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| cell and gene therapy manufacturing and commercialization: Cell Therapy Adrian Gee, 2009-09-18 Cell Therapy: cGMP Facilities and Manufacturing is the source for a complete discussion of facility design and operation with practical approaches to a variety of day-to-day activities, such as staff training and competency, cleaning procedures, and environmental monitoring. This in-depth book also includes detailed reviews of quality, the framework of regulations, and professional standards. It meets a previously unmet need for a thorough facility-focused resource, Cell Therapy: cGMP Facilities and Manufacturing will be an important addition to the cell therapy professional’s library. Additional topics in Cell Therapy: cGMP Facilities and Manufacturing...Standard operating procedures - Supply management - Facility equipment - Product manufacturing, review, release and administration - Facility master file. |
| cell and gene therapy manufacturing and commercialization: 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 manufacturing and commercialization: Handbook of Cell and Gene Therapy Hazel Aranha, Humberto Vega-Mercado, 2023-03-17 This handbook provides an in-depth review of information across the developmental spectrum of gene and cell therapy products. From introductory information to state-of-the-art technologies and concepts, the book provides insights into upstream processes such as vector design and construction, purification, formulation and fill/finish, as well as delivery options. Planning steps for compliance with current good manufacturing practice (cGMP) to readiness for chemistry, manufacturing and controls (CMC) are also discussed. This book wraps up with examples of successes and pitfalls addressed by experts who have navigated the multiple challenges that are part of any innovative endeavor. Features Provides the most up-to-date information on the development of gene therapy, from the technology involved to gene correction and genome editing Discusses siRNA, mRNA, and plasmid manufacturing Describes the importance of supplier-sponsor synergies on the path to commercialization Written for a diverse audience with a large number of individuals in the core technologies and supportive practices It is intended as a one-stop resource for the availability of state-of-the-art information related to cell and gene therapy products for researchers, scientists, management and other academic and research institutions. |
| cell and gene therapy manufacturing and commercialization: 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 manufacturing and commercialization: Therapeutic Oligonucleotides Jens Kurreck, 2008 This book provides a compelling overall update on current status of RNA interference |
| cell and gene therapy manufacturing and commercialization: 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 manufacturing and commercialization: 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 manufacturing and commercialization: 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 manufacturing and commercialization: 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 manufacturing and commercialization: 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 manufacturing and commercialization: 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 manufacturing and commercialization: Therapy with Cultured Cells Howard Green, 2019-05-08 In this book the author describes the discoveries in his laboratory that led to therapy with cultured cells. The first cultured cell type used for therapy was the keratinocyte of the epidermis, for the treatment of burns. Subsequent developments led to the use of cultured cells for the treatment of diseases of the eye, of the joints and of other diseases. Cultured cells for therapy are now being prepared by industries in the US, Japan and Korea and are used in the aforesaid countries, as well as in France, Sweden and Greece, for the treatment of disease. |
| cell and gene therapy manufacturing and commercialization: Perinatal Stem Cells Anthony Atala, Kyle J. Cetrulo, Rouzbeh R. Taghizadeh, Curtis L Cetrulo, Sean Murphy, 2018-06-14 Perinatal Stem Cells provides researchers and clinicians with a comprehensive description of the current clinical and pre-clinical applications of stem cells derived from perinatal sources, such as amniotic fluid, placenta and placental membranes, the umbilical cord and Wharton's jelly. It's compiled by leading experts in the field, offering readers detailed insights into sources of perinatal stem cells and their potential for disease treatment. Therapeutic applications of perinatal stem cells include the treatment of in utero and pregnancy related diseases, cardiac disease, liver disease, pulmonary disease, inflammatory diseases, for hematopoietic regeneration, and for neural protection after stroke or traumatic brain injury. In addition, the rapid advance in clinical translation and commercialization of perinatal stem cell therapies is highlighted in a section on Clinical and Industry Perspective which provides insight into the new opportunities and challenges involved in this novel and exciting industry. - Explores current clinical and pre-clinical application of stem cells derived from perinatal sources - Offers detailed insight into sources of perinatal stem cells and their potential for disease treatment - Discusses progress in the manufacturing, banking and clinical translation of perinatal stem cells - Edited by a world-renowned team to present a complete story of the development and promise of perinatal stem cells |
| cell and gene therapy manufacturing and commercialization: The Business of Healthcare Innovation Lawton Robert Burns, 2005-08-25 The Business of Healthcare Innovation is the first wide-ranging analysis of business trends in the manufacturing segment of the health care industry. In this leading edge volume, Professor Burns focuses on the key role of the 'producers' as the main source of innovation in health systems. Written by professors of the Wharton School and industry executives, this book provides a detailed overview of the pharmaceutical, biotechnology, genomics/proteomics, medical device and information technology sectors. It analyses the market structures of these sectors as well as the business models and corporate strategies of firms operating within them. Most importantly, the book describes the growing convergence between these sectors and the need for executives in one sector to increasingly draw upon trends in the others. It will be essential reading for students and researchers in the field of health management, and of great interest to strategy scholars, industry practitioners and management consultants. |
| cell and gene therapy manufacturing and commercialization: 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 manufacturing and commercialization: Monoclonal Antibody Production National Research Council, Institute for Laboratory Animal Research, Committee on Methods of Producing Monoclonal Antibodies, 1999-05-06 The American Anti-Vivisection Society (AAVS) petitioned the National Institutes of Health (NIH) on April 23, 1997, to prohibit the use of animals in the production of mAb. On September 18, 1997, NIH declined to prohibit the use of mice in mAb production, stating that the ascites method of mAb production is scientifically appropriate for some research projects and cannot be replaced. On March 26, 1998, AAVS submitted a second petition, stating that NIH failed to provide valid scientific reasons for not supporting a proposed ban. The office of the NIH director asked the National Research Council to conduct a study of methods of producing mAb. In response to that request, the Research Council appointed the Committee on Methods of Producing Monoclonal Antibodies, to act on behalf of the Institute for Laboratory Animal Research of the Commission on Life Sciences, to conduct the study. The 11 expert members of the committee had extensive experience in biomedical research, laboratory animal medicine, animal welfare, pain research, and patient advocacy (Appendix B). The committee was asked to determine whether there was a scientific necessity for the mouse ascites method; if so, whether the method caused pain or distress; and, if so, what could be done to minimize the pain or distress. The committee was also asked to comment on available in vitro methods; to suggest what acceptable scientific rationale, if any, there was for using the mouse ascites method; and to identify regulatory requirements for the continued use of the mouse ascites method. The committee held an open data-gathering meeting during which its members summarized data bearing on those questions. A 1-day workshop (Appendix A) was attended by 34 participants, 14 of whom made formal presentations. A second meeting was held to finalize the report. The present report was written on the basis of information in the literature and information presented at the meeting and the workshop. |
| cell and gene therapy manufacturing and commercialization: Continuous Manufacturing for the Modernization of Pharmaceutical Production National Academies of Sciences, Engineering, and Medicine, Division on Earth and Life Studies, Board on Chemical Sciences and Technology, 2019-04-05 On July 30-31, 2018, the National Academies of Sciences, Engineering, and Medicine held a workshop titled Continuous Manufacturing for the Modernization of Pharmaceutical Production. This workshop discussed the business and regulatory concerns associated with adopting continuous manufacturing techniques to produce biologics such as enzymes, monoclonal antibodies, and vaccines. The participants also discussed specific challenges for integration across the manufacturing system, including upstream and downstream processes, analytical techniques, and drug product development. The workshop addressed these challenges broadly across the biologics domain but focused particularly on drug categories of greatest FDA and industrial interest such as monoclonal antibodies and vaccines. This publication summarizes the presentations and discussions from the workshop. |
| cell and gene therapy manufacturing and commercialization: Improving and Accelerating Therapeutic Development for Nervous System Disorders Institute of Medicine, Board on Health Sciences Policy, Forum on Neuroscience and Nervous System Disorders, 2014-02-06 Improving and Accelerating Therapeutic Development for Nervous System Disorders is the summary of a workshop convened by the IOM Forum on Neuroscience and Nervous System Disorders to examine opportunities to accelerate early phases of drug development for nervous system drug discovery. Workshop participants discussed challenges in neuroscience research for enabling faster entry of potential treatments into first-in-human trials, explored how new and emerging tools and technologies may improve the efficiency of research, and considered mechanisms to facilitate a more effective and efficient development pipeline. There are several challenges to the current drug development pipeline for nervous system disorders. The fundamental etiology and pathophysiology of many nervous system disorders are unknown and the brain is inaccessible to study, making it difficult to develop accurate models. Patient heterogeneity is high, disease pathology can occur years to decades before becoming clinically apparent, and diagnostic and treatment biomarkers are lacking. In addition, the lack of validated targets, limitations related to the predictive validity of animal models - the extent to which the model predicts clinical efficacy - and regulatory barriers can also impede translation and drug development for nervous system disorders. Improving and Accelerating Therapeutic Development for Nervous System Disorders identifies avenues for moving directly from cellular models to human trials, minimizing the need for animal models to test efficacy, and discusses the potential benefits and risks of such an approach. This report is a timely discussion of opportunities to improve early drug development with a focus toward preclinical trials. |
| cell and gene therapy manufacturing and commercialization: Advances in Application of Stem Cells: From Bench to Clinics Firdos Alam Khan, 2021-09-14 The field of stem cell biology is expanding with a continued surge of new information related to its applications. Over past few years, stem cells have been extensively used in cell therapy, tissue engineering, in vitro drug testing among others. At the moment there is no single book available which comprehensively describes the significance of various application of stem cells derived from embryonic and adult sources from lab to clinics. In this edited volume, we discuss basics and advanced topics of stem cells to help researchers, students and professional find the most important information in a single source of updated information about stem cells and relevant applications. This book is divided in 12 chapters and covers topics such as in vitro cell culture, 3D cell culture, cell therapy, tissue engineering, cell factory, cell functionality, in vitro drug testing, organ development, autologous transplantation, allogeneic transplantation, adult stem cells, multipotent stem cells, induced pluripotent stem cells, a pluripotent and embryonic stem cells. |
| cell and gene therapy manufacturing and commercialization: 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. |
| cell and gene therapy manufacturing and commercialization: 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 manufacturing and commercialization: We, the Almighty Fires Anna Rose Welch, 2019-10-01 These thought-provoking and spiritual poems focus on faith, relationships, and the role of God in life and in the bedroom. Female empowerment is at the heart of this collection, as well as perceptions of humanity as beings full of light. |
| cell and gene therapy manufacturing and commercialization: Putting Biotechnology to Work National Research Council, Division on Earth and Life Studies, Commission on Life Sciences, Committee on Bioprocess Engineering, 1992-02-01 The ability of the United States to sustain a dominant global position in biotechnology lies in maintaining its primacy in basic life-science research and developing a strong resource base for bioprocess engineering and bioproduct manufacturing. This book examines the status of bioprocessing and biotechnology in the United States; current bioprocess technology, products, and opportunities; and challenges of the future and what must be done to meet those challenges. It gives recommendations for action to provide suitable incentives to establish a national program in bioprocess-engineering research, development, education, and technology transfer. |
| cell and gene therapy manufacturing and commercialization: Cell Line Development Mohamed Al-Rubeai, 2009-08-11 Mammalian cell lines command an effective monopoly for the production of therapeutic proteins that require post-translational modifications. This unique advantage outweighs the costs associated with mammalian cell culture, which are far grater in terms of development time and manufacturing when compared to microbial culture. The development of cell lines has undergone several advances over the years, essentially to meet the requirement to cut the time and costs associated with using such a complex hosts as production platforms. This book provides a comprehensive guide to the methodology involved in the development of cell lines and the cell engineering approach that can be employed to enhance productivity, improve cell function, glycosylation and secretion and control apoptosis. It presents an overall picture of the current topics central to expression engineering including such topics as epigenetics and the use of technologies to overcome positional dependent inactivation, the use of promoter and enhancer sequences for expression of various transgenes, site directed engineering of defined chromosomal sites, and examination of the role of eukaryotic nucleus as the controller of expression of genes that are introduced for production of a desired product. It includes a review of selection methods for high producers and an application developed by a major biopharmaceutical industry to expedite the cell line development process. The potential of cell engineering approch to enhance cell lines through the manipulation of single genes that play important roles in key metabolic and regulatory pathways is also explored throughout. |
| cell and gene therapy manufacturing and commercialization: Human Genetics and Genomics Bruce R. Korf, Mira B. Irons, 2012-11-19 This fourth edition of the best-selling textbook, Human Genetics and Genomics, clearly explains the key principles needed by medical and health sciences students, from the basis of molecular genetics, to clinical applications used in the treatment of both rare and common conditions. A newly expanded Part 1, Basic Principles of Human Genetics, focuses on introducing the reader to key concepts such as Mendelian principles, DNA replication and gene expression. Part 2, Genetics and Genomics in Medical Practice, uses case scenarios to help you engage with current genetic practice. Now featuring full-color diagrams, Human Genetics and Genomics has been rigorously updated to reflect today’s genetics teaching, and includes updated discussion of genetic risk assessment, “single gene” disorders and therapeutics. Key learning features include: Clinical snapshots to help relate science to practice 'Hot topics' boxes that focus on the latest developments in testing, assessment and treatment 'Ethical issues' boxes to prompt further thought and discussion on the implications of genetic developments 'Sources of information' boxes to assist with the practicalities of clinical research and information provision Self-assessment review questions in each chapter Accompanied by the Wiley E-Text digital edition (included in the price of the book), Human Genetics and Genomics is also fully supported by a suite of online resources at www.korfgenetics.com, including: Factsheets on 100 genetic disorders, ideal for study and exam preparation Interactive Multiple Choice Questions (MCQs) with feedback on all answers Links to online resources for further study Figures from the book available as PowerPoint slides, ideal for teaching purposes The perfect companion to the genetics component of both problem-based learning and integrated medical courses, Human Genetics and Genomics presents the ideal balance between the bio-molecular basis of genetics and clinical cases, and provides an invaluable overview for anyone wishing to engage with this fast-moving discipline. |
| cell and gene therapy manufacturing and commercialization: Somatic Gene Therapy P.L. Chang, 2018-01-17 As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy. |
| cell and gene therapy manufacturing and commercialization: Innovation and Invention in Medical Devices Institute of Medicine, Board on Health Sciences Policy, Roundtable on Research and Development of Drugs, Biologics, and Medical Devices, 2001-12-01 The objective of the workshop that is the subject of this summary report was to present the challenges and opportunities for medical devices as perceived by the key stakeholders in the field. The agenda, and hence the summaries of the presentations that were made in the workshop and which are presented in this summary report, was organized to first examine the nature of innovation in the field and the social and economic infrastructure that supports such innovation. The next objective was to identify and discuss the greatest unmet clinical needs, with a futuristic view of technologies that might meet those needs. And finally, consideration was given to the barriers to the application of new technologies to meet clinical needs. |
| cell and gene therapy manufacturing and commercialization: Handbook of Cell and Gene Therapy Hazel Aranha, Humberto Vega-Mercado, 2023-03-17 This handbook provides an in-depth review of information across the developmental spectrum of gene and cell therapy products. From introductory information to state-of-the-art technologies and concepts, the book provides insights into upstream processes such as vector design and construction, purification, formulation and fill/finish, as well as delivery options. Planning steps for compliance with current good manufacturing practice (cGMP) to readiness for chemistry, manufacturing and controls (CMC) are also discussed. This book wraps up with examples of successes and pitfalls addressed by experts who have navigated the multiple challenges that are part of any innovative endeavor. Features Provides the most up-to-date information on the development of gene therapy, from the technology involved to gene correction and genome editing Discusses siRNA, mRNA, and plasmid manufacturing Describes the importance of supplier-sponsor synergies on the path to commercialization Written for a diverse audience with a large number of individuals in the core technologies and supportive practices It is intended as a one-stop resource for the availability of state-of-the-art information related to cell and gene therapy products for researchers, scientists, management and other academic and research institutions. |
| cell and gene therapy manufacturing and commercialization: Biotechnology Entrepreneurship Craig Shimasaki, 2014-04-08 As an authoritative guide to biotechnology enterprise and entrepreneurship, Biotechnology Entrepreneurship and Management supports the international community in training the biotechnology leaders of tomorrow. Outlining fundamental concepts vital to graduate students and practitioners entering the biotech industry in management or in any entrepreneurial capacity, Biotechnology Entrepreneurship and Management provides tested strategies and hard-won lessons from a leading board of educators and practitioners. It provides a 'how-to' for individuals training at any level for the biotech industry, from macro to micro. Coverage ranges from the initial challenge of translating a technology idea into a working business case, through securing angel investment, and in managing all aspects of the result: business valuation, business development, partnering, biological manufacturing, FDA approvals and regulatory requirements. An engaging and user-friendly style is complemented by diverse diagrams, graphics and business flow charts with decision trees to support effective management and decision making. - Provides tested strategies and lessons in an engaging and user-friendly style supplemented by tailored pedagogy, training tips and overview sidebars - Case studies are interspersed throughout each chapter to support key concepts and best practices. - Enhanced by use of numerous detailed graphics, tables and flow charts |
| cell and gene therapy manufacturing and commercialization: Stem Cell Therapies Adam C. Berger, Sarah H. Beachy, Board on Health Sciences Policy, Steve Olson, Board on Life Sciences, Division on Earth and Life Sciences, Institute of Medicine, National Academy of Sciences, 2014-06-18 Stem cells offer tremendous promise for advancing health and medicine. Whether being used to replace damaged cells and organs or else by supporting the body's intrinsic repair mechanisms, stem cells hold the potential to treat such debilitating conditions as Parkinson's disease, diabetes, and spinal cord injury. Clinical trials of stem cell treatments are under way in countries around the world, but the evidence base to support the medical use of stem cells remains limited. Despite this paucity of clinical evidence, consumer demand for treatments using stem cells has risen, driven in part by a lack of available treatment options for debilitating diseases as well as direct-to-consumer advertising and public portrayals of stem cell-based treatments. Clinics that offer stem cell therapies for a wide range of diseases and conditions have been established throughout the world, both in newly industrialized countries such as China, India, and Mexico and in developed countries such as the United States and various European nations. Though these therapies are often promoted as being established and effective, they generally have not received stringent regulatory oversight and have not been tested with rigorous trials designed to determine their safety and likely benefits. In the absence of substantiated claims, the potential for harm to patients - as well as to the field of stem cell research in general - may outweigh the potential benefits. To explore these issues, the Institute of Medicine, the National Academy of Sciences, and the International Society for Stem Cell Research held a workshop in November 2013. Stem Cell Therapies summarizes the workshop. Researchers, clinicians, patients, policy makers, and others from North America, Europe, and Asia met to examine the global pattern of treatments and products being offered, the range of patient experiences, and options to maximize the well-being of patients, either by protecting them from treatments that are dangerous or ineffective or by steering them toward treatments that are effective. This report discusses the current environment in which patients are receiving unregulated stem cell offerings, focusing on the treatments being offered and their risks and benefits. The report considers the evidence base for clinical application of stem cell technologies and ways to assure the quality of stem cell offerings. |
| cell and gene therapy manufacturing and commercialization: The Role of NIH in Drug Development Innovation and Its Impact on Patient Access National Academies of Sciences, Engineering, and Medicine, Health and Medicine Division, Board on Health Sciences Policy, Board on Health Care Services, 2020-01-27 To explore the role of the National Institutes of Health (NIH) in innovative drug development and its impact on patient access, the Board on Health Care Services and the Board on Health Sciences Policy of the National Academies jointly hosted a public workshop on July 24â€25, 2019, in Washington, DC. Workshop speakers and participants discussed the ways in which federal investments in biomedical research are translated into innovative therapies and considered approaches to ensure that the public has affordable access to the resulting new drugs. This publication summarizes the presentations and discussions from the workshop. |
| cell and gene therapy manufacturing and commercialization: Regenerative Medicine and Stem Cell Biology Nagwa El-Badri, 2020-11-27 This textbook covers the basic aspects of stem cell research and applications in regenerative medicine. Each chapter includes a didactic component and a practical section. The book offers readers insights into: How to identify the basic concepts of stem cell biology and the molecular regulation of pluripotency and stem cell development. How to produce induced pluripotent stem cells (iPSCs) and the basics of transfection. The biology of adult stem cells, with particular emphasis on mesenchymal stromal cells and hematopoietic stem cells, and the basic mechanisms that regulate them. How cancer stem cells arise and metastasize, and their properties. How to develop the skills needed to isolate, differentiate and characterize adult stem The clinical significance of stem cell research and the potential problems that need to be overcome. Evaluating the use of stem cells for tissue engineering and therapies (the amniotic membrane) The applications of bio-nanotechnology in stem cell research. How epigenetic mechanisms, including various DNA modifications and histone dynamics, are involved in regulating the potentiality and differentiation of stem cells. The scientific methods, ethical considerations and implications of stem cell research. |
| cell and gene therapy manufacturing and commercialization: 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 manufacturing and commercialization: Animal Cell Technology Manuel J.T. Carrondo, Bryan Griffiths, José L.P. Moreira, 2012-12-06 Animal cell technology has undergone a rapid transformation over the last decade from a research tool and highly specialised technology to a central resource for innovation in pharmaceutical research and development. These proceedings of the 14th Meeting of the European Society for Animal Cell Technology (Vilamoura, Portugal, May 1996) bring up to date the historical perspective of animal cell technology for the benefit of society, `From Vaccines to Genetic Medicine', and will charter this vital technology for the years to come. Strong contributions are grouped in the traditional ESACT areas of 'Cell and Physiology Engineering' dealing with cell state, including genetics, and its environment, and 'Animal Cell Process Engineering' covering integration of bioreaction with bioseparation coupled with on-line monitoring to improve protein production and consistency. Extensive coverage of metabolic engineering on synthesis, folding, assembly, transiting and secretion is dealt with in the session on 'Recombinant Proteins: Biosynthesis and Bioprocessing'. Two traditional but expanding areas of animal cell technology relevance are highlighted in the broad sessions of 'Animal Cells as Tools for Discovery and Testing' and 'Animal Cell Vaccines: Present and Future'. Two sessions finally cover the more recent domains of animal cell technology work - 'Tissue Engineering and Biomedical Devices' and 'Cells and Vectors for Genetic Medicine' - where one can foresee a very bright future. |
| cell and gene therapy manufacturing and commercialization: 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. |
| cell and gene therapy manufacturing and commercialization: Modern Methods of Clinical Investigation Institute of Medicine, Committee on Technological Innovation in Medicine, 1990-02-01 The very rapid pace of advances in biomedical research promises us a wide range of new drugs, medical devices, and clinical procedures. The extent to which these discoveries will benefit the public, however, depends in large part on the methods we choose for developing and testing them. Modern Methods of Clinical Investigation focuses on strategies for clinical evaluation and their role in uncovering the actual benefits and risks of medical innovation. Essays explore differences in our current systems for evaluating drugs, medical devices, and clinical procedures; health insurance databases as a tool for assessing treatment outcomes; the role of the medical profession, the Food and Drug Administration, and industry in stimulating the use of evaluative methods; and more. This book will be of special interest to policymakers, regulators, executives in the medical industry, clinical researchers, and physicians. |
| cell and gene therapy manufacturing and commercialization: Global Trends 2040 National Intelligence Council, 2021-03 The ongoing COVID-19 pandemic marks the most significant, singular global disruption since World War II, with health, economic, political, and security implications that will ripple for years to come. -Global Trends 2040 (2021) Global Trends 2040-A More Contested World (2021), released by the US National Intelligence Council, is the latest report in its series of reports starting in 1997 about megatrends and the world's future. This report, strongly influenced by the COVID-19 pandemic, paints a bleak picture of the future and describes a contested, fragmented and turbulent world. It specifically discusses the four main trends that will shape tomorrow's world: - Demographics-by 2040, 1.4 billion people will be added mostly in Africa and South Asia. - Economics-increased government debt and concentrated economic power will escalate problems for the poor and middleclass. - Climate-a hotter world will increase water, food, and health insecurity. - Technology-the emergence of new technologies could both solve and cause problems for human life. Students of trends, policymakers, entrepreneurs, academics, journalists and anyone eager for a glimpse into the next decades, will find this report, with colored graphs, essential reading. |
| cell and gene therapy manufacturing and commercialization: Operations Research and Health Care Margaret L. Brandeau, Francois Sainfort, William P. Pierskalla, 2006-04-04 In both rich and poor nations, public resources for health care are inadequate to meet demand. Policy makers and health care providers must determine how to provide the most effective health care to citizens using the limited resources that are available. This chapter describes current and future challenges in the delivery of health care, and outlines the role that operations research (OR) models can play in helping to solve those problems. The chapter concludes with an overview of this book – its intended audience, the areas covered, and a description of the subsequent chapters. KEY WORDS Health care delivery, Health care planning HEALTH CARE DELIVERY: PROBLEMS AND CHALLENGES 3 1.1 WORLDWIDE HEALTH: THE PAST 50 YEARS Human health has improved significantly in the last 50 years. In 1950, global life expectancy was 46 years [1]. That figure rose to 61 years by 1980 and to 67 years by 1998 [2]. Much of these gains occurred in low- and middle-income countries, and were due in large part to improved nutrition and sanitation, medical innovations, and improvements in public health infrastructure. |
| cell and gene therapy manufacturing and commercialization: Oncoimmunology Laurence Zitvogel, Guido Kroemer, 2017-12-13 In this book, leading experts in cancer immunotherapy join forces to provide a comprehensive guide that sets out the main principles of oncoimmunology and examines the latest advances and their implications for clinical practice, focusing in particular on drugs with FDA/EMA approvals and breakthrough status. The aim is to deliver a landmark educational tool that will serve as the definitive reference for MD and PhD students while also meeting the needs of established researchers and healthcare professionals. Immunotherapy-based approaches are now inducing long-lasting clinical responses across multiple histological types of neoplasia, in previously difficult-to-treat metastatic cancers. The future challenges for oncologists are to understand and exploit the cellular and molecular components of complex immune networks, to optimize combinatorial regimens, to avoid immune-related side effects, and to plan immunomonitoring studies for biomarker discovery. The editors hope that this book will guide future and established health professionals toward the effective application of cancer immunology and immunotherapy and contribute significantly to further progress in the field. |
| cell and gene therapy manufacturing and commercialization: Soft Machines Richard Anthony Lewis Jones, 2004 Enthusiasts look forward to a time when tiny machines reassemble matter and process information but is their vision realistic? 'Soft Machines' explains why the nanoworld is so different to the macro-world that we are all familar with and shows how it has more in common with biology than conventional engineering. |
Cell: Cell - Cell Press
Cell publishes findings of unusual significance in any area of experimental biology, including but not limited to cell biology, molecular biology, neuroscience, immunology, virology and …
Cell (biology) - Wikipedia
The cell is the basic structural and functional unit of all forms of life. Every cell consists of cytoplasm enclosed within a membrane; many cells contain organelles, each with a specific …
Cell | Definition, Types, Functions, Diagram, Division, Theory,
Apr 25, 2025 · cell, in biology, the basic membrane-bound unit that contains the fundamental molecules of life and of which all living things are composed. A single cell is often a complete …
The cell: Types, functions, and organelles - Medical News Today
Dec 19, 2023 · A cell is the smallest living organism and the basic unit of life on earth. Together, trillions of cells make up the human body. Cells have three parts: the membrane, the nucleus, …
Cell – Definition, Structure, Types, Functions, Examples
Apr 7, 2024 · A cell is the basic structural and functional unit of all living organisms, responsible for various life processes and containing essential biological
What Is a Cell? | Learn Science at Scitable - Nature
All cells evolved from a common ancestor and use the same kinds of carbon-based molecules. Learn how cell function depends on a diverse group of nucleic acids, proteins, lipids, and sugars.
What is a cell? - MedlinePlus
Feb 22, 2021 · Cells are the basic building blocks of all living things. The human body is composed of trillions of cells. They provide structure for the body, take in nutrients from food, …
Cell Definition - BYJU'S
Jan 14, 2018 · Cells are the structural, functional, and biological units of all living beings. A cell can replicate itself independently. Hence, they are known as the building blocks of life. Each …
Introduction to cells - Basic Biology
Aug 30, 2020 · A cell is the simplest unit of life and they are responsible for keeping an organism alive and functioning. This introduction to cells is the starting point for the area of biology that …
Overview of Cells - Visible Body
What are cells and what do they do? The nucleus of a eukaryotic cell contains its DNA. Cells are the microscopic units that make up humans and every other living organism. Some organisms …
Cell: Cell - Cell Press
Cell publishes findings of unusual significance in any area of experimental biology, including but not limited to cell biology, molecular biology, neuroscience, immunology, virology and …
Cell (biology) - Wikipedia
The cell is the basic structural and functional unit of all forms of life. Every cell consists of cytoplasm enclosed within a membrane; many cells contain organelles, each with a specific …
Cell | Definition, Types, Functions, Diagram, Division, Theory,
Apr 25, 2025 · cell, in biology, the basic membrane-bound unit that contains the fundamental molecules of life and of which all living things are composed. A single cell is often a complete …
The cell: Types, functions, and organelles - Medical News Today
Dec 19, 2023 · A cell is the smallest living organism and the basic unit of life on earth. Together, trillions of cells make up the human body. Cells have three parts: the membrane, the nucleus, …
Cell – Definition, Structure, Types, Functions, Examples
Apr 7, 2024 · A cell is the basic structural and functional unit of all living organisms, responsible for various life processes and containing essential biological
What Is a Cell? | Learn Science at Scitable - Nature
All cells evolved from a common ancestor and use the same kinds of carbon-based molecules. Learn how cell function depends on a diverse group of nucleic acids, proteins, lipids, and sugars.
What is a cell? - MedlinePlus
Feb 22, 2021 · Cells are the basic building blocks of all living things. The human body is composed of trillions of cells. They provide structure for the body, take in nutrients from food, …
Cell Definition - BYJU'S
Jan 14, 2018 · Cells are the structural, functional, and biological units of all living beings. A cell can replicate itself independently. Hence, they are known as the building blocks of life. Each …
Introduction to cells - Basic Biology
Aug 30, 2020 · A cell is the simplest unit of life and they are responsible for keeping an organism alive and functioning. This introduction to cells is the starting point for the area of biology that …
Overview of Cells - Visible Body
What are cells and what do they do? The nucleus of a eukaryotic cell contains its DNA. Cells are the microscopic units that make up humans and every other living organism. Some organisms …
