severe hypercholesterolemia and premature cardiovascular disease. Repair Biotechnologies Inc. has developed REP-0003, a therapeutic based on the Cholesterol Degrading Platform (CDP) that degrades excessive intracellular free cholesterol into a non-toxic and excretable catabolite to safely reverse cholesterol-rich vulnerable plaque.
Repair Biotechnologies Inc.’s REP-0003 has been awarded orphan drug designation by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH).
Repair Biotechnologies Inc. has received encouraging feedback from a pre-IND meeting with the FDA as it works toward an IND filing to conduct a phase Ib study of its REP-0003 mRNA therapy in patients with homozygous familial hypercholesterolemia (HoFH).
