HTT splicing modulator 27

Chemical Structure : HTT splicing modulator 27

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货号: PC-21151Not For Human Use, Lab Use Only.

HTT splicing modulator 27 is a potent, orally bioavailable, and brain-penetrant small-molecule HTT pre-mRNA splicing modulator with mHTT protein EC50 of 66 nM.

规格	价格	库存	数量
50 mg		Get quote
100 mg		Get quote
200 mg		Get quote
1 g		Get quote

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生物&药学活性

HTT splicing modulator 27 is a potent, orally bioavailable, and brain-penetrant small-molecule HTT pre-mRNA splicing modulator with mHTT protein EC50 of 66 nM.
HTT splicing modulator 27 (10 mg/kg) significantly reduces HTT e49–50 RNA in mouse brain in an acute model of Huntington's disease (HD).
HTT splicing modulator 27 (10 mg/kg) significantly reduces mHTT in brain in subchronic rodent models of HD.
HTT splicing modulator 27 shows minimal efflux ratios in both the MDR1 and the BCRP transporter assays and demonstrates a higher CNS effect (relative to peripheral tissues) when compared to branaplam (Cat# PC-45659).

物理化学性质&存储条件

分子量	395.44
分子式	C20H22FN7O
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	(R)-5-(3-(Cyclopropylamino)pyrrolidin-1-yl)-N-(8-fluoro-2-methylimidazo[1,2-a]pyridin-6-yl)pyrazine-2-carboxamide

参考文献

1. Longbin Liu, et al. J Med Chem. 2023 Sep 15. doi: 10.1021/acs.jmedchem.3c01173.