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Parkinson's Disease to be Targeted by Novel RNA Interference Drug Development CAMBRIDGE, Mass., and ROCHESTER, Minn., October 30, 2003 -Alnylam Pharmaceuticals, Inc., the leading therapeutic RNA interference (RNAi) company, today announced the formation of a research collaboration to apply its proprietary RNAi- based therapeutic technology to a discovery by researchers at Mayo Clinic and NIH of a causal pathway in Parkinson's disease. Through the collaboration, Alnylam will provide RNAi technology and fund research at Mayo Clinic to develop a drug that suppresses the expression of a specific gene, alpha-synuclein, found to be over-expressed in Parkinson's patients. The findings are published in the current issue of Science. Under the terms of the research collaboration, Alnylam will identify, synthesize and provide RNAi-based drug compounds targeted to the alpha synuclein gene expression. Mayo Clinic will test and select the RNAi compounds for their efficacy in a series of in vitro and in vivo studies. "This collaboration with Alnylam creates the possibility of previously unimagined therapeutic advances for Parkinson's patients," says Matthew Farrer, Ph.D., Assistant Professor of Neuroscience and the director of a neurogenetics laboratory at Mayo Clinic in Jacksonville, Fla. "RNA interference is a promising technology that offers the potential to halt progression of Parkinson's disease through a targeted mechanism of action." Demetrius Maraganore, M.D., Professor of Neurology at Mayo Clinic in Rochester, Minn., adds "Alpha-synuclein gene over-expression is the 'Rosetta stone' of Parkinson's disease. We anticipate that therapy to reduce alpha-synuclein gene expression will benefit not only Parkinson's disease patients who carry the rare gene mutation, but also persons who carry common susceptibility variants of the gene, or persons who aggregate the alpha-synuclein protein via other genetic and non-genetic mechanisms." "The groundbreaking research of Mayo Clinic offers us an exciting platform to apply RNA interference to silence a specific gene pathway in Parkinson's disease," says Thomas Ulich, M.D., Senior Vice President of Research and Development at Alnylam. "For this collaboration we have targeted Parkinson's disease. This is one of many diseases in which we plan to apply RNAi-based therapies, to create a new class of medicines for patients." Alpha Synuclein Mechanism Alpha synuclein is found in various body tissues, primarily in the brain, where scientists believe it may play a role in synaptic vesicle recycling (how nerve cells transmit their signals). The findings in Science highlight that simple over-expression of normal, wild-type alpha-synuclein is sufficient to cause disease in a family with multiple affected members. Previous work by the Mayo Clinic group, published in Human Molecular Genetics, demonstrated that over- expression of wild-type alpha-synuclein may confer susceptibility in Parkinson's disease patients population-wide. Parkinson's Disease Parkinson's disease is a serious disorder that affects nerve cells (neurons) in the part of the brain controlling muscle movement. Nearly one million Americans currently live with Parkinson's and approximately 50,000 more receive a diagnosis of the disease every year. People with Parkinson's often experience trembling, muscle rigidity, difficulty walking, and problems with balance and coordination. These symptoms generally develop after age 50, although the disease affects a small percentage of younger people as well. About Alnylam Alnylam is the leading company in the emerging field of RNA interference (RNAi) whose vision is to Harness a Revolution in Biology for Human HealthTM. Growing from its foundation as the world's first company focused on RNAi-based therapeutics, the company is built around the leading capabilities of its two operating units, Alnylam Pharmaceuticals of Cambridge, Massachusetts, and Ribopharma of Kulmbach, Germany. Over the last several years, RNAi has been identified as a fundamental cellular mechanism for gene silencing. The company's leadership in the field of RNAi is supported by its preeminent founders and advisors and its strengths in fundamental patents, technology, and know-how that underlie the commercialization of RNAi-based therapeutics. The company's focus is to discover, develop, and commercialize therapeutic products based on RNAi for a broad range of therapeutic indications including viral, oncologic, metabolic, CNS and autoimmune diseases. The company's mission is to lead research in the field of RNAi, to build the leading pipeline of RNAi-based therapeutics, and to emerge as business leaders. Its global headquarters are in Cambridge, MA. Additional information is available at www.alnylam.com. ### CONTACT Barbara Yates tel.: 781-862-1967 fax: 781-863-0933 email: [log in to unmask] SOURCE: Atlas Venture http://www.atlasventure.com/home/news_content.asp?ne_id=1726 * * * ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn