LNP78

LNP78 is a novel lipid nanoparticle (LNP) delivery system developed by researchers at Emory University, specifically designed for the functional delivery of mRNA to hematopoietic stem cells (HSCs) in vivo. Composed of a biodegradable ionizable lipid, DMG-PEG, cholesterol, and DSPC, LNP78 has demonstrated high potency in targeting bone marrow LSK cells and long-term HSCs, as well as liver endothelial cells and splenic immune cells, while avoiding significant delivery to the lung, kidney, or heart. Its primary therapeutic potential lies in enabling in vivo gene editing for hematological and immune disorders, such as sickle cell disease, by bypassing the need for ex vivo cell manipulation. Preclinical studies have shown that LNP78 can achieve over 70% transfection efficiency in mouse HSCs after a single dose, with a favorable safety profile and minimal upregulation of serum cytokines.