siRNA nanoparticle for the treatment of cancer

Offentlig prosjektsammendrag

Sense Biopharma is an early-phase biotechnology company based in Oslo. Sense develops highly innovative treatments for cancers and auto-immune diseases. Our first indication is brain cancer (glioblastoma). About 300 Norwegians every year are diagnosed with aggressive brain cancer. The treatment has not changed significantly for the past 30 years and the life expectancy at diagnosis is only 14 months, one of the lowest in all cancer. Thus, the unmet medical need is very high. Sense harnesses the promise of RNA interference technology (RNAi) to develop effective and safe novel medicines. RNAi enables us to temporarily shut down genes that the tumor cells need to survive. RNAi technology is safe because it is targeted and temporary. The main problem that has for long bedeviled the field of RNAi is delivery of RNA into the cells. RNA is not stable in blood and does not cross the cell membrane. We thus need a delivery strategy. The most advanced delivery vehicles are nanoparticles. Nanoparticles are tiny spheres into which RNA can be packaged. The RNA is shielded from blood and remains stable. The first generation of nanoparticles was based on lipids. The nanoparticles were tiny lipid droplets that carried RNA inside. These work well for RNA delivery, but the main problem is that the majority of lipid nanoparticles get metabolized in the liver. The function of the liver is to filter nutrients (including lipids) out of the blood and to process them. Not surprisingly, the few nanoparticle-based RNAi medicines that have so far been approved target diseases in the liver, exploiting to their advantage their natural livertropism. For the treatment of brain tumor, we need a nanoparticle that shows little liver retention and can reach the brain. That's why we have chosen polymer nanoparticles, which largely pass unhindered through the liver. Our nanoparticle is currently at pre-clinical stage of development.