Proprietary Tools
Chemistry:
Key to our ability to develop oligonucleotides that modulate microRNAs is our proprietary "tool box" of chemical modifications for optimizing oligonucleotide drugs. These modifications allow Regulus scientists to utilize medicinal chemistry approaches for improving the specificity, potency and drug-like properties of oligonucleotides. We have versatile chemistries that can be tailored to each microRNA and are not restricted to a singular approach.
Bioinformatics:
Developing microRNA therapeutics requires an understanding of microRNA targets, and the pathways they regulate. Since microRNAs regulate networks of genes, understanding links between microRNA targets and function is essentially a challenge in network biology. Regulus scientists are developing novel methods and algorithms to monitor the activity of microRNA-modulating drugs and understand the functional consequences of network alterations.
Pharmacology/Biology:
Using microRNA therapeutics to treat human diseases requires knowledge of the functional consequences of microRNA modulation via pharmacologic agents. Optimal therapeutic targets will result from skillful alignment of the properties of oligonucleotide drugs with therapeutically relevant biological functions of microRNAs. Moreover, development of microRNA-modulating drugs requires optimization of their pharmacological properties (biodistribution, pharmacokinetics, safety, etc). Regulus scientists are experienced at performing experiments in animals to pharmacologically modulate microRNAs using oligonucleotides.
At Regulus, we believe that there is a tremendous synergy in maintaining all of these technologies, and that the successful integration of all is a pre-requisite to the development of effective microRNA therapeutics.
