- Docente: Sanzio Candeletti
- Credits: 5
- SSD: BIO/14
- Language: Italian
- Moduli: Sanzio Candeletti (Modulo 1) Sabrina Angelini (Modulo 2)
- Teaching Mode: Traditional lectures (Modulo 1) Traditional lectures (Modulo 2)
- Campus: Rimini
- Corso: Single cycle degree programme (LMCU) in Pharmacy (cod. 9223)
Learning outcomes
At the end of the module the student: - knows the structure, production and post-translational processes of recombinant proteins, the purification processes and therapeutic targets of the biotechnological drugs on the market; - knows the different classes of therapeutic proteins and monoclonal proteins and their pharmacodynamic and pharmacokinetic profile; - knows the activity, immunogenicity and safety profile of biotechnological drugs and biosimilars; - will know how to apply the knowledge learned for carrying out the profession, for the conservation, conservation and dispensing of biotechnological drugs and to inform and educate patients on their use and monitor the management of the appropriateness in their use and the continuity of adherence to drug prescription in chronic therapy.
Course contents
MODULE 1
GENETIC ENGINEERING AND RECOMBINANT DNA
Gene cloning. Cellular genetic engineering for drug-protein production. Methods of DNA transfer in cultured cells. Cell systems for the study and production of recombinant proteins. Cloning of a human gene. Post-translational modifications of proteins. Pharmacological applications of recombinant cell lines. Transgenic animals in drug research, development and production
MONOCLONAL ANTIBODIES
Structure of antibodies. Activity and recycling of antibodies. Biotechnological synthesis of antibodies. Growth and selection of hybridomas. Chimeric, humanized and fully human monoclonal antibodies. Modifications that improve the pharmacological properties of monoclonal antibodies. Small molecule conjugated antibodies
OLIGONUCLEOTIDES AS DRUGS
Classification of oligonucleotides. Mechanism of action of oligonucleotides.
PHARMACODYNAMICS
Mechanisms of action of monoclonal antibodies. Antibodies directed to tumor antigens. Modification of the organism's response. Mechanisms of action in non-anticancer therapies. Monoclonal antibodies conjugated with cytotoxic agents. Selectivity of action. Side effects of monoclonal antibodies.
PHARMACOKINETICS
Routes of administration of biotechnological drugs. ADME. Salvage pathway. Properties of the antigen. Immunogenicity
CLINICAL USE OF BIOTECHNOLOGICAL DRUGS
DRUGS FOR THE CARDIOVASCULAR SYSTEM
Dyslipidemias: cardiovascular risk factors. Monoclonal antibodies for the treatment of dyslipidemias. Fibrinolytic drugs. New potential therapeutic targets
Diseases of the bone and calcium metabolism.
The recombinant parathyroid hormone. Denosumab (RANKL inhibitor). Recombinant Calcitonin
DRUGS FOR THE CENTRAL NERVOUS SYSTEM
Multiple sclerosis therapy. Migraine therapy. Anti-CGRP monoclonal antibodies. Alzheimer's disease, biotech drugs in development
DRUGS FOR THE RESPIRATORY SYSTEM
Biotechnological and innovative drugs in asthma therapy. Anti-IgE, Anti-IL5, Anti-IL5 receptor, Anti-IL4 receptor
New molecules or new targets in development for asthma therapy Anti-IL13 / IL17, Anticalins, Aptamers.
Safety of biosimilar drugs.
MODULE 2
ADVANCED THERAPIES:
Cells as drugs
GENE THERAPY
Definition and concept of gene therapy. Types of gene therapy approaches. Ex vivo gene therapy. In vivo gene therapy.
The tools of gene therapy. Viruses as safe vectors for gene therapy. The clinical applications of gene therapy. CAR-T. In vivo gene therapy drugs
TARGET THERAPY
Signal transducers. Gene mutations in human tumors. Tyrosine kinase inhibitors. Targeted therapy on the Bcr-Abl protein.
Targeted therapy on EGFR1, HER2 / neu. Inhibitors of VEGF and angiogenesis. RAF and MEK kinase inhibitors. PI3K kinase inhibitors
TARGETED DRUGS IN ONCOLOGY
Development of targeted anticancer chemotherapy. Drugs acting on tyrosine kinase receptors.
EPIGENETIC DRUGS
CLINICAL USE OF BIOTECHNOLOGICAL DRUGS
DRUGS FOR ONCOLOGY
Therapeutic strategies. Monoclonal antibody therapies. Active immunotherapy. Checkpoint inhibitors. Clinical information. Cell-mediated therapies. Cytokine therapies. Inhibitors of IL6. Inhibitors of IL1. Inhibitors of IL12 and IL23. Anti-IL17. Inhibitors of surface antigens of B and T lymphocytes
DRUGS FOR THE IMMUNE SYSTEM
Biotechnological drugs for chronic autoimmune inflammatory diseases. Inhibitors of TNFalpha
ADVERSE EFFECTS of Biotechnological Drugs
Adverse effects related to the mechanism of action. Tumor lysis syndrome. Infections. Tumors. Inhibition of angiogenesis. Adverse immunological effects. Anti-drug antibodies. Immunogenicity. Autoimmune Diseases. Cytokine release syndrome.
Interaction with an antigen other than the target. Toxicity of monoclonal antibody derivatives
Readings/Bibliography
Vegeto E, Maggi A, Minghetti P., Farmaci biotecnologici: aspetti farmacologici e clinici. Casa Editrice Ambrosiana. Zanichelli
Teaching methods
Frontal lessons
Assessment methods
Verification of learning consists in an oral exam, for each module, which aims to verify and evaluate the achievement of the learning objectives.
The student evaluations takes into account the correctness of the answers, the understanding of the principles learned showed by the student. In addition, the level of exposure, also in relation to the previous knowledge necessary for the understanding of the specific exam subject will be considered. The duration of the oral exam is on average 20-30 minutes.
Teaching tools
PC, Projector. PowerPoint; the slides used during lessons will be available on the VIRTUALE platform
Office hours
See the website of Sanzio Candeletti
See the website of Sabrina Angelini