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New Data Shows Breakthrough microRNA-Targeted Therapy Developed Using Santaris Pharma A/S Proprietary LNA Technology Holds Promise as New Treatment for Hepatitis C
Date:12/3/2009

C3649 inhibits miR-122, an important microRNA involved in HCV replication, the HCV is blocked from replicating without the apparent selection of resistant mutants. SPC3649 has other important properties that make it attractive as a therapeutic agent for HCV. The preclinical data show changes in the expression of key genes that may help patients who do not respond to interferon treatment to become responsive.

SPC3649 is the first microRNA-targeted drug to enter human clinical trials and is currently undergoing Phase 1 clinical studies in healthy volunteers. These preclinical data provide even greater impetus to further examine the potential of SPC3649 for treating patients infected with HCV.

"Advancing the first microRNA-targeted therapy, SPC3649, into human clinical trials was certainly a breakthrough in science and we are very encouraged by these preclinical findings demonstrating that SPC3649 has the potential to be an effective treatment for patients infected with the Hepatitis C virus," said Henrik 0rum, PhD, Vice President and Chief Scientific Officer of Santaris Pharma A/S. "In drug discovery and development programs internally and with our partners, we continue to demonstrate that our proprietary LNA Drug Platform is fundamental in developing effective RNA-targeted therapies with high affinity, target specificity and remarkable potency for a range of diseases."

Santaris Pharma A/S has a robust product pipeline based on its proprietary LNA technology including mRNA and microRNA drug discovery and development partnerships and collaborations with Shire (rare genetic disorders), Wyeth, now part of Pfizer, (delivery of lead candidates against up to ten targets), GlaxoSmithKline (four viral disease drug candidates) and Enzon Pharmaceuticals (eight cancer targets successfully delivered).

About Locked Nucleic Acid (LNA) Drug Platform

The Locked Nucleic Acid (LNA) Drug Platform developed b
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