Preferred Label : Sapablursen;
NCIt synonyms : TMPRSS6-ASO IONIS-TMPRSS6-LRx; AS ODN IONIS-TMPRSS6-LRx; TMPRSS6-targeting Antisense Oligonucleotide IONIS-TMPRSS6-LRx;
NCIt definition : An antisense oligonucleotide (ASO) targeting the transmembrane protease serine 6 (TMPRSS6)
mRNA and conjugated to the liver-targeted ligand N-acetylgalactosamine (GalNAc), that
can potentially be used to regulate TMPRSS6, hepcidin and iron levels and treat iron
overload and anemia associated with certain disorders such as beta thalassemia and
myelodysplastic syndromes. Upon subcutaneous administration of sapablursen, the GalNAc
moiety targets and binds with high affinity to asialoglycoprotein receptors (ASGPRs)
expressed on hepatocytes. Once inside the cell, the ASO blocks the translation of
the TMPRSS6 protein. Reduction of the TMPRSS6 level prevents TMPRSS6-mediated suppression
of the bone morphogenetic protein (BMP)-SMAD signaling pathway and leads to an increase
in hepcidin levels. Hepcidin decreases the uptake of dietary iron and the release
of iron from cellular storage, which reduces serum iron, normalizes iron levels and
stimulates erythropoiesis. Hepcidin plays a key role in the homeostasis of systemic
iron; low levels of hepcidin are associated with iron overload in certain diseases,
such as beta-thalassemia and polycythemia vera. TMPRSS6 negatively regulates hepcidin
expression and plays a key role in iron homeostasis.;
UNII : DXS4HJC32N;
CAS number : 2273007-95-5;
Molecule name : IONIS-TMPRSS6-LRx; ISIS-702843; IONIS TMPRSS6-LRx; IONIS-TMPRSS6 LRx; ISIS 702843;
NCI Metathesaurus CUI : CL1648393;
Origin ID : C179118;
UMLS CUI : C5555482;
Semantic type(s)
concept_is_in_subset