Cemdisiran
Based on 1 Customer Validation
Cemdisiran (ALN-CC5) is an N-acetylgalactosamine-conjugated RNAi agent and also a complement component C5 inhibitor. Cemdisiran targets C5 mRNA, cleaves C5 mRNA via the endogenous RNA interference pathway, and inhibits the production of C5 protein in the liver. Cemdisiran exerts a dose-dependent inhibitory effect on total C5 concentrations in cynomolgus monkeys. When used in combination with Pozelimab (HY-P99786) in cynomolgus monkeys, Cemdisiran achieves a more sustained and complete inhibitory effect on complement activity. Cemdisiran can be used in the research of paroxysmal nocturnal hemoglobinuria and other complement-mediated diseases.
商品は「研究用試薬」です。人や動物の医療用・臨床診断用・食品用の製品ではありません。
研究用途以外に使用した場合、当社は一切の責任を負いかねます。
- 純度: 98.43%
- CAS 番号: 1639264-46-2
- 分子式: C542H711F17N169O330P45S6
- 分子量:16782.53
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保管条件:
4°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
生物活性
Combination of cemdisiran (5-25 mg/kg; subcutaneous injection; single dose) and pozelimab (5-10 mg/kg; subcutaneous injection; single dose) achieves sustained, complete inhibition of in vitro complement hemolysis in male cynomolgus monkeys[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:Male cynomolgus monkeys (aged 2-4 years, weighing 2-4.9 kg)[1]
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Dosage:5 mg/kg; 25 mg/kg
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Administration:s.c.; single dose
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Result:Increased total mean C5 concentrations 1.6-1.7-fold above baseline at 4-8 hours post-dose, returned to baseline by Day 3, then dropped to <25 μg/mL, and recovered to baseline by approximately Week 10.
Did not achieve >80% suppression of ex vivo classical pathway hemolytic activity (5 mg/kg dose).
Increased total mean C5 concentrations 1.6-1.7-fold above baseline at 4-8 hours post-dose, returned to baseline by Day 3, then dropped to <5 μg/mL, achieving >90% maximum suppression of total C5, recovered to only 30-45% of baseline by Week 16.
Required 2 weeks to achieve >80% suppression of ex vivo classical pathway hemolytic activity, which was maintained for 5 weeks (25 mg/kg dose).
化学情報
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CAS 番号 1639264-46-2
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性状 Solid
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分子量 16782.53
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分子式 C542H711F17N169O330P45S6
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Color White to off-white
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SMILES
[Cemdisiran]
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別名
ALN-CC5
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配列
RNA, (Am-sp-Am-sp-(2′-deoxy-2′-fluoro)G-Cm-(2′-deoxy-2′-fluoro)A-Am-(2′-deoxy-2′-fluoro)G-Am-(2′-deoxy-2′-fluoro)U-(2′-deoxy-2′-fluoro)A-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)U-Um-Um-(2′-deoxy-2′-fluoro)A-Um-(2′-deoxy-2′-fluoro)A-Am-Um-Am), 3′-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]-1,12,19,25-tetraoxo-16-oxa-13,20,24-triazanonacos-1-yl]-4-hydroxy-2-pyrrolidinyl]methyl hydrogen phosphate], complex with RNA (Um-sp-(2′-deoxy-2′-fluoro)A-sp-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)A-Um-Am-(2′-deoxy-2′-fluoro)A-Am-(2′-deoxy-2′-fluoro)A-Am-Um-Am-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)U-Um-(2′-deoxy-2′-fluoro)G-Cm-Um-Um-sp-Um-sp-Um-dT-dT) (1:1)
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輸送条件
Room temperature in continental US; may vary elsewhere.
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保管条件
4°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
溶剤 & 溶解度
H2O : ≥ 20 mg/mL (1.19 mM)
* "≥" means soluble, but saturation unknown.
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
濃度 (開始) × 体積 (開始) = 濃度 (終了) × 体積 (終了)
純度とドキュメンテーション
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データシート (278 KB)
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SDS (252 KB)
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- Portuguese - PT (252 KB)
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取扱説明書 (2242 KB)
参考文献
[1]. Devalaraja-Narashimha K, et al. Pharmacokinetics and pharmacodynamics of pozelimab alone or in combination with cemdisiran in non-human primates. PLoS One. 2022;17(6):e0269749. Published 2022 Jun 16. [Content Brief]
[2]. Badri P, et al. Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria. Clin Pharmacokinet. 2021;60(3):365-378. [Content Brief]
Complete Stock Solution Preparation Table
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
| Optional Solvent | Concentration Solvent Mass | 1 mg | 5 mg | 10 mg | 25 mg |
|---|---|---|---|---|---|
| H2O | 1 mM | 0.0596 mL | 0.2979 mL | 0.5959 mL | 1.4896 mL |
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.