基因治疗药物开发：转化生物标志物、生物分析和伴随诊断

English | 2024 | ISBN: 1119852781 | 478 pages | PDF, True EPUB | 18.05 MB

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.

基因治疗药物开发

从产业视角来看，基因疗法的转化和生物分析挑战以及最佳实践

基因治疗药物开发重点关注基因治疗模式的转化和生物分析挑战及最佳实践，并呈现在一系列数据中，包括与安全性和有效性相关的信息，以推进通过研发管道并用于临床使用。该文本涵盖了一系列生物分析方法和技术平台，例如患者筛选试验、不同的PCR测试、酶活性测定、ELISpot、NGS、LC/MS和免疫分析法，以及来自FDA和EMA的基因治疗安全性及有效性指导原则，还包括从美国和欧盟视角下的伴随诊断法规。

章节内容深入讨论了基因疗法在制药和生物制药行业中的转化生物标志物、生物分析及开发伴随诊断或实验室测试的最佳实践。为了帮助读者理解，文本中还包含相关章节内的临床实例。一些核心话题包括研究设计、免疫原性、各种生物分析方法及其应用以及全球监管问题。

由在该领域经验丰富的两位作者撰写的《基因治疗药物开发》涵盖了：

检测腺病毒载体（AAV）壳体上存在的抗腺相关病毒（AAV）的预存抗体的方法 检测对病毒壳体和转基因蛋白的细胞免疫及血清应答，以及基因疗法产品的免疫原性 非临床和临床研究中用于分布和脱落的相关考量和方法 基因治疗转录因子蛋白表达量的定量及生物化学功能分析 针对基因治疗的代谢物和远端药效动力学生物标志物测定 检测并定量逆转录病毒载体（rAAV）整合及脱靶编辑 关于基因疗法产品开发的当前监管环境以及伴随诊断中生物标志物及一般性监管考量

《基因治疗药物开发》内容全面，是制药、生物制药和合同研究组织（CROs）领域研究人员和开发者的必备资源，也是化学、生物学、 biomedical工程学、医药学和医学科学相关领域的教授、研究人员及高级学生的重要参考。

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