Second Generation Cell and Gene-based Therapies

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 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.

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

Since the publication of the second edition of this book in 2004, gene therapy and cell therapy clinical trials have yielded some remarkable successes and some disappointing failures. Now in its third edition, Gene and Cell Therapy: Therapeutic Mechanisms and Strategies assembles many of the new technical advances in gene delivery, clinical applications, and new approaches to the regulation and modification of gene expression. New Topics Covered in this Edition: Gene and Cell Therapies for Diabetes and Cardiovascular Diseases Clinical Trials Human Embryonic Stem Cells Tissue Engineering Combined with Cell Therapies Novel Polymers Relevant Nanotechnologies SiRNA Therapeutic Strategies Dendrimer Technologies Comprised of contributions from international experts, this book begins with a discussion of delivery systems and therapeutic strategies, exploring retroviral vectors and adenovirus vectors, as well as other therapeutic strategies. The middle section focuses on gene expression and detection, followed by an examination of various therapeutic strategies for individual diseases, including hematopoietic disorders, cardiovascular conditions, cancer, diabetes, cystic fibrosis, neurological disorders, and childhood-onset blindness. The final section discusses recent clinical trials and regulatory issues surrounding the new technology. This compendium is assembled by noted molecular biologist and biochemist Nancy Smyth Templeton. Baylor College of Medicine and several other institutions have used Dr. Templeton’s non-viral therapeutics in clinical trials for the treatment of lung, breast, head and neck, and pancreatic cancers, as well as Hepatitis B and C. She continues to work at the forefront of research in gene and cell therapies. Her contributions, as well as those contained in this volume, are sure to advance the state of the art of these revolutionary life-saving technologies.

The Second Edition of Gene Therapy of Cancer provides crucial updates on the basic science and ongoing research in this field, examining the state of the art technology in gene therapy and its therapeutic applications to the treatment of cancer. The clinical chapters are improved to include new areas of research and more successful trials. Chapters emphasize the scientific basis of gene therapy using immune, oncogene, antisense, pro-drug activating, and drug resistance gene targets, while other chapters discuss therapeutic approaches and clinical applications. This book is a valuable reference for anyone needing to stay abreast of the latest advances in gene therapy treatment for cancer. Key Features * Provides in-depth description of targeted systems and treatment strategies * Explains the underlying cancer biology necessary for understanding a given therapeutic approach * Extensively covers immune therapeutics of vaccines, cytokines, and peptide-induced responses * Presents translational focus with emphasis on requirements for clinical implementation * Incorporates detailed illustrations of vectors and therapeutic approaches ideal for classroom presentations and general reference

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.

Regenerative medicine – stem cell and gene-based therapy – offers a new approach for restoring function of damaged organs and tissues. This is the first book to cover the major new aspects and field of regenerative medicine. This title is therefore a timely addition to the literature. It brings together the major approaches to regenerative medicine in one text, which ensures that techniques learnt in one discipline are disseminated across other areas of medicine.

Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement Demonstrates noninvasive imaging of adenovirus-mediated gene transfer Discusses utility of adenoviral vectors in animal disease models Considers Federal Drug Administration regulations for human clinical trials

Ideal for cardiologists who need to keep abreast of rapidly changing scientific foundations, clinical research results, and evidence-based medicine, Braunwald’s Heart Disease is your indispensable source for definitive, state-of-the-art answers on every aspect of contemporary cardiology, helping you apply the most recent knowledge in personalized medicine, imaging techniques, pharmacology, interventional cardiology, electrophysiology, and much more! Practice with confidence and overcome your toughest challenges with advice from the top minds in cardiology today, who synthesize the entire state of current knowledge and summarize all of the most recent ACC/AHA practice guidelines. Locate the answers you need fast thanks to a user-friendly, full-color design with more than 1,200 color illustrations. Learn from leading international experts, including 53 new authors. Explore brand-new chapters, such as Principles of Cardiovascular Genetics and Biomarkers, Proteomics, Metabolomics, and Personalized Medicine. Access new and updated guidelines covering Diseases of the Aorta, Peripheral Artery Diseases, Diabetes and the Cardiovascular System, Heart Failure, and Valvular Heart Disease. Stay abreast of the latest diagnostic and imaging techniques and modalities, such as three-dimensional echocardiography, speckle tracking, tissue Doppler, computed tomography, and cardiac magnetic resonance imaging. Consult this title on your favorite e-reader, conduct rapid searches, and adjust font sizes for optimal readability.

Ever since the birth of molecular biology, the tantalizing possibility of treating disease at its genetic roots has become increasingly feasible. Gene therapy - though still in its infancy - remains one of the hottest areas of research in medicine. Its approach utilizes a gene transfer vehicle ( vector) to deliver therapeutic DNA or RNA to cells of the body in order to rectify the defect that is causing the disease. Successful therapies have been reported in humans in recent years such as cures in boys with severe immune deficiencies. Moreover, gene therapy strategies are being adapted in numerous biomedical laboratories to obtain novel treatments for a variety of diseases and to study basic biological aspects of disease. Correction of disease in animal studies, is steadily gaining ground, highlighting the immense potential of gene therapy in the medical profession. This book will cover topics that are at the forefront of biomedical research such as RNA interference, viral and non-viral gene transfer systems, treatment of hematological diseases and disorders of the central nervous system.Leading experts on the respective vector or disease will contribute the individual chapters and explain cutting-edge technologies. It also gives a broad overview of the most important gene transfer vectors and most extensively studied target diseases. This comprehensive guide is therefore a must-read for anyone in the biotechnology, biomedical or medical industries seeking to further their knowledge in the area of human gene therapy.

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.

Stem cells have generated a lot of excitement among the researchers, clinicians and the public alike. Various types of stem cells are being evaluated for their regenerative potential. Marginal benefit resulting by transplanting autologus stem cells (deemed to be absolutely safe) in various clinical conditions has been proposed to be a growth factor effect rather than true regeneration. In contrast, various pre-clinical studies have been undertaken, using differentiated cells from embryonic stem cells or induced pluripotent stem cells have shown promise, functional improvement and no signs of teratoma formation. The scientists are not in a rush to reach the clinic but a handful of clinical studies have shown promise. This book is a collection of studies/reviews, beginning with an introduction to the pluripotent stem cells and covering various aspects like derivation, differentiation, ethics, etc., and hence would provide insight into the recent standing on the pluripotent stem cells biology. The chapters have been categorized into three sections, covering subjects ranging from the generation of pluripotent stem cells and various means of their derivation from embryonic as well as adult tissues, the mechanistic understanding of pluripotency and narrating the potential therapeutic implications of these in vitro generated cells in various diseases, in addition to the associated pros and cons in the same.

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.

Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.

We all know that the field of neuro-oncology is heterogeneous and under continuous development with the addition of new knowledge and information on a regular basis. The present book "Brain Tumor - An Update" is an attempt to share the personal experiences of experts who are involved in neuro-oncology-related research. Through this book, the authors share their experiences and provide details about the pathophysiology, neuroimaging approaches, and management options, and how to go about decision-making in patients with brain tumors. We hope that the valuable contributions from the authors shall facilitate understanding about brain tumors. I am grateful to all the authors who have contributed their tremendous expertise, and I would like to acknowledge the outstanding support of Ms. Danijela Sakic, Author Service Manager, IntechOpen Science, who collaborated tirelessly in crafting this book.

A scientist assesses the potential of stem cell therapies for treating such brain disorders as stroke, Alzheimer's disease, and Parkinson's disease. Stem cell therapies are the subject of enormous hype, endowed by the media with almost magical qualities and imagined by the public to bring about miracle cures. Stem cells have the potential to generate new cells of different types, and have been shown to do so in certain cases. Could stem cell transplants repair the damaged brain? In this book, neurobiologist Jack Price assesses the potential of stem cell therapies to treat such brain disorders as stroke, Alzheimer's disease, Parkinson's disease, and spinal cord injuries. Certainly brain disorders are in need of effective treatments. These disorders don't just kill, they disable, and conventional drug therapies have not had much success in treating them. Price explains that repairing the human brain is difficult, largely because of its structural, functional, and developmental complexity. He examines the self-repairing capacity of blood and gut cells—and the lack of such capacity in the brain; describes the limitations of early brain stem cell therapies for neurodegenerative disorders; and discusses current clinical trials that may lead to the first licensed stem cell therapies for stroke, Parkinson's and macular degeneration. And he describes the real promise of pluripotential stem cells, which can make all the cell types that constitute the body. New technologies, Price reports, challenge the very notion of cell transplantation, instead seeking to convince the brain itself to manufacture the new cells it needs. Could this be the true future of brain repair?

Gene therapy is becoming a promising technology for the management of many human diseases. Hereditary and acquired disorders can both be tackled using the technique of gene therapy. This book provides detailed, up-to-date topics addressing basic principles of gene therapy and discussing some of the challenges encountered by scientists in developing this relatively novel technology. The development of new and efficient gene transfer vectors is of utmost importance in the progress of the field of gene therapy. Both viral and non-viral vectors are extensively discussed. A detailed chapter elaborates the problem of host immune rejection of transplanted donor cells or engineered tissue that can be avoided using the encapsulation of transgenic cells, thus avoiding the use of drugs that achieve immunosuppression.

This book presents state-of-the-art pre-clinical models and clinical applications of stem-cell-based therapies applied to different lung diseases, with a special focus on the translation of bench data into clinical studies. Starting with the assumption that abnormal lung tissue repair and regeneration has emerged as the driving force underlying pathogenesis and progression in many lung diseases, it sheds new light on the potential of stem/stromal cells as drivers of repair and sources of reparative factors in the lung. The first part of the book offers an overview of stem cell types and mechanisms involved in lung development, homeostasis, repair and regeneration, and reveals the crucial role of the extracellular matrix within the lung microenvironment. In the second part, leading experts present the latest pre-clinical evidence and clinical applications of stem-cell-based therapies in a wide variety of lung diseases, ranging from COPD and lung fibrosis to other rare lung diseases. The last section discusses stem cell delivery systems and devices, such as aerosolised spray application. This book appeals to pneumologists, stem cell and matrix biologists, as well as bioengeneers with a special interest in regenerative medicine applied to pulmonary diseases.

Parkinson's disease (PD) is the second most common neurodegenerative disorder results due to loss of dopamine producing brain cells. Knowledge relating to PD condition has been known since 5000BC, however no effective therapeutic strategies are available till today. Therefore it is important for neurobiologists to work further by taking advantage of modern scientific methods and develop appropriate therapeutic strategies. Efforts in this direction are worthy as they will reduce the burden of PD among elderly, who are already burdened with age related systemic degenerative processes. This book is a humble effort in that progressive direction. It has chapters covering multiple aspects relating to etiology, pathophysiology of PD, available and futuristic therapeutics strategies. Therefore it will be of interest to common man, biomedical researchers and clinicians. This is one small step in a direction "to reduce the burden of neurological disease."

Site-specific endonucleases create double-strand breaks within the genome and can be targeted to literally any genetic mutation. Together with a repair template, a correction of the defective locus becomes possible. This book offers insight into the modern tools of genome editing, their hurdles and their huge potential. A new era of in vivo genetic engineering has begun.