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.
Para uso exclusivo en investigación. No vendemos a pacientes.
- Pureza: 98.43%
- No. CAS: 1639264-46-2
- Fòrmula: C542H711F17N169O330P45S6
- Peso molecular:16782.53
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Almacenamiento:
4°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
To place orders, for customer services and technical support, please contact: MedChemExpress USA
Tel: 609-228-6898 E-mail: [email protected] [email protected]
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Actividad biológica
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Chemical Information
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Solvente y solubilidad
- Pureza y Documentación
- Referencias
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Help & FAQs
Actividad biológica
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).
Chemical Information
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No. CAS 1639264-46-2
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Appearance Solid
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Peso molecular 16782.53
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Fòrmula C542H711F17N169O330P45S6
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Color White to off-white
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SMILES
[Cemdisiran]
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Synonyms
ALN-CC5
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Sequence
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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Envío
Room temperature in continental US; may vary elsewhere.
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Almacenamiento
4°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Solvente y solubilidad
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.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Pureza y Documentación
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Ficha de datos (278 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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Instrucciones de manejo (2242 KB)
Referencias
[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.
Cemdisiran Related Classifications
HY-145720 Related Classifications
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