| Bioactivity | SLN124 is a GalNac-siRNA targeting transmembrane serine protease 6 (Tmprss6). SLN124 is composed of a trimeric GalNAc ligand conjugated to TMPRSS6‐siRNA. SLN124 has the potential for an siRNA to restore hepcidin expression and normalise iron homeostasis in β‐thalassaemia[1]. |
| Invitro | SLN124 comprises a double‐stranded 19mer RNA oligonucleotide with 2′‐O‐methyl,2′‐fluoro‐2′‐deoxy modifications and phosphorothioate bonds targeting TMPRSS6, which is linked to a tri‐antennary GalNAc unit at the 5′ end of the sense strand[1]. |
| In Vivo | SLN124 (s.c.; 3 mg/kg; two dose on days 1 and 14), alone or in combination with the oral iron chelator deferiprone (DFP), not only suppresses Tmprss6 expression, but also normalises serum hepcidin levels, significantly improves erythropoiesis and reduces splenomegaly in 6‐month‐old female Hbbth3/+ mice[1]. |
| Name | SLN124 |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | Please store the product under the recommended conditions in the Certificate of Analysis. |
| Reference | [1]. Jim Vadolas, 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 Jul;194(1):200-210. |