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Oligonucleotide-Based Drugs and Therapeutics: Preclinical and Clinical Considerations for Development

Oligonucleotide-Based Drugs and Therapeutics: Preclinical and Clinical Considerations for Development

Nicolay Ferrari, Rosanne Seguin

ISBN: 978-1-119-07030-6

Jul 2018

560 pages


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Antisense-based drugs are part of a growing number of pharmaceutical and biotech programs to treat diseases including cancer, diabetes, and ALS. Reviewing fundamentals and offering guidelines for drug discovery and development, this book is a practical guide from the safety assessment to clinical experience with antisense oligonucleotides.

• Examines principles and tools for scientists in both pre-clinical and clinical settings and how to apply them to antisense oligonucleotides
• Reviews fundamentals and provides guidelines for scientists in drug design and discovery to increase efficiency, assessment, and success of drug candidates
• Helps readers understand antisense molecules and their targets, biochemistry and toxicity mechanisms, roles in disease, and applications for safety and therapeutics
• Includes interdisciplinary perspectives, from both academia and research, on a hot topic that combines pharmacology, toxicology, biology, and medicinal chemistry to advance our understanding and application of therapeutics

List of Contributors


Table of Contents

Chapter 1 Mechanisms of Oligonucleotide Actions

Chapter 2 The Medicinal Chemistry of Antisense Oligonucleotides

Chapter 3 Cellular Pharmacology of Antisense Oligonucleotides

Chapter 4 Pharmacokinetics and Pharmacodynamics of Antisense Oligonucleotides

Chapter 5 Tissue Distribution, Metabolism and Clearance

Chapter 6 Hybridization-independent Effects - Principles and Specific Considerations for Oligonucleotide Drugs

Chapter 7 Hybridization-Dependent Effects – The Prediction, Evaluation and Consequences of Unintended Target Hybridization

Chapter 8 Class-related proinflammatory effects

Chapter 9 Exaggerated Pharmacology

Chapter 10 Genotoxicity Tests for Novel Oligonucleotide-Based Therapeutics

Chapter 11 Reproductive and Developmental Toxicity Testing Strategies for Oligonucleotide-based Therapeutics

Chapter 12 Specific Considerations for Preclinical Development of Inhaled Oligonucleotides

Chapter 13 Lessons Learned in Oncology Programs

Chapter 14 Inhaled Antisense for Treatment of Respiratory Disease

Chapter 15 Antisense oligonucleotides for treatment of neurology diseases

Chapter 16 Nucleic Acids as Adjuvants

Chapter 17 Splice-switching Oligonucleotides

Chapter 18 CMC Aspects for the Clinical Development of Spiegelmers