SMIM23004U Biotherapeutics Drug Development

The scientific and commercial prominence of biotherapeutics is beyond dispute. Molecules obtained from expression systems, (semi)synthetic macromolecules as well as biological material intended for therapy or prophylaxis require specialization on all levels to overcome the great challenges andcomplexity in regard to pharmaceutical formulation design, technological processing, and assessment in the process of developing and regulating such novel safe medicines. In this course, leading experts in the field will present selected topics and case-based approaches will be used to introduce examples of some of the newest advances related to different parts of the drug development process.

This course provides an overview of the drug development process and highlights crucial aspects of specific importance for the capability to develop biologically active macromolecules like peptides, antibodies, oligonucleotides, and other biological material such as living organisms and stem cells into effective medicines.

At first sight, utilization of biotherapeutics as drugs appears straightforward since the pharmacological activity of endogenous compounds such as hormones and coagulation factors are given. Avenues of oligonucleotide and cell therapy may seem less obvious, but due to tremendous advances in understanding disease biology, new targets for biotherapeutics are continuously identified, and this overall excels novel opportunities for new and improved products. Provided that the potent macromolecules or other biological material is druggable. This requires efficient formulation designs and novel delivery approaches, which are continuously emerging. Also, due to the structural complexity and instability of the active material, drug development requires specific processes and models for efficacy testing and safety assessment.

Course content

The course will address and highlight topics of relevance for the design and development of biotherapeutics. Experts from selected relevant fields will lecture on and discuss both fundamental concepts and specific selected issues related to biotherapeutics drug development. Topics of special attention are picked from the discovery phase through development and assessment, and include e.g. molecular engineering, pharmaceutical drug design and formulation, experimental models, efficacy and safety assessment and regulatory aspects. Case studies will form the basis for interactive discussion sessions.