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Santaris Pharma A/S Advances RNA-Targeted Drug Development Candidate Against PCSK9, an Important New Target for the Treatment of High Cholesterol
Date:4/26/2010

ompound miravirsen, the International Nonproprietary Name (INN) or generic name for the compound formerly known as SPC3649.

Miravirsen, the first microRNA-targeted drug to enter human clinical trials, is a specific inhibitor of miR-122, a liver-specific microRNA that the Hepatitis C virus requires for replication. Unlike Hepatitis C therapies that directly target the virus, miravirsen works by removing a "helper" molecule (miR-122) that the virus needs in order to make new copies. Miravirsen is being studied in a Phase 1 trial multiple-ascending dose study in healthy volunteers and a Phase 2 study in patients infected with Hepatitis C is expected to begin in the second half of 2010.

Santaris Pharma A/S also has a broad range of drug discovery and development programs with its partners: Shire plc (lead candidates against up to five targets for rare genetic disorders), Pfizer (lead candidates against up to ten targets), GlaxoSmithKline (options to drug candidates from up to four viral disease programs) and Enzon Pharmaceuticals (lead candidates against eight cancer targets successfully delivered).

About Locked Nucleic Acid (LNA) Drug Platform

The LNA Drug Platform utilizing Santaris Pharma A/S proprietary LNA chemistry provides the key to delivering on the promise of RNA-targeted therapies today by addressing these concerns and overcoming the limitations of earlier antisense and siRNA technologies to deliver potent single-stranded LNA-based drug candidates across a multitude of disease states. The unique combination of small size and very high affinity, which is only achievable with LNA-based drugs, allows this new class of drugs to potently and specifically inhibit RNA-targets in many different tissues without the need for complex delivery vehicles. LNA-based drugs are a promising new type of therapy that enables scientists to develo
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SOURCE Santaris Pharma A/S
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5. Santaris Pharma A/S Forms Strategic Alliance With Shire plc to Develop RNA-Based Medicines for the Treatment of Rare Genetic Disorders
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