NanoAssemblr™ Chosen for Horizon 2020 Project to Develop Novel RNA therapeutics

Precision NanoSystems NanoAssemblr™ platform has been selected for the controlled manufacture of nanomedicines as part of the multinational B-SMART (Brain-Specific, Modular and Active RNA Therapeutics) translational research project. This €6 million initiative, funded by the EU Horizon 2020 program, aims to use RNA-based therapeutics to silence the production of disease-causing proteins for a range of neurodegenerative disorders, such as Parkinson’s, Alzheimer’s and Huntington’s. Professor Raymond Schiffelers from UMC Utrecht is leading the project, and explained its goals: “RNA medicines are interesting because you can use what is essentially the same polynucleotide molecule to treat multiple diseases, just by changing the nucleotide sequence. Our goal is therefore to design modular nanoparticles capable of delivering a payload of therapeutic RNAs to the brain, allowing them to prevent the biosynthesis of harmful proteins at source.”

“One of the key aims for Horizon 2020 projects is to demonstrate that these technologies are transferrable from a research to a clinical setting. This means that the manufacturing processes must be reproducible and scalable. Precision NanoSystem’s NanoAssemblr platform is ideal from this perspective, allowing us to make many different types of particles in a reliable and reproducible manner. This technology also allows you to accurately predict the particle size based on the mixing speed, PEG concentration and mixing ratios, which is a significant step forward. Equally importantly, it can be easily scaled to manufacture batch volumes sufficient for clinical trials. Having a NanoAssemblr Benchtop instrument in each laboratory involved in the project – eight in total across the Netherlands, Belgium, Norway, the UK, Spain and Italy – will allow us to manufacture the same particles at each site, helping to accelerate our research.”