Drug Development for Gene Therapy: Translational Biomarkers, Bioanalysis, and Companion Diagnostics

Drug Development for Gene Therapy provides a comprehensive perspective on translational and bioanalytical challenges and best practices for gene therapies, with a focus on industry-centric perspectives and a significant body of data. The text covers bioanalytical methods and platforms, including patient screening assays, PCR tests, enzyme activity assays, ELISpot, NGS, LC/MS, and immunoassays, with FDA and EMA guidelines on gene therapy safety and efficacy. It also includes clinical examples of relevant therapies and discusses study design, immunogenicity, bioanalytical methods, global regulatory issues, and more. Written by two highly qualified authors with significant experience in the field, Drug Development for Gene Therapy is a valuable resource for researchers and developers in the pharmaceuticals, biopharmaceuticals, and contract research organizations (CROs), as well as professors, researchers, and advanced students in chemistry, biological, biomedical engineering, pharmaceuticals, and medical sciences.

Format: Hardback
Length: 496 pages
Publication date: 31 January 2024
Publisher: John Wiley & Sons Inc

Drug Development for Gene Therapy is a comprehensive guide that provides an industry-centric perspective on the translational and bioanalytical challenges and best practices associated with gene therapies. This text presents a substantial body of data, encompassing information related to safety and efficacy, which is essential for advancing gene therapy modalities through the development pipeline and into clinical use.

The book covers a wide range of bioanalytical methods and platforms, including patient screening assays, various PCR tests, enzyme activity assays, ELISpot, NGS, LC/MS, and immunoassays. It also provides guidelines from the FDA and EMA on gene therapy safety and efficacy, as well as companion diagnostics regulations from the US and EU perspectives.

The chapters in this text 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, clinical examples of relevant therapies are included in related chapters. Some of the core topics covered include study design, immunogenicity, various bioanalytical methods and their applications, and global regulatory issues.

Drug Development for Gene Therapy is written by two highly qualified authors with extensive experience in the field. The book includes information on bioanalytical methods to detect pre-existing antibodies against adeno-associated viruses (AAV) capsids, the detection of cellular immunity and humoral response to viral capsids and transgene proteins, and the immunogenicity of gene therapy products. It also covers nonclinical and clinical study considerations and methods for biodistribution and shedding, as well as quantification of transgene protein expression and biochem.

This comprehensive guide is an invaluable resource for researchers, scientists, and professionals involved in gene therapy development, providing them with the necessary knowledge and tools to overcome the challenges and advance this promising therapeutic approach.

Weight: 908g
Dimension: 159 x 237 x 31 (mm)
ISBN-13: 9781119852780