1867157-35-4
1867157-35-4 structure
- Name: Vutrisiran
- Chemical Name: Vutrisiran
- CAS Number: 1867157-35-4
- Molecular Formula:
- Molecular Weight:
- Catalog: Research Areas Neurological Disease
- Create Date: 2022-01-05 07:52:59
- Modify Date: 2024-01-11 17:44:39
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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].
| Name | Vutrisiran |
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| Description | 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]. |
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| Related Catalog | |
| 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]. |
| 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]. |
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| No Any Chemical & Physical Properties |