国庆充电必备 | 20本细胞基因治疗药物书籍：研发、生产、注册、临床及市场

中国在 CGT 领域的崛起成为全球亮点。截至 2025 年 8 月，我国已有 9 款 CGT 产品获批上市，形成 7 款 CAR-T 疗法、1 款干细胞疗法、1 款基因疗法的产品矩阵，其中传奇生物的西达基奥仑赛 2024 年销售额达 9.6 亿美元，同比增长 93%。研发管线方面，截至 2025 年第一季度，全球 4418 项 CGT 在研项目中，我国贡献了重要力量，仅 CDE 就累计受理 765 项 IND 申请，553 项获批进入临床试验。政策层面，“提前介入、一企一策” 的审评机制与雄安、湖南等地的产业化平台建设相互支撑，推动 CAR-T 疗法制备成本逐步下降，部分干细胞疗法费用已降至 8万元/疗程，加速了创新疗法的临床可及性。

从全球范围看，CGT 市场正持续扩张，2024 年全球畅销 TOP10 CGT 疗法总销售额超 70 亿美元，同比增长 32%，预计 2031 年全球市场规模将突破 520 亿美元。随着监管体系的完善与支付模式的创新，这种 “终极治疗手段” 正从 “天价疗法” 逐步走向普惠，为人类健康事业开辟全新赛道。

值国庆之际，小编精选了细胞基因治疗在研发、生产、注册、临床及市场的20本书籍和大家一起学习交流，有兴趣的老师可以扫码登记，符合要求方可加入我们学习行列。快快加入我们喔，一起过一个充实而又愉快的国庆！

INSPIRATION

Handbook of Cell and Gene Therapy

This book is intended as a one-stop resource for availability of state-of-the-art information related to cell and gene therapy products for researchers, scientists, management and other academic and research institutions working in the field.‍

Cancer Immunotherapies: Solid Tumors and Hematologic Malignancies

This book presents the clinical scope of cancer immunotherapeutic agents for solid tumors and hematological malignancies, elaborates on the scientific details of their modes of action, and presents the impact of these agents on oncology, patients and the broader healthcare system. At present, cancer immunotherapies fall broadly into three categories: immune checkpoint inhibitors (ICIs), adoptive T cell therapies, and cancer vaccines which have distinct mechanisms of action. Immune checkpoint inhibitors rely upon disrupting tumor antigen recognition as self by the immune system through inhibition of checkpoint molecules. Adoptive T cell therapies involve the engineering of T cells ex vivo to target and destroy tumor cells. The first part of this book will provide an overview of the discovery and mechanistic details of the technology. The second part will be devoted to elaborating on the clinical outcomes, successes and limitations for specific tumor subtypes, which includes both solid tumors and hematological malignances for both pediatric and adult populations. As such, the book offers a valuable resource for oncologists, hematologists, and all those seeking an up-to-date overview of cancer immunotherapies.

In Vitro Models for Stem Cell Therapy

This volume looks at in vitro disease models representing the respiratory, hepatobiliary, osteochondral, nervous, dermal, ocular, immune system, and pathological biological processes like tumorigenesis for stem cell research. The chapters in this book cover a range of diseases and application of various stem cells such as adult stem cells and iPS. Chapters also discuss new methods to characterize and manipulate stem cells with the aim to better understand and improve their biological performance. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Cutting-edge and thorough, In Vitro Models for Stem Cell Therapy: Methods and Protocols is a valuable resource for researchers and scientists interested in learning more about this exciting field

Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective

This book discusses the different regulatory pathways for Advanced Therapy Medicinal Products implemented by national agencies in North and South America, Europe and Asia and by international bodies in the effort of international harmonization.

This book represents an update of the first edition, as it covers regulatory novelties and accumulated experience in the regions already addressed. In addition, this new edition offers a wider international perspective: new chapters are included covering Advanced Therapy Medicinal Products regulations in India, Malaysia, Spain and Thailand, the European Pharmacopoeia texts for gene therapy medicinal products as well as international harmonization programs.

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 and therapeutic use of these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of Advanced Therapy Medicinal Products in the areas of product quality, pharmacology and toxicology, clinical trial design and HTA pathways, 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 & 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.‍

Biotechnologies for Gene Therapy: RNA, CRISPR, Nanobots, and Preclinical Applications

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The purpose of this book is to highlight some of latest developments and applications of CRISPR, RNA, and DNA to treat diseases ranging from cancers to cardiovascular and degenerative disorders. It also features innovations of the delivery methods for nucleic acids ranging from nanodevices made from DNA and pseudo amino acids to viral vectors. This is an ideal book for academics, clinicians, and students interested in gene therapy.

Cell and Gene Therapies

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 Therapy: cGMP Facilities and Manufacturing

This new edition presents a fully-updated and expanded look at current Good Manufacturing Practice (cGMP) for cell therapy products. It provides a complete discussion of facility design and operation including details specific to cord blood banking, cell processing, vector production and qualification of a new facility. Several chapters cover facility infrastructure including cleaning and maintenance, vendor qualification, writing a Standard Operating Procedure, staff training, and process validation. The detailed and invaluable product information covers topics like labelling, release and administration, transportation and shipment, et al. Further chapters cover relevant topics like writing and maintaining investigational new drug applications, support opportunities in North America and the European Union, commercial cell processing and quality testing services, and financial considerations for academic GMP facilities. A chapter on future directions rounds out Cell Therapy: cGMP Facilities and Manufacturing making it essential reading for any cell therapy professional involved in the development, use, or management of this type of facility.

Regulation of Genome Editing in Human iPS Cells : A Comparative Legal Analysis of National Regulatory Frameworks for iPSC-based Cell/Gene Therapies

The book provides a concise overview of currently applicable regulatory frameworks of states which are among the world leaders in research and development (R&D) of cell and gene therapies. Developments in genome editing are expected to lead to new possibilities for the treatment of hereditary diseases in humans. The treatment of such often severe but hitherto uncurable diseases can be based on genome-edited induced pluripotent stem cells (iPS cells). Such treatments constitute combined cell/gene therapies. These therapies need to be governed by a regulatory framework which ensures quality, safety, and efficacy of the relevant therapeutic products. On the other hand, such regulations may retard product approval and impede R&D. Accordingly, national regulations for therapies based on genome-edited iPS cells are an important and, as the case may be, decisive factor for both researchers and industry regarding their decision where to locate their R&D activities. Therefore, regulatory frameworks impact significantly on the competitiveness of states and their economies. This is why a comparative analysis of laws and regulations of different countries matters. Such a comparative legal analysis provides an important insight into regulatory concepts which, in turn, may inspire adjustments of, or amendments to, domestic legal regimes. For this purpose, experts present country reports on France, Germany, Japan, South Korea, Switzerland, and the USA. The reports on France and Germany also refer to the parameters and implications arising from pertinent EU law. This contributed volume is aimed at researchers, but also at, e.g., legal scholars, lawmakers, regulators, and political decision makers.

Handbook of Stem Cell Therapy

The handbook comprehensively reviews the therapeutic potential of stem cells and stem cell-based cell-free strategies for the treatment of diseases. The initial chapters discuss the diverse applications of the stem and progenitor cells, including skeletal myoblasts, endothelial progenitor cells, adipose tissue-derived stem cells, induced pluripotent stem cells, mesenchymal stem cells, and neuronal stem cells in myocardial repair, inflammatory bowel disease, cognitive deficits, wound healing, retinal disorders, and COVID-19. The subsequent chapter summarizes methods for identifying and characterizing mesenchymal stem cells and discusses their properties and surface markers used for their prospective isolation. It also covers the therapeutic potential of mesenchymal stem cells in regenerative medicine. In turn, the book reviews the impact of stem cells derived secretome on various biological processes such as angiogenesis, neurogenesis, tissue repair, immunomodulation, musculoskeletal pathologies, wound healing, anti-fibrotic, and anti-tumor for tissue maintenance and regeneration.

Lastly, it summarizes treatment advantages, opportunities, and shortcomings in stem cell-based therapy, potentially helping to refine future trials and translate them from experimental to clinical studies. This reference book is a valuable resource for researchers involved in stem cell research to understand the multifaceted therapeutic applications of stem cells.‍

Potency Assays for Advanced Stem Cell Therapy Medicinal Products

This volume of the Springer book series Advances in Experimental Medicine and Biology covers potency assays, one of the most complex yet fundamental evaluations that critically influence stem cell regenerative medicine. Developing potency assays for cell-based medicinal products comes with numerous challenges due to the highly specialised nature of the application and purpose. This book provides the reader with the knowledge necessary to understand issues governing the successful development of potency assays, highlighting an international outlook of how the various challenges raised are being managed. Stakeholders concerned with potency assay development range from patient and clinician to contract research organisations, small and medium enterprise, regulatory authorities and even politicians. The value of potency assays is poised to increase given the inevitable watershed as early-stage clinical trials addressing safety progress to trials testing efficacy. Contributors from clinical, academic, industrial and regulatory sectors establish a broad point of view for guidance and timely debate. Potency assays require extensive collaboration across disciplines and sectors, as well as compromise and the authors aim to constructively address the many key aspects involved.

Potency assays provide a quantitative measure of the biological activity of advanced therapy medicinal products (ATMPs) and thus are required for their market authorization. As the pace of ATMP development accelerates, the need to develop specific, accurate, and robust potency assays for each product is also accelerating. The volume Potency Assays for Stem Cell Advanced Therapy Medicinal Products presents a broad outlook on the development, quality attributes, and implementation of potency assays for ATMPs. The first few chapters introduce a nuanced historical perspective on the science of potency assay development, describe specific quality attributes of an idealized potency assay, indicate pitfalls associated with developing such assays for ATMPs, and review guidance recommended by regulatory authorities on assay suitability for product approval. Subsequent chapters highlight efforts to develop potency assays for specific ATMPs, including skeletal stem cells, mesenchymal stromal cells, extracellular vesicles, CAR T-cells, and discuss emerging technologies/platforms for potency assay design. The volume concludes with a chapter reviewing potency assays used for the release of commercial ATMP products, which amalgamates information contained in previous chapters. Overall, the knowledge contributed from leading authorities in both academia and industry is an ideal resource for technicians, scientists, clinicians, process engineers, and regulators working with ATMPs.

―Donald G. Phinney, PhD Professor, Department of Molecular Medicine, Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology‍

Stem Cells in Tissue Regeneration, Therapy and Drug Discovery

Much research has focused on the basic cellular and molecular biological aspects of stem cells. Much of this research has been fueled by their potential for use in regenerative medicine applications, which has in turn spurred growing numbers of translational and clinical studies. However, more work is needed if the potential is to be realized for improvement of the lives and well-being of patients with numerous diseases and conditions. This book series 'Cell Biology and Translational Medicine (CBTMED)' as part of Springer Nature’s longstanding and very successful Advances in Experimental Medicine and Biology book series, has the goal to accelerate advances by timely information exchange. Emerging areas of regenerative medicine and translational aspects of stem cells are covered in each volume. Outstanding researchers are recruited to highlight developments and remaining challenges in both the basic research and clinical arenas. This current book is the 16th volume of a continuing series. Chapter Epithelial Stem Cells: Making, Shaping and Breaking the Niche is available open access under a Creative Commons Attribution 4.0 International License via link.springer.com.

Gene Therapy of Cancer: Methods and Protocols, 3rd Edition

This third edition provides new and updated chapters on gene therapeutic strategies of cancer. Chapters guide readers through suicide and oncolytic gene therapy, gene replacement and gene suppression therapy, vector development and refinement, immunogene therapy, TCR and CAR engineering, tumor vaccination using DNA or RNA vaccines, and antitumoral immune stimulation at different levels. Written in the format of the highly successful Methods in Molecular Biology series, each chapter includes an introduction to the topic, lists necessary materials and reagents, includes tips on troubleshooting and known pitfalls, and step-by-step, readily reproducible protocols.

Authoritative and cutting-edge, Gene Therapy of Cancer: Methods and Protocols, Third Edition aims to be a useful and practical guide to new researchers and experts looking to expand their knowledge.‍

Gene Therapy

Gene Therapy describes the delivery systems now available to target a given tissue with specific gene or oligonucleotide sequences, and explores the utility of animal modules as test systems. In the context of selected disease states, it summarises in vitro and in vivo studies and clinical trials performed to date.;Scope and limitations of gene therapy, K. Sikora. Tissue-directed gene delivery systems, C.R. Middaugh et al. Retroviral vectors, W.H. Gunzburg and B. Salmons. Adenovirus vectors, C.J.A. Ring. Adeno-associated virus vectors for human gene therapy, J.S. Bartlett and R.J. Samulski. Liposome delivery systems, R.K. Scheule and S.H. Cheng. Development of mammalian artificial chromosome vectors: prospects for somatic gene transfer, Z. Larin. Infectious herpes vectors for gene therapy, J.-M.H. Vos et al. Role of animal models in gene therapy, J.R. Dorin and D.J. Porteous. Gene targeting as an approach to gene therapy, A. Porter. Cystic fibrosis, N.J. Caplen and E.W.F.W. Alton. Gene therapy for haemophilia B, K. Kurachi and J.-M. Wang. Gene therapy for adenosine deaminase deficiency, H.B. Gaspar and C. Kinnon. Cardiovascular disease, B.A. French. Cancer gene therapy I: genetic intervention strategies, L.-A. Martin and N.R. Lemoine. Cancer gene therapy II: immunomodulation strategies, J. Galea-Lauri and J. Gaken. The muscular dystrophies, C.A. Sewry and T.A. Partridge. Recent developments in gene therapy for neurological disorders, P.R. Lowenstein.

Advances in Application of Stem Cells: From Bench to Clinics

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.

Stem Cell Therapy in Dermatological Disorders

This book provides a comprehensive understanding of the transformative potential of stem cell therapies for improving skin health and treating debilitating dermatological disorders.

Stem Cell Therapy in Dermatological Disorders delves into the emerging field of stem cell therapy as a revolutionary approach to treating various dermatological conditions. This book provides a comprehensive overview of the science behind stem cell technology, focusing on its applications in skin regeneration, wound healing, and the management of chronic skin diseases. It examines the cellular and molecular mechanisms that make stem cells uniquely suited for dermatological use, exploring their ability to promote tissue repair, modulate inflammation, and restore the skin’s structural integrity. This book aims to bridge the gap between experimental research and clinical application, presenting up-to-date findings on the different types of stem cells used in dermatology, including mesenchymal stem cells, induced pluripotent stem cells, and embryonic stem cells. It highlights the role of stem cells in addressing disorders such as atopic dermatitis, psoriasis, vitiligo, and chronic non-healing wounds. Readers will find in-depth discussions on current therapeutic techniques, the challenges of translating preclinical studies to human trials, and the ethical considerations associated with stem cell therapies.

In addition to discussing the state-of-the-art in stem cell-based treatments, the book also casts an eye on the future, identifying gaps in existing knowledge and potential areas for innovation. The authors provide insights into novel delivery systems, genetic engineering advancements, and combinatory approaches that may enhance the efficacy and safety of stem cell therapies in dermatology. This volume serves as an essential resource for dermatologists, researchers, and clinicians seeking to understand the transformative potential of stem cell therapies in improving skin health and treating debilitating dermatological disorders.

The EBMT Handbook: Hematopoietic Cell Transplantation and Cellular Therapies

This Open Access 8th edition of the European Society for Blood and Marrow Transplantation (EBMT) handbook addresses the latest developments and innovations in hematopoietic stem cell transplantation and cellular therapy. Consisting of 94 chapters, it has been written by 215 leading experts in the field. Discussing all types of stem cell and bone marrow transplantation, including selection of stem cell sources, it also covers the indications for transplantation, the management of early and late complications as well as the new and rapidly evolving field of cellular therapies and new infections (COVID-19). This book provides an unparalleled description of current practices to enhance readers’ knowledge and practice skills.

Handbook of Cell and Gene Therapy

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.

Cell Therapy：Stem Cells and Regenerative Medicine

This book provides an overview of cell-based therapy for human diseases including the definition, history, and clinical applications of human stem cells and their use in regenerative medicine. It covers human pluripotent stem cells (human embryonic stem cells and human induced pluripotent stem cells), mesenchymal stem/stromal cells, and hematopoietic stem cells, bone marrow mononuclear cells. In terms of clinical applications, this book also provides an update on recent human trials using these cells to treat various diseases, including neurological disorders, pulmonary dysfunctions, metabolic/endocrine-related diseases, frailty, and cancer treatment. In addition, it discusses the authors’ clinical trial experiences related to these conditions in a clinical setting to provide additional insight into regenerative medicine, especially cell-based therapy. The field of regenerative medicine has witnessed tremendous advancement in stem cell research and therapy in recent years, with thousands of preclinical studies supporting thousands of human clinical trials conducted worldwide. Emerging evidence in regenerative medicine and stem cell research suggests the treatments are safe, whereas their therapeutic potential, effectiveness, and mode of action require deeper investigation. It is important to introduce the science behind stem cell-based therapy to patients, clinicians, and researchers to further enhance understanding of responsible clinical conduct and so to educate and thus prevent the further emergence of unregulated, deceitful stem cell clinics solely operating for the sake of profit.

Pluripotent Stem Cell: Current Understanding and Future Directions

This Special Issue Reprint synthesizes the latest research articles about induced pluripotent stem cells (iPSCs) for easier study. Researchers have contributed ten new studies and an editorial overview of iPSC reprogramming as well as explored genetic editing tools, neural and organoid growing methods, new immunological solutions, and computer-based discoveries. The research shows that along with their present use in managing genetic diseases, iPSCs can become effective treatments through reprogramming and editing using CRISPR. The studies address modern technology, including machine learning that can identify cell characteristics and developmental signaling that can form new cell lines. The reprinted articles showcase the development of iPSC research and suggest methods by which scientists can use stem cells for medical advancement today.

Stem Cell Therapeutics

Discover the revolutionary potential of stem cell therapy and gain a comprehensive understanding of its applications, challenges, and ethical considerations across various medical fields with Stem Cell Therapeutics, a must-read for anyone interested in the future of medicine. Stem Cell Therapeutics delves into the forefront of medical research, exploring the revolutionary potential of stem cell therapy in combating a wide array of diseases. This comprehensive volume provides a thorough examination of the subject matter, offering insights into the latest advancements, methodologies, and applications of stem cell therapeutics. The book adopts a multidisciplinary approach, drawing upon expertise from fields such as biology, medicine, biotechnology, and bioengineering. Readers can expect to encounter in-depth discussions on the use of stem cells in treating various medical conditions, including neurodegenerative disorders, cardiovascular diseases, autoimmune conditions, and cancer. Each chapter provides a comprehensive overview of the current state of research for each disease, highlighting the challenges, successes, and prospects of stem cell-based therapies. Additionally, the book explores ethical considerations, regulatory frameworks, and commercialization strategies surrounding the field, ensuring a well-rounded understanding of the subject matter for readers at all levels of expertise.

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