Timetable, Themes & Courses

Here you will find the latest updates on the ULLA 2022 Timetable, Themes & Courses • This years focus: Challenges and opportunities in drug development

ULLA 22 • TIMETABLE

ULLA 22 • Timetable

SESSIONS BY DATE

SESSION 1 • 3 JULY

Shozeb Haider, Francesco Gervasio	Day 1. Workshop on Protein Structure Exploration in Drug Design and Discovery for Non-Computational Scientists
Iwan de Esch	Basic concepts in medicinal chemistry
Gabriele Costantino	Functional cosmetics and Skincare: From research to market opportunity
Ruggero Bettini	Inhalation route for local and systemic delivery of biological drugs
Johan Heldin	Methods to visualize protein interactions
Natalja Genina	Advances in 3D printing of pharmaceuticals
Rik Gijsbers	Viral vectors and gene therapy: turning infectious viruses into efficient vehicles to halt and cure genetic disorders
Marco van de Weert	Formulation of therapeutic proteins
Gerard van Westen	Advanced Computational Methods in Drug Discovery: AI and Physics Based simulations
Jordi Carreras-Puigvert	High Content imaging and lab automation for drug discovery and life sciences: Insights from assay design to disease models
Ben Boyd	Getting off to a flying start for a great finish to your PhD
Jukka Rantanen	Quality by Design (QbD) in Drug Development
Erik Jansson	Proteomics and metabolomics with mass spectrometry for drug discovery
Albert J. Kooistra, Jacqueline E. van Muijlwijk-Koezen	A hands-on introduction into computational medicinal chemistry

SESSION 2 • 4 JULY

Li Wei, Ruth Brauer	Post-marketing research into the safety of drugs using Big Data
Irena Loryan	Barriers of the CNS and their impact on brain drug disposition
Shozeb Haider, Francesco Gervasio	Day 2. Workshop on Protein Structure Exploration in Drug Design and Discovery for Non-Computational Scientists
Dr. Sara Garfield	Patient and public involvement (PPI) in research: Integration and dissemination
Matthias Barz	An introduction to Nanomedicine: From Concepts to Therapies
Isabel Braadbaart	Intro Science Teaching
Sergei Strelkov	Protein X-ray Crystallography in Drug Design
Silvia Pescina	Ex vivo ocular models for drug delivery studies
Henning Henschel	Introduction to computational medicinal chemistry
Pieroni Marco	Medicinal Chemistry In The Managing Of Antimicrobial Resistance
Ola Spjuth	Introduction to Artificial Intelligence and machine learning in drug discovery
Erik Jacobsson	Bioprocessing lecture and study visit to Cytiva (formerly GE Healthcare)
Ben Boyd	Size matters: Advanced characterisation and concepts in colloidal drug delivery
Erik Sjögren	Physiologically Based Pharmacokinetic modelling
Susanne Bredenberg	Entrepreneurship and Innovation

SESSION 3 • 7 JULY

Isabelle Turbica	Therapeutic Antibodies : From Bench to Clinic
Dr. Eric Morel, Dr. Dorine Bonte	Day 1. Biotech Challenge
Jörg P. Kutter	Day 1: Modern tools for pharmaceutical analysis – sample preparation and separation formats
Maurizio Sessa	Day 1. Fundamentals of drug utilization research using real-world data
Oscar Della Pasqua	Biomarker development and implementation in clinical development
Ali Makky	Smart nanomaterials responding to light and their biomedical applications
Guy Van den Mooter	Physicochemical and biopharmaceutical profiling of drug candidates
Jonas Rydfjord	Chemistry in early drug discovery – principles, strategies, and tools
Alessio Lodola	Computer-Aided Drug Design
Karin Steffen	Introduction to R (RStudio) and making nice figures with ggplot2
Anders Backlund	Applications of chemography

SESSION 4 • 8 JULY

Tâp Ha-Duong	Experimental and computational tools to design peptide-based inhibitors of protein-protein interactions
Dr. Eric Morel, Dr. Dorine Bonte	Day 2. Biotech Challenge
Jörg P. Kutter	Day 2: Modern tools for pharmaceutical analysis – sample preparation and separation formats
Maurizio Sessa	Day 2. Fundamentals of drug utilization research using real-world data.
Oscar Della Pasqua	Model-informed Drug Development
Jonas Rydfjord	Early drug discovery in an academic setting
Lene Jørgensen	Pharmaceutical policy and basic regulatory affairs
Eva Garmendia	Science communication: bringing your science to the public!
Blazej Slazak, Sunithi Gunasekera	Introduction to peptide discovery and characterisation

Themes & courses

Drug Discovery

• Workshop on Protein Structure Exploration in Drug Design and Discovery for Non-Computational Scientists
• Basic concepts in medicinal chemistry
• Advanced Computational Methods in Drug Discovery: AI and Physics Based simulations
• Protein X-ray Crystallography in Drug Design
• Introduction to Computational Medicinal Chemistry
• Medicinal Chemistry In The Managing Of Antimicrobial Resistance
• Computer-Aided Drug Design
• Early drug discovery in an academic setting
• Introduction to peptide discovery and characterisation
• Applications of chemography
• Experimental and Computational Tools to Design Peptide-based Inhibitors of Protein-protein Interactions
• Proteomics and metabolomics with mass spectrometry for drug discovery

Drug Development

• Formulation of therapeutic proteins
• Quality by Design (QbD) in Drug Development
• Barriers of the CNS and their impact on brain drug disposition
• Ex vivo ocular models for drug delivery studies
• Size matters: Advanced characterisation and concepts in colloidal drug delivery
• Biomarker development and implementation in clinical development
• Physicochemical and biopharmaceutical profiling of drug candidates
• Chemistry in early drug discovery – principles, strategies, and tools
• Model-informed Drug Development

Drug Usage

• Fundamentals of drug utilization research using real-world data
• Post-marketing research into the safety of drugs using Big Data

Biologics

• Inhalation route for local and systemic delivery of biological drugs
• Therapeutic Antibodies • From Bench to Clinic

Modern methods and techniques in pharmaceutical sciences        

• A hands-on introduction into computational medicinal chemistry
• Modern tools for pharmaceutical analysis – sample preparation and separation formats
• Methods to visualize protein interactions
• Advances in 3D printing of pharmaceuticals
• Viral vectors and gene therapy: turning infectious viruses into efficient vehicles to halt and cure genetic disorders
• An introduction to Nanomedicine: From Concepts to Therapies
• Introduction to artificial intelligence and machine learning in drug discovery
• Physiologically Based Pharmacokinetic modelling
• Smart nanomaterials responding to light and their biomedical applications
• High Content imaging and lab automation for drug discovery and life sciences: Insights from assay design to disease models
• Bioprocessing lecture and study visit to Cytiva (formerly GE Healthcare)

Innovation and entrepreneurship

• Functional cosmetics and Skincare: From research to market opportunity
• Entrepreneurship and Innovation

General Skills

• Science communication: bringing your science to the public!
• Pharmaceutical policy and basic regulatory affairs
• Introduction to R (RStudio) and making nice figures with ggplot2
• Biotech Challenge
• Patient and public involvement (PPI) in research: Integration and dissemination
• Intro Science Teaching
• Getting off to a flying start for a great finish to your PhD

• Duration: Two day
• Level: Basic
• Course leaders: Prof Shozeb Haider and Prof Francesco Gervasio
• Institution: UCL School of Pharmacy/University of Geneva Pharmaceutical Sciences
• Teaching staff: Ioannis Galdadas (University of Geneva)
• Requirements: Laptop Essential

This hands-on workshop will employ molecular modelling tools to explore structural data resources to understand protein structure and function for drug discovery. The main aim is to interpret and exploit biomolecular structures, focusing on how best to use structural information to gain the most from it in specific research contexts.

This course will include structural/sequence database searching, visualising protein structure, predicting protein structures with machine learning (AlphaFold2), and exploring protein interactions with ligands.

The hands-on workshop will use theory and experiment simultaneously. There will be an opportunity to discuss your own research interests to maximise effectiveness. This course is aimed at students wanting to learn more about (a) protein structure, (b) the application of structural information in drug discovery and (c) how to use some of the key bioinformatics resources that are available for drug discovery.  No previous understanding in the field of structural biology is required. Basic knowledge of protein structure and chemistry is essential.

Syllabus, tools, resources

• Introduction Protein Structure and Function
• Basics of Molecular Visualisation Tools
• Computational Approaches to Database searching, Sequence and Structural alignments
• Introduction to Protein Analysis
• De Novo Protein Structure Prediction with AlphaFold2
• Automated Ligand Docking
• Tools and resources for drug discovery

Outcomes

After this course you should be able to:

• Access and browse structural data repositories to determine whether appropriate structural information exists, including the use of structure-quality information
• Visualise the 3D structure of proteins
• Sequence searching and homology comparisons
• Predict the Structure of a Protein from its sequence using Machine Learning
• Evaluate the potential impact of genetic variation on a structure
• Able to carry out ligand docking

• Duration: One day
• Level: Basic
• Course leader: Iwan de Esch
• Institution: VU University Amsterdam
• Teaching staff: To be confirmed
• Requirements: NA

This  course  is  designed  for  students  with  an  interest  in  life  sciences  and  the biotech/pharmaceutical industry but without prior education in this field. A general introduction will be given to the process of drug discovery, drug design and synthesis, drug development and drug safety assessment.

Subsequently, potential drug targets,  mechanisms  of  drug  actions  (including  drug-receptor/enzyme Using various drug classes, relationships between chemical structures and biological activities will be derived and illustrated. Finally, various modern developments and tools will be illustrated by recent applications in the field of drug research, medicinal chemistry and toxicology.

Topics include:

• Drug Targets, proteins
• Enzymes as drug targets
• GPCRs as drug targets
• Kinases, ion channels and other drug targets;
• Pharmacokinetics
• Hit finding & optimization
• Computational design & QSAR

• Duration: One day
• Level: Basic
• Course leader: Prof. Gerard van Westen
• Institution: Leiden Academic Center for Drug Research (LACDR)
• Teaching staff: Dr. Willem Jespers
• Requirements: Laptop

Pharmaceutical science is changing; while perhaps not a paradigm shift, the influence and catalytic effect of data science on drug discovery cannot be denied. Scientific data is becoming public and even open access. Moreover, better computing capabilities and more data make it easier to use prior data to improve ongoing work.  

Chemical Biology explores biology via chemical tools. In practice this means that the molecular interaction space of protein targets is probed. Computational Chemical Biology is the computational addition to these goals and is located in between the fields of medicinal chemistry, cheminformatics, bioinformatics, and computational biology. This course consists of bio and cheminformatic approaches which are coupled to structure-based methods using crystal structures. Herein students learn to computationally analyze protein sequences as well as ‘small molecules’, and ultimately model interactions between them using publicly accessible databases and state of the art tools.

Theory will be explained in the form of three lectures. After each lecture, the students will have time to perform practical work related to the lecture.

• Duration: One day
• Level: Basic
• Course leader: Prof. Sergei Strelkov
• Institution: KU Leuven
• Teaching staff: To be confirmed
• Requirements: Laptops. Students will be asked to install some software (free educational versions) in advance on their laptops.

Knowledge of the atomic structure of the (protein) target and structural analysis of its complexes with candidate molecules play a central role in rational drug design. The course will include a lecture in the morning and a practical exercise in the afternoon.

The lecture will discuss the basic principles of the protein crystallography (crystallization, X-ray diffraction experiment, building and analysis of the three-dimensional structure). In addition, further important methods of 3D structure determination including cryoelectron microscopy and in silico prediction will be introduced.

In the afternoon a hands-on practical session on the use of software tools will follow. Students will learn the CCP4/Coot crystallographic suite through performing phasing, model building and refinement of a protein-drug complex. Thereafter Pymol will be used towards graphical presentation and analysis of the structure.

This course was very popular during several previous ULLA schools, which motivates us to offer it again.

• Duration: One day
• Level: Basic
• Course leader: Henning Henschel
• Institution: Uppsala University
• Teaching staff: To be confirmed
• Requirements: NA

Computational methods have in recent years evolved to become a commonplace tool in medicinal chemistry. These methods can for example be used to screen potential ligands against the binding site of a known target in the early drug discovery process or to guide the drug design process by establishing a three-dimensional quantitative structure-activity relationship based on compound data.

This course comprises a short introduction to the field, focussing on getting hands-on experience with some basic molecular modelling techniques in a computational lab. Exercises will also give an introduction to the Schrödinger software suite. 

• Duration: One day
• Level: Basic
• Course leader: Pieroni Marco
• Institution: University of Parma
• Teaching staff: Giannamaria Annunziato
• Requirements: NA

The course will be based on the medicinal chemistry of innovative therapeutic agents for the fight of bacterial infections. The first part of the course will be dedicated to a short introduction of one of the most threatening insults to public health nowadays, that is antimicrobial resistance (AMR). How AMR affects public health, both under the clinical and socio-economical point of view, will be explained and commented with the help of epidemiological and socio-economical data.

After this introductory part, the state of the art of the current therapeutic arsenal will be described, with particular emphasis on the most recent FDA/EMA approved drugs and a critical comment on the reasons that have made the antibacterial drug discovery as a neglected research area. Then, the main mechanisms of bacterial resistance will be illustrated, with a brief description of the most innovative strategies to counteract AMR. In this context, a broad deepening will be dedicated to the exploitation of the so called “non-essential” targets as novel strategies to fight bacterial resistance. Also, drugs with innovative mechanism of action will be introduced.

The course will be focused on the medicinal chemistry aspects lining up the design and synthesis of efflux pumps inhibitors, biofilm disruptors and inhibitors of metabolic pathways which are dispensable during bacterial life cycle but become indispensable for bacterial colonization and infection persistence. In these regards, focused case studies will be reported and commented.

• Duration: One day
• Level: Advanced
• Course leader: Alessio Lodola
• Institution: University of Parma
• Teaching staff: -
• Requirements: Students must bring a laptop. More detailed instruction for software access/installation will be provided before the course starts.

Computer-Aided Drug Design (CADD) is a common approach in medicinal chemistry projects aimed at finding new hits for a given target or in lead optimization campaigns.

The fundamentals to understand and exploit many CADD tools often reside in complex equations for energy estimation and geometry optimization of molecular models. Medicinal chemists demand graphical outputs to focus their attention on chemical interpretation, but sometimes a careful consideration of numerical recipes underlying these outputs helps to provide a more realistic sight to CADD predictions or explanations.

This course will deal with the theoretical aspects and the applications of CADD. The theory of force fields, docking methods, and commonly applied computational strategies (i.e., virtual screening methods) and their application to drug design will be presented and discussed.

• Duration: One day
• Level: Advanced
• Course leader: Jonas Rydfjord
• Institution: Uppsala University
• Teaching staff: Ulrika Yngve, Ylva Gravenfors, Ulrika Rosenström
• Requirements: Prior knowledge of medicinal chemistry and the drug discovery process will be helpful, but is not strictly required.

There is a demand for new and more efficient medicines to treat a range of different illnesses like viral infections, neurological diseases, and cancer. The process of drug discovery is in most cases the same even though the drug target differs. In this joint course between the department of Medicinal Chemistry at Uppsala University and the SciLifeLab Drug Discovery platform you will learn conceptually how to work in the very earliest parts of the drug discovery process.

The course will combine an introduction to the general workflow with an example of a successful drug discovery project targeting Acute Myeloid Leukemia (AML) run by the SciLifeLab platform. You will also get the chance to practice and put your knowledge to the test in an interactive game-like workshop were you will form small drug discovery teams working with molecules, assay data and decision making in a fictive project.

• Duration: One day
• Level: Basic
• Course leaders: Blazej Slazak and Sunithi Gunasekera
• Institution: Uppsala University
• Teaching staff: -
• Requirements: NA

This course will contain an introduction to peptide discovery, and in particular characterisation of peptides using NMR and MS.

The course contains introductory lectures demonstrations/practicals (e.g. preparing and running a peptide sample on LCMS to collect fragmentation data for sequencing de novo; and introductions to experimental design for structure determination by NMR in solution, and sequential assignments).

• Duration: One day
• Level: Basic
• Course leader: Anders Backlund
• Institution: Uppsala University
• Teaching staff: -
• Requirements: A laptop is essential and spread sheet (ExCel, Numbers, etc) and text editing software (BBedit, emacs, WordPad, etc) should be installed. In addition, chemical structure drawing software capable of turning structures into SMILES (and vice versa) is advantageous.

Chemography, or the art of navigating chemical space, can be done using the tool ChemGPS-NP. With the 'maps' retrieved, rapid assessment and comparison of large sets of molecules, evaluation of chemical similarity, and prediction of biological activities can be made - even on entirely hypothetical molecules.

The course explores various uses of chemography and introduces general concepts of analysis using available anti-viral chemistry data.

• Duration: One day
• Level: Advanced
• Course leader: Tâp Ha-Duong
• Institution: Université Paris-Saclay
• Teaching staff: Nicolo Tonali
• Requirements: Prior knowledge in molecular biology is useful

The proposed course aims at providing an overview of both the experimental (3h) and the computational (3h) strategies and tools used for identifying and optimizing peptides that target protein-protein interfaces with high affinity and specificity. Indeed, protein-protein interactions play crucial roles in many biological processes and modifications of their fine mechanisms generally result in severe diseases.

Peptide derivatives are very promising therapeutic agents for modulating protein-protein associations with size and specificity between those of small compounds and antibodies. For the same reasons, rational design of peptide-based inhibitors naturally borrows and combines approaches from both protein-ligand and protein-protein research fields.

Duration: One day
Level: Advanced
Course leaders: Erik Jansson
Institution: Uppsala University
Teaching staff: Theodosia Vallianatou
Requirements: Prior knowledge in analytical chemistry and molecular biology is useful

You will learn how mass spectrometry can provide metabolomic and proteomic information pertinent to any biological model system you are working with. An overview of sample preparation and experimental data acquisition with mass spectrometry will be provided.

We will provide hands-on exploration of metabolomic and proteomic datasets, including standard LC-MS based metabolomics and proteomics but also exploration of datasets acquired with mass spectrometry imaging and proteomic workflows targeted at identification of protein interactions within molecular pathways affected by drug treatments.

• Duration: One Day
• Level: Advanced
• Course leader: Marco van de Weert
• Institution: University of Copenhagen
• Teaching staff: Vito Foderà
• Requirements: basic understanding of thermodynamics, general analytical chemistry, general biochemistry (structure and function of proteins).

Proteins are an increasingly important class of drug molecules for the treatment of various serious diseases. Their administration and formulation differs markedly from those of small molecules, e.g. they are generally administered by injection, which limits formulation choices.

Moreover, proteins are chemically and physically labile, which puts further demands on the formulation, but also on the analytical methodology. In this course several important aspects of protein formulation will be discussed: a) protein folding and aggregation; b) analytical characterisation and its use in formulation design; and c) immunogenicity of therapeutic proteins.

These topics will be discussed through lectures by the course organisers as well as group discussions. The lectures will mainly cover protein folding and aggregation as well as immunogenicity. Analytical methods and formulation approaches will be discussed in an “open forum” format with group discussions.

At the end of the course the participants are expected to be able to describe the various challenges in protein formulation design, with an emphasis on physical instability and analytical characterization.

• Duration: One Day
• Level: Basic
• Course leader: Jukka Rantanen
• Institution: University of Copenhagen
• Teaching staff: Anette Müllertz, University of Copenhagen, Clare Strachan, University of Helsinki
• Requirements: NA

Quality by Design (QbD) approach is underpinning the importance of a risk-based approach as a foundation for the broader drug development process. This regulatory framework is characterized by the use risk management strategies aiming for patient-centric identification of critical quality attributes (CQA) and related material properties and process parameters, including careful investigation of the relationship between them using design of experiments (DoE). This gives a solid basis for constructing the Design Space for a final medicinal product.

There is a solid scientific foundation behind a thorough QbD based product design and a detailed product understanding should be based not only on experimental approach, but also on the utilization of modelling from different perspectives. Molecular modelling is a natural starting point for formulation design, followed by investigation of multi-dimensional analytical signals and complex process data, as well as the use of pure physical modelling for explaining and simulation of the phenomena related to the performance of the final product.

This course will give a basic introduction of risk assessment and DoE tools, therapy-driven construction of target drug delivery profiles, effective use of spectroscopy (near-IR, Raman, and different spectral imaging modalities) both within process analysis and product design, and further, state-of-art insight into selected examples of modelling as a part of QbD based drug development for future pharmaceuticals [1]. 

[1] M. de Matas, T. De Beer, S. Folestad, J. Ketolainen, H. Lindén, J. Lopes, W. Oostra, M. Weimer, P. Ohrngren, J. Rantanen, 2016. Strategic framework for education and training in Quality by Design (QbD) and process analytical technology (PAT). Eur.J.Pharm.Sci. 90: 2-7

• Duration: One Day
• Level: Basic
• Course leader: Assoc. Prof. Irena Loryan
• Institution: Uppsala University
• Teaching staff: Mohammed A. Saleh, Leiden Academic Center for Drug Research, Frida Bällgren, Uppsala University.
• Requirements: basic principles in pharmacokinetics

The course is intended for junior researchers working on drug discovery, development and optimization of drugs for CNS action (or to be avoided), for development of novel CNS drug delivery systems, as well as clinical pharmacists working in the field of CNS disorders.

Background lectures and case studies will cover the following topics:

• Mechanisms of drug transport across the blood-brain barrier and the blood-CSF barrier
• The free drug theory and general concepts about drug disposition at the CNS target site(s)
• In vitro, and in vivo methodologies to assess brain drug delivery and disposition in animals and humans, in healthy and diseased conditions.
• Physiologically-based pharmacokinetic (PBPK) modelling of CNS drug disposition

State-of-the-art approaches recommended in drug discovery and development

This course will give the participants a thorough introduction to the basic principles and methods used for assessment of brain drug exposure in preclinical and clinical settings, and will give advice on design of studies for evaluation of brain drug delivery. Analysis of case studies will offer students the opportunity to learn how to interpret the results and make strategic decisions.

• Duration: One Day
• Level: Advanced
• Course leader: Silvia Pescina
• Institution: University of Parma
• Teaching staff: Sara Nicoli
• Requirements: basic knowledge on hurdles of the ocular delivery and on conventional ophthalmic formulations

In the last decades, the research in the field of ophthalmic delivery has addressed several unmet clinical needs in ophthalmology. As a consequence, innovative formulations allowing for minimally and less invasive administration, amongst all the nanosystems, led to valuable results, even already implemented as marketed products.

A full in-depth knowledge of an ophthalmic formulation is attained by combining the results from in vitro experiments with those obtained using ex vivo animal models. Particularly, working with ex vivo models based on animal ocular tissues allows for permeation and retention studies, crucial for the comprehension of formulation behaviour and consequently for its evaluation and optimization.

Ex vivo ocular models can be either simple, such as an isolated membrane, like cornea, sclera, choroid, or very complex, as a whole eye. Regardless of the complexity, the set-up and validation of ex vivo ocular models are often a challenge since reliability and reproducibility are the two main mandatory requirements.

Topics of the course will be the development, validation, and application of ex vivo ocular models for drug delivery studies. Several examples will be provided, involving both the anterior and the posterior segment of the eye, as well as conventional and innovative dosage forms. During the course PhD students will be directly involved, taking an active part to the discussion, even by working in small groups.

• Duration: One Day
• Level: Advanced
• Course leader: Professor Ben Boyd
• Institution: University of Copenhagen
• Teaching staff: Professor Alex Bunker (UHelsinki), Professor Camilla Foged (UCPH), Professor Anette Mullertz (UCPH), Professor Thomas Rades (UCPH), Professor Jukka Rantanen (UCPH), Professor Clare Strachan (UHelsinki)
• Requirements: NA

Colloids have had a profound impact on human society in the last few years.  For example, the LNP-based Covid-19 vaccines are colloidal particles, requiring a controlled size in the colloidal range for their interaction with biology. Although only relatively recently hitting the headlines, they have been in development for over 40 years, with decades of colloid science underpinning their development. Indeed, colloid science has an impact across all facets of drug delivery.

Of course, colloids and colloidal structure is not only about particles – it captures issues where structure and interfaces are important to function, often not achievable with the bulk material from which they are prepared. Understanding the challenges with different types of colloidal materials, their structure and composition, how they interact with the body and how they can be translated into medicines are all important topics for the contemporary pharmaceutical scientist to be exposed to.

To this end, this course will give a contemporary view of challenges and opportunities across colloidal systems in drug delivery, where you will hear the latest thinking and developments across the materials, their biointeractions, analytical methods to study composition and structure and the production of colloid-based medicines.

9.00–9.10      Introduction to the course (BB)

Session 1     Colloidal materials in drug delivery I

9.10–9.50      Oral drug delivery and colloidal lipid systems (AM)

9.50–10.30    Colloidal materials – on the way to and in the solid state (TR)

10.30–10.45   Break

Session 2      Colloidal materials in drug delivery II

10.45–11.30   Colloidal vaccines – thank you World for listening.  (CF)

11.30–12.15   Computational approaches to the design of colloidal drug delivery systems (AB)

12.15–13.45   Lunch

Session 3      Contemporary analytical approaches to colloidal systems

13.45–14.30    Spectroscopic characterization of colloidal drug delivery systems (CS)

14.30–15.15   Structural characterization of colloidal drug delivery systems (BB)

15.15–15.30   Break

Session 4      Preparation and translation at the commercial scale

15.30–16.15   Unique considerations and challenges in the manufacturing of colloidal materials as pharmaceutical products (JR)

16.15–16.45   Colloidal pub quiz – a fine way to round out the course!

16.45–17.00   Final comments (BB)

• Duration: One Day
• Level: Basic
• Course leader: Oscar Della Pasqua
• Institution: University College London
• Teaching staff: Salvatore D’Agate
• Requirements: Prior knowledge in pharmacokinetics and pharmacodynamics. Students should bring their own laptop.

This course is aimed at demonstrating how clinical pharmacology concepts can be applied to the development and validation of biomarkers to: 1) support evidence generation,  2) optimise trial design (patient selection, inclusion/exclusion criteria, dose rationale) and  3) establish the relevance of personalised regimens in specific group of patients, for whom the benefit-risk balance may differ from the overall target patient population.

Attention is given to the assessment of the operating characteristics of biomarkers and their use in early and late clinical drug development, including an overview of biomarker guided clinical trial designs.   

The following topics will be presented and discussed:

• Biomarker identification, definition and applications
• Biomarker evaluation and validation: receiver operating characteristics
• Biomarkers as predictors of treatment response and clinical decision making
• Biomarker-guided clinical trial design

In addition, the clinical and theoretical concepts participants will have the opportunity to further understand the regulatory requirements for an effective development program. The use of quantitative pharmacology methods and real-world data are introduced as a tool for evidence synthesis and personalisation of treatment.

• Duration: One Day
• Level: Advanced
• Course leader: Guy Van den Mooter
• Institution: KULeuven
• Teaching staff: -
• Requirements: NA

Physicochemical and biopharmaceutical profiling is an essential step in the pharmaceutical development by which candidate drugs are characterized with respect to the appropriateness to be formulated and processed to a useful dosage form.

During this phase of the development, information about the physicochemical and biopharmaceutical properties, and stability profile (physical, chemical, compatibility with excipients, etc.) of the drug candidate is collected. This information guides the formulation scientist as it will dictate many of the possible formulation and processing approaches.

In this course, fundamental physicochemical and biopharmaceutical concepts are discussed including solubility and dissolution rate, ionization behavior, partitioning, solid state properties (polymorphism, amorphous and crystalline state), salt formation and salt/ counterion selection, physical and chemical stability, powder properties and drug absorption profiling. Analytical techniques to assess solid state properties such as X-ray diffraction, modulated differential scanning calorimetry, dynamic vapour sorption, thermogravimetric analysis and vibrational spectroscopy (IR and Raman) are briefly discussed and illustrated with examples.

• Duration: One Day
• Level: Advanced
• Course leader: Jonas Rydfjord
• Institution: Uppsala University
• Teaching staff: Luke Odell and Johan Wannberg
• Requirements: Prior knowledge of organic chemistry.

A fundamental part of medicinal chemistry is the synthesis of new organic compounds. By wise choices of strategies for compound generation, large and diverse compound libraries can be synthesized for later use in screening efforts. Enabling methods for late-stage functionalization, incorporation of stereocenters, and green chemistry, are also ever-evolving and applied in medicinal chemistry.

In this course we will take a look at some of the strategies that can guide the choice of synthetic route as well as the larger application of compound library generation. There will be a mix of an overview of strategies and methods with more in-depth lectures on photocatalysis, late-stage C-H functionalization, and DNA-encoded libraries, for example.

• Duration: One Day
• Level: Basic
• Course leader: Oscar Della Pasqua
• Institution: University College London
• Teaching staff: Alessandro di Deo
• Requirements: Laptop with R-studio. Basic knowledge of pharmacokinetics, pharmacodynamics and clinical pharmacology is required. Students are also expected to have good understanding of biostatistics.

For more than five decades empirical evidence of the benefit and risk of medicines has been used to support the evaluation, approval and use of medicines. Consequently, results from clinical studies are assessed without considering the constraints of the underlying experimental design or the implications of inclusion and exclusion criteria for the efficacy and safety profile of a medicinal product in the overall population.

By contrast, model-informed drug development (MIDD) has evolved as a quantitative approach and is now widely applied in clinical pharmacology as a tool to integrate knowledge and optimise experimental protocols, thereby supporting the dose rationale and providing further opportunities for the personalisation of treatment. 

This course offers:

• An overview of the principles of MIDD
• Its role in regulatory approval  
• A comprehensive introduction to clinical trial simulations. 

The session will comprise lectures in which theoretical principles will be presented followed by practical computer exercises, during which the impact of study design, covariate factors and disease conditions will be evaluated.

In addition to gaining basic understanding of the requirements for performing and interpreting the results from clinical trial simulations, attention will be given to assumptions and model integration (e.g. selection of PK and PKPD models, epidemiology and clinical characteristics of disease and patient population).

• Duration: Two Day
• Level: Basic
• Course leader: Maurizio Sessa
• Institution: University of Copenhagen
• Teaching staff: Björn Wettermark (Uppsala University), Mohammadhossein Hajiebrahimi (Uppsala University), Morten Andersen (University of Copenhagen)
• Requirements: All applicants must have basic and documented skills in using statistical software (R) for data management, and have - at a minimum - basic level knowledge in epidemiology.

The students are expected to obtain an epidemiologic competence, which makes them able to contribute to planning and carrying out drug utilization studies based on real-world data. In particular, the course will provide knowledge and skills on three aspects of drug utilization research: 1) selection of data sources, 2) study design, 3) data management & data analysis.

The participants will learn which types of real-world data sources are available to conduct drug utilization research with examples from Denmark and Sweden. A wide range of national and regional data sources available on drug dispensing, biomarkers, health outcomes, and socioeconomic status will be presented and so will the processes when linking data from various sources.

Additionally, the participants will have the possibility of learning the basic principles of pharmacoepidemiological study design, and data management of real-world data using simulated datasets. In particular, the participants will be trained to choose of available study design and operationalization of epidemiological concepts in algorithms for defining exposures and auxiliary variables in real-world data. We will also provide examples of multi-countries studies using real-world data.

Learning objectives

A student who has met the objectives of the course will be able to:

• Identify available real-world data relevant for drug utilization research with examples from Denmark and Sweden.
• Recognize ethical aspects, laws & regulations for real-world data.
• Plan and design drug utilization analyses using real-world data.
• Define drug exposures and auxiliary variables related to drug exposure in real-world data.
• Perform basic data management procedures on simulated real-world data.
• Understand the principles of multi-country research in drug utilization research.

• Duration: One Day
• Level: Basic
• Course leaders: Prof Li Wei and Dr Ruth Brauer
• Institution: University College London
• Teaching staff: -
• Requirements: NA

This day course is designed for students who are interested in pharmacoepidemiology or Phase 4 trial research. In recent years, due to the pandemic, we have all become familiar with epidemiological information presented in figures and graphs. We would like to share how we conduct studies into the use and safety of commonly used drugs with our ULLA summer school students.

We aim to equip students with a basic understanding of the concepts and practice of pharmacoepidemiology. Formally, pharmacoepidemiology is the science that applies epidemiologic approaches to studying the use, effectiveness and safety of drugs in a large population.

Students will be introduced to observational study designs (ie. cohort/case control studies) and their different strengths and limitations compared to experimental study designs. We will also discuss various sources of information (ie. CPRD/HES) that are often used in pharmaco-epidemiological research. Special emphasis will be put on the knowledge and skills to critically appraise studies.

Throughout the day we will use a mixture of both workshops and more traditional lectures. There will be plenty of opportunity for students to ask questions related to their own research.

• Duration: One Day
• Level: Advanced
• Course leader: Ruggero Bettini
• Institution: University of Parma
• Teaching staff: Annalisa Bianchera
• Requirements: NA

Biopharmaceuticals are going to dominate the pharmaceutical market in the future. However, their characteristics limited their administration to parenteral route in severe illnesses and hospital use. The application of safe and efficient alternative route of delivery could open new opportunities and application to these potent drugs.

The airways epithelia have emerged as a safe and efficient absorption route for peptide and proteins and could represent the solution for non-invasive biopharmaceutical delivery. Pulmonary administration of insulin has been the first scientific breakthrough in this sense for lower airways demonstrating the feasibility of such an approach.

This short course will take in consideration some of the aspects related to the design, development, and characterization of biological drug formulations to be administered to the airways with particular focus on the pro and cons of selecting liquid or solid formulation and the matching of a suitable device for drug administration.

The course will address in particular the technological aspects related to drug delivery systems and platforms for these administration route considering also some specific applications such as vaccination. The course will also address, in interactive manner, some practical aspect related to the characterization the formulation for inhalation.

• Duration: One Day
• Level: Basic
• Course leader: Dr Isabelle Turbica
• Institution: Université Paris-Saclay
• Teaching staff: Prof. Philippe Billiald
• Requirements: Basic knowledge in molecular immunology and pharmaceutical development of biologics. Students bring their own computer

Major rapid advances in antibody engineering and biopharmaceutical manufacturing have made antibodies and their derivatives the fastest growing class of therapeutics. However, designing a therapeutic antibody candidate, driving its pharmaceutical development and regulatory pre-clinical evaluation are not an easy task and address special issues, compared to small molecule drugs.

This one-day course will be a walk from the bench design to the clinic, from academic research to early stage clinical evaluation carried out in a university spin-off prior to industrial development in pharmaceutical companies. It will be supported by morning lecture courses, followed by afternoon bioinformatic practicals, critical review of scientific papers and a case study analysis.

The main following areas will be covered:

• Immunoglobulins as distinct therapeutic class
• Monoclonal antibody sources (hybridomas, transgenic mice and repertoire libraries)
• Converting an antibody into a drug candidate: antibody engineering methods; residual immunogenicity evaluation, host cells and expression systems in use for high scale production (productivity, protein homogeneity and cost of goods)
• Manufacturing consistency and drug substance quality.
• Regulatory pre-clinical studies to be conducted prior to first-in-man studies.
• First-in-man case study: an antithrombotic Fab for the treatment of cardiovascular emergencies.

• Duration: One Day
• Level: Basic
• Course leader: Eva Garmendia
• Institution: Uppsala Antibiotic Center
• Teaching staff: To be confirmed
• Requirements: NA

The ultimate goal of scientific advances is to have an impact in society. In this respect, communication of new scientific methods and results, and the popular outreach of science are of extreme importance to fill the gap between research and broad social environments. There are different sectors of society and consequently many ways that science can be brought to the public in effective ways: i.e. from spreading the news of scientific discoveries to stimulating kids into this fascinating world.

In this course, we will first go through various ways by which science can be brought to the public, and then we will work hands on creating different outreach projects in groups. Ready to make your science fun and engaging? This is the course for you!

• Duration: One Day
• Level: Basic
• Course leader: Lene Jørgensen
• Institution: University of Copenhagen
• Teaching staff: Sofia Kälvemark Sporrong, Uppsala University
• Requirements: NA

Knowledge about and analysis of stakeholders is important in all contexts, and when working with medicines knowing the ‘who’ and ‘why’ in regulatory affairs and pharmaceutical policy is key. Pharmaceutical policy and regulatory affairs play a role throughout, from development to use of medicines and the more everyone knows and understands the easier it becomes to provide medicines efficiently. You will have learned some during your master’s studies but are you up to date?

To put regulatory affairs into the broader perspective of pharmaceutical policy this course will first focus on the rationales for the highly detailed regulations of medicines, including their consequences. The global diversity when it comes to regulations of medicines will be reviewed in a cultural and economic context. Regulations and different rationales behind them are elucidated from the perspectives of different stakeholders, such as patients, governments and the pharmaceutical industry.

Second, we will briefly touch upon aspects from the various regulatory phases in drug development and in addition, you will learn and practice some of the specific vocabulary that is used within the field of regulatory affairs. So that you can use this vocabulary in a potential job interview.

• Duration: One Day
• Level: Basic
• Course leader: Karin Steffen
• Institution: Uppsala University
• Teaching staff: -
• Requirements: People need to bring their own computers and need to have administrator rights (for downloading packages).

Data analysis and visualisation are key skills in modern life sciences. One platform that integrates both parts, is freely available and open-source is R.

If you have never worked with R, RStudio or scripting, it may be a steep learning curve and maybe even a threshold to get started at all. However, R is immensely versatile and finding answers  to issues online is easy once you know the basics. This is meant as a fundamental beginners course.

The goal with this course is to enable you to use R so that after the course, you’ll be able to figure out your own analyses.

The course will touch upon:

• The RStudio GUI
• Importing data
• Manipulating and re-arranging data
• Example analyses relevant to pharmaceutical research
• Visualizing your results using ggplot2

Learn more

• R (free on all platforms, from CRAN https://cran.r-project.org/)
• ​RStudio (free, from https://www.rstudio.com/products/rstudio/download/)

• Duration: Two Day
• Level: Basic
• Course leaders: Dr Eric Morel and Dr Dorine Bonte
• Institution: University Paris-Saclay
• Teaching staff: -
• Requirements: An open mind and any basic knowledge in the ULLA broad themes: Drug discovery, Drug development, Drug usage, Biologics, Modern methods and techniques in pharmaceutical sciences, Innovation and entrepreneurship, and General skills.

Attendees will have to find a yet undeveloped innovative project that could be an idea, an object, a device, a service, an association, a concept, an app, a company, etc. based on research in the scientific literature or scientific web sites in Health domain. One can provide as an example the Google/Novartis association for a real time measurement of glycaemia for diabetic patients with lens device.

PhD students will first have to constitute their own team of 4 people and will be questioned by mentors to build their project by taking into account hard Sciences and technical aspects but also sustainability, social, ethical and environmental aspects, etc. Then, during a second session, they will have to work on their project and prepare a powerpoint (8 slides) for the project final defence during the last session.

This will be a virtual mini-symposium with an oral defence of the innovative projects that will be performed in front of a jury composed of PSaclay University and ULLA members. The top ranking will get rewards.

Attendees will develop the following skills:

Thoughts ordering

• Clear problematic
• Comprehensive objective
• Setup of a totally original project

Scientific knowledge

• Scientific background and basis (pathologies, mechanisms, technics…)
• Preclinical data and clinical trial as a basis

Transversal knowledge

• Financial development and economical insertion
• Regulations
• Ethics and societal development
• Environmental impact

Soft skills

• Management of team and communication
• Organisation

• Duration: One Day
• Level: Basic
• Course leader: Dr Sara Garfield
• Institution: University College London
• Teaching staff: Prof Cate Whittlesea, Dr Mine Orlu
• Requirements: Zoom capacity on PC with camera so some of our patient and public colleagues can attend virtually

Session 1 – How to make Patient and public involvement (PPI) an integral part of your research

By the end of the session participants will be able to:

• Discuss how an established PPI model can be used to engage patients and the public in both fundamental and applied science, and healthcare research
• Describe strategies to engage a diverse group of patients and the public in research and the outline the benefit of this collaboration
• Using the information provided, produce an initial plan to include patients and the public in your research with input from PPI representatives

Session 2 – Disseminating your research to a lay audience

By the end of the session participants will be able to:

• Explain the importance and rational for disseminating research findings to lay audiences e.g. patients, and public

• Discuss important considerations and appropriate strategies to disseminate findings from both fundamental and applied science, and healthcare research patients to lay audiences e.g. patients and the public
• Using the information provided, produce an initial plan to disseminate your research findings to lay audiences e.g. patients and the public

Each of the sessions starts with a patient and public involvement Ice breaker activity in mixed groups. This is followed by a presentation including PPI representatives (Session 1: Outlining a PPI engagement model, and examples of PPI involvement in healthcare and pharmaceutical science research; Session 2 :Outlining important aspects to consider and strategies to disseminate research results to lay audiences).

The third session is a workshop where participants draft a plan to include PPI representatives in their research (session 1) or lay audience dissemination plan (session 2) with input from the UCL School of Pharmacy team, PPI representatives and participants. The final session focuses on action planning. Individuals will write up to 3 SMART objectives to apply over the following 3 months.

• Duration: One Day
• Level: Basic
• Course leader: Isabel Braadbaart
• Institution: VU Amsterdam
• Teaching staff: Jacqueline van Muijlwijk
• Requirements: NA

An essential responsibility for PhD students is sharing knowledge and expertise through teaching and supervision responsibilities. But what is the best way to tutor students? This course will allow you to practice your teaching skills in mini-lessons and reflect critically on your role in higher education through group and whole-class discussions.

Topics to be covered include: creating a productive environment for learning, guiding experimental discussions, the function and purpose of assessment, and supporting novice problem-solvers.

At the end of the day you will have improved your conceptual knowledge of what good education is and you will have acquired some practical guidelines to support you own development as a teacher.

The day will begin with an introductory session where we learn about our different educational backgrounds and explore our existing beliefs about good educational practices. Next students will teach a brief, prepared lesson on a key concept in their discipline or research to a small group with the goal of promoting discussion. This will be followed by a group plenary and feedback. After lunch, we will dive deeper into the cultural differences in assessment practices, the goals of assessment, and what the research tells us about effective assessment practices that improve student learning.

Lastly, we will examine to role of the PhD student as an ‘expert’ and how we can support problem-solving strategies in novice learners. After a brief input, students will work in small groups to prepare another short lesson which they will teach to their peers, this time based on problem-solving. This will be followed by a plenary discussion and feedback, as well as a final closing activity.

• Duration: One Day
• Level: Basic
• Course leader: dr Albert J. Kooistra and prof dr Jacqueline E. van Muijlwijk-Koezen
• Institution: VU Amsterdam and University of Copenhagen
• Teaching staff:
• Requirements: We will use MOE, which will be provided during the course

This entry-level course will provide you with the knowledge and hands-on experience with chem(o)informatics tools to gain a better understanding of what happens at the interface between proteins and their ligands, i.e. the protein-ligand interaction space. The integration of protein structures, ligand structures, and their bioactivities allows you to provide a (structural) rationale for observed behavior.

During this course you will learn to:

• Search target-annotated chemical databases for ligand information and bioactivity data
• Derive essential features for target-binding based on ligand structures
• Search protein-structure databases
• Analyze protein-ligand complexes
• Explain the effects of site-directed mutations on ligand binding
• Use protein-ligand interaction analysis to perform docking studies
• And (if necessary for your protein target) create a homology model

During this course we will primarily use examples based on G protein-coupled receptors (GPCRs). However, there will also be plenty of time to directly apply the acquired knowledge to your own targets!

• Duration: Two Day
• Level: Advanced
• Course leader: Jörg P. Kutter
• Institution: Univ. of Copenhagen
• Teaching staff: Deirdre Cabooter and Soraya Chapel (both KULeuven)
• Requirements: Basic knowledge on chromatographic separations

The course will introduce the students to modern tools for pharmaceutical analysis with an emphasis on advanced liquid chromatographic techniques as well as methods exploiting the microfluidic format. These approaches offer new ways to perform bioanalytical procedures for pharmaceutically relevant problems.

The first part of the course will introduce state-of-the-art liquid chromatographic techniques and focus on novel chromatographic support materials from a fundamental point of view. An overview of methods that allow evaluating and improving the performance of these materials will be given. Subsequently, method development will be reviewed based on fundamental principles.

In the second part, two-dimensional liquid chromatography (2D-LC) techniques will be introduced as an alternative way to tune the selectivity of a separation. The motivations for doing 2D-LC will be explained and an introduction to the basic principles and main concepts of the technique will be given. The most common modes of implementation will be described (on-line or off-line, heart-cutting or comprehensive…). The most critical steps when setting up a 2D-LC experiment will be discussed. These include the selection of complementary separation modes and the choice of column dimensions and operating conditions. The students will learn about best practices for the development of successful 2D-LC methods. Finally, relevant examples of applications in the field of proteomics will be given.

In the third part, the students will hear about microfluidic formats to perform sample preparation and separation for applications in the pharmaceutical sciences. We will discuss advantages, possibilities and challenges of this format and see examples of how standard approaches can be improved or enhanced in the microchip format. Applications examples include chips to perform extraction of drug metabolites, chips including enzyme reactors for metabolism studies, chips for hydrogen-deuterium exchange to elucidate protein structure, chips as front-ends for small angle X-scattering to investigate protein structure, separation systems on chip, and coupling of chip devices to mass spectrometric detection.

• Duration: One Day
• Level: Basic-Advanced
• Course leader: Johan Heldin
• Institution: Uppsala University
• Teaching staff: Group members
• Requirements: Some basic immunofluorescence knowledge

The course focuses on methods for identifying protein interactions and posttranslational modifications (PTM), which has been developed in our lab, of which several are in the process/have been commercialized.

The course lets the students finish a proximity ligation assay using a brand-new kit from Navinci. During incubations, theoretical lectures covering conventual isPLA, proxHCR, UnFold/NaveniFlex, and molboolean will be given.

• Duration: One Day
• Level: Advanced
• Course leader: Natalja Genina
• Institution: University of Copenhagen
• Teaching staff: Assoc. Prof. Dr Pamela Robles Martinez (UCL), Prof. Sofia K. Sporrong (UU), Postdoctoral students from Prof. Christel Berstrom lab (UU)
• Requirements: Tinkercad (online web-based software)

The proposed course will give inside into the new advances regarding printed technologies in the pharmaceutical field. During the course, the participants will get familiar with the most common printing techniques used to produce medical devices and dosage forms.

The technicalities (formulation design, printing challenges, characterization techniques) regarding each printing technique would be discussed in the interactive manner. The students will actively participate during the course by doing scientific ‘battles’, answering the ‘Millionaire’ questions and working in a groups to make a ‘tender’.

The students will also get hands-on experience by designing their pharmaceutical dosage forms in the computer-aided design software (Tinkercad) by working on a case study. Most importantly, the participant will have a chance to visit the local laboratory at Uppsala University and see the printers in action and have a chance to print their design dosage form. Before the course, the participant will get a short video (made by the organizer) to have a smooth start during a course. 

• Duration: One Day
• Level: Basic
• Course leader: Rik Gijsbers
• Institution: KULeuven
• Teaching staff: -
• Requirements: Basic molecular biology, biochemistry

The idea that the introduction of exogenous genetic material could serve as an effective treatment for inherited disorders was hypothesized by visionary scientists more than 50 years ago. Initially, this strategy was envisioned to provide a functional version of a gene to replace a non-functional one, and that was the underlying cause of the disease the patient was suffering from.

Over the past decades, gene therapy has evolved from science fiction into clinical reality with several products reaching the market providing new treatment options for otherwise untreatable patients in multiple fields of medicine, offering the potential of a durable and possibly even curative clinical benefit for the patient.

Previously untreatable diseases such as Spinal Muscular Atrophy, sickle cell disease, ADA-SCID, and others can now be cured or at least halted with single intravenous injections of gene therapy vectors or viral vector corrected cells. In the years to come, gene and cell therapy (and ATMPs in general) will become an intrinsic part of our standard armamentarium to treat human disease.

In this lecture, we will discuss some of the technologies underpinning the silent revolution of the past few decades, we will review the critical discoveries and milestones in the development of gene therapies, and discuss some of the success stories currently available on the market. In addition, we will touch upon the current portfolio of gene therapeutic tools, and where challenges still lie (manufacturing, biosafety, payment models, ethics).

• Duration: One Day
• Level: Basic
• Course leader: Prof. Matthias Barz
• Institution: Leiden University
• Teaching staff: To be confirmed
• Requirements: Background knowledge in chemistry, cell biology, immunology, pharmacy

Nanomedicine is defined as the use of nanotechnology in medicine, which covers a broad spectra of in vivo and ex vivo applications ranging from diagnosis to therapeutic interventions. Over the last decades nanomedicine has already started to change the standard procedures how patients are diagnosed or treated.

Besides a general introduction to the concept of nanomedicine, we will focus on the use of nanoparticles in diagnosis and therapy, which made it to clinical realty or advanced stages of clinical development. Thus, we will address liposomes and lipid formulations, therapeutic antibodies and antibody drug conjugates, polymeric drugs, polymeric drug delivery systems and nanoparticle based diagnostics/theranostics.

• Duration: One Day
• Level: Basic
• Course leader: Ola Spjuth
• Institution: Uppsala University
• Teaching staff: To be confirmed
• Requirements: Students are expected to bring their own laptops. Internet connection required (Eduroam or UPUNET works fine) – The free software Orange (https://orangedatamining.com/) should be installed.

This course gives an introduction to artificial intelligence and machine learning and its applications in drug discovery and development, including challenges and opportunities.

The morning session consists of a 2h lecture on basic concepts and methods for AI/ML mixed with different areas of drug discovery where these methods can be applied.

The afternoon session consists of a 2h hands-on exercise where students will get introduced to the Orange data mining software and will carry out basic exercises for unsupervised and supervised learning on data from drug discovery projects.

• Duration: One Day
• Level: Basic
• Course leader: Erik Sjögren
• Institution: Uppsala University
• Teaching staff: Ilse Dubbelboer
• Requirements: Laptops with OSP suite installed (https://github.com/Open-Systems-Pharmacology/Suite/releases/tag/v10.0)

This is an introductory course to familiarize yourself with the Physiologically based pharmacokinetic (PBPK) methodology and its usefulness in drug development via lectures and basic hands-on exercises using the PBPK platform PK-Sim^®.

PBPK models are quantitative descriptions of the absorption, distribution, metabolism and excretion (ADME) of drugs in the organism based on interrelationships between key physiological, biochemical and physicochemical determinants of these processes. This approach for mechanistic in silico modeling is used across the phases of drug development from early discovery and phase-0 upto late life cycle management and clinical use.

Key elements of PBPK is a clear distinction between organism and the entities of investigation as well as mechanistic representations of processes relevant for drug disposition and pharmacokinetics. This allows for translations and quantitation of intrinsic and extrinsic factors influencing drug pharmacokinetics in both healthy volunteers, patients and special populations.

• Duration: One Day
• Level: Basic
• Course leader: Ali Makky
• Institution: Université Paris-Saclay
• Teaching staff:
• Requirements:

Nanomaterials responsible to external stimuli are considered nowadays as one of the most promising nanoparticulate systems with multifunctional properties. Such nanomaterials can be used in imaging applications and/or for the treatment of several diseases including cancer and localized resistant bacterial infections.

Different external stimuli for the development of smart nanomaterials have been reported in the last few years. This includes magnetic field, electrical field, temperature, ultrasound and light. Among these stimuli, the light appeared to be the most reliable one for conceiving such responsive nanomaterials.

Indeed, light-responsive materials can (i) exert a direct therapeutic effect such in the case of photothermal (PTT) and photodynamic therapies (PDT); (ii) allow the delivery of therapeutic agents in a spatiotemporal manner at a specific target; (iii) offer imaging possibilities of the treated organs by fluorescence or photoacoustic mechanisms. In this course, we will focus on (i) the recent developments with light responsive nanomaterials within the last 10 years, (ii) the formulation requirements for their successful design, (iii) their applications in vitro and/or in vivo for cancer therapy, antimicrobial therapy and imaging (iv) their limitations and the future prospects for their successful clinical translation.

• Duration: One Day
• Level: Basic
• Course leader: Jordi Carreras-Puigvert
• Institution: Uppsala University
• Teaching staff: Rikard Nyström
• Requirements: NA

This 2-block course describes the use of high content screening for drug discovery, and introduces the students to automated liquid handling.

The course includes:

1) lecture about the steps, processes and approaches needed for drug discovery with a focus on high content screening,

2) a hands-on workshop on designing a broad purpose pipetting protocol for life sciences applications using liquid handling instrumentation.

The participants will learn about current image-based drug discovery techniques, from assay development to model selection. Aspects of clinical testing and precision medicine using high content imaging will also be addressed. At the end of the course, participants should have a good overview and understanding of how high content imaging can aid drug discovery efforts, allowing them to pinpoint potential career directions for their own scientific paths.

The participants will also have the opportunity to learn how to use liquid handling instrumentation for automated pipetting, a tool widely used in modern laboratory setups and industry.

The main blocks included in the lecture are:

• General screening considerations,
• Models for screening (2D, 3D and Primary cells),
• Image-based strategies for screening   (Unbiased assays (Cell Painting) and Biology-directed assays),
• Image and Data Analysis (Traditional approaches, AI guided, Data storage considerations, Analysis methods suitable for large datasets (eg KNIME, R, Orange) and Data visualization) and 5) Target identification for phenotypic screens and follow-up (Chemical Proteomics and target ID, Genetic approaches and Transcriptomics). For the hands-on automation lab, the students will learn how to design and execute automated pipetting protocols using instruments from Flow Robotics.

• Duration: One Day
• Level: Basic
• Course leader: Prof. Gabriele Costantino
• Institution: University of Parma
• Teaching staff: -
• Requirements: NA

Skin is a complex organ which manages the relationships between us and the exterior world. Skin is not only a physical barrier but also a dynamic sensory apparatus which cross-talks with the brain and with the microbiota.

Recently, a lot of attention is given to functional cosmetics, or cosmeceuticals, which are intended to contribute to the one’s own wellbeing and wellness through a combination of biomimetic formulations, active ingredients and matching with individual expectation. The science of skincare is therefore acquiring a particular interest with respect to both the potential for contributing to the preservation of a healthy and pleasant state and the potential for market growth.

This course, for which a basic level of knowledge is required, aspires to help students in developing innovative concepts and operational instruments for a better valorisation of their professional skills as pharmacists, in the consideration of the increased role that functional cosmetics will play in the close future in both community pharmacies and pharmaceutical industry.

 The following topics will be covered:

• the science of beauty and the beauty of science
• the skin, the skin-brain-gut axis and health preservation
• cosmetics and functional cosmetics: regulatory issues and market opportunities
• neurocosmeceuticals: understanding the growing landscape of functional cosmetics
• biochemical pathways to pleasantness
• bioprospecting towards new ingredients for skincare

• Duration: One day
• Level: Basic
• Course leaders: Susanne Bredenberg (UU Innovation)
• Institution: UU Innovation and Drivhuset
• Teaching staff: Olivia Tolan (UU Innovation), Philip Gratell (Drivhuset)
• Requirements: NA

There is a growing demand for entrepreneurial skills, both within research and in business. Innovation stems from a challenge: an unmet need prompts an idea. If that idea for a product, method, or service arrives at the right time, and is thoughtfully developed, it can advance science, business, even society. Pharmaceutical innovations in particular are nearly always characterised by strategic use of intellectual property protection. So in this course we will combine the subjects of innovation and intellectual property.

The course will be organised in four sections. In the first section you will be challenged to think differently and to think like a designer using the mindset of "Design Thinking". This mindset can transform the way we develop products, services, processes, and strategies, but also in research when tackling quandaries. The mindset can be applied to any field and uses abductive reasoning and iterative exercises to innovate based on reality.

The next section is starting with a presentation of the NABC model, Need – Approach – Benefit – Competition. The NABC model originated in Stanford Research Institute (SRI) as a way to develop ideas for innovative projects. Its main focus is the value proposition: what is the added value of the idea for the client/customer – putting the audience in the heart of development. Then it is everyone’s turn to practice their pitch skills using the NABC model in interactive, smaller groups.

Finally we will guide participants through the field of intellectual property and demonstrate how to access vast amounts of technical and commercial information online using free databases.

At the end of the course, participants should have an understanding of challenged based learning and the NABC model and how it can help you to bring clarity and to organise your ideas and practical skills to use when pitching ideas or projects - whether they be scientific, commercial, or both. Furthermore, participants should have an overview of intellectual property and how different types of rights can be used to build or retain value in innovations. They will also have experimented with locating information in an intellectual property database.

• Duration: One Day
• Level: Basic
• Course leader: Erik Jacobsson
• Institution: Uppsala University
• Teaching staff: -
• Requirements: NA (Maximal number of students 20)

The biopharmaceutical market has grown tremendously in the last years with 10-12 of the top 20 drugs by retail sales being biologicals. To produce these drugs in larger quantities, expertise in bioprocessing (growing cells in bio-reactors, harvesting and purification of the product) is necessary. In this introductory course to bioprocessing we have invited specialists from Cytiva (former GE-healthcare) and Testa Center to discuss some of the key elements in the bioprocessing workflow.

In addition to the lecture, we will take you on study visit to Testa Center. Testa Center is a private-public owned “test bed” for bioprocessing located at the Cytiva site in Uppsala. At Testa Center small (and big) companies and the academia can test, scale up and develop bioprocesses in a factory-like environment and build know-how and value into the projects. Testa Center is also utilized as an education facility for professionals and universities in order for students to meet the increasing need for bioprocess specialists.

Testa Center harbours a unique instrumentation park, not commonly found in university accessible laboratories, with the ability to grow cells at a scale from 1 to 500 L, harvest and purify the products produced.

• Introduction to key elements in bioprocessing
• Study visit to Testa Center/Cytiva

Duration: One day
Level: Basic
Course leaders: Ben Boyd
Institution: University of Copenhagen
Teaching staff: Professor Alex Bunker (UHelsinki), Professor Camilla Foged (UCPH), Professor Thomas Rades (UCPH)
Requirements: NA

This course is targeted towards starting/early stage PhD students, to provide an opportunity to learn non-technical but critical concept that will help students to make the most of their PhD. The course instructors have graduated more than 200 PhD students collectively, and their wisdom on organisation, communication, publication, networking and conferences, and finalising the synopsis/defence will prove invaluable for students that attend this course.

Session 1
10.45-10.50      Introduction to course (BB)
10.50-11.30      Getting off to a flying start – organisation beats motivation (TBD)
11.30-12.15      Publication and writing (TR)

12.15-13.45      Lunch

Session 2
13.45-14.30      Scientific communication and dissemination (CF)
14.30-15.15      Importance of networking & how to handle conferences (BB)

15.15-15.30      Break

Session 3
15.30-16.15      Finalising your thesis: writing a great synopsis & gunning the defence (AB)
16.15-16.45      Perspectives from late stage students (AB’s student)
16.45-17.00      Final comments (BB)