Industry Practices in Oligonucleotide Tissue Biodistribution Assessment: An IQ consortium Cross-Industry Survey of Current Approaches and Emerging Trends
Rattachement africain : jp, us, ch, dk, br. Niveau de preuve : code pays fourni par la source.
Le résumé fourni par la source
Accurate assessment of tissue distribution for oligonucleotide therapeutic (ONT) drug candidates is essential for understanding pharmacokinetic behavior and predicting therapeutic efficacy. ONTs present a unique challenge with their rapid systemic clearance coupled with prolonged tissue retention, making comprehensive tissue concentration evaluation critical for successful drug development. The IQ Consortium Tissue Concentration Working Group surveyed member companies about their current tissue concentration assessment methods to understand industry practices and identify areas for improvement. Most companies reported that ONTs still represent a relatively small portion of their pre-candidate selection portfolios, reflecting the evolving nature of this therapeutic modality. siRNAs dominated development efforts across surveyed organizations, followed by antisense oligonucleotides, indicating clear therapeutic class preferences within the industry. Assessment strategies varied considerably across organizations, highlighting different approaches to resource allocation and risk management. While some companies routinely evaluate tissue concentrations for all ONT programs regardless of indication or target, others take a more selective, program-dependent approach based on compound characteristics and therapeutic objectives. Despite this strategic variability, there was universal reliance on LC-MS for quantification, often supplemented with qPCR/RT-qPCR and hybridization assays for comprehensive analytical coverage. All surveyed companies integrate tissue concentration data into translational pharmacokinetic modeling efforts, yet few have adopted physiologically-based pharmacokinetic (PBPK) models as standard practice. Companies recognize the value of improving ONT tissue distribution assessment through standardized methodology tailored to specific oligonucleotide classes.
Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.
Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Industry Practices in Oligonucleotide Tissue Biodistribution Assessment: An IQ consortium Cross-Industry Survey of Current Approaches and Emerging Trends
- Date Crossref
- 29/01/2026
- Éditeur
- Springer Science and Business Media LLC
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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Takeda (Japan) pays non établi dans la noticeEntreprise
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GlaxoSmithKline (United States) pays non établi dans la noticeEntreprise
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Novartis (Switzerland) pays non établi dans la noticeEntreprise
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Novo Nordisk (Denmark) pays non établi dans la noticeEntreprise
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Novo Nordisk (United States) pays non établi dans la noticeEntreprise
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Boehringer Ingelheim (Brazil) pays non établi dans la noticeEntreprise
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Johnson & Johnson (United States) pays non établi dans la noticeEntreprise
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AngioDynamics (United States) pays non établi dans la noticeEntreprise
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DMPK & Modeling pays non établi dans la noticeInstitution
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Collegeville GlaxoSmithKline pays non établi dans la noticeUniversité ou école supérieure
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PK Sciences pays non établi dans la noticeInstitution
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Translational Biomarkers and Bioanalytics pays non établi dans la noticeInstitution
Takeda (Japan), GlaxoSmithKline (United States) et Novartis (Switzerland), avec 9 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.