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Article

Cross-Species Translation of Biophase Half-Life and Potency of GalNAc-Conjugated siRNAs

22 Jul 2022

Abstract excerpt

Small interfering RNAs (siRNAs) with N -acetylgalactosamine (GalNAc) conjugation for improved liver uptake represent an emerging class of drugs to treat liver diseases. Understanding how pharmacokinetics and pharmacodynamics translate is pivotal for in vivo study design and human dose prediction. However, the literature is sparse on translational data for this modality, and pharmacokinetics in the liver is seldom...

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PMID: 35867041
DOI: 10.1089/nat.2022.0010

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