Book Overview
What this book is:
a narrative of the stunning technological advances of molecular and cellular biology in the late 20th and early 21st centuries, and their application to the development of the industry and its products—told through [the author’s] lens on the companies that were set up to exploit them.
What this book is not: a comprehensive review of technologies and companies, since it relies on the author’s personal experience for book structure and conclusions.
Chapter breakdown:
- Chapters 1, 2, 3: early days and forming of recombinant DNA-based companies on both coasts of US and in UK
- Chapters 4, 5: development of monoclonal antibodies (mAbs), companies founded on mAbs, companies and leaders who developed blockbuster mAb products
- Chapter 6: author “draws on [his] experiences at Glaxo Group Research (early 1990s) to describe what pharma companies were beginning to do w/ these new technologies and the strategic alliances they formed” (p.14). he also describes how smaller biotech companies—using combinatorial chemistry and screening—developed new small molecule drugs for old and new targets, when this used to be pharma’s core strength.
- Chapter 7, 8: advances in genetics and genomics technologies and their applications in the late 20th century - 21st century.
- excitement around cloning genes that cause major forms of inherited Mendelian diseases (e.g. CF, Huntington’s) and companies formed on this idea (Sequena Tx)
- Human Genome Project
- Public (The Institute for Genome Research) vs. private (Human Genome Sciences, Celera, Incyte) race to sequence the human genome
- Chapter 9: functional genomics (science of understanding primary function of genes associated w/ diseases), companies founded on idea that less complex organisms (e.g. worms, fruit flies) would reveal function of unknown genes of relevance to human disease, uncovering protein function by understanding protein 3D structure using crystallography technologies informed by genomics-driven approach (e.g. Structural GenomiX - Chapter 10)
- Chapter 11: companies applying genomics to get insights into rare diseases, finding and developing medicines to treat them (e.g. Genzyme) + their business models, despite trad pharma expressing business concerns over this business model
- Chapter 12: antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs)—technologies that can reliably alter gene expression by blocking mRNA translation—signature companies founded on these technologies, and products made from applying these technologies
- Chapter 13: early stages of gene therapy (directly replacing or repairing a defective gene)—technologies used, their applications, activities of some gene therapy companies
- Chapter 14: cell therapy—important new companies are still being formed, leading to development of new cell therapy products e.g. CAR T cells to treat blood cancers
- early days mirror what happened in gene therapy
- Chapter 15: earlier-developed DNA-editing technologies and companies that exploited them, companies founded on CRISPR-Cas9 gene-editing technology (editing in vivo and ex vivo) and locked in patent battles
- Chapter 16: vaccines developed using biotech techniques and their impact on human health and some historical perspectives, diseases caused by viruses, how biotech has influenced vaccine design
- biotech success stories of development of Hep B and HPV vaccines
- development of more recent mRNA-based vaccines against SARS-CoV-2
- Chapter 17, “Fortunes and Unicorns” - dark art of valuation, challenges of determining, justifying, and maintaining a high valuation, especially as a unicorn (company w/ valuation of > $1B)
- Chapter 18, “The Essence of Biotech” - defines primary traits that determine success or failure of companies in biotech. How the biotech industry has changed / not changed from the 1970s - 2020s
Biotech is not just science! If biotech is successfully applied or not depends on the company (founders, finances, leadership), stakeholders (patient groups, regulatory bodies), personalities, events, geography, etc.