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The AAPS Journal · 2026 · Vol. 28 · Issue 2 · Springer
Antisense oligonucleotide (ASO) therapeutics present new opportunities for treating challenging-to-treat diseases. Prediction of concentration–time course in systemic circulation and tissues within the context of large molecule physiologically based pharmacokinetic (PBPK) modelling has proven to be useful in animal-to-human extrapolation and first-in-human dose selection. A human PBPK model was developed and verified using bep...