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首页-小分子抑制剂&激动剂-Others-SMN2 Modulator-RG7800
RG7800

Chemical Structure : RG7800

CAS No.: 1449598-06-4

RG7800 (RG-7800, RO6885247)

货号: 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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纯度 & COA & 质检文件 纯度: >98% (HPLC) Select Batch:

生物&药学活性

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.

物理化学性质&存储条件

分子量 416.51872
分子式 C24H28N6O
外观性状 Solid
CAS No.
储存条件
固体粉末
-20 °C 12 个月; 4°C 6 个月
配置液
-80 °C 6 个月; -20°C 6 个月
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)-

参考文献

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

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