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001			10.1002/9781119852810
003			Wiley
005			20251030120314.0
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008			240216t2024||||xxu fs| |0|| 0|eng||
020			_a9781119852810 _qelectronic
024	7		_a10.1002/9781119852810 _2doi
041			_aeng
245	0	0	_aDrug Development for Gene Therapy _bTranslational Biomarkers, Bioanalysis, and Companion Diagnostics _h[electronic resource] _cEdited by Yanmei Lu, Boris Gorovits.
250			_a1
260			_bWiley _c2024
264		1	_bWiley _c2024
300			_a496pp.;
336			_atext _btxt _2rdacontent
337			_acomputer _bc _2rdamedia
338			_aonline resource _bcr _2rdacarrier
347			_atext file _2rdaft
347			_bPDF
520			_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.
650		4	_aDrug Discovery & Development _991621
650		4	_aMolecular Techniques _991931
650		4	_aTissue, Cell, and Genetic Engineering _991405
653	0	0	_agene therapy modalities; Clinical Laboratory Improvement Amendments; CLIA; pcr tests; immunoassays; humoral immune responses; AAV capsid proteins; gene therapy regulation; gene therapy safety; cellular immunity
700	1		_aLu, Yanmei _4edt _991932
700	1		_aGorovits, Boris _4edt _991933
776	0	8	_iPrinted edition: _z9781119852780
856	4	0	_uhttps://doi.org/10.1002/9781119852810 _aWiley Online Library
856	4	0	_uhttps://onlinelibrary.wiley.com/action/showBook?doi=10.1002%2F9781119852810 _aWiley Online Library
856	4	2	_3Cover Image _uhttps://onlinelibrary.wiley.com/cms/asset/17752019-d00d-409c-9cc8-a904fe0ba998/9781119852810.cover.gif _aWiley Online Library
999			_c67151 _d67151