RICERCA E SVILUPPO DI FARMACI BIOLOGICI E PICCOLE MOLECOLE E ASPETTI OMICI DELLA PROGETTAZIONE RAZIONALE DI FARMACI

12 CFU - 2° Semester

Teaching Staff

SALVATORE GUCCIONE - Module Biological Drugs and Small Molecules Research and Development - CHIM/08 - 6 CFU
SIMONE RONSISVALLE - Module ASPETTI OMICI DELLA PROGETTAZIONE RAZIONALE DEI FARMACI - CHIM/08 - 6 CFU

Learning Objectives

Biological Drugs and Small Molecules Research and Development
Biological drug products play a central role in combating human diseases; however, developing new successful biological drugs presents many challenges, including labor intensive production processes, tighter regulatory controls, and increased market competition.

After finalizing this course, the student will be able to
- compare biological with small molecule-drugs and understand how they differ in terms of discovery and development; understand the singularities of peptide drugs as a “transition” case in between both extremes;
- describe the key features of pharmaceutical biotechnology and biopharmaceuticals;
- describe and classify the most important biopharmaceuticals according to their production methods, characteristics and indications;
- compare the biotechnological production of drugs using plants, microbes and animal cells;
- characterize the production of recombinant proteins, antibodies and blood-derived products;
- understand the basic theoretical principles of advanced therapies (gene and cell therapies) and their practical challenges;
- get familiarized with the most important current regulatory aspects of biological drugs,
- get in-deep knowledge of one model biological drug, via completion of the case-studies.
ASPETTI OMICI DELLA PROGETTAZIONE RAZIONALE DEI FARMACI
Students will have to learn what are the scientific needs that led to the birth of the "omic" sciences, the basic scientific fields that felt the need for an application of the "omic" sciences and how they differ or which additional information gives respect to classical techniques. Furthermore genomics, transcriptomics, epigenomics, proteomics and metabolomics will be treated with hints referring to classical techniques so that the student can recall the studies carried out on these already learned during his course of study and use them as a comparison for the "homic" counterpart. ". Some fields of application will be evaluated. Furthermore, the technological bases through which these sciences can help design or rationally reposition new drugs will be learned. For this reason, hints of mass spectrometry (instrumentation and theory) and of molecular modeling (with hints of bioinformatics) will be reported. Finally, the student will be able to work on real pathological applications and deepen his knowledge through case studies.

Course Structure

Biological Drugs and Small Molecules Research and Development
Frontal Lessons. Attendance of lessons is recommended.

Perequisites: Organic Chemistry and General Biochemistry.

According to "Regolamento Didattico di Ateneo (R.D.A.) i.e. University Didactic Regulations attendance of lessons is mandatory.

Should teaching be carried out in mixed mode or remotely, it may be necessary to introduce changes with respect to previous statements, in line with the programme planned and outlined in the syllabus.

Learning assessment may also be carried out on line, should the conditions require it.
ASPETTI OMICI DELLA PROGETTAZIONE RAZIONALE DEI FARMACI
Frontal lessons

Detailed Course Content

Biological Drugs and Small Molecules Research and Development
Course will consist of lectures, content of which will include classification of biological drugs, their production and their therapeutic use. Experimental biological drugs of particular interest will also be discussed .

Text-book Pharmaceutical Biotechnology: Concepts and Applications by Gary Walsh downloadable from :

http://site.iugaza.edu.ps/tbashiti/files/2013/02/2.Pharmaceutical_Biotechnology_ConceptsApplications-Gary_Walsh.pdf

Chapter 1,4,5, 11(subpar. 1 and 2),13,14.

Other chapters in the text-book can be used at the student' s best convenience as "ad hoc" background or as useful readings to master the course.
ASPETTI OMICI DELLA PROGETTAZIONE RAZIONALE DEI FARMACI
Introduction to the omic aspects of biological science
Scientific techniques for drug design: Instrumentation and basic theory (electrophoretic, chromatographic techniques and mass spectrometry); Molecular modeling theoretical bases (outline of Bioinformatics)
Discussion of: - Genomics (theoretical basis, sequencing, technology development, genome analysis, "Shotgun" sequencing, "high-throughput sequencing, sequence and approach recombination, databases), transcriptomics (RNA Isolation, sequence expression , serial and cap analysis of gene expression, Microarrays, RNA-Seq), epigenomics (DNA methylation, histone modification, Chip-Chip and Chip-Seq, "wide" and "non-wide" approaches, accessibility test chromatoin, direct detection), proteomic (protein detection with or without antibodies, hybrid technologies) and matbolomic (separation methods, detection methods, statistical methods)
Fields of application: Genomics (functional and structural genomics, model systems), transcriptomics (diagnostics, human-apathological transcriptomics, environmental stress, gene functioning, aging), epigenomics, proteomics (proteomic interaction and protein network, protein expression, biomarkers, proteogenomics, protein identification, analysis of protein structure, analysis of post-translational modifications) and metabolomics
Pathological applications and case studies

Textbook Information

Biological Drugs and Small Molecules Research and Development
1) Pharmaceutical Biotechnology: Concepts and Applications by Gary Walsh, WILEY, ISBN: 978-0-470-01244-4

downloadable from http://site.iugaza.edu.ps/tbashiti/files/2013/02/2.Pharmaceutical_Biotechnology_ConceptsApplications-Gary_Walsh.pdf

2)Biological Drug Products: Development and Strategies, Wei Wang (Editor), Manmohan Singh (Editor), WILEY, ISBN: 978-1-118-14889-1 November 2013.
ASPETTI OMICI DELLA PROGETTAZIONE RAZIONALE DEI FARMACI

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