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Votoplam hydrochloride

Chemical Structure : Votoplam hydrochloride

CAS No.: 2407851-28-7

Votoplam hydrochloride (PTC518 hydrochloride)

货号: PC-23056Not For Human Use, Lab Use Only.

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

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纯度 & COA & 质检文件 纯度: >98% (HPLC)

生物&药学活性

Votoplam (PTC518) hydrochloride 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 455.95
分子式 C21H26ClN9O
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

Phenol, 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)-, hydrochloride (1:1)

参考文献

1. Patent WO2020005873 A1.

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