– C12-200 is a cationic lipid for Lipid NanoParticles as delivery systems for enabling the therapeutic potential of siRNA, mRNA or CRISPR as they exhibit remarkable in vivo potencies at low doses.C12-200 is a common positive control ionizable lipid for
exploring new ionizable lipids. C12-200 is an ionizable
lipid with five tails. The tri-palmitoyl-Sglyceryl
cysteine linked to the pentapeptide (Pam3)-modified C12-200 iLNPs was developed to deliver tumor antigen mRNA
for enhancing the mRNA-mediated cancer immunotherapy.
The results of the therapeutic evaluation showed that Pam3-
modified C12-200 iLNPs could almost inhibit tumor growth in
tumor-bearing mice. To improve delivery efficiency, the
formulation was optimized to replace the phospholipids from
DSPC to DOPE, and the result showed that optimized
formulation induced EPO protein expression was seven times
that of the initial formulation,[35] and the formulation has been
used in the study for mRNA-mediated human ?-galactosidase
protein replacement therapy in mice and nonhuman
primates.