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Second Generation Cell and Gene-Based Therapies

  • ID: 4539987
  • Book
  • January 2020
  • Region: Global
  • 846 Pages
  • Elsevier Science and Technology
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Second Generation Cell and Gene-Based Therapies: Biological Advances, Clinical Outcomes, and Strategies for Capitalization serves as the only book on 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 that will ultimately facilitate clinical delivery of cutting-edge, curative products.

Chapters cover hematopoietic stem cells, mesenchymal stem cells, tissue engineering, CAR-T cells, and cells of the immune system, as well as enabling technologies, such as gene and genome editing. Additionally, deep dives in product fundamentals, the history of the science, the pathobiology of diseases, the scientific and technological bases, and financing and technology adoption constraints are taken in order to unravel what will shape the cytotherapy industry in 2025 and beyond.

  • 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
  • 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
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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?

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Vertes, Alain
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. Vertès 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).
Dowden, Nathan J.
Smith, Devyn
. 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.
Qureshi, Nasib
US Department of Agriculture, National Center for Agricultural Utilization Research, Bioenergy Research Unit, Peoria, IL US
US Department of Agriculture, National Center for Agricultural Utilization Research, Peoria, IL US
. Senior Researcher/Senior Scientist, United States Department of Agriculture, National Center for Agricultural Utilization Research, Bioenergy Research Unit.
. Affiliate Faculty, Institute for Genomic Biology, Univ. of Illinois.
. Adjunct Professor (Biochemical Engineering), University of Illinois.
. EIC, World Journal of Microbiology & Biotechnology (Springer).
. Co-Editor, Stem Cells in Regenerative Medicine: Science, Regulation and Business Strategies (Wiley, 2015).

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