Alnylam Reports Progress for Systemic Delivery of RNAi Therapeutics; Results Presented at 2nd International Symposium on Triglycerides and HDL.CAMBRIDGE, Mass. -- Alnylam Pharmaceuticals Alnylam Pharmaceuticals Inc. (NYSE: ALNY), a biopharmaceutical company, engages in the development and commercialization of therapeutic products based on RNA interference (RNAi) in the United States. , Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced further progress on its research in systemic delivery of RNAi therapeutics targeting apolipoprotein B (apoB), a disease-associated gene in hypercholesterolemia Hypercholesterolemia Definition Hypercholesterolemia refers to levels of cholesterol in the blood that are higher than normal. Description Cholesterol circulates in the blood stream. It is an essential molecule for the human body. , which it presented at the 2nd International Symposium on Triglycerides Triglycerides Fatty compounds synthesized from carbohydrates during the process of digestion and stored in the body's adipose (fat) tissues. High levels of triglycerides in the blood are associated with insulin resistance. and HDL (Hardware Description Language) A language used to describe the functions of an electronic circuit for documentation, simulation or logic synthesis (or all three). Although many proprietary HDLs have been developed, Verilog and VHDL are the major standards. in New York New York, state, United States New York, Middle Atlantic state of the United States. It is bordered by Vermont, Massachusetts, Connecticut, and the Atlantic Ocean (E), New Jersey and Pennsylvania (S), Lakes Erie and Ontario and the Canadian province of . Alnylam scientists reported an improved dosage regimen for the systemic delivery of short interfering RNAs (siRNAs) that silence the gene for apoB. The siRNAs, the molecules that mediate RNA interference RNA interference n. A process in which the introduction of double-stranded RNA into a cell inhibits the expression of genes. (RNAi), were chemically modified and optimized by Alnylam researchers. Further reported data included the demonstration of siRNA-mediated therapeutic efficacy in an animal model of hypercholesterolemia. "These data represent important progress in the advancement of systemic RNAi therapeutics towards clinical development," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam Pharmaceuticals. "We have been able to demonstrate silencing of an endogenous, disease-associated gene using a chemically modified and optimized siRNA at significantly lower doses than in our previous landmark study." The new pre-clinical dosing studies in mice used a lower daily dose regimen (5 mg/kg) of the systemically delivered siRNA to achieve a 60% silencing of apoB in the target organ target organ n. A tissue or organ that is affected by a specific hormone. target organ, n the organ or body part whose activity levels demonstrate change in the course of biofeedback. . Overall doses of siRNA in this regimen were 6-fold lower than those published by Alnylam scientists in Nature in November 2004. Alnylam's Nature article was the first-ever published 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. demonstration of RNAi-mediated silencing of an endogenous gene in mammals by a method applicable to systemic RNAi therapeutics for human disease. Systemic delivery of siRNAs through the bloodstream potentially enables RNAi therapeutics to be developed for a broad range of diseases. About RNA Interference (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 and regulate such genes selectively through RNAi could provide a means to treat a wide range of human diseases. The discovery of RNAi has been heralded by many as a major breakthrough, and the journal Science named RNAi the top scientific achievement of 2002, as well as one of the top 10 scientific advances of 2003. About Alnylam Alnylam is a biopharmaceutical company seeking to develop and commercialize novel therapeutics based on RNA interference, or RNAi. Growing from its foundation as the world's first company focused on RNAi therapeutics, 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 therapeutics. Alnylam is developing a pipeline of RNAi products using Direct RNAi(TM) to treat ocular, central nervous system, and respiratory diseases and Systemic RNAi(TM) to treat a broad range of diseases, including oncology, metabolic, and autoimmune diseases Autoimmune diseases A group of diseases, like rheumatoid arthritis and systemic lupus erythematosus, in which immune cells turn on the body, attacking various tissues and organs. Mentioned in: Complement Deficiencies, Premature Menopause . The company's global headquarters are in Cambridge, Massachusetts. For additional information, please visit www.alnylam.com. Alnylam Forward-Looking Statements Various statements in this release concerning our future expectations, plans, prospects and future operating results constitute forward-looking statements for the purposes of the safe harbor Safe Harbor 1. A legal provision to reduce or eliminate liability as long as good faith is demonstrated. 2. A form of shark repellent implemented by a target company acquiring a business that is so poorly regulated that the target itself is less attractive. 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; 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 Form 10-Q Form 10-Q See 10-Q. filed 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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