
RG7800
CAS No. 1449598-06-4
RG7800 ( RG-7800 | RG 7800 )
产品货号. M11934 CAS No. 1449598-06-4
RG7800 (RG-7800、RG 7800) 是一种有效的口服小分子 SMN2 剪接修饰剂,对于 SMN2 剪接和 SMN2 蛋白的 EC1.5X 分别为 23 nM 和 87 nM。
纯度: >98% (HPLC)






规格 | 价格/人民币 | 库存 | 数量 |
2MG | ¥316 | 有现货 |
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5MG | ¥518 | 有现货 |
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10MG | ¥713 | 有现货 |
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100MG | 获取报价 | 有现货 |
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200MG | 获取报价 | 有现货 |
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500MG | 获取报价 | 有现货 |
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1G | 获取报价 | 有现货 |
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生物学信息
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产品名称RG7800
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注意事项本公司产品仅用于科研实验,不得用于人体或动物的临床与诊断
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产品简述RG7800 (RG-7800、RG 7800) 是一种有效的口服小分子 SMN2 剪接修饰剂,对于 SMN2 剪接和 SMN2 蛋白的 EC1.5X 分别为 23 nM 和 87 nM。
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产品描述RG7800 (RG-7800, RG 7800) is a potent, orally available, small molecule SMN2 splicing modifier with EC1.5X of 23 nM and 87 nM for SMN2 splicing and SMN2 protein, respectively; specifically modifies the alternative splicing of SMN2 exon 7 in SMA patient-derived cells and in two SMA mouse models; exhibits excellent pharmacokinetic and in vivo efficacy and has a favorable safety profile.Spinal Muscular Atrophy (SMA),Phase 2 Clinical.
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体外实验RG7800 increases the SMN protein level via induction of alternative splicing of the SMN2 mRNA. RG7800 is shown to promote the inclusion of exon 7 in SMN2 mRNA, generating full-length mRNA in vitro using fibroblasts from an SMA type I patient.
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体内实验RG7800 shows favorable drug metabolism and pharmacokinetic profile in the rat and in cynomolgus monkey with good oral bioavailability. In SMA mouse model, treatment of RG7800 shows a clear dose dependent increase in SMN protein levels. Mice treated with RG7800 demonstrate a dose dependent increase in survival beginning at the low dose (0.3/1 mg/kg). In the middle and high dose groups (1/3 and 3/10 mg/kg, respectively), approximately 80?90% survive beyond PND50/PND60 with profound body weight gain when the study is terminated. RG7800 dose-dependently corrects SMN2 splicing by including exon 7 to create FL mRNA, suggesting that RG7800 corrects alternative splicing of the human SMN2 gene in the brain of transgenic SMA model mice, leading to an increase of the SMN protein in the brain.
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同义词RG-7800 | RG 7800
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通路Others
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靶点SMN2
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受体SMN2
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研究领域Neurological Disease
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适应症Spinal Muscular Atrophy(SMA)
化学信息
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CAS Number1449598-06-4
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分子量416.51872
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分子式C24H28N6O
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纯度>98% (HPLC)
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溶解度10 mM in DMSO
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SMILESO=C1C=C(C2=NN3C(C(CC)=NC(C)=C3)=C2)N=C4N1C=C(C5CCN(C)CC5)C=C4C
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化学全称4H-Pyrido[1,2-a]pyrimidin-4-one, 2-(4-ethyl-6-methylpyrazolo[1,5-a]pyrazin-2-yl)-9-methyl-7-(1-methyl-4-piperidinyl)-
运输与储存
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储存条件(-20℃)
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运输条件With Ice Pack
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稳定性≥ 2 years
参考文献
1. Ratni H, et al. J Med Chem. 2016 Jul 14;59(13):6086-100.