2. Epigenetics
    Neuronal Signaling
  3. Small Interfering RNA (siRNA)
    Transthyretin (TTR)
  4. Vutrisiran

Vutrisiran  (Synonyms: ALN-TTRsc02)

Cat. No.: HY-132589 Purity: 96.37%
COA Handling Instructions

Vutrisiran (ALN-TTRsc02) is a liver-directed, investigational, small interfering ribonucleic acid (siRNA) agent. Vutrisiran can be used for transthyretin (TTR)-mediated amyloidosis research.

For research use only. We do not sell to patients.

RNA, (Um-sp-(2'-deoxy-2'-fluoro)C-sp-Um-Um-Gm-(2'-deoxy-2'-fluoro)G-UmUm-(2'-deoxy-2'-fluoro)A-Cm-Am-Um-Gm-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy2'-fluoro)A-Um-Cm-Cm-Cm-Am-sp-Um-sp-Cm), complex with RNA (Um-sp-Gm-sp-Gm-Gm-Am-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'-fluoro)U-Gm-Um-Am-Am-Cm-Cm-Am-Am-GmAm) 3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]-1,12,19,25-tetraoxo-16-oxa-13,20,24-triazanonacos-1-yl]-4-hydroxy-2-pyrrolidinyl]methyl hydrogen phosphate] (1:1)

Vutrisiran Chemical Structure

CAS No. : 1867157-35-4

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Based on 1 publication(s) in Google Scholar

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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].

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].

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].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.
Clinical Trial
Molecular Weight

16345 (AS: 7556.3; SS: 8788.7 )

Appearance

Solid

CAS No.
Sequence

RNA, (Um-sp-(2'-deoxy-2'-fluoro)C-sp-Um-Um-Gm-(2'-deoxy-2'-fluoro)G-UmUm-(2'-deoxy-2'-fluoro)A-Cm-Am-Um-Gm-(2'-deoxy-2'-fluoro)A-Am-(2'-deoxy2'-fluoro)A-Um-Cm-Cm-Cm-Am-sp-Um-sp-Cm), complex with RNA (Um-sp-Gm-sp-Gm-Gm-Am-Um-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)C-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'-fluoro)U-Gm-Um-Am-Am-Cm-Cm-Am-Am-GmAm) 3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]-1,12,19,25-tetraoxo-16-oxa-13,20,24-triazanonacos-1-yl]-4-hydroxy-2-pyrrolidinyl]methyl hydrogen phosphate] (1:1)
SMILES

[Vutrisiran]
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
In Vitro: 

H2O : ≥ 20 mg/mL

*"≥" means soluble, but saturation unknown.
Purity & Documentation

Purity: 96.37%

COA (250 KB) RP-HPLC (250 KB)

Handling Instructions (2659 KB)
References
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Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

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Keywords:

Vutrisiran1867157-35-4ALN-TTRsc02Small Interfering RNA (siRNA)Transthyretin (TTR)thyroxine-binding prealbuminsiRNAtransthyretinamyloidosissubcutaneousInhibitorinhibitorinhibit

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