Gene and Cell Therapies: Principles of Pharmaceutical Development explores increasing interest in gene and cell therapies following powerful demonstrations of their capability to treat rare genetic disorders, advanced cancers, and regenerate or repair tissues. Sections cover the design of viral vectors, oncolytic viruses, ribonucleic acid cancer vaccines, and autologous and allogeneic cell therapies, along with impacts on their pharmaceutical development. Advances in molecular and cell biology that have led to efficacious and safe gene and cell therapies are also covered, including their relationship to the process and analytical developments that ensure quality.
Gene and Cell Therapies: Principles of Pharmaceutical Development scope and style, along with included case studies, learning points, and lecture slides will allow non-specialists to develop a foundational understanding, while new concepts urge further advancements. Although these new, advanced medicines are continually being improved upon, their complex manufacture remains a key obstacle to increasing patient access.
1. Chapter 1. Introduction - p. 1 2. Chapter 2. Gene Therapies - p. 11 3. 2 Gene Therapies - p. 11 4. 2.1 Viral Vectors - p. 11 5. 2.2 CAR T-Cell Therapies - p. 36 6. 2.3 Messenger RNA Therapeutics - p. 48 7. 2.3.1 Regulatory Categorization - p. 48 8. 2.3.2 Mechanism of Action - p. 49 9. 2.3.3 Optimization of mRNA Immunogenicity and Stability - p. 52 10. 2.3.4 Self-Amplifying mRNA - p. 54 11. 2.3.5 Production of Synthetic mRNA - p. 54 12. 2.3.6 Future Perspectives for mRNA Gene Therapies - p. 61 13. 2.4 Oncolytic Viruses - p. 62 14. 2.5 Electroporation - p. 69 15. 2.6 Health Economics - p. 70 16. Chapter 3. Somatic Cell Therapies - p. 77 17. 3.1 Somatic Cell Therapies - p. 77 18. 3.1.1 Overview - p. 77 19. 3.2.1 Classification by US and EU Regulatory Agencies - p. 79 20. 3.3.1 Blood Products - p. 83 21. 3.4.1 Example Somatic Cell Therapy Medicinal Products (sCTMPs), Currently or Previously Marketed - p. 85 22. 3.5.1 Advances in Mesenchymal Stem Cell (MSC)–Based Therapies - p. 94 23. 3.6.1 The Manufacture of Mesenchymal Stem Cells (MSC)–Based Drug Products - p. 97 24. 3.7.1 The Therapeutic Potential of Human Embryonic Stem Cells (hESCs) - p. 103 25. 3.8.1 The Therapeutic Potential of Human Induced Pluripotent Stem Cells (hiPSCs) - p. 113 26. 3.8.1.1 Emergence to Current State - p. 113 27. 3.8.1.2 Human Leukocyte Antigen (HLA) Haplobanks and Hypoimmunogenic Cells - p. 114 28. 3.8.1.3 Quality Control Tests and Standardization - p. 116 29. 3.8.1.4 Manufacturing Considerations - p. 118 30. 3.8.1.5 Intellectual Property - p. 132 31. 3.8.1.6 Example Human Induced Pluripotent Stem Cell (hiPSC)–Derived Therapeutic Interventions - p. 132 32. 3.9.1 Tissue Engineered Products - p. 142 33. 3.10.1 Future Perspectives - p. 146 34. Chapter 4. Pharmaceutical Development - p. 153 35. 4.1 Quality by Design - p. 155 36. 4.2 The Formulation of Viral Vectors - p. 168 37. 4.3 Formulation of mRNA Cancer Vaccines - p. 184 38. 4.4 Formulation of Cell Therapy Products - p. 199 39. 4.5 Future Perspectives - p. 216 40. Glossary - p. 221 41. Index - p. 225
Gene and Cell Therapies: Principles of Pharmaceutical Development explores increasing interest in gene and cell therapies following powerful demonstrations of their capability to treat rare genetic disorders, advanced cancers, and regenerate or repair tissues. Sections cover the design of viral vectors, oncolytic viruses, ribonucleic acid cancer vaccines, and autologous and allogeneic cell therapies, along with impacts on their pharmaceutical development. Advances in molecular and cell biology that have led to efficacious and safe gene and cell therapies are also covered, including their relationship to the process and analytical developments that ensure quality.
Gene and Cell Therapies: Principles of Pharmaceutical Development scope and style, along with included case studies, learning points, and lecture slides will allow non-specialists to develop a foundational understanding, while new concepts urge further advancements. Although these new, advanced medicines are continually being improved upon, their complex manufacture remains a key obstacle to increasing patient access.
1. Chapter 1. Introduction - p. 1 2. Chapter 2. Gene Therapies - p. 11 3. 2 Gene Therapies - p. 11 4. 2.1 Viral Vectors - p. 11 5. 2.2 CAR T-Cell Therapies - p. 36 6. 2.3 Messenger RNA Therapeutics - p. 48 7. 2.3.1 Regulatory Categorization - p. 48 8. 2.3.2 Mechanism of Action - p. 49 9. 2.3.3 Optimization of mRNA Immunogenicity and Stability - p. 52 10. 2.3.4 Self-Amplifying mRNA - p. 54 11. 2.3.5 Production of Synthetic mRNA - p. 54 12. 2.3.6 Future Perspectives for mRNA Gene Therapies - p. 61 13. 2.4 Oncolytic Viruses - p. 62 14. 2.5 Electroporation - p. 69 15. 2.6 Health Economics - p. 70 16. Chapter 3. Somatic Cell Therapies - p. 77 17. 3.1 Somatic Cell Therapies - p. 77 18. 3.1.1 Overview - p. 77 19. 3.2.1 Classification by US and EU Regulatory Agencies - p. 79 20. 3.3.1 Blood Products - p. 83 21. 3.4.1 Example Somatic Cell Therapy Medicinal Products (sCTMPs), Currently or Previously Marketed - p. 85 22. 3.5.1 Advances in Mesenchymal Stem Cell (MSC)–Based Therapies - p. 94 23. 3.6.1 The Manufacture of Mesenchymal Stem Cells (MSC)–Based Drug Products - p. 97 24. 3.7.1 The Therapeutic Potential of Human Embryonic Stem Cells (hESCs) - p. 103 25. 3.8.1 The Therapeutic Potential of Human Induced Pluripotent Stem Cells (hiPSCs) - p. 113 26. 3.8.1.1 Emergence to Current State - p. 113 27. 3.8.1.2 Human Leukocyte Antigen (HLA) Haplobanks and Hypoimmunogenic Cells - p. 114 28. 3.8.1.3 Quality Control Tests and Standardization - p. 116 29. 3.8.1.4 Manufacturing Considerations - p. 118 30. 3.8.1.5 Intellectual Property - p. 132 31. 3.8.1.6 Example Human Induced Pluripotent Stem Cell (hiPSC)–Derived Therapeutic Interventions - p. 132 32. 3.9.1 Tissue Engineered Products - p. 142 33. 3.10.1 Future Perspectives - p. 146 34. Chapter 4. Pharmaceutical Development - p. 153 35. 4.1 Quality by Design - p. 155 36. 4.2 The Formulation of Viral Vectors - p. 168 37. 4.3 Formulation of mRNA Cancer Vaccines - p. 184 38. 4.4 Formulation of Cell Therapy Products - p. 199 39. 4.5 Future Perspectives - p. 216 40. Glossary - p. 221 41. Index - p. 225
