Efficacy and safety of RNAi candidates successfully tested
The research team led by Prof. Armin Braun, Head of Preclinical Pharmacology and Toxicology at Fraunhofer ITEM, and Prof. Axel Schambach, Head of the Institute of Experimental Hematology at MHH, is focusing on RNA-based drugs that can be quickly adapted to different viruses and thus provide protection against emerging infectious diseases. To this end, they are initially focusing on the parainfluenza virus. The aim is to use RNA interference (RNAi) to prevent the virus from multiplying and spreading in the body. RNAi is a natural mechanism in our cells for switching off genes in a targeted manner. The research team has developed suitable RNAi building blocks that specifically paralyze different sections of the parainfluenza virus genome that the virus needs to replicate. The efficacy and safety of these RNAi candidates have been successfully tested on human lung tissue sections as well as in 3D cell cultures from human lung epithelium and in animal models. The RNAi candidates were able to suppress the parainfluenza virus by 95 percent in vitro and in vivo. The researchers have also developed an inhalable therapeutic agent to deliver the therapeutic RNA directly to the target cell infected by the virus in the lung. The studies on the ex-vivo model of isolated perfused lungs showed that the drug preparation can be nebulized well and reaches the lung epithelium as desired.
Creating the regulatory conditions for clinical testing of the inhalable active ingredient
In the third funding stage, the researchers now want to create the regulatory requirements for the clinical trial. This means that the efficacy and safety of the approach will now be worked out even more clearly and important steps regarding clinical application will be developed. The long-term goal is to use the developed iGUARD platform to construct corresponding RNA therapeutics for other known viral diseases. Ultimately, to quickly adapt active substances to unknown, newly emerging virus types and thus be able to develop customized therapies very quickly.
Cooperation partenrs in the iGUARD project
The iGUARD project is a cooperation between the MHH Institute for Experimental Hematology (Prof. Axel Schambach, Philippe Vollmer Barbosa), the MHH Clinic for Hematology, Hemostaseology, Oncology and Stem Cell Transplantation (Dr. Adrian Schwarzer) and the Fraunhofer ITEM Division of Preclinical Pharmacology and Toxicology (Prof. Armin Braun, Philippe Vollmer Barbosa).