Large grant for development of new drugs
Jørgen Kjems and Ken Howard are awarded a three-year grant of DKK 9,8 million from The Danish Council for Strategic Research for a project that focuses on the development of new drugs for cystic fibrosis and inflammatory bowel disease.
The aim of this project is to develop better nanodrugs for the treatment of these chronic diseases of mucosal surfaces. This is achieved by designing small RNAs surrounded by polymers to form nanoparticles that facilitate penetration across pulmonary and gastrointestinal mucous membranes and downregulate the expression of several genes involved in cystic fibrosis (CF) and inflammatory bowel disease (IBD).
Project description
A promising molecular medicine approach is based on inactivation of disease-associated genes using small intervening RNA (siRNA) and antisense oligonucleotides (ASO).
Nanoparticles containing siRNA or ASO are designed to improve the therapeutic potential of the otherwise unstable oligonucleotides by improving delivery across biological barriers. These systems allow non-invasive routes of administration such as oral and pulmonary routes that are an attractive alternative to injections.
Both lung and intestinal tissues are covered with an overlying mucosa, which effectively prevents direct contact between the cells and the drug. This project aims to develop advanced nanoparticle systems with improved mucus attachment and mucus penetration properties optimised for efficient delivery of siRNA or ASO to the lung and intestine. Delivery of nanoparticles to the mucosa and the lung epithelium will be optimised by using the built-in chains of biodegradable proteins or sugars that are active within the mucosal environment.
The therapeutic potential of these systems will be evaluated in animal models for CF and IBD.
In addition to the research groups of Professor Kjems and Dr Howard, researchers at Aarhus University Hospital and international experts in cell and disease animal models participate in this project providing a unique platform for developing better nanodrugs for the treatment of diseases of mucosal surfaces.
The project is conducted in collaboration with Aarhus University Hospital, the Norwegian University of Science and Technology Norway (NTNU), University of Iowa, USA; Alexander Fleming Biomedical Science Research Center, Greece; Ribotask; Integrated DNA Technologies, USA.
More information
Professor Jørgen Kjems
Department of Molecular Biology and Genetics & Interdisciplinary Nanoscience Center (iNANO)
Aarhus University, Denmark
jk@mb.au.dk – +45 2899 2086
Text: Jørgen Kjems, Sys Zoffmann og Lisbeth Heilesen
Translation: Lisbeth Heilesen