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RG7800 hydrochloride is an orally active modulator of SMN2 splicing.

| Pack Size | Price | USA Warehouse | Global Warehouse | Quantity |
|---|---|---|---|---|
| 5 mg | $108 | Inquiry | Inquiry | |
| 10 mg | $171 | Inquiry | Inquiry | |
| 50 mg | $584 | Inquiry | Inquiry | |
| 100 mg | $873 | Inquiry | Inquiry | |
| 200 mg | Inquiry | Inquiry | Inquiry | |
| 500 mg | Inquiry | Inquiry | Inquiry |
| Description | RG7800 hydrochloride is an orally active modulator of SMN2 splicing. |
| Targets&IC50 | SMN protein:, SMN2 splicing: 87 nM , EC1.5x: 23 nM |
| In vitro | RG7800 hydrochloride is an orally active modulator of SMN2 splicing(EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein).RG7800 hydrochloride is the first small molecule SMN2 splicing modifier to enter human clinical trials. |
| In vivo | RG7800 dose-dependently elevates the SMN protein level in the brain and in peripheral tissue of Δ7 mice. RG7800 is a favorable agent metabolism and pharmacokinetic (DMPK) profile in the rat and in cynomolgus monkey with good oral bioavailability. |
| Synonyms | RO6885247 hydrochloride, RG7800 hydrochloride |
| Formula | C24H28N6O.xHCl |
| Smiles | CC1=CC(C2CCN(C)CC2)=CN3C1=NC(C4=NN(C=C(C)N=C5CC)C5=C4)=CC3=O.[xHCl] |
| Relative Density. | no data available |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature. |
| Solubility Information | H2O: 113.5 mg/mL, Sonication is recommended. |
Dissolve 2 mg of the compound in 100 μL DMSO
to obtain a stock solution at a concentration of 20 mg/mL . If the required concentration exceeds the compound's known solubility, please contact us for technical support before proceeding.
1) Add 100 μL of the DMSO
stock solution to 400 μL PEG300
and mix thoroughly until the solution becomes clear.
2) Add 50 μL Tween 80 and mix well until fully clarified.
3) Add 450 μL Saline,PBS or ddH2O
and mix thoroughly until a homogeneous solution is obtained.
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