Release Details

CAMBRIDGE, Mass. and CARLSBAD, Calif., Oct 25, 2007 (BUSINESS WIRE) -- Regulus Therapeutics, a joint venture between Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) and Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) formed to discover, develop, and commercialize microRNA therapeutics, announced today the publication of new research in the Journal of Virology on the role of microRNAs in regulating pathways of viral infection. The research was performed in collaboration with the Max von Pettenkofer Institute for Virology in Munich, Germany and the National Center for Scientific Research in Strasbourg, France. microRNAs are small non-coding RNAs expressed in mammalian genomes and certain viral genomes and are believed to regulate whole networks of genes that can be involved in discrete disease processes. The new findings add to a growing body of research demonstrating that certain viral microRNAs play a role in the viral life cycle, likely by regulating host gene expression, and suggest that microRNA therapeutics that target specific viral microRNAs could provide a novel approach for anti-viral medicines.

"Our new research is an important step forward in understanding the broad biological roles for microRNAs in health and disease, particularly the role of viral microRNAs in regulating the expression of host genes that might otherwise limit the severity of a viral infection," said Juergen Soutschek, Ph.D., Senior Director of Scientific Development for Regulus. "The publication of these new results is representative of Regulus' strategy to collaborate with leading academic groups in the area of microRNA biology, and this research is another example of the role microRNA therapeutics may play in treating disease."

Human cytomegalovirus (CMV) can be a significant health problem for patients with compromised or deficient immune systems, leading to severe and potentially life-threatening disease. The infection of the mouse with CMV is the best established model to investigate the biology of CMV infections in vivo. In this new research (Doelken et al. (2007) Journal of Virology 81), microRNAs encoded by mouse CMV were isolated and characterized from infected fibroblasts. These viral microRNAs increased over time during infection and became even more abundant than some of the most highly expressed host-derived microRNAs suggesting that the viral microRNAs have an important role during viral infection. Viral microRNAs that are highly expressed during infection may compete with the function of cellular microRNAs, and therefore perturb the fine-tuned regulation of certain biological pathways to the advantage of the virus. The infection of mice with CMV can now be used to study the role of viral microRNAs in viral infections and as a model to evaluate microRNA therapeutics.

About microRNAs

microRNAs are a recently discovered class of genetically encoded small RNAs, approximately 20 nucleotides in length, and are believed to regulate the expression of a large number of human genes. microRNA therapeutics represent a new approach for the treatment of a broad range of human disease When inappropriately encoded, microRNAs represent potential disease targets whose selective antagonism can result in the correction of an entire disease pathway in a manner unachievable by today's medicines. In fact, the inappropriate absence or presence of specific microRNA molecules in various cells has been shown to be associated with specific human diseases including cancer, viral infection, and metabolic disorders.

About Regulus

Regulus Therapeutics LLC is a biopharmaceutical company formed to discover, develop and commercialize microRNA therapeutics. The company was created as a joint venture between Alnylam Pharmaceuticals, a leader in RNAi therapeutics, and Isis Pharmaceuticals, a leader in antisense technologies and therapeutics. Isis and Alnylam scientists and collaborators were the first to discover microRNA antagonist strategies that work in vivo in animal studies (Krutzfeldt et al. (2005) Nature 438, 685-689; Esau et al. (2006) Cell Metab., 3, 87-98). Isis and Alnylam have also created and consolidated key intellectual property believed by the companies to be required for development and commercialization of microRNA therapeutics. The company, founded in 2007, maintains facilities in Carlsbad, California. For more information, visit www.regulusrx.com.

About Alnylam Pharmaceuticals

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is applying its therapeutic expertise in RNAi to address significant medical needs, many of which cannot effectively be addressed with small molecules or antibodies, the current major classes of drugs. Alnylam is leading the translation of RNAi as a new class of innovative medicines with peer-reviewed research efforts published in the world's top scientific journals including Nature, Nature Medicine, and Cell. The company is leveraging these capabilities to build a broad pipeline of RNAi therapeutics; its most advanced program is in Phase II human clinical trials for the treatment of respiratory syncytial virus (RSV) infection. In addition, the company is developing RNAi therapeutics for the treatment of influenza, hypercholesterolemia, and liver cancers, among other diseases. The company's leadership position in fundamental patents, technology, and know-how relating to RNAi has enabled it to form major alliances with leading companies including Medtronic, Novartis, Biogen Idec, and Roche. The company, founded in 2002, maintains headquarters in Cambridge, Massachusetts. For more information, visit www.alnylam.com.

About Isis Pharmaceuticals

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 18 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 www.isispharm.com.

Alnylam Forward-Looking Statements

Various statements in this release concerning Alnylam's future expectations, plans and prospects, including statements concerning the potential of microRNA therapeutics and the importance of Alnylam's intellectual property, know-how, and other technology in the discovery, development and commercialization of microRNA therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including risks related to: Alnylam's approach to discover and develop novel drugs, which is unproven and may never lead to marketable products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to enforce its patents against infringers and to defend its patent portfolio against challenges from third parties; Alnylam's ability to obtain additional funding to support its business activities; Alnylam's dependence on third parties for development, manufacture, marketing, sales and distribution of products; obtaining regulatory approval for products; competition from others using technology similar to Alnylam's and others developing products for similar uses; Alnylam's dependence on collaborators; and Alnylam's short operating history; as well as those risks more fully discussed in the "Risk Factors" section of its most recent quarterly report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam does not assume any obligation to update any forward-looking statements.

Isis Forward Looking Statements

This press release includes forward-looking statements regarding the future therapeutic and commercial potential of Isis' technologies and intellectual property related to microRNA therapeutics being discovered and developed by Regulus. 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 this and other documents are available from the Company.

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

SOURCE: Regulus Therapeutics

Alnylam Pharmaceuticals, Inc.
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