Drug Development for Gene Therapy
John Wiley & Sons Inc (Verlag)
978-1-119-85278-0 (ISBN)
»Drug Development for Gene Therapy« focuses on the translational and bioanalytical challenges and best practices for gene therapy modalities, presenting a significant body of data, including information related to safety and efficacy, necessary to advance through the development pipeline into clinical use. The text covers bioanalytical methods and platforms including patient screening assays, different PCR tests, enzyme activity assays, ELISpot, NGS, LC/MS, and immunoassays, with FDA and EMA guidelines on gene therapy safety and efficacy, along with companion diagnostics regulations from US and EU perspectives.
The chapters offer an in-depth discussion of the basics and best practices for translational biomarkers, bioanalysis, and developing companion diagnostics / lab tests for gene therapies in the pharma and biopharma industries. To aid in reader comprehension, the text includes clinical examples of relevant therapies in related chapters. Some of the core topics covered include study design, immunogenicity, various bioanalytical methods and their applications, and global regulatory issues.
Written by two highly qualified authors with significant experience in the field, »Drug Development for Gene Therapy« includes information on:
- Bioanalytical methods to detect pre-existing antibodies against adeno-associated viruses (AAV) capsids
- Detection of cellular immunity and humoral response to viral capsids and transgene proteins, and immunogenicity of gene therapy products/li>
- Nonclinical and clinical study considerations and methods for biodistribution and shedding/li>
- Quantification of transgene protein expression and biochemical function, and substrate and distal pharmacodynamic biomarker measurements for gene therapy/li>
- Detection and quantification of rAAV integration and off-target editing/li>
- Current regulatory landscape for gene therapy product development and the role of biomarkers and general regulatory considerations for gene therapy companion diagnostics/li>
With comprehensive coverage of the subject, »Drug Development for Gene Therapy« is a must-have resource for researchers and developers in the areas of pharmaceuticals, biopharmaceuticals, and contract research organizations (CROs), along with professors, researchers, and advanced students in chemistry, biological, biomedical engineering, pharmaceuticals, and medical sciences.
Yanmei Lu, PhD is currently the Vice President of Biomarker and BioAnalytical Sciences at Sangamo Therapeutics and previously worked at Genentech. Dr. Lu has a PhD in Biochemistry and Molecular Biology and has published 50+ peer-reviewed articles and book chapters.
Boris Gorovits, PhD is currently the Principal at Gorovits BioSolutions, LLC and previously worked at Sana Biotherapeutics, Pfizer and Wyeth Research. Dr. Gorovits has published 80+ journal articles and book chapters.
List of contributors
Preface
Section I: Introduction
1. Introduction to AAV-based in vivo gene therapy
2. Recent development in in vivo clinical gene therapy platforms
Section II: Translational biomarkers for gene therapy
3. Overview of biomarkers for AAV gene therapy
4. Nonclinical and Clinical Study Considerations for Biodistribution, Shedding and Pharmacokinetics / Pharmacodynamics
5. Immunogenicity of gene therapy products
Section III: Bioanalysis for gene therapy
6. Bioanalytical Methods To Detect Pre-existing and Post-administration Humoral Immune Responses Against AAV Capsid Proteins
7. Bioanalytical methods to study biodistribution and shedding of AAV-based gene therapy vectors
8. Transgene mRNA expression analysis
9. Quantification of transgene protein expression and biochemical function
10. Substrate and Distal Pharmacodynamic Biomarker Measurements for Gene Therapy
11. Detection of cellular immunity to viral capsids and transgene proteins
12. Detection of humoral response to viral capsids and transgene proteins
13. rAAV integration: Detection and Risk Assessment
14. Gene modification and integration for patient monitoring
Section IV: Companion diagnostic development for gene therapy
15. Introduction to companion diagnostics for gene therapy
16. Validation for Gene Therapy Companion Diagnostics
17. Regulatory considerations for gene therapy companion diagnostics
Section V: Regulatory perspectives on gene therapy
18. Current regulatory landscape for gene therapy product development and the role of biomarkers
Index
| Erscheinungsdatum | 02.02.2024 |
|---|---|
| Verlagsort | New York |
| Sprache | englisch |
| Gewicht | 1012 g |
| Einbandart | gebunden |
| Themenwelt | Medizin / Pharmazie ► Medizinische Fachgebiete ► Biomedizin |
| Medizin / Pharmazie ► Medizinische Fachgebiete ► Pharmakologie / Pharmakotherapie | |
| Medizin / Pharmazie ► Pharmazie | |
| Naturwissenschaften ► Biologie ► Biochemie | |
| Naturwissenschaften ► Chemie ► Analytische Chemie | |
| Schlagworte | Antiangiogene Gentherapie • Arzneimittelentwicklung • Bioanalyse • Biomarker • Biomarker (Zelle) • Gentherapie • Translationale Medizin |
| ISBN-10 | 1-119-85278-1 / 1119852781 |
| ISBN-13 | 978-1-119-85278-0 / 9781119852780 |
| Zustand | Neuware |
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