Screening-based DoE approach of cationic LNPs for pulmonary mRNA delivery
발표자
이재성 (숭실대학교)
연구책임자
심가용 (숭실대학교 의생명시스템학부)
공동저자
이재성 (숭실대학교), 심가용 (숭실대학교 의생명시스템학부)
초록
내용
mRNA therapeutics are a flexible next-generation platform. Lipid nanoparticles (LNPs) are widely used for delivery, but conventional versions predominantly accumulate in the liver. To overcome this, we used cationic lipids to control surface charge, enabling lung-specific targeting. In this study, we optimized lung-targeting LNPs using a Design of Experiments (DoE) approach, specifically a Definitive Screening Design (DSD), to identify key factors with minimal experiments. Thirteen LNPs were manufactured using five lipids across three levels. We evaluated physicochemical properties, cell viability, and mRNA expression in lung epithelial cells and in vivo mice. Statistical analysis identified optimal candidates. This study demonstrates the potential of targeted LNP strategies for lung diseases and the efficiency of DoE-based screening.