Third Rock Ventures, a venture capital firm of Boston, Mass., has formed new company MyoKardia with a $38 million Series A financing. MyoKardia is developing a pipeline of novel small molecule therapeutics that address key clinical needs for patients with genetic heart disease.

MyoKardia’s first programs include hypertrophic and dilated cardiomyopathy, for which no novel therapeutics have been brought to market in over a decade. 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 disease “at its source.” This genetically targeted approach has the potential to revolutionize the treatment of cardiomyopathies, and ultimately a broader spectrum of cardiovascular disease, including heart failure.

The company is founded by experts in the fields of muscle biology and cardiovascular genetics: James Spudich, Ph.D., professor of biochemistry, Stanford University; Leslie Leinwand, Ph.D., professor of molecular, cellular and developmental biology, University of Colorado; Christine Seidman, M.D., professor of medicine and genetics, Harvard Medical School and director of the cardiovascular genetics center at Brigham and Women's Hospital; and Jonathan Seidman, Ph.D., professor of genetics, Harvard Medical School.

"The last decade has been challenging for those pursuing novel therapeutics in the cardiovascular space, in part because most treatments target symptoms far downstream of the root cause," said Leinwand. "MyoKardia's approach addresses this challenge head on by employing genetics to more precisely define the disease and ‘who we want to treat,’ and by employing cutting-edge muscle biochemistry and a novel platform to determine ‘how we want to treat.’ Our initial targets are genetic cardiomyopathies, but this could very well be a novel and tractable therapeutic discovery approach to even larger diseases like heart failure."

MyoKardia's platform brings together recent assay and protein expression advances pioneered by its founders with genetic insights to enable a personalized medicine approach. This allows for the rapid development of mutation-specific sarcomeric allosteric modulators that rebalance contractility, therefore stopping and potentially reversing the course of disease. MyoKardia's approach will leverage resident 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 DCM, HCM and other genetic cardiomyopathies and heart disease related to sarcomere dysfunction.

"With MyoKardia's platform, we have the ability to exquisitely characterize the biochemistry and biophysics of the human mutated sarcomere," said Spudich. "This not only allows us to better understand what drives pathophysiology, it also points us toward potential mutant specific solutions."