New consortium to develop RNA-based treatment for Crohn's disease
30 September 2022
With over 5 million euros in funding from Horizon Europe, new knowledge consortium GENEGUT will develop an RNA-based therapy against inflammatory disease in the gastrointestinal tract. "It is inspiring to take on a challenge with such extensive medical needs," says Per Artursson, Professor of Dosage Form Design.
RNA-based therapies are expected to become increasingly important in modern healthcare, and with the mRNA vaccines against covid-19, the technology took another important step towards a full scale breakthrough. The next scientific challenge is to safely deliver the RNA therapeutics to and into the target cell, and now nine universities and pharmaceutical companies are uniting in GENEGUT, a European knowledge consortium aiming to build the basis for an RNA therapy against Crohn's disease, an inflammatory disease of the gastrointestinal tract.
“It is extremely inspiring for our research team to take an active role in GENEGUT. With 5.4 million euros in funding from Horizon Europe, we will over the next four years take on a challenge associated with extensive medical needs, and the fact that the consortium has succeeded in recruiting several leading organisations in our field gives us good prospects to reach our goals,” states Per Artursson, Professor of Dosage Form Design at Uppsala University.
Crohn's disease can occur in all parts of the gastrointestinal tract, but most often affect the end of the small intestine. In Europe alone, nearly three million people develop the disease each year, causing society costs of approximately SEK 50 billion. Healthcare still lacks a curative treatment for Crohn's disease, but with the right therapy, the patient can basically live a normal life.
“With GENEGUT, we aim to create the foundation for a completely new form of RNA-based therapies, where we will use biomaterials and capsule technology to bind RNA to nanoparticles designed specifically to penetrate the barriers of the gastrointestinal tract. Our team provides expertise in, among other things, the development of relevant cell models, mechanisms of RNA nanoparticle uptake and intracellular localisation,” says Madlen Hubert, Associate Professor at the Department of Pharmacy.
The consortium's goal is to, within four years, develop an RNA-based drug that can be swallowed in tablet or capsule form and progress it to clinical studies. If and when the drug reaches healthcare, the ambition is to enable an orally available therapy against Crohn's disease that will selectively and effectively target the inflamed intestinal cells.
“RNA-based therapies is an exciting field with enormous potential. The number of approved treatments is increasing in both the US and Europe, but every new step forward requires that we can deliver the substances into the body and activate uptake in the target cell. Our group has extensive experience in the type of research required, and I hope that our contributions will be of great value to the consortium and, in the long term, to everyone living with Crohn's disease,” says Madlen Hubert.
- GENEGUT is an interdisciplinary consortium coordinated at University College Cork.
- The consortium unites 9 partners in 8 European countries whose collective expertise runs from laboratory to clinical studies.
- The consortium includes Uppsala University's research environment for Drug delivery.