Scholar Rock

Scholar Rock Holding Corporation, a biopharmaceutical company, focuses on improving the lives of children and adults with spinal muscular atrophy (SMA) and other rare, severe, and debilitating neuromuscular diseases. The company’s novel understanding of the molecular mechanisms of growth factor activation within the transforming growth factor beta (TGFß) superfamily has enabled the development of a proprietary platform. This platform is used for the development of monoclonal antibodies that locally and selectively target the precursor, or latent, forms of growth factors. The company is developing Apitegromab, an inhibitor of the activation of myostatin, which is in a Phase 2 clinical trial for the treatment of SMA and facioscapulohumeral muscular dystrophy (FSHD).