1499251-18-1
1499251-18-1
Chemical Structure
Fitusiran
Synonym(s): ALN-AT3SC; SAR439774
- CAS No.: 1499251-18-1
- Formula:N/A
- Molecular Weight:16248.2 (AS: 7675 + SS: 8573.2)
SMILES: [Fitusiran]
Biological Activity: Fitusiran (ALN-AT3SC) is a siRNA strategy. Fitusiran delivers siRNA targeting the SERPINC1 gene in hepatocytes to inhibit antithrombin synthesis. Fitusiran enhances thrombin activity and increases Thrombin generation, thereby restoring hemostatic function. Fitusiran can be used in hemophilia-related research[1][2][3].
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Fitusiran | 98.70% | Fitusiran (ALN-AT3SC) is a siRNA strategy. Fitusiran delivers siRNA targeting the SERPINC1 gene in hepatocytes to inhibit antithrombin synthesis. Fitusiran enhances thrombin activity and increases Thrombin generation, thereby restoring hemostatic function. Fitusiran can be used in hemophilia-related research. | ||||||||||||||||||||
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References
- [1]. Lu D, et al. Fitusiran: The first approved siRNA therapy for hemophilia via reducing plasma antithrombin levels. Drug Discov Ther. 2025;19(2):131-132. [Content Brief]
- [2]. Srivastava A, et al. Fitusiran, an investigational siRNA therapeutic targeting antithrombin for the treatment of hemophilia: first results from a phase 3 study to evaluate efficacy and safety in people with hemophilia A or B without inhibitors (ATLAS-A/B)[J]. Blood, 2021, 138: LBA-3.
- [3]. Pasi KJ, et al. Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran-Results of the phase 1 inhibitor cohort. J Thromb Haemost. 2021;19(6):1436-1446. [Content Brief]
Keywords
