Viltolarsen
Based on 1 Customer Validation
Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research.
For research use only. We do not sell to patients.
- Purity: 95.25%
- CAS No.: 2055732-84-6
- Formula: C244H381N113O88P20
- Molecular Weight:6924.82
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Storage:
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
To place orders, for customer services and technical support, please contact: MedChemExpress USA
Tel: 609-228-6898 E-mail: [email protected] [email protected]
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Biological Activity
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Chemical Information
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Solvent & Solubility
- Purity & Documentation
- References
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Help & FAQs
Biological Activity
Viltolarsen (NS-065/NCNP-01; 2 days) induces exon 53 skipping in cells from a DMD model with a deletion of exons 45-52 with an EC50 value of 0.63 μM and in cells with a deletion of exons 48-52 with an EC50 value of 2.3 μM[2].
Viltolarsen (10 μM; 3 days) causes the dystrophin protein expression in cells from a DMD model[2].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
| NCT Number | Sponsor | Condition | Start Date |
Phase
|
|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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CAS No. 2055732-84-6
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Appearance Solid
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Molecular Weight 6924.82
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Formula C244H381N113O88P20
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Color White to off-white
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SMILES
OC[C@H]1O[C@@H](n(ccc(N)n2)c2=O)CN(P(N(C)C)(OC[C@H]3O[C@@H](n(ccc(N)n4)c4=O)CN(P(N(C)C)(OC[C@H]5O[C@@H](n(cc(C)c(=O)[nH]6)c6=O)CN(P(N(C)C)(OC[C@H]7O[C@@H](n(ccc(N)n8)c8=O)CN(P(N(C)C)(OC[C@H]9O[C@@H](n(ccc(N)n%10)c%10=O)CN(P(N(C)C)(OC[C@H]%11O[C@@H](n(cnc%12%13)c%12nc(N)[nH]c%13=O)CN(P(N(C)C)(OC[C@H]%14O[C@@H](n(cnc%15%16)c%15nc(N)[nH]c%16=O)CN(P(N(C)C)(OC[C@H]%17O[C@@H](n(cc(C)c(=O)[nH]%18)c%18=O)CN(P(N(C)C)(OC[C@H]%19O[C@@H](n(cc(C)c(=O)[nH]%20)c%20=O)CN(P(N(C)C)(OC[C@@H]%21CN(P(N(C)C)(OC[C@H]%22O[C@@H](n(cc(C)c(=O)[nH]%23)c%23=O)CN(P(N(C)C)(OC[C@H]%24O[C@@H](n(cnc%25%26)c%25nc(N)[nH]c%26=O)CN(P(N(C)C)(OC[C@H]%27O[C@@H](n%28c%29ncnc(N)c%29nc%28)CN(P(N(C)C)(OC[C@H]%30O[C@@H](n%31c%32ncnc(N)c%32nc%31)CN(P(N(C)C)(OC[C@H]%33O[C@@H](n(cnc%34%35)c%34nc(N)[nH]c%35=O)CN(P(N(C)C)(OC[C@H]%36O[C@@H](n(cnc%37%38)c%37nc(N)[nH]c%38=O)CN(P(N(C)C)(OC[C@H]%39O[C@@H](n(cc(C)c(=O)[nH]%40)c%40=O)CN(P(N(C)C)(OC[C@H]%41O[C@@H](n(cnc%42%43)c%42nc(N)[nH]c%43=O)CN(P(N(C)C)(OC[C@H]%44O[C@@H](n(cc(C)c(=O)[nH]%45)c%45=O)CN(P(N(C)C)(OC[C@H]%46O[C@@H](n(cc(C)c(=O)[nH]%47)c%47=O)CN(P(N(C)C)(OC[C@@H]%48CNC[C@H](n(ccc(N)n%49)c%49=O)O%48)=O)C%46)=O)C%44)=O)C%41)=O)C%39)=O)C%36)=O)C%33)=O)C%30)=O)C%27)=O)C%24)=O)C%22)=O)C[C@H](n(ccc(N)n%50)c%50=O)O%21)=O)C%19)=O)C%17)=O)C%14)=O)C%11)=O)C9)=O)C7)=O)C5)=O)C3)=O)C1
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Synonyms
NS-065/NCNP-01
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Sequence
RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-Gm5U-m5U-C)
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
Solvent & Solubility
H2O : 100 mg/mL (14.44 mM; Need ultrasonic)
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
For the following dissolution methods, please prepare the working solution directly:
It is recommended to prepare fresh solutions and use them promptly within a short period of time.
The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.
Add each solvent one by one: PBS
Solubility: 50 mg/mL (7.22 mM); Clear solution; Need ultrasonic
Purity & Documentation
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Data Sheet (272 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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Handling Instructions (2242 KB)
References
[1]. Sohita Dhillon. Viltolarsen: First Approval. Drugs. 2020 Jul;80(10):1027-1031. [Content Brief]
[2]. Naoki Watanabe, et al. NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2018 Dec 7:13:442-449. [Content Brief]
Complete Stock Solution Preparation Table
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
| Optional Solvent | Concentration Solvent Mass | 1 mg | 5 mg | 10 mg | 25 mg |
|---|---|---|---|---|---|
| H2O | 1 mM | 0.1444 mL | 0.7220 mL | 1.4441 mL | 3.6102 mL |
| 5 mM | 0.0289 mL | 0.1444 mL | 0.2888 mL | 0.7220 mL | |
| 10 mM | 0.0144 mL | 0.0722 mL | 0.1444 mL | 0.3610 mL |
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Viltolarsen Related Classifications
HY-132586 Related Classifications
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