This week in therapeutics

Indication

Target/marker/pathway

Summary

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Publication and contact information

Cardiovascular disease

Cardiovascular disease

MicroRNA-33a (miR-33a);
miR-33b

Mouse and primate studies suggest an 8mer anti-miRNA that targets both miR-33a and miR-33b could help treat cardiovascular disease. miR-33a and miR-33b inhibit the expression of genes involved in lipid metabolism and insulin signaling. In mice, an 8mer anti-miRNA targeting the seed regions of miR-33a and miR-33b (anti-miR-33a/b) increased high-density lipoprotein (HDL) cholesterol compared with a scrambled anti-miRNA. In nonhuman primate models of metabolic disease, anti-miR-33a/b increased HDL cholesterol up to 39% compared with vehicle or anti-miRNAs targeting either miR-33a or
miR-33b. In primates treated for more than 100 days, there were no adverse events, injection site reactions, liver inflammation or fibrosis. Next steps include toxicology studies in rodents and nonhuman primates.
Regulus Therapeutics Inc. and AstraZeneca plc have an anti-miRNA targeting miR-33 in preclinical testing to treat cardiovascular disease (see Family seeds, page 8).

SciBX 6(47); doi:10.1038/scibx.2013.1349
Published online Dec. 12, 2013

Patent application filed covering therapeutic targeting of
miR-33a and miR-33b for treatment of lipid-related disorders; available for licensing

Rottiers, V. et al. Sci. Transl. Med.; published online Nov. 20, 2013;
doi:10.1126/scitranslmed.3006840
Contact: Anders M. Näär, Massachusetts General Hospital Cancer Center, Charlestown, Mass.
e-mail:
naar@helix.mgh.harvard.edu

Contact: Sakari Kauppinen, Santaris Pharma A/S, Hørsholm, Denmark
e-mail:
sk@bio.aau.dk