Chemical Structure : BIO-6553
CAS No.:
BIO-6553
(BIO6553, BIO 6553)
Catalog No.: PC-26407Not For Human Use, Lab Use Only.
BIO-6553 is a potent, CNS-penetrant, selective, and orally bioavailable Huntington gene (HTT)-splicing modulator with HTT HTRF IC50 of 54 nM.
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Purity & Documentation Purity:
>98% (HPLC)
Biological Activity
BIO-6553 is a potent, CNS-penetrant, selective, and orally bioavailable Huntington gene (HTT)-splicing modulator with HTT HTRF IC50 of 54 nM.
BIO-6553 demonstrates enhanced solubility and HTT potency while maintaining a favorable FOXM1 selectivity margin.
BIO-6553 (30 mg/kg, oral) achieved a 71% peak whole blood HTT mRNA reduction at 2 h with sustained effects at 24 h in both cortex and blood of YAC128 transgenic mouse model of HD in RT-qPCR assays.
BIO-6553 exhibits strong in vivo HTT potency, with a free brain IC50 of 36 nM.
BIO-6553 significantly reduces canonical HTT transcript levels in both the brain and blood of the YAC128 HD mice with sustained effects.
Physicochemical Properties
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M.Wt
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481.55
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Formula
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C23H24FN7O2S
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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)-3-(3-(Azetidin-1-yl)pyrrolidin-1-yl)-N-(8-fluoro-7-methoxy-2-methylimidazo[1,2-a]pyridin-6-yl)thieno[2,3-b]pyrazine-6-carboxamide
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References
1. Xu C, et al. J Med Chem. 2026 Mar 17. doi: 10.1021/acs.jmedchem.6c00224.
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