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AstraZeneca buys miRNA know how
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AstraZeneca buys miRNA know how

16.08.2012 - British-Swedish pharma giant AstraZeneca has inlicenced three miRNA targets from Regulus Therapeutics.

AstraZeneca said it will make an US$28m upfront payment and equity investment for the exclusive commercialisation rights for compounds directed against three miRNA targets identified by the US joint venture of Alnylam and Isis Pharmaceuticals. The deal includes Regulus’ lead programme that targets miRNA33 in atherosclerosis. Three other programmes will be focused on cardiovascular (miRNA24) and metabolic diseases as well as cancer. Under the terms of the agreement, Regulus will lead preclinical development, whereas AstraZeneca will be responsible for clinical development and commercialisation. In case of success, Regulus will receive preclinical and clinical milestones and royalties.

Atherosclerosis is the build-up of plaque that occurs when cholesterol and inflammatory cells accumulate in blood vessels. These plaques can rupture, leading to slowing or blockage of blood flow and ultimately resulting in a heart attack or stroke. Scientific research has shown a strong correlation between high cholesterol levels and cardiovascular disease which, according to the Centers for Disease Control, is the leading cause of death in the United States.

Regulus’ lead program for atherosclerosis targets microRNA-33, which has a unique mechanism of action for the management of cholesterol levels. The inhibition of microRNA-33 with our anti-miRs promotes reverse cholesterol transport, or RCT, which is the efflux of cholesterol from specific cholesterol-laden inflammatory cells called macrophages in atherosclerotic plaques. Treatment with anti-microRNA-33 in an atherosclerotic mouse model led to reduction in arterial plaque size by 35% (Rayner et al., J Clin Invest. 2011) and treatment in non-human primates increased circulating levels of HDL-C by 50%. By enhancing RCT, anti-miR-33 differs from other emerging therapeutic strategies that focus only on raising HDL-C in circulation.

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