04148nam a22003854u 450000100220000000300060002200500170002800700150004500800410006002000300010102400310013104100080016224501650017025000060033526000160034126400160035730000120037333600260038533700260041133800360043734700210047334700080049452025040050265000400300665000320304665000490307865302150312770000270334270000320336977600360340185600640343785601020350185601420360399900170374510.1002/9781119852810Wiley20251030120314.0cr nn 008mam|a240216t2024||||xxu    fs|   |0|| 0|eng||  a9781119852810qelectronic7 a10.1002/97811198528102doi  aeng00aDrug Development for Gene TherapybTranslational Biomarkers, Bioanalysis, and Companion Diagnosticsh[electronic resource]cEdited by Yanmei Lu, Boris Gorovits.  a1  bWileyc2024 1bWileyc2024  a496pp.;  atextbtxt2rdacontent  acomputerbc2rdamedia  aonline resourcebcr2rdacarrier  atext file2rdaft  bPDF  aDrug 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. 4aDrug Discovery & Development991621 4aMolecular Techniques991931 4aTissue, Cell, and Genetic Engineering99140500agene therapy modalities; Clinical Laboratory Improvement Amendments; CLIA; pcr tests; immunoassays; humoral immune responses; AAV capsid proteins; gene therapy regulation; gene therapy safety; cellular immunity1 aLu, Yanmei4edt9919321 aGorovits, Boris4edt99193308iPrinted edition:z978111985278040uhttps://doi.org/10.1002/9781119852810aWiley Online Library40uhttps://onlinelibrary.wiley.com/action/showBook?doi=10.1002%2F9781119852810aWiley Online Library423Cover Imageuhttps://onlinelibrary.wiley.com/cms/asset/17752019-d00d-409c-9cc8-a904fe0ba998/9781119852810.cover.gifaWiley Online Library  c67151d67151