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Oct 08, 2012
 |  BioCentury  |  Emerging Company Profile

MyoKardia: Genetic heartthrob

MyoKardia targeting myosin mutations to treat genetic cardiomyopathies

MyoKardia Inc. plans to use its scientific founders' insights about the genetic underpinnings of cardiomyopathies to discover and develop small molecules to treat subsets of the heart disease. It hopes to achieve preclinical proof-of-concept within 24-36 months.

The company represents the convergence of work in muscle biology and cardiovascular genetics that recently have emerged from Harvard Medical School, Stanford University and the University of Colorado.

The aggregator of the discoveries was Third Rock Ventures, which launched MyoKardia with $38 million in series A money last month (see BioCentury, Sept. 24).

Third Rock's Charles Homcy, who is interim CEO of MyoKardia, said the drug discovery technology combines innovative approaches to protein expression, assay development and medicinal chemistry.

"There are hundreds of genetic mutations that create the biochemical abnormalities in motor functioning of the heart" and cause cardiomyopathy, he said.

In the past, interrogating the connection between mutation and cardiomyopathy was not possible in...

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