Third Rock bankrolls a new biotech to tackle genetic heart disease

Third Rock Ventures has dipped back into its second fund for a $38 million A round to launch a new drug developer which plans to get into the genetic nitty-gritty of heart disease. 

In many ways the newly created South San Francisco-based MyoKardia is a classic Third Rock creation. Helmed initially by Charles Homcy, a partner in the venture group, it gathered together some marquee scientific talent to go after the root causes of hypertrophic and dilated cardiomyopathy, a condition that afflicts a million people in the U.S. The biotech will target personalized small-molecule treatments that can address protein mutations in the sarcomere--part of the heart muscle--for small "buckets" of patients, Homcy tells FierceBiotech

In many ways the development strategy for MyoKardia will look a lot like Vertex's ($VRTX) approach to Kalydeco, says Homcy, which started out being proven for a small percentage of the cystic fibrosis population and then advanced into new studies for larger groups. Duchenne muscular dystrophy, a rare disease that has been carefully segmented into patient groups often no bigger than a single-digit percentage of the total population, offers another example of this approach. Each of the genetically defined "buckets" in MyoKardia's sights represents a separate program offering opportunities for partnering at a later stage. The big idea here is to start with precisely-defined groups of patients, driving for proof-of-concept data in small human studies.

Christine and Jonathan Seidman at Harvard Medical School, Stanford's James Spudich, and the University of Colorado's Leslie Leinwand proffered the scientific insights that will drive MyoKardia's research and development work. 

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

Third Rock has been wheeling and dealing since it launched its second, $426 million fund two years ago. It's not unusual to see the group launch startups like MyoKardia and Ember Therapeutics--a 2012 Fierce 15 company--where it puts together tranched packages of venture rounds to tackle new drugs spotlighted by breakthrough scientific work. 

- here's the press release

Special Report: Ember Therapeutics – 2012 Fierce 15

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