Company Overview - Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases with high unmet medical needs [2][3] - The company's lead clinical program is pociredir, designed to increase fetal hemoglobin expression for treating sickle cell disease (SCD) [2][3] Upcoming Presentations - Fulcrum Therapeutics will present at the 5th Global Congress on Sickle Cell Disease in Abuja, Nigeria, from June 3-6, 2025, and at the 2025 European Hematology Association Congress in Milan, Italy, from June 12-15, 2025 [2] - Specific presentations include preclinical and early clinical results of pociredir, a Phase 1b study evaluating safety and pharmacokinetics in SCD patients, and pharmacokinetics and safety in healthy adults [5] Pociredir Details - Pociredir is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (EED), leading to downregulation of fetal globin repressors and increased fetal hemoglobin levels [3] - Initial data showed proof-of-concept for pociredir in SCD, with well-tolerated results in clinical trials prior to the FDA lifting the clinical hold in August 2023 [3] Sickle Cell Disease Overview - Sickle cell disease is a genetic disorder caused by a mutation in the HBB gene, leading to inefficient oxygen transport and sickle-shaped red blood cells [4] - Patients with SCD face serious health issues, including anemia, pain, infections, and reduced life expectancy [4]