Biological Drugs and Advanced Therapies
A.Y. 2025/2026
Learning objectives
Recent advances in genetic engineering, cellular, and tissue biology have led to the development of
new biologically-based medical products, offering treatment options that were previously
unavailable. This course aims to: i) provide an overview of this emerging area in modern
pharmacology; ii) teach students how to identify and classify biologic drugs and Advanced Therapy
Medicinal Products (ATMPs); iii) examine European regulations on the classification, production,
and use of these products.
new biologically-based medical products, offering treatment options that were previously
unavailable. This course aims to: i) provide an overview of this emerging area in modern
pharmacology; ii) teach students how to identify and classify biologic drugs and Advanced Therapy
Medicinal Products (ATMPs); iii) examine European regulations on the classification, production,
and use of these products.
Expected learning outcomes
By the end of the course, students will understand how new therapeutic molecules are developed
and will have knowledge of the properties of biologic drugs, with a focus on the molecular and cellular
mechanisms behind their action. Students will also be familiar with the regulations and procedures
for conducting pharmacological clinical trials.
and will have knowledge of the properties of biologic drugs, with a focus on the molecular and cellular
mechanisms behind their action. Students will also be familiar with the regulations and procedures
for conducting pharmacological clinical trials.
Lesson period: Second 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
Second semester
Course syllabus
1. Biological and Biotechnological Drugs
- Historical background and classification
- Design and production of biological and biotechnological drugs
- Comparison between biological drugs and conventional (small-molecule) drugs
- Safety considerations
2. Biotechnological Drugs for Central Nervous System Diseases
Alzheimer's disease
Multiple sclerosis
Migraine
3. Biotechnological Drugs for Oncological Diseases
Hormone therapy
Immunotherapy
Monoclonal antibodies
- Engineering and production; characteristics
- Types of monoclonal antibodies: Chimeric, Humanized, Fully human
- Bispecific antibodies: structure and therapeutic applications
- Antibody-Drug Conjugates (ADCs)
- Antibody fragments
- Side effects and toxicity of monoclonal antibodies
- Therapeutic applications
Cancer vaccines
4. RNA-Based Drugs
Introduction to RNA interference and genome editing
Types of RNA-based drugs, mechanisms of action, and therapeutic applications
- Small interfering RNAs (siRNAs)
- Antisense oligonucleotides (ASOs)
- Aptamers
- Therapeutic RNAs targeting genomic DNA: clinical applications
- mRNA vaccines
Formulation, chemical modifications, delivery systems, cellular uptake and intracellular distribution of RNA therapeutics
Potential toxicity, immunogenicity, and mutagenic risk
5. Advanced Therapy Medicinal Products (ATMPs)
- Definition and classification of ATMPs
- Technologies and platforms for production
- Regulatory pathway for marketing authorization
- Role of the CAT (Committee for Advanced Therapies) and the CHMP (Committee for Medicinal Products for Human Use)
- Good Manufacturing Practices (GMP)
- Examples of ATMPs approved in Italy and in Europe
- Compassionate use of ATMPs
- Ethical challenges and economic sustainability for healthcare systems
- Historical background and classification
- Design and production of biological and biotechnological drugs
- Comparison between biological drugs and conventional (small-molecule) drugs
- Safety considerations
2. Biotechnological Drugs for Central Nervous System Diseases
Alzheimer's disease
Multiple sclerosis
Migraine
3. Biotechnological Drugs for Oncological Diseases
Hormone therapy
Immunotherapy
Monoclonal antibodies
- Engineering and production; characteristics
- Types of monoclonal antibodies: Chimeric, Humanized, Fully human
- Bispecific antibodies: structure and therapeutic applications
- Antibody-Drug Conjugates (ADCs)
- Antibody fragments
- Side effects and toxicity of monoclonal antibodies
- Therapeutic applications
Cancer vaccines
4. RNA-Based Drugs
Introduction to RNA interference and genome editing
Types of RNA-based drugs, mechanisms of action, and therapeutic applications
- Small interfering RNAs (siRNAs)
- Antisense oligonucleotides (ASOs)
- Aptamers
- Therapeutic RNAs targeting genomic DNA: clinical applications
- mRNA vaccines
Formulation, chemical modifications, delivery systems, cellular uptake and intracellular distribution of RNA therapeutics
Potential toxicity, immunogenicity, and mutagenic risk
5. Advanced Therapy Medicinal Products (ATMPs)
- Definition and classification of ATMPs
- Technologies and platforms for production
- Regulatory pathway for marketing authorization
- Role of the CAT (Committee for Advanced Therapies) and the CHMP (Committee for Medicinal Products for Human Use)
- Good Manufacturing Practices (GMP)
- Examples of ATMPs approved in Italy and in Europe
- Compassionate use of ATMPs
- Ethical challenges and economic sustainability for healthcare systems
Prerequisites for admission
Students are expected to have prior knowledge in pharmacology, molecular biology, and biochemistry
Teaching methods
Interactive lectures supported by slides and video materials. Students are encouraged to attend regularly and to participate actively in class. Discussion is promoted to ensure proper understanding, stimulate critical thinking, and enhance communication skills. Seminars held by guest speakers may also be included. All teaching materials will be made available through the ARIEL platform.
Teaching Resources
Articles and review papers.
Farmaci biotecnologici e terapie avanzate. Calabrese - Molteni - Racagni Edra 2025
Farmaci biotecnologici e terapie avanzate. Calabrese - Molteni - Racagni Edra 2025
Assessment methods and Criteria
The exam consists of an oral assessment during which students are required to discuss three topics. The oral exam lasts approximately 30 minutes.
The examination is designed to:
- assess the achievement of the learning objectives in terms of knowledge and understanding;
- evaluate the ability to apply acquired knowledge through the discussion of topics covered during the course;
- verify the correct use of scientific terminology in the fields of pharmacology and genetics, as well as the ability to present content clearly and logically, making appropriate connections with topics from other courses within the degree programme.
The examination is designed to:
- assess the achievement of the learning objectives in terms of knowledge and understanding;
- evaluate the ability to apply acquired knowledge through the discussion of topics covered during the course;
- verify the correct use of scientific terminology in the fields of pharmacology and genetics, as well as the ability to present content clearly and logically, making appropriate connections with topics from other courses within the degree programme.
Professor(s)
Reception:
by appointment previously agreed by e-mail
INGM, Padiglione Invernizzi, Via F. Sforza 35 4 piano Ala A