Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on developing small-molecule therapies for genetically defined rare diseases with high unmet medical need. The company's lead candidate, pociredir, is an oral small molecule designed to increase fetal hemoglobin levels for treating sickle cell disease, and has demonstrated clinical efficacy in early-stage trials with potential registration-enabling studies planned. Beyond its lead program, Fulcrum operates a discovery platform to identify and validate cellular drug targets that modulate gene expression to address the root causes of rare genetic diseases, with a pipeline including preclinical candidates in oncology. The company plans to commercialize approved products through a targeted commercial infrastructure for rare diseases while retaining full rights to pociredir and exploring partnerships for select markets and candidates.