ALC-0315

ALC-0315 is an ionizable cationic lipid (pKa 6.09) that has been used in the generation of lipid nanoparticles (LNPs) for the delivery of mRNA, siRNA, and plasmid DNA in vitro and in vivo. LNPs containing ALC-0315 and encapsulating siRNA targeting Factor VII mRNA decrease Factor VII mRNA levels in hepatocytes in mice. Administration of severe acute respiratory coronavirus 2 (SARS-CoV-2) mRNA in ALC-0315-containing LNPs induces the production of IgG that binds to the SARS-CoV-2 receptor-binding domain (RBD) in rhesus macaques, with a boost in antigen-specific IgG geometric mean titers (GMT) seven and 14 days after a second dose. LNPs containing ALC-0315 and encapsulating ovalbumin mRNA reduce tumor volume in an ovalbumin-expressing B16/F10 murine melanoma model. Formulations containing ALC-0315 have been used in the development of LNPs for the delivery of mRNA-based vaccines.. ALC-0315 is an ionizable cationic lipid (pKa 6.09) that has been used in the generation of lipid nanoparticles (LNPs) for the delivery of mRNA, siRNA, and plasmid DNA in vitro and in vivo. LNPs containing ALC-0315 and encapsulating siRNA targeting Factor VII mRNA decrease Factor VII mRNA levels in hepatocytes in mice. Administration of severe acute respiratory coronavirus 2 (SARS-CoV-2) mRNA in ALC-0315-containing LNPs induces the production of IgG that binds to the SARS-CoV-2 receptor-binding domain (RBD) in rhesus macaques, with a boost in antigen-specific IgG geometric mean titers (GMT) seven and 14 days after a second dose. LNPs containing ALC-0315 and encapsulating ovalbumin mRNA reduce tumor volume in an ovalbumin-expressing B16/F10 murine melanoma model. Formulations containing ALC-0315 have been used in the development of LNPs for the delivery of mRNA-based vaccines.. SMILES: OCCCCN(CCCCCCOC(C(CCCCCC)CCCCCCCC)=O)CCCCCCOC(C(CCCCCC)CCCCCCCC)=O. InChi Code: InChI=1S/C48H95NO5/c1-5-9-13-17-19-27-37-45(35-25-15-11-7-3)47(51)53-43-33-23-21-29-39-49(41-31-32-42-50)40-30-22-24-34-44-54-48(52)46(36-26-16-12-8-4)38-28-20-18-14-10-6-2/h45-46,50H,5-44H2,1-4H3. InChi Key: QGWBEETXHOVFQS-UHFFFAOYSA-N