94191 - Advanced Pharmaceutical Analysis

Learning outcomes

The student, as a result of attendance and after passing the final exam, acquires the knowledge of the analytical problems peculiar to the different phases of the drug discovery process, the main techniques and the analytical approaches to support this process, with particular focus on a specific phase of the process itself.

Course contents

The ultimate goal of a drug discovery program is to generate a candidate drug compound that, besides being active and selective in vitro on the isolated target, is safe and efficacious in humans. In order to develop such a compound, many criteria including affinity, activity, selectivity, physico-chemical properties, and pharmacokinetics must be optimized. The definition of these criteria involves the availability and knowledge of appropriate analytical methodologies.

In detail, the course will address the most advanced analytical methods required to ensure quality during the devopment of drugs for gene and cell therapies. Those drugs are included in the so called Advanced therapy medicinal products (ATMPs). 

There will also be a special session dedicated to case reports discussed by R&D managers of companies operating in the field of drug discovery. Lectures will be delivered by recognized experts from the academic and industrial sectors.

Classes will take place at the Rimini Campus from September 22 to September 24, 2025.

The course is delivered in English.

Main topics of the 2025-26 course:

- Developing cell and gene therapies: a journey from concepts to patients

- ATMPs: regulatory aspects

- Navigating the analytical lifecycle: strategic management for streamlined product development

- Characterization of synthetic oligonucleotides by high-performance liquid chromatography-mass spectrometry

- Analytical challenges in mRNA and pDNA production - Enhancing mRNA-based therapeutics safety and efficiency

- Mass Spectrometry toolbox for the analysis of adeno-associated virus capsids used in gene therapy treatments.

- LC-MS bioanalysis of oligonucleotide drugs: technology considerations and analytical challenges

- Separation and identification of therapeutic oligonucleotides impurities - Nusinersen case study for the treatment of spinal muscular atrophy

- Challenges in cell therapy characterization and analysis

- Immunogenicity of ATMs: evaluation of the presence of anti-drug antibodies using ligand binding assays

- The experience of mRNA and AdV vaccines: humoral and cellular biomarkers for the evaluation of the immune response to SARS-CoV-2 vaccination and breakthrough infections

- Flow cytometry validation to assess the number of circulating cells in preclinical studies

- qPCR validation and application to biodistribution studies qPCR validation and application to biodistribution studies

Readings/Bibliography

A pdf file with the presented slides will be provided.

Teaching methods

The course is delivered in English. Few sessions are scheduled for lectures which will be delivered by experts from the academic and industrial sector. the course is delivered as intensive course and classes are scheduled to take place within 3 days in September. Specifically, for the 2025-26 academic year classes will take place from September 22 to September 24, 2025. To enrol pplease contact  me in due time before the starting date.

The course is delivered in English.

Assessment methods

An oral exam will be set up to assess the acquisition on the learning outcomes.

Teaching tools

Video projector, PC. Educational material as pdf file

Office hours

See the website of Manuela Bartolini