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Product details:
ISBN13:9781032136554
ISBN10:1032136553
Binding:Hardback
No. of pages:490 pages
Size:254x178 mm
Weight:1070 g
Language:English
Illustrations: 1 Illustrations, black & white; 36 Illustrations, color; 1 Line drawings, black & white; 36 Line drawings, color; 19 Tables, black & white; 1 Tables, color
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Category:

Development of Gene Therapies

Strategic, Scientific, Regulatory, and Access Considerations
 
Edition number: 1
Publisher: Chapman and Hall
Date of Publication:
 
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Publisher's listprice:
GBP 150.00
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Short description:

Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development.

Long description:

Cell and gene therapies have become the third major drug modality in pharmaceutical medicine of the 21st century after low molecular weight and antibody drugs. The gene therapy (GTx) field is rapidly advancing, and yet there are still fundamental scientific questions that remain to be answered. Development of GTx products poses unique challenges and opportunities for drug developers. However, there is lack of a systematic exposition of the GTx product development and the pivotal role of the biostatistician in this process. Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development. Intended to provide an exposition to the GTx new product development through peer-reviewed papers written by subject matter experts in this emerging field, this book will be useful for researchers in gene therapy drug development, biostatisticians, regulators, patient advocates, graduate students, and the finance and business development community .  


Key Features:



  • A collection of papers covering a wide spectrum of topics in gene therapies (GTx), written by leading subject matter experts

  • An exposition of the core principles of GTx product development, emerging business models, industry standards, best practices, and regulatory pathways

  • An exposition of statistical and innovative modeling tools for design and analysis of clinical trials of GTx

  • Insights into commercial models, access hurdles, and health economics of gene therapies

  • Case studies of successful GTx approvals from core team members that developed the first two FDA-approved AAV gene therapies: Luxturna and Zolgensma

  • A discussion of potential benefits and hurdles to be overcome for GTx in coming years from a multi-stakeholder perspective
Table of Contents:

1. Introduction: The Road to Gene Therapy  2. Driving AAV Drug Design to the Right Place, Right Amount, and Right Time  3. A Practical Guide to the Nonclinical Development of In Vivo Gene Therapies  4. Quantitative Systems Pharmacology Modeling of Adeno-Associated Virus Gene Therapies: Mechanistic Identification of Species-Translation Using Preclinical and Clinical Data  5. Bringing Gene Therapy to Patients: A Clinical Development Perspective Based on Brain and Neuromuscular Diseases  6. The Ethics of Gene Therapy  7. AAV Vector Immunogenicity in Gene Therapy: Mechanisms, Assessment, and Immunomodulation Strategies  8. Prenatal Somatic Cell Gene Therapy  9. Development of Gene Therapies for Ultra-Rare Disease  10. Statistical Innovation for Gene Therapy Development: Clinical Trial Design and Analysis Considerations  11. Biomarkers in Gene Therapy Development for Rare Diseases  12. Manufacturing, Analytical, and Process Comparability Challenges for Recombinant Adeno-Associated Virus (rAAV) Gene Therapy  13. Regulatory Considerations in the Development of Gene Therapy Products  14. Gene Therapy Clinical Safety Considerations: Short- and Long-Term  15. Development of Gene Therapies from an Academic Perspective  16. Commercial Models, Access Hurdles, and Health Economics of Gene Therapies  17. The Zolgensma Journey: A Groundbreaking Therapy for SMA  18. History and Development Story of Luxturna: Scientific and Regulatory Challenges  19. The Future of in vivo rAAV Gene Therapies for Rare Neurological Diseases