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Alnylam Scientists Discover Novel Method for Monitoring RNAi Activity in Blood Samples

Circulating Extracellular RNA Detection (cERD) Method Enables Quantitative Measurement of Tissue-Specific mRNA Silencing

Company to Enable Academic Research and to Provide Broad Licenses to Industry

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 9, 2012-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that its scientists have discovered a novel method for monitoring RNAi activity in blood samples, representing a potential major advance for future development of RNAi therapeutics, other therapeutic modalities, and diagnostics. The new pre-clinical research discovery results were presented at the Keystone Gene Silencing by Small RNAs meeting, being held February 7 – 12, 2012 in Vancouver, B.C. This new method, called “circulating extracellular RNA detection” (cERD), allows quantitative measurement of tissue-specific mRNA silencing from biological fluids including blood and cerebrospinal fluid (CSF). Alnylam intends to enable use of the new technology in academic research and license industry to deploy the technology.

“We are very excited about this new discovery by Alnylam scientists on a novel approach for monitoring RNAi activity in blood samples. The cERD method enables routine and frequent measurement of tissue-specific target gene silencing, without the need for invasive tissue biopsies,” said Rachel Meyers, Ph.D., Vice President, Research at Alnylam. “We believe that this approach could be transformative for the development of RNAi therapeutics, in addition to having potential applications with other therapeutic modalities. In this regard, we are pleased to launch our efforts enabling industrial applications with a technology license to Isis.”

New pre-clinical results describing the cERD method demonstrate the ability to quantitatively monitor tissue-specific mRNA modulation in a non-invasive manner. Specifically, the new findings demonstrate that tissue-specific mRNAs can be detected in blood and CSF. Based on analysis of serum samples, mRNAs appear to circulate in blood via exosomes, a newly discovered class of cell-derived particles. The cERD method allows analysis of all circulating RNA, whether or not it is associated with exosomes. Administration of RNAi therapeutics toward the liver-specific target genes transthyretin (TTR) and transmembrane protease, serine 6 (Tmprss6) was found to result in dose-dependent silencing of target genes in both tissue mRNA, as measured in tissue samples, and blood-derived mRNA, as measured by the cERD method. Both onset and duration of mRNA silencing were found to be closely matched between tissue and blood-derived mRNA. Pre-clinical results were confirmed in a variety of animal models. Further, administration of RNAi therapeutics targeting the alpha-synuclein gene in the central nervous system (CNS) was found to result in silencing of the target gene in both tissue and CSF-derived mRNA, as measured by the cERD method. In addition, the cERD method was successfully applied to monitor activity of microRNA therapeutics and gene therapy vectors. Research on the new cERD method was done in collaboration with Derrick Gibbings at the Swiss Federal Institute of Technology, Zurich.

Alnylam intends to broadly enable academic research and also support industry efforts on RNAi therapeutics and other modalities with the newly described cERD method via a broad-based licensing effort. Alnylam announced today that its initial cERD license has been provided to Isis Pharmaceuticals.

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 leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, ALN-APC for the treatment of hemophilia, and ALN-TMP for the treatment of hemoglobinopathies. As part of its “Alnylam 5x15TM” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in clinical development, including programs in advanced stages, on its own or with a partner by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and Sanofi. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for applications in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam’s VaxiRNA™ platform applies RNAi technology to improve the manufacturing processes for vaccines; GlaxoSmithKline is a collaborator in this effort. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.

Alnylam Forward-Looking Statements

Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including, without limitation, statements regarding its expectations for the use of its cERD method and its potential impact of the development of RNAi therapeutics, in addition to its potential applications with other therapeutic modalities, its plans to enable use of its new technology in academic and industry research efforts with licenses, and its “Alnylam 5x15” product strategy, 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, without limitation, Alnylam’s ability to discover and develop novel drug candidates and delivery approaches, successfully demonstrate the efficacy and safety of its drug candidates, which may not support further development of product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, Alnylam’s ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, obtaining regulatory approval for products, competition from others using technology similar to Alnylam’s and others developing products for similar uses, Alnylam’s ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, including new licenses relating to its cERD method, Alnylam’s dependence on third parties for access to intellectual property, and the development, manufacture, marketing, sales and distribution of products, and the outcome of litigation, 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.

Source: Alnylam Pharmaceuticals, Inc.

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"Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995: Statements in this press release regarding Alnylam Pharmaceuticals's business which are not historical facts are "forward-looking statements" that involve risks and uncertainties. For a discussion of such risks and uncertainties, which could cause actual results to differ from those contained in the forward-looking statements, see "Risk Factors" in the Company's Annual Report or Form 10-K for the most recently ended fiscal year.