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| bespoke gene therapy consortium: Development of Gene Therapies Avery McIntosh, Oleksandr Sverdlov, 2024-05-23 Cell and gene therapies have become the third major drug modality in pharmaceutical medicine of the 21st century after low molecular weight and antibody drugs. The gene therapy (GTx) field is rapidly advancing, and yet there are still fundamental scientific questions that remain to be answered. Development of GTx products poses unique challenges and opportunities for drug developers. However, there is lack of a systematic exposition of the GTx product development and the pivotal role of the biostatistician in this process. 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. Intended to provide an exposition to the GTx new product development through peer-reviewed papers written by subject matter experts in this emerging field, this book will be useful for researchers in gene therapy drug development, biostatisticians, regulators, patient advocates, graduate students, and the finance and business development community . Key Features: A collection of papers covering a wide spectrum of topics in gene therapies (GTx), written by leading subject matter experts. An exposition of the core principles of GTx product development, emerging business models, industry standards, best practices, and regulatory pathways. An exposition of statistical and innovative modeling tools for design and analysis of clinical trials of GTx. Insights into commercial models, access hurdles, and health economics of gene therapies. Case studies of successful GTx approvals from core team members that developed the first two FDA-approved AAV gene therapies: Luxturna and Zolgensma. A discussion of potential benefits and hurdles to be overcome for GTx in coming years from a multi-stakeholder perspective. |
| bespoke gene therapy consortium: Personal Genome Medicine Michael J. Malinowski, 2023-08-31 In the years following FDA approval of direct-to-consumer, genetic-health-risk testing, millions of people in the United States have sent their DNA to companies to receive personal genetic health risk information without physician or other learned medical professional involvement. In Personal Genome Medicine, Michael J. Malinowski examines the ethical, legal, and social implications of this development. Drawing from the past and present of medicine in the U.S., Malinowski applies law, policy, public and private sector practices, and governing norms to analyze the commercial personal genome sequencing and testing sectors and to assess their impact on the future of U.S. medicine. Written in relatable and accessible language, the book also proposes regulatory reforms for government and medical professionals that will enable technological advancements while maintaining personal and public health standards. |
| bespoke gene therapy consortium: Genetically Modified Organisms , 2024-10-02 This book showcases the most recent advancements in genomics and biotechnology and the ongoing challenges and prospects in creating genetically modified organisms (GMOs). Readers will be acquainted with cutting-edge progress and patterns in gene and genome editing technologies and their diverse applications in medicine, biotechnology, and industry across various organisms. Furthermore, the text delves into the safety considerations and potential uses of GMOs and the regulatory frameworks in different countries. It also presents case studies illustrating how GMOs have catalyzed advancements in medicine, agriculture, and industry. This book consolidates recent discoveries and addresses the informational needs of students and researchers in the field. |
| bespoke gene therapy consortium: AAV Gene Therapy: Immunology and Immunotherapeutics Jose Martinez-Navio, Nicole K. Paulk, Guangping Gao, 2022-02-09 Dr. Gao is the co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics. His research laboratory receives financial support from sponsored research agreements with various companies including Merck and LuYe Pharma. The other Topic Editors declare no conflict of interest with regards to the Research Topic theme |
| bespoke gene therapy consortium: The Forever Fix Ricki Lewis, 2012-03-13 Fascinating narrative science that explores the next frontier in medicine and genetics through the very personal prism of the children and families gene therapy has touched. Eight-year-old Corey Haas was nearly blind from a hereditary disorder when his sight was restored through a delicate procedure that made medical history. Like something from a science fiction novel, doctors carefully injected viruses bearing healing genes into the DNA of Corey's eyes—a few days later, Corey could see, his sight restored by gene therapy. THE FOREVER FIX is the first book to tell the fascinating story of gene therapy: how it works, the science behind it, how patients (mostly children) have been helped and harmed, and how scientists learned from each trial to get one step closer to its immense promise, the promise of a forever fix, - a cure that, by fixing problems at their genetic root, does not need further surgery or medication. Told through the voices of the children and families who have been the inspiration, experimental subjects, and successes of genetic science, THE FOREVER FIX is compelling and engaging narrative science that tells explores the future of medicine as well as the families and scientists who are breaking new ground every day. |
| bespoke gene therapy consortium: Welcome to Your Child's Brain Sandra Aamodt, Sam Wang, 2011-09-08 Neuroscientists Aamodt and Wang illuminate how children's brains grow - and how they can be nurtured, scientifically, to reach their full potential. The authors investigate common child-rearing wisdom, exposing bad brain trainingA products and the ways parents most influence a child's personality. They explain why playing outside improves vision, why teenagers stay up late, and why learning a second language increases empathy. And they share amusing experiments that will let every parent watch a child's grey matter at work. Filled with myth-busting facts and clever advice, this is an indispensable, entertaining guide to your child's brain. |
| bespoke gene therapy consortium: Rare Disease Drug Development Raymond A. Huml, 2021-11-08 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. |
| bespoke gene therapy consortium: Improving Healthcare Quality in Europe Characteristics, Effectiveness and Implementation of Different Strategies OECD, World Health Organization, 2019-10-17 This volume, developed by the Observatory together with OECD, provides an overall conceptual framework for understanding and applying strategies aimed at improving quality of care. Crucially, it summarizes available evidence on different quality strategies and provides recommendations for their implementation. This book is intended to help policy-makers to understand concepts of quality and to support them to evaluate single strategies and combinations of strategies. |
| bespoke gene therapy consortium: Rare Diseases and Orphan Products Institute of Medicine, Board on Health Sciences Policy, Committee on Accelerating Rare Diseases Research and Orphan Product Development, 2011-04-03 Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development. |
| bespoke gene therapy consortium: Nano's Journey Aldo Pourchet, 2021-06 The human body contains many organs, and every organ is made up of countless tiny cells-cells that are born specific to each organ. These Normal Cells understand from the start the job they need to do to keep their home organ healthy.Little Nano is a special kind of cell, a cell with an evolving story: she is a Stem Cell. Born in the Bone Marrow, Nano will eventually become a Normal Cell assigned to one specific organ. But what organ will she eventually call home?To prepare for this big decision, the little Stem Cell will go exploring and get to know the organs of the human body. Ready to meet Nano? She's about to begin her wonderful journey! |
| bespoke gene therapy consortium: The Hippocampus in Clinical Neuroscience K. Szabo, M.G. Hennerici, 2014-04-23 The hippocampus is one of the most intriguing structures of the human brain. Damage to this part causes symptoms ranging from transient disorders accompanied by tiny lesions to severely debilitating cognitive disorders with marked tissue loss. This publication provides a predominantly clinical approach to the complex workings of the hippocampus from different perspectives, ranging from basic principles to specific diseases. The first part of the book summarizes current knowledge regarding the structure and physiology of the hippocampus and establishes the ties to basic neuroscience. The second part deals with the function and assessment of the human hippocampus, including memory function, neuropsychological measures, and conventional and functional imaging studies. The chapters of the third part are devoted to the hippocampus in neurological disorders, e.g. the interaction between stress and memory function, and the pathological conditions of common as well as selected rare neurological diseases affecting the hippocampus. The book is highly recommended to clinical neurologists who wish to gain a broad understanding of this complex and fascinating organ in terms of basic principles, modern imaging findings, and specific diseases. |
| bespoke gene therapy consortium: Rare Diseases Epidemiology: Update and Overview Manuel Posada de la Paz, Domenica Taruscio, Stephen C. Groft, 2017-12-06 The fields of rare diseases research and orphan products development continue to expand with more products in research and development status. In recent years, the role of the patient advocacy groups has evolved into a research partner with the academic research community and the bio-pharmaceutical industry. Unique approaches to research and development require epidemiological data not previously available to assist in protocol study design and patient recruitment for clinical trials required by regulatory agencies prior to approval for access by patents and practicing physicians. |
| bespoke gene therapy consortium: Orphan Drugs and Rare Diseases David Pryde, Michael J Palmer, 2014-07-30 Orphan drugs are designated drug substances that are intended to treat rare or ‘orphan’ diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world’s population; however, individually, any single, rare disease may only affect a handful of people making them commercially unattractive for the biopharmaceutical industry to target. Ground breaking legislation, starting with the Orphan Drug Act that was passed in the US in 1983 to provide financial incentives for companies to develop orphan drugs, has sparked ever increasing interest from biopharmaceutical companies to tackle rare diseases. These developments have made rare diseases, and the orphan drugs that treat them, sufficiently attractive to pharmaceutical development and many pharmaceutical companies now have research units dedicated to this area of research. It is therefore timely to review the area of orphan drugs and some of the basic science, drug discovery and regulatory factors that underpin this important, and growing, area of biomedical research. Written by a combination of academic and industry experts working in the field, this text brings together expert authors in the regulatory, drug development, genetics, biochemistry, patient advocacy group, medicinal chemistry and commercial domains to create a unique and timely reference for all biomedical researchers interested in finding out more about orphan drugs and the rare diseases they treat. Providing an up-to-date monograph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs and will appeal to medicinal and pharmaceutical chemists, biochemists and anyone working within the fields of rare disease research and drug development or pharmaceuticals in industry or academia. |
| bespoke gene therapy consortium: Clinical Mitochondrial Medicine Patrick F. Chinnery, Michael J. Keogh, 2018-05-17 This interactive clinical textbook takes a system- and case-based approach in understanding mitochondrial disorders in clinical practice. |
| bespoke gene therapy consortium: The Health Benefits of Smoking Cessation United States. Public Health Service. Office of the Surgeon General, 1990 |
| bespoke gene therapy consortium: Stem Cells and Prostate Cancer Scott D. Cramer, 2015-02-08 Recent evidence demonstrates that normal prostate tissue contains stem cells. There is also accumulating evidence that prostate cancer contains a population of cells with stem cell-like characteristics referred to as cancer stem cells, or tumor initiating cells. Both the normal prostate stem cell and cancer stem cell populations have important implications for the generation, therapeutic targeting, and prevention of prostate cancer. The purpose of this book is to explore the role of stem cells in prostate cancer, which is becoming an increasingly hot trend in cancer research. |
| bespoke gene therapy consortium: Health Technology Assessment in Japan Isao Kamae, 2019-09-03 Representing the first book on the topic, this work offers the reader an introduction to the Japanese systems for health technology assessment (HTA) officially introduced by the Ministry of Health, Labour and Welfare (MHLW) in 2016. Policy and guidelines are discussed, with the relevant methods and conditions of cost-effectiveness analysis explained alongside. Numerous instructive examples and exercises, ranging from basic to advanced, impart valuable knowledge and insight on the quantitative methods for economic evaluation, which will appeal to both beginners and experts. This guidebook is authored by Japan’s foremost expert in HTA and pharmacoeconomics, with a view to strengthening the reader’s expertise in value-based healthcare and decision-making. The methods presented are essential to informing regulatory, local and patient decisions; as such, the book is equally recommended to industry and government, as well as academia, and anyone with an interest in Japanese HTA. |
| bespoke gene therapy consortium: Superconvergence Jamie Metzl, 2024-06-11 Superconvergence is brilliant. I can't recommend it more strongly.―Sanjay Gupta MD, bestselling author, neurosurgeon, and Emmy-award winning chief medical correspondent (CNN) In Superconvergence, leading futurist and OneShared.World founder Jamie Metzl explores how artificial intelligence, genome sequencing, gene editing, and other revolutionary technologies are transforming our lives, world, and future. These accelerating and increasingly interconnected technologies have the potential to improve our health, feed billions of people, supercharge our economies, store essential information for millions of years, and save our planet, but they can also―if we are not careful―do immeasurable harm. The challenge we face is that while our ability to engineer the world around us is advancing exponentially, our processes for understanding the scope, scale, and implications of these changes, and for managing our godlike powers wisely, are only inching forward glacially. Luckily, in Jamie Metzl, we have a leading expert who integrates science, technology, history, politics, and international affairs to envision a future that most specialists, almost by definition, cannot see. In this bold and inspiring exploration of transformative human knowledge, Metzl gives us the definitive account of the technological precipice on which we stand and the map to where we go from here. |
| bespoke gene therapy consortium: Gene Therapy and Ethics Anders Nordgren, 1999 |
| bespoke gene therapy consortium: Practical Psychopharmacology Joseph F. Goldberg, Stephen M. Stahl, 2021-04-29 A practical guide translating clinical trials findings, across major psychiatric disorders, to devise tailored, evidence-based treatments. |
| bespoke gene therapy consortium: Ultimate Pulmonary Wellness Noah Greenspan, 2017-09-21 Ultimate Pulmonary Wellness is a resource for all people living with respiratory disease including patients, their families and caretakers; and clinicians. This well-rounded guidebook is the fusion of twenty-five years of clinical practice, education and research by Dr. Noah Greenspan, board-certified clinical specialist in cardiovascular and pulmonary physical therapy; and Program Director of the Pulmonary Wellness & Rehabilitation Center in New York City. It is one of the most comprehensive works of its kind. This brand new first edition draws together a complex variety of threads, clearly defining the key components of living well with a pulmonary disease; including the anatomy, physiology and pathophysiology of the respiratory system; the multifactorial and multi-systemic nature of breathing; the role of medicine (physician, diagnosis and treatment) in the management and prevention of respiratory disease; and the importance of lifestyle factors, such as exercise, nutrition and managing your emotions, as well as the prevention of infection; in ultimate pulmonary wellness; and living your absolute best life with respiratory disease. |
| bespoke gene therapy consortium: Mammalian Synthetic Biology Jamie A. Davies, Paul S. Freemont, 2020 This primer introduces the challenges and opportunities of applying synthetic biological techniques to mammalian cells, tissues, and organisms. It covers the special features that make engineering mammalian systems different from engineering bacteria, fungi, and plants, and provides an overview of current techniques. A variety of cutting-edge examples illustrate the different purposes of mammalian synthetic biology, including pure biomedical research, drug production, tissue engineering, and regenerative medicine. |
| bespoke gene therapy consortium: Toward Precision Medicine National Research Council, Division on Earth and Life Studies, Board on Life Sciences, Committee on A Framework for Developing a New Taxonomy of Disease, 2012-01-16 Motivated by the explosion of molecular data on humans-particularly data associated with individual patients-and the sense that there are large, as-yet-untapped opportunities to use this data to improve health outcomes, Toward Precision Medicine explores the feasibility and need for a new taxonomy of human disease based on molecular biology and develops a potential framework for creating one. The book says that a new data network that integrates emerging research on the molecular makeup of diseases with clinical data on individual patients could drive the development of a more accurate classification of diseases and ultimately enhance diagnosis and treatment. The new taxonomy that emerges would define diseases by their underlying molecular causes and other factors in addition to their traditional physical signs and symptoms. The book adds that the new data network could also improve biomedical research by enabling scientists to access patients' information during treatment while still protecting their rights. This would allow the marriage of molecular research and clinical data at the point of care, as opposed to research information continuing to reside primarily in academia. Toward Precision Medicine notes that moving toward individualized medicine requires that researchers and health care providers have access to very large sets of health- and disease-related data linked to individual patients. These data are also critical for developing the information commons, the knowledge network of disease, and ultimately the new taxonomy. |
| bespoke gene therapy consortium: The Coming Wave Mustafa Suleyman, 2023-09-05 NEW YORK TIMES BESTSELLER • An urgent warning of the unprecedented risks that AI and other fast-developing technologies pose to global order, and how we might contain them while we have the chance—from a co-founder of the pioneering artificial intelligence company DeepMind and current CEO of Microsoft AI “A fascinating, well-written, and important book.”—Yuval Noah Harari “Essential reading.”—Daniel Kahneman “An excellent guide for navigating unprecedented times.”—Bill Gates A Best Book of the Year: CNN, Economist, Bloomberg, Politico Playbook, Financial Times, The Guardian, CEO Magazine, Semafor • Winner of the Inc. Non-Obvious Book Award • Finalist for the Porchlight Business Book Award and the Financial Times and Schroders Business Book of the Year Award We are approaching a critical threshold in the history of our species. Everything is about to change. Soon you will live surrounded by AIs. They will organize your life, operate your business, and run core government services. You will live in a world of DNA printers and quantum computers, engineered pathogens and autonomous weapons, robot assistants and abundant energy. None of us are prepared. As co-founder of the pioneering AI company DeepMind, part of Google, Mustafa Suleyman has been at the center of this revolution. The coming decade, he argues, will be defined by this wave of powerful, fast-proliferating new technologies. In The Coming Wave, Suleyman shows how these forces will create immense prosperity but also threaten the nation-state, the foundation of global order. As our fragile governments sleepwalk into disaster, we face an existential dilemma: unprecedented harms on one side, the threat of overbearing surveillance on the other. Can we forge a narrow path between catastrophe and dystopia? Is it possible to contain the threat of AI? This groundbreaking book from the ultimate AI insider establishes “the containment problem”—the task of maintaining control over powerful technologies—as the essential challenge of our age. |
| bespoke gene therapy consortium: Toxicity Testing in the 21st Century National Research Council, Division on Earth and Life Studies, Institute for Laboratory Animal Research, Board on Environmental Studies and Toxicology, Committee on Toxicity Testing and Assessment of Environmental Agents, 2007-10-05 Advances in molecular biology and toxicology are paving the way for major improvements in the evaluation of the hazards posed by the large number of chemicals found at low levels in the environment. The National Research Council was asked by the U.S. Environmental Protection Agency to review the state of the science and create a far-reaching vision for the future of toxicity testing. The book finds that developing, improving, and validating new laboratory tools based on recent scientific advances could significantly improve our ability to understand the hazards and risks posed by chemicals. This new knowledge would lead to much more informed environmental regulations and dramatically reduce the need for animal testing because the new tests would be based on human cells and cell components. Substantial scientific efforts and resources will be required to leverage these new technologies to realize the vision, but the result will be a more efficient, informative and less costly system for assessing the hazards posed by industrial chemicals and pesticides. |
| bespoke gene therapy consortium: Breast Cancer Management for Surgeons Lynda Wyld, Christos Markopoulos, Marjut Leidenius, Elżbieta Senkus-Konefka, 2017-11-29 This textbook provides a comprehensive overview of the state of the art knowledge of breast cancer management for the modern breast surgeon. It covers all aspects of multidisciplinary care including primary breast and axillary surgery, reconstruction and oncoplastic techniques, external beam radiotherapy and newer techniques such as tomotherapy, intraoperative radiotherapy and brachytherapy. In addition, systemic therapies including chemotherapy, the latest biological targeted therapies and endocrine therapies are covered. Readers can find out about other important aspects of breast cancer such as genetics, screening, imaging and long-term health among others. Chapters take the reader through the basics up to the highest levels of knowledge in an easy to understand format with management algorithms to aid clinical care, generous referencing of the best literature and figures and photographs to illustrate each section. Published with the official approval of the European Society of Surgical Oncology (ESSO) and the European Society of Breast Cancer Specialists (EUSOMA), the book is written by a panel of recognised leaders in the field and is an indispensable guide for the practicing breast specialist and senior specialists in training, wishing to update their knowledge with the latest trends or polish off their training before accreditation. |
| bespoke gene therapy consortium: Newborn Screening for Pompe Disease Wuh-Liang Hwu, Yin-Hsiu Chien, Raymond Wang, 2021-09-02 Pompe disease, also known as acid maltase deficiency or acid alpha-glucosidase deficiency, in its most severe form results in a rapidly progressive, neonatal-onset skeletal and cardiomyopathy, leading to early infantile death without treatment. The development of treatment with recombinant enzyme replacement therapy radically transformed the clinical trajectory of those affected, enabling long-term ventilator-free survival with resolution of cardiomyopathy. These positive clinical outcomes resulted in the implementation of newborn screening programs for Pompe disease across the world. This Special Issue highlights some of the experiences of Pompe screening programs worldwide and discusses public policy and ethical issues elicited by presymptomatic screening for Pompe disease. |
| bespoke gene therapy consortium: Textbook of Personalized Medicine Kewal K. Jain, 2015-03-17 Advances in the technology used in personalized medicine and increased applications for clinical use have created a need for this expansion and revision of Kewal K. Jain’s Textbook of Personalized Medicine. As the first definitive work on this topic, this book reviews the fundamentals and development of personalized medicine and subsequent adoptions of the concepts by the biopharmaceutical industry and the medical profession. It also discusses examples of applications in key therapeutic areas, as well as ethical and regulatory issues, providing a concise and comprehensive source of reference for those involved in healthcare management, planning and politics. Algorithms are included as a guide to those involved in the management of important diseases where decision-making is involved due to the multiple choices available. Textbook of Personalized Medicine, Second Edition will serve as a convenient source of information for physicians, scientists, decision makers in the biopharmaceutical and healthcare industries and interested members of the public. |
| bespoke gene therapy consortium: The Biomedical Sciences in Society Iain Crinson, 2021-03-02 This textbook provides a comprehensive introduction to the interdisciplinary field of the Social Studies of Science and Technology (SSST). Over the past two decades, the biomedical sciences have transformed our understanding of the relationship between the social and natural worlds, while its ‘promissory visions’ are seen to offer extraordinary opportunities for economic and social development. But alongside these scientific innovations have emerged new, and frequently unanticipated social, political, bioethical, and legal dilemmas and challenges. This cutting-edge text explores ‘post-genomic’ developments in the field of pharmacogenomics and the prospects for a new ‘precision’ or personalised medicine; the potential of environmental epigenetics to reconfigure the boundaries of the social and natural worlds; the emergence of an array of ‘neuro-disciplines’, seeking to identify the neural basis of a whole range of social and economic behaviours; and the challenges of constructing a coherent and robust governance framework for the conduct of biomedical science research and innovation, responsive to the social and health needs of the whole population. |
| bespoke gene therapy consortium: Registries for Evaluating Patient Outcomes Agency for Healthcare Research and Quality/AHRQ, 2014-04-01 This User’s Guide is intended to support the design, implementation, analysis, interpretation, and quality evaluation of registries created to increase understanding of patient outcomes. For the purposes of this guide, a patient registry is an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves one or more predetermined scientific, clinical, or policy purposes. A registry database is a file (or files) derived from the registry. Although registries can serve many purposes, this guide focuses on registries created for one or more of the following purposes: to describe the natural history of disease, to determine clinical effectiveness or cost-effectiveness of health care products and services, to measure or monitor safety and harm, and/or to measure quality of care. Registries are classified according to how their populations are defined. For example, product registries include patients who have been exposed to biopharmaceutical products or medical devices. Health services registries consist of patients who have had a common procedure, clinical encounter, or hospitalization. Disease or condition registries are defined by patients having the same diagnosis, such as cystic fibrosis or heart failure. The User’s Guide was created by researchers affiliated with AHRQ’s Effective Health Care Program, particularly those who participated in AHRQ’s DEcIDE (Developing Evidence to Inform Decisions About Effectiveness) program. Chapters were subject to multiple internal and external independent reviews. |
| bespoke gene therapy consortium: Vaccination Ethics and Policy Jason L. Schwartz, Arthur L. Caplan, 2021-08-24 A comprehensive overview of important and contested issues in vaccination ethics and policy by experts from history, science, policy, law, and ethics. Vaccination has long been a familiar, highly effective form of medicine and a triumph of public health. Because vaccination is both an individual medical intervention and a central component of public health efforts, it raises a distinct set of legal and ethical issues—from debates over their risks and benefits to the use of government vaccination requirements—and makes vaccine policymaking uniquely challenging. This volume examines the full range of ethical and policy issues related to the development and use of vaccines in the United States and around the world. Forty essays, articles, and reports by experts in the field look at all aspects of the vaccine life cycle. After an overview of vaccine history, they consider research and development, regulation and safety, vaccination promotion and requirements, pandemics and bioterrorism, and the frontier of vaccination. The texts cover such topics as vaccine safety controversies; the ethics of vaccine trials; vaccine injury compensation; vaccine refusal and the risks of vaccine-preventable diseases; equitable access to vaccines in emergencies; lessons from the eradication of smallpox; and possible future vaccines against cancer, malaria, and Ebola. The volume intentionally includes texts that take opposing viewpoints, offering readers a range of arguments. The book will be an essential reference for professionals, scholars, and students. Contributors Jeffrey P. Baker, Seth Berkley, Luciana Borio, Arthur L. Caplan, R. Alta Charo, Dave A. Chokshi, James Colgrove, Katherine M. Cook, Louis Z. Cooper, Edward Cox, Douglas S. Diekema, Ezekiel J. Emanuel, Claudia I. Emerson, Geoffrey Evans, Ruth R. Faden, Chris Feudtner, David P. Fidler, Fiona Godlee, D. A. Henderson, Alan R. Hinman, Peter Hotez, Robert M. Jacobson, Aaron S. Kesselheim, Heidi J. Larson, Robert J. Levine, Donald W. Light, Adel Mahmoud, Edgar K. Marcuse, Howard Markel, Michelle M. Mello, Paul A. Offit, Saad B. Omer, Walter A. Orenstein, Gregory A. Poland, Lance E. Rodewald, Daniel A. Salmon, Anne Schuchat, Jason L. Schwartz, Peter A. Singer, Michael Specter, Alexandra Minna Stern, Jeremy Sugarman, Thomas R. Talbot, Robert Temple, Stephen P. Teret, Alan Wertheimer, Tadataka Yamada |
| bespoke gene therapy consortium: Oncoplastic Breast Surgery Techniques for the General Surgeon V. Suzanne Klimberg, Tibor Kovacs, Isabel T. Rubio, 2020-05-22 This book covers an up-to-date review of advances in the management strategies for patients with breast cancer and their co-morbidities. Oncoplasty has become the standard of care for breast conservation surgery and can lead to improving aesthetic outcomes of breast cancer surgery, without compromising oncological outcomes. The goal of the text is to increase the competencies and performance of healthcare professionals involved in treating this patient population, which will ultimately improve the aesthetic outcomes, quality of life and overall survival of patients with breast disease and breast cancer. Written by experts in the field, chapters address a wide range of breast surgery techniques that help the general surgeon provide improved oncologic and aesthetic results for patients. The authors have detailed not only best practice for conventional procedures but also new and evolving techniques. Oncoplastic Breast Surgery Techniques for the General Surgeon serves as a valuable resource not only for medical students but also for the general surgeon seeking to improve results in private practice and for professors learning and teaching new breast surgery techniques. |
| bespoke gene therapy consortium: High Quality Care for All Secretary of State for Health, 2008 This review incorporates the views and visions of 2,000 clinicians and other health and social care professionals from every NHS region in England, and has been developed in discussion with patients, carers and the general public. The changes proposed are locally-led, patient-centred and clinically driven. Chapter 2 identifies the challenges facing the NHS in the 21st century: ever higher expectations; demand driven by demographics as people live longer; health in an age of information and connectivity; the changing nature of disease; advances in treatment; a changing health workplace. Chapter 3 outlines the proposals to deliver high quality care for patients and the public, with an emphasis on helping people to stay healthy, empowering patients, providing the most effective treatments, and keeping patients as safe as possible in healthcare environments. The importance of quality in all aspects of the NHS is reinforced in chapter 4, and must be understood from the perspective of the patient's safety, experience in care received and the effectiveness of that care. Best practice will be widely promoted, with a central role for the National Institute for Health and Clinical Excellence (NICE) in expanding national standards. This will bring clarity to the high standards expected and quality performance will be measured and published. The review outlines the need to put frontline staff in control of this drive for quality (chapter 5), with greater freedom to use their expertise and skill and decision-making to find innovative ways to improve care for patients. Clinical and managerial leadership skills at the local level need further development, and all levels of staff will receive support through education and training (chapter 6). The review recommends the introduction of an NHS Constitution (chapter 7). The final chapter sets out the means of implementation. |
| bespoke gene therapy consortium: Foundations of Regenerative Medicine Anthony Atala, 2009-09-04 The interdisciplinary field of regenerative medicine holds the promise of repairing and replacing tissues and organs damaged by disease and of developing therapies for previously untreatable conditions, such as diabetes, heart disease, liver disease, and renal failure. Derived from the fields of tissue engineering, cell and developmental biology, biomaterials science, nanotechnology, physics, chemistry, physiology, molecular biology, biochemistry, bioengineering, and surgery, regenerative medicine is one of the most influential topics of biological research today.Derived from the successful Principles of Regenerative Medicine, this volume brings together the latest information on the advances in technology and medicine and the replacement of tissues and organs damaged by disease. Chapters focus on the fundamental principles of regenerative therapies that have crossover with a broad range of disciplines. From the molecular basis to therapeutic applications, this volume is an essential source for students, researchers, and technicians in tissue engineering, stem cells, nuclear transfer (therapeutic cloning), cell, tissue, and organ transplantation, nanotechnology, bioengineering, and medicine to gain a comprehensive understanding of the nature and prospects for this important field. - Highlights the fundamentals of regenerative medicine to relate to a variety of related science and technology fields - Introductory chapter directly addresses why regenerative medicine is important to a variety of researchers by providing practical examples and references to primary literature - Includes new discoveries from leading researchers on restoration of diseased tissues and organs |
| bespoke gene therapy consortium: Rare Diseases Epidemiology Manuel Posada de la Paz, Stephen C. Groft, 2010-09-18 In our etiologic research, we epidemiologists need to leave behind the concepts of ‘cohort’ study and ‘case–control’ study and adopt that of the etiologic study as the singular substitute for these. With this sentence, the famous epidemiologist Professor Olli S. Miettinen began his personal re ection on the future of the epidemiology [1]. He sought to highlight the fact that the role of the epidemiologist should be mainly focused on aetiological research. Nevertheless, the widespread idea still exists that epidemiology is limited to purely providing gures and descriptive data on the frequency and distribution of disease. Indeed, it is more than likely that the precise aim of those rst classic epidemiological steps, i. e. , methods essentially based on describing the distri- tion of a given disease, is still not all that well understood by many scientists, let alone the general public. Such descriptions seek to generate hypotheses and afford explanations for key factors (be these risk factors or the presumable causes th- selves), which might justify differences in terms of persons, time or place and, in turn, ultimately serve to develop preventive measures and/or gain quality-adjusted life years. To restrict the goals of epidemiology to activities exclusively concerned with reporting gures or even complex statistical results is a great mistake, one that renders it dif cult to take full advantage of the epidemiologist’s true role, which is “to study disease determinants and to assess the actual impact of factors involved in their development, distribution and dissemination”. |
| bespoke gene therapy consortium: Rethinking the Clinical Gaze John Gardner, 2017-05-31 This book draws on medical sociology and science and technology studies to develop a novel conceptual framework for understanding innovation processes, using the case study of deep brain stimulation in paediatric neurology. It addresses key questions, including: How are promising and potentially disruptive new health technologies integrated into busy resource-constrained clinical contexts? What activities are involved in establishing a new clinical service? How do social and cultural forces shape these services, and importantly, how are understandings of ‘health’ and ‘illness’ reconfigured in the process? The book explores how the ideals of patient-centred medicine influence innovation in the clinic, and it introduces the concept of patient-centred proto-platforms. It argues that patient-centred innovation can constitute an expansion of medical power, as the clinical gaze is directed not only towards the body but also towards the patient as a social being. This will be an innovative and insightful read for academics and advanced students, as well as health service researchers with an interest in technology adoption processes. |
| bespoke gene therapy consortium: Chasing My Cure David Fajgenbaum, 2019-09-10 LOS ANGELES TIMES AND PUBLISHERS WEEKLY BESTSELLER • The powerful memoir of a young doctor and former college athlete diagnosed with a rare disease who spearheaded the search for a cure—and became a champion for a new approach to medical research. “A wonderful and moving chronicle of a doctor’s relentless pursuit, this book serves both patients and physicians in demystifying the science that lies behind medicine.”—Siddhartha Mukherjee, New York Times bestselling author of The Emperor of All Maladies and The Gene David Fajgenbaum, a former Georgetown quarterback, was nicknamed the Beast in medical school, where he was also known for his unmatched mental stamina. But things changed dramatically when he began suffering from inexplicable fatigue. In a matter of weeks, his organs were failing and he was read his last rites. Doctors were baffled by his condition, which they had yet to even diagnose. Floating in and out of consciousness, Fajgenbaum prayed for a second chance, the equivalent of a dramatic play to second the game into overtime. Miraculously, Fajgenbaum survived—only to endure repeated near-death relapses from what would eventually be identified as a form of Castleman disease, an extremely deadly and rare condition that acts like a cross between cancer and an autoimmune disorder. When he relapsed while on the only drug in development and realized that the medical community was unlikely to make progress in time to save his life, Fajgenbaum turned his desperate hope for a cure into concrete action: Between hospitalizations he studied his own charts and tested his own blood samples, looking for clues that could unlock a new treatment. With the help of family, friends, and mentors, he also reached out to other Castleman disease patients and physicians, and eventually came up with an ambitious plan to crowdsource the most promising research questions and recruit world-class researchers to tackle them. Instead of waiting for the scientific stars to align, he would attempt to align them himself. More than five years later and now married to his college sweetheart, Fajgenbaum has seen his hard work pay off: A treatment he identified has induced a tentative remission and his novel approach to collaborative scientific inquiry has become a blueprint for advancing rare disease research. His incredible story demonstrates the potency of hope, and what can happen when the forces of determination, love, family, faith, and serendipity collide. Praise for Chasing My Cure “A page-turning chronicle of living, nearly dying, and discovering what it really means to be invincible in hope.”—Angela Duckworth, #1 New York Times bestselling author of Grit “[A] remarkable memoir . . . Fajgenbaum writes lucidly and movingly . . . Fajgenbaum’s stirring account of his illness will inspire readers.”—Publishers Weekly |
| bespoke gene therapy consortium: Drug Safety Data: How to Analyze, Summarize and Interpret to Determine Risk Michael J. Klepper, Barton Cobert, 2010-09-15 Drug Safety Data: How to Analyze, Summarize and Interpret to Determine Risk was selected for The First Clinical Research Bookshelf - Essential reading for clinical research professionals by the Journal of Clinical Research Best Practices. Drug Safety Data: How to Analyze, Summarize and Interpret to Determine Risk provides drug safety/pharmacovogilance professionals, pharmaceutical and clinical research scientists, statisticians, programmers, medical writers, and technicians with an accessible, practical framework for the analysis, summary and interpretation of drug safety data. The only guide of its kind, Drug Safety Data: How to Analyze, Summarize and Interpret to Determine Risk is an invaluable reference for pre- and post-marketing risk assessment. With decades of pharmaceutical research and drug safety expertise, authors Dr. Klepper and Dr. Cobert discuss how quality planning, safety training, and data standardization result in significant cost, time, and resource savings. Through illustrative, step-by-step instruction, Drug Safety Data: How to Analyze, Summarize and Interpret to Determine Risk is the definitive guide to drug safety data analysis and reporting. Key features include: * Step-by-step instruction on how to analyze, summarize and interpret safety data for mandatory governmental safety reports * Pragmatic tips...and mistakes to avoid * Simple explanations of what safety data are collected, and what the data mean * Practical approaches to determining a drug effect and understanding its clinical significance * Guidance for determining risk throughout the lifecycle of a drug, biologic or nutraceutical * Examples of user-friendly data displays that enhance safety signal identification * Ways to improve data quality and reduce the time, resources and costs involved in mandatory safety reporting * Relevant material for the required training of drug safety/pharmacovigilance professionals * SPECIAL FEATURE: Actual examples of an Integrated Analysis of Safety (IAS) -used in the preparation of the Integrated Summary of Safety (ISS) and the Summary of Clinical Safety (SCS) reports -, and the Periodic Safety Update Report (PSUR) |
| bespoke gene therapy consortium: The Oxford Handbook of Public Health Ethics Anna C. Mastroianni, Jeffrey P. Kahn, Nancy E. Kass, 2019-07-23 Natural disasters and cholera outbreaks. Ebola, SARS, and concerns over pandemic flu. HIV and AIDS. E. coli outbreaks from contaminated produce and fast foods. Threats of bioterrorism. Contamination of compounded drugs. Vaccination refusals and outbreaks of preventable diseases. These are just some of the headlines from the last 30-plus years highlighting the essential roles and responsibilities of public health, all of which come with ethical issues and the responsibilities they create. Public health has achieved extraordinary successes. And yet these successes also bring with them ethical tension. Not all public health successes are equally distributed in the population; extraordinary health disparities between rich and poor still exist. The most successful public health programs sometimes rely on policies that, while improving public health conditions, also limit individual rights. Public health practitioners and policymakers face these and other questions of ethics routinely in their work, and they must navigate their sometimes competing responsibilities to the health of the public with other important societal values such as privacy, autonomy, and prevailing cultural norms. This Oxford Handbook provides a sweeping and comprehensive review of the current state of public health ethics, addressing these and numerous other questions. Taking account of the wide range of topics under the umbrella of public health and the ethical issues raised by them, this volume is organized into fifteen sections. It begins with two sections that discuss the conceptual foundations, ethical tensions, and ethical frameworks of and for public health and how public health does its work. The thirteen sections that follow examine the application of public health ethics considerations and approaches across a broad range of public health topics. While chapters are organized into topical sections, each chapter is designed to serve as a standalone contribution. The book includes 73 chapters covering many topics from varying perspectives, a recognition of the diversity of the issues that define public health ethics in the U.S. and globally. This Handbook is an authoritative and indispensable guide to the state of public health ethics today. |
| bespoke gene therapy consortium: Digital Pathology Liron Pantanowitz, Anil V. Parwani, 2017 The definitive, complete reference of digital pathology! An extraordinarily comprehensive and complete book for individuals with anything from minimal knowledge to deep, accomplished experience in digital pathology. Easy to read and plainly written, Digital Pathology examines the history and technological evolution of digital pathology, from the birth of scanning technology and telepathology to three-dimensional imaging on large multi-touch displays and computer aided diagnosis. A must-have book for anyone wishing to learn more about and work in this exciting and critical information environment including pathologists, laboratory professionals, students and any other medical practitioners with a particular interest in the history and future of digital pathology. It can also be a useful reference for anyone, medical or non-medical, who have an interest in learning more about the field. Digital pathology is truly a game changer, and this book is a crucial tool for anyone wishing to know more. Subjects discussed in depth include: Static digital imaging; basics and clinical use. Digital imaging processes. Telepathology. While slide imaging. Clinical applications of whole slide imaging. Digital pathology for educational, quality improvement, research and other settings. Forensic digital imaging. |
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Natural History Studies and Registries in the Development of …
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Minutes of the Consortium Assembly Meeting - IRDiRC
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fnih.org
Feb 5, 2024 · The Accelerating Medicines Partnerships® (AMP®) Bespoke Gene Therapy Consortium (BGTC) recognized the need for a comprehensive playbook that would serve as a …
Foundation for the National Institutes of Health Disease …
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fnih.org
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CELL & GENE THERAPY INSIGHTS
ACCELERATES BESPOKE GENE THERAPIES FOR RARE DISEASES While rare diseases imply small patient populations, collectively it is estimated that between 263–446 million people …
NIAMS Research – Focus on Genomics and Genetics
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Creating a sustainable business model for cell and gene …
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Souad Messahel, PhD - California
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Généthon rejoint le consortium américain Bespoke Gene …
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Accelerating Medicines Partnership - FNIH
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Cell and Gene Therapy F14: The Ethical Conduct of Research: …
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Elpida Therapeutics Announces Two Programs to be …
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Gene therapy for the eyes and ears: hopes and challenges
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Rare Disease Community & Federal Agencies – A Critical …
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The 7th International Forum of Lysosomal Disorders 第7回国際
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May 2024 NCATS Advisory Council Minutes - National Center …
Platform Vector Gene Therapy (PaVe-GT) and the Bespoke Gene Therapy Consortium (BGTC), and is anticipated to move more quickly as a CAN-specific activity. President Joseph R. Biden …
June 22, 2021 Joni L. Rutter, Ph.D. Acting Director Bethesda …
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2024 Issue Number 1 NEWSLETTER - Généthon
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Alert | Health Care & FDA Practice
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ELSI Friday Forum Friday, January 10, 2025 12-1:30 PM ET …
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January 22, 2024 The Honorable Bill Cassidy, M.D. Ranking …
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Neurological Cell and Gene Therapy Development: Industry …
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ARM Gene Therapy Advisory Committee Town Hall
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* WG Co-Chairs - FNIH
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Alert | Health Care & FDA Practice
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Patients with Rare Diseases” - Congress.gov
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NCATS FY 2024–2029 Draft Strategic Plan Framework
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