PROGRAMME SUMMARY
The European Rare Diseases Research Alliance (ERDERA) has been established to further help in coordinating the research efforts of European, Associated and non-European countries in the field of rare diseases and implement the objectives of the International Rare Disease Research Consortium (IRDiRC). These actions follow the five Joint Transnational Calls for rare diseases research projects launched previously by the European Joint Programme on Rare Diseases (EJP RD) since 2019. The call will open on 10 December 2024.
Aim of the Call
A number of national and regional funding organisations intend to participate in the ERDERA Joint Transnational Call (JTC) 2025 to fund research projects on rare diseases. The call is supposed to open simultaneously with the involvement of the following funding organisations in their respective countries/regions.
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 the expected impact being future use of the results to benefit patients.
Projects will focus on 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. Applicants are encouraged to assemble groups of rare diseases based on relevant criteria and commonalities if this leverages added value in sharing resources or expertise.
Topic: “Pre-clinical therapy studies for rare diseases using small molecules and biologicals – development and validation”
Topics list
Research studies on therapies using small molecules, small non-coding chemically synthesized nucleic acid-based therapies, repurposed drugs or biologicals (e.g., antibodies or proteins such as enzymes, immune modulators or growth factors etc.). Proposals must cover at least one of the following areas:
development of novel therapies in a preclinical setting through cell, organoid and animal model studies, molecule screening or use of in silico or artificial intelligence models
- development of predictive and pharmacodynamics biomarkers correlated to the efficiency of the therapy in a preclinical setting that could serve as surrogate endpoints
- replication of pre-clinical studies in an independent lab to increase validity of exploratory findings
- pre-clinical proof of concept studies for evidence of pharmacological activity in vitro and in vivo, pharmaco-kinetics and pharmaco-dynamics of the drug and first toxicology and safety data
- studies to support readiness for initiating clinical trial authorization conforming to regulatory requirements
Translatability into humans should be the key focus of the project, and applicants should demonstrate access to relevant scientific or regulatory expertise (e.g., through innovation task forces or competent national authorities).
Excluded approaches and topics
The following approaches and topics are excluded from the scope of the JTC 2025:
ATMP therapies (gene therapy medicinal product (including mRNA-based therapies), somatic cell therapy medicinal product, tissue engineered product, according to EMA definition).
- Development of new cell/organoid/animal models, which should already be established.
- Set-up or extension of natural history studies / patient registries.
- Interventional clinical trials to prove efficacy of drugs/treatments/surgical procedures/medical procedures. This includes studies comparing efficacy, e.g., two surgical techniques or therapies, and projects whose main objective is the implementation of a clinical phase IV pharmacovigilance study.
- Projects focusing only on rare neurodegenerative diseases that are within the focus of the Joint Programming Initiative on Neurodegenerative Disease Research (JPND). These are: Alzheimer’s disease and other dementias; Parkinson’s disease (PD) and PD-related disorders; Prion diseases; Motor Neuron Diseases; Huntington’s disease; Spinal Muscular Atrophy and dominant forms of Spinocerebellar Ataxia. Interested researchers should refer to the relevant JPND calls. However, childhood dementias/neurodegenerative diseases are eligible.
- Rare infectious diseases, rare cancers and rare adverse drug events in treatments of common diseases. Rare diseases with a predisposition to cancer are eligible.
FUNDING SPECIFICS
The FNR supports Luxembourg partners in this Call with a maximum of 300,000 EUR.
APPLICATION INFORMATION
Submission
Both pre- and full proposals must be written in English and must be submitted to the Joint Call Secretariat (JCS) by the coordinator through the electronic submission system exclusively. Specific information to follow.
In addition, Luxembourg partners must submit both pre and full proposals, along with the FNR INTER Call documents to the FNR no later than 7 working days after the EJP-RD deadline. Applications to the FNR must be submitted via the FNR’s Online Grant Management System.