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

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 nonclinic...

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Autores principales: Berman, Cindy L., Antonsson, Madeleine, Batkai, Sandor, Bosgra, Sieto, Chopda, Girish R., Driessen, Wouter, Foy, Jeffrey, Hassan, Chopie, Hu, Xiao Shelley, Jang, Hyun Gyung, Meena,  , Sanseverino, Mark, Thum, Thomas, Wang, Yanfeng, Wild, Martin, Wu, Jing-Tao
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10561745/
https://www.ncbi.nlm.nih.gov/pubmed/37590469
http://dx.doi.org/10.1089/nat.2023.0011
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author Berman, Cindy L.
Antonsson, Madeleine
Batkai, Sandor
Bosgra, Sieto
Chopda, Girish R.
Driessen, Wouter
Foy, Jeffrey
Hassan, Chopie
Hu, Xiao Shelley
Jang, Hyun Gyung
Meena,  
Sanseverino, Mark
Thum, Thomas
Wang, Yanfeng
Wild, Martin
Wu, Jing-Tao
author_facet Berman, Cindy L.
Antonsson, Madeleine
Batkai, Sandor
Bosgra, Sieto
Chopda, Girish R.
Driessen, Wouter
Foy, Jeffrey
Hassan, Chopie
Hu, Xiao Shelley
Jang, Hyun Gyung
Meena,  
Sanseverino, Mark
Thum, Thomas
Wang, Yanfeng
Wild, Martin
Wu, Jing-Tao
author_sort Berman, Cindy L.
collection PubMed
description 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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spelling pubmed-105617452023-10-10 OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides Berman, Cindy L. Antonsson, Madeleine Batkai, Sandor Bosgra, Sieto Chopda, Girish R. Driessen, Wouter Foy, Jeffrey Hassan, Chopie Hu, Xiao Shelley Jang, Hyun Gyung Meena,   Sanseverino, Mark Thum, Thomas Wang, Yanfeng Wild, Martin Wu, Jing-Tao Nucleic Acid Ther Issues in Development 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. Mary Ann Liebert, Inc., publishers 2023-10-01 2023-10-03 /pmc/articles/PMC10561745/ /pubmed/37590469 http://dx.doi.org/10.1089/nat.2023.0011 Text en © Cindy L. Berman et al., 2023; Published by Mary Ann Liebert, Inc. https://creativecommons.org/licenses/by/4.0/This Open Access article is distributed under the terms of the Creative Commons License [CC-BY] (http://creativecommons.org/licenses/by/4.0 (https://creativecommons.org/licenses/by/4.0/) ), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Issues in Development
Berman, Cindy L.
Antonsson, Madeleine
Batkai, Sandor
Bosgra, Sieto
Chopda, Girish R.
Driessen, Wouter
Foy, Jeffrey
Hassan, Chopie
Hu, Xiao Shelley
Jang, Hyun Gyung
Meena,  
Sanseverino, Mark
Thum, Thomas
Wang, Yanfeng
Wild, Martin
Wu, Jing-Tao
OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides
title OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides
title_full OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides
title_fullStr OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides
title_full_unstemmed OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides
title_short OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides
title_sort oswg recommended approaches to the nonclinical pharmacokinetic (adme) characterization of therapeutic oligonucleotides
topic Issues in Development
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10561745/
https://www.ncbi.nlm.nih.gov/pubmed/37590469
http://dx.doi.org/10.1089/nat.2023.0011
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