When Boston Celtics star Reggie Lewis dropped dead during a shootaround 20 years ago, little was known about his underlying heart problem. It took an autopsy to confirm that he had a genetic defect known as hypertrophic cardiomyopathy, which leads to thickening of the heart tissue and irregular heartbeats.
Years later, as a slew of genetic technologies have come together, Third Rock Ventures is betting $38 million that a startup called MyoKardia can make drugs that treat that underlying genetic defect, and others like it that affect 1 million people in the U.S.
MyoKardia, a new company setting up shop in San Francisco, is being led by Third Rock venture partner Charles Homcyand a team of top academic scientists who study the genetics of cardiovascular disease and muscle biology. The group of advisors includes James Spudich of Stanford University, Leslie Leinwand of the University of Colorado, and Christine Seidman and Jonathan Seidman, at Harvard Medical School.
The basic concept at MyoKardia is to connect the dots between abnormal genes, proteins, and tissues in certain genetically defined types of heart disease. As those connections become more clear, it will look to develop conventional small-molecule pills that work for certain segments of people with common gene mutations, and which people can take daily to keep the symptoms at bay, Homcy says. The initial plan is to go after hypertrophic cardiomyopathy and dilated cardiomyopathy, with a handful of drugs matched up to specifically treat a handful of different genetic forms of the disease. Longer term, the company hopes that work will be useful for more of the 5 million patients in the U.S. with congestive heart failure, who have a complex disease that may be the result of a complex interplay of genetics and environmental factors.
“This may be the first time that we can really get a sense of the mechanisms that regulate cardiac form and function,” Homcy says.
MyoKardia is getting started in San Francisco, Homcy says, partly to be near Spudich, who recently won the Albert Lasker award for his basic research for his studies of how muscles contract. Homcy also has
