RNA Epithelial Approach for Crohn’s Treatment

Descrizione del progetto

Terapia a base di RNA per la malattia di Crohn

La malattia di Crohn (CD) è un disturbo infiammatorio cronico associato a un rivestimento intestinale danneggiato. I trattamenti convenzionali si concentrano sulla soppressione del sistema immunitario ma non promuovono la guarigione della mucosa e una remissione sostenibile. Servono strategie capaci di affrontare direttamente e risolvere la causa alla base della disfunzione epiteliale. Per affrontare questo problema, il progetto RE-ACT finanziato dal CER, propone una terapia altamente innovativa basata su RNA lunghi non codificanti (lncRNA) incapsulati in nanoparticelle lipidiche. Questo approccio si basa sulla recente scoperta che i pazienti con CD presentano una disregolazione degli lncRNA. I ricercatori intendono valutare l’efficacia di questo trattamento innovativo e prepararsi alla traduzione clinica. Questa strategia ha il potenziale per essere un’alternativa più sicura ed efficace alle terapie esistenti per la CD.

Obiettivo

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.