Delivery Platform

Effective siRNA delivery to the right cells is a challenge given the complexity of the human body.

We attach a GalNAc ligand to our siRNA molecule at one or more different sites for highly targeted delivery to specific liver tissues/cells.

 

 GalNAc conjugates

GalNAc (N-Acetylgalactosamine) is a naturally occurring sugar that binds to the asialoglycoprotein receptor (ASGPR), which is highly expressed in the membrane of hepatocytes (liver cells). Coupling GalNAc sugars to stabilized siRNA molecules allows them to target liver cells and carry out their function, being shuttled into the cytoplasm as a result of the GalNAc-ASGPR binding.

GalNAc-siRNA conjugates can be administered subcutaneously, in a more patient-friendly way that removes the need for invasive intravenous infusions. This key advantage lowers the patient burden of GalNAc-siRNA drugs in comparison to more complex delivery systems such as liposomes, meaning that GalNAc-based therapies are an attractive solution for a wider selection of liver-centric disease areas. Given the size of this opportunity, developing GalNAc-siRNA therapies is the core focus of our Company.