PHARMACEUTICAL BIOTECHNOLOGY: BIOTHERAPEUTIC DRUG DISCOVERY, ENGINEERING, AND DELIVERY

The "Pharmaceutical biotechnology: biotherapeutic drug discovery, engineering and delivery" course is one of the training activities of the PhD course in Bio and Nanomaterials Science and Technology. The course allows students to acquire the knowledge of the main concepts related to the properties, the development and the applications of the major biomacromolecules and cell-based therapies.

The aim of the course is to introduce the students to biotherapeutics, engineered biomacromolecules and cells that are developed by means of biological processes involving recombinant technologies. Particular attention will be dedicated to the properties and the methodologies used to develop most of the biomacromolecules (e.g. nucleic acids, peptides and proteins) and cells (e.g. immune cells, stem cells, red blood cells) currently used in the clinic. The students are supposed to be able to appreciate the advantages and the disadvantages of the different biotherapeutics and being able to recognise and evaluate, through a critical analysis, which molecular format may be more suitable for a given therapeutic application.

Basic knowledge of pharmacology, molecular, chemical, structural and cellular biology.

Contents of the course:
• Principles of pharmacology
• Peptide therapeutics: advantages and disadvantages. Natural product derived peptides as starting points for drug discovery. Display technologies in peptide lead discovery: phage display, mRNA display and ribosome display. Stapled peptides. Strategies to extend the half-life of peptides;
• Cytokine-based therapies: benefits and limitations. Interleukins approved for therapy. Engineering and synthetic cytokines signalling for therapeutics.
• Non-antibody scaffold drugs: advantages and major challenges. Strategies for the generation of differentiated non-antibody scaffold drugs. Examples of non-antibody scaffold molecules for therapeutic, diagnostic and biotechnological applications.
• Therapeutic antibodies: properties and mode of actions. Multidimensional engineering of antibodies: antigen binding, Fc receptor binding and modularity. Multispecific antibodies. Antibody fragments. Antibody drug conjugates (ADCs).
• Cell-based therapies. Adoptive cell therapy using tumour-infiltrating lymphocytes (TIL). Engineered T cell expressing chimeric antigen receptors (CAR-T). Therapeutic approaches to enhance natural killer (NK) cell cytotoxicity against cancer. Macrophages in immunotherapy. Engineering stem cells for cancer therapy. Dendritic cell (DC) in cancer immunotherapy. Antigen-specific therapeutic approaches for autoimmunity. Red blood cell-based therapies.
• Nucleic acid therapeutics: advantages and disadvantages. Principle of systematic evolution of ligands by exponential enrichment (SELEX). DNA, RNA aptamers. Antisense oligonucleotide (AON). RNA interference (RNAi). Ribozymes.
• Development of small organic DNA-encoded molecules.

All needed material will be provided in class.

The assessment of learning takes place by means of a short oral presentation. The objective of the exam consists in verifying and evaluating the ability of the student to include at least one content of the course in her/his current PhD research project. The students must demonstrate a critical thinking and the ability to expose the topic in a formal and concise manner using an appropriate scientific language. The constant and active participation to the lessons will be taken into account in the final evaluation.

Teaching is organized in frontal theoretical lectures in the classroom. PowerPoint slides will be used during the lessons. The teaching material is present and downloadable from the University's Moodle e-learning platform.

Accessibility, Disability and Inclusion

Accommodation and support services for students with disabilities and students with specific learning impairments: Ca’ Foscari abides by Italian Law (Law 17/1999; Law 170/2010) regarding support services and accommodation available to students with disabilities. This includes students with mobility, visual, hearing and other disabilities (Law 17/1999), and specific learning impairments (Law 170/2010). In the case of disability or impairment that requires accommodations (i.e., alternate testing, readers, note takers or interpreters) please contact the Disability and Accessibility Offices in Student Services: disabilita@unive.it

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This subject deals with topics related to the macro-area "Human capital, health, education" and contributes to the achievement of one or more goals of U. N. Agenda for Sustainable Development