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.
One of the recent advances in 21st century medicine is the emergence of gene therapies, drugs that affect the basic biology of genetic disease. The field has seen some notable setbacks in the past, but in recent years has exploded as decades of basic science have been successfully translated into the most complex biologics ever constructed, leading to regulatory approval of several gene therapy products in oncology, hematology, neurology, and ophthalmology indications. These drugs are at the apex of biological manufacturing complexity, and have the potential to be disease modifying or even curative. Evidence-based and innovative quantitative clinical development and lifecycle management strategies will be required as fixtures in the development for these unique drugs in order to reach patients in need. Development of Gene Therapies: Strategic, Scientific, and Regulatory Considerations is an unparalleled summary of the current scientific, statistical, developmental, and regulatory aspects of gene therapies, which is fast becoming a core area of the biopharmaceutical industry. This edited volume provides a systematic description of core development topics in gene therapies through 19 peer-reviewed chapters written by subject matter experts in the field. This edited volume is an invaluable resource for business leaders and investors hoping to understand the scientific principles and strategy of a company they may potentially invest in; the family members of someone affected by a genetic disease who wish to understand better how these therapies work and what they might expect as a treatment for a loved one; academic professionals, who want to learn and teach incoming medical, public health, or business students; and seasoned drug developers, who wish to learn more about the about the cutting edge of biopharmaceutical drug development. Key Features: Provides a thorough background on the scientific, manufacturing, and translational concepts and competencies for gene therapies. Covers important strategic aspects of the gene therapy industry, thereby helping investors, drug developers, and regulators gain a better appreciation of the potential value of gene therapies. Expounds on many existing and emerging state-of-the art scientific and technological advances, as well as ethical, pharmacovigilance, and regulatory considerations for gene therapy product development. Presents several case studies of successful development of gene therapies, including two of the most remarkable FDA-approved gene therapy products: Zolgensma and Luxturna. Provides perspectives and forward-looking statements on the future of gene therapies in neurological, in utero, and ultra-rare indications.
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. 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
The three sections of this volume present currently available cancer gene therapy techniques. Part I describes the various aspects of gene delivery. In Part II, the contributors discuss strategies and targets for the treatment of cancer. Finally, in Part III, experts discuss the difficulties inherent in bringing gene therapy treatment for cancer to the clinic. This book will prove valuable as the volume of preclinical and clinical data continues to increase.
The idea of human gene therapy was accepted by the medical community and society at large long before believable clinical benefits began to emerge. In this book, some of the fields most distinguished contributors chronicle the evolution of this momentous direction for medicine, illustrating how imaginative concepts shaped the development of technologies and brought the daring new idea to its current position of imminent practical success. This is a book designed to endure as clinical advances accumulate, a clear-eyed work of reference that will anchor the further development of this revolution in therapy. It is an essential addition to libraries of clinical medicine, biotechnology, and public policy, and a resource that no laboratory investigator with an interest in the biology of gene transfer should be without.
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.
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.
Gene therapy has tremendous potential for the treatment of neurological disorders. There has been substantial progress in the development of gene therapy strategies for neurological disorders over the last two decades. Gene Therapy in Neurological Disorders thoroughly reviews currently available gene therapy tools and presents examples of their application in a variety of neurological diseases. The book begins with general reviews of gene therapy strategies with a focus on neurological disorders. The remainder of the chapters present approaches to specific neurological disorders. Each chapter gives an in-depth introduction to the relevant field before diving into the specific tool or application. The book aims to help investigators, students and research staff better understand the principles of gene therapy and its application in the nervous system. Provides background information and experimental details of gene therapy tools applied for neuroscience research and neurological disorders Covers a broad range of gene delivery and regulation tools, therapeutic agents, and target cells, including emerging new technologies such as CRISPR/Cas9 genome editing Discusses applications of gene therapy tools to neurological disorders including neurodegeneration, muscular dystrophy, trauma and chronic pain, and neoplastic diseases
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.
