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RxGen and Santaris Pharma Use LNA-antimiR Technology to antagonize the liver-expressed microRNA-122 in Non-Human Primates and to Safely Lower Serum Cholesterol

HAMDEN, Conn. & H0RSHOLM, Denmark--(BUSINESS WIRE)--Mar 27, 2008 - In a study published in the journal Nature today, Santaris Pharma and RxGen Inc., a specialty pharma services company demonstrated that a systemically delivered Locked Nucleic Acid (LNA) compound could safely and effectively silence a microRNA target in African green monkeys. The study shows that a specific LNA-antimiR that binds to the liver-specific microRNA-122 can readily enter the liver and antagonize the miRNA target molecule. Remarkably, administration of just three doses over the course of one week markedly altered serum lipid profiles for 7 weeks, resulting in a significant decrease in total cholesterol, with no evidence of adverse toxicity.

"LNA-antimiR compounds are an effective approach to silence microRNAs in primates," said RxGen President & CSO Matthew Lawrence. "We are all very excited to see how experimentally straightforward and safe it was to achieve a sustained and pronounced physiologic change with silencing of the liver-specific microRNA-122 using Santaris Pharma's LNA technology."

This is the first demonstration that microRNAs can be silenced in non-human primates and suggests that they might represent a new class of therapeutic targets in humans. Moreover, the demonstration that unconjugated LNA compounds are stable and active in non-human primates opens many possibilities for targeting microRNAs and mRNAs to explore biological pathways and therapeutic targets.

"This study is very exciting for both companies in terms of demonstrating the power and utility of LNA for the development of novel therapeutic approaches based on disease-associated microRNAs," commented Sakari Kauppinen, Director of MicroRNA Research at Santaris Pharma and visiting Professor at Wilhelm Johannsen Centre for Functional Genome Research, University of Copenhagen.

About RxGen

RxGen, Inc. is a privately held biotechnology and preclinical research organization based in New Haven, CT, providing high value animal modeling services related to central nervous system, metabolism, and ophthalmic related disorders to the pharmaceutical and biotechnology industries. In addition to providing specialty CRO services, RxGen leverages its unique resources and capabilities to validate novel therapeutic products and technologies on a contract and partnership basis. For more information about RxGen, please visit www.rx-gen.com.

About Santaris Pharma

Santaris Pharma is a Danish clinical stage biopharmaceutical company. The company was formed in 2003 and currently employs 105 people. Santaris Pharma has the exclusive rights to LNA technology, a 3rd generation antisense chemistry used to develop new classes of RNA medicines, called RNA antagonists and microRNA antagonists. Santaris Pharmas RNA antagonists and microRNA antagonists are being developed to silence RNAs associated with cancer, metabolic disorders and viral diseases. Santaris Pharma completed a Euro 40m second round of equity financing in May 2006 and a Euro 20m third round of equity financing in December 2007. Santaris Pharma has a global alliance with Enzon Pharmaceuticals of New Jersey to develop and co-commercialise a series of Santaris Pharma RNA antagonists for the treatment of cancer and a worldwide strategic alliance with GlaxoSmithKline for the discovery, development and commercialisation of novel medicines against viral diseases.

Contact

RxGen
Christopher Stanley, 512-897-7020
Director, Corporate Development
cstanley@rx-gen.com
or
Santaris Pharma
Randi Krogsgaard, +45 45179879
Director, Corporate Communications
rmk@santaris.com

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