Pharmaceutical Aspects Of Oligonucleotides

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Pharmaceutical Aspects Of Oligonucleotides


Pharmaceutical Aspects Of Oligonucleotides
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Oligonucleotide Based Drugs And Therapeutics


Oligonucleotide Based Drugs And Therapeutics
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Author : Nicolay Ferrari
language : en
Publisher: John Wiley & Sons
Release Date : 2018-06-06

Oligonucleotide Based Drugs And Therapeutics written by Nicolay Ferrari and has been published by John Wiley & Sons this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-06-06 with Medical categories.


A comprehensive review of contemporary antisense oligonucleotides drugs and therapeutic principles, methods, applications, and research Oligonucleotide-based drugs, in particular antisense oligonucleotides, are part of a growing number of pharmaceutical and biotech programs progressing to treat a wide range of indications including cancer, cardiovascular, neurodegenerative, neuromuscular, and respiratory diseases, as well as other severe and rare diseases. Reviewing fundamentals and offering guidelines for drug discovery and development, this book is a practical guide covering all key aspects of this increasingly popular area of pharmacology and biotech and pharma research, from the basic science behind antisense oligonucleotides chemistry, toxicology, manufacturing, to safety assessments, the design of therapeutic protocols, to clinical experience. Antisense oligonucleotides are single strands of DNA or RNA that are complementary to a chosen sequence. While the idea of antisense oligonucleotides to target single genes dates back to the 1970's, most advances have taken place in recent years. The increasing number of antisense oligonucleotide programs in clinical development is a testament to the progress and understanding of pharmacologic, pharmacokinetic, and toxicologic properties as well as improvement in the delivery of oligonucleotides. This valuable book reviews the fundamentals of oligonucleotides, with a focus on antisense oligonucleotide drugs, and reports on the latest research underway worldwide. • Helps readers understand antisense molecules and their targets, biochemistry, and toxicity mechanisms, roles in disease, and applications for safety and therapeutics • Examines the principles, practices, and tools for scientists in both pre-clinical and clinical settings and how to apply them to antisense oligonucleotides • Provides guidelines for scientists in drug design and discovery to help improve efficiency, assessment, and the success of drug candidates • Includes interdisciplinary perspectives, from academia, industry, regulatory and from the fields of pharmacology, toxicology, biology, and medicinal chemistry Oligonucleotide-Based Drugs and Therapeutics belongs on the reference shelves of chemists, pharmaceutical scientists, chemical biologists, toxicologists and other scientists working in the pharmaceutical and biotechnology industries. It will also be a valuable resource for regulatory specialists and safety assessment professionals and an important reference for academic researchers and post-graduates interested in therapeutics, antisense therapy, and oligonucleotides.

Handbook Of Analysis Of Oligonucleotides And Related Products


Handbook Of Analysis Of Oligonucleotides And Related Products
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Author : Jose V. Bonilla
language : en
Publisher: CRC Press
Release Date : 2011-02-23

Handbook Of Analysis Of Oligonucleotides And Related Products written by Jose V. Bonilla and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2011-02-23 with Medical categories.


Oligonucleotides represent one of the most significant pharmaceutical breakthroughs in recent years, showing great promise as diagnostic and therapeutic agents for malignant tumors, cardiovascular disease, diabetes, viral infections, and many other degenerative disorders. The Handbook of Analysis of Oligonucleotides and Related Products is an essential reference manual on the practical application of modern and emerging analytical techniques for the analysis of this unique class of compounds. A strong collaboration among thirty leading analytical scientists from around the world, the book provides readers with a comprehensive overview of the most commonly used analytical techniques and their advantages and limitations in assuring the identity, purity, quality, and strength of an oligonucleotide intended for therapeutic use. Topics discussed include: Strategies for enzymatic or chemical degradation of chemically modified oligonucleotides toward mass spectrometric sequencing Purity analysis by chromatographic or electrophoretic methods, including RP-HPLC, AX-HPLC, HILIC, SEC, and CGE Characterization of sequence-related impurities in oligonucleotides by mass spectrometry and chromatography Structure elucidation by spectroscopic methods (IR, NMR, MS) as well as base composition and thermal melt analysis (Tm) Approaches for the accurate determination of molar extinction coefficient of oligonucleotides Accurate determination of assay values Assessment of the overall quality of oligonucleotides, including microbial analysis and determination of residual solvents and heavy metals Strategies for determining the chemical stability of oligonucleotides The use of hybridization techniques for supporting pharmacokinetics and drug metabolism studies in preclinical and clinical development Guidance for the presentation of relevant analytical information towards meeting current regulatory expectations for oligonucleotide therapeutics This resource provides a practical guide for applying state-of-the-art analytical techniques in research, development, and manufacturing settings.

Pharmaceutical Aspects Of Oligonucleotides


Pharmaceutical Aspects Of Oligonucleotides
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Author : Patrick Couvreur
language : en
Publisher: CRC Press
Release Date : 2003-09-02

Pharmaceutical Aspects Of Oligonucleotides written by Patrick Couvreur and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2003-09-02 with Medical categories.


Oligonucleotides diffuse poorly through biological barriers, including cell membranes. They are also rapidly degraded in vivo by nucleuses. Aiming to improve the administration of compounds, the book studies the development of nucleotide chemistry.

Synthesis Of Therapeutic Oligonucleotides


Synthesis Of Therapeutic Oligonucleotides
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Author : Satoshi Obika
language : en
Publisher: Springer
Release Date : 2018-12-20

Synthesis Of Therapeutic Oligonucleotides written by Satoshi Obika and has been published by Springer this book supported file pdf, txt, epub, kindle and other format this book has been release on 2018-12-20 with Medical categories.


This book presents the latest knowledge on a broad range of topics relating to the synthesis of natural and artificial oligonucleotides with therapeutic potential. Nucleic acid-based therapeutics are attracting much attention, and numerous therapeutic oligonucleotides, such as antisense oligonucleotides, siRNAs, splice-switching oligonucleotides, and nucleic acid aptamers, are being evaluated in clinical trials for the treatment of a variety of diseases. Synthesis of Therapeutic Oligonucleotides covers a broad range of topics in the field that are of high relevance to researchers, including the synthesis of natural and chemically modified oligonucleotides, the development of novel nucleic acid analogs, industrial scale synthesis and purification of oligonucleotides, and important aspects of chemistry, manufacturing, and controls (CMC). The aim is to provide new insights and inspire fresh ideas in nucleic acid chemistry that may ultimately lead to novel concepts and techniques and the discovery of more effective nucleic acid drugs. The book will be of high value for both established researchers in the field and students intending to specialize in nucleic acid chemistry research.

Therapeutic Oligonucleotides


Therapeutic Oligonucleotides
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Author : Jens Kurreck
language : en
Publisher: Royal Society of Chemistry
Release Date : 2008

Therapeutic Oligonucleotides written by Jens Kurreck and has been published by Royal Society of Chemistry this book supported file pdf, txt, epub, kindle and other format this book has been release on 2008 with Medical categories.


This book provides a compelling overall update on current status of RNA interference

Pharmaceutical Stress Testing


Pharmaceutical Stress Testing
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Author : Steven W. Baertschi
language : en
Publisher: CRC Press
Release Date : 2011-07-27

Pharmaceutical Stress Testing written by Steven W. Baertschi and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2011-07-27 with Medical categories.


The second edition of Pharmaceutical Stress Testing: Predicting Drug Degradation provides a practical and scientific guide to designing, executing and interpreting stress testing studies for drug substance and drug product. This is the only guide available to tackle this subject in-depth. The Second Edition expands coverage from chemical stability into the physical aspects of stress testing, and incorporates the concept of Quality by Design into the stress testing construct / framework. It has been revised and expanded to include chapters on large molecules, such as proteins and antibodies, and it outlines the changes in stress testing that have emerged in recent years. Key features include: A renowned Editorial team and contributions from all major drug companies, reflecting a wealth of experience. 10 new chapters, including Stress Testing and its relationship to the assessment of potential genotoxic degradants, combination drug therapies, proteins, oligonucleotides, physical changes and alternative dosage forms such as liposomal formulations Updated methodologies for predicting drug stability and degradation pathways Best practice models to follow An expanded Frequently Asked Questions section This is an essential reference book for Pharmaceutical Scientists and those working in Quality Assurance and Drug Development (analytical sciences, formulations, chemical process, project management).

Pharmaceutical Biotechnology


Pharmaceutical Biotechnology
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Author : Daan J. A. Crommelin
language : en
Publisher: Springer Science & Business Media
Release Date : 2013-10-22

Pharmaceutical Biotechnology written by Daan J. A. Crommelin and has been published by Springer Science & Business Media this book supported file pdf, txt, epub, kindle and other format this book has been release on 2013-10-22 with Medical categories.


This introductory text explains both the basic science and the applications of biotechnology-derived pharmaceuticals, with special emphasis on their clinical use. It serves as a complete one-stop source for undergraduate/graduate pharmacists, pharmaceutical science students, and for those in the pharmaceutical industry. The Fourth Edition will completely update the previous edition, and will also include additional coverage on the newer approaches such as oligonucleotides, siRNA, gene therapy and nanotech.