Fulcrum Therapeutics

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing novel therapies for patients with genetically defined diseases that have significant unmet medical needs. The company's lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy, a rare condition characterized by progressive muscle wasting and disability. Another key candidate, FTX-6058, aims to upregulate fetal hemoglobin to treat sickle cell disease and beta-thalassemia. Fulcrum is also actively researching drug targets for other genetic disorders, including Duchenne muscular dystrophy and Friedreich ataxia, as well as neurological and pulmonary diseases. The company has established collaborations with Acceleron Pharma to explore biological targets in pulmonary disease and with MyoKardia to develop therapies for genetic cardiomyopathies. Founded in 2015, Fulcrum Therapeutics continues to focus on improving patient outcomes in rare disease contexts.