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RG7800 hydrochloride
本产品不向个人销售,仅用作科学研究,不用于任何人体实验及非科研性质的动物实验。
RG7800 hydrochloride图片
包装与价格:
包装价格(元)
5mg电议
10mg电议
50mg电议
100mg电议
200mg电议
500mg电议

产品名称
RO6885247 hydrochloride
产品介绍
RG7800 hydrochloride 是一种可口服的SMN2剪接调节剂,对SMN2剪接及SMN蛋白的EC1.5x值分别为 23 nM 和 87 nM。
生物活性

RG7800 hydrochloride is an orally activeSMN2splicing modulator, withEC1.5xs of 23 nM and 87 nM forSMN2splicing andSMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy.

IC50& Target

EC1.5x: 23 nM (SMN2splicing), 87 nM (SMN protein)[1]

体外研究
(In Vitro)

RG7800 hydrochloride is an orally activeSMN2splicing modulator, with EC1.5xs of 23 nM and 87 nM forSMN2splicing and SMN protein; and is the first small moleculeSMN2splicing modifier to enter human clinical trials[1].

体内研究
(In Vivo)

RG7800 is a favorable agent metabolism and pharmacokinetic (DMPK) profile in the rat and in cynomolgus monkey with good oral bioavailability. RG7800 (1, 3, 10 mg/kg, p.o.) dose-dependently elevates the SMN protein level in the brain and in peripheral tissue of Δ7 mice[1].

Clinical Trial
性状

Solid

Formula

C24H28N6O.xHCl

运输条件

Room temperature in continental US; may vary elsewhere.

储存方式

4°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

溶解性数据
In Vitro: 

H2O : ≥ 113.5 mg/mL

*"≥" means soluble, but saturation unknown.