ATX-100 is an ionizable cationic lipid (pKa = 6.38).1,2 It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of siRNA. Intravenous administration of LNPs containing ATX-100 and encapsulating Factor VII siRNA decrease Factor VII blood levels in mice.2
1.Rajappan, K., Tanis, S.P., Roberts, S., et al.Development of a safe and scalable process for the production of a high-purity thiocarbamate-based ionizable lipid as an excipient in mRNA-encapsulating lipid nanoparticlesOrg. Process Res. Dev.25(6)1383-1390(2021) 2.Rajappan, K., Tanis, S.P., Mukthavaram, R., et al.Property-driven design and development of lipids for efficient delivery of siRNAJ. Med. Chem.63(21)12992-13012(2020)