RG7800
This product is for research use only, not for human use. We do not sell to patients.
For small sizes, please check our retail website as below: www.invivochem.com
| Size | Price | Stock |
|---|---|---|
| 250mg | $1250 | Check With Us |
| 500mg | $2100 | Check With Us |
| 1g | $3150 | Check With Us |
: Cat #: V5680 CAS #: 1449598-06-4 Purity ≥ 98%
Description: RG7800 (also known as RG-7800; RO6885247) is a potent and selective survival motor neuron 2 (SMN2) splicing modifier, which is currently being studies in human clinical trials for the treatment of Spinal Muscular Atrophy (SMA).
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| Molecular Weight (MW) | 416.52 |
|---|---|
| Molecular Formula | C24H28N6O |
| CAS No. | 1449598-06-4 |
| Protocol | In Vitro | RG7800 increases the SMN protein level via induction of alternative splicing of the SMN2 mRNA. RG7800 is shown to promote the inclusion of exon 7 in SMN2 mRNA, generating full-length mRNA in vitro using fibroblasts from an SMA type I patient |
|---|---|---|
| In Vivo | RG7800 shows favorable drug metabolism and pharmacokinetic profile in the rat and in cynomolgus monkey with good oral bioavailability. In SMA mouse model, treatment of RG7800 shows a clear dose dependent increase in SMN protein levels. Mice treated with RG7800 demonstrate a dose dependent increase in survival beginning at the low dose (0.3/1 mg/kg). In the middle and high dose groups (1/3 and 3/10 mg/kg, respectively), approximately 80−90% survive beyond PND50/PND60 with profound body weight gain when the study is terminated. RG7800 dose-dependently corrects SMN2 splicing by including exon 7 to create FL mRNA, suggesting that RG7800 corrects alternative splicing of the human SMN2 gene in the brain of transgenic SMA model mice, leading to an increase of the SMN protein in the brain |
These protocols are for reference only. InvivoChem does not
independently validate these methods.
| Solvent volume to be added | Mass (the weight of a compound) | |||
|---|---|---|---|---|
| Mother liquor concentration | 1mg | 5mg | 10mg | 20mg |
| 1mM | 2.4008 mL | 12.0042 mL | 24.0085 mL | 48.0169 mL |
| 5mM | 0.4802 mL | 2.4008 mL | 4.8017 mL | 9.6034 mL |
| 10mM | 0.2401 mL | 1.2004 mL | 2.4008 mL | 4.8017 mL |
| 20mM | 0.1200 mL | 0.6002 mL | 1.2004 mL | 2.4008 mL |
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Step Two: Enter the in vivo formulation
%DMSO
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%ddH2O
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Method for preparing in vivo formulation:
Take
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DMSO master liquid, next add
µL
PEG300, mix and clarify, next add
µL
Tween 80,mix and clarify, next add
µL
ddH2O,mix and clarify.
Note:
- (1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
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