Idera Pharmaceuticals, Inc. (Nasdaq: IDRA) today announced the presentation of preclinical data of its novel gene-silencing oligonucleotide (GSO) technology including observations that demonstrate GSOs exert gene-silencing activity through a similar cellular pathway as siRNA. The presentation, entitled "Novel Oligonucleotides Containing Two 3'-Ends Complementary to Target mRNA Show Optimal Gene-Silencing Activity", is being made at the TIDES: Oligonucleotide and Peptide® Research, Technology and Product Development conference being held in Boston, Massachusetts from May 22-25, 2011.
The data presented today are from studies of GSOs targeting MyD88, a signal transduction protein utilized in Toll-like receptor pathways. In these studies, MyD88 GSOs suppressed MyD88 mRNA in cell-based assays, and inhibited MyD88-dependent induction of cytokines and chemokines in mice. In vivo inhibition of MyD88-dependent immune responses was achieved by administering GSOs subcutaneously without the use of any delivery enhancement technology. In cell-based assays, using 5' RLM-RACE analysis, MyD88 GSO was shown to cleave the targeted mRNA at the same site as does MyD88 siRNA, thereby suggesting that GSOs exert gene-silencing activity through a similar mechanism as siRNA.
"GSOs are single-stranded oligonucleotides with two exposed 3'-ends that show length-dependent gene-silencing activity. Based on our ongoing research we believe that GSOs act through a common pathway as does siRNA," commented Nicola La Monica, Ph.D., Vice President of Biology at Idera Pharmaceuticals. "Additionally, the gene-silencing activity of GSOs has been achieved by systemic administration without the use of any carrier, which is one of the major limitations of the use of siRNA."