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 blood diseases, such as polycythemia vera (PV).

About Polycythemia Vera (PV)

PV is a rare, clonal and potentially fatal disease characterized 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 and commonly have symptoms of fatigue.

Safety and efficacy have not been evaluated by any regulatory authorities for the indication described.

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