Fulcrum’s two lead programs in clinical development are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and other hemoglobinopathies, including beta-thalassemia.

The company’s proprietary product engine, FulcrumSeek, identifies drug targets that can modulate gene expression to treat the known root cause of gene mis-expression.