RG7800 tetrahydrochloride
Based on 5 publication(s) in Google Scholar
RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy.
For research use only. We do not sell to patients.
- Purity: 99.79%
- Formula: C24H32Cl4N6O
- Molecular Weight:562.36
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Storage:
4°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
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Biological Activity
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Chemical Information
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Solvent & Solubility
- Protocol
- Purity & Documentation
- References
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Help & FAQs
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Cell Cycle/DNA Damage Compound Library
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Publications Citing Use of MedChemExpress (MCE) RG7800 tetrahydrochloride
MoreAll DNA/RNA Synthesis Isoforms
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Biological Activity
EC1.5x: 23 nM (SMN2 splicing), 87 nM (SMN protein)[1]
RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; and is the first small molecule SMN2 splicing modifier to enter human clinical trials[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
Chemical Information
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Appearance Solid
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Molecular Weight 562.36
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Formula C24H32Cl4N6O
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Color Light yellow to yellow
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SMILES
CC1=CC(C2CCN(C)CC2)=CN3C1=NC(C4=NN(C=C(C)N=C5CC)C5=C4)=CC3=O.[H]Cl.[H]Cl.[H]Cl.[H]Cl
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Synonyms
RO6885247 tetrahydrochloride
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
4°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Publications (5)
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Journal Impact Factor
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Most Recent
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Nature
2021 Aug;596(7871):291-295. PMID: 34321659 -
J Mol Med (Berl)
2019 Aug;97(8):1183-1193. PMID: 31201471 -
Life Sci Alliance
Drug screening with human SMN2 reporter identifies SMN protein stabilizers to correct SMA pathology. [Abstract]2019 Mar 25;2(2):e201800268. PMID: 30910806 -
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Solvent & Solubility
H2O : ≥ 113.5 mg/mL (201.83 mM)
* "≥" means soluble, but saturation unknown.
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)
Protocol
Mice[1]
Homozygous Δ7 mice are dosed ip with RG7800 or vehicle (100% DMSO, 2.5 mL/kg) once per day from PND3 through postnatal day 3 (PND23), and the dosing regimen is switched on PND24 to a 3-fold higher oral dose once daily in 0.5% HPMC and 0.1% Tween 80. Litters are randomized across groups. Body weight and survival are assessed daily. Survival analysis is done using GraphPad Prism (log-rank test), and a p <0.05 is considered as significant[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
Purity & Documentation
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Data Sheet (275 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 (2659 KB)
References
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 | 1.7782 mL | 8.8911 mL | 17.7822 mL | 44.4555 mL |
| 5 mM | 0.3556 mL | 1.7782 mL | 3.5564 mL | 8.8911 mL | |
| 10 mM | 0.1778 mL | 0.8891 mL | 1.7782 mL | 4.4456 mL | |
| 15 mM | 0.1185 mL | 0.5927 mL | 1.1855 mL | 2.9637 mL | |
| 20 mM | 0.0889 mL | 0.4446 mL | 0.8891 mL | 2.2228 mL | |
| 25 mM | 0.0711 mL | 0.3556 mL | 0.7113 mL | 1.7782 mL | |
| 30 mM | 0.0593 mL | 0.2964 mL | 0.5927 mL | 1.4819 mL | |
| 40 mM | 0.0445 mL | 0.2223 mL | 0.4446 mL | 1.1114 mL | |
| 50 mM | 0.0356 mL | 0.1778 mL | 0.3556 mL | 0.8891 mL | |
| 60 mM | 0.0296 mL | 0.1482 mL | 0.2964 mL | 0.7409 mL | |
| 80 mM | 0.0222 mL | 0.1111 mL | 0.2223 mL | 0.5557 mL | |
| 100 mM | 0.0178 mL | 0.0889 mL | 0.1778 mL | 0.4446 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.
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