A1-28 is a disulfide bond-containing ionizable cationic lipid.1 It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of CRISPR complementary single-guide RNA (sgRNA) and Cas9 mRNA for genome editing in vitro.

VL422 is an ionizable cationic lipid.1 It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of CRISPR complementary single-guide RNA (sgRNA) and Cas9 mRNA for gene editing in vitro and in vivo. LNPs containing VL422 and encapsulating Cas9 mRNA and sgRNA targeting the gene encoding angiopoietin-related protein 3 (ANGPTL3), a protein whose loss-of-function decreases LDL, HDL, and cholesterol plasma levels, induce a deletion in a premature stop codon in ANGPTL3 in the livers of cynomolgus monkeys.

CL15F6 is an ionizable cationic lipid (pKa = 6.75).1 It has been used in the formation of lipid nanoparticles (LNPs) for the delivery of mRNA and polymer-lipid hybrid nanoparticles for the delivery of plasmid DNA in vitro.1,2

C3-K2-E14 is an ionizable cationic lipid (pKa = 5.5).1 It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of mRNA or siRNA in vitro and in vivo. LNPs containing C3-K2-E14 and encapsulating siRNA targeting the gene encoding colony-stimulating factor 1 (Csf1) reduce the percentage of circulating Ly6Chi monocytes and increase the percentage of circulating Ly6Cint monocytes in mice.

(S)-C12-200 is an ionizable cationic lipid (pKa = 7.12) and an isomer of C12-200 (Item No. 36699).1 It has been used in the formation of lipid nanoparticles (LNPs) for the delivery of mRNA in vivo. Intravenous administration of LNPs containing (S)-C12-200 and encapsulating mRNA encoding Cre recombinase increase the percentage of tdTomato+ hepatocytes, endothelial cells, and Kupffer cells in the livers of Ai14 mice greater than (R)-C12-200-containing and Cre mRNA-encapsulating LNPs.

PPPDA-O16B is a disulfide bond-containing ionizable cationic lipid that has been used in the generation of lipid nanoparticles (LNPs) for plasmid delivery in vitro and in vivo.1 LNPs containing PPPDA-O16B and encapsulating plasmids encoding Ras/Rap1-specific endopeptidase (RRSP) increase RRSP levels and decrease Ras levels in, and reduce the viability of, HeLa cervical cancer cells. LNPs containing PPPDA-O16B and encapsulating plasmids encoding RRSP selectively localize to tumors over the heart, liver, spleen, lungs, and kidneys, as well as decrease tumor volume without reducing body weight, in an HCT116 colorectal cancer mouse xenograft model.

Lipid C3 is an ionizable cationic lipid (pKa = 5.05-5.671).1,2 It has been used in the formation of lipid nanoparticles (LNPs) for the delivery of mRNA in vitro and in vivo.1 Intracerebroventricular administration of LNPs containing lipid C3 and encapsulating mRNA encoding α-L-iduronidase (Idua) to neonates increase midbrain and hippocampal α-L-iduronidase enzyme activity and decrease forebrain, midbrain, and hippocampal glycosaminoglycan levels in an Idua-W392X mouse model of the lysosomal storage disorder mucopolysaccharidosis type I, also known as Hurler syndrome. Intracerebroventricular administration of LNPs containing lipid C3 and encapsulating an mRNA reporter to fetal macaques in utero increase brain GFP expression.

BAmP-O16B is an ionizable cationic amino lipid that has been used in the generation of lipid nanoparticles (LNPs).1 LNPs containing BAmP-O16B and encapsulating NSA-inactivated RNase A reduce the viability of SiHa and HeLa cancer cells, but not non-cancerous NIH3T3 and HEK293T cells, upon intracellular reactivation of RNase A by glutathione (GSH).

Lipid OA2 is an ionizable cationic lipid that has been used in the generation of single-component lipid nanoparticles (LNPs) for the delivery of siRNA.1 LNPs containing lipid OA2 and siRNA targeting the gene encoding suppressor of cytokine signaling 1 (Socs1) decrease Socs1 expression by approximately 50% in isolated mouse bone marrow dendritic cells (BMDCs). Vaccination with ovalbumin-induced isolated mouse BMDCs transfected with LNPs composed of lipid OA2 and Socs1-targeting siRNA increases survival and decreases tumor volume in a B16-OVA murine melanoma model.

Compound 11 (EBP inhibitor) is a selective orally bioavailable, brain-penetrant emopamil binding protein (EBP) inhibitor with EC50 of 44 nM. Compound 11 enhances the formation of oligodendrocytes in human cortical organoids.

Lung endothelium-targeted Lipid-1 is used in pulmonary endothelium targeted mRNA delivery. Lung endothelium-targeted Lipid-1 formulated nanoparticle, Lung-LNP (LuLNP) is capable of delivering therapeutic mRNA cargo for vascular repair in influenza infection.

CRT0066101 is a PRKDs inhibitor. CRT0066101 suppressed the proliferation and migration of four bladder cancer cell lines in vitro. CRT0066101 blocked tumor growth in a mouse flank xenograft model of bladder cancer. CRT0066101 treatment or PKD2 silencing arrested bladder cancer cells at the G2/M phase, the arrest being accompanied by decreases in the levels of cyclin B1, CDK1 and phospho-CDK1 (Thr161) and increases in the levels of p27Kip1 and phospho-CDK1 (Thr14/Tyr15). CRT0066101 suppresses bladder cancer growth by inhibiting PKD2 through induction of G2/M cell cycle arrest, leading to the blockade of cell cycle progression.

HA-155 is a potent and selective autotaxin (ATX) inhibitor with an IC50 of 5.7 nM.

PF-CBP1, also known as PF-06670910, is potent and highly-selective inhibitor of the bromodomain of CREB binding protein (CBP BRD) that down regulates targets of CBP in macrophages primary neurons.

GSK-3 Inhibitor XIII is a potent and ATP-competitive GSK-3 inhibitor with a Ki of 24 nM.