Drug Development For Gene Therapy
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Development Of Gene Therapies
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Author : Avery McIntosh
language : en
Publisher: Chapman & Hall/CRC Biostatistics Series
Release Date : 2024-03-13
Development Of Gene Therapies written by Avery McIntosh and has been published by Chapman & Hall/CRC Biostatistics Series this book supported file pdf, txt, epub, kindle and other format this book has been release on 2024-03-13 with categories.
Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development.
Drug Development For Gene Therapy
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Author : Yanmei Lu
language : en
Publisher: John Wiley & Sons
Release Date : 2024-02-28
Drug Development For Gene Therapy written by Yanmei Lu and has been published by John Wiley & Sons this book supported file pdf, txt, epub, kindle and other format this book has been release on 2024-02-28 with Medical categories.
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.
Biopharmaceutical Drug Design And Development
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Author : Susanna Wu-Pong
language : en
Publisher: Springer Science & Business Media
Release Date : 2010-01-11
Biopharmaceutical Drug Design And Development written by Susanna Wu-Pong and has been published by Springer Science & Business Media this book supported file pdf, txt, epub, kindle and other format this book has been release on 2010-01-11 with Medical categories.
This book provides a comprehensive examination of the newest biopharmaceutical drugs. Among the drugs discussed are ones in the categories of monoclonal antibodies for in-vivo use, cytokines, growth factors, enzymes, immunomodulators, thrombolytics, and immonotherapies including vaccines. Additionally, the volume examines new and emerging technologies, and contains a review of the Human Genome Project.
Gene Profiles In Drug Design
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Author : Brett A. Lidbury
language : en
Publisher: CRC Press
Release Date : 2008-07-10
Gene Profiles In Drug Design written by Brett A. Lidbury and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2008-07-10 with Medical categories.
With the successful mapping of the human genome, we have entered an age of unprecedented opportunity in which researchers are beginning to apply this vast repository of knowledge to the treatment of human disease. Gene-profiling technologies and the concept of individualized medicine are leading to the development of drugs with enhanced specificity
Rare Disease Drug Development
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Author : Raymond A. Huml
language : en
Publisher: Springer Nature
Release Date : 2021-11-08
Rare Disease Drug Development written by Raymond A. Huml and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-11-08 with Medical categories.
This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case for real-world data and evidence generation in orphan medicinal drug development. Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers.
Current Topics In Nonclinical Drug Development
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Author : Pritam S. Sahota
language : en
Publisher: CRC Press
Release Date : 2020-12-23
Current Topics In Nonclinical Drug Development written by Pritam S. Sahota and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-12-23 with Medical categories.
The inaugural volume in the Current Topics in Nonclinical Drug Development Series explores the critical issues and current topics in nonclinical drug development. This first volume covers individual topics and strategies in drug development from compound characterization to drug registration. Written by a variety of experts in the field, recent and rapid advances in technologies and associated changes in regulatory guidance are discussed. Additional features include: Deals with day-to-day issues in study design, evaluation of findings, and presentation of data. Explains new approaches in the development of medical devices. Includes dedicated chapters on the use of bioinformatics in drug development. Addresses strategies for photosafety testing of drugs. Current Topics in Nonclinical Drug Development, Volume I will aid toxicologists, toxicologic pathologists, consultants, regulators, Study Directors, and nonclinical scientists dealing with day-to-day issues in study design, evaluation of findings, and presentation of data. In addition, the book will be a valuable reference for academicians and graduate students pursuing research related to nonclinical drug development.
Genome Based Therapeutics
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Author : Institute of Medicine
language : en
Publisher: National Academies Press
Release Date : 2012-11-21
Genome Based Therapeutics written by Institute of Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2012-11-21 with Medical categories.
The number of new drug approvals has remained reasonably steady for the past 50 years at around 20 to 30 per year, while at the same time the total spending on health-related research and development has tripled since 1990. There are many suspected causes for this trend, including increases in regulatory barriers, the rising costs of scientific inquiry, a decrease in research and development efficiency, the downstream effects of patient expirations on investment, and the lack of production models that have successfully incorporated new technology. Regardless, this trajectory is not economically sustainable for the businesses involved, and, in response, many companies are turning toward collaborative models of drug development, whether with other industrial firms, academia, or government. Introducing greater efficiency and knowledge into these new models and aligning incentives among participants may help to reverse the trends highlighted above, while producing more effective drugs in the process. Genome-Based Therapeutics explains that new technologies have the potential to open up avenues of development and to identify new drug targets to pursue. Specifically, improved validation of gene-disease associations through genomics research has the potential to revolutionize drug production and lower development costs. Genetic information has helped developers by increasing their understanding of the mechanisms of disease as well as individual patients' reactions to their medications. There is a need to identify the success factors for the various models that are being developed, whether they are industry-led, academia-led, or collaborations between the two. Genome-Based Therapeutics summarizes a workshop that was held on March 21, 2012, titled New Paradigms in Drug Discovery: How Genomic Data Are Being Used to Revolutionize the Drug Discovery and Development Process. At this workshop the goal was to examine the general approaches being used to apply successes achieved so far, and the challenges ahead.
Gene Therapy
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Author : Gavin Brooks
language : en
Publisher:
Release Date : 2002
Gene Therapy written by Gavin Brooks and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2002 with Medical categories.
Considers the genetic basis of disease and gene therapies, concentrating in detail on specific genetic illnesses, such as AIDS, cancer and cardiovascular diseases, and their treatment. This text should be of interest to pharmacists and to those involved in the biological or medical sciences.
Biotechnology In The Modern Medicinal System
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Author : Rajesh K. Kesharwani
language : en
Publisher: CRC Press
Release Date : 2021-12-23
Biotechnology In The Modern Medicinal System written by Rajesh K. Kesharwani and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-12-23 with Science categories.
Biotechnology in the Modern Medicinal System: Advances in Gene Therapy, Immunotherapy, and Targeted Drug Delivery presents an informative picture of the state-of-the-art research and development of actionable knowledge in medical biotechnology, specifically involving gene therapy, immunotherapy, and targeted drug delivery systems. The book includes novel approaches for therapy of various ailments and the real-world challenges and complexities of the current drug delivery methodologies and techniques. The volume helps to bridge the gap between academic research and real-time clinical applications and the needs of medical biotechnology methods. This edited book also provides a detailed application of medical biotechnology in drug discovery and the treatment of various deadly diseases. Chapters discuss targeted drug delivery to specific sites to avoid possible entry to non-targeted sites, minimizing adverse effects. The volume provides information about the roles of alternative routes of drug targeting, like intranasal and transdermal, resulting in improving patient compliance. Targeted drug delivery is explored for several health issues, such as neurodegenerative disorders, cancer, malaria, and hemoglobin disorders. Also considered is the role of genes in various genetic diseases and gene therapy, and immunogene therapy as alternative approaches to conventional cancer therapy. Finally, the book investigates the important role of computers in biotechnology to accelerate research and development in the modern medicinal field for better and optimum results. Studies show that significant improvement has been observed in the development of a faster and less invasive diagnostic system for the treatment of diseases by utilizing both artificial intelligence (AI) and biotechnology. This valuable volume provides a wealth of information that will be valuable to scientists and researchers, faculty, and students.
Exploring Novel Clinical Trial Designs For Gene Based Therapies
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Author : National Academies of Sciences, Engineering, and Medicine
language : en
Publisher: National Academies Press
Release Date : 2020-08-27
Exploring Novel Clinical Trial Designs For Gene Based Therapies written by National Academies of Sciences, Engineering, and Medicine and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2020-08-27 with Medical categories.
Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.