Operating Grant : E-Rare-2: Innovative Therapeutic Approaches (2014)

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.

The specific objective of this call is to promote the development of innovative therapeutic approaches for rare diseases. In order to achieve this goal, the necessary expertise and resources should be brought together from academia, clinical/public health sector and private companies.

Projects shall involve a group of rare diseases or a single rare disease following the European definition (a seriously debilitating and/or life-threatening disease affecting not more than 5 in 10.000 people). Rare infectious diseases, rare cancers and rare adverse drug events in treatments of common diseases are excluded from the scope of the call.

The research projects have to focus 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 combinations 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 following approaches and topics are excluded from the scope of the call:

• Development of new cell or animal models. The relevant cell or animal models do already need to be established for the purpose of the project
• Interventional clinical trials or any projects enrolling patients
• Therapeutic approaches concerning rare infectious diseases, rare cancers and rare adverse drug events in treatments of common diseases
• Surgery or radiation therapies