Alnylam Announces Progress in Pre-clinical Programs at the 35th Annual Society for Neuroscience Meeting; Pre-clinical Data from Parkinson's Disease, Huntington's Disease, and Neuropathic Pain Programs.CAMBRIDGE, Mass. -- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it presented pre-clinical data at the 35th Annual Society for Neuroscience For other uses, see SFN (disambiguation). The Society for Neuroscience (SfN) is a professional society for basic scientists and physicians around the world whose research is focused on the study of the brain and nervous system. Meeting being held in Washington, D.C. from November 12-16, 2005. Alnylam and its academic collaborators presented data from three ongoing neurology programs focused on Parkinson's disease Parkinson's disease or Parkinsonism, degenerative brain disorder first described by the English surgeon James Parkinson in 1817. When there is no known cause, the disease usually appears after age 40 and is referred to as Parkinson's disease. , Huntington's disease Huntington's disease, hereditary, acute disturbance of the central nervous system usually beginning in middle age and characterized by involuntary muscular movements and progressive intellectual deterioration; formerly called Huntington's chorea. , and neuropathic pain. "Data emerging from the neuroscience community continues to point to the potential of RNAi therapeutics for the treatment of neurological diseases," said Victor Kotelianski, M.D., Ph.D., Vice President of Research at Alnylam Pharmaceuticals. "Results from our ongoing pre-clinical studies support the strategy of using siRNAs to treat these diseases. We are excited about the data presented at the Society for Neuroscience meeting, which we believe support an RNAi approach for neurological diseases and demonstrate the progress that Alnylam continues to make in this area." Parkinson's Disease Alnylam has designed and synthesized siRNAs that are specific to the alpha-synuclein gene, a gene associated with Parkinson's disease. The siRNAs are stable in cerebrospinal fluid cerebrospinal fluid (CSF) Clear, colourless liquid that surrounds the brain and spinal cord and fills the spaces in them. It helps support the brain, acts as a lubricant, maintains pressure in the skull, and cushions shocks. , and potently silence the alpha-synuclein gene in neurons in vitro in vitro /in vi·tro/ (in ve´tro) [L.] within a glass; observable in a test tube; in an artificial environment. in vi·tro adj. In an artificial environment outside a living organism. . In collaboration with Dr. Matthew Farrer and Dr. Jada Lewis at Mayo Clinic, these siRNAs are now being tested in animal models to examine alpha-synuclein silencing in vivo in vivo /in vi·vo/ (ve´vo) [L.] within the living body. in vi·vo adj. Within a living organism. in vivo adv. . Preliminary data show that the siRNAs are taken up by central nervous system neurons in vivo and can be transported to brain regions distant from the site of injection, which could be beneficial for the treatment of Parkinson's disease. Huntington's Disease In collaboration with the laboratory of Dr. Neil Aronin, Alnylam scientists applied proprietary technology to siRNAs identified by Dr. Aronin and Dr. Philip Zamore, both of the University of Massachusetts Medical School UMMS is ranked fourth in primary care education among the nation’s 125 medical schools in the 2006 U.S.News & World Report annual guide, “America’s Best Graduate Schools”. UMMS is also a major center for research. , that silence huntingtin, the gene that is mutated in Huntington's disease. In this pre-clinical study, the siRNAs administered into the brain were taken up by the same type of neurons that are most affected in the early stages of Huntington's disease. Ongoing studies are examining the effect these siRNAs have in inhibiting the disease process in animal models of Huntington's disease. Neuropathic Pain Neuropathic pain is chronic pain associated with damage to the nervous system and can be attributable to a range of causes including lower back injury, diabetic neuropathy Diabetic Neuropathy Definition Diabetic neuropathy is a nerve disorder caused by diabetes mellitus. Diabetic neuropathy may be diffuse, affecting several parts of the body, or focal, affecting a specific nerve and part of the body. , cancer treatment, and shingles. Data were presented demonstrating that siRNAs can be used to silence targets associated with neuropathic pain, potentially providing pain relief. In the first pre-clinical studies, the laboratories of Dr. Frank Porreca and Dr. Josephine Lai at the University of Arizona (body, education) University of Arizona - The University was founded in 1885 as a Land Grant institution with a three-fold mission of teaching, research and public service. evaluated siRNAs that target the gene for NPY NPY neuropeptide-Y. , a pain-associated peptide. The set of siRNAs were designed at the University of Arizona and Alnylam with modifications using proprietary technology at Alnylam. An siRNA was then administered by intrathecal intrathecal /in·tra·the·cal/ (-the´k'l) within a sheath; through the theca of the spinal cord into the subarachnoid space. Intrathecal injection, and over the course of treatment, results showed significant pain relief. In a second study, siRNAs were designed by Alnylam to target a gene, NaV1.8, which encodes an ion channel ion channel n. See channel. . NaV1.8 is recognized to play a significant role in chronic neuropathic pain, but has proved extremely difficult to inhibit selectively with traditional small molecule drugs. The pre-clinical data demonstrated that siRNAs targeting NaV1.8 administered by intrathecal injection can provide nearly complete pain relief. About RNA Interference RNA interference n. A process in which the introduction of double-stranded RNA into a cell inhibits the expression of genes. (RNAi) RNA interference, or RNAi, is a naturally occurring mechanism within cells for selectively silencing and regulating specific genes. Since many diseases are caused by the inappropriate activity of specific genes, the ability to silence genes selectively through RNAi could provide a new way to treat a wide range of human diseases. RNAi is induced by small, double-stranded RNA RNA: see nucleic acid. RNA in full ribonucleic acid One of the two main types of nucleic acid (the other being DNA), which functions in cellular protein synthesis in all living cells and replaces DNA as the carrier of genetic molecules. One method to activate RNAi is with chemically synthesized small interfering RNAs, or siRNAs, which are double-stranded RNAs that are targeted to a specific disease-associated gene. The siRNA molecules are used by the natural RNAi machinery in cells to cause highly targeted gene silencing. About Alnylam Alnylam is a biopharmaceutical company developing novel therapeutics based on a breakthrough in biology known as RNA interference, or RNAi. The company, founded in 2002 by scientific pioneers in the field of RNAi, maintains a leadership position in fundamental patents, technology, and know-how relating to RNAi. Alnylam is applying its expertise in RNAi to address multiple therapeutic opportunities that cannot effectively be addressed with small molecules or antibodies, the two current major classes of drugs. The company's expertise in designing and optimizing RNAi therapeutics has enabled Alnylam to form major alliances with leading companies including Merck, Medtronic, and Novartis. The company's global headquarters are in Cambridge, Massachusetts, with an additional operating unit operating unit A type of operating company that engages in transactions with outsiders and that is owned by another business. For example, in 1995 the stockholders of Capital Cities/ABC approved a $19 billion merger with the Walt Disney Company, whereupon in Kulmbach, Germany. For more information, please visit www.alnylam.com. Alnylam Forward-Looking Statements Various statements in this release concerning our future expectations, plans and prospects, including our views with respect to the potential of using siRNAs to treat neurological diseases, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act The Private Securities Litigation Reform Act of 1995 (PSLRA) implemented several significant substantive changes affecting certain cases brought under the federal securities laws, including changes related to pleading, discovery, liability, class representation and awards fees and 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: our approach to discover and develop novel drugs, which is unproven and may never lead to marketable products; our ability to obtain additional funding to support our business activities; our dependence on third parties for development, manufacture, marketing, sales and distribution of our products; the successful development of products, all of which are in early stages of development; obtaining regulatory approval for products; competition from others using technology similar to ours and others developing products for similar uses; obtaining, maintaining and protecting intellectual property utilized by our products; our dependence on collaborators; and our short operating history; as well as those risks more fully discussed in the "Certain Factors That May Affect Future Results" section of our most recent quarterly report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We do not assume any obligation to update any forward-looking statements. |
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