Idera Pharmaceuticals, Inc. (NASDAQ: IDRA) today presented new data on its novel gene-silencing oligonucleotide (GSO) technology at the Cell Symposium on Regulatory RNAs in Chicago, IL. In preclinical studies, systemic delivery of GSOs targeted to ApoB or PCSK9 mRNA caused a reduction in the level of the targeted mRNA and associated protein and resulted in a decrease in serum total cholesterol and LDL-cholesterol concentration. ApoB and PCSK9 are two validated targets associated with cardiovascular diseases.
"These data are an important step for Idera's GSO program as they show significant in vivo gene-silencing activity following systemic delivery of GSOs without using any carrier technologies," said Nicola La Monica, Ph.D., Vice President of Biology at Idera Pharmaceuticals. "These attributes demonstrate the potential for GSOs to overcome some of the issues associated with other gene-silencing technologies."
The presentation, entitled "Design and characterization of novel Gene Silencing Oligonucleotides", was authored by Idera scientists Nicola La Monica, Weiwen Jiang, Lakshmi Bhagat, Ekambar R. Kandimalla and Sudhir Agrawal.
In this study, Idera created 19mer GSOs for apolipoprotein B (ApoB) and proprotein convertase subtilisin/kexin type 9 (PCSK9) mRNA and evaluated their in vivo activity in mice following subcutaneous administration. The data demonstrate that treatment with each GSO led to a significant reduction in the concentration of the target associated mRNAs and protein. The effects were specific, with no significant effects being observed on ABCA1, ABCG1 or LXR mRNA levels. In addition, treatment with GSOs for either ApoB or PCSK9 resulted in a decrease in total serum cholesterol and LDL-cholesterol.
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