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Scholar Rock and Collaborators at Harvard Medical School and Northeastern University Elucidate Molecular Basis of Myostatin Activation - a Key Physiological Process in Muscle HealthScholar Rock, a biotechnology company focused on discovering and developing drugs that selectively target growth factors in the disease microenvironment, today announced the publication of "Tolloid cleavage activates latent GDF8 by priming the pro-complex for dissociation" in The EMBO Journal in collaboration with the laboratories of Prof. Timothy Springer (Dept. of Biological Chemistry and Molecular Pharmacology, Harvard Medical School, and Boston Children's Hospital) and Prof. John R. Engen (Dept. of Chemistry and Chemical Biology, Northeastern University (News - Alert)). Myostatin (also known as GDF8) is a key signaling protein that contributes to the regulation of muscle mass and function. Initially produced by muscle in a latent inactive form, myostatin can be activated under certain conditions by sequential enzymatic steps. For the first time, the new study provides an understanding at the molecular level of the structural changes that take place in the protein during this activation process, and the central role of the tolloid enzyme in generating active myostatin. Insight into the activation mechanism of myostatin and other related proteins is central to the drug discovery platform established at Scholar Rock for the development of novel therapies for the treatment of many severe diseases. "Deploying deep structural understanding of growth factors and their activation is opening a profound new way to intervene in human disease," said Alan J. Buckler, Ph.D., Chief Scientific Officer of Scholar Rock. "SRK-015, our clinical canddate for the treatment of muscle atrophy and wasting disorders, exemplifies the strong potential of targeting specific structural states of myostatin with the objective of providing superior therapeutic outcomes." The proprietary therapeutic antibody, SRK-015, was discovered and designed by Scholar Rock to selectively and locally target the latent form of myostatin with the ability to specifically block its intramuscular activation. In a variety of preclinical models of muscle atrophy, SRK-015 has demonstrated improvement in muscle function. SRK-015 is initially being developed by Scholar Rock for the improvement of muscle strength and function in patients with Spinal Muscular Atrophy (SMA (News - Alert)) with the treatment of additional neuromuscular diseases to follow.
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