Scholar Rock Holding Corporation

Scholar Rock Holding Corporation is a late-stage biopharmaceutical company focused on discovering, developing, and delivering innovative medicines for serious diseases where protein growth factor signaling plays a central role. It employs a proprietary platform targeting the transforming growth factor beta (TGFβ) superfamily to create monoclonal antibodies that selectively inhibit precursor activation of these growth factors. Key product candidates include apitegromab, a selective anti-latent myostatin antibody in Phase 3 for spinal muscular atrophy in patients aged two years and older, with additional Phase 2 programs in spinal muscular atrophy for younger patients and facioscapulohumeral muscular dystrophy, plus preclinical efforts in other neuromuscular diseases. The pipeline also features SRK-181 in Phase 1 for immuno-oncology in cancers resistant to checkpoint inhibitors, SRK-373 in preclinical for fibrotic diseases, and SRK-256 in preclinical for iron-restricted anemia. Scholar Rock Holding Corporation operates in the biotechnology sector, addressing neuromuscular disorders, obesity, cancer, fibrosis, and hematology through this novel approach. Founded in 2012 and headquartered in Cambridge, Massachusetts, it manages its activities as a single operating segment dedicated to advancing these investigational therapies.