Drisapersen
Based on 1 Customer Validation
Drisapersen (Kyndrisa) is a 2'-O-methyl phosphorothioate RNA antisense oligonucleotide that induces exon 51 skipping. Drisapersen induces skipping of exon 51 during Dystrophin pre-mRNA splicing, allowing the synthesis of partially functional Dystrophin. Drisapersen can be used in research related to Duchenne muscular dystrophy.
For research use only. We do not sell to patients.
- Purity: 97.92%
- CAS No.: 1251830-50-8
- Formula: C211H275N76O119P19S19
- Molecular Weight:6977.62
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Storage:
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, 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
All DNA/RNA Synthesis Isoforms
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Biological Activity
Drisapersen induces exon 51 skipping during Dystrophin pre-mRNA splicing, leading to the synthesis of Dystrophin, and is used for research related to Duchenne muscular dystrophy[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. 1251830-50-8
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Appearance Solid
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Molecular Weight 6977.62
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Formula C211H275N76O119P19S19
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Color White to off-white
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SMILES
CO[C@@H]1[C@H](O)[C@@H](COP(S)(O[C@@H]2[C@@H](COP(S)(O[C@@H]3[C@@H](COP(S)(O[C@@H]4[C@@H](COP(S)(O[C@@H]5[C@@H](COP(S)(O[C@@H]6[C@@H](COP(S)(O[C@@H]7[C@@H](COP(S)(O[C@@H]8[C@@H](COP(S)(O[C@@H]9[C@@H](COP(S)(O[C@@H]%10[C@@H](COP(S)(O[C@@H]%11[C@@H](COP(S)(O[C@@H]%12[C@@H](COP(S)(O[C@@H]%13[C@@H](COP(S)(O[C@@H]%14[C@@H](COP(S)(O[C@@H]%15[C@@H](COP(S)(O[C@@H]%16[C@@H](COP(S)(O[C@@H]%17[C@@H](COP(S)(O[C@@H]%18[C@@H](COP(S)(O[C@@H]%19[C@@H](COP(S)(O[C@@H]%20[C@@H](CO)O[C@H]([C@@H]%20OC)N%21C=CC(NC%21=O)=O)=O)O[C@H]([C@@H]%19OC)N%22C=CC(N)=NC%22=O)=O)O[C@H]([C@@H]%18OC)n%23c%24ncnc(N)c%24nc%23)=O)O[C@H]([C@@H]%17OC)n%25c%26ncnc(N)c%26nc%25)=O)O[C@H]([C@@H]%16OC)n%27cnc%28c%27N=C(N)NC%28=O)=O)O[C@H]([C@@H]%15OC)n%29cnc%30c%29N=C(N)NC%30=O)=O)O[C@H]([C@@H]%14OC)n%31c%32ncnc(N)c%32nc%31)=O)O[C@H]([C@@H]%13OC)n%33c%34ncnc(N)c%34nc%33)=O)O[C@H]([C@@H]%12OC)n%35cnc%36c%35N=C(N)NC%36=O)=O)O[C@H]([C@@H]%11OC)n%37c%38ncnc(N)c%38nc%37)=O)O[C@H]([C@@H]%10OC)N%39C=CC(NC%39=O)=O)=O)O[C@H]([C@@H]9OC)n%40cnc%41c%40N=C(N)NC%41=O)=O)O[C@H]([C@@H]8OC)n%42cnc%43c%42N=C(N)NC%43=O)=O)O[C@H]([C@@H]7OC)N%44C=CC(N)=NC%44=O)=O)O[C@H]([C@@H]6OC)n%45c%46ncnc(N)c%46nc%45)=O)O[C@H]([C@@H]5OC)N%47C=CC(NC%47=O)=O)=O)O[C@H]([C@@H]4OC)N%48C=CC(NC%48=O)=O)=O)O[C@H]([C@@H]3OC)N%49C=CC(NC%49=O)=O)=O)O[C@H]([C@@H]2OC)N%50C=CC(N)=NC%50=O)=O)O[C@H]1N%51C=CC(NC%51=O)=O
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Synonyms
Kyndrisa; GSK2402968A; PRO051
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Sequence
RNA, (P-thio)(Um-Cm-Am-Am-Gm-Gm-Am-Am-Gm-Am-Um-Gm-Gm-Cm-Am-Um-Um-Um-Cm-Um)
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Solvent & Solubility
H2O : 100 mg/mL (14.33 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 (sealed storage, 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 (sealed storage, 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)
Purity & Documentation
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Data Sheet (271 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]. Goemans NM, et al. Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study. PLoS One. 2016;11(9):e0161955. Published 2016 Sep 2. [Content Brief]
[2]. Flanigan KM, et al. Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial. Neuromuscul Disord. 2014;24(1):16-24. [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 (sealed storage, 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 | 0.1433 mL | 0.7166 mL | 1.4332 mL | 3.5829 mL |
| 5 mM | 0.0287 mL | 0.1433 mL | 0.2866 mL | 0.7166 mL | |
| 10 mM | 0.0143 mL | 0.0717 mL | 0.1433 mL | 0.3583 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.
Drisapersen Related Classifications
HY-145724 Related Classifications
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