Alnylam and Collaborators Receive LEAPS Award from Michael J. Fox Foundation for Parkinson's Research; Enter into Research Collaboration

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that it was part of a research team awarded a new $3.8 million grant from The Michael J. Fox Foundation (MJFF) to further develop an RNAi therapeutic for the treatment of Parkinson’s disease. The four-year grant, which is part of the Foundation’s LEAPS (Linked Efforts to Accelerate Parkinson’s Solutions) initiative, was awarded to Alnylam, Mayo Clinic of Jacksonville, Florida, and the Parkinson’s Institute and Clinical Center of Sunnyvale, California. MJFF fosters collaboration between industry and academia through its LEAPS program as one strategy to accelerate progress toward the development of new and improved interventions for Parkinson’s disease. "We are excited to be continuing our work with Mayo Clinic and the Parkinson’s Institute that builds upon a MJFF Target Validation award granted to Alnylam in 2005. We look forward and remain committed to advancing this therapeutic strategy to patients,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We believe that RNAi therapeutics, due to their novel mechanism of action, may have a significant impact in the treatment of Parkinson’s disease and address this neurodegenerative disorder in a fundamentally new way.” "Available Parkinson’s treatments mask symptoms but do nothing to halt or slow underlying disease progression,” said Katie Hood, chief executive officer of MJFF. "More and more scientific evidence supports the hypothesis that lowering alpha-synuclein levels in the brain could achieve the so-called ‘Holy Grail’ of PD research, a neuroprotective therapy. But no drugs have been identified to date that are capable of reducing alpha-synuclein expression; new approaches are needed. This LEAPS grant is characteristic of how The Michael J. Fox Foundation goes about its work – making big bets on fresh ideas with great potential to impact patients’ quality of life.” The LEAPS award will fund research focused on the further development of an RNAi therapeutic targeting alpha-synuclein. Alpha-synuclein may play a role in the development of Parkinson’s disease. Continued research will look at whether reducing levels of alpha-synuclein in the brain can slow or halt disease progression. Alnylam and collaborators have designed and synthesized small interfering RNAs (siRNAs), the molecules that mediate RNAi, that are specific to the alpha-synuclein gene. Previously reported in vivo data show that siRNAs administered into the brain were effective in reducing alpha-synuclein levels. To date, no drugs have been identified that are capable of lowering alpha-synuclein levels in the brain. These data suggest the applicability of RNAi therapeutics as a possible disease-modifying therapy for Parkinson’s disease. "With this generous and important grant we share an exciting optimism regarding RNAi therapeutics. This completely novel approach may well be the next big step toward releasing people with Parkinson’s from the disabilities they endure every day,” said J. William Langston, M.D., Scientific Director and Chief Executive Officer, Parkinson’s Institute and Clinical Center. "We are proud to join in taking this major scientific leap forward.” 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. RNAi therapeutics target the cause of diseases by potently silencing specific messenger RNAs (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. 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. Alnylam is a joint owner of Regulus Therapeutics LLC, 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, visit www.alnylam.com. About The Michael J. Fox Foundation Founded in 2000, The Michael J. Fox Foundation for Parkinson’s Research is dedicated to ensuring the development of a cure for Parkinson’s disease within this decade through an aggressively funded research agenda. The Foundation has funded approximately $110 million in research to date. Alnylam Forward-Looking Statements 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 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.