Idera Pharmaceuticals, Inc. (Nasdaq: IDRA) today announced the online publication in the Journal of Medicinal Chemistry of its studies of a novel class of compounds, which we refer to as gene-silencing oligonucleotides (GSOs). In these studies, Idera's GSOs efficiently inhibited gene expression while overcoming some of the most intractable issues associated with other gene-silencing technologies: stability, specificity, and systemic delivery.
In the publication, entitled Novel Oligonucleotides Containing Two 3'-Ends complementary to Target mRNA Show Optimal Gene Silencing Activity, data is presented on the gene-silencing activity of GSOs in both cell-based assays and in mouse models. GSOs are single-stranded oligonucleotides comprised of RNA or DNA with two exposed 3'-ends. The key observations of the study are that the gene-silencing activity of GSOs was length dependent, with 19-mer GSOs having the greatest activity, and that GSOs were able to be delivered systemically without using a delivery technology. In the studies, GSOs demonstrated greater activity and a longer duration of activity against multiple targets including MyD88, VEGF, and TLR9 mRNAs, as compared to the traditional antisense compounds evaluated.
"The discovery that GSOs have length-dependent gene-silencing activity, which is analogous to observations with siRNA, was an unexpected but very important finding as it suggests that GSOs are engaging a similar cellular pathway for their activity," commented Nicola La Monica, Ph.D., Vice President of Biology at Idera Pharmaceuticals. "We are conducting studies of GSOs targeted to messenger RNA and micro RNA and we are very pleased with the results of these ongoing studies."
"The data reported in the publication demonstrate the robust gene-silencing activity of our proprietary GSOs and their efficient systemic delivery without the use of any delivery technology," commented Sudhir Agrawal, D.Phil, Chairman and Chief Executive Officer of Idera. "Our focus remains on advancing the clinical development of our TLR-targeted drug candidates. However, the GSOs provide us with a new technology platform that we are currently using for validating targets and for identifying new drug candidates for potentially undruggable targets."
Idera Pharmaceuticals, Inc.