pociredir

Pociredir (FTX-6058) is an investigational oral small-molecule inhibitor of Embryonic Ectoderm Development (EED), a component of the polycomb repressive complex 2 (PRC2). By inhibiting EED, pociredir downregulates key repressors of fetal globin genes such as BCL11A, leading to increased expression of fetal hemoglobin (HbF). This mechanism is intended to ameliorate symptoms in patients with sickle cell disease by increasing HbF levels and reducing red blood cell destruction and anemia. Pociredir was discovered and developed by Fulcrum Therapeutics using proprietary gene modulation technology. It has received Orphan Drug Designation and Fast Track status from the FDA for sickle cell disease. Clinical trials have shown dose-dependent increases in HbF with a favorable safety profile over short-term exposure[1][2][3][5][6].

Other names

pociredir