Ateneo - UPO

Module 1 – In vitro and in vivo Preclinical models: cellular models (2D/3D, organoids, organ-on-chip) and animal models used in the preclinical phase of drug and biotechnology development, with reference to pharmacokinetics, pharmacodynamics and toxicology.
Module 2 – Synthetic Biology: principles of genetic engineering and synthetic biology, genetic circuits, genome editing technologies (e.g. CRISPR-Cas), biosensors and biotechnological/biomedical applications, with attention to biosafety aspects.
Module 3 – Project design and management: methodologies for designing and managing biotechnology research projects, from objective-setting to resource planning, through to the drafting of project proposals (including the context of competitive calls, e.g. Horizon Europe).

• Instructor-provided material (slides, selected scientific articles, ICH/EMA/FDA guidelines)
• Module 1: selected chapters from preclinical pharmacology and toxicology textbooks (e.g. Hodgson's Textbook of Modern Toxicology)
• Module 2: Synthetic Biology: A Primer (Ellis, Baldwin, et al.); articles from Nature Biotechnology, ACS Synthetic Biology
• Module 3: A Guide to the Project Management Body of Knowledge (PMBOK Guide) – selected sections; material on Horizon Europe / EU funding schemes

To provide students with the scientific, technical and managerial competencies needed to design, conduct and evaluate preclinical studies in the biomedical biotechnology field, to understand the principles of applied synthetic biology, and to manage a research project from conception to execution, in line with international regulatory standards.

Basic knowledge of molecular biology, cell biology, genetics and general pharmacology, acquired during a bachelor's degree in biological/biomedical/biotechnological sciences.

• Lectures (Modules 1 and 2)
• Practical tutorials and case-study discussion (Modules 1 and 2)
• Group work on the design of a biotechnology research project, with final presentation (Module 3)
• Seminars with external experts (regulatory affairs, project management)

Students with disabilities, Specific Learning Disorders (SLD), or Special Educational Needs (SEN) may request specific services and tools dedicated to them by contacting the Staff for Career Development and Coordination and Services for Students, and by consulting the dedicated page on the University's website. After getting in touch with the University Staff, they may contact the instructor responsible for the course to agree on exam procedures and teaching-related aspects. Attendance: attendance at in-person lectures is mandatory for all degree programs (minimum 50%-66% of hours). Students who do not reach the minimum threshold will not be admitted to the exam in the current session.
Working students and individuals in particular situations (caregivers, etc.) are invited to contact the instructor within the first week of classes to agree on alternative arrangements, in compliance with the University Regulations.
Accessible materials: slide PDFs are provided in text format (not scanned); recorded distance-learning
(DAD) lectures are accompanied by subtitles or transcripts.

Written exam

Module 1 – In vitro and in vivo Preclinical models (12 hours)
1. Introduction to preclinical development: from discovery to clinical candidate selection
2. 2D and 3D cellular models: primary cultures, cell lines, spheroids
3. Organoids and organ-on-chip: principles and translational applications
4. Animal models: species selection, disease models, the 3Rs principle
5. Pharmacokinetic (ADME) studies and preclinical pharmacodynamics
6. Elements of preclinical toxicology (acute/chronic toxicity, genotoxicity)
7. Good Laboratory Practice (GLP) and preclinical data quality
Module 2 – Synthetic Biology (24 hours)
8. Fundamentals of synthetic biology: principles of applied genetic engineering
9. Standardized biological parts (BioBricks) and synthetic genetic circuits
10. Genome editing technologies: CRISPR-Cas9 and variants
11. Design of biosensors and controlled gene expression systems
12. Applications in therapy (cell/gene therapy) and diagnostics
13. Synthetic biology for biotechnological production (microbial chassis)
14. Biosafety, biocontainment and ethical-regulatory aspects of synthetic biology
15. Case studies and discussion of recent scientific articles
Module 3 – Project design and management (12 hours)
16. Life cycle of a research project: from idea to proposal
17. Defining objectives, milestones and deliverables
18. Resource planning: budget, timelines (Gantt charts), team
19. Risk analysis and management of unforeseen events
20. Writing a competitive project proposal (e.g. Horizon Europe)
21. Elements of intellectual property and technology transfer
22. Presentation and peer evaluation of student-developed projects

By the end of the course, students will be able to:
• Knowledge and understanding: describe the main in vitro and in vivo preclinical models, the principles of synthetic biology and project management methodologies.
• Applying knowledge and understanding: select the most appropriate preclinical model for a specific experimental question; design simple synthetic genetic circuits; plan the phases of a biotechnology research project.
• Making judgements: critically evaluate the methodological and regulatory robustness of a preclinical study or a synthetic biology project.
• Communication skills: present, in written and oral form, including in English, the results of a biotechnology research project.
• Learning skills: independently keep up to date on new preclinical/synthetic-biology technologies and project management tools.