Regulus relies on its proprietary “tool box” of chemical modifications to develop oligonucleotides that effectively modulate microRNAs. These modifications allow Regulus scientists to utilize medicinal chemistry approaches for improving the specificity, potency, and drug-like properties of oligonucleotides. Regulus has versatile chemistries that can be tailored to each microRNA and therapeutic indication. Our therapeutic product platform has benefited from more than 20 years of innovation in RNA technology, including the use of a phosphorothioate backbone to increase metabolic stability and tissue half-life of oligonucleotides. In addition, Regulus scientists apply new chemistries that dramatically alter the pharmacokinetic properties of these molecules. The introduction of chemical modifications such as 2’ methoxyethyl (MOE) and 2’, 4’-constrained 2’O-ethyl (cEt) into the ribose sugar ring significantly improve both the pharmacokinetic and safety profile. Given the high water solubility of anti-miR oligonucleotides (due to their polyanionic chemical structure), these compounds can be systemically delivered in simple aqueous solutions such as buffered saline and rapidly partitioned from the plasma to be taken up by cells of multiple different tissues. Regulus has also built an oligonucleotide synthesis and analytical development expertise to help to advance therapeutic programs successfully from discovery to development.
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RNA-based therapeutics hold significant potential as promising treatment options for human disease.
Nature Reviews Genetices
View a poster on the Regulation of microRNA biogenesis, function, and degradation published in Nature Reviews Genetics.