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Second Generation Cell and Gene-Based Therapies
Biological Advances, Clinical Outcomes and Strategies for Capitalisation

The only volume on the market to bridge the fields of basic science, clinical therapy, technology development and business

Alain Vertes (Edited by), Nathan J. Dowden (Edited by), Devyn Smith (Edited by), Nasib Qureshi (Edited by)

9780128120347, Elsevier Science

Hardback, published 10 February 2020

846 pages
23.4 x 19 x 4.2 cm, 1.86 kg

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.

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?

Subject Areas: Biomedical engineering [MQW], Clinical & internal medicine [MJ]

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