From 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 benefitted 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 therapy took to reach approval, Prasad uncovers the building blocks of biotechnology innovation and the investments that must be made to catalyze the development of future breakthroughs. He also explores issues of scientific communication and misinformation, providing recommendations for improvements in the future. For those seeking to understand the vitally important processes that lead to new medicines and the surrounding ecosystem that is enabling the next generation of innovative medicines that have the potential to transform patient outcomes, Building Breakthroughs is essential reading.
Raju Prasad, PhD (CHICAGO, IL), is a partner and biotechnology analyst at William Blair, focusing on cell therapy, gene therapy, and gene editing. Dr. Prasad previously worked as a research associate with the University of North Carolina at Chapel Hill's Gillings School of Global Public Health and as an independent consultant with the US Environmental Protection Agency.
Introduction 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
From mRNA vaccines to gene therapies, the next frontier of medical innovation is here.
