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美國(guó)Isis Pharmaceuticals, Inc.
位于美國(guó)加州CarIsbad的Isis制藥公司是全球反義藥物研究領(lǐng)域的領(lǐng)先公司.美國(guó)Isis制藥公司(Isis公司98年生產(chǎn)全球第一個(gè)反義核酸藥物)完成世界最大的反義核酸合成, 一次反義核酸合成量達(dá)到4KG.(600mMol)

Isis Pharmaceuticals, Inc. is a leading drug discovery and development company, focused exclusively on the therapeutic target, RNA. Founded in 1989, Isis' mission is to develop products from its RNA-based technologies, such as antisense. Very little was known about antisense when the Company was first established, but its founders envisioned an opportunity to revolutionize the pharmaceutical industry with a more efficient and targeted means of drug discovery. Since then, Isis scientists have made tremendous progress advancing the science of antisense, unveiling capabilities that have far exceeded their expectations.

Antisense drugs are the first class of drugs targeted to control expression of genes through interactions with RNA. The goal of antisense drugs is to intervene at the RNA level to prevent the production of proteins involved in disease. Antisense drugs are designed to complement and bind (hybridize) to a segment of messenger RNA (mRNA) to form a duplex. mRNA contains instructions to make proteins, including those proteins involved in disease. The formation of this duplex or two stranded molecule prevents the mRNA from functioning normally and from producing a specific protein. Antisense drugs are designed to interact specifically with the intended mRNA target. This high degree of specificity may make antisense drugs more effective and less toxic than traditional drugs.

In addition to expanding our broad pipeline of antisense drugs, our technology supports therapeutic targeting of non-messenger RNA molecules, including microRNAs. In September 2007, we formed Regulus Therapeutics LLC as a joint venture to focus on the discovery and development of microRNA-based drugs to treat disease.

Beyond antisense, Company scientists have created another technology that exploits their knowledge of RNA. Ibis Biosciences, Inc., a majority-owned subsidiary of Isis, has developed and is commercializing the Ibis T5000 Biosensor System for rapid identification and characterization of organisms.

Isis is capitalizing on its expertise through the following assets:

Antisense Research and Drug Development

Antisense drug discovery and development is the cornerstone of Isis. The Company is the leader in all aspects of antisense technology advancement, including mechanism research, the development of new chemistries, novel formulations and innovative drugs.

Gene functionalization and target validation are the first steps in drug discovery. Gene functionalization is the identification of the role a particular gene plays in disease. Target validation is the determination of a gene’s value as a drug target. The Company uses optimized, target-specific antisense inhibitors to generate this critical information. The resulting insights direct the drug discovery research of Isis.

Isis has a broad antisense pipeline with drugs in all phases of preclinical and clinical development to treat a wide variety of health conditions, including cardiovascular, metabolic, neurodegenerative, ocular and inflammatory diseases, and cancer.

Isis was granted marketing clearance for the world’s first antisense drug, Vitravene? (fomivirsen), in 1998. Vitravene is marketed by Novartis Ophthalmics.

Ibis Biosciences, Inc.

Ibis Biosciences, Inc., a majority-owned subsidiary of Isis Pharmaceuticals, has developed and is commercializing the Ibis T5000? Biosensor System for rapid identification and characterization of infectious agents. The Ibis T5000 is currently intended for research use only and not for use in diagnostic procedures. It is capable of identifying virtually all bacteria, viruses and fungi, and can provide information about drug resistance, virulence and strain type of these pathogens. Commercial applications for the Ibis T5000 Biosensor System include epidemiologic surveillance, monitoring of pandemic diseases, identification of emerging or previously unknown pathogens, forensic characterization of human samples, identification of sources of hospital-associated infections, and, in the future, human infectious disease diagnostics. Ibis develops, manufactures and markets Ibis T5000 instruments and assay kits. Ibis is a product of Isis' innovation and research that has generated more than $70 million in revenue from various government agencies to develop its Ibis T5000 Biosensor System and related assay kits.

At the beginning of 2008, Abbott invested $20 million in Ibis. In July 2008, Abbott invested an additional $20 million in Ibis, bringing Abbott total investment to $40 million. Abbott owns 18.6% of Ibis at a post money valuation of $215 million, with the option to purchase the remaining shares of Ibis by June 30, 2009. Abbott and Ibis have also entered into a distribution relationship under which Abbott will be selling Ibis products.

Regulus Therapeutics LLC

Regulus was established as a joint venture with our partners Alnylam Pharmaceuticals to discover, develop and commercialize microRNA-based therapeutics. Because microRNAs regulate whole networks of genes that can be involved in discrete disease processes, microRNA-based therapeutics represent a new approach to target the pathways of human disease. Regulus combines the strengths and assets of our and Alnylam’s technologies, know-how, and intellectual property relating to microRNA therapeutics.

Intellectual Property

With over 1,500 issued patents worldwide, Isis is the owner of one of the largest antisense and RNA patent estates in the pharmaceutical industry. The Company holds key patents covering antisense mechanisms, biology, chemistry, inhibitors of gene expression and manufacturing of antisense drugs. This patent estate is a valuable asset to the Company and its shareholders, as it protects Isis' inventions in RNA-based drug discovery and encourages revenue-generating partnerships. As of September 30, 2008, Isis has generated more than $118 million from licensing its intellectual property.
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