The development and manufacture of biological medicines from a company’s perspective 3900-BT1-11
University training in molecular biology produces graduates who can design an experiment but who have rarely seen how an experiment becomes a product. This series closes that gap by bringing in people who have done it. The organisations represented were chosen to span the whole value chain and, deliberately, to span outcomes: programmes that reached patients, programmes that were sold, programmes that were stopped, and business models that exist because of how the sector is financed rather than because of any particular molecule. The intent is not to survey companies. Each lecture is a case study in a decision made under uncertainty, with incomplete data and finite money, which is the condition under which nearly all translational work is actually done. Students are asked throughout to separate the scientific question from the development question, because a mechanism can be correct and a programme still fail, and the reasons for that are systematic enough to be taught.
The series opens at the source of the material everything else operates on: the large-scale academic discovery engine. A research institute organised around shared platforms and data rather than around individual laboratories generates target hypotheses through genomics, functional genetics and chemical biology at scale, and this opening session examines how that environment works, how it differs from a conventional department, and how a finding leaves it for a company. It also sets the standard of target validation on which every later lecture depends, since the whole chain inherits whatever confidence, or false confidence, is established here.
The core of the series then takes the observation that the same disease biology can be addressed with a small molecule, a protein, a nucleic acid, a living cell or a diagnostic, and that the choice of modality determines everything downstream: manufacturing, regulatory route, cost of goods, clinical design, and the questions a company must answer before it can raise money. Nucleic-acid therapeutics are treated first, through messenger and circular RNA, covering both the chemistry of the molecule itself — cap structure, nucleoside modification, sequence and topology — and the delivery problem that governs whether any of it reaches the intended tissue; students see how a chemical insight developed in an academic laboratory becomes a platform on which several indications can be built, and why a platform is financed quite differently from a single drug. Biologics follow, with antibody and protein therapeutics used to show how discovery and engineering interact with immunological target selection, how manufacturing complexity constrains a biologic from the first day of development rather than at scale-up, and where the translational risk lies when mouse models are weak predictors of an immunological mechanism. Cell and gene therapy carries the material that nothing else in a molecular biology curriculum covers: a product that is a cell population rather than a compound, manufactured for one patient at a time, released against a specification that must be fixed before the clinical data exist, under a regulatory framework for advanced therapies and manufacturing conditions that together dominate the company's cost structure. The microbiome sessions treat a defined community as both a therapeutic and a diagnostic target, and use it to confront the exceptional difficulty of establishing causality and a reproducible product specification in that setting. Diagnostics and medical devices close the block, serving two purposes at once: diagnostics development follows a different regulatory and commercial logic from therapeutics, resting on analytical and clinical validation, the in-vitro diagnostics framework, and purchasing decisions made by health systems rather than by prescribers; and the build-and-divest model common in the field illustrates a route to value that does not require becoming a commercial pharmaceutical company at all.
Development at scale is then examined from inside an established, research-driven pharmaceutical company specialising in a therapeutic area where efficacy is notoriously hard to demonstrate. This session covers how a global portfolio is governed, how compounds are prioritised and killed, and what late-stage clinical development and lifecycle management actually involve. Its placement after the modality block is deliberate: with concrete cases already in hand, students can compare how the same decision is reached in a twenty-person start-up and in an organisation running global phase III trials, and see which differences are matters of scale and which are matters of kind.
The final block addresses the environment without which none of the preceding work reaches a patient, since science does not travel on scientific merit alone. One session covers the institutional support structure for biomedical innovation — public funding instruments, non-commercial clinical trials, and the mechanisms through which research is translated in the national and European setting. A second turns to the sector's labour market seen from the recruiter's side: how biotechnology and medical-technology organisations are actually staffed, what a scientific advisory board is for, which competences are scarce, and how a scientific career maps onto the roles that industry offers. A third takes the stakeholder environment in which reimbursement and public trust are decided, and examines how evidence is communicated and contested among patients, clinicians, payers, regulators, media and government.
Individual speakers are scheduled according to availability, and the order of sessions within a block may vary between editions; the opening and closing sessions are fixed, because the series is constructed to begin with the origin of the evidence and to end with the conditions under which it is judged.
Course coordinators
Type of course
Mode
Prerequisites (description)
Learning outcomes
Knowledge:
1. Describe the key stages of biotechnology product development, from early discovery through preclinical and clinical development to regulatory approval and commercialisation.
2. Explain the principal business models, funding strategies and partnership structures used in the biotech sector.
3. Identify the regulatory frameworks governing the development of biologics, nucleic acid medicines and cell and gene therapies in major jurisdictions.
4. Describe common causes of failure in biotech product development programmes and explain how risk is managed at each stage.
Skills:
1. Analyse real-world biotech case studies and identify the scientific, regulatory, financial and strategic decisions that shaped their outcomes.
2. Evaluate the translational potential of early-stage research findings using criteria applied in industry settings.
3. Extract and synthesise information from scientific publications, regulatory documents, investor reports and company communications.
4. Present a structured case-study analysis to a mixed scientific and non-scientific audience.
Competences:
1. Critically assess the scientific and commercial viability of a biotechnology programme.
2. Engage productively with industry professionals and formulate informed questions regarding strategic, scientific and operational decisions.
3. Recognise and reflect on the ethical responsibilities associated with biotechnology product development, including patient access, equity and dual use.
4. Apply an interdisciplinary perspective — integrating scientific, regulatory and business considerations — when evaluating decisions made in a biotech context.
Assessment criteria
The assessment takes the form of an individual written assignment, to be submitted within one week of the final lecture. Students are provided with a brief description of a realistic biotechnology industry scenario—such as a decision to advance a program to clinical trials, licensing negotiations, the selection of a platform technology, or a response to regulatory hurdles—and are tasked with preparing a structured written analysis of 800 to 1,200 words.
The analysis must identify key scientific, regulatory, financial, and strategic issues; present a coherent and well-substantiated argument; and draw upon concepts and examples discussed during guest lectures. Students are expected to critically evaluate the available options and propose and justify a specific course of action, while employing precise and appropriate scientific and industry terminology.
Grading will be based on the depth and relevance of the analysis, the quality of the argumentation, the extent to which course material is utilized, and the clarity of expression. Top marks will be awarded to papers that demonstrate original, critical thinking and a coherent, effective integration of scientific, regulatory, and business perspectives. Passing the assignment requires identifying the main issues, presenting a sound line of reasoning, and demonstrating a command of the course material. Papers that fail to meaningfully address the scenario or do not demonstrate a sufficient understanding of the topics covered will receive a failing grade.
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Recomended books:
1) Hill RG, Rang HP (eds). Drug Discovery and Development: Technology in Transition. Elsevier, 3rd ed., 2021. — The single best overview of the technical pipeline; recommended as the course's main reference.
2) Crommelin DJA, Sindelar RD, Meibohm B (eds). Pharmaceutical Biotechnology: Fundamentals and Applications. Springer, 5th ed., 2019. — Biologics, formulation and delivery; supports L4, L6, L9, L12.
3) Walsh G. Biopharmaceuticals: Biochemistry and Biotechnology. Wiley. — Manufacturing-oriented treatment of protein therapeutics; use alongside the Biopharmaceutical benchmarks series.
4) Strohl WR, Strohl LM. Therapeutic Antibody Engineering. Woodhead/Elsevier, 2012. — Detailed reference for L6; dated on formats, still sound on principles.
5) Friedman LM, Furberg CD, DeMets DL, Reboussin DM, Granger CB. Fundamentals of Clinical Trials. Springer, 5th ed., 2015.
6) Drummond MF, Sculpher MJ, Claxton K, Stoddart GL, Torrance GW. Methods for the Economic Evaluation of Health Care Programmes. Oxford University Press, 4th ed., 2015.
7) Shimasaki C (ed). Biotechnology Entrepreneurship: Leading, Managing and Commercializing Innovative Technologies. Academic Press, 2nd ed., 2020.
8) Simon F, Giovannetti G. Managing Biotechnology: From Science to Market in the Digital Age. Wiley, 2017..
9) Onetti A, Zucchella A. Business Modeling for Life Science and Biotech Companies. Routledge, 2014.
10) Goldacre B. Bad Pharma: How Medicine Is Broken, and How We Can Fix It. Fourth Estate, 2012.
11) Kinch MS. A Prescription for Change: The Looming Crisis in Drug Development. University of North Carolina Press, 2016.
12) Werth B. The Billion-Dollar Molecule: The Quest for the Perfect Drug. Simon & Schuster, 1994 (and its sequel The Antidote, 2014).
13) Isaacson W. The Code Breaker. Simon & Schuster, 2021.
14) Mukherjee S. The Gene: An Intimate History. Bodley Head, 2016.