Drug development for gene therapy : translational biomarkers, bioanalysis, and companion diagnostics / edited by Yanmei Lu, Boris Gorovits.

Other author Lu, Yanmei, 1966- editor.
Other author Gorovits, Boris, editor.
Format Electronic
PublicationHoboken, New Jersey : John Wiley & Sons, Inc., [2024]
Description1 online resource (xxiv, 465 pages) : illustrations (chiefly color)
Supplemental ContentDirect link to eBook
Subjects
Gene therapy.
Drugs--Design.
Genetic Therapy--methods.
Biomarkers, Pharmacological--analysis.
Drug Development--methods.
Genetic Therapy.
Drug Design.

Contents Introduction to AAV-based in vivo gene therapy -- Recent development in in vivo clinical gene therapy platforms -- Overview of biomarkers for AAV gene therapy -- Nonclinical and Clinical Study Considerations for Biodistribution, Shedding and Pharmacokinetics / Pharmacodynamics -- Immunogenicity of gene therapy products -- Bioanalytical Methods To Detect Pre-existing and Post-administration Humoral Immune Responses Against AAV Capsid Proteins -- Bioanalytical methods to study biodistribution and shedding of AAV-based gene therapy vectors -- Transgene mRNA expression analysis -- Quantification of transgene protein expression and biochemical function -- Substrate and Distal Pharmacodynamic Biomarker Measurements for Gene Therapy -- Detection of cellular immunity to viral capsids and transgene proteins -- Detection of humoral response to viral capsids and transgene proteins -- rAAV integration : Detection and Risk Assessment -- Gene modification and integration for patient monitoring -- Introduction to companion diagnostics for gene therapy -- Validation for Gene Therapy Companion Diagnostics -- Regulatory considerations for gene therapy companion diagnostics -- Current regulatory landscape for gene therapy product development and the role of biomarkers.
Abstract "This book focuses on the translational and bioanalytical challenges and best practices for gene therapy modalities. Some of the topics covered include study design, immunogenicity, various bioanalytical methods and their applications, and global regulatory issues. Altogether, 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 industry and research"-- Provided by publisher.
Bibliography noteIncludes bibliographical references and index.
Source of descriptionDescription based on online resource; title from digital title page (viewed on March 26, 2024).
Issued in other formPrint version: Drug development for gene therapy Hoboken, New Jersey : John Wiley & Sons, Inc., [2024] 9781119852780
LCCN 2023049873
ISBN9781119852810 electronic book
ISBN1119852811 electronic book
ISBN1119852803 electronic book
ISBN9781119852797 electronic book
ISBN111985279X electronic book
ISBN9781119852803 (electronic bk.)
ISBNhardcover
Standard identifier# 10.1002/9781119852810