Biopharmaceutical company focused on developing therapies for rare genetic diseases.
Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical firm based in Cambridge, Massachusetts, is dedicated to advancing treatments for genetically defined diseases with significant unmet medical needs across the United States. The company's primary focus lies in developing innovative therapies aimed at improving the lives of patients afflicted with rare neuromuscular, muscular, central nervous system, hematologic disorders, as well as cardiomyopathies and pulmonary diseases.
At the forefront of Fulcrum Therapeutics' pipeline are promising product candidates such as losmapimod, a small molecule designed to address facioscapulohumeral muscular dystrophy, and FTX-6058, an oral fetal hemoglobin inducer targeting sickle cell disease and other hemoglobinopathies, including beta-thalassemia. The company leverages its expertise in drug discovery to identify and develop effective treatments that modulate specific biological pathways associated with targeted indications.
Fulcrum Therapeutics collaborates closely with industry leaders to bolster its research efforts. Notably, it has established research and discovery partnerships with Acceleron Pharma Inc. to explore biological targets within the pulmonary disease domain. Additionally, a strategic collaboration and license agreement with MyoKardia, Inc. underscores its commitment to advancing novel therapies for genetic cardiomyopathies. Founded in 2015, Fulcrum Therapeutics continues to drive innovation in biopharmaceuticals, aiming to transform the treatment landscape for genetically defined diseases.