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RNA Epithelial Approach for Crohn’s Treatment

Project description

RNA-based therapy for Crohn’s disease

Crohn’s disease (CD) is a chronic inflammatory disorder associated with a damaged gut lining. Conventional treatments focus on immune system suppression but do not promote mucosal healing and sustainable remission. There is a need for strategies that directly target and restore the underlying cause of epithelial dysfunction. To address this issue, the ERC-funded RE-ACT project proposes a highly innovative therapy based on long non-coding RNAs (lncRNAs) encapsulated in lipid nanoparticles. This approach builds on the recent discovery that patients with CD exhibit lncRNA dysregulation. Researchers aim to evaluate the efficacy of this innovative treatment and prepare for clinical translation. This strategy has the potential to serve as a safer and more effective alternative to existing CD therapies.

Objective

RE-ACT aims to translate groundbreaking ERC-funded research on lncRNA-based therapy for Crohn's Disease (CD) into a commercially viable treatment option. Our innovative approach targets the gut epithelial lining using lncRNA encapsulated in lipid nanoparticles (LNPs), addressing a critical gap in current CD treatments which primarily focus on immune suppression. By restoring epithelial function, we aim to achieve better mucosal healing and long-term patient outcomes. Building on our discovery of widespread lncRNA dysregulation in CD patients, we will conduct comprehensive preclinical studies to validate the efficacy and safety of our lncRNA-LNP therapy. Our work will involve both ex-vivo studies using human cell models and in-vivo studies in mouse models of epithelial injury and gut inflammation, focusing on optimizing delivery, assessing therapeutic effects, and evaluating potential systemic impacts. Concurrently, we will develop a robust IP strategy to protect our innovation, conduct thorough market assessments to understand the commercial landscape and engage in targeted business development activities to secure partnerships and funding for future clinical development. Our integrated approach combines scientific rigor with commercial acumen, maximizing the potential for successful translation from laboratory to clinic. The project addresses a significant unmet medical need in CD treatment, potentially offering a more effective and targeted therapy with fewer systemic side effects. If successful, our lncRNA-based approach could not only improve outcomes for CD patients but also pave the way for similar therapies in other gastrointestinal disorders. By combining cutting-edge science with strategic commercial development, this project represents a significant step towards addressing the limitations of current CD treatments and improving the lives of millions affected by this challenging condition.

Fields of science (EuroSciVoc)

Programme(s)

Topic(s)

Funding Scheme

HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants

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Call for proposal

(opens in new window) ERC-2024-POC

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Host institution

MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER
Net EU contribution
€ 150 000,00
Total cost
No data

Beneficiaries (1)