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抑制剂/激活剂

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凋亡与自噬

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Branaplam LMI070; NVS-SM1

Branaplam (LMI070; NVS-SM1) 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 抑制 hERG，IC50 为 6.3 μM。Branaplam 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。

货号：
TK0241
规格：

2mg

5mg
价格：
￥0.00

产品参数

CAS No.	1562338-42-4
生物活性	Branaplam (LMI070; NVS-SM1) is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model.
分子式	C22H27N5O2
分子量	393.48
运输条件	Room temperature in continental US; may vary elsewhere.
储存条件	4°C, sealed storage, away from moisture
溶解性数据	DMSO : 6.12 mg/mL (15.55 mM; Need ultrasonic and warming)
体内研究	Branaplam (LMI070; NVS-SM1; 3, 10, 30 mg/kg; oral) produces dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord. Branaplam (1 mg/kg of IV; 3 mg/kg of PO) has a CL of 25 mL/min/kg and an AUC of 3.03 μM•h. A single Branaplam (oral; 30 mg/kg) results in significant and durable SMN protein elevation in brain for up to 160 hours in C/+ mice. Branaplam (oral; 0.03, 0.1, 0.3, 1, 3 mg/kg) improves body weight and extendes lifespan in n SMNΔ7 mice.
体外研究	Branaplam (LMI070; NVS-SM1) treatment induces changes in the levels of 175 genes in human fibroblasts.
文献	•Nature. 2021 Aug;596(7871):291-295. •Nucleic Acids Res. 2021 Sep 7;49(15):8462-8470. •bioRxiv. 2020 Jun. •bioRxiv. 2020 Feb.
纯度及产品资料	98%

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