fetal hemoglobin (HbF) increase

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Fulcrum Therapeutics to Present Results from the 12 mg Dose Cohort of the Phase 1b PIONEER Trial of Pociredir in Sickle Cell Disease
Globenewswireยท 2025-07-28 20:01
Company Overview - Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases, particularly in areas with high unmet medical need [3] - The company's lead clinical program is pociredir, aimed at increasing fetal hemoglobin (HbF) levels for the treatment of sickle cell disease (SCD) [3][4] Upcoming Event - Fulcrum will host a conference call and webcast on July 29, 2025, at 8:00 a.m. ET to present topline results from the 12 mg dose cohort of the Phase 1b PIONEER trial of pociredir in SCD [1] Pociredir Details - Pociredir is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (EED), discovered using Fulcrum's proprietary technology [4] - The drug works by downregulating fetal globin repressors, leading to increased HbF levels, which may benefit patients with SCD [4] - Pociredir has received FDA Fast Track designation and Orphan Drug Designation for SCD treatment [4] Sickle Cell Disease (SCD) Background - SCD is a genetic disorder caused by a mutation in the HBB gene, leading to inefficient oxygen transport and sickle-shaped red blood cells [5] - Patients with SCD face serious health issues, including anemia, pain, infections, and reduced life expectancy [5]