Mass. General Grants License for Drug Platform to Regulate Cholesterol


March 7, 2011

March 7, 2011 – Massachusetts General Hospital (MGH) has granted an exclusive license to Santaris Pharma A/S for intellectual property related to the regulation of microRNA for the treatment of cardiovascular disorders.

The company will utilize its Locked Nucleic Acid (LNA) drug platform to develop an LNA-based drug targeting miR-33, an important microRNA that regulates high density lipoprotein (HDL) levels, or “good” cholesterol.

Cholesterol levels that are out of balance or too high overall lead to the formation of atherosclerotic plaques that cause heart attacks or strokes. According to the World Health Organization, cardiovascular diseases are the number one cause of death globally and by 2030, almost 23.6 million people will die from cardiovascular diseases, mainly from heart disease and stroke.

Last year, MGH researchers published data in Science identifying tiny segments of RNA, microRNAs, which play an important role in the body's regulation of cholesterol and lipids. Their study found that the miR-33 family of microRNAs suppresses a protein known to be important for generation of HDL and for the removal of cholesterol from peripheral tissues, including cells that form atherosclerotic plaques. Data show that turning off miR-33 raises HDL levels, suggesting miR-33 as a novel target in the treatment of cardiovascular and metabolic disorders.

"Current treatments for such cholesterol abnormalities as low circulating HDL levels are only modestly effective, and there is an urgent need for new therapeutic strategies," said Anders Näär, Ph.D., of the MGH Center for Cancer Research, who led the study. "Our discovery of miR-33 as a key regulator of HDL has provided a novel therapeutic target for antisense-based technologies to ameliorate cardiometabolic disorders."

MicroRNAs have emerged as an important class of small RNAs encoded in the genome. They act to control the expression of sets of genes and entire pathways and are thus thought of as master regulators of gene expression. Because they are single molecular entities that dictate the expression of fundamental regulatory pathways, microRNAs represent potential drug targets for controlling many biological and disease processes.

The LNA Drug Platform overcomes the limitations of earlier antisense and siRNA technologies to deliver potent single-stranded LNA-based drug candidates across a multitude of disease states. The combination of small size and very high affinity allows this class of drugs candidates to potently and specifically inhibit RNA targets in many different tissues without the need for complex delivery vehicles.

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