The GENEGUT project will transform the treatment of ileal CD by developing a first-in-class oral RNA-based therapy, tackling inflammation locally in the intestinal tissue, while avoiding systemic side effects.
Despite advances in new immune modulators and biological treatment, up to 30% of patients become non-responders. Besides, intestinal fibrosis is a significant challenge in Crohn’s Disease with limited efficacy of current drugs. And lastly, despite all the advances in diagnosis, there has been no significant decrease in mortality in patients with Crohn’s Disease over the last several decades. This highlights how urgently new therapies are needed as an alternative to surgical interventions and to avoid the associated complications of ileal resection.
The GENEGUT consortium, in which EFCCA is a partner, is determined to push beyond the state of the art in RNA-based therapies: “Crohn’s Disease, as a highly prevalent disease with an unmet need for targeted treatment, is an ideal candidate for which to develop a safe, effective, targeted treatment exploring non-invasive routes of administration, such as oral delivery.”
The GENEGUT approach
Over the course of four years, GENEGUT aims to develop a non-invasive, safe, effective, and targeted treatment that will be realised in the form of a first-in-class, orally administered RNA-based therapy.
This breakthrough RNA therapy will be developed using complementary technologies that selectively target immunomodulatory pathways within inflamed intestinal cells that reside in specific regions of the small intestine. The delivery of RNA will be enabled by a combination approach where novel biomaterials, designed to overcome the barriers in the gastrointestinal tract, are synthesised into nanoparticles (NP) which encapsulate the RNA. Using an industry partner’s emergent capsule platform technology, the RNA is orally administered and locally tackles the inflammation in the intestinal tissue, avoiding systemic side effects.
As a pre-clinical study, GENEGUT will be the first, potentially revolutionary step toward delivering an advanced form of treatment that more than three million patients in Europe alone could benefit from. This would be the first option for these patients to finally manage the chronic disease and lead a life of improved quality.
The project starting in October of 2022, is coordinated by the School of Pharmacy of the University College Cork and, through a patient-centred approach, brings together the European Federation of Crohn’s and Ulcerative Colitis Associations with renowned researchers, expert clinical scientists, SMEs and large pharma companies.
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