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

Drug Metabolism and Pharmacokinetics of Antisense Oligonucleotide Therapeutics: Typical Profiles, Evaluation Approaches, and Points to Consider Compared with Small Molecule Drugs

(2023) Hideo Takakusa et al.   Nucleic Acid Therapeutics

Considerations and recommendations for assessment of plasma protein binding and drug–drug interactions for siRNA therapeutics

(2022) Sara C Humphreys et al.   NUCLEIC ACIDS RESEARCH

Considerations in the Immunogenicity Assessment Strategy for Oligonucleotide Therapeutics (ONTs)

(2022) Nazneen Bano et al.   AAPS Journal

Assessment of the Immunogenicity Potential for Oligonucleotide-Based Drugs

(2022) Scott P. Henry et al.   Nucleic Acid Therapeutics

FDA and industry collaboration: Identifying opportunities to further reduce reliance on nonhuman primates for nonclinical safety evaluations

(2022) David Ackley et al.   REGULATORY TOXICOLOGY AND PHARMACOLOGY

Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides

(2022) Aurélie Goyenvalle et al.   Nucleic Acid Therapeutics

Impact of Serum Proteins on the Uptake and RNA Interference Activity of N-Acetylgalactosamine-Conjugated Small Interfering RNAs

(2021) Saket Agarwal et al.   Nucleic Acid Therapeutics

A Drug‐Drug Interaction Study Evaluating the Effect of Givosiran, a Small Interfering Ribonucleic Acid (siRNA), on Cytochrome P450 Activity in Liver

(2021) Daphne Vassiliou et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Phosphorothioate modified oligonucleotide–protein interactions

(2020) Stanley T Crooke et al.   NUCLEIC ACIDS RESEARCH

Advances in oligonucleotide drug delivery

(2020) Thomas C. Roberts et al.   NATURE REVIEWS DRUG DISCOVERY

Immunogenicity Assessment of Inotersen, a 2′-O-(2-Methoxyethyl) Antisense Oligonucleotide in Animals and Humans: Effect on Pharmacokinetics, Pharmacodynamics, and Safety

(2020) Rosie Z. Yu et al.   Nucleic Acid Therapeutics

Clinical Pharmacology Studies Supporting Oligonucleotide Therapy Development: An Assessment of Therapies Approved and in Development Between 2012‐2018

(2020) Hobart Rogers et al.   CTS-Clinical and Translational Science

Targeting RNA : A Transformative Therapeutic Strategy

(2019) Wei Yin et al.   CTS-Clinical and Translational Science

Plasma and liver protein binding of GalNAc conjugated siRNA

(2019) Sara C Humphreys et al.   DRUG METABOLISM AND DISPOSITION

In Vitro Drug-Drug Interaction Evaluation of GalNAc conjugated siRNAs Against CYP450 Enzymes and Transporters

(2019) Diane Ramsden et al.   DRUG METABOLISM AND DISPOSITION

Metabolism and Disposition of Volanesorsen, a 2′ MOE Antisense Oligonucleotide, Across Species

(2019) Noah Post et al.   DRUG METABOLISM AND DISPOSITION

Pharmacokinetics and Clinical Pharmacology Considerations of GalNAc3-Conjugated Antisense Oligonucleotides

(2019) Yanfeng Wang et al.   Expert Opinion on Drug Metabolism & Toxicology

The Pharmacokinetics of 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides: Experiences from Developing Exon Skipping Therapies for Duchenne Muscular Dystrophy

(2019) Sieto Bosgra et al.   Nucleic Acid Therapeutics

Discovery of a novel deaminated metabolite of a single-stranded oligonucleotide in vivo by mass spectrometry

(2019) Jing Li et al.   Bioanalysis

Evaluation of electrophoretic mobility shift assay as a method to determine plasma protein binding of siRNA

(2019) Carrie Rocca et al.   Bioanalysis

Development of Protecting Groups for Prodrug-Type Oligonucleotide Medicines

(2018) Hisao Saneyoshi et al.   CHEMICAL & PHARMACEUTICAL BULLETIN

Drug metabolism and pharmacokinetic strategies for oligonucleotide- and mRNA-based drug development

(2018) Shalini Andersson et al.   DRUG DISCOVERY TODAY

An Assessment of the In Vitro Inhibition of Cytochrome P450 Enzymes, UDP-Glucuronosyltransferases, and Transporters by Phosphodiester- or Phosphorothioate-Linked Oligonucleotides

(2018) Faraz Kazmi et al.   DRUG METABOLISM AND DISPOSITION

The Nonclinical Safety Profile of GalNAc-conjugated RNAi Therapeutics in Subacute Studies

(2018) Maja M. Janas et al.   TOXICOLOGIC PATHOLOGY

Impurities in Oligonucleotide Drug Substances and Drug Products

(2017) Daniel Capaldi et al.   Nucleic Acid Therapeutics

Recommendations of the Oligonucleotide Safety Working Group's Formulated Oligonucleotide Subcommittee for the Safety Assessment of Formulated Oligonucleotide-Based Therapeutics

(2017) Jennifer L. Marlowe et al.   Nucleic Acid Therapeutics

Assessment of the Drug Interaction Potential of Unconjugated and GalNAc 3 -Conjugated 2′-MOE-ASOs

(2017) Colby S. Shemesh et al.   Molecular Therapy-Nucleic Acids

Disposition and Pharmacokinetics of a GalNAc3-Conjugated Antisense Oligonucleotide Targeting Human Lipoprotein (a) in Monkeys

(2016) Rosie Z. Yu et al.   Nucleic Acid Therapeutics

Exposure to siRNA-GalNAc Conjugates in Systems of the Standard Test Battery for Genotoxicity

(2016) Maja M. Janas et al.   Nucleic Acid Therapeutics

Elucidation of the Biotransformation Pathways of a Galnac3-conjugated Antisense Oligonucleotide in Rats and Monkeys

(2016) Colby S Shemesh et al.   Molecular Therapy-Nucleic Acids

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

(2015) Richard S. Geary et al.   ADVANCED DRUG DELIVERY REVIEWS

Predictive Dose-Based Estimation of Systemic Exposure Multiples in Mouse and Monkey Relative to Human for Antisense Oligonucleotides With 2′-O-(2-Methoxyethyl) Modifications

(2015) Rosie Z Yu et al.   Molecular Therapy-Nucleic Acids

Assessment and Reporting of the Clinical Immunogenicity of Therapeutic Proteins and Peptides—Harmonized Terminology and Tactical Recommendations

(2014) G. Shankar et al.   AAPS Journal

Considerations for Assessment of Reproductive and Developmental Toxicity of Oligonucleotide-Based Therapeutics

(2014) Joy Cavagnaro et al.   Nucleic Acid Therapeutics

Assessing unintended hybridization-induced biological effects of oligonucleotides

(2012) Morten Lindow et al.   NATURE BIOTECHNOLOGY

In Vitro Metabolic Stabilities and Metabolism of 2′-O-(Methoxyethyl) Partially Modified Phosphorothioate Antisense Oligonucleotides in Preincubated Rat or Human Whole Liver Homogenates

(2010) Min-Son Baek et al.   OLIGONUCLEOTIDES

Antisense oligonucleotide pharmacokinetics and metabolism

(2009) Richard S Geary   Expert Opinion on Drug Metabolism & Toxicology

Safety Evaluation of Ocular Drug Delivery Formulations: Techniques and Practical Considerations

(2008) Brian G. Short   TOXICOLOGIC PATHOLOGY