Maria G. Majorek, Monika Gutowska, Joanna Hucz, Monika Lamparska-Przybysz, Maciej Wieczorek
The discovery of RNA interference (RNAi) in eukaryotic cells has opened new possibilities for drug development. Target-specific gene silencing via short interfering RNA (siRNA) displays many advantages over traditional pharmaceuticals. The unique siRNA nucleotide sequence benefits in higher drug specificity. Besides designing target-optimized siRNA oligonucleotides, one of the major challenges is to establish the efficient delivery system facilitating selective targeting altered tissues. The proper sequence-delivery combination provides these innovative therapeutics with unrivalled specificity and tolerability.
Another pivotal issue which has to be taken into account designing siRNA drugs is to provide them with an appropriate pharmacokinetic and pharmacodynamic properties. This is often achieved by an introduction of variety of chemical modifications into the siRNA molecules e.g. O-methyl groups added to the 2′ position of the ribosyl ring.
The ongoing extensive studies on RNAi resulted in introduction of several potential siRNA drugs into clinical trials. Celon Pharma takes part in a global research on siRNA therapeutics with its deep pipeline of innovative products including RNAi-based drugs.