Transnational Research Projects for Innovative Therapeutic Approaches for Rare Diseases
Muscular Dystrophy Canada is partnering with the Canadian Institutes of Health Research in the ninth E-Rare joint call for funding multilateral research projects on rare diseases (JTC2017). The aim of the call is to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with a clear translational research approach. Projects shall involve a group of rare diseases or a single rare disease following the European definition i.e. a disease affecting not more than five in 10,000 persons in the European Community, EC associated states and Canada.
The specific objective of this call is to promote research projects focusing on the pre-clinical development of therapeutic approaches in suitable existing animal or cell models.
Therapeutic approaches can include:
- Cell-based therapy (e.g. somatic cell therapy, cell-based regenerative medicine, tissue engineering, therapies based on combination of cells with scaffolds or substrates, etc.);
- Gene therapy (e.g. transfer of nucleic acids for therapeutic purposes including DNA, RNA, oligonucleotides, etc.);
- Pharmacological therapy (e.g. use of chemicals or biopharmaceuticals including repurposing approaches, high throughput screening of molecules, etc.).
- Project proposals must clearly demonstrate the potential health impact as well as the added-value of transnational collaboration: gathering a critical mass of patients/biological material, sharing of resources (models, databases, diagnosis etc.), harmonization of data, sharing of specific know-how and/or innovative technologies, etc.
- Each transnational collaborative project should represent the critical mass to achieve ambitious scientific goals. Consortia are encouraged to demonstrate engagement with industry for its active participation including areas of collaboration, sharing of resources, capabilities and expertise, in order to ensure an efficient transfer of pre-clinical results into clinical utility. Likewise, patient organizations are invited to participate where appropriate as their engagement has the potential to provide new insights that could lead to innovative discoveries, and ensures that research is relevant to patients’ concerns.
The following approaches are excluded:
- Therapeutic approaches concerning rare infectious diseases, rare cancers and rare adverse drug events in treatments of common diseases;
- Interventional clinical trials or any other interventional treatment of patients within the project (e.g. compassionate use)
- The set-up of new patient registries/databases
- Development of new cell or animal models. The relevant cell or animal model must be already established for the purpose of the project;
- Surgery or radiation therapies.