规格 | 价格 | 库存 | 数量 |
---|---|---|---|
1mg |
|
||
2mg |
|
||
5mg |
|
||
10mg |
|
||
25mg |
|
||
50mg |
|
||
100mg |
|
||
Other Sizes |
|
靶点 |
SMN2
|
---|---|
体外研究 (In Vitro) |
Risdiplam 提高 SMN 蛋白的量并控制 SMN2 前 mRNA 的剪接以生成全长 SMN2 mRNA。 Risdiplam 是 SMN2 的剪接调节剂,可以增加全长 SMN2 蛋白的量,从而增强 SMN 蛋白的功能。导致婴儿死亡的最常见遗传病仍然是SMA(SMA)。由于东南运动神经元 1 (SMN1) 基因中的双联体,早期运动神经元蛋白 (SMN) 水平低是导致这种常染色体隐性神经病的原因,其特征是进行性运动和呼吸肌减弱。残留失活和基因丢失的其他来源[1]。
|
参考文献 |
[1]. Poirier A, et al. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018 Nov 29;6(6):e00447.
|
分子式 |
C22H23N7O
|
---|---|
分子量 |
401.464323282242
|
精确质量 |
401.2
|
元素分析 |
C, 65.82; H, 5.77; N, 24.42; O, 3.99
|
CAS号 |
1825352-65-5
|
相关CAS号 |
Risdiplam-d4
|
外观&性状 |
Solid powder
|
SMILES |
CC1=CC(=NN2C1=NC(=C2)C)C3=CC(=O)N4C=C(C=CC4=N3)N5CCNC6(C5)CC6
|
InChi Key |
ASKZRYGFUPSJPN-UHFFFAOYSA-N
|
InChi Code |
InChI=1S/C22H23N7O/c1-14-9-18(26-29-11-15(2)24-21(14)29)17-10-20(30)28-12-16(3-4-19(28)25-17)27-8-7-23-22(13-27)5-6-22/h3-4,9-12,23H,5-8,13H2,1-2H3
|
化学名 |
7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one
|
别名 |
RG7916; RO703406; RG-7916; RO-7034067;RG 7916; RO 7034067; Evrysdi
|
HS Tariff Code |
2934.99.9001
|
存储方式 |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month |
运输条件 |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
|
溶解度 (体外) |
DMSO: ~1.7 mg/mL (~4.2 mM)
Ethanol: < 1 mg/mL H2O: < 0.1 mg/mL |
---|---|
溶解度 (体内) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80: Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 :Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400 请根据您的实验动物和给药方式选择适当的溶解配方/方案: 1、请先配制澄清的储备液(如:用DMSO配置50 或 100 mg/mL母液(储备液)); 2、取适量母液,按从左到右的顺序依次添加助溶剂,澄清后再加入下一助溶剂。以 下列配方为例说明 (注意此配方只用于说明,并不一定代表此产品 的实际溶解配方): 10% DMSO → 40% PEG300 → 5% Tween-80 → 45% ddH2O (或 saline); 假设最终工作液的体积为 1 mL, 浓度为5 mg/mL: 取 100 μL 50 mg/mL 的澄清 DMSO 储备液加到 400 μL PEG300 中,混合均匀/澄清;向上述体系中加入50 μL Tween-80,混合均匀/澄清;然后继续加入450 μL ddH2O (或 saline)定容至 1 mL; 3、溶剂前显示的百分比是指该溶剂在最终溶液/工作液中的体积所占比例; 4、 如产品在配制过程中出现沉淀/析出,可通过加热(≤50℃)或超声的方式助溶; 5、为保证最佳实验结果,工作液请现配现用! 6、如不确定怎么将母液配置成体内动物实验的工作液,请查看说明书或联系我们; 7、 以上所有助溶剂都可在 Invivochem.cn网站购买。 |
制备储备液 | 1 mg | 5 mg | 10 mg | |
1 mM | 2.4909 mL | 12.4545 mL | 24.9091 mL | |
5 mM | 0.4982 mL | 2.4909 mL | 4.9818 mL | |
10 mM | 0.2491 mL | 1.2455 mL | 2.4909 mL |
1、根据实验需要选择合适的溶剂配制储备液 (母液):对于大多数产品,InvivoChem推荐用DMSO配置母液 (比如:5、10、20mM或者10、20、50 mg/mL浓度),个别水溶性高的产品可直接溶于水。产品在DMSO 、水或其他溶剂中的具体溶解度详见上”溶解度 (体外)”部分;
2、如果您找不到您想要的溶解度信息,或者很难将产品溶解在溶液中,请联系我们;
3、建议使用下列计算器进行相关计算(摩尔浓度计算器、稀释计算器、分子量计算器、重组计算器等);
4、母液配好之后,将其分装到常规用量,并储存在-20°C或-80°C,尽量减少反复冻融循环。
计算结果:
工作液浓度: mg/mL;
DMSO母液配制方法: mg 药物溶于 μL DMSO溶液(母液浓度 mg/mL)。如该浓度超过该批次药物DMSO溶解度,请首先与我们联系。
体内配方配制方法:取 μL DMSO母液,加入 μL PEG300,混匀澄清后加入μL Tween 80,混匀澄清后加入 μL ddH2O,混匀澄清。
(1) 请确保溶液澄清之后,再加入下一种溶剂 (助溶剂) 。可利用涡旋、超声或水浴加热等方法助溶;
(2) 一定要按顺序加入溶剂 (助溶剂) 。
NCT Number | Recruitment | interventions | Conditions | Sponsor/Collaborators | Start Date | Phases |
NCT04888702 | Active Recruiting |
Device: Actimyo | Spinal Muscular Atrophy | Dr. Stéphanie Delstanche | August 8, 2018 | Not Applicable |
NCT05156320 | Active Recruiting |
Drug: Apitegromab Drug: Placebo |
SMA Atrophy |
Scholar Rock, Inc. | February 24, 2022 | Phase 3 |
NCT03779334 | Active Recruiting |
Drug: Risdiplam | Muscular Atrophy, Spinal | Hoffmann-La Roche | August 7, 2019 | Phase 2 |
NCT03032172 | Active Recruiting |
Drug: Risdiplam | Spinal Muscular Atrophy | Hoffmann-La Roche | March 3, 2017 | Phase 2 |
NCT02913482 | Active Recruiting |
Drug: Risdiplam | Muscular Atrophy, Spinal | Hoffmann-La Roche | December 23, 2016 | Phase 2 |
Risdiplam tissues concentration vs risdiplam plasma concentration: A, brain (n = 189) (Studies 1‐4, 6‐10). Pharmacol Res Perspect . 2018 Nov 29;6(6):e00447. td> |
Risdiplam tissue distribution in cynomolgus monkeys. Pharmacol Res Perspect . 2018 Nov 29;6(6):e00447. td> |
Time course of plasma and tissue risdiplam concentrations in mice or rats following PO or IP, single or repeat administration of risdiplam. Pharmacol Res Perspect . 2018 Nov 29;6(6):e00447. td> |
SMN protein increase in muscle vs brain in SMA mice following administration of risdiplam. Pharmacol Res Perspect . 2018 Nov 29;6(6):e00447. td> |