PHARMACOLOGY AND MOLECULAR THERAPIES

PHARMACOLOGY:
Origin of new drugs – Small molecules versus biopharmaceuticals; the drug discovery phase; preclinical development; clinical development.
Pharmacokinetics – Physical processes underlying drug disposition; drug absorption and routes of administration; distribution of drugs in the body; drug metabolism; drug and metabolite excretion; clinical pharmacokinetics: temporal characteristics of drug effects and relationship to the therapeutic window; first-order kinetics; effect of repeated dosing.
Pharmacogenetics and Pharmacogenomics – Individual variation in drug response: genetic factors; biomarkers as predictors of efficacy and safety; clinical validity and clinical utility of genomic biomarkers; moving from bench to bedside.
Biopharmaceuticals – Examples of therapeutic proteins; biosimilars; problems in manufacture; evolution of monoclonal antibodies; safety issues; pharmacokinetics and pharmacodynamics.
MOLECULAR THERAPIES:
Introduction to advanced molecular therapies: definition, advantages and main current limitations, ethical issues; concepts and tools of gene and cell therapy; delivery systems and therapeutic strategies for cell and gene therapy.
Gene therapy: development and application of virus-based vectors: retroviruses, lentiviruses, adenoviruses, adeno-associated viruses, herpes simplex virus, alphaviruses; oncolytic viruses for treatment of cancer; applications of gene therapy to non-neoplastic disorders; gene therapy approaches for HIV-1 infection. Gene editing strategies: delivery technologies and platforms for genome editing: zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR–Cas9 system engineered nucleases to correct genetic defects and to cure chronic viral infections: design, mechanism of action, limitations and examples.
Regenerative Medicine: advances in the use of adult stem cells and induced pluripotent stem cells as “medicinal products” for therapies. The identification of natural or the development of artificial scaffolds capable to mimic the niche: a major area of enquiry to optimize the outcome from stem cell transplantation and tissue engineering. Stem cell aging: mechanisms, regulators and therapeutic opportunities. The development of new biotechnologies and their application to Precision Medicine in cancer treatment.
Process of developing products starting from lab-based research, from the crucial step of protecting intellectual property, marketing strategies, basic knowledge on how a company works, and the status of the biotechnology sector in Italy. Rare and neglected diseases, orphan drugs; challenges and potential of developing new peptide-based therapeutics; nucleic acids as therapeutic agents/therapeutic targets; nanoparticles as delivery systems.