Projektbeschreibung
Neue Technologie soll Entdeckung von Wirkstoffen mit RNA-Target erleichtern
RNA-Moleküle sind die Hauptregulatoren vieler biologischer Abläufe und immer mehr Studien belegen ihre zentrale Rolle bei Krankheiten. Es gab Versuche, RNA mit niedermolekularen Verbindungen als therapeutische Angriffspunkte zu nutzen, doch diese erwiesen sich technologisch als schwierig. Darum stellt ein Forschungsteam im EU-finanzierten Projekt incPRINT-DRUG jetzt seine Hochdurchsatztechnologie für RNA-Protein-Interaktionen vor. Sie soll diejenigen niedermolekularen Verbindungen finden, die auf spezifische pathogene RNA-Protein-Komplexe abzielen. Die Technologie bewertet gleichzeitig die Wirksamkeit, Spezifität und Toxizität von Wirkstoffen in lebenden Zellen. Ziel des Projekts incPRINT-DRUG ist es, die Methode für die Entdeckung von RNA-Wirkstoffen anzupassen und so einen Beitrag zur Biomedizin zu leisten.
Ziel
RNA transcripts have emerged as key regulators of diverse biological phenomena and are implicated in many human diseases including neurodegenerative disorders, cancers, viral infections and antibiotic resistance. As such, RNA represents an untapped source of therapeutic targets. Bioavailable small molecules have been recently successfully applied to target RNA. One caveat, however, is that lead compounds are not easily designed from RNA sequence or structure and therefore targeting RNA by small molecules remains challenging. One of the strategies to target RNA with small molecules is to target RNA-protein interactions or RNA elements stabilized by protein binding. However, the unbiased in vivo identification of small molecules that selectively target RNA-protein interactions with little cellular toxicity remains unachieved due to the lack of technology. In the incPRINT-DRUG project, we will adapt our innovative high-throughput RNA-protein interaction technology for unbiased identification of small molecules that target specific pathogenic RNA-protein interactions. Our unique technology enables drug testing in living cells combined with the simultaneous testing of drug specificity and toxicity. Our core technology has been extensively validated over the past years and proved to be robust to be optimized and adapted for the RNA drug discovery. Our business development strategies will follow several possible scenarios: (1) license the technology, (2) generate new IP with the technology and license the IP to industrial partners, (3) industrial partnership by providing service to biotech and pharma companies. All three options will be explored with our Technology Transfer & Industrial Partnership Office. incPRINT-DRUG is expected to have a strong biomedical and economic impact given the growing interest in finding small molecules targeting pathogenic RNAs and the technological gap to achieve it using data-driven experimental methods.
Wissenschaftliches Gebiet
- medical and health sciencesbasic medicinepharmacology and pharmacydrug discovery
- natural sciencesbiological sciencesbiochemistrybiomoleculesproteins
- medical and health sciencesclinical medicineoncology
- natural sciencesbiological sciencesgeneticsRNA
- medical and health sciencesbasic medicinepharmacology and pharmacydrug resistanceantibiotic resistance
Programm/Programme
Thema/Themen
Finanzierungsplan
ERC-POC - Proof of Concept GrantGastgebende Einrichtung
75231 Paris
Frankreich