Alnylam Scientists and Collaborators Publish Research in Nature Chemical Biology on a Novel Mechanism for Regulation of Gene Expression with Anti-Gene RNAs (agRNAs)

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has published a new paper in the journal Nature Chemical Biology with collaborators at the University of Texas Southwestern Medical Center (UT Southwestern). These data demonstrate a novel mechanism whereby gene expression can be selectively regulated by anti-gene RNAs (agRNAs), double-stranded RNAs that target non-coding RNA sequences.

“We are encouraged by the progress we are making in understanding basic molecular mechanisms whereby non-coding RNA transcripts of genes can be targeted to effect desired changes in gene expression”

“In collaboration with leading academic labs, Alnylam continues to lead the translation of RNAi research toward clinical applications,” said Muthiah Manoharan, Ph.D., Senior Vice President, Drug Discovery at Alnylam. “We are very excited about our collaboration with the Corey lab at UT Southwestern, where we have explored novel mechanisms for control of gene expression with novel RNA interference and RNA activation mechanisms. This new paper, our 12th peer-reviewed paper published this year to date, is an excellent example of the novel insights we are learning from these and many other efforts.”

The new research results were published in Nature Chemical Biology (Yue et al., Nature Chemical Biology, 10.1038/nchembio.400). The new findings describe the design and characterization of agRNAs complementary to non-coding RNAs. agRNAs can act as inhibitors or activators of gene expression, and were previously described to be complementary to non-coding transcripts that span the promoter region of genes (Li, L.C. et al., Proc. Natl. Acad. Sci. USA 103, 17337–17342 (2006); Janowski, B.A. et al., Nat. Chem. Biol. 3, 166–173 (2007); Schwartz, J.C. et al., Nat. Struct. Mol. Biol. 15, 842–848 (2008)). In this new work, agRNAs that target non-coding RNAs corresponding to regions beyond the 3' termini of genes were also found to be active in silencing or activating genes in a sequence-specific manner. The use of agRNAs for RNA activation (RNAa) may have applications in a range of human disorders such as genetic and metabolic diseases and cancer where the aberrantly low expression of specific proteins defines disease etiology.

“We are encouraged by the progress we are making in understanding basic molecular mechanisms whereby non-coding RNA transcripts of genes can be targeted to effect desired changes in gene expression,” said David Corey, Ph.D., Professor of Pharmacology and Biochemistry at UT Southwestern. “In this new study, we have discovered yet a new approach whereby agRNAs can lead to either silencing or activation of target genes.”

About RNA Interference (RNAi)

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.

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 for the treatment of a wide range of disease areas, including respiratory syncytial virus (RSV), liver cancers, TTR-mediated amyloidosis (ATTR), hypercholesterolemia, and Huntington’s disease. In addition, Alnylam formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in manufacturing processes for biotherapeutic products, including recombinant proteins and monoclonal antibodies. 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, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. Alnylam and Isis are joint owners of Regulus Therapeutics Inc., a company focused on the discovery, development, and commercialization of microRNA therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.

Alnylam Forward-Looking Statement

Various statements in this release concerning Alnylam’s future expectations, plans and prospects, 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 Alnylam’s ability to discover and develop novel drug candidates, 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.