[CAS NO. 1449598-06-4] RG7800
PRODUCTS SPECIFICATIONS [1449598-06-4]
DESCRIPTION [1449598-06-4]
Overview
| MDL | MFCD31382130 |
|---|---|
| Molecular Weight | 416.52 |
| Molecular Formula | C24H28N6O |
| SMILES | O=C1C=C(C2=NN3C(C(CC)=NC(C)=C3)=C2)N=C4N1C=C(C5CCN(C)CC5)C=C4C |
4 Publications Citing Use of MCE
Summary
RG7800 is a SMN2 splicing modifier. RG7800 has the potential for spinal muscular atrophy treatment.
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 [1] .
MCE has not independently confirmed the accuracy of these methods. They are for reference only.
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 [1] .
MCE has not independently confirmed the accuracy of these methods. They are for reference only.
Clinical Trial
| NCT Number | Sponsor | Condition | Start Date | Phase |
|---|---|---|---|---|
| NCT02240355 | Hoffmann-La Roche |
Muscular Atrophy, Spinal
|
November 2014 | Phase 1 |
Appearance
Solid
Shipping
Room temperature in continental US; may vary elsewhere.
Storage
| Powder | -20°C | 3 years |
|---|---|---|
| 4°C | 2 years | |
| In solvent | -80°C | 6 months |
| -20°C | 1 month |
Solvent & Solubility
Ethanol : 2.5 mg/mL ( 6.00 mM ; Need ultrasonic)
DMSO : 1.4 mg/mL ( 3.36 mM ; Need ultrasonic and warming)
H 2 O : 1.25 mg/mL ( 3.00 mM ; ultrasonic and adjust pH to 3 with HCl)
H 2 O : < 0.1 mg/mL (insoluble)
Stock Solutions
| Concentration Solvent Mass | 1 mg | 5 mg | 10 mg |
|---|
| 1 mM | 2.4008 mL | 12.0042 mL | 24.0085 mL |
| 5 mM | 0.4802 mL | 2.4008 mL | 4.8017 mL |
| 10 mM | --- | --- | --- |
