[CAS NO. 1867157-35-4] Vutrisiran
PRODUCTS SPECIFICATIONS [1867157-35-4]
DESCRIPTION [1867157-35-4]
Overview
| MDL | - |
|---|---|
| Molecular Weight | 16345 (AS: 7556.3; SS: 8788.7 ) |
| Molecular Formula | - |
| SMILES | [Vutrisiran] |
Summary
Vutrisiran (ALN-TTRsc02) is a liver-directed, investigational, small interfering ribonucleic acid (siRNA) agent. Vutrisiran can be used for transthyretin (TTR)-mediated amyloidosis research [1] .
In Vitro
Vutrisiran (previously ALN-TTRSC02) is a second-generation investigational RNAi therapeutic under development for the study of ATTR amyloidosis. Vutrisiran contains an siRNA that targets a sequence within the TTR mRNA which is conserved across wt and all known TTR variants. However, the Vutrisiran siRNA utilizes enhanced stabilization chemistry (ESC) and is conjugated to a triantennary GalNAc ligand, with the aim of enabling infrequent, subcutaneous (SC) dosing [1] .
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
In Vivo
In non-human primates, single SC doses of Vutrisiran 0.3 and 1 mg/kg achieved mean maximum TTR reductions (nadirs) of 55% and 96%, respectively, with serum TTR reductions persisting beyond 4 months for the 1 mg/kg dose. In the same study, monthly doses of 1 and 3 mg/kg maintained a reduction of TTR levels at 96%, relative to baseline [1] .
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
Clinical Trial
| NCT Number | Sponsor | Condition | Start Date | Phase |
|---|---|---|---|---|
| NCT04153149 | Alnylam Pharmaceuticals |
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
|
November 26, 2019 | Phase 3 |
| NCT03759379 | Alnylam Pharmaceuticals |
Amyloidosis, Hereditary|Transthyretin Amyloidosis
|
February 14, 2019 | Phase 3 |
Appearance
Solid
Shipping
Room temperature in continental US; may vary elsewhere.
Storage
4°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Solvent & Solubility
H 2 O : ≥ 20 mg/mL
* "≥" means soluble, but saturation unknown.