| Identification | Back Directory | [Name]
RO6885247) | [CAS]
1449598-06-4 | [Synonyms]
RG7800
CS-2490
RO6885247)
RG-7800; RG 7800
RG7800(RO6885247)
RG7800; RG-7800; RG 7800; RO6885247; RO-6885247; RO 6885247.
2-(4-Ethyl-6-methylpyrazolo[1,5-a]pyrazin-2-yl)-9-methyl-7-(1-methylpiperidin-4-yl)-4H-pyrido[1,2-a]pyrimidin-4-one
4H-Pyrido[1,2-a]pyrimidin-4-one, 2-(4-ethyl-6-methylpyrazolo[1,5-a]pyrazin-2-yl)-9-methyl-7-(1-methyl-4-piperidinyl)- | [Molecular Formula]
C24H28N6O | [MDL Number]
MFCD31382130 | [MOL File]
1449598-06-4.mol | [Molecular Weight]
416.52 |
| Chemical Properties | Back Directory | [density ]
1.31±0.1 g/cm3(Predicted) | [storage temp. ]
Store at -20°C | [solubility ]
DMSO:1.4(Max Conc. mg/mL);3.4(Max Conc. mM)
Ethanol:2.5(Max Conc. mg/mL);6.0(Max Conc. mM) | [form ]
Solid | [pka]
9.13±0.10(Predicted) | [color ]
Off-white to yellow |
| Hazard Information | Back Directory | [Uses]
RG7800 is a SMN2 splicing modifier. RG7800 has the potential for spinal muscular atrophy treatment. | [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]. | [storage]
Store at -20°C | [References]
[1] Ratni H, et al. Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy. J Med Chem. 2016 Jul 14;59(13):6086-100. DOI:10.1021/acs.jmedchem.6b00459 |
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