BIO-6553 | HTT-splicing modulator | Probechem Biochemicals

Chemical Structure : BIO-6553

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BIO-6553 (BIO6553, BIO 6553)

货号: 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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250 mg		Get quote

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生物&药学活性

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.

物理化学性质&存储条件

分子量	481.55
分子式	C23H24FN7O2S
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)-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

参考文献

1. Xu C, et al. J Med Chem. 2026 Mar 17. doi: 10.1021/acs.jmedchem.6c00224.

BIO-6553 (BIO6553, BIO 6553)

生物&药学活性

物理化学性质&存储条件

参考文献

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BIO-6553 (BIO6553, BIO 6553)

生物&药学活性

物理化学性质&存储条件

参考文献

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