Advertisement
| cell and gene therapy insights: Cell and Gene Therapies Miguel-Angel Perales, Syed A. Abutalib, Catherine Bollard, 2018-11-27 In this book, experts in the field express their well-reasoned opinions on a range of complex, clinically relevant issues across the full spectrum of cell and gene therapies with the aim of providing trainee and practicing hematologists, including hematopoietic transplant physicians, with information that is relevant to clinical practice and ongoing research. Each chapter focuses on a particular topic, and the concise text is supported by numerous working tables, algorithms, and figures. Whenever appropriate, guidance is provided regarding the availability of potentially high-impact clinical trials. The rapid evolution of cell and gene therapies is giving rise to numerous controversies that need to be carefully addressed. In meeting this challenge, this book will appeal to all residents, fellows, and faculty members responsible for the care of hematopoietic cell transplant patients. It will also offer a robust, engaging tool to aid vital activities in the daily work of every hematology and oncology trainee. |
| cell and gene therapy insights: Exploring Novel Clinical Trial Designs for Gene-Based Therapies National Academies of Sciences, Engineering, and Medicine, Health and Medicine Division, Board on Health Sciences Policy, Forum on Regenerative Medicine, 2020-08-27 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. |
| cell and gene therapy insights: Gene and Cell Therapies Eve Hanna, Mondher Toumi, 2020-05-19 The major advances in the field of biotechnology and molecular biology in the twenty-first century have led to a better understanding of the pathophysiology of diseases. A new generation of biopharmaceuticals has emerged, including a wide and heterogeneous range of innovative cell and gene therapies. These therapies aim to prevent or treat chronic and serious life-threatening diseases, previously considered incurable. This book describes the evolution and adaptation of the regulatory environment to assess these therapies in contrast with the resistance of health technology assessment (HTA) agencies and payers to acknowledge the specificity of cell and gene therapies and the need to adapt existing decision-making frameworks. This book provides insights on the learnings from the experience of current cell and gene therapies (regulatory approval, HTA, and market access), in addition to future trends to enhance patient access to these therapies. Key Features: Describes the potential change of treatment paradigm and the specificity of cell and gene therapies, including the gradual move from repeated treatment administration to one-time single administration with the potential to be definite cure Highlights the challenges at the HTA level Discusses the affordability of future cell and gene therapies and the possible challenges for health insurance systems Provides potential solutions to address these challenges and ensure patient access to innovation while maintaining the sustainability of healthcare systems |
| cell and gene therapy insights: Regulatory Aspects of Gene Therapy and Cell Therapy Products Maria Cristina Galli, Mercedes Serabian, 2015-09-15 This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent must-know guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals. |
| cell and gene therapy insights: Therapeutic Oligonucleotides Jens Kurreck, 2008 This book provides a compelling overall update on current status of RNA interference |
| cell and gene therapy insights: Ex Vivo Cell Therapy Klaus Schindhelm, Robert Nordon, 1999 R.E. Nordon and K. Schindhelm, Introduction. -- L. Robb, A.G. Elefanty, and C.G. Begley, Transcriptional Control of Hematopoieses. -- R. Starr and N.A. Nicola, Cell Signaling by Hemopoietic Growth Factor Receptors. -- P.J. Simmons, D.N. Haylock, and J.-P. Lévesque, Influence of Cytokines and Adhesion Molecules on Hematopoietic Stem Cell Development. -- P.A. Rowlings, Allogeneic Hematopoietic Stem Cell Transplantation. -- U. Hahn and L.B. To, Autologous Stem Cell Transplantation. -- M.R. Vowels, Cord Blood Stem Cell Transplantation. -- S.R. Riddell, E.H. Warren, D. Lewinsohn, C. Yee, and P.D. Greenberg, Reconstitution of Immunity by Adoptive Immunotherapy with T Cells. -- L.Q. Sun, M. Miller, and G. Symonds, Exogenous Gene Transfer into Lymphoid and Hematopoietic Progenitor Cells. -- C. Dowding, T. Leemhuis, A. Jakubowski, and C. Reading, Process Development for Ex Vivo Cell Therapy. -- R.E. Nordon and K. Schindhelm, Cell Separation. -- P.W. Zandstra, C.J. Eaves, and J.M. Piret, Environ ... |
| cell and gene therapy insights: Stem Cell Biology and Gene Therapy Peter J. Quesenberry, Gary S. Stein, Bernard G. Forget, Sherman M. Weissman, 1998-09-10 STEM CELL BIOLOGY AND GENE THERAPY Edited by Peter J. Quesenberry, Gary S. Stein, Bernard Forget, and Sherman Weissman Advances in molecular genetics and recombinant DNA technology have ushered in a new era in medical therapeutic research. New insights into the molecular basis of human disease and the role played by biological regulatory mechanisms have precipitated tremendous drug development efforts backed by intensive research into human gene therapy worldwide. Stem Cell Biology and Gene Therapy is the first book to thoroughly cover major advances in the field and their applications to novel molecular therapies. This self-contained volume integrates biological and clinical components of stem cell biology, examines some of the most difficult aspects of gene therapy, and provides a systematic review of advanced gene modification techniques. Twenty essays by leading researchers address some of the most compelling topics in contemporary medical research, including: * Fundamental regulatory mechanisms that operate in stem cells * Stem cells from a therapeutic perspective, including preparations of stem cells and their therapeutic potential as vehicles for gene therapy * Delivery systems for therapeutic genes, including an overview of the most promising vectors * Clinical applications for gene therapy, covering a broad range of diseases such as hemophilia, cancers, neurological disease, and more Complete with illustrations and real-world examples of a variety of disorders, Stem Cell Biology and Gene Therapy is essential for researchers in gene therapy and members of the biotechnology industry who are developing human molecular therapies for commercial use. It is also an important reference for molecular biologists, cell biologists, immunologists, molecular geneticists, hematologists, cancer researchers, biochemists, and anyone working in internal medicine. |
| cell and gene therapy insights: Stem Cell Manufacturing Joaquim M.S. Cabral, Claudia Lobato da Silva, Lucas G. Chase, M. Margardia Diogo, 2016-07-24 Stem Cell Manufacturing discusses the required technologies that enable the transfer of the current laboratory-based practice of stem cell tissue culture to the clinic environment as therapeutics, while concurrently achieving control, reproducibility, automation, validation, and safety of the process and the product. The advent of stem cell research unveiled the therapeutic potential of stem cells and their derivatives and increased the awareness of the public and scientific community for the topic. The successful manufacturing of stem cells and their derivatives is expected to have a positive impact in the society since it will contribute to widen the offer of therapeutic solutions to the patients. Fully defined cellular products can be used to restore the structure and function of damaged tissues and organs and to develop stem cell-based cellular therapies for the treatment of cancer and hematological disorders, autoimmune and other inflammatory diseases and genetic disorders. - Presents the first 'Flowchart' of stem cell manufacturing enabling easy understanding of the various processes in a sequential and coherent manner - Covers all bioprocess technologies required for the transfer of the bench findings to the clinic including the process components: cell signals, bioreactors, modeling, automation, safety, etc. - Presents comprehensive coverage of a true multidisciplinary topic by bringing together specialists in their particular area - Provides the basics of the processes and identifies the issues to be resolved for large scale cell culture by the bioengineer - Addresses the critical need in bioprocessing for the successful delivery of stem cell technology to the market place by involving professional engineers in sections of the book |
| cell and gene therapy insights: Cellular Transplantation Craig Halberstadt, Dwaine F. Emerich, 2011-10-10 There have been tremendous strides in cellular transplantation in recent years, leading to accepted practice for the treatment of certain diseases, and use for many others in trial phases. The long history of cellular transplantation, or the transfer of cells from one organism or region of the body to another, has been revolutionized by advances in stem cell research, as well as developments in gene therapy. Cellular Transplants: From Lab to Clinic provides a thorough foundation of the basic science underpinning this exciting field, expert overviews of the state-of-the-art, and detailed description of clinical success stories to date, as well as insights into the road ahead. As highlighted by this timely and authoritative survey, scale-up technologies and whole organ transplantation are among the hurdles representing the next frontier. The contents are organized into four main sections, with the first covering basic biology, including transplant immunology, the use of immunosuppressive drugs, stem cell biology, and the development of donor animals for transplantation. The next part looks at peripheral and reconstructive applications, followed by a section devoted to transplantation for diseases of the central nervous system. The last part presents efforts to address the key challenges ahead, such as identifying novel transplantable cells and integrating biomaterials and nanotechnology with cell matrices. - Provides detailed description of clinical trials in cell transplantation - Review of current therapeutic approaches - Coverage of the broad range of diseases addressed by cell therapeutics - Discussion of stem cell biology and its role in transplantation |
| cell and gene therapy insights: Gene Therapy for Viral Infections Patrick Arbuthnot, 2015-06-01 Gene Therapy for Viral Infections provides a comprehensive review of the broader field of nucleic acid and its use in treating viral infections. The text bridges the gap between basic science and important clinical applications of the technology, providing a systematic, integrated review of the advances in nucleic acid-based antiviral drugs and the potential advantages of new technologies over current treatment options. Coverage begins with the fundamentals, exploring varying topics, including harnessing RNAi to silence viral gene expression, antiviral gene editing, viral gene therapy vectors, and non-viral vectors. Subsequent sections include detailed coverage of the developing use of gene therapy for the treatment of specific infections, the principles of rational design of antivirals, and the hurdles that currently face the further advancement of gene therapy technology. - Provides coverage of gene therapy for a variety of infections, including HBV, HCV, HIV, hemorrhagic fever viruses, and respiratory and other viral infections - Bridges the gap between the basic science and the important medical applications of this technology - Features a broad approach to the topic, including an essential overview and the applications of gene therapy, synthetic RNA, and other antiviral strategies that involve nucleic acid engineering - Presents perspectives on the future use of nucleic acids as a novel class of antiviral drugs - Arms the reader with the cutting-edge information needed to stay abreast of this developing field |
| cell and gene therapy insights: Bioprocessing for Cell-Based Therapies Che J. Connon, 2017-02-06 With contributions from leading, international academics and industrial practitioners, Bioprocessing for Cell-Based Therapies explores the very latest techniques and guidelines in bioprocess production to meet safety, regulatory and ethical requirements, for the production of therapeutic cells, including stem cells. An authoritative, cutting-edge handbook on bioprocessing for the production of therapeutic cells with extensive illustrations in full colour throughout An authoritative, cutting-edge handbook on bioprocessing for the production of therapeutic cells with extensive illustrations in full colour throughout In depth discussion of the application of cell therapy including methods used in the delivery of cells to the patient Includes contributions from experts in both academia and industry, combining a practical approach with cutting edge research The only handbook currently available to provide a state of the art guide to Bioprocessing covering the complete range of cell-based therapies, from experts in academia and industry |
| cell and gene therapy insights: Pharmaceutical Biotechnology Daan J. A. Crommelin, Robert D. Sindelar, 2002-11-14 The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well. |
| cell and gene therapy insights: The Challenge of CMC Regulatory Compliance for Biopharmaceuticals John Geigert, 2019-05-08 Biopharmaceuticals (i.e., biological medicines sourced from genetically-engineered living systems) for treatment of human diseases have become a significant percentage of the pharmaceutical industry. And not just the recombinant DNA-derived proteins and monoclonal antibodies (both from the innovators and biosimilars); but now, an increasing awareness of the importance of gene therapy and genetically engineered cellular medicinal products. These biopharmaceuticals are being developed by many companies whose Chemistry, Manufacturing & Control (CMC) teams have varying degrees of familiarity or experience with the CMC strategy and regulatory compliance requirements for these challenging products. Companies clearly plan out the strategy for their clinical study plans, but frequently, the development of a strategy for CMC is an afterthought. Coupled with the complexity of the biopharmaceutical manufacturing processes and products, and this can be a recipe for disaster. The third edition of this book provides insights and practical guidance for the CMC teams to develop an acceptable cost-effective, risk-based CMC regulatory compliance strategy for all biopharmaceuticals (recombinant proteins, monoclonal antibodies, genetically engineered viruses and genetically engineered human cells) from early clinical stage development through market approval. The third edition of this book provides added coverage for the biosimilars, antibody drug conjugates (ADCs), bispecific antibodies, genetically engineered viruses, and genetically engineered cells. This third edition of the book also addresses the heightened pressure on CMC regulatory compliance timelines due to the introduction of expedited clinical pathways moving the clinical development closer to a seamless phase process (e.g., FDA Breakthrough Therapy designation, CBER Regenerative Medicine Advanced Therapy (RMAT) designation, EMA Priority Medicines (PRIME) designation). The Challenge of CMC Regulatory Compliance for Biopharmaceuticals is essential, practical information for all pharmaceutical development scientists, Manufacturing and Quality Unit staff, Regulatory Affairs personnel, and senior management involved in the manufacture of biopharmaceuticals. |
| cell and gene therapy insights: A Handbook of Gene and Cell Therapy Clévio Nóbrega, Liliana Mendonça, Carlos A. Matos, 2020-06-27 This is a reference handbook for young researchers exploring gene and cell therapy. Gene therapy could be defined as a set of strategies modifying gene expression or correcting mutant/defective genes through the administration of DNA (or RNA) to cells, in order to treat disease. Important advances like the discovery of RNA interference, the completion of the Human Genome project or the development of induced pluripotent stem cells (iPSc) and the basics of gene therapy are covered. This is a great book for students, teachers, biomedical researchers delving into gene/cell therapy or researchers borrowing skills from this scientific field. |
| cell and gene therapy insights: A Guide to Human Gene Therapy Roland W. Herzog, Sergei Zolotukhin, 2010 1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne |
| cell and gene therapy insights: Primary and Stem Cells Uma Lakshmipathy, Bhaskar Thyagarajan, 2011-10-31 This book describes basic cell engineering methods, emphasizing stem cell applications, and use of the genetically modified stem cells in cell therapy and drug discovery. Together, the chapters introduce and offer insights on new techniques for engineering of stem cells and the delivery of transgenes into stem cells via various viral and non-viral systems. The book offers a guide to the types of manipulations currently available to create genetically engineered stem cells that suit any investigator's purpose, whether it's basic science investigation, creation of disease models and screens, or cells for therapeutic applications. |
| cell and gene therapy insights: Biodefense in the Age of Synthetic Biology National Academies of Sciences, Engineering, and Medicine, Division on Earth and Life Studies, Board on Life Sciences, Board on Chemical Sciences and Technology, Committee on Strategies for Identifying and Addressing Potential Biodefense Vulnerabilities Posed by Synthetic Biology, 2019-01-05 Scientific advances over the past several decades have accelerated the ability to engineer existing organisms and to potentially create novel ones not found in nature. Synthetic biology, which collectively refers to concepts, approaches, and tools that enable the modification or creation of biological organisms, is being pursued overwhelmingly for beneficial purposes ranging from reducing the burden of disease to improving agricultural yields to remediating pollution. Although the contributions synthetic biology can make in these and other areas hold great promise, it is also possible to imagine malicious uses that could threaten U.S. citizens and military personnel. Making informed decisions about how to address such concerns requires a realistic assessment of the capabilities that could be misused. Biodefense in the Age of Synthetic Biology explores and envisions potential misuses of synthetic biology. This report develops a framework to guide an assessment of the security concerns related to advances in synthetic biology, assesses the levels of concern warranted for such advances, and identifies options that could help mitigate those concerns. |
| cell and gene therapy insights: Mesenchymal Stem Cell Therapy Lucas G. Chase, Mohan C Vemuri, 2012-12-12 Over the past decade, significant efforts have been made to develop stem cell-based therapies for difficult to treat diseases. Multipotent mesenchymal stromal cells, also referred to as mesenchymal stem cells (MSCs), appear to hold great promise in regards to a regenerative cell-based therapy for the treatment of these diseases. Currently, more than 200 clinical trials are underway worldwide exploring the use of MSCs for the treatment of a wide range of disorders including bone, cartilage and tendon damage, myocardial infarction, graft-versus-host disease, Crohn’s disease, diabetes, multiple sclerosis, critical limb ischemia and many others. MSCs were first identified by Friendenstein and colleagues as an adherent stromal cell population within the bone marrow with the ability to form clonogenic colonies in vitro. In regards to the basic biology associated with MSCs, there has been tremendous progress towards understanding this cell population’s phenotype and function from a range of tissue sources. Despite enormous progress and an overall increased understanding of MSCs at the molecular and cellular level, several critical questions remain to be answered in regards to the use of these cells in therapeutic applications. Clinically, both autologous and allogenic approaches for the transplantation of MSCs are being explored. Several of the processing steps needed for the clinical application of MSCs, including isolation from various tissues, scalable in vitro expansion, cell banking, dose preparation, quality control parameters, delivery methods and numerous others are being extensively studied. Despite a significant number of ongoing clinical trials, none of the current therapeutic approaches have, at this point, become a standard of care treatment. Although exceptionally promising, the clinical translation of MSC-based therapies is still a work in progress. The extensive number of ongoing clinical trials is expected to provide a clearer path forward for the realization and implementation of MSCs in regenerative medicine. Towards this end, reviews of current clinical trial results and discussions of relevant topics association with the clinical application of MSCs are compiled in this book from some of the leading researchers in this exciting and rapidly advancing field. Although not absolutely all-inclusive, we hope the chapters within this book can promote and enable a better understanding of the translation of MSCs from bench-to-bedside and inspire researchers to further explore this promising and quickly evolving field. |
| cell and gene therapy insights: Stem Cells and the Future of Regenerative Medicine Institute of Medicine, Board on Neuroscience and Behavioral Health, National Research Council, Division on Earth and Life Studies, Board on Life Sciences, Committee on the Biological and Biomedical Applications of Stem Cell Research, 2002-01-25 Recent scientific breakthroughs, celebrity patient advocates, and conflicting religious beliefs have come together to bring the state of stem cell researchâ€specifically embryonic stem cell researchâ€into the political crosshairs. President Bush's watershed policy statement allows federal funding for embryonic stem cell research but only on a limited number of stem cell lines. Millions of Americans could be affected by the continuing political debate among policymakers and the public. Stem Cells and the Future of Regenerative Medicine provides a deeper exploration of the biological, ethical, and funding questions prompted by the therapeutic potential of undifferentiated human cells. In terms accessible to lay readers, the book summarizes what we know about adult and embryonic stem cells and discusses how to go about the transition from mouse studies to research that has therapeutic implications for people. Perhaps most important, Stem Cells and the Future of Regenerative Medicine also provides an overview of the moral and ethical problems that arise from the use of embryonic stem cells. This timely book compares the impact of public and private research funding and discusses approaches to appropriate research oversight. Based on the insights of leading scientists, ethicists, and other authorities, the book offers authoritative recommendations regarding the use of existing stem cell lines versus new lines in research, the important role of the federal government in this field of research, and other fundamental issues. |
| cell and gene therapy insights: The Forever Fix Ricki Lewis, 2012-03-13 Lewis pens 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 the promise of a cure. |
| cell and gene therapy insights: Essential Current Concepts in Stem Cell Biology Beate Brand-Saberi, 2020-01-03 This textbook describes the biology of different adult stem cell types and outlines the current level of knowledge in the field. It clearly explains the basics of hematopoietic, mesenchymal and cord blood stem cells and also covers induced pluripotent stem cells. Further, it includes a chapter on ethical aspects of human stem cell research, which promotes critical thinking and responsible handling of the material. Based on the international masters program Molecular and Developmental Stem Cell Biology taught at Ruhr-University Bochum and Tongji University Shanghai, the book is a valuable source for postdocs and researchers working with stems cells and also offers essential insights for physicians and dentists wishing to expand their knowledge. This textbook is a valuable complement to Concepts and Applications of Stem Cell Biology, also published in the Learning Materials in Biosciences textbook series. |
| cell and gene therapy insights: Molecular and Cellular Therapeutics David Whitehouse, Ralph Rapley, 2012-02-17 Molecular and Cellular Therapeutics aims to bring together key developments in the areas of molecular diagnostics, therapeutics and drug discovery. The book covers topics including diagnostics, therapeutics, model systems, clinical trials and drug discovery. The developing approaches to molecular and cellular therapies, diagnostics and drug discovery are presented in the context of the pathologies they are devised to treat. |
| cell and gene therapy insights: Evolution of Translational Omics Institute of Medicine, Board on Health Sciences Policy, Board on Health Care Services, Committee on the Review of Omics-Based Tests for Predicting Patient Outcomes in Clinical Trials, 2012-09-13 Technologies collectively called omics enable simultaneous measurement of an enormous number of biomolecules; for example, genomics investigates thousands of DNA sequences, and proteomics examines large numbers of proteins. Scientists are using these technologies to develop innovative tests to detect disease and to predict a patient's likelihood of responding to specific drugs. Following a recent case involving premature use of omics-based tests in cancer clinical trials at Duke University, the NCI requested that the IOM establish a committee to recommend ways to strengthen omics-based test development and evaluation. This report identifies best practices to enhance development, evaluation, and translation of omics-based tests while simultaneously reinforcing steps to ensure that these tests are appropriately assessed for scientific validity before they are used to guide patient treatment in clinical trials. |
| cell and gene therapy insights: Cell Culture Engineering Wei-Shu Hu, 2006-08-16 Since the introduction of recombinant human growth hormone and insulin a quarter century ago, protein therapeutics has greatly broadened the ho- zon of health care. Many patients suffering with life-threatening diseases or chronic dysfunctions, which were medically untreatable not long ago, can attest to the wonder these drugs have achieved. Although the ?rst generation of p- tein therapeutics was produced in recombinant Escherichia coli, most recent products use mammalian cells as production hosts. Not long after the ?rst p- duction of recombinant proteins in E. coli, it was realized that the complex tasks of most post-translational modi?cations on proteins could only be ef?ciently carried out in mammalian cells. In the 1990s, we witnessed a rapid expansion of mammalian-cell-derived protein therapeutics, chie?y antibodies. In fact, it has been nearly a decade since the market value of mammalian-cell-derived protein therapeutics surpassed that of those produced from E. coli. A common characteristic of recent antibody products is the relatively large dose required for effective therapy, demanding larger quantities for the treatment of a given disease. This, coupled with the broadening repertoire of protein drugs, has rapidly expanded the quantity needed for clinical applications. The increasing demand for protein therapeutics has not been met exclusively by construction of new manufacturing plants and increasing total volume capacity. More - portantly the productivity of cell culture processes has been driven upward by an order of magnitude in the past decade. |
| cell and gene therapy insights: Stem Cells for Cancer and Genetic Disease Treatment Phuc Van Pham, Ahmed El-Hashash, 2018-11-15 This invaluable resource discusses insights ranging from basic biological mechanisms of various types of stem cells through the potential applications in the treatment of human diseases, including cancer and genetic disorders. These discoveries are placed within the structural context of tissue and developmental biology in sections dealing with recent advances in understanding different types of stem cell biology and their potential applications in tissue repair and regeneration and in the treatment different types of human cancer and genetic diseases or disorders. Stem Cells for Cancer and Genetic Disease Treatment and the other books in the Stem Cells in Clinical Applicationsseries will be invaluable to scientists, researchers, advanced students and clinicians working in stem cells, regenerative medicine or tissue engineering as well as cancer or genetics research. |
| cell and gene therapy insights: Cell Therapy Adrian P. Gee, 2021-11-10 This new edition presents a fully-updated and expanded look at current Good Manufacturing Practice (cGMP) for cell therapy products. It provides a complete discussion of facility design and operation including details specific to cord blood banking, cell processing, vector production and qualification of a new facility. Several chapters cover facility infrastructure including cleaning and maintenance, vendor qualification, writing a Standard Operating Procedure, staff training, and process validation. The detailed and invaluable product information covers topics like labelling, release and administration, transportation and shipment, et al. Further chapters cover relevant topics like writing and maintaining investigational new drug applications, support opportunities in North America and the European Union, commercial cell processing and quality testing services, and financial considerations for academic GMP facilities. A chapter on future directions rounds out Cell Therapy: cGMP Facilities and Manufacturing making it essential reading for any cell therapy professional involved in the development, use, or management of this type of facility. |
| cell and gene therapy insights: Human Genome Editing National Academies of Sciences, Engineering, and Medicine, National Academy of Medicine, National Academy of Sciences, Committee on Human Gene Editing: Scientific, Medical, and Ethical Considerations, 2017-08-13 Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions. Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing. |
| cell and gene therapy insights: Heritable Human Genome Editing The Royal Society, National Academy of Sciences, National Academy of Medicine, International Commission on the Clinical Use of Human Germline Genome Editing, 2021-01-16 Heritable human genome editing - making changes to the genetic material of eggs, sperm, or any cells that lead to their development, including the cells of early embryos, and establishing a pregnancy - raises not only scientific and medical considerations but also a host of ethical, moral, and societal issues. Human embryos whose genomes have been edited should not be used to create a pregnancy until it is established that precise genomic changes can be made reliably and without introducing undesired changes - criteria that have not yet been met, says Heritable Human Genome Editing. From an international commission of the U.S. National Academy of Medicine, U.S. National Academy of Sciences, and the U.K.'s Royal Society, the report considers potential benefits, harms, and uncertainties associated with genome editing technologies and defines a translational pathway from rigorous preclinical research to initial clinical uses, should a country decide to permit such uses. The report specifies stringent preclinical and clinical requirements for establishing safety and efficacy, and for undertaking long-term monitoring of outcomes. Extensive national and international dialogue is needed before any country decides whether to permit clinical use of this technology, according to the report, which identifies essential elements of national and international scientific governance and oversight. |
| cell and gene therapy insights: Regenerative Medicine Tingting Qiu, Mondher Toumi, 2023-05-23 A comprehensive review of the challenges that exist in patient accessibility to regenerative medicines (RMs), presenting clinical trials, marketing authorization, HTA, pricing, reimbursement, affordability, payment and partnership agreements of RMs and commercialization. Specfically, we investigated how COVID-19 has impacted the RM industry by elaborating on the disruptions it caused but also the new opportunities it brought. The ultimate goal of this work is to make strategic recommendations for manufacturers and decisions-makers on effective strategies to address the above obstacles and facilitate patient access to promising regenerative medicines. FEATURES Regenerative medicine (RM) is an emerging interdisciplinary field aiming to replace or regenerate human cells, tissues, or organs in order to restore normal function. RM holds the promise of revolutionizing treatment in the 21st century. RMs bring new hope for some previously untreatable diseases, as well as holding promise for the treatment of common chronic diseases. Rapid advancements in biotechnology and improved understanding of disease pathophysiology have attracted tremendous interests in the development of RMs. Discusses the high cost of RMs which may challenge the sustainability of healthcare insurers (public and private). |
| cell and gene therapy insights: Immunoinformatics Christian Schönbach, Shoba Ranganathan, Vladimir Brusic, 2007-11-21 In contrast to existing books on immunoinformatics, this volume presents a cross-section of immunoinformatics research. The contributions highlight the interdisciplinary nature of the field and how collaborative efforts among bioinformaticians and bench scientists result in innovative strategies for understanding the immune system. Immunoinformatics is ideal for scientists and students in immunology, bioinformatics, microbiology, and many other disciplines. |
| cell and gene therapy insights: RNA Worlds: New Tools for Deep Exploration Thomas R. Cech, Joan A. Steitz, John F. Atkins, 2018-12-31 A Subject Collection from Cold Spring Harbor Perspectives in Biology. |
| cell and gene therapy insights: Technologies in Cell Culture , 2024-06-26 Technologies in Cell Culture - A Journey From Basics to Advanced Applications is a comprehensive book that offers a broad overview of the subject, encompassing fundamental concepts, modern techniques, and their diverse applications across various fields. Comprising eleven chapters authored by leading international experts in their respective fields, this book adeptly navigates the complexities of cell culture. It provides valuable insights into bioprocessing, cancer biology, regenerative medicine, and more. The book explores innovative strategies for restoring eyesight in individuals with age-related macular degeneration through retinal pigment epithelium monolayers derived from autologous adipose tissue stem cells. We discover novel approaches for utilizing in vitro techniques to evaluate new medications targeting crucial molecular pathways. A deeper comprehension of the tumor microenvironment can be achieved by conducting coculture studies on cancer cells and adipocytes. The readers will gain insight into organoid intelligence, a culmination of advancements in stem cell technologies, bioengineering, and artificial intelligence. For anyone intrigued by unraveling the mysteries of cellular life, Technologies in Cell Culture - A Journey From Basics to Advanced Applications is an indispensable resource, catering to both novice scientists and seasoned researchers seeking to expand their knowledge. Join us on this journey as we explore the myriad ways in which cell culture technology can impact biomedicine and beyond. |
| cell and gene therapy insights: Chimeric Antigen Receptor T-Cell Therapies for Cancer E-Book Daniel W. Lee, Nirali N. Shah, 2019-11-30 From patient referral to post-therapy management, Chimeric Antigen Receptor (CAR) T-Cell Therapies for Cancer: A Practical Guide presents a comprehensive view of CAR modified T-cells in a concise and practical format. Providing authoritative guidance on the implementation and management of CAR T-cell therapy from Drs. Daniel W. Lee and Nirali N. Shah, this clinical resource keeps you up to date on the latest developments in this rapidly evolving area. - Covers all clinical aspects, including patient referral, toxicities management, comorbidities, bridging therapy, post-CAR monitoring, and multidisciplinary approaches to supportive care. - Includes key topics on associated toxicities such as predictive biomarkers, infections, and multidisciplinary approaches to supportive care. - Presents current knowledge on FDA approved CAR T-cell products as well as developments on the horizon. - Editors and authors represent leading investigators in academia and worldwide pioneers of CAR therapy. |
| cell and gene therapy insights: Allocation of Staff , 1983 |
| cell and gene therapy insights: The Road to Discovery Jan Anthony Witkowski, 2016 The Road to Discovery: A Short History of Cold Spring Harbor Laboratory was published in 2015 to mark the 125th anniversary of Cold Spring Harbor Laboratory. At Cold Spring Harbor, in a bucolic setting on the north shore of New York's Long Island, two interdependent research centers in biology were founded as Charles Darwin's insights into heredity and evolution shook the world of science. Fifty years later, those centers would emerge as a single institution that would cradle another revolution, the new science of molecular biology, and advance to world renown in research and professional education. It is a remarkable story, with a path of progress that was neither simple nor assured. The Road to Discovery traces half a century of changes in name, leadership, governance, and financial fortune. And scientific missteps, most notoriously in eugenics, were triumphed by innovative work in genetics, human metabolism, and cancer. From the 1940s through the 1960s, the Laboratory was home to fundamental discoveries about the nature of genetic material and a cauldron of critical assessment of ideas about genes by sharp-tongued summer visitors. James D. Watson, a junior member of that group, would go on to deduce the structure of DNA with Francis Crick in 1953 and help create the new field of molecular genetics before returning to Cold Spring Harbor as Director 15 years later. As the book shows, his Bold Plan would inspire, cajole, and goad into existence an era of expansion, new research directions, and initiatives in conferences, courses, publishing, and education that redefined the scope of the Laboratory. Under Bruce Stillman's leadership, that scope has grown still more, making the Laboratory unique among research institutions worldwide--envied, imitated, but not reproduced. The book's author is the science historian Jan Witkowski. His knowledge of the subject is wide and his affection for it deep. He brings to his task insights that only a decades-long career as a staff member can provide. For over a century, the Laboratory has been influenced by exceptional personalities, outstanding achievements, and dramatic events. The Road to Discovery captures that history in a lively narrative illuminated by vignettes on the importance of individual scientists and their discoveries. Abundantly documented with material from the Laboratory's archives, it is an accessible book that will appeal to anyone interested in the development of biomedical science and biotechnology through the 20th century to the present day. |
| cell and gene therapy insights: From Research to Reality The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada, 2020-11-05 From Research to Reality describes the stages involved in the approval and use of gene therapies in Canada, and examines challenges associated with regulatory oversight, manufacturing, access, and affordability, and identifies promising approaches to address them. |
| cell and gene therapy insights: 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. |
| cell and gene therapy insights: Bioprocess and Analytics Development for Virus-based Advanced Therapeutics and Medicinal Products (ATMPs) Saurabh Gautam, Abhilash I. Chiramel, Roland Pach, 2023-08-10 This book reviews the knowledge, methods and available techniques in the rapidly advancing field of virus based vaccines and gene therapeutics. It also highlights new innovative tools and interdisciplinary techniques for bioprocess development and analytics of viruses and viral vectors. As such, it provides a timely and highly relevant resource, since current advances in pharmaceutical research have seen the rise of vaccines and advanced therapeutics and medicinal products (ATMPs), that rely on the power of viruses. However, developing bioprocesses and analytics required to create this often called “magic bullet” (i.e. gene therapy) remains an extremely challenging and costly task. This book offers strategies for overcoming hurdles and difficulties within in all the necessary steps of viral vector development - from scalability to purification methods and quality control. The book is intended for researchers working in academia or industry, as well as graduate students pursuing a career in virology. |
| cell and gene therapy insights: Leucine-Rich Repeat Kinase 2 (LRRK2) Hardy J. Rideout, 2017-03-28 This is the first book to assemble the leading researchers in the field of LRRK2 biology and neurology and provide a snapshot of the current state of knowledge, encompassing all major aspects of its function and dysfunction. The contributors are experts in cell biology and physiology, neurobiology, and medicinal chemistry, bringing a multidisciplinary perspective on the gene and its role in disease. The book covers the identification of LRRK2 as a major contributor to the pathogenesis of Parkinson's Disease. It also discusses the current state of the field after a decade of research, putative normal physiological roles of LRRK2, and the various pathways that have been identified in the search for the mechanism(s) of its induction of neurodegeneration. |
| cell and gene therapy insights: Mutation-Specific Gene Editing for Blood Disorders Carsten Werner Lederer, Pietro Genovese, Annarita Miccio, Sjaak Philipsen, 2021-11-25 Dr. Miccio holds patents related to viral gene delivery vectors. The Topic Editors acknowledge the use of image material from kindpng.com and from Crystal and Annie Spratt on unsplash.com. |
CELL & GENE THERAPY INSIGHTS
458 Cell & Gene Therapy Insights; DOI: 10.18609/cgti.2024.059 launched in October 2021 to develop plat-forms and standards to speed the develop-ment and delivery of gene therapies for …
Cell & Gene Therapy Insights 2022; 8(3), 341 DOI: …
In a recent Expert Roundtable discussion, we spoke to five experts about strategies for scale up of lentivirus-based cell and gene therapies. Here, we sum up some of their key thoughts.
The state of cell and gene therapy in 2023 - Cell Press
Dec 12, 2023 · Gene, cell, and RNA therapies, collectively called cell and gene ther-apies (CGTs) in this review, represent the cutting edge of drug discov-ery (Figure 1). Owing to their precise …
CE GENE THERAPY INSIGHTS - cdn.cytivalifesciences.com
gene therapies is to work on stable cell lines. The ELEVECTA stable producer cell line is a transfection-free and inducible AAV producer cell line that does not require a helper virus. It is …
Current and future directions for tumor infiltrating …
CELL GENE THERAPY INSIGHTS 856 DOI: 10.18609/cgti.2020.088 Immunotherapy represents a potentially life-saving option in the treatment of pa-tients with cancer. Because of the …
CELL & GENE THERAPY INSIGHTS
Advanced Therapy Medicinal Products (AT-MPs) (which include cell and gene therapy (CGT) products) pose many new challeng-es, and also bring new concepts and oppor-tunities that are …
Business Insight: cell & gene therapy - Arnold & Porter
the broad nature of regulatory challenges for the biopharmaceutical industry as a whole stemming from COVID-19 epidemic, this section focuses on the challenges specific to the cell and gene …
CELL THERAPY INSIGHTS
Cell and gene therapies have the potential to facilitate disease-modifying treatment of both rare and chronic conditions. Whilst approved treatments are now available, several chal-
CELL & GENE THERAPY INSIGHTS
CELL & GENE THERAPY INSIGHTS SPOTLIGHT ON: Vector bioprocessing & materials Guest Editor: Andrew Tustian, Regeneron Pharmaceuticals APRIL 2023 Volume 9, Issue 3
CELL & GENE THERAPY INSIGHTS
Apr 6, 2024 · Adoptive cell therapy (ACT) is a form of immunotherapy in which cancer-specific T cells are modified and expanded ex vivo and re-infused to target and eradicate the tumor. …
CELL THERAPY INSIHTS - cdn.insights.bio
CELL & GENE THERAPY INSIGHTS 428 DOI: 10.18609/cgti.2021.070 INTRODUCTION Preclinical and clinical research and develop-ment for cell therapy and immunotherapy have …
CELL & GENE THERAPY INSIGHTS
Apr 6, 2024 · gene therapy is a more advanced, safer and precise option for monoge-netic life threatening disorders. The efficacy of gene therapy, however, does not only rely on the gene …
DIAKIT 2025 THERAPY CELL & GENE INSIGHTS
Presenting a webinar with Cell & Gene Therapy Insights gives you an efficient and cost-effective way to: generate qualified leads from amongst the global cell and gene therapy community; …
CELL & GENE THERAPY INSIGHTS
CELL & GENE THERAPY INSIGHTS SPOTLIGHT ON: Scale-up and scale-out – what do we really need and how will we get there? Guest Editor: Jan Thirkettle, Transine Therapeutics …
CELL & GENE THERAPY INSIGHTS
CRISPR gene editing for sickle cell disease: one disease, differentiated approaches Cell & Gene Therapy Insights 2021; 7(7), 851–856 DOI: 10.18609/cgti.2021.116 CRISPR-Cas-mediated …
CELL & GENE THERAPY INSIGHTS
own research and development. This grew into three different areas: antibody discovery, gene therapy manufacturing strategies, and CRISPR engineering. We have evolved as the markets …
CELL & GENE THERAPY INSIGHTS
CELL & GENE THERAPY INSIGHTS EXPERT INSIGHT INTERVIEW INNOVATOR INSIGHT High dose systemic gene ® therapy: emerging trends on safety and efficacy Ying Kai Chan, …
CELL & GENE THERAPY INSIGHTS
CELL & GENE THERAPY INSIGHTS 448 DOI: 10.18609cgti.2017.041 manufacturing solutions are off-the-shelf (allogeneic) therapies. These can encompass therapies such as those that …
CELL THERAPY INSIGHTS
the efficacy of a gene therapy, playing roles in receptor binding during cell entry, intracellu-lar trafficking and genome release. Correct expression of the viral vector cap-sid with the right …
CELL & GENE THERAPY INSIGHTS
458 Cell & Gene Therapy Insights; DOI: 10.18609/cgti.2024.059 launched in October 2021 to develop plat-forms and standards to speed the develop-ment and delivery of gene therapies …
Cell & Gene Therapy Insights 2022; 8(3), 341 DOI: …
In a recent Expert Roundtable discussion, we spoke to five experts about strategies for scale up of lentivirus-based cell and gene therapies. Here, we sum up some of their key thoughts.
The state of cell and gene therapy in 2023 - Cell Press
Dec 12, 2023 · Gene, cell, and RNA therapies, collectively called cell and gene ther-apies (CGTs) in this review, represent the cutting edge of drug discov-ery (Figure 1). Owing to their precise …
CE GENE THERAPY INSIGHTS - cdn.cytivalifesciences.com
gene therapies is to work on stable cell lines. The ELEVECTA stable producer cell line is a transfection-free and inducible AAV producer cell line that does not require a helper virus. It is …
CELL THERAPY INSIGHTS - pdfs.semanticscholar.org
Cell & Gene Therapy Insights 2021; 7(1), 1–7 DOI: 10.18609/cgti.2021.010 RAAVS AT CENTER STAGE FOR GENE THERAPY Adeno-associated virus (AAV) are recombi-nant viral vectors …
Current and future directions for tumor infiltrating lymphocyte …
CELL GENE THERAPY INSIGHTS 856 DOI: 10.18609/cgti.2020.088 Immunotherapy represents a potentially life-saving option in the treatment of pa-tients with cancer. Because of the …
CELL & GENE THERAPY INSIGHTS
Advanced Therapy Medicinal Products (AT-MPs) (which include cell and gene therapy (CGT) products) pose many new challeng-es, and also bring new concepts and oppor-tunities that …
Business Insight: cell & gene therapy - Arnold & Porter
the broad nature of regulatory challenges for the biopharmaceutical industry as a whole stemming from COVID-19 epidemic, this section focuses on the challenges specific to the cell and gene …
CELL THERAPY INSIGHTS
Cell and gene therapies have the potential to facilitate disease-modifying treatment of both rare and chronic conditions. Whilst approved treatments are now available, several chal-
CELL & GENE THERAPY INSIGHTS
CELL & GENE THERAPY INSIGHTS SPOTLIGHT ON: Vector bioprocessing & materials Guest Editor: Andrew Tustian, Regeneron Pharmaceuticals APRIL 2023 Volume 9, Issue 3
CELL & GENE THERAPY INSIGHTS
Apr 6, 2024 · Adoptive cell therapy (ACT) is a form of immunotherapy in which cancer-specific T cells are modified and expanded ex vivo and re-infused to target and eradicate the tumor. …
CELL THERAPY INSIHTS - cdn.insights.bio
CELL & GENE THERAPY INSIGHTS 428 DOI: 10.18609/cgti.2021.070 INTRODUCTION Preclinical and clinical research and develop-ment for cell therapy and immunotherapy have …
CELL & GENE THERAPY INSIGHTS
Apr 6, 2024 · gene therapy is a more advanced, safer and precise option for monoge-netic life threatening disorders. The efficacy of gene therapy, however, does not only rely on the gene …
DIAKIT 2025 THERAPY CELL & GENE INSIGHTS
Presenting a webinar with Cell & Gene Therapy Insights gives you an efficient and cost-effective way to: generate qualified leads from amongst the global cell and gene therapy community; …
CELL & GENE THERAPY INSIGHTS
CELL & GENE THERAPY INSIGHTS SPOTLIGHT ON: Scale-up and scale-out – what do we really need and how will we get there? Guest Editor: Jan Thirkettle, Transine Therapeutics …
CELL & GENE THERAPY INSIGHTS
CRISPR gene editing for sickle cell disease: one disease, differentiated approaches Cell & Gene Therapy Insights 2021; 7(7), 851–856 DOI: 10.18609/cgti.2021.116 CRISPR-Cas-mediated …
CELL & GENE THERAPY INSIGHTS
own research and development. This grew into three different areas: antibody discovery, gene therapy manufacturing strategies, and CRISPR engineering. We have evolved as the markets …
CELL & GENE THERAPY INSIGHTS
CELL & GENE THERAPY INSIGHTS EXPERT INSIGHT INTERVIEW INNOVATOR INSIGHT High dose systemic gene ® therapy: emerging trends on safety and efficacy Ying Kai Chan, …
CELL & GENE THERAPY INSIGHTS
CELL & GENE THERAPY INSIGHTS 448 DOI: 10.18609cgti.2017.041 manufacturing solutions are off-the-shelf (allogeneic) therapies. These can encompass therapies such as those that …
CELL THERAPY INSIGHTS
the efficacy of a gene therapy, playing roles in receptor binding during cell entry, intracellu-lar trafficking and genome release. Correct expression of the viral vector cap-sid with the right …
