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OverviewDrug 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. Full Product DetailsAuthor: Yanmei Lu (Sangamo Therapeutics) , Boris Gorovits (Gorovits BioSolutions, LLC)Publisher: John Wiley & Sons Inc Imprint: John Wiley & Sons Inc ISBN: 9781119852780ISBN 10: 1119852781 Pages: 496 Publication Date: 31 January 2024 Audience: Professional and scholarly , Professional & Vocational Format: Hardback Publisher's Status: Active Availability: In stock We have confirmation that this item is in stock with the supplier. It will be ordered in for you and dispatched immediately. Table of ContentsReviewsAuthor InformationYanmei 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. Tab Content 6Author Website:Countries AvailableAll regions |