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incPRINT as a platform for RNA-targeted drug discovery

Project description

Novel technology assists the discovery of RNA-targeting drugs

RNA molecules are master regulators of many biological processes, and emerging evidence underscores their role in disease. However, using small molecules to target RNA therapeutically has proven technologically challenging. To address this problem, scientists of the EU-funded incPRINT-DRUG project propose to use their high-throughput RNA–protein interaction technology designed to identify small molecules that target specific pathogenic RNA–protein complexes. This technology simultaneously assesses the efficacy, specificity and toxicity of drugs in living cells. The aim of the incPRINT-DRUG project is to adapt this tool for RNA drug discovery and contribute to the biomedical field.

Objective

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.

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Coordinator

INSTITUT CURIE
Net EU contribution
€ 150 000,00
Address
Rue d ulm 26
75231 Paris
France

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Region
Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
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Other funding
€ 0,00

Beneficiaries (1)