News | March 07, 2011

Mass. General Grants License for Drug Platform to Regulate Cholesterol

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.

For more information: www.santaris.com

Related Content

News | Pharmaceuticals

January 5, 2022 — The Feinstein Institutes for Medical Research, the science arm of Northwell Health, recently received ...

Home January 05, 2022
Home
News | Pharmaceuticals

January 4, 2022 — The U.S. Food and Drug Administration (FDA) has cleared Novartis' inclisiran (Leqvio), the first small ...

Home January 04, 2022
Home
News | Pharmaceuticals

December 16, 2021 – Spanish pharmaceutical company Ferrer and the Spanish National Center for Cardiovascular Research ...

Home December 16, 2021
Home
Videos | Pharmaceuticals

Susan Cheng, M.D., MPH, director of the Institute for Research on Healthy Aging in the Department of Cardiology at ...

Home November 22, 2021
Home
News | Pharmaceuticals

November 19, 2021 — Results from two early clinical studies show the first version of an oral PCSK9-inhibitor ...

Home November 19, 2021
Home
News | Pharmaceuticals

November 15, 2021 — Esperion announced the presentation of three abstracts highlighting Nexletol tablets at the American ...

Home November 15, 2021
Home
News | Pharmaceuticals

November 15, 2021 — Novartis announced results from a pooled post-hoc analysis of Phase III ORION-9, -10 and -11 trials ...

Home November 15, 2021
Home
Feature | Pharmaceuticals | By Dave Fornell, DAIC Editor

There has been debate in recent years over balancing the risks vs. benefits of aspirin for the prevention of heart ...

Home October 13, 2021
Home
News | Pharmaceuticals

October 7, 2021 – The U.S. Food and Drug Administration (FDA) recently granted the breakthrough therapy designation for ...

Home October 07, 2021
Home
News | Pharmaceuticals

May 15, 2021 — A new analysis of the Phase 3 EXPLORER-HCM study evaluating mavacamten, an investigational, first-in ...

Home May 15, 2021
Home
Subscribe Now