Advertisement
| cell and gene therapy consulting: 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 consulting: 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 consulting: Gene Therapy, An Issue of Hematology/Oncology Clinics of North America Daniel E. Bauer, Donald B Kohn, 2017-09-27 This issue of Hematology/Oncology Clinics will focus on Gene Therapy. Topics include, but are not limited to Historical Perspective and Current Renaissance, Integrating Vectors, Nonintegrating Vectors, Gene Editing, Conditioning Therapies for Autologous HSCT, Approaches to Immunodeficiency, Approaches to Hemoglobinopathy, Approaches to Hemophilia, Hematopoietic Gene Therapies for Neurologic and Metabolic Disease, Gene Therapy Approaches to HIV and other Infectious Diseases, HSC Approaches to Cancer, and Gene Modified T Cell Therapies for Cancer. |
| cell and gene therapy consulting: 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 consulting: Stem Cells in Regenerative Medicine Alain A. Vertes, Nasib Qureshi, Arnold I. Caplan, Lee E. Babiss, 2015-09-14 This book is a unique guide to emerging stem cell technologies and the opportunities for their commercialisation. It provides in-depth analyses of the science, business, legal, and financing fundamentals of stem cell technologies, offering a holistic assessment of this emerging and dynamic segment of the field of regenerative medicine. • Reviews the very latest advances in the technology and business of stem cells used for therapy, research, and diagnostics • Identifies key challenges to the commercialisation of stem cell technology and avenues to overcome problems in the pipeline • Written by an expert team with extensive experience in the business, basic and applied science of stem cell research This comprehensive volume is essential reading for researchers in cell biology, biotechnology, regenerative medicine, and tissue engineering, including scientists and professionals, looking to enter commercial biotechnology fields. |
| cell and gene therapy consulting: Advances In Pharmaceutical Cell Therapy: Principles Of Cell-based Biopharmaceuticals Christine Guenther, Andrea Josefine Hauser, Ralf Huss, 2015-09-23 This textbook is a comprehensive overview of the development of cell-based biopharmaceuticals. Beginning with the underlying biology of stem cell and cell-based products, it traces the long and complex journey from preclinical concept to initiation of a pivotal clinical trial and the potential business model behind it.The book also takes into consideration the different regulatory landscapes and their continuous evolution in Europe, North America and other parts of the world. The authors describe a path to manufacture a clinical grade therapeutic that passes all necessary quality measures as a robust and marketable product including an outlook on next generation products and innovative strategies.This reference book is a must-have guide for any professional already active in biopharmaceuticals and anyone interested in getting involved in a scientific, medical or business capacity. |
| cell and gene therapy consulting: The Gene Therapy Plan Mitchell L. Gaynor, MD, 2016-04-05 Dr. Mitchell Gaynor, integrative medicine pioneer, details what we can each do individually to keep our bodies healthy. |
| cell and gene therapy consulting: Mergers and Acquisitions Mark Thomas, Janna L. Rose, 2024-04-05 Covid-19 has brought so much uncertainty, but one certainty is that the vaccine race will generate winners and losers in the pharmaceutical and biotechnology industries. This will have a major impact on merger and acquisition activity. While the plethora of merger and acquisition deals are abundantly reported by the news media, there is a clear lack of in-depth analysis on the multiple rationales and various challenges in the life sciences industry. By offering contributions from a variety of experts in the biotechnology and pharmaceutical industries, as well as experts on mergers and acquisitions, this edited collection will draw upon the knowledge of a variety of different actors within the fields of pharma and biotech. This book offers a timely exploration of the complexities of mergers and acquisitions in the pharmaceutical and biotechnology industries while seeking to bridge the gap between theory and practice. It presents a critical analysis of the rationale for acquisitions and studies the challenges of ensuring a successful deal. In the light of the Covid-19 pandemic, it will also explore the impact this may have on the industry, which may further stimulate merger and acquisition activity. It will be of interest to researchers, academics, policymakers, and students in the fields of strategy, management, governance, and the biotechnology and pharmaceutical industries. |
| cell and gene therapy consulting: Phase Appropriate GMP for Biological Processes Trevor Deeks, 2018-04 |
| cell and gene therapy consulting: 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 consulting: 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 consulting: 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 consulting: 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 consulting: Quality Management and Accreditation in Hematopoietic Stem Cell Transplantation and Cellular Therapy Mahmoud Aljurf, John A. Snowden, Patrick Hayden, Kim H. Orchard, Eoin McGrath, 2021-02-19 This open access book provides a concise yet comprehensive overview on how to build a quality management program for hematopoietic stem cell transplantation (HSCT) and cellular therapy. The text reviews all the essential steps and elements necessary for establishing a quality management program and achieving accreditation in HSCT and cellular therapy. Specific areas of focus include document development and implementation, audits and validation, performance measurement, writing a quality management plan, the accreditation process, data management, and maintaining a quality management program. Written by experts in the field, Quality Management and Accreditation in Hematopoietic Stem Cell Transplantation and Cellular Therapy: A Practical Guide is a valuable resource for physicians, healthcare professionals, and laboratory staff involved in the creation and maintenance of a state-of-the-art HSCT and cellular therapy program. |
| cell and gene therapy consulting: Gene Therapy Mauro Giacca, 2010-11-01 I entered the gene therapy field in the mid-1990s, being fascinated by the immense potential of genes as drugs for the treatment of human disease. Since then, I have experienced the ups and downs of this discipline, and tried to contribute with my work and that of my laboratory to the development of innovative approaches to the treatment of cardiovascular disorders. During these years, I have had several opp- tunities to speak on gene therapy at lectures and academic lessons, and have often noticed that the field is very attractive to scientists of all disciplines. However, as yet no comprehensive book on the subject has been published. Indeed, most books in the field are either a collection of gene transfer laboratory protocols or deal with the subject in a rather superficial manner. Hence the idea to write a gene therapy textbook that is broad and comprehensive, but at the same time provides sufficient molecular and clinical detail to be of interest to students, professors, and specialists in the various disciplines that contribute to gene therapy. I have tried to keep the language plain and, whenever possible, non-technical. Since the book is intended to be a textbook in the field of gene therapy in both the basic science and clinical areas, whenever technical descriptions are required, they are provided. |
| cell and gene therapy consulting: 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 consulting: 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 consulting: Regulatory Toxicology Franz-Xaver Reichl, Michael Schwenk, 2014-03-27 This book will be written by experts for professionals, scientists and all those involved in toxicological data generation and decision-making. It is the updated and expanded version of a monograph published in German in 2004. Chemical safety is regulated on various levels including production, storage, transport, handling, disposal or labelling. This book deals comprehensively with the safety-ensuring methods and concepts employed by regulatory agencies, industry and academics. Toxicologists use experimental and scientific approaches for data collection, e.g. about chemical hazards, physicochemical features or toxicokinetics. The respective experimental methods are described in the book. Toxicologists also deal with much insecurity in the exposure and effect scenarios during risk assessment. To overcome these, they have different extrapolation methods and estimation procedures at their disposal. The book describes these methods in an accessible manner. Differing concepts from one regulation area to another are also covered. Reasons and consequences become evident when reading the book. Altogether, the book Regulatory Toxicology will serve as an excellent reference. |
| cell and gene therapy consulting: 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 consulting: Career Opportunities in Biotechnology and Drug Development Toby Freedman, 2008 An essential guide for students in the life sciences, established researchers, and career counselors, this resource features discussions of job security, future trends, and potential career paths. Even those already working in the industry will find helpful information on how to take advantage of opportunities within their own companies and elsewhere. |
| cell and gene therapy consulting: Innovation Breakdown Joseph V. Gulfo, 2017-03-07 How do you convert a potentially life-saving new idea into an actual medical product and then make it available to doctors and patients? Joseph Gulfo thought he knew what to do but he thought wrong. |
| cell and gene therapy consulting: Official Gazette of the United States Patent and Trademark Office , 2003 |
| cell and gene therapy consulting: Development of Gene Therapies Avery McIntosh, Oleksandr Sverdlov, 2024-05-23 Cell and gene therapies have become the third major drug modality in pharmaceutical medicine of the 21st century after low molecular weight and antibody drugs. The gene therapy (GTx) field is rapidly advancing, and yet there are still fundamental scientific questions that remain to be answered. Development of GTx products poses unique challenges and opportunities for drug developers. However, there is lack of a systematic exposition of the GTx product development and the pivotal role of the biostatistician in this process. Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development. Intended to provide an exposition to the GTx new product development through peer-reviewed papers written by subject matter experts in this emerging field, this book will be useful for researchers in gene therapy drug development, biostatisticians, regulators, patient advocates, graduate students, and the finance and business development community . Key Features: A collection of papers covering a wide spectrum of topics in gene therapies (GTx), written by leading subject matter experts. An exposition of the core principles of GTx product development, emerging business models, industry standards, best practices, and regulatory pathways. An exposition of statistical and innovative modeling tools for design and analysis of clinical trials of GTx. Insights into commercial models, access hurdles, and health economics of gene therapies. Case studies of successful GTx approvals from core team members that developed the first two FDA-approved AAV gene therapies: Luxturna and Zolgensma. A discussion of potential benefits and hurdles to be overcome for GTx in coming years from a multi-stakeholder perspective. |
| cell and gene therapy consulting: Assessing Genetic Risks Institute of Medicine, Committee on Assessing Genetic Risks, 1994-01-01 Raising hopes for disease treatment and prevention, but also the specter of discrimination and designer genes, genetic testing is potentially one of the most socially explosive developments of our time. This book presents a current assessment of this rapidly evolving field, offering principles for actions and research and recommendations on key issues in genetic testing and screening. Advantages of early genetic knowledge are balanced with issues associated with such knowledge: availability of treatment, privacy and discrimination, personal decision-making, public health objectives, cost, and more. Among the important issues covered: Quality control in genetic testing. Appropriate roles for public agencies, private health practitioners, and laboratories. Value-neutral education and counseling for persons considering testing. Use of test results in insurance, employment, and other settings. |
| cell and gene therapy consulting: 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 consulting: 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 consulting: Stem Cell and Gene Therapy for Cardiovascular Disease Emerson c. Perin, Leslie W. Miller, Doris Taylor, James T. Willerson, 2015-08-21 Stem Cell and Gene Therapy for Cardiovascular Disease is a state-of-the-art reference that combines, in one place, the breadth and depth of information available on the topic. As stem cell and gene therapies are the most cutting-edge therapies currently available for patients with heart failure, each section of the book provides information on medical trials from contributors and specialists from around the world, including not only what has been completed, but also what is planned for future research and trials. Cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals will find this book an invaluable resource for further study on the topic. - Provides information on stem and gene therapy medical trials from contributors and specialists around the world, including not only what has been completed, but also what is planned for future research and trials - Presents topics that can be applied to allogeneic cells, mesenchymal cells, gene therapy, cardiomyoctyes, iPS cells, MAPC's, and organogenesis - Covers the three areas with the greatest clinical trials to date: chronic limb ischemia, chronic angina, and acute MI - Covers the prevailing opinions on how to harness the body's natural repair mechanisms - Ideal resource for cardiology researchers, basic science clinicians, fellows, residents, students, and industry professionals |
| cell and gene therapy consulting: Biopharmaceutical Manufacturing Ralf Pörtner, 2024-02-11 This volume “Cell Engineerring 11 - Biopharmaceutical Manufacturing: Progress, Trends and Challenges” is a source of the latest innovative research and technical development in biomanufacturing systems. It is organised into 2 parts: 1) Manufacturing of recombinant therapeutic proteins (e.g. therapeutic antibodies, biosimilars/biogenerics) and 2) Manufacturing aspects of cell and gene therapy. Each with selected chapters on the following topics for both up- and downstream, such as: Advanced process strategies, especially continuous manufacturing, Advanced culture techniques, especially single-use systems, Process transfer, scale-up/scale-down models, Processing advances/Manufacturing productivity/efficiency, Model-assisted process understanding and development/Digital Twins, Process controls and analytics, Quality control, Quality by design, Facility design and full-scale commercial systems, manufacturing technology innovation. The book comprises contributions of experts from academia and industry active in the field of cell culture development for the production of recombinant proteins, cell therapy and gene therapy, with consideration of Digital Twin ́s and facility design. The knowledge and expertise of the authors cover disciplines like cell biology, engineering, biotechnology and biomedical sciences. Inevitably, some omissions will occur in the test, but the authors have sought to avoid duplications by extensive cross-referencing to chapters in other volumes of this series and elsewhere. We hope the volume provides a useful compendium of techniques for scientists in industrial and research laboratories active in this field. |
| cell and gene therapy consulting: Lead from the Future Mark W. Johnson, Josh Suskewicz, 2020-04-14 Gold Medal Winner for Best Leadership Book in the 2021 Axiom Business Book Awards Named one of the Top Ten Technology Books Of 2020 — Forbes Named one of the 10 Best New Business Books of 2020 by Inc. magazine Johnson and Suskewicz have raised a battle cry for the kind of leadership we need in these uncertain times. -- Sandi Peterson, Member, Board of Directors, Microsoft We all know a visionary leader when we see one. They're bold and prophetic and at the same time pragmatic. They don't just promote change--they drive it, while inspiring and mobilizing others to do the same. Visionaries like Steve Jobs and Jeff Bezos possess a host of innate qualities that make them extraordinary, but what truly sets them apart is their ability to turn vision into action. In Lead from the Future, Innosight's Mark W. Johnson and Josh Suskewicz introduce a new way of thinking and managing, called future-back, that enables any manager to become a practical visionary. Addressing the many barriers to change that exist in established organizations, they present a systematic approach to overcoming them that includes: The principles and mind-set that allow leadership teams to look beyond typical short-term planning horizons A method for turning emerging challenges into the growth opportunities that can define an organization's future A step-by-step approach for translating a vision into a strategic plan that teams can align around and commit to Ways to ensure that visionary thinking becomes a repeatable organizational capability As practical as it is inspiring, Lead from the Future is the guide you and your team need to develop a vision and translate it into transformative growth. |
| cell and gene therapy consulting: Drug Development for Gene Therapy Yanmei Lu, Boris Gorovits, 2024-02-28 Drug Development for Gene Therapy Industry-centric perspective on translational and bioanalytical challenges and best practices for gene therapies Drug Development for Gene Therapy focuses on the translational and bioanalytical challenges and best practices for gene therapy modalities, presenting a significant body of data, including information related to safety and efficacy, necessary to advance through the development pipeline into clinical use. The text covers bioanalytical methods and platforms including patient screening assays, different PCR tests, enzyme activity assays, ELISpot, NGS, LC/MS, and immunoassays, with FDA and EMA guidelines on gene therapy safety and efficacy, along with companion diagnostics regulations from US and EU perspectives. The chapters offer an in-depth discussion of the basics and best practices for translational biomarkers, bioanalysis, and developing companion diagnostics / lab tests for gene therapies in the pharma and biopharma industries. To aid in reader comprehension, the text includes clinical examples of relevant therapies in related chapters. Some of the core topics covered include study design, immunogenicity, various bioanalytical methods and their applications, and global regulatory issues. Written by two highly qualified authors with significant experience in the field, Drug Development for Gene Therapy includes information on: Bioanalytical methods to detect pre-existing antibodies against adeno-associated viruses (AAV) capsids Detection of cellular immunity and humoral response to viral capsids and transgene proteins, and immunogenicity of gene therapy products Nonclinical and clinical study considerations and methods for biodistribution and shedding Quantification of transgene protein expression and biochemical function, and substrate and distal pharmacodynamic biomarker measurements for gene therapy Detection and quantification of rAAV integration and off-target editing Current regulatory landscape for gene therapy product development and the role of biomarkers and general regulatory considerations for gene therapy companion diagnostics With comprehensive coverage of the subject, Drug Development for Gene Therapy is a must-have resource for researchers and developers in the areas of pharmaceuticals, biopharmaceuticals, and contract research organizations (CROs), along with professors, researchers, and advanced students in chemistry, biological, biomedical engineering, pharmaceuticals, and medical sciences. |
| cell and gene therapy consulting: 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 consulting: Gene and Cell Therapies for Beta-Globinopathies Punam Malik, John Tisdale, 2017-11-09 Hemoglobin defects, specifically sickle cell disease & thalassemia, combined, constitute the most common monogenic disorders in the world. In fact, nearly 2% of the world’s population carries a globin gene mutation. The transfer of the corrective globin gene through the HSC compartment by allogeneic HSC transplantation (HSCT) has already proven curative in both SCD and thalassemia patients, and provides the proof of concept that genetic manipulation of the defective organ might be equally therapeutic. However, procedural toxicities and the requirement of an HLA-matched sibling donor limit this approach to a fraction of affected individuals. The editors review the progress & the state of the field in HSCT for hemoglobinopathies & shed light on the major changes expected in the next decade. Although allogeneic HSCT is a curative option, it is limited by the availability of matched donors, which are often available only to 15-20% of patients. An alternative to allogeneic HS CT is genetic correction of autologous HSCs, to overcome donor availability & immune side effects. This Book reviews the progress made on additive gene therapy approaches & the current state of the field. Finally, targeted genetic correction is emerging as a novel therapeutic strategy in the hemoglobinopathies. Although ideal, the inefficiency of targeted correction was rate limiting for translation of this technology to the clinic. With advancements in zinc finger nucleases and TALE endonuclease mediated targeted correction, correction frequencies in hematopoietic stem cells is now reaching levels that may become clinically relevant. Furthermore, the ability to generate autologous embryonic stem cell like cells from primary somatic cells (skin fibroblasts or hematopoietic cells) of the affected individual has allowed for the potential application of genetic correction strategies.This Book reviews upcoming genetic strategies to reactivate fetal hemoglobin production and research advances. |
| cell and gene therapy consulting: Gene Therapy and Cell Therapy Through the Liver Shuji Terai, Takeshi Suda, 2015-10-16 This book reports the recent progress in gene and cell therapy through the liver and aims to facilitate a comprehensive understanding of the current aspects and future prospects from basic research to clinical therapies. Edited by pioneering researchers, this volume presents extensive information to principal investigators, researchers, postdocs and clinicians for examining the wide varieties of pathological conditions both inside and outside the liver. Providing not only the basic and clinical aspects of therapy, this volume is special in that it focuses on the administrative and regulatory difficulties of actual clinical application and legal regulations in different parts of the globe. By indicating the advantages and limitations of the most promising gene and cell therapies targeting the liver, this book will inspire readers to develop a feasible treatment in the next generation. |
| cell and gene therapy consulting: The Changing Economics of Medical Technology Institute of Medicine, Committee on Technological Innovation in Medicine, 1991-02-01 Americans praise medical technology for saving lives and improving health. Yet, new technology is often cited as a key factor in skyrocketing medical costs. This volume, second in the Medical Innovation at the Crossroads series, examines how economic incentives for innovation are changing and what that means for the future of health care. Up-to-date with a wide variety of examples and case studies, this book explores how payment, patent, and regulatory policiesâ€as well as the involvement of numerous government agenciesâ€affect the introduction and use of new pharmaceuticals, medical devices, and surgical procedures. The volume also includes detailed comparisons of policies and patterns of technological innovation in Western Europe and Japan. This fact-filled and practical book will be of interest to economists, policymakers, health administrators, health care practitioners, and the concerned public. |
| cell and gene therapy consulting: New Bioprocessing Strategies: Development and Manufacturing of Recombinant Antibodies and Proteins Bob Kiss, Uwe Gottschalk, Michael Pohlscheidt, 2018-12-06 This book review series presents current trends in modern biotechnology. The aim is to cover all aspects of this interdisciplinary technology where knowledge, methods and expertise are required from chemistry, biochemistry, microbiology, genetics, chemical engineering and computer science. Volumes are organized topically and provide a comprehensive discussion of developments in the respective field over the past 3-5 years. The series also discusses new discoveries and applications. Special volumes are dedicated to selected topics which focus on new biotechnological products and new processes for their synthesis and purification. In general, special volumes are edited by well-known guest editors. The series editor and publisher will however always be pleased to receive suggestions and supplementary information. Manuscripts are accepted in English. |
| cell and gene therapy consulting: Lung Stem Cells in Development, Health and Disease Nikolic, Marko Z., Hogan, Brigid L.M., 2021-04-01 Most organs in the adult human body are able to maintain themselves and undergo repair after injury; these processes are largely dependent on stem cells. In this Monograph, the Guest Editors bring together leading authors in the field to provide information about the different classes of stem cells present both in the developing and adult lung: where they are found, how they function in homeostasis and pathologic conditions, the mechanisms that regulate their behaviour, and how they may be harnessed for therapeutic purposes. The book focuses on stem cells in the mouse and human lung but also includes the ferret as an increasingly important new model organism. Chapters also discuss how lung tissue, including endogenous stem cells, can be generated in vitro from pluripotent stem cell lines. This state-of-the-art collection comprehensively covers one of the most exciting areas of respiratory science |
| cell and gene therapy consulting: Monthly Catalog of United States Government Publications , |
| cell and gene therapy consulting: Concepts of Biology XII , |
| cell and gene therapy consulting: Translating Gene Therapy to the Clinic Jeffrey Laurence, Michael Franklin, 2014-11-14 Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme. - Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application - Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details specific progress and barriers for current translational use - Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials |
| cell and gene therapy consulting: Second Generation Cell and Gene-Based Therapies Alain Vertes, Nathan J. Dowden, Devyn Smith, Nasib Qureshi, 2020-02-07 Second Generation Cell and Gene-Based Therapies: Biological Advances, Clinical Outcomes, and Strategies for Capitalisation serves as the only volume to the market to bridge basic science, clinical therapy, technology development, and business in the field of cellular therapy/cytotherapy. After more than two decades of painstaking fundamental research, the concept of therapeutic cells (stem cells, genes, etc.), beyond the concept of vaccines, is reaching clinical trial, with mounting confidence in the safety and efficacy of these products. Nonetheless, numerous incremental technical advances remain to be achieved. Thus, this volume highlights the possible R&D paths, which will ultimately facilitate clinical delivery of cutting edge curative products.The next waves of innovation are reviewed in depth for hematopoietic stem cells, mesenchymal stem cells, tissue engineering, CAR-T cells, and cells of the immune system, as well as for enabling technologies such as gene and genome editing. Additionally, deep dives in product fundamentals, history of science, pathobiology of diseases, scientific and technological bases, and financing and technology adoption constraints are taken to unravel what will shape the cytotherapy industry to the horizon 2025 and beyond. The outcome is not simply a scientific book, but a global perspective on the nascent field combining science, business, and strategic fundamentals. - Helps readers learn about the most current trends in cell-based therapy, their overall effectiveness from a clinical prospective, and how the industry is moving therapies forward for capitalization - Perspectives section at the end of each chapter summarizes key learnings, hypotheses, and objectives highlighted and combines scientific and business insights - Edited and authored by scientists representing both basic and clinical research and industry, presenting a complete story of the current state and future promise of cellular therapies |
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 …
