RNA Epithelial Approach for Crohn’s Treatment

Description du projet

Thérapie à base d’ARN pour la maladie de Crohn

La maladie de Crohn (MC) est une affection inflammatoire chronique associée à une détérioration de la muqueuse intestinale. Les traitements conventionnels se concentrent sur la suppression du système immunitaire mais ne favorisent pas la guérison des muqueuses et une rémission durable. Il est nécessaire d’élaborer des stratégies qui ciblent directement et restaurent la cause sous-jacente du dysfonctionnement épithélial. Pour résoudre ce problème, le projet RE-ACT, financé par le CER, propose une thérapie très innovante basée sur de longs ARN non-codants (ARNlnc) encapsulés dans des nanoparticules lipidiques. Cette approche s’appuie sur la découverte récente que les patients atteints de MC présentent une dysrégulation des ARNnc. Les chercheurs entendent évaluer l’efficacité de ce traitement innovant et préparer sa traduction clinique. Cette stratégie pourrait constituer une alternative plus sûre et plus efficace aux thérapies existantes contre la MC.

Objectif

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.