A call for European projects on innovative therapeutic approaches for rare diseases

The French National Research Agency will launch, in partnership with 16 countries, the ninth E-Rare joint call for funding multilateral research projects on rare diseases on “Innovative therapeutic approaches for rare diseases”.

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.

Towards innovative therapeutic approaches

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.).

The research projects submitted within this call must be based on novel ideas stemming from consolidated previous results and must be clearly endowed with a strong translational research orientation, i.e. bench to bed studies allowing a rapid implementation into public health-related decisions or into the clinics.

The use of existing European health research infrastructures or initiatives is strongly encouraged when appropriate. The following European Research Infrastructures or Initiatives were identified as potentially useful for this kind of study: BBMRI; EATRIS; ECRIN; ELIXIR; EU-OPENSCREEN; INFRAFRONTIER; RD-Connect and European Medicines Agency.

Submission and evaluation of projects will be carried out in two stages and integrated with other countries. Each country shall fund its national teams according to its own procedures.

The deadline for proposal pre-registration is February 1st, 2017.
The deadline for proposal submission is June 2nd, 2017.