Second-Generation Cell and Gene-Based Therapies

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

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

PART I: SCIENCE
1. Riding the technology S-curves in cytotherapies
2. Gene therapy
3. Therapeutic potential of cells of the immune system
4. T-cell engineering
5. Engineered hematopoietic stem cells
6. Second generation MSCs
7. First generation induced Pluripotent Stem Cells
8. Editing technologies for the mammalian genomes
9. The Next Wave:  Tissue Replacement and Organ Replacement

PART II: TRANSLATION
10. Gene Therapy clinical trials
11. CAR T-cells clinical trials
12. Cytotherapy clinical trials in genetic disorders of the blood
13. Late stage Clinical trials data from minimally manipulated MSCs and translational advances with genetically engineered MSCs

PART III: THE NEXT FRONTIER
14. Second generation genome editing technologies in drug discovery
15. Harnessing the therapeutic potential of dendritic cells
16. Second generation CAR T-cells
17. Capitalising on the potential of neural stem cells in oncology and CNS
18. Combinatory cytotherapy

PART IV: PERSPECTIVES
19. Cytotherapy manufacturing: service-based business model vs. off-the-shelf blockbuster business model
20. Second generation cytotherapy business models: the case of CAR-T cells business sustainability
21. Financing the new wave of transformational oncology treatments
22. Strategic alliances in cytotherapies and gene therapies. Funding the new wave of cell therapeutics
23. Solid tumor immune-oncology