Bioactivity | GalNAc unconjugated/naked Fitusiran, an small interfering RNA without GalNAc conjugation, specifically targets antithrombin (AT) messenger RNA to lower production of AT in the liver. GalNAc unconjugated/naked Fitusiran increases thrombin generation and has the potential for the research of the hemophilia[1]. |
Name | GalNAc unconjugated/naked Fitusiran |
Molar Mass | 14459.60 |
Appearance | Solid |
Transport | Room temperature in continental US; may vary elsewhere. |
Storage | -20°C, sealed storage, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture) |
Reference | [1]. 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. |