GlpBio

Risdiplam (RG7916)

目录号: GC30845纯度: >99.00%同义词: RG7916; RO7034067

Risdiplam (RG7916) 是一种小分子 SMN2 前体 mRNA 剪接修饰剂,可促进外显子 7 的包含和全长 SMN2 mRNA 的产生,从而补偿 SMN1 的损失 。

Risdiplam (RG7916)
Cas No.: 1825352-65-5
规格价格库存数量操作
1mg¥450.00现货
1
2mg¥660.00现货
1
5mg¥1,100.00现货
1
10mg¥1,780.00现货
1
25mg¥3,340.00现货
1
50mg¥4,800.00现货
1
100mg¥6,770.00现货
1
500mg¥13,320.00现货
1
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文献被引

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    183(7):1867-1883 (2020)
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产品描述 Description

Risdiplam (RG7916) is a small molecule SMN2 pre-mRNA splicing modifier that promotes the inclusion of exon 7 and production of full-length SMN2 mRNA, which can compensate for the loss of SMN1 [1-2].

Risdiplam (RG7916)(0-1μM) was active in vitro in SMA patient-derived fibroblasts and in motor neurons generated from induced pluripotent stem cells (iPSCs) derived from SMA type 1 patient fibroblasts, promoting the inclusion of exon 7, to generate full-length (FL) mRNA[1].

Risdiplam (RG7916) potently increases SMN protein in both brain and muscle tissues of transgenic mouse models of SMA[1]. In infants with type 1 spinal muscular atrophy, treatment with oral risdiplam(0.08-0.2mg/kg/day)led to an increased expression of functional SMN protein in the blood[3].

References:
[1]. Ratni H, Ebeling M,et,al. Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Aug 9;61(15):6501-6517. doi: 10.1021/acs.jmedchem.8b00741. Epub 2018 Jul 25. PMID: 30044619.
[2]. Singh RN, Ottesen EW, et,al. The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy. Neurosci Insights. 2020 Nov 23;15:2633105520973985. doi: 10.1177/2633105520973985. PMID: 33283185; PMCID: PMC7691903.
[3]. Baranello G, Darras BT, et,al. FIREFISH Working Group. Risdiplam in Type 1 Spinal Muscular Atrophy. N Engl J Med. 2021 Mar 11;384(10):915-923. doi: 10.1056/NEJMoa2009965. Epub 2021 Feb 24. PMID: 33626251.

Risdiplam (RG7916) 是一种小分子 SMN2 前体 mRNA 剪接修饰剂,可促进外显子 7 的包含和全长 SMN2 mRNA 的产生,从而补偿 SMN1 的损失 [1-2] 。

Risdiplam (RG7916)(0-1μM) 在 SMA 患者来源的成纤维细胞和源自 SMA 1 型患者成纤维细胞的诱导多能干细胞 (iPSC) 生成的运动神经元中具有体外活性,促进外显子 7 的包含,生成全长 (FL) mRNA[1]。

Risdiplam (RG7916) 有效增加 SMA 转基因小鼠模型大脑和肌肉组织中的 SMN 蛋白[1]。在患有1型脊髓性肌萎缩症的婴儿中,口服risdiplam(0.08-0.2mg/kg/day)治疗导致血液中功能性SMN蛋白的表达增加[3]。

实验参考方法 Experimental Reference Method

Cell experiment [1]:

Cell lines

SMA type I fibroblasts; SMA type I motor neurons

Reaction Conditions

0-1µM

Applications

Risdiplam (RG7916) was active in vitro in SMA patient-derived fibroblasts and in motor neurons generated from induced pluripotent stem cells (iPSCs) derived from SMA type 1 patient fibroblasts, promoting the inclusion of exon 7, to generate full-length (FL) mRNA.
Animal experiment [1]:

Animal models

Adult C/C-allele miceand Neonatal Δ7 Mouse Model

Preparation Method

Adult C/C-allele mice with mild SMA phenotype were treated for 10 days withRisdiplam (RG7916) given once a day orally at three different doses (1, 3, and 10 mg/kg). The level of SMN protein was assessed in brain and in quadriceps muscle. In the Δ7 mouse model of severe SMA, Risdiplam (RG7916) was administered by intraperitoneal (ip) injection once daily starting on postnatal day (PND) 3 and continued through PND9. The level of SMN protein was assessed in brain and in quadriceps muscle as well.

Dosage form

Adult C/C-allele mice：1, 3, and 10 mg/kg;10 days;p.o. Δ7 mouse model：0-3mg/kg;6 days;i.p.

Applications

Risdiplam (RG7916) potently increases SMN protein in both brain and muscle tissues of transgenic mouse models of SMA.

References:

[1].Ratni H, Ebeling M,et,al. Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Aug 9;61(15):6501-6517. doi: 10.1021/acs.jmedchem.8b00741. Epub 2018 Jul 25. PMID: 30044619.

产品文档 Product Documents

Purity:>99.00%

化学性质Chemical Properties

CAS 号
1825352-65-5
同义词
RG7916; RO7034067
SMILES
O=C(C=C(C(C=C1C)=NN2C1=NC(C)=C2)N=C3C=C4)N3C=C4N(CCN5)CC65CC6
分子式
C22H23N7O
分子量
401.46 g/mol
溶解性
Ethanol : < 1 mg/mL (insoluble);DMSO : < 1 mg/mL (insoluble or slightly soluble)
保存条件
Store at -20°C
General tips
请根据产品在不同溶剂中的溶解度选择合适的溶剂配制储备液；一旦配成溶液，请分装保存，避免反复冻融造成的产品失效。
储备液的保存方式和期限：-80°C 储存时，请在 6 个月内使用，-20°C 储存时，请在 1 个月内使用。
为了提高溶解度，请将管子加热至 37°C，然后在超声波浴中震荡一段时间。
Shipping Condition
评估样品解决方案：配备蓝冰进行发货。所有其他可用尺寸：配备 RT，或根据请求配备蓝冰。

计算工具摩尔浓度 / 稀释 / 分子量 / 单位换算 / 体内配方 / 溶解度

g/mol