Delivery for RNA based drugs

To deliver drug-encapsulated nanoparticles across the blood-brain barrier (BBB), several target proteins have been investigated (LRP1, TfR1, and Glut1). We developed BDDS™, our proprietary DDS by introducing a ligand moiety of the specific transporter to the surface of a PEG-based micelle as a targeting strategy to cross the BBB and to help the uptake of drugs by the cells in brain, such as by the neurons, astrocytes, and microglial cells.

The strength of BDDS™ lies in the capacity to adjust the composition of adjuvant moieties during the formulation process. Eventually, we can produce an optimized drug product with highly controlled biophysical properties, regardless of different electrochemical properties of the cargo material (ASO, mRNA, and siRNA).

The quantitative biodistribution analysis shows a high yield of BBB penetration with an outstanding enrichment in the brain, even to the deep brain, spinal cords, and skeletal muscle, supporting a unique strength on the drug delivery for the neurodegenerative disease and neuromuscular disease treatment.