1. Knowledge and Understanding

At the end of the course, students will be able to:

• Describe the fundamental principles of pharmaceutical biotechnology;
• Illustrate the basics of recombinant DNA technology;
• Explain strategies for the production of recombinant proteins in prokaryotic and eukaryotic systems;
• Identify the main categories of biotechnological drugs;
• Describe the principles of purification and characterization techniques for biological macromolecules;
• Recognize the therapeutic applications of recombinant proteins, monoclonal antibodies, vaccines, and synthetic oligonucleotides.

2. Applying Knowledge and Understanding

Students will be able to:

• Apply bacterial transformation techniques;
• Perform cell selection procedures and evaluate gene expression;
• Carry out extraction and purification procedures for recombinant proteins;
• Interpret experimental results generated during laboratory activities;
• Calculate transformation efficiency and evaluate the quality of the obtained results;
• Use molecular visualization software for the structural analysis of biomacromolecules.

3. Making Judgements

Students will be able to:

• Critically analyze experimental results;
• Evaluate alternative strategies for the production of biotechnological drugs;
• Compare advantages and limitations of different expression platforms;
• Interpret data related to protein purification and molecular characterization processes;
• Critically discuss the advantages and limitations of modern biotechnological technologies.

4. Communication Skills

Students will be able to:

• Clearly and comprehensively explain biotechnological concepts and methodologies;
• Use the specialized terminology of pharmaceutical biotechnology appropriately;
• Justify methodological choices employed in biotechnological production processes;
• Discuss clinical applications of biotechnological drugs.

5. Learning Skills

Students will be able to:

• Independently retrieve scientific information from specialized bibliographic sources;
• Continuously update their knowledge in a field characterized by rapid scientific and technological development;
• Independently explore new biotechnological methodologies;
• Integrate knowledge derived from biological, pharmacological, chemical, and pharmaceutical technological disciplines.

The course consists of lectures (4 ECTS credits; 28 hours) and individual laboratory practical sessions (2 ECTS credits; 30 hours).

The course may be adapted to blended or distance-learning formats when necessary, while ensuring full coverage of the syllabus.

Should the course be delivered in blended or remote mode, the necessary modifications may be introduced to ensure compliance with the syllabus and learning objectives.

Students are expected to have acquired knowledge from the following courses: general and inorganic chemistry, animal biology and plant biology, molecular biology, human anatomy, organic chemistry, analytical chemistry, microbiology, biochemistry and applied biochemistry, general physiology, medicinal and toxicological chemistry, pharmaceutical analysis laboratory, pharmacology and pharmacotherapy.

Attendance is mandatory, as required by the degree regulations of the CTF program.

Absences are permitted for up to 30% of the total course hours, considering all teaching activities.

Theoretical Part

Module 1 - Fundamentals of Pharmaceutical Biotechnology

• Definition of pharmaceutical biotechnology: multidisciplinary convergence in biotechnology.
• Development of biotechnological processes and biotechnology companies.
• Recombinant DNA technology.
• Large-scale production of recombinant proteins: design and production of biotechnological drugs.
• Manipulation of gene expression in prokaryotes.
• Production of recombinant proteins in eukaryotic cells.
• Analytical and purification techniques for biological macromolecules.

Module 2 - Therapeutic Proteins

• Insulin and insulin analogues.
• GLP-1 receptor agonists (incretin mimetics).
• Hematopoietic growth factors: G-CSF, GM-CSF, EPO, IL-2.
• Interferons.

Module 3 - Oligonucleotides (ONs)

• Medicinal chemistry, pharmacokinetics, and toxicology of oligonucleotides.
• Antisense oligonucleotides.
• Antigene strategies.
• Ribozymes.
• Aptamers.
• Decoys.

Special Topics

• RNA interference.
• MicroRNAs.
• Genome editing using CRISPR/Cas9.
• Vaccines.
• Monoclonal antibodies.
• CAR-T therapies.

Laboratory Activities

• Bacterial transformation, selection, and evaluation of gene expression.
• Extraction and purification of recombinant protein products.
• Propagation, characterization, and amplification of transformation constructs.
• Structural bioinformatics: visualization and manipulation of macromolecules using molecular graphics software.

1. Bibliography and notes provided during the course available online at: http://studium.unict.it/dokeos/2026/
2. D. J. A. Crommelin & R. D. Sindelar, BIOTECNOLOGIE FARMACEUTICHE, Zanichelli.
3. B. R. Glick, J. J. Pasternak, BIOTECNOLOGIA MOLECOLARE, Zanichelli.
4. M. L. Calabrò, Compendio di BIOTECNOLOGIE FARMACEUTICHE, EdiSES.
5. A. Maggi, BIOTECNOLOGIE FARMACOLOGICHE, MASSON.
6. E. Vegeto, A. Maggi, P. Minghetti, FARMACI BIOTECNOLOGICI: aspetti farmacologici e clinici, Casa Editrice Ambrosiana.
7. T. A. Brown, Biotecnologie molecolari: Principi e tecniche, Seconda edizione italiana, Zanichelli.
8. G. A. Petsko, D. Ringe, Struttura e funzione delle proteine, Zanichelli.
9. D. J. A. Crommelin, R. D. Sindelar, B. Meibohm. Pharmaceutical Biotechnology: Fundamentals and Applications, Fifth Edition.  Springer.
10. G. Walsh. Pharmaceutical Biotechnology: Concepts and Applications, Wiley.
11. T. Brown & Tom Brown (Jnr). Nucleic Acids Book, Atdbio.  https://atdbio.com/nucleic-acids-book
12. J. Sambrook, E. F. Fritsch, T. Maniatis, Molecular cloning: A laboratory manual, Second edition, Cold Spring Harbor Laboratory Press.

Assessment Methods

Learning outcomes will be assessed through an oral examination aimed at verifying:

• Knowledge of the theoretical foundations of pharmaceutical biotechnology, recombinant DNA technology, gene expression, and the main strategies for the development and clinical application of biotechnological drugs.
• Ability to apply theoretical and methodological principles to bacterial transformation, gene expression, recombinant protein production and purification, in silico analysis of macromolecules, and interpretation of laboratory results.
• Ability to critically evaluate experimental results and select appropriate biotechnological strategies and expression systems.
• Ability to explain concepts, methodologies, and applications of pharmaceutical biotechnology using appropriate scientific terminology.
• Ability to independently integrate knowledge acquired throughout the course and connect biological, biochemical, pharmacological, and pharmaceutical aspects involved in biotechnological drug development.

Typically, the examination consists of:

• One question selected from two advanced topics;
• Three questions covering different sections of the course program (one question per module).

The examination generally lasts between 40 and 60 minutes.

Learning assessment may also be conducted remotely if required by specific circumstances.

GRADING CRITERIA

Knowledge and Understanding

• Accuracy of theoretical knowledge.
• Completeness of information provided.
• Ability to identify and describe the biological, biochemical, and pharmaceutical principles underlying the production and characterization of therapeutic biomolecules.

Applying Knowledge

• Ability to apply theoretical concepts to laboratory methodologies.
• Ability to interpret experimental data.
• Ability to address issues related to the design, production, and characterization of biotechnological drugs.

Making Judgements

• Critical analysis of experimental results.
• Identification of limitations and challenges.
• Evaluation of the effectiveness of experimental and production strategies.

Communication Skills

• Appropriate use of scientific terminology.
• Clarity and consistency in presenting concepts and methodologies.
• Ability to integrate biological, biochemical, pharmacological, and pharmaceutical perspectives.

Learning Skills

• Integration of knowledge from different parts of the course and prerequisite disciplines.
• Appropriate use of scientific literature and reference materials.
• Ability to independently update knowledge regarding emerging technologies and recent developments in pharmaceutical biotechnology.

GRADING SCALE

Fail: The student does not demonstrate the minimum required knowledge and shows inadequate command of technical terminology.

18-21/30: The student demonstrates essential knowledge of the subjects with limited critical integration skills and uses simple but generally correct scientific language.

22-25/30: The student demonstrates a satisfactory knowledge of the subjects, with appropriate ability to establish connections and use technical terminology.

26-28/30: The student demonstrates a good mastery of the course contents and proper application of acquired knowledge with adequate technical language.

29-30/30 cum laude: The student demonstrates in-depth knowledge, strong critical thinking skills, independent judgment, and full command of specialized terminology.

INFORMATION FOR STUDENTS WITH DISABILITIES AND/OR SPECIFIC LEARNING DISORDERS (SLD)

To ensure equal opportunities and compliance with current regulations, students may request an individual meeting to discuss compensatory and/or dispensatory measures based on educational objectives and specific needs.

Students may contact Prof. Santina Chiechio (santina.chiechio@unict.it), Department Coordinator for CInAP (Center for Active and Participatory Inclusion - Services for Students with Disabilities and/or SLDs), Department of Drug and Health Sciences.

• Classification of vaccines.
• Antisense oligonucleotides (ASOs).
• Ultra-rapid-acting insulin analogues.
• Fusion proteins.