Lipids

6-Trehalose monooleate can be used as antiaggregation agent for stabilization of proteins. It can also be used as a delivery vehicle for topical drugs by forming solid lipid nanoparticles.

C12-113 is an ionizable lipid used to formulate lipid nanoparticles (LNPs) for delivering RNA to cells. C12-113 LNPs delivered mRNA encoding luceriferase and the SARS-Cov2 spike protein into a mouse model.

Lipid M is an ionizable lipid used to form lipid nanoparticles for delivering RNA. LNPs formulated with Lipid M delivered luciferase mRNA in mice resulting in higher protein expression compared to the benchmark lipid DLin-MC3-DMA. Lipid M LNPs delivering IgG mRNA showed prolonged expression compared to MC3 in non-human primates after intramuscular injection.

4-O10b1 is an ionizable lipid used to generate lipid nanoparticles (LNPs) for delivering RNA to cells. LNPs comprised of 4-O10b1 and conjugated with the macrophage antibody F4/80 were able to delivery siRNA targeting TAK1 to RAW264.7 cells resulting in suppressed activation of NF-kB. Intranasal administration reduced lung injury in an influenza mouse model.

FTT5 (Echelon Cat#: N-1115) is an analog of TT3 (Echelon Cat#: N-1113). FTT5 lipid nanomaterials (LNPs) displayed two-fold potency in the delivery efficiency of mRNA to the liver compared to TT3 LNPs. FTT5 LNPs efficiently delivered long mRNAs such as human factor VIII (hFVIII) mRNA (~4.5 kb) for expression of hFVIII protein in hemophilia A mice in vivo.

80-O18 is an ionizable lipid used to generate lipid nanoparticles (LNPs) for delivery of proteins. A GFP-Cre recombinase fusion protein encapsulated in 80-O18 LNPs was delivered to the cytosol of HeLa-HGM cells. Cytosolic delivery was measured by a chloroalkane penetration assay.

OC2-K3-E10 is an ionizable lipid for delivery of mRNA in lipid nanoparticles (LNPs). OC2-K3-E10 LNPs delivered Firefly luciferase mRNA when injected intramuscularly in mice and resulted in comparable levels of luminescence as SM-102 LNPs. LNPs were also able to deliver Cas9 mRNA and sgRNA into genetically modified mice. LNPs containing OC2-K3-E10 were able to encapsulate four-fold more mRNA copies per particle compared to SM-102.

PPZ-A10 is an ionizable lipid used for formulating lipid nanoparticles to deliver RNA in vitro and in vivo. PPZ-A10 LNPs preferentially delivered Cre mRNA to liver and spleen immune cells when injected intravenously into mice at concentrations as low as 0.3 mg/kg. PPZ-A10 LNPs containing RNA to generate chimeric antigen receptor macrophages were injected into a hepatocellular carcinoma mouse model resulting in decreased tumor burden and increased survival.

The linker-degradable ionizable lipid (LDIL) 4A3-SCC-10 possesses superior endosomal escape and rapid mRNA release abilities thanks to its unique GSH-responsive cone-shaped architecture. Lipid nanoparticles (LNPs) of 4A3-SCC-10 showed improved mCherry mRNA delivery in livers by 87-fold compared to DLin-MC3-DMA in vivo. 4A3-SCC-10 also demonstrated improved mRNA delivery in vivo compared to its parent counterpart without a disulfide bond or control counterpart with a disulfide bond in the tail.