Translational Medicine

Translational Medicine: Tools and Techniques provides a standardized path from basic research to the clinic and brings together various policy and practice issues to simplify the broad interdisciplinary field. With discussions from academic and industry leaders at international institutions who have successfully implemented translational medicine techniques and tools in various settings, readers will be guided through implementation strategies relevant to their own needs and institutions. The book also addresses regulatory processes in USA, EU, Japan and China. By providing details on omics sciences techniques, biomarkers, data mining and management approaches, case reports from industry, and tools to assess the value of different technologies and techniques, this book is the first to provide a user-friendly go-to guide for key opinion leaders (KOLs), industry administrators, faculty members, clinicians, researchers, and students interested in translational medicine. Includes detailed and standardized information about the techniques and tools used in translational medicine Provides specific industry case scenarios Explains how to use translational medicine tools and techniques to plan and improve infrastructures and capabilities while reducing cost and optimizing resources

Principles of Translational Science in Medicine: From Bench to Bedside, Second Edition, provides an update on major achievements in the translation of research into medically relevant results and therapeutics. The book presents a thorough discussion of biomarkers, early human trials, and networking models, and includes institutional and industrial support systems. It also covers algorithms that have influenced all major areas of biomedical research in recent years, resulting in an increasing numbers of new chemical/biological entities (NCEs or NBEs) as shown in FDA statistics. The book is ideal for use as a guide for biomedical scientists to establish a systematic approach to translational medicine. Provides an in-depth description of novel tools for the assessment of translatability of trials to balance risk and improve projects at any given stage of product development New chapters deal with translational issues in the fastest growing population (the elderly), case studies, translatability assessment tools, and advances in nanotherapies Details IPR issues of translation, especially for public-private-partnerships Contains contributions from world leaders in translational medicine, including the former NIH director and authorities from various European regulatory institutions

Biomaterials in Translational Medicine delivers timely and detailed information on the latest advances in biomaterials and their role and impact in translational medicine. Key topics addressed include the properties and functions of these materials and how they might be applied for clinical diagnosis and treatment. Particular emphasis is placed on basic fundamentals, biomaterial formulations, design principles, fabrication techniques and transitioning bench-to-bed clinical applications. The book is an essential reference resource for researchers, clinicians, materials scientists, engineers and anyone involved in the future development of innovative biomaterials that drive advancement in translational medicine. Systematically introduces the fundamental principles, rationales and methodologies of creating or improving biomaterials in the context of translational medicine Includes the translational or commercialization status of these new biomaterials Provides the reader with enough background knowledge for a fundamental grip of the difficulties and technicalities of using biomaterial translational medicine Directs the reader on how to find other up-to-date sources (i.e. peer reviewed journals) in the field of translational medicine and biomaterials

Translational Regenerative Medicine is a reference book that outlines the life cycle for effective implementation of discoveries in the dynamic field of regenerative medicine. By addressing science, technology, development, regulatory, manufacturing, intellectual property, investment, financial, and clinical aspects of the field, this work takes a holistic look at the translation of science and disseminates knowledge for practical use of regenerative medicine tools, therapeutics, and diagnostics. Incorporating contributions from leaders in the fields of translational science across academia, industry, and government, this book establishes a more fluid transition for rapid translation of research to enhance human health and well-being. Provides formulaic coverage of the landscape, process development, manufacturing, challenges, evaluation, and regulatory aspects of the most promising regenerative medicine clinical applications Covers clinical aspects of regenerative medicine related to skin, cartilage, tendons, ligaments, joints, bone, fat, muscle, vascular system, hematopoietic /immune system, peripheral nerve, central nervous system, endocrine system, ophthalmic system, auditory system, oral system, respiratory system, cardiac system, renal system, hepatic system, gastrointestinal system, genitourinary system Identifies effective, proven tools and metrics to identify and pursue clinical and commercial regenerative medicine

Translational Medicine: Optimizing Preclinical Safety Evaluation of Biopharmaceuticals provides scientists responsible for the translation of novel biopharmaceuticals into clinical trials with a better understanding of how to navigate the obstacles that keep innovative medical research discoveries from becoming new therapies or even making it to clinical trials. The book includes sections on protein-based therapeutics, modified proteins, oligonucleotide-based therapies, monoclonal antibodies, antibody–drug conjugates, gene and cell-based therapies, gene-modified cell-based therapies, combination products, and therapeutic vaccines. Best practices are defined for efficient discovery research to facilitate a science-based, efficient, and predictive preclinical development program to ensure clinical efficacy and safety. Key Features: Defines best practices for leveraging of discovery research to facilitate a development program Includes general principles, animal models, biomarkers, preclinical toxicology testing paradigms, and practical applications Discusses rare diseases Discusses "What-Why-When-How" highlighting different considerations based upon product attributes. Includes special considerations for rare diseases About the Editors Joy A. Cavagnaro is an internationally recognized expert in preclinical development and regulatory strategy with an emphasis on genetic medicines.. Her 40-year career spans academia, government (FDA), and the CRO and biotech industries. She was awarded the 2019 Arnold J Lehman Award from the Society of Toxicology for introducing the concept of science-based, case-by-case approach to preclinical safety evaluation, which became the foundation of ICH S6. She currently serves on scientific advisory boards for advocacy groups and companies and consults and lectures in the area of preclinical development of novel therapies. Mary Ellen Cosenza is a regulatory toxicology consultant with over 30 years of senior leadership experience in the biopharmaceutical industry in the U.S., Europe, and emerging markets. She has held leadership position in both the American College of Toxicology (ACT) and the International Union of Toxicology (IUTOX) and is also an adjunct assistant professor at the University of Southern California where she teaches graduate-level courses in toxicology and regulation of biologics.

Examines Critical Decisions for Transitioning Lab Science to a Clinical Setting The development of therapeutic pharmaceutical compounds is becoming more expensive, and the success rates for getting such treatments approved for marketing and to the patients is decreasing. As a result, translational medicine (TM) is becoming increasingly important in the healthcare industry – a means of maximizing the consideration and use of information collected as compounds transition from initial lab discovery, through pre-clinical testing, early clinical trials, and late confirmatory studies that lead to regulatory approval of drug release to patients. Translational Medicine: Strategies and Statistical Methods suggests a process for transitioning from the initial lab discovery to the patient’s bedside with minimal disconnect and offers a comprehensive review of statistical design and methodology commonly employed in this bench-to-bedside research. Documents Alternative Research Approaches for Faster and More Accurate Data Judgment Calls Elaborating on how to introduce TM into clinical studies, this authoritative work presents a keen approach to building, executing, and validating statistical models that consider data from various phases of development. It also delineates a truly translational example to help bolster understanding of discussed concepts. This comprehensive guide effectively demonstrates how to overcome obstacles related to successful TM practice. It contains invaluable information for pharmaceutical scientists, research executives, clinicians, and biostatisticians looking to expedite successful implementation of this important process.

Clinical and Translational Science: Principles of Human Research, Second Edition, is the most authoritative and timely resource for the broad range of investigators taking on the challenge of clinical and translational science, a field that is devoted to investigating human health and disease, interventions, and outcomes for the purposes of developing new treatment approaches, devices, and modalities to improve health. This updated second edition has been prepared with an international perspective, beginning with fundamental principles, experimental design, epidemiology, traditional and new biostatistical approaches, and investigative tools. It presents complete instruction and guidance from fundamental principles, approaches, and infrastructure, especially for human genetics and genomics, human pharmacology, research in special populations, the societal context of human research, and the future of human research. The book moves on to discuss legal, social, and ethical issues, and concludes with a discussion of future prospects, providing readers with a comprehensive view of this rapidly developing area of science. Introduces novel physiological and therapeutic strategies for engaging the fastest growing scientific field in both the private sector and academic medicine Brings insights from international leaders into the discipline of clinical and translational science Addresses drug discovery, drug repurposing and development, innovative and improved approaches to go/no-go decisions in drug development, and traditional and innovative clinical trial designs

Translational Cardiometabolic Genomic Medicine, edited by Dr. Annabelle Rodriguez-Oquendo, is an important resource to postgraduate (medical, dental and graduate) students, postdoctoral fellows, basic scientists, and physician scientists seeking to understand and expand their knowledge base in the field of genomic medicine as it is applied to cardiometabolic diseases. This handbook integrates cutting-edge experimental approaches such as chromatin immunoprecipitation paired end tagging (CHIA-PET), to population studies such as the Multi-Ethnic Study of Atherosclerosis. It encompasses a range of book chapters that highlight bioinformatic approaches to better understanding functionality of the noncoding regions of the human genome to the use of molecular diagnostic testing in predicting increased risk of cardiovascular diseases. Where applicable, this reference also includes chapters related to therapeutic options specifically aligned to molecular targets. Provides comprehensive research on translational genomic medicine Explains state-of-the-art genome editing for stem cells and mouse models with significant relevance to human cardiometabolic disease Includes discussions on the functional effects of single nucleotide polymorphisms and cardiometabolic diseases, stratified by sex and race Encompasses a range of book chapters that highlight bioinformatic approaches to better understanding functionality of the noncoding regions of the human genome