FCT PhD Programme i3DU
Course: Biomarkers and Assay Development

18 June 2018

5 days
Faculdade de Farmácia, Universidade de Lisboa
Amphitheatre B

The FCT PhD Programme in Medicines and Pharmaceutical Innovation (i3DU) trains students in target discovery, drug design, pre-clinical development, and drug safety, bridging the translational gap from discoveries on disease targets and mechanisms into novel diagnostic and therapeutic agents.

The i3DU Programme offers a training course on Biomarkers and Assay Development that will be held at the Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, in Lisbon, 18-22 June 2018.

This course is designed to cover principles and applications of biomarkers and assay development, from identification to validation, to impact in drug discovery, and disease diagnosis and prognosis.

We welcome the participation of external academic and scientific community members. Registration is free but mandatory.

Organizing committee at iMed.ULisboa/FF

Cecília Rodrigues (Coordinator)
Adelaide Fernandes
Elsa Rodrigues
Paula Leandro
Rui Castro
Susana Solá

Course outline

Biomarkers are now an integral part of the drug discovery and development process, acting as indicators of mechanism of action, efficacy, safety and disease progression, as well as assisting in diagnosis, and patient selection and design of clinical trials. Biomarkers also offer the potential to inform treatment decisions and bring personalized medicine into clinical practice.

In early stage drug discovery, biomarkers are used to validate in vitro target modulation. As projects progress, biomarker assays are developed for pharmacokinetic/pharmacodynamic (PK/PD) models, profiling molecules prior to testing in disease models as initial proof of concept. PK/PD models can also assist in dose-to-man scaling predictions for use in clinical trials. The biomarker assays developed during the in vitro discovery phases are frequently used as efficacy or toxicity endpoints in the clinic. Clinical trials, particularly in oncology, are frequently designed around these biomarkers, in addition to biomarkers of disease progression. In contrast, less evidence exists to recommend the use of any biomarker of pathological processes in neurodegenerative diseases, opening the door for conducting future disease progression biomarker studies.

Goals and Learning Outcomes

• Understand the development of both efficacy and translational biomarker assays in cell lines, primary cells and tissues;
• Explore formats ranging from nucleic acids, protein markers and signaling pathways to cellular phenotypic changes;
• Apply on-target cellular readouts to support medicinal chemistry optimization projects;
• Use a range of assays for pharmacodynamic models to determine dosing regimens, and in disease models to correlate target coverage with disease-modifying effects, including functional readouts showing inhibition of target function.


The course is divided into a balanced blend of lectures on theoretical, practical and laboratory case-based discussions presented during a dedicated course with limited attendance. The training programme has specific slots allocated to seminars and workshops, including informal discussions with lecturers.

Early discovery requires broad expertise in biomarker identification and assay development across many therapeutic areas. We will focus on oncology, central nervous system, and metabolic diseases and address the development of quantitative assays in primary or immortalized cells, stem cells or induced pluripotent stem cells, or disease tissue that can be used as pharmacodynamic or disease models. Ultimately, these models will act as efficacy and translational markers from the in vivo phase to the clinic. A broad range of endpoints can be employed to support biomarker selection, including genomic, proteomic, phosphoprotein and epigenetic markers. Assay technologies will be discussed.