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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 and Alnylam established Regulus Therapeutics Inc., a company focused on the discovery and development of microRNA-based drugs to treat disease.

Beyond antisense, Isis scientists created another technology that exploits their knowledge of RNA. Ibis Biosciences, Inc. was created to develop and commercialize this technology. In 2009, Ibis was acquired by Abbott Molecular, which is advancing the technology into larger commercial markets including clinical diagnostics. 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, 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, a product of Isis’ innovation, provides a revolutionary approach to the discovery and detection of infectious agents with applications in many different areas including hospital-associated infections and infectious disease diagnosis. During 2008, Abbott invested $40 million in Ibis and in early 2009, Abbott purchased the remaining equity in Ibis for a total purchase price of $215 million. Isis will continue to benefit from the sale of Ibis through an earn out based on sales of Ibis instruments and assay kits.

Regulus Therapeutics Inc.

Regulus was established 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 1,600 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. To date, Isis has generated more than $334 million from licensing its intellectual property.