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

A new biomedical company involving the �鶹ӰԺ, Stanford University and the Harvard Medical School has been launched with $38 million in financing from headquartered in Boston and San Francisco to develop therapeutic treatments for genetic heart diseases.

The company, , is developing small molecule therapeutics that address key clinical needs for patients with genetic heart disease, said CU-�鶹ӰԺ Professor and BioFrontiers Institute Chief Scientific Officer 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.

In addition to Leinwand, the company’s co-founders include Professor James Spudich of Stanford University’s Department of Biochemistry, Dr. Christine Seidman of Harvard Medical School’s Department of Genetics and director of the Cardiovascular Genetics Center at Brigham and Women’s Hospital in Boston, and Professor Jonathan Seidman of Harvard Medical School’s Department of Genetics.

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-�鶹ӰԺ’s Department of Molecular, Cellular and Developmental Biology and Chief Scientific Officer of CU-�鶹ӰԺ’s BioFrontiers Institute. “These therapies will address the actual causes of the disease instead of its symptoms.”

Leinwand and Spudich have seven decades of collective experience working with the protein in cells responsible for muscle contraction, known as myosin. Jonathan and Christine Seidman are a husband-and-wife team renowned for discovering the genetic basis of hypertrophic cardiomyopathy.

“I think that we have a convergence of the development of approaches to be able to screen for these new drugs with the perfect group of founders with which to do it,” said Leinwand. “Third Rock Ventures is visionary in that their executives could see all of this coming together.”

Third Rock Ventures is a venture capital firm formed in 2007 with the mission of launching transformative life sciences companies. Third Rock Ventures has launched MyoKardia with a $38 million “Series A” round of financing. With more than $800 million and two funds under management, Third Rock Ventures works with entrepreneurs to build companies that will make a difference in the lives of patients.

Abnormalities in the basic units of heart muscle, called sarcomeres, have been identified as the driving cause for a variety of heart diseases and the most common cause of hypertrophic and dilated cardiomyopathy. Mutations in the proteins of the sarcomere cause disease by affecting heart muscle contractions. MyoKardia brings together recent assay and protein expression advances with genetic insights to enable a personalized medicine approach to stop and potentially reverse the course of disease.

MyoKardia’s approach will leverage expertise in sarcomere genetics, in-vivo and in-vitro disease models, next-generation biochemical and biophysical assay development, and medicinal chemistry. Together, these capabilities will provide for the efficient progression of multiple programs in genetic cardiomyopathies and heart disease related to sarcomere dysfunction.

Hypertrophic and dilated cardiomyopathies are the most common forms of heart muscle disease and the most common diagnosis leading to heart transplants. More than 60 million people worldwide have cardiomyopathy or carry a cardiomyopathy-causing gene mutation.

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