Prediction of Interspecies Translation for Targeting Delivery Coefficients of GalNAc-siRNA Silencing Apolipoprotein C-III Using a Mechanistic Minimal Physiologically …
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Z Tian, H Luo, Y Chu, Y Liu, S Gao, L Song…
Clinical …, 2025
Springer
Methods A mechanistic minimal physiologically based pharmacokinetic/pharmacodynamic (mPBPK-PD) model for an investigational new apolipoprotein C-III (APOC3)-silencing GalNAc-siRNA (RBD5044) was developed using available pharmacokinetic/pharmacodynamic (PK/PD) data. The aim was to explore hepatic-targeting delivery processes, the PK/PD relationship, and interspecies translation. Results First, multiple PK/PD datasets from mice were satisfactorily fitted using the mPBPK-PD model. Second, we …
