Lipid Nanoparticle Library For Non-Viral Delivery Of mRNA And saRNA Towards Vaccine And Cell & Gene Therapy Applications

Recent FDA approval of ONPATTRO^® by Alnylam, BioNTech/Pfizer and Moderna mRNA vaccine and the various clinical trials with mRNA-based drugs or vaccines have provided momentum to further develop LNP-based genetic medicine candidates. Efficient ionizable lipids (one of the important component of LNPs) are needed that are non-toxic, can effectively encapsulate nucleic acids, and can help with endosomal release. Scarcity of lipids that are suitable for vaccination, cell and gene therapy continue to be a problem in advancing many potential therapeutic/vaccine candidates to the clinic. PNI developed a proprietary ionizable lipid library which has shown promising potency towards various applications involving cell therapy, protein replacement therapy and RNA-based Vaccines.