Alnylam to Participate in Major Cardiovascular Disease Research Grant Awarded to Scientists at University of Pennsylvania

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today its participation in a $2.4 million research grant awarded to the University of Pennsylvania under the "American Recovery and Reinvestment Act of 2009”. The new grant will fund research over two years in the laboratories of Daniel Rader, M.D. of the University of Pennsylvania School of Medicine, Sekar Kathiresan, M.D. of Massachusetts General Hospital (MGH) and Harvard Medical School, and Alnylam.

The goal of this initiative is to mechanistically evaluate the metabolic and molecular effects of 38 novel genes implicated by human genetic studies in prevention of cardiovascular disease and to discover and develop RNAi therapeutics towards these gene candidates. Through an agreement with the University of Pennsylvania and MGH, Alnylam will retain the rights to develop and commercialize RNAi therapeutics from the research effort.

"We are excited to participate in this important work led by Dr. Dan Rader of the University of Pennsylvania, who is widely recognized for his outstanding work in lipid research and lipoprotein metabolism,” said Victor Kotelianski, M.D., Ph.D., D.Sc., Senior Vice President, Distinguished Alnylam Fellow. "We are also pleased to be collaborating in this research effort with Dr. Kathiresan of MGH, where we will be able to interrogate the findings of large-scale genome-wide association studies in cardiovascular disease, including genes associated with abnormal blood lipid levels and early onset myocardial infarction. As evidenced by our RNAi therapeutic program targeting PCSK9, cardiovascular disease remains an area of great interest for Alnylam as we believe there is significant potential for an RNAi therapeutic to make an impact in the lives of patients.”

"We are honored to be awarded this new grant as part of the NIH stimulus,” said Rader, the Cooper-McClure Professor of Medicine at the University of Pennsylvania School of Medicine. "Together with my team at the University of Pennsylvania and my collaborators at the Massachusetts General Hospital and the Broad Institute, I am looking forward to working with Alnylam as leaders in the RNAi therapeutics field. Our knowledge of lipid disorders and their association with human disease combined with Alnylam’s expertise of development of RNAi therapeutics represents a powerful and rare opportunity to advance new innovative medicines.”

The work conducted under this grant intends to validate novel genes as targets for new therapies for lipid disorders and heart disease and will establish early proof of concept on how genetic association studies can yield important new insights into biology that have the potential to impact cardiovascular disease. Coronary heart disease is the leading cause of mortality in both men and women worldwide and its incidence is highly correlated with levels of cholesterol and triglycerides in the blood. Plasma concentrations of low-density lipoprotein (LDL, or "bad” cholesterol) and high-density lipoprotein (HDL, or "good” cholesterol), as well as triglycerides, have a strong inherited basis and recent genetic association studies have linked previously unsuspected genes to these complex traits.

The project described was supported by Award Number RC2HL101864 from the National Heart, Lung, And Blood Institute. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Heart, Lung, And Blood Institute or the National Institutes of Health.

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; its most advanced program is in Phase II human clinical trials for the treatment of respiratory syncytial virus (RSV) infection and is partnered with Cubist and Kyowa Hakko. In addition, the company is developing RNAi therapeutics for the treatment of a wide range of disease areas, including liver cancers, hypercholesterolemia, Huntington’s disease, and TTR amyloidosis. 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, and Cubist. To reflect its outlook for key scientific, clinical, and business initiatives, Alnylam established "RNAi 2010” in January 2008 which includes the company’s plan to significantly expand the scope of delivery solutions for RNAi therapeutics, have four or more programs in clinical development, and to form four or more new major business collaborations, all by the end of 2010. Alnylam is a joint owner of Regulus Therapeutics, a joint venture focused on the discovery, development, and commercialization of microRNA therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit http://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 the company's ability to successfully research and develop products and to successfully prosecute and enforce its patents around the world, 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.