Sapablursen, formerly known as IONIS-TMPRSS6-LRx, is an investigational ligand-conjugated antisense (LICA) medicine designed to target the TMPRSS6 gene to modulate the production of hepcidin, which is the key regulator of iron homeostasis. By modulating hepcidin expression, sapablursen has the potential to positively impact diseases characterized by iron deficiency, such as polycythemia vera (PV).
About Polycythemia Vera
Polycythemia Vera (PV) is a rare, non-genetic and potentially fatal disease caused by overproduction of red blood cells. This overproduction leads to a thickening of the blood, which increases patients’ risk of life-threatening blood clots, including in the lungs, heart and brain. Patients with PV also experience severe iron deficiency due to hepcidin overexpression. There are no approved disease-modifying treatments for PV.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.