Learning outcomes of the course unit
The main goal of the course is to provide basic information to know and understand the properties, application and problems related to the production, characterization and use of drugs of biotechnological origin.
The comprehensive introduction on methodologies for production, characterization and purification of protein therapeutics, and the description of their pharmacodynamic and pharmacokinetic properties, toxicity and immunogenicity will allow the student to apply his/her knowledge to the identification and evaluation of the relevant properties and issues related to each biotech drug.
Basic concepts of medicinal chemistry, biochemistry, organic chemistry, immunology and pharmacology are needed to understand the structure, action, application and limitations of recombinant therapeutic proteins.
Course contents summary
The first part of the course provides basic knowledge about biotech methodologies and biotechnology-related techniques and their application to the design, production, downstream processing and characterization of biotech drugs.
The second part of the course will cover the most important classes of biotech drugs in different therapeutic areas, such as hormones, enzymes, cytokines, monoclonal antibodies. For each drug, relevant aspects of its production, mechanism of action, therapeutic application, as well as its pharmacokinetic properties and toxicity will be treated.
Structure-activity and structure-property relationships will be described for biotech drugs differing from the structure of physiological proteins, to understand their modified/improved pharmacodynamic or pharmacokinetic properties.
Introduction to biotechnology.
Basic elements of molecular biotechnology.
Production of biotech compounds: cultivation and downstream processing.
Biophysical and biochemical analyses of recombinant proteins.
Chemical and physicochemical properties and reactivity of recombinant proteins; stability of proteins and mechanisms of degradation.
Pegylation of therapeutic proteins
Fusion proteins and soluble receptors.
Hormones: insulin, glucagon, growth hormone, follicle-stimulating hormone, luteinizing hormone, parathyroid hormone.
Cytokines: interleukines (IL-1, IL-2); interferons; hematopoietic growth factors (G-SCF, erythropoietin).
Blood proteins: tissue-type plasminogen activator, clotting factors (VII, VIII, IX).
Enzymes: human deoxyribonuclease, beta-glucocerebrosidase, alpha-galactisodase.
Recombinant vaccines: genetically improved live vaccines, live vectors, genetically improved subunit vaccines
Monoclonal antibodies, immunoconjugates and radioimmunoconjugates: rituximab, ofatumumab, ibritumomab tiuxetan, gemtuzumab ozogamicin, muromonab, catumaxomab, daclizumab, basiliximab, infliximab, adalimumab, certolizumab pegol, cetuximab, bevacizumab, ranibizumab.
Soluble receptors: etanercept, abatacept.
Maria Luisa Calabrò, Compendio di Biotecnologie Farmaceutiche, EdiSES, Napoli, 2008.
Daan J.A. Crommelin, Robert D. Sindelar, Biotecnologie Farmaceutiche, Zanichelli, Bologna.
Thomas L. Lemke, David A. Williams, Victoria F. Roche, S. William Zito, Foye’s Principi di Chimica Farmaceutica, sesta edizione italiana, Piccin, Padova, 2013. (for the specific chapter related to pharmaceutical biotechnology. An English version of this book is available)
Daan J.A. Crommelin, Robert D. Sindelar, Bernd Meibohm, Pharmaceutical Biotechnology, Fourth Edition, Springer, 2013.
Teacher-led lessons with slides.
Assessment methods and criteria
An oral examination, with questions related to all the topics treated during the course, will be used to assess the knowledge and comprehension of the contents of the course achieved by the student. The ability of the student to apply the acquired knowledge will be also evaluated through connections among topics.
Preparation is be considered sufficient if the student proves knowledge and understanding of the basic aspects of each topic and is able to apply this knowledge to the discussion. During the examination use of an appropriate terminology will also be assessed.
The grade for the course of Biotechnological Drugs/ Supplement to Pharmaceutical Chemistry will be the average grade obtained for the two modules.