Description
Book SynopsisFrom mRNA vaccines to gene therapies, the next frontier of medical innovation is here. In Building Breakthroughs, Raju Prasad tells the story of important advancements in biotechnology and medical innovation from gene therapies to mRNA vaccines, providing historical context and examining cutting-edge research. Based on in-depth interviews with both the scientists who developed these discoveries and the patients who have benefited from them, Building Breakthroughs reveals the key players behind drug development and the inner workings of this essential business. Through stories of patients and their families, and of the researchers creating new treatments, Prasad reveals how cell therapies are advancing to treat childhood leukemia and a form of lymphoma, how a gene therapy was established to treat the rare disease spinal muscular atrophy type I, and potentially curative therapies that are being developed for sickle cell disease. By examining the clinical trial and regulatory paths each
Table of ContentsIntroduction
Part I. The Journey from Idea to Approval
Chapter 1. Leukemia and Lymphoma
Chapter 2. Chimeric Antigen Receptor (CAR)-T Cell Therapy
Chapter 3. Spinal Muscular Atrophy
Chapter 4. In Vivo Gene Therapy
Chapter 5. Sickle Cell Anemia
Chapter 6. Ex Vivo Gene-Modified Cell Therapy
Chapter 7. A Brief History of FDA Regulation
Chapter 8. The Path to Approval
Part II. Enabling the Next Wave Of Innovation
Chapter 9. Building Blocks
Chapter 10. Recognizing Value
Chapter 11. Financial and Human Capital Investments in Biotechnology
Chapter 12. Looking Forward
Epilogue. COVID-19
Acknowledgments
References
Index
