Problem
of cell types remain untargetable for RNA therapeutics
With programmable RNAi medicines drug developers have the extraordinary ability to target the genetic root cause of disease. However the inability to deliver these therapeutic RNA molecules to cell types beyond the liver is holding back the progress of RNA medicine. Of the 500 or so cell types in our body, today only one cell (liver hepatocyte) has a clinically approved delivery system for RNAi.
Solution
RNA is shuttled via an ecosystem of unencapsulated mechanisms — the delivery is encoded in the RNA
To address the delivery problem, we investigated how RNA is naturally delivered to different cell and tissue types. What we realized is that nature has evolved an ecosystem of RNA delivery mechanisms to facilitate cell-cell communication. While conventional thought had assumed this RNA must be packaged in lipidic capsules, such as exosomes or vesicles, over 99% of RNA is not encapsulated.
