Lipid C3 is an ionizable cationic lipid (pKa = 5.05-5.67) utilized in the development of lipid nanoparticles (LNPs) for mRNA delivery in vitro and in vivo. In an Idua-W392X mouse model of mucopolysaccharidosis type I (Hurler syndrome), intracerebroventricular administration of LNPs containing lipid C3 and mRNA encoding α-L-iduronidase (Idua) enhances α-L-iduronidase enzyme activity in the midbrain and hippocampus while reducing glycosaminoglycan levels in the forebrain, midbrain, and hippocampus. Additionally, fetal macaques administered LNPs with lipid C3 and an mRNA reporter in utero exhibit increased GFP expression in the brain.
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