Fulcrum Therapeutics Inc. is a clinical-stage biopharmaceutical company focused on developing small-molecule therapies to treat genetically defined rare diseases in areas of high unmet medical need. The company employs proprietary technology to identify drug targets that modulate gene expression, addressing the root causes of gene mis-expression. Its lead product candidate, losmapimod, targets facioscapulohumeral muscular dystrophy (FSHD), a rare and debilitating muscle disorder. Additional programs include FTX-6058 and pociredir, small molecules designed to increase fetal hemoglobin expression for sickle cell disease, beta-thalassemia, and other hemoglobinopathies, as well as efforts in diamond-blackfan anemia, bone marrow failure syndromes, and fibrotic disorders. Fulcrum Therapeutics Inc. emphasizes a patient-centered approach, aiming to transform the lives of patients and caregivers through innovative gene control mechanisms. Founded in 2015 and headquartered in Cambridge, Massachusetts, the company plays a key role in advancing treatments for underserved rare disease populations.