Design, Development, and Therapeutic Application of Biotechnological Drugs
A.Y. 2024/2025
Learning objectives
The objective of the course is to provide students with the appropriate tools to comprehend concepts and strategies underlying innovation in the field of biotechnological drug discovery and development, with attention to the advanced knowledge in the design, optimization, and therapeutic use of biotechnological drugs.
Expected learning outcomes
At the end of the course, it is expected that the student, through the acquisition of advanced knowledges, will have the conceptual and technological basis to project him into the creative process of innovation in the field of the design, optimization and the evaluation of efficacy and mechanism of action of biotechnological drugs. Students are also expected to acquire the ability to present and explain their understanding of the subject appropriately and effectively.
Lesson period: Second semester
Assessment methods: Esame
Assessment result: voto verbalizzato in trentesimi
Single course
This course can be attended as a single course.
Course syllabus and organization
Single session
Responsible
Lesson period
Second semester
Prerequisites for admission
The student should have acquired advanced knowledge on molecular biology, pharmacology and biotechnology, pharmaceutical chemistry and organic chemistry applied to macromolecules, to understand the innovative strategies applied to the development of the biotechnological drug.
Assessment methods and Criteria
Students will be evaluated on the basis of an oral test. The student will be asked at least 4 questions on the arguments of the teaching unit "Synthetic methodologies in the preparation of biotechnological drugs", while for the teaching unit "Design and therapeutic activity of biotechnological drugs" the exam consists in an oral discussion on at least three topics treated during the teaching unit.
The student will have to demonstrate mastery and ability to show the knowledge learned and reasoning skills in integrating the topics covered in class.
The final mark (in thirtieths) will be calculated as weighted average (on the basis of the CFU) of the marks of the two teaching units.
The student will have to demonstrate mastery and ability to show the knowledge learned and reasoning skills in integrating the topics covered in class.
The final mark (in thirtieths) will be calculated as weighted average (on the basis of the CFU) of the marks of the two teaching units.
Design and therapeutic activity of biotechnological drugs
Course syllabus
Didactic Unit Prof. Romeo
· Introduction: pharmaco-kinetic and -dynamic issues associated with biotechnological drugs
· Site-specific drug development: prodrug chemistry, passive and active targeting, examples of site-specific release strategies
· Design of bioconjugates: vascular targeting; enzyme-mediated tissue selectivity; enzyme-prodrug therapies
· Nanoparticles and their applications in gene- and drug- delivery
· Strategies for the delivery of biotechnological drugs in the central nervous system
· Aptamers as diagnostic and therapeutic means
· Examples of chemical development of biotechnological drugs
Didactic Unit Prof.ssa Fumagalli
Biotechnology drugs for the central nervous system
· Multiple sclerosis: epidemiology, symptoms, clinical forms, pathogenesis. Therapy: biotechnology drugs and other approved treatments.
· Migraine: pathogenesis and pharmacological treatments. Monoclonal antibodies in therapy.
· ALS: disease description and approved drugs. Therapeutic strategies with antisense oligonucleotides.
miRNAs as drugs: how they are studied in vitro and in vivo and their potential therapeutic use
Gene therapy: goals, problems and technological challenges.
· Examples of diseases treated with in-vivo and ex-vivo gene therapy, approved drugs, and ongoing studies
· Therapeutic strategies based on Crispr/Cas9 technology
Cell therapy: types and properties of stem cells (embryonic, fetal and adult).
· Hematopoietic, skin, corneal, and muscle stem cells: description and examples of therapeutic use
· Mesenchymal, neural, and iPS stem cells: description and examples of therapeutic use
Extracellular vesicles in regenerative and precision medicine
· Studies in multiple sclerosis, cerebral ischemia, and in oncology
· Extracellular vesicles and synaptic dysfunction in Alzheimer's disease
· Introduction: pharmaco-kinetic and -dynamic issues associated with biotechnological drugs
· Site-specific drug development: prodrug chemistry, passive and active targeting, examples of site-specific release strategies
· Design of bioconjugates: vascular targeting; enzyme-mediated tissue selectivity; enzyme-prodrug therapies
· Nanoparticles and their applications in gene- and drug- delivery
· Strategies for the delivery of biotechnological drugs in the central nervous system
· Aptamers as diagnostic and therapeutic means
· Examples of chemical development of biotechnological drugs
Didactic Unit Prof.ssa Fumagalli
Biotechnology drugs for the central nervous system
· Multiple sclerosis: epidemiology, symptoms, clinical forms, pathogenesis. Therapy: biotechnology drugs and other approved treatments.
· Migraine: pathogenesis and pharmacological treatments. Monoclonal antibodies in therapy.
· ALS: disease description and approved drugs. Therapeutic strategies with antisense oligonucleotides.
miRNAs as drugs: how they are studied in vitro and in vivo and their potential therapeutic use
Gene therapy: goals, problems and technological challenges.
· Examples of diseases treated with in-vivo and ex-vivo gene therapy, approved drugs, and ongoing studies
· Therapeutic strategies based on Crispr/Cas9 technology
Cell therapy: types and properties of stem cells (embryonic, fetal and adult).
· Hematopoietic, skin, corneal, and muscle stem cells: description and examples of therapeutic use
· Mesenchymal, neural, and iPS stem cells: description and examples of therapeutic use
Extracellular vesicles in regenerative and precision medicine
· Studies in multiple sclerosis, cerebral ischemia, and in oncology
· Extracellular vesicles and synaptic dysfunction in Alzheimer's disease
Teaching methods
The program will be carried out through lectures (6 CFU) with the help of teaching material (slides) and digital tools (such as Wooclap) to improve interactive learning
Lessons attendance is strongly advised.
Lessons attendance is strongly advised.
Teaching Resources
Didactic materials and bibliography will be provided by teachers and are available on MyAriel
Synthetic methodologies in the preparation of biotechnological drugs
Course syllabus
Organic chemistry overview on:
- functional groups
- stereochemistry, racemic mixture, enantiomeric excess.
- stereochemical outcome of the reactions.
Peptides and proteins
- general features of amino acids and peptides
- protecting groups: the orthogonal approach
- peptide bond formation: activation and coupling
- solid phase and liquid phase peptide synthesis
- microwave assisted peptide synthesis
- chemical ligation: native chemical ligation, expressed protein ligation
- secondary structures of peptides and proteins and related analytical techniques: circular dichroism, FT-IR, NMR
Peptidomimetics
- main features and role
- bioactive peptide conformation and tools to introduce constraints
- peptoids and foldamers
Supramolecular assemblies
- protein-ligand interaction and related techniques: microscale thermoforesis, surface plasmon resonance, calorimetry,
- peptide nanomaterials: from self-assembly to applications. Overview on dynamic light scattering and electronic microscopy.
Nucleic Acids
- Structure and nomenclature: nucleosides and nucleotides
- Nucleic acids synthesis: the formation of the phosphodiester bond
- Oligonucleotides synthesis
- Modified oligonucleotides: PNA and Morpholinos.
- functional groups
- stereochemistry, racemic mixture, enantiomeric excess.
- stereochemical outcome of the reactions.
Peptides and proteins
- general features of amino acids and peptides
- protecting groups: the orthogonal approach
- peptide bond formation: activation and coupling
- solid phase and liquid phase peptide synthesis
- microwave assisted peptide synthesis
- chemical ligation: native chemical ligation, expressed protein ligation
- secondary structures of peptides and proteins and related analytical techniques: circular dichroism, FT-IR, NMR
Peptidomimetics
- main features and role
- bioactive peptide conformation and tools to introduce constraints
- peptoids and foldamers
Supramolecular assemblies
- protein-ligand interaction and related techniques: microscale thermoforesis, surface plasmon resonance, calorimetry,
- peptide nanomaterials: from self-assembly to applications. Overview on dynamic light scattering and electronic microscopy.
Nucleic Acids
- Structure and nomenclature: nucleosides and nucleotides
- Nucleic acids synthesis: the formation of the phosphodiester bond
- Oligonucleotides synthesis
- Modified oligonucleotides: PNA and Morpholinos.
Teaching methods
The program will be carried out through lectures (3 CFU) with the help of teaching material (slides).
Lessons attendance is strongly advised.
Lessons attendance is strongly advised.
Teaching Resources
Didactic material and bibliography will be provided by teachers and are available on MyAriel.
Design and therapeutic activity of biotechnological drugs
BIO/14 - PHARMACOLOGY - University credits: 3
CHIM/08 - PHARMACEUTICAL CHEMISTRY - University credits: 3
CHIM/08 - PHARMACEUTICAL CHEMISTRY - University credits: 3
Lessons: 48 hours
Professors:
Fumagalli Marta, Romeo Sergio
Synthetic methodologies in the preparation of biotechnological drugs
CHIM/06 - ORGANIC CHEMISTRY - University credits: 3
Lessons: 24 hours
Professor:
Pellegrino Sara
Professor(s)
Reception:
Monday 10:30-12:30, by appointment via email
Via Mangiagalli 25, second floor, office 2062