Tech Forum: Accelerating LNP-mediated Nucleic Acid Medicine via a Powerful Ionizable Lipid Library Toolbox

Lipid nanoparticle (LNP)-mediated nucleic-acid (NA) delivery has gotten a tremendous boost over the last years, leading to the development of life-changing vaccines and therapeutics. While the effectivity of LNP to reach the liver has been well established, the cell type-specific, extrahepatic RNA delivery is still a major hurdle, hampering the development of novel therapeutic modalities. Critical for the future success of these lipid-based delivery systems is to identify top-performing and safe ionizable lipid excipients to enable the organ‐ and cell-specific delivery of NA payloads. To that end, we have created a state-of-the-art ionizable lipid toolbox, based on the most promising ionizable lipids from multiple and structurally diverse proprietary libraries with a proven track record of success in different applications. Here, we present case studies where we have evaluated our powerful “lipid toolbox” in terms of their intrinsic tissue‐specificity in vivo.