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Sep 20, 2012

New Firm Targets Genetic Heart Diseases

  • Third Rock Ventures has ploughed $38 million into establishing MyoKardia, a drug discovery and development firm focused on developing small molecule drugs targeting genetic heart diseases, and in particular hypertrophic and dilated cardiomyopathy.

    MyoKardia is harnessing a drug discovery platform that combines expertise in cardiovascular genomics with heart muscle biology and assay and protein expression capabilities to develop drugs that target the causes of heart dysfunction at its source. The firm says abnormalities at the level of the muscle sarcomere have been identified as causal in a range of heart conditions, and in particular, mutations in sarcomere proteins have been identified as responsible for hypertrophic and dilated cardiomyopathy, by rendering the muscle either 'hyper' or 'hypo' contractile.

    MyoKardia's platform allows the design of mutation-specific sarcomeric allosteric modulators that effectively restore balance in heart muscle contractility, to stop and potentially reverse the course of disease. The firm hopes that ultimately its therapeutic approach could be applied to treat a broader spectrum of cardiovascular disorders, including heart failure.

    “The last decade has been challenging for those pursuing novel therapies in the cardiovascular space, in part because most treatments target symptoms far downstream of the root cause,” explains MyoKardia cofounder Leslie Leinwand, Ph.D., professor of molecular, cellular, and developmental biology at the University of Colorado. “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.”

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