Press Releases
Isis Pharmaceuticals Demonstrates The Potential Of Single-Stranded RNA-Like Antisense Technology To Activate The RNAi Pathway
In the published study titled 'Single-stranded siRNAs activate RNAi in animals', Isis scientists evaluated the absorption, distribution, metabolism and activity of the ss-siRNAs designed to utilize the RNAi pathway. These data show that single-stranded RNA-like antisense oligonucleotides are broadly distributed to and active in multiple organs and effectively engage and trigger the RNAi pathway without the need for complex formulations or delivery vehicles typically necessary for double-stranded RNAi molecules.
"Double-stranded siRNA drugs require complex formulations to achieve sufficient delivery for systemic activity. This requirement has severely limited the development of safe and effective drugs that work through the RNAi pathway. In contrast, single-stranded RNA-like antisense drugs can be administered subcutaneously and distribute to tissues without the need for formulations. In addition, approaching RNAi with single-stranded drugs eliminates the need for the complementary strand, the 'sense' strand, which limits the risk of adverse effects, and reduces the cost of manufacturing," said
In a separate study also published today in Cell titled 'Single-stranded RNAs act through RNAi, allele-selectively target expanded CAG repeats, and potently inhibit huntingtin expression', Isis and collaborators from
"Together, these data provide compelling evidence that single-stranded oligonucleotides can be designed to exploit the RNAi pathway and silence gene expression of specific mRNAs in target tissues. In our initial experiments with animals, the compounds were well-tolerated even when used at high doses," said
"Working with our colleagues at the
The journal articles titled, "Single-Stranded siRNAs Activate RNAi in Animals" and "Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression" and a leading edge preview titled "Singles Engage the RNA Interference Pathway" by Davidson, B.L. and Monteys A.M. are available in the
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Isis is exploiting its leadership position in antisense technology to discover and develop novel drugs for its product pipeline and for its partners. Isis' broad pipeline consists of 25 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, and cancer. Isis' partner, Genzyme, plans to commercialize Isis' lead product, KYNAMRO™, following regulatory approval. Isis' patents provide strong and extensive protection for its drugs and technology. Additional information about Isis is available at www.isispharm.com.
This press release includes forward-looking statements regarding Isis' antisense technology, the discovery, development and therapeutic potential of Isis' antisense drugs and the potential for Isis to develop antisense drugs that work through the RNAi pathway. Any statement describing Isis' goals, expectations, financial or other projections, intentions or beliefs, including the planned commercialization of KYNAMRO, is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Isis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Isis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis' programs are described in additional detail in Isis' annual report on Form 10-K for the year ended
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SOURCE
D. Wade Walke, Ph.D., Executive Director, Corporate Communications and Investor Relations, +1-760-603-2741; or Amy Blackley, Ph.D., Associate Director, Corporate Communications, +1-760-603-2772