Votoplam | HTT splicing modulator | Probechem Biochemicals

Chemical Structure : Votoplam

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Votoplam (PTC-518, PTC518)

货号: PC-23055Not For Human Use, Lab Use Only.

Votoplam (PTC518) is a small molecule HTT gene modulator and splicing modifier, selectively lowers huntingtin mRNA and protein.

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生物&药学活性

Votoplam (PTC518) is a small molecule HTT gene modulator and splicing modifier, selectively lowers huntingtin mRNA and protein.
Votoplam (PTC518) promotes the inclusion of a novel pseudoexon containing a premature termination codon, thus triggering HTT mRNA degradation and subsequent reduction in HTT protein levels.
Votoplam (PTC518) demonstrated a dose-dependent lowering of HTT mRNA and protein to the targeted 30-50% reduction in a Phase 1 healthy volunteer study of PTC518 for Huntington's disease.

物理化学性质&存储条件

分子量	419.49
分子式	C21H25N9O
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	2-[3-(2,2,6,6-Tetramethyl-4-piperidinyl)-3H-1,2,3-triazolo[4,5-c]pyridazin-6-yl]-5-(2H-1,2,3-triazol-2-yl)phenol

参考文献

1. Patent WO2020005873 A1.

Votoplam (PTC-518, PTC518)

生物&药学活性

物理化学性质&存储条件

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Votoplam (PTC-518, PTC518)

生物&药学活性

物理化学性质&存储条件

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