Branaplam hydrochloride(Synonyms: LMI070 hydrochloride; NVS-SM1 hydrochloride)

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上海金畔生物科技有限公司为生命科学和医药研发人员提供生物活性分子抑制剂、激动剂、特异性抑制剂、化合物库、重组蛋白，专注于信号通路和疾病研究领域。

Branaplam hydrochloride (Synonyms: LMI070 hydrochloride; NVS-SM1 hydrochloride)

Branaplam (LMI070; NVS-SM1) hydrochloride 是一种口服有效和选择性的 SMN2 拼接调节剂，对 SMN 的 EC50 为 20 nM。Branaplam hydrochloride 抑制 hERG，IC50 为 6.3 μM。Branaplam hydrochloride 在严重的脊髓性肌萎缩症 (SMA) 小鼠模型中可提高全长 SMN 蛋白并延长其生存期。

Branaplam hydrochloride(Synonyms: LMI070 hydrochloride; NVS-SM1 hydrochloride)

Branaplam hydrochloride Chemical Structure

CAS No. : 1562338-39-9

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Branaplam hydrochloride 的其他形式现货产品:

Branaplam

生物活性

Branaplam (LMI070; NVS-SM1) hydrochloride is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam hydrochloride inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam hydrochloride elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model[1][2].

体外研究
(In Vitro)

Branaplam (LMI070; NVS-SM1) hydrochloride treatment induces changes in the levels of 175 genes in human fibroblasts[1].

Shanghai Jinpan Biotech Co Ltd has not independently confirmed the accuracy of these methods. They are for reference only.
体内研究
(In Vivo)

Branaplam (LMI070; NVS-SM1; 3, 10, 30 mg/kg; oral) hydrochloride produces dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord[1].
Branaplam (1 mg/kg of IV; 3 mg/kg of PO) hydrochloride has a CL of 25 mL/min/kg and an AUC of 3.03 μM•h[2].
A single Branaplam (oral; 30 mg/kg) hydrochloride results in significant and durable SMN protein elevation in brain for up to 160 hours in C/+ mice[1].
Branaplam (oral; 0.03, 0.1, 0.3, 1, 3 mg/kg) hydrochloride improves body weight and extendes lifespan in n SMNΔ7 mice[1].

Shanghai Jinpan Biotech Co Ltd has not independently confirmed the accuracy of these methods. They are for reference only.
分子量

429.94

Formula

C22H28ClN5O2
CAS 号

1562338-39-9
运输条件

Room temperature in continental US; may vary elsewhere.
储存方式

Please store the product under the recommended conditions in the Certificate of Analysis.
溶解性数据
In Vitro: 

H2O : 200 mg/mL (465.18 mM; Need ultrasonic)

配制储备液
浓度 溶剂体积 质量 1 mg 5 mg 10 mg
1 mM 2.3259 mL 11.6295 mL 23.2591 mL
5 mM 0.4652 mL 2.3259 mL 4.6518 mL
10 mM 0.2326 mL 1.1630 mL 2.3259 mL

参考文献

  • [1]. Palacino J, et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat Chem Biol. 2015 Jul;11(7):511-517.

    [2]. Cheung AK, et al. Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Dec 27;61(24):11021-11036.

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