LC-1 lipid nanoparticles

LC-1 lipid nanoparticles are a non-viral delivery platform optimized for the encapsulation and systemic delivery of large mRNA cargos, such as those encoding CRISPR-Cas9 gene editors and adenine base editors (ABE). Developed by researchers at the University of Toronto, the LC-1 ionizable lipid was identified through a large-cargo-informed discovery strategy using a library of over 300 lipids. Mechanistically, LC-1 LNPs promote the formation of inverse-hexagonal phase structures, which facilitate membrane fusion and enhance endosomal escape of the mRNA payload. In preclinical studies, LC-1 LNPs have demonstrated superior gene editing efficiency in the liver, lung, and brain compared to benchmark lipids like ALC-0315 and LP-01. Therapeutic applications include targeting PCSK9 for hypercholesterolemia and CFTR for cystic fibrosis.

Other names

LC-1 LNPsLC1 LNPsLC 1 LNPs