Everyone is born with variations in genetic material — DNA and RNA — which serves as the blueprints for building the proteins that control cell function. Our company’s founders recognized that our bodies have a natural system for making changes to our RNA. By leveraging this system, we aim to reverse harmful genetic variations that cause missing or malfunctioning proteins, which can lead to serious diseases like alpha-1 antitrypsin deficiency (AATD).

RNA is made up of a sequence of molecular subunits, often compared to letters in a code, that determine how proteins are built and function. The body’s natural RNA editing system uses a specialized protein called ADAR to make precise changes to the RNA by swapping one of the letters – switching an adenosine (A) to a new letter called an inosine (I), which is read as a guanosine (G) by the body’s genetic machinery. We are developing an investigational therapy that is designed to guide ADAR to make precise edits at the correct location. These small edits to RNA can lead to important improvements in how proteins function.