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).

Repair Biotechnologies Inc. and Genevant Sciences Corp. have entered into a collaboration and nonexclusive license agreement to combine Repair’s Cholesterol Degrading Platform (CDP) mRNA technology with Genevant’s proprietary lipid nanoparticle (LNP) technology in the development of a potential novel treatment for atherosclerosis.

Repair Biotechnologies Inc. has reported new data showing that its lipid nanoparticle (LNP)/messenger RNA (mRNA) therapy rapidly reversed the progression of atherosclerosis in mouse models applicable to familial hypercholesterolemia as well as atherosclerosis in the broader population.