Key information

🔎 Fund Overview

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need. The Company's lead clinical program is pociredir, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease (SCD). It uses technology to identify drug targets that can modulate gene expression to treat the known root cause of gene mis-expression. In addition to its product candidates, the Company has developed a discovery approach that it employs to systematically identify and validate cellular drug targets that can potentially modulate gene expression to treat known root causes of genetically defined rare diseases. The Company continues to advance its program for the potential treatment of inherited aplastic anemias, such as Diamond-Blackfan anemia (DBA), Shwachman-Diamond syndrome, and Fanconi anemia.