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RG7800

Chemical Structure : RG7800

CAS No.: 1449598-06-4

RG7800 (RG-7800, RO6885247)

Catalog No.: PC-42423Not For Human Use, Lab Use Only.

RG7800 (RG-7800, RO6885247) is a potent, orally available, small molecule SMN2 splicing modifier with EC1.5X of 23 nM and 87 nM for SMN2 splicing and SMN2 protein, respectively.

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Purity & Documentation Purity: >98% (HPLC) Select Batch:

Biological Activity

RG7800 (RG-7800, RO6885247) is a potent, orally available, small molecule SMN2 splicing modifier with EC1.5X of 23 nM and 87 nM for SMN2 splicing and SMN2 protein, respectively.
RG7800 specifically modifies the alternative splicing of SMN2 exon 7 in SMA patient-derived cells and in two SMA mouse models.
RG7800 exhibits excellent pharmacokinetic and in vivo efficacy and has a favorable safety profile.

Physicochemical Properties

M.Wt 416.51872
Formula C24H28N6O
Appearance Solid
CAS No.
Storage
Solide Powder
-20 °C 12 Months; 4°C 6 Months
In Solvent
-80 °C 6 Months; -20°C 6 Months
Shipping
Solubility

10 mM in DMSO

Chemical Name/SMILES

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)-

References

1. Ratni H, et al. J Med Chem. 2016 Jul 14;59(13):6086-100.

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