Innovative Biotechnological Drugs
A.Y. 2023/2024
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 production of the biotec drug.
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 production of the biotec drug.
Lesson period: First 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
First 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 aspects in the preparation of biopharmaceuticals", while for the teaching unit "Biotechnological approaches to drug design and optimization" 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.
Innovative aspects in drug design and optimization
Course syllabus
Teaching Unit: Novel therapeutic strategies (3 CFU)
· Introduction to biotechnological drugs- recombinant therapeutic protein and monoclonal antibodies
- Biotechnological drugs for multiple sclerosis
· Innovative therapies based on miRNA
· Extracellular vesicles as innovative vectors for targeted therapies
- Studies in experimental models of multiple sclerosis and Alzheimer's disease
· Innovation in the gene therapy
- Technological problems and challenges in the gene therapy: generalities
- The development of safe vectors
- Innovative systems of the stable expression of the therapeutic gene
- Genome editing: the CRISP/Cas9 system
- The approved drugs and those under study
· Stem cells and innovation in the regenerative medicine
- Types and properties: embryonic stem cells, adult stem cells and iPS
- Potential applications of cell therapy
- Neural stem cells and potential clinical applications
Teaching Unit: Biotechnological approaches to drug design and optimization (3CFU)
Pharmacokinetic issues associated with peptide drugs
- innovative strategies of formulation and pre-formulation
- specific methods for the controlled and prolonged release of bioactive: carries, pegylation and other (bio)technologies
Pharmacodynamics: biotechnological techniques for the development of site-specific drugs
- prodrugs
- passive and active targeting
- cancer therapy: examples of biotechnological site-specific release strategies for anticancer molecules and peptides. Design of bioconjugates; vascular targeting; enzymes-mediated tissue selectivity; enzyme-prodrug therapies.
Examples of biotech drugs.
- Central nervous system targeting
- Aptamers as diagnostic and therapeutic agents
· Introduction to biotechnological drugs- recombinant therapeutic protein and monoclonal antibodies
- Biotechnological drugs for multiple sclerosis
· Innovative therapies based on miRNA
· Extracellular vesicles as innovative vectors for targeted therapies
- Studies in experimental models of multiple sclerosis and Alzheimer's disease
· Innovation in the gene therapy
- Technological problems and challenges in the gene therapy: generalities
- The development of safe vectors
- Innovative systems of the stable expression of the therapeutic gene
- Genome editing: the CRISP/Cas9 system
- The approved drugs and those under study
· Stem cells and innovation in the regenerative medicine
- Types and properties: embryonic stem cells, adult stem cells and iPS
- Potential applications of cell therapy
- Neural stem cells and potential clinical applications
Teaching Unit: Biotechnological approaches to drug design and optimization (3CFU)
Pharmacokinetic issues associated with peptide drugs
- innovative strategies of formulation and pre-formulation
- specific methods for the controlled and prolonged release of bioactive: carries, pegylation and other (bio)technologies
Pharmacodynamics: biotechnological techniques for the development of site-specific drugs
- prodrugs
- passive and active targeting
- cancer therapy: examples of biotechnological site-specific release strategies for anticancer molecules and peptides. Design of bioconjugates; vascular targeting; enzymes-mediated tissue selectivity; enzyme-prodrug therapies.
Examples of biotech drugs.
- Central nervous system targeting
- Aptamers as diagnostic and therapeutic agents
Teaching methods
The program will be carried out through lectures (6 CFU) with the help of teaching material (slides).
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 Ariel
Synthetic approaches in the preparation of biomolecules
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 Ariel.
Innovative aspects in drug design and optimization
BIO/14 - PHARMACOLOGY
CHIM/08 - PHARMACEUTICAL CHEMISTRY
CHIM/08 - PHARMACEUTICAL CHEMISTRY
Lessons: 48 hours
Professors:
Fumagalli Marta, Romeo Sergio
Synthetic approaches in the preparation of biomolecules
CHIM/06 - ORGANIC CHEMISTRY - University credits: 3
Lessons: 24 hours
Professor:
Sani Monica
Educational website(s)
Professor(s)
Reception:
Monday 10:30-12:30, by appointment via email
Via Mangiagalli 25, second floor, office 2062