2646704-10-9
Chemical Structure
Manusiran
Synonym(s): Divesiran; SLN124
- CAS No.: 2646704-10-9
- Formula:C434H570F19N145O270P40S10
- Molecular Weight:14058.53
SMILES: [Manusiran]
Biological Activity: Manusiran (Divesiran) is a GalNac-siRNA targeting liver and transmembrane serine protease 6 (Serine protease 6). Manusiran increases hepatic Hepcidin synthesis and plasma levels by silencing TMPRSS6, a negative regulator of hepcidin production, and limits the availability of iron required for erythropoiesis. Combined use of Manusiran with Deferiprone (HY-B0568) reduces ineffective erythropoiesis and hepatic iron overload in a mouse model of β-thalassemia. Manusiran can be used for research on polycythemia vera, type 1 hereditary hemochromatosis, and β-thalassemia[1][2][3].
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Manusiran | Manusiran (Divesiran) is a GalNac-siRNA targeting liver and transmembrane serine protease 6 (Serine protease 6). Manusiran increases hepatic Hepcidin synthesis and plasma levels by silencing TMPRSS6, a negative regulator of hepcidin production, and limits the availability of iron required for erythropoiesis. Combined use of Manusiran with Deferiprone (HY-B0568) reduces ineffective erythropoiesis and hepatic iron overload in a mouse model of β-thalassemia. Manusiran can be used for research on polycythemia vera, type 1 hereditary hemochromatosis, and β-thalassemia. | |||||||||||||||||||||
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References
- [1]. Kremyanskaya M, et al. Initial Results from a Phase 1/2 Study Evaluating Divesiran, a Novel Galnac Conjugated siRNA, in Patients with Polycythemia Vera (SANRECO)[J]. Blood, 2024, 144: 656.
- [2]. Altamura S, et al. SLN124, a GalNAc-siRNA Conjugate Targeting TMPRSS6, Efficiently Prevents Iron Overload in Hereditary Haemochromatosis Type 1. Hemasphere. 2019 Oct 1;3(6):e301. [Content Brief]
- [3]. Vadolas J, et al. SLN124, a GalNac-siRNA targeting transmembrane serine protease 6, in combination with deferiprone therapy reduces ineffective erythropoiesis and hepatic iron-overload in a mouse model of β-thalassaemia. Br J Haematol. 2021;194(1):200-210. [Content Brief]
