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Precursor form of myostatin identified as promising target
March 2018
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CAMBRIDGE, Mass.—New research out of biotechnology company Scholar Rock has shown that blocking the activation of the latent precursor form of myostatin via monoclonal antibodies could represent a more effective approach to this target. The precursor form, the researchers found, is the predominant form of myostatin in muscles, and is localized to the extracellular matrix in that it coats individual muscle fibers. Lead drug candidate SRK-015 was found to bind to the myostatin precursor with high selectivity and affinity, enabling it to block the activation of myostatin and resulting atrophy and encourage muscle mass. SRK-015 is being advanced as a potential therapeutic for improving muscle strength and function in patients with spinal muscular atrophy. The results of this research appeared in Nature in a paper titled “Blocking extracellular activation of myostatin as a strategy for treating muscle wasting.”

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