Nature Publishes Santaris Pharma Study on microRNA Silencing

A study by scientists and collaborators on LNA-mediated microRNA silencing in non-human primates has been published in Nature's Advance Online Publication.

The groundbreaking peer-reviewed study is the first demonstration of microRNA silencing in non-human primates, providing an important validation of Santaris Pharma's emerging clinical program to develop a novel class of LNA-based therapeutics capable of silencing disease-associated microRNAs.

The study authors was led by Dr Sakari Kauppinen, Director of MicroRNA Research at Santaris Pharma and visiting Professor at Wilhelm Johannsen Centre for Functional Genome Research, University of Copenhagen.

- Even though further studies will be needed to optimize the dosing regimen and to assess the safety of LNA-antimiR compounds after long-term treatment, our findings represent an important step towards the development of LNA-based microRNA therapeutics, says Kauppinen.

Human Testing in 2008

MicroRNAs are small regulatory RNAs that play important roles in development and disease and, thus, represent a potential new class of targets for therapeutic intervention. Santaris Pharma has the proprietary worldwide pharmaceutical rights to the LNA technology, which was employed in the study published by Nature to target microRNAs. The use of LNA significantly enhances the potency of microRNA antagonists, while retaining target specificity, which is essential in silencing of specific microRNAs.

Santaris Pharma is preparing to advance its first LNA-antimiR compound, targeting miR-122, into human clinical testing in the first half of 2008. The first trial will be a Phase I safety and pharmacokinetic study in healthy volunteers.

Read the full press release here or read the publication in Nature here.

Source: Press release, Santaris Pharma, 26.03.2008