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Isis Awarded $1.5 Million NIH Grant to Improve Chemical Properties of RNAi-Based Therapeutics
Date:10/3/2007

Phase 2 SBIR Grant Funding to Advance the Development of Single-Stranded

Antisense RNAi Drugs

CARLSBAD, Calif., Oct. 3 /PRNewswire-FirstCall/ -- Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) today announced that it has been awarded a multi-year Phase 2 Small Business Innovation Research (SBIR) grant by the National Institutes of Health (NIH) for up to $1.5 million to design oligonucleotide drugs that can exploit the RNA interference (RNAi) antisense mechanism for disease treatment. The Phase 2 grant builds upon a successfully completed Phase 1 program that demonstrated the feasibility of using single-stranded antisense drugs to target the RNAi pathway.

"We are pleased with the continued support from the NIH in our ongoing efforts to apply our RNA technologies, oligonucleotide chemistries and expertise to exploit antisense mechanisms, such as RNAi, for therapeutic benefit. Based on our extensive work with single-stranded antisense drugs that work through an RNase H mechanism and the feasibility studies we have completed with single-stranded antisense drugs that harness the RNAi pathway, we are optimistic that the two drug families will share certain characteristics, including bioavailability and tissue distribution," said C. Frank Bennett, PhD, Senior Vice President of Research at Isis Pharmaceuticals, Inc. "At Isis, we have a rich history of pioneering work in RNA-targeted drug discovery, and with this research we are continuing to expand the ways in which we can harness biological phenomena with compounds exhibiting chemical properties that enable development of practical, safe and attractive drugs."

The multi-year grant will fund research by Isis to improve the stability and tissue distribution of RNAi drugs. Much of the work will focus on optimizing the chemical properties of single-stranded oligonucleotides that trigger the RNAi pathway. In addition to demonstrating that compounds optimized with Isis' chemistries produce superior results in animal models when compared to unoptimized compounds, the grant funds the discovery of RNAi-based drugs.

About RNAi

RNAi is an antisense mechanism that involves using RNA to target a specific messenger RNA sequence (mRNA) for degradation. The most common RNAi agents are double-stranded RNAs, which ultimately associate with proteins to form an RNA-induced silencing complex (RISC). The antisense RNA within active RISC binds to its mRNA target, which is then cleaved and degraded by the RNase enzyme in the complex. Although most currently practiced methods of inducing the RNAi mechanism utilize double-stranded RNAs, RISC requires only the antisense strand to function. This provides the opportunity to develop simplified single-stranded antisense drugs that can utilize the RNAi pathway to treat human disease.

About Isis Pharmaceuticals, Inc.

Isis is exploiting its expertise in RNA to discover and develop novel drugs for its product pipeline and for its partners. The Company has successfully commercialized the world's first antisense drug and has 17 drugs in development. Isis' drug development programs are focused on treating cardiovascular and metabolic diseases. Isis' partners are developing drugs for a wide variety of diseases. Ibis Biosciences, Inc., Isis' wholly owned subsidiary, is developing and commercializing the Ibis T5000(TM) Biosensor System, a revolutionary system to identify infectious organisms. As an innovator in RNA-based drug discovery and development, Isis is the owner or exclusive licensee of over 1,500 issued patents worldwide. Additional information about Isis is available at http://www.isispharm.com.

This press release includes forward-looking statements regarding Isis' research and drug discovery capabilities, and the potential for advancing RNAi-based therapeutics with funding from the NIH. Any statement describing Isis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement, including those statements that are described as Isis' goals. 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 products. 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 December 31, 2006, and its quarterly report on Form 10-Q for the quarter ended June 30, 2007, which are on file with the SEC. Copies of these and other documents are available from the Company.

In this press release, unless the context requires otherwise, "Isis," "Company," "we," "our," and "us" means Isis Pharmaceuticals and its subsidiaries.

Isis Pharmaceuticals, Ibis Biosciences and Ibis T5000 are registered trademarks or trademarks of Isis Pharmaceuticals, Inc.


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SOURCE Isis Pharmaceuticals, Inc.
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