OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides

Overview

Journal Nucleic Acid Ther

Specialties Biochemistry
Molecular Biology
Pharmacology

Date 2023 Aug 17

PMID 37590469

Authors

Cindy L Berman

Madeleine Antonsson

Sandor Batkai

Sieto Bosgra

Girish R Chopda

Wouter Driessen

Jeffrey Foy

Chopie Hassan

Xiao Shelley Hu

Hyun Gyung Jang

Meena

Mark Sanseverino

Thomas Thum

Yanfeng Wang

Martin Wild

Jing-Tao Wu

Affiliations

Soon will be listed here.

Abstract

This white paper summarizes the recommendations of the absorption, distribution, metabolism, and excretion (ADME) Subcommittee of the Oligonucleotide Safety Working Group for the characterization of absorption, distribution, metabolism, and excretion of oligonucleotide (ON) therapeutics in nonclinical studies. In general, the recommended approach is similar to that for small molecule drugs. However, some differences in timing and/or scope may be warranted due to the greater consistency of results across ON classes as compared with the diversity among small molecule classes. For some types of studies, a platform-based approach may be appropriate; once sufficient data are available for the platform, presentation of these data should be sufficient to support development of additional ONs of the same platform. These recommendations can serve as a starting point for nonclinical study design and foundation for discussions with regulatory agencies.

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