RNA interference is a natural phenomenon which can be used to selectively turn OFF the genes expressed in some diseases. Genes are the blue prints for proteins, the building blocks of life, and RNA interference can prevent these being translated in to the proteins involved in these diseases. This ability to target specifically the expression of these proteins could open the way to therapeutics that neither classical small molecules nor antibodies can open.
Silence's intellectual property and know-how have permitted the creation of the strongest portfolio in the RNA interference's field with:
- A RNAi platform built around the AtuRNAi® patented technology and based on: exclusive features, stabilisation technology, and specific siRNA molecules.
- A delivery platform using proprietary molecules such as AtuFECT to target the largest variety of organs in the field with: AtuPLEX® (for endothelial cells of the vascular system); DBTC (for hepatocytes and hepathic vascular system), and DACC (for vascular endothelial cells of the lung)
- A promising pipeline with five clinical stage programs: three in phase II and two in phase I. Silence has developed, for example, an internal program (Atu027) using its proprietary AtuPLEX® and AtuRNAi® technologies to target the expression of the protein PKN3. Atu027 is currently being tested in a Phase I dose-escalation trial and the results of this study should be presented around mid-2012.
Silence's pipeline made of internal and external programs provide validity for the RNAi and the internally developed delivery systems.