nor-MC3 is an MC3 -type ionizable lipid with C 17 unsaturated chains, formulated in lipid nanoparticles for nucleic acid ( mRNA and siRNA) delivery and supports in vitro and in vivo transfection [1].
In Vivo, Nor-MC3-containing lipid nanoparticles (1 mg/kg mRNA; i.v.; single injection) deliver mRNA to mouse liver and spleen with high efficacy[1].
In Vitro, nor-MC3-based mRNA LNPs (0.03-10 μg/mL mRNA; 24 h) exhibit superior in vitro transfection efficiency in HuH7 cells compared to MC3-based LNPs, with enhanced activity at 10 μg/mL mRNA[1]. nor-MC3-based siRNA LNPs (0.03-1 μg/mL siRNA) exhibit comparable in vitro knockdown efficiency in stably luciferase-expressing 22Rv1 cells to MC3-based LNPs, with enhanced activity at 0.5 μg/mL siRNA and a similar EC50 value of 0.1644 μg/mL siRNA[1].
References:
[1]. Ciufolini M A, et al. MC3-TYPE LIPIDS AND USE THEREOF IN THE PREPARATION OF LIPID NANOPARTICLES. WO/2022/246571. WIPO. 01.12.2022.
In Vivo, Nor-MC3-containing lipid nanoparticles (1 mg/kg mRNA; i.v.; single injection) deliver mRNA to mouse liver and spleen with high efficacy[1].
In Vitro, nor-MC3-based mRNA LNPs (0.03-10 μg/mL mRNA; 24 h) exhibit superior in vitro transfection efficiency in HuH7 cells compared to MC3-based LNPs, with enhanced activity at 10 μg/mL mRNA[1]. nor-MC3-based siRNA LNPs (0.03-1 μg/mL siRNA) exhibit comparable in vitro knockdown efficiency in stably luciferase-expressing 22Rv1 cells to MC3-based LNPs, with enhanced activity at 0.5 μg/mL siRNA and a similar EC50 value of 0.1644 μg/mL siRNA[1].
References:
[1]. Ciufolini M A, et al. MC3-TYPE LIPIDS AND USE THEREOF IN THE PREPARATION OF LIPID NANOPARTICLES. WO/2022/246571. WIPO. 01.12.2022.