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

Scientists and students in stem cell biology, pharmaceutical science, regenerative medicine, cancer research, clinical trials, gene therapy, corporate strategists & investors in these fields

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?