04194nam a22003974u 4500 10.1002/9781119852810 Wiley 20251030120314.0 cr nn 008mam|a 240216t2024||||xxu fs| |0|| 0|eng|| 9781119852810 electronic 10.1002/9781119852810 doi eng Drug Development for Gene Therapy Translational Biomarkers, Bioanalysis, and Companion Diagnostics [electronic resource] Edited by Yanmei Lu, Boris Gorovits. 1 Wiley 2024 Wiley 2024 496pp.; text txt rdacontent computer c rdamedia online resource cr rdacarrier text file rdaft PDF Drug Development for Gene Therapy Industry-centric perspective on translational and bioanalytical challenges and best practices for gene therapies 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 Nonclinical and clinical study considerations and methods for biodistribution and shedding Quantification of transgene protein expression and biochemical function, and substrate and distal pharmacodynamic biomarker measurements for gene therapy Detection and quantification of rAAV integration and off-target editing Current regulatory landscape for gene therapy product development and the role of biomarkers and general regulatory considerations for gene therapy companion diagnostics 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. Drug Discovery & Development 91621 Molecular Techniques 91931 Tissue, Cell, and Genetic Engineering 91405 gene therapy modalities; Clinical Laboratory Improvement Amendments; CLIA; pcr tests; immunoassays; humoral immune responses; AAV capsid proteins; gene therapy regulation; gene therapy safety; cellular immunity Lu, Yanmei edt 91932 Gorovits, Boris edt 91933 Printed edition: 9781119852780 https://doi.org/10.1002/9781119852810 Wiley Online Library https://onlinelibrary.wiley.com/action/showBook?doi=10.1002%2F9781119852810 Wiley Online Library Cover Image https://onlinelibrary.wiley.com/cms/asset/17752019-d00d-409c-9cc8-a904fe0ba998/9781119852810.cover.gif Wiley Online Library 67151 67151