TPO-ASO

TPO-ASO (specifically referring to the candidate AB022, also known as ISIS 558047) is a liver-targeted antisense oligonucleotide (ASO) designed to reduce platelet counts by inhibiting the production of thrombopoietin (TPO). Developed through a collaboration between Ionis Pharmaceuticals and Aronora, the molecule silences TPO mRNA expression in the liver, leading to a controlled reduction in circulating TPO levels and a subsequent decrease in megakaryocyte maturation and platelet production. Research in animal models, including mice, baboons, and cynomolgus monkeys, has demonstrated that TPO-ASO can achieve a sustained reduction in platelet counts (typically around 50%) without significantly impairing primary hemostasis or causing bleeding complications. It is being investigated for the prevention of arterial and venous thrombosis, as well as for attenuating the hypofibrinolytic phenotype associated with sickle cell disease.

Other names

thrombopoietin antisense oligonucleotideTPO antisense oligonucleotide