CU professor co-founds new company to develop genetic heart disease treatment

September 21, 2012

A new biomedical company involving CU-Boulder, Stanford University and the Harvard Medical School has been launched with $38 million in financing from Third Rock Ventures LLC headquartered in Boston and San Francisco to develop therapeutic treatments for genetic heart diseases.

The company, MyoKardia Inc., is developing small molecule therapeutics that address key clinical needs for patients with genetic heart disease, said CU-Boulder Professor Leslie Leinwand, one of four company founders who are all world leaders in the fields of muscle biology and cardiovascular genetics. Based in San Francisco, MyoKardia Inc. initially will target two genetically driven types of a heart muscle disease known as cardiomyopathy, both of which weaken and enlarge heart muscles and which can lead to heart failure.  

The two genetic heart diseases first being targeted by MyoKardia -- hypertrophic cardiomyopathy and dilated cardiomyopathy -- afflict roughly 1 million people in the United States. No new therapeutics to treat the diseases have been brought to market in more than a decade. MyoKardia is developing a pipeline of novel, small molecule therapeutics to target the mutated proteins that cause genetic cardiomyopathies.

Hypertrophic cardiomyopathy produces thickening of the heart walls and is best known as a leading cause of sudden cardiac death in young athletes. Dilated cardiomyopathy produces weakening of the heart walls and enlargement of the heart chambers. Cardiomyopathy can occur at any age, and more than 30,000 children, from newborns to 18-year-olds, suffer from some form of cardiomyopathy in the United States -- a patient population comparable to the number of people living with cystic fibrosis.

MyoKardia’s proprietary drug discovery platform brings together advances from the fields of cardiovascular genomics and heart muscle biology to enable its scientists to target certain heart diseases at the genetic level. This genetically targeted approach has the potential to revolutionize the treatment of cardiomyopathies, and ultimately a broader spectrum of cardiovascular disease, including heart failure.

“This is personalized medicine at its best,” said Leinwand, a professor in CU-Boulder’s Department of Molecular, Cellular and Developmental Biology and Chief Scientific Officer of CU-Boulder’s BioFrontiers Institute. “These therapies will address the actual causes of the disease instead of its symptoms.”

For more information about MyoKardia Inc. visit http://www.myokardia.com/. For more information about Third Rock Ventures visit http://www.thirdrockventures.com. For more information about CU’s BioFrontiers Institute visit http://biofrontiers.colorado.edu/.

Give FeedbackSee More Photos View Photo