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Chemical Structure : 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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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).
| M.Wt | 395.44 | |
| Formula | C20H22FN7O | |
| Appearance | Solid | |
| Storage |
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| Solubility |
10 mM in DMSO |
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1. Longbin Liu, et al. J Med Chem. 2023 Sep 15. doi: 10.1021/acs.jmedchem.3c01173.
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