RNAi PlatformRNAi Platform
Potent siRNA molecules are fundamental for powerful RNAi applications. Silence Therapeutics developed early on its own multidisciplinary research and discovery approach for advancing a novel proprietary technology platform and RNAi therapeutic approach into clinical development. These platform technologies encompass the generation of highly potent chemically stabilized siRNA molecules (proprietary "AtuRNAi®") which differed from the "classical Tuschl"-type molecules in several aspects (Czauderna et al. 2003). Consequently, it led to the creation of their own patent estate for therapeutic application.
Silence Therapeutics has developed novel proprietary siRNA, so-called AtuRNAi®, which has been chemically modified to result in a blunt-ended, chemically stabilized molecule which contains only naturally-occurring RNA. This modification pattern offers advantages over standard siRNA such as stability against nuclease degradation and prevention from innate immune response. The result of using only naturally occurring building blocks for manufacturing of AtuRNAi® is: the elimination of toxic metabolites, lower manufacturing costs through cheaper building blocks, as well as increased yield and faster synthesis through improved process development can be guaranteed. Unlike most existing therapeutics where each new chemical entity has a distinct composition and synthetic protocol, the structure and synthesis of AtuRNAi® type siRNA is similar.