Summary of CIHR Training Grant in Biological Therapeutics

In the context of this program, biological therapeutics is broadly defined as encompassing nucleic acids such as small interfering RNAs (siRNA) and short hairpin RNAs (shRNA), viruses and viral vectors, aptamers, peptides and proteins including antibodies. The focus is on the study and development of macromolecule based drugs rather than small molecule based drugs, the latter of which has traditionally been the main supply pipeline for new drug entities. The first long term objective of this strategic training program is to bring together leaders in the fields of biological drug development in the Toronto research core to address a rapidly growing yet unmet need, namely to train young scientists in the design, development, and delivery of biologicallybased medicines. A second objective is to foster university and industrial collaborative research and provide a trans-disciplinary training environment. The research focus is on drug target validation, drug development, and therapeutics, (but not biomarkers). Research into novel methods for delivering biologically based drugs to target organs in animal models and humans is also be an integral aspect of this training program. A third objective is to provide the necessary communication skills needed for trainees to succeed as independent researchers in the study of biologically targeted drug therapy.

This training program encompasses three major therapeutic areas of application: neuroscience/CNS drugs, cancer, and infection and immunity. Within these areas of application, the focus is  on three key parameters: Design, Development and Delivery of biologically based medicines. The 14 researchers in this training program have laboratories based at the University of Toronto, Ontario Cancer Institute, MaRS, the Toronto Western Research Institute, and Toronto General and St. Michaels Hospitals. Postdoctoral Fellows (PDFs) and graduate students will receive training in techniques in bioinformatics, the use of RNAi, new methods and formulations for the delivery of nucleic acids and proteins (e.g. liposome packaging of siRNA), and the analysis of the metabolism and fate of these macromolecules (pharmacokinetics) in in vivo animal models. The collaborative environment inherent in our training program will (a) provide a deeper level of knowledge and substantially enhance the technical skills of the students and PDFs, (b) facilitate the ability of the trainees to work collaboratively together and promote team oriented research with colleagues, (c) greatly increase the exposure of each trainee to cross-disciplinary research in the fields of neuroscience, cancer, immunology, and drug delivery, and (d) through an industrial internship program, provide an opportunity to work temporarily in the private sector where they will acquire additional technical skills and potentially make connections for future job placements.