Pre-clinical therapy studies for rare diseases using small molecules and biologicals – development and validation ERDERA 2025

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:

1. 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 ;
    
2. development of predictive and pharmacodynamics biomarkers correlated to the efficiency of the therapy in a preclinical setting that could serve as surrogate endpoints
    
3. replication of pre-clinical studies in an independent lab to increase validity of exploratory findings
    
4. 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
    
5. 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).

ERDERA is holding an event to officially present the European Partnership on Rare Diseases, a groundbreaking initiative aimed at transforming the lives of 30 million rare disease patients across Europe and beyond. The event will take place online on 28 October 2024, from 14:00 to 16:15 CET.

Be Part of a Major Milestone in Rare Disease Research: Join ERDERA’s Launch Event! - ERDERA