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Tiny Locked Nucleic Acid-based compounds inhibit entire disease-associated microRNA families

May 17, 2017

The study published in Nature Genetics was carried out by Santaris Pharma A/S scientists and collaborators at Cold Spring Harbor Laboratory, New York. In this study, scientists demonstrated that tiny LNA-based compounds inhibited both single microRNAs and entire microRNA families in cultured cells, as well as in vivo in several mice tissues and in a mouse breast tumor model. The tiny LNA-based compounds were well tolerated by the mice and could be delivered without the use of complex delivery vehicles.

The Santaris Pharma A/S LNA Drug Platform is the only RNA technology with both mRNA and microRNA targeted drugs in clinical trials, demonstrating the broad utility of the proprietary platform. In September 2010, Santaris Pharma A/S successfully advanced miravirsen, a lead microRNA drug candidate targeting miR-122, into Phase 2 studies for the treatment of patients infected with the Hepatitis C virus. In addition, Santaris Pharma A/S is advancing two mRNA-targeted drugs, SPC5001 targeting PCSK9 and SPC4955 targeting apoB, for the treatment of high cholesterol into Phase 1 in the first half of 2011.

Santaris Pharma A/S also has a robust product pipeline with its partners consisting of mRNA and microRNA drug discovery and development collaborations. These include partnerships with Pfizer, Inc. (delivery of lead candidates against up to 20 targets), miRagen Therapeutics (cardiovascular diseases), Shire plc (rare genetic disorders), GlaxoSmithKline (four viral disease drug candidates) and Enzon Pharmaceuticals (eight cancer targets successfully delivered - three are now in Phase 1 clinical studies).

SOURCE Santaris Pharma