LA JOLLA, Calif., June 6, 2011 – Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced publication in the Journal of Clinical Investigation of new pre-clinical data in mice on the antagonism of microRNA-33 (miR-33). The study, performed with collaborators at NYU Langone Medical Center, demonstrated that antagonism of miR-33 with proprietary chemically modified anti-miR oligonucleotides can promote clearance of excess cholesterol and statistically significant regression of atherosclerosis in mice with established atherosclerotic plaques.
Recent advances in lipid metabolism have identified miR-33 as a "master switch" of cholesterol transport genes, such as ATP-binding cassette transporter A1 (ABCA1), a regulator of high density lipoprotein cholesterol (HDL-C), or 'good' cholesterol. Inhibition of miR-33 results in increased ABCA1 expression and elevations in HDL-C, suggesting that miR-33 antagonism may be atheroprotective [Rayneret al. Science 328, 1570 (2010)]. In this new study, in collaboration with Kathryn Moore, Ph.D., associate professor in the Department of Medicine at NYU Langone Medical Center, the impact of miR-33 inhibition was assessed in mice with established atherosclerotic plaques. Treating mice with anti-miR-33 led to increased HDL-C, enhanced reverse cholesterol transport to the plasma, liver and feces, and reductions in plaque size and lipid content.
"We are encouraged by the continuing progress being made on our miR-33 program and the ongoing work done in collaboration with Dr. Moore's lab at NYU Langone," said Hubert Chen, M.D., vice president of translational medi