LNP-siSAT1

LNP-siSAT1 is a lipid nanoparticle-based siRNA delivery system designed to selectively knockdown the Spermidine/Spermine N1-Acetyltransferase 1 (SAT1) enzyme. SAT1 is involved in polyamine catabolism and is frequently overexpressed in glioblastoma multiforme (GBM), where it contributes to tumor survival and resistance to radiation therapy. The delivery vehicle utilizes the ionizable lipid DODAP and is formulated via microfluidic mixing to produce nanoparticles approximately 80 nm in size with a neutral surface charge. In preclinical models, LNP-siSAT1 has demonstrated the ability to reduce SAT1 mRNA and protein levels in various glioblastoma cell lines (e.g., U251, LN229, 42MGBA) and significantly enhance the efficacy of radiation treatment.