Pharmaceutical Biotechnology

The course is designed to provide fundamental pharmacological knowledge on the main classes of drugs used in clinical therapy. Particular emphasis will be placed on the cellular and molecular mechanisms and on the rationale underlying the development of new drugs. During the course, students will also be introduced to the basic concepts of in vitro and in vivo experimental models currently used in drug development, as well as to the approaches used for analyzing and evaluating experimental results. In addition, the main resources (databases, online bibliographic research, etc.) used for studying and staying updated with the scientific literature will be presented. DETAILED PROGRAM Drugs acting at synapses and neuroeffector junctions: Neurotransmission – The autonomic and somatic motor nervous systems – Muscarinic receptor agonists and antagonists – Anticholinesterase agents – Drugs acting at the neuromuscular junction and autonomic ganglia – Catecholamines, sympathomimetics, and adrenergic receptor antagonists. Drugs acting on the central nervous system: Neurotransmission and the central nervous system (classical, atypical, retrograde, and gaseous transmitters) – Neuromodulation – General and local anesthetics – Hypnotics and sedatives – Drugs for the treatment of psychiatric disorders (psychosis, anxiety, and depression) – Drugs for epilepsy – Drugs for migraine treatment – Pharmacological treatment of neurodegenerative diseases (Parkinson’s Disease, Alzheimer’s Disease) – Opioid analgesics and their antagonists – Drug addiction and substance abuse. Pharmacological treatment of inflammation: Analgesic, antipyretic, and anti-inflammatory agents – Drugs used in the treatment of bronchial asthma. Drugs affecting renal and cardiovascular function: Diuretics, vasopressin, and other agents affecting renal water balance – Renin and angiotensin – Drugs for myocardial ischemia – Antihypertensives and pharmacological treatment of hypertension – Pharmacological treatment of heart failure – Antiarrhythmic drugs. Drugs acting on gastrointestinal function: Drugs for gastric acid control and treatment of peptic ulcers – Prokinetic agents – Emetic and antiemetic drugs – Agents used in diarrhea and constipation – Drugs used in pancreatic and biliary diseases. Antiinfective and antitumor chemotherapy: Antibiotics – Antiprotozoal drugs – Drugs for the chemotherapy of helminthic infections – Antifungal agents – Antiviral and antiretroviral drugs – Antineoplastic agents – Immunomodulatory drugs – Drugs acting on blood and hematopoietic organs. Anticoagulants, thrombolytics, and antiplatelet agents. Hormones and their antagonists: Adenohypophyseal hormones and hypothalamic releasing factors – Thyroid drugs – Estrogens and progestins – Androgens – Adrenocorticotropic hormone – Adrenal corticosteroids and synthetic analogs – Inhibitors of corticosteroid synthesis and action – Insulin, oral hypoglycemic agents, and endocrine pancreas pharmacology – Agents influencing bone calcification and turnover (calcium, phosphate, parathyroid hormone, vitamin D, calcitonin, and related compounds). Drugs used in the treatment of hyperlipoproteinemias. Water- and fat-soluble vitamins. Biotechnological and Advanced Therapies: Biotechnological drugs for metabolic diseases: Cell therapy for type 1 diabetes – SGLT2 inhibitors: mechanisms, clinical and pharmacokinetic applications – Glucagon-Like Peptide-1 Receptor (GLP-1R) agonists. Biotechnological drugs for dyslipidemias: PCSK9 inhibitors – First-, second-, and third-generation inhibitors – Gene therapy – Drugs targeting apolipoprotein B-100 – Lipoprotein(a)-lowering agents – Drugs acting on ANGPTL3 and APOC-III. Biotechnological drugs for central nervous system (CNS) disorders: Anti-Aβ antibodies and Alzheimer’s disease – Multiple sclerosis – Migraine – Botulinum toxin – Biotechnological drugs modulating the calcitonin gene-related peptide (CGRP). Cancer and targeted therapy strategies: The concept of “agnostic” drugs – Cancer therapy: evolution and approaches – Hormonal therapy – Immunotherapy – Antibodies in cancer treatment. Cell therapy, regenerative medicine, and advanced therapies: Pharmacokinetic and pharmacodynamic features – Molecular mechanisms underlying the therapeutic effects of RNA-based therapies – RNA metabolism modulation: antisense oligonucleotides (GAPmers) – Exogenous administration of mRNA transcripts – CAR-T cell therapies.

To effectively address the course contents and achieve the intended learning objectives, it is recommended that students possess basic knowledge of general pharmacology, physiology, and biochemistry. These prerequisites are not mandatory, but they are strongly advised to ensure a deeper understanding of the lectures and the successful completion of the final examination.

The achievement of the expected learning outcomes is verified through an oral examination. The assessment considers not only the student’s specific preparation on the topics covered in the lectures and indicated in the syllabus, but also critical reasoning skills, the logical organization of answers, and the appropriate use of technical-scientific terminology. The duration of the oral examination is not strictly fixed, as it depends on the student’s ability to present and discuss topics; on average, it lasts around 20–25 minutes. The course does not include mid-term assessments: examinations take place only at the end of the course, according to the official schedule published on Infostud and on the e-learning platform. • To pass the exam, students must achieve a grade of at least 18/30, demonstrating sufficient knowledge of the fundamental topics and the ability to communicate clearly using adequate technical terminology. • A final grade of 30/30 with honours is awarded to students who show an excellent and comprehensive knowledge of all course topics (including those not directly discussed in class), the ability to critically and coherently integrate concepts, and a mastery of scientific language with outstanding communication skills.