LP-01

LP-01 is an ionizable cationic amino lipid (pKa = ~6.1). It has been used in the generation of lipid nanoparticles (LNPs). LNPs containing LP-01 and encapsulating both Cas9 mRNA and modified single-guide RNA (sgRNA) for the transport protein transthyretin (Ttr) induce gene editing in liver cells in mice in a dose-dependent manner resulting in reduced serum Ttr levels for at least 12 months.Formal Name: 9Z,12Z-octadecadienoic acid, 3-[4,4-bis(octyloxy)-1-oxobutoxy]-2-[[[[3-(diethylamino)propoxy]carbonyl]oxy]methyl]propyl ester. CAS Number: 1799316-64-5. Synonyms: CIN-16645. Molecular Formula: C50H93NO9. Formula Weight: 852.3. Purity: >95%. Formulation: (Request formulation change), A solution in methyl acetate. Solubility: Dichloromethane: Soluble, DMF: Soluble, DMSO: Soluble. SMILES: CCCCCCCCOC(OCCCCCCCC)CCC(OCC(COC(CCCCCCC/C=C\C/C=C\CCCCC)=O)COC(OCCCN(CC)CC)=O)=O. InChi Code: InChI=1S/C50H93NO9/c1-6-11-14-17-20-21-22-23-24-25-26-27-28-29-32-36-47(52)58-43-46(45-60-50(54)57-42-35-39-51(9-4)10-5)44-59-48(53)37-38-49(55-40-33-30-18-15-12-7-2)56-41-34-31-19-16-13-8-3/h20-21,23-24,46,49H,6-19,22,25-45H2,1-5H3/b21-20-,24-23-. InChi Key: CHTXXFZHKGGQGX-IFLFXUNCSA-N.