Chemical Structure : HTT splicing modulator 27
CAS No.:
2743336-70-9
HTT splicing modulator 27
Catalog No.: 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.
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Purity & Documentation Purity:
>98% (HPLC)
Biological Activity
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).
Physicochemical Properties
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M.Wt
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395.44
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Formula
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C20H22FN7O
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Appearance
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Solid
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CAS No.
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Storage
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- Solide Powder
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-20°C 12 Months; 4°C 6 Months
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- In Solvent
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-80°C 6 Months; -20°C 6 Months
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Shipping
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Solubility
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10 mM in DMSO
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Chemical Name/SMILES
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(R)-5-(3-(Cyclopropylamino)pyrrolidin-1-yl)-N-(8-fluoro-2-methylimidazo[1,2-a]pyridin-6-yl)pyrazine-2-carboxamide
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References
1. Longbin Liu, et al. J Med Chem. 2023 Sep 15. doi: 10.1021/acs.jmedchem.3c01173.
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