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.
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Table of Contents
PART 1 Science 1. Innovation S- Curves in Living Drugs Development and Their Commercialisation 2. Development and Deployment of Gene Therapies: An ADA- SCID Case Study 3. Therapeutic Potential of Cells of the Immune System 4. T Cell Engineering and the Rise of CAR- T Cell Therapies 5. Harnessing Natural Killer Cells' Killing Function in Cancer 6. Pluripotent Stem Cell-Derived Islet Replacement Therapy for Diabetes 7. Harnessing in Silico Technologies to Develop and Augment Second- Generation Cell- Based Therapies 8. Second Generation Genome Editing Technologies in Drug Discovery 9. The Next Wave: Tissue Replacement and Organ Replacement 10. Combining Stem Cells and Materials for Nerve Tissue Regeneration
PART 2 Translation 11. Gene Therapy Clinical Trials: Past, Present and Future 12. CAR-T Cell Clinical Trials Experience Past, Present and Future 13. T Cell Receptor Engineered T Cell Therapy in Oncology 14. Cytotherapy Clinical Trials in Genetic Disorders of the Blood and Options for Reimbursement
PART 3 The Next Frontier 15.Mesenchymal Stromal Cell Therapies The Next Frontiers 16. Regenerative Medicine and Ageing: Is Senescence Reprogrammable? 17. Deciphering the Systems Architecture of the Brain Using Molecular Can Openers 18. Harnessing the Therapeutic Potential of Dendritic Cells 19. Brief Overview of Chimeric Antigen Receptor-Mediated Immunotherapy for Glioblastoma Multiforme 20. Combination Therapies in Solid Tumour Oncology
PART 4 Perspectives 21. Gene- Editing Technologies in Adoptive T Cell Therapy for Cancer: An Ethical Analysis 22. Decisions in the Development Lifecycle of Cell and Gene Therapies 23 CAR- T: From Concepts to Products Now What? 24. Strategic Alliances in Cytotherapies and Gene Therapies: Funding the New Wave of Cell Therapeutics 25. The Role of Governments in the Commercial Emergence of Radical Innovation: The Case of the United Kingdom 26. Patenting Trends in Chimeric Antigen Receptor Technologies 27. Reimbursement and Payment Models for Therapies With Transformative and Curative Intent 28. Commercialising CAR- T Therapies: The Evolution of a Revolution 29. Will You Move the Needle of Medicine?
Authors
Alain Vertes NxR Biotechnologies GmbH, Basel, Switzerland.University of Illinois at Urbana-Champaign, Institut Pasteur Paris, and University of Lille Flanders-Artois
. Sloan Fellow from London Business School and a microbiologist by training (University of Illinois at Urbana-Champaign, Institut Pasteur Paris, and University of Lille Flandres-Artois).
. Consulting, Partnering & Fund Raising, Founder, Managing Director, NxR Biotechnologies, Switzerland.
. Dr. Vert?s is a strategy and business development consultant for cell therapeutic biotech companies, and works to enable seed funding in the field. Focusing on technology deployment and innovation commercialization, he has contributed to both industrial and pharmaceutical biotechnology, in different functions including research, manufacturing, contract research, and strategic alliances in pharmaceuticals (Lilly, Pfizer, Roche).
. Championed radical innovation for bringing to patients disease-modifying, paradigm-changing therapeutics such as siRNA, and led in a scientific and business manner Roche's global cell therapeutics strategy and implementation team resulting in Roche's entry in 2010 in the field of regenerative medicine.
. Member, Industry Committee-Europe, Tissue Engineering and Regenerative Medicine International Society
. Co-Editor, Stem Cells in Regenerative Medicine: Science, Regulation and Business Strategies (Wiley, 2015).
Nathan J. Dowden Senior Vice President, Corporate Development, Rubius Therapeutics.
Devyn Smith Head of Operations and Strategy, Medicinal Sciences Division of R&D, Pfizer Inc..
. Head of Operations & Strategy, Medicinal Sciences Division of R&D, Pfizer Inc.
. Responsible for all cell & gene therapy products, as well as previously developing Pfizer's Regenerative Medicine program.
. PhD in Genetics from Harvard Medical School.
. Adjunct faculty member at Northeastern University.
Nasib Qureshi USDA-ARS-NCAUR, IL, USA. Affiliate Faculty, Institute for Genomic Biology, University of Illinois at Urbana-Champaign (UIUC), IL, USA (2004-2011). Adjunct Professor, UIUC (Biochemical Engineering, 2003-2018)..
Research Chemical/Biochemical Engineer, United States Department of Agriculture (USDA), Agricultural Research Service (ARS), National Center for Agricultural Utilization Research (NCAUR), Peoria, IL, USA.
PhD (Chemical/Biological Engineering), PhD (Fermentation Technology)
Fellow in American Institute of Chemical Engineers (AIChE), Society for Industrial Microbiology & Biotechnology (SIMB), and American Institute of Chemists (AIC).
Co-Editor, Stem cells in Regenerative Medicine (Wiley, 2015)
