B-SMART has achieved first results in the development of RNA-based nanomedicines to treat neurodegenerative diseases. The scientists succeeded in developing an effective nanoparticle with customisable characteristics which is now being tested in a preclinical trial. In order to ensure scalability and a safe transfer of the scientific results to the clinic, the researchers have been focusing on a reliable and reproducible nanoparticle production method based on microfluidics.
“We are very pleased to see that our frontrunner nanoparticle behaves as expected,” said Prof. Raymond Schiffelers from the University Medical Center Utrecht who coordinates the project. “With a slight advance in time, we are confident to move on to our next big challenge, which will be to demonstrate therapeutic activity in mouse models of Alzheimer’s disease.”
The researchers are already working on the selection of the special nanobodies needed for the development of the so-called targeting ligands which enable the RNA-based nanoparticles to cross the blood-cerebrospinal fluid barrier and reach the affected brain cells.