THPO-ASO

THPO-ASO is an antisense oligonucleotide designed to selectively silence the hepatic expression of the thrombopoietin (THPO) gene. Thrombopoietin is a key regulator of platelet production via stimulation of the myeloproliferative leukemia virus oncogene (cMPL) receptor on megakaryocytes. By inhibiting hepatic THPO mRNA, THPO-ASO reduces circulating thrombopoietin levels and consequently lowers platelet counts in vivo. Preclinical studies in mice and baboons have demonstrated that repeated administration leads to a sustained reduction in plasma thrombopoietin and platelet count without affecting extrahepatic sources of THPO. In disease models such as myeloproliferative neoplasms (MPNs) and metastatic breast cancer, this reduction delays disease progression by limiting pathological processes dependent on elevated platelets[1][3][6]. The drug is being developed primarily for chronic myeloproliferative disorders but may also have applications in other conditions where modulation of platelet count is beneficial.

Other names

thrombopoietin antisense oligonucleotideTPO-ASO