Cemdisiran sodium
Based on 1 Customer Validation
Cemdisiran sodium (ALN-CC5 sodium) is an N-acetylgalactosamine-conjugated RNAi agent and also a complement component C5 inhibitor. Cemdisiran sodium targets C5 mRNA, cleaves C5 mRNA via the endogenous RNA interference pathway, and inhibits the production of C5 protein in the liver. Cemdisiran sodium 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 sodium achieves a more sustained and complete inhibitory effect on complement activity. Cemdisiran sodium can be used in the research of paroxysmal nocturnal hemoglobinuria and other complement-mediated diseases.
For research use only. We do not sell to patients.
- Purity: 96.30%
- Formula: C542H665F17N169Na46O330P45S6
- Molecular Weight:16782.66 (free acid)
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Storage:
4°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Biological Activity
Combination of cemdisiran (5-25 mg/kg; subcutaneous injection; single dose) sodium 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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Appearance Solid
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Molecular Weight 16782.66 (free acid)
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Formula C542H665F17N169Na46O330P45S6
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Color White to off-white
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SMILES
[Cemdisiran (sodium)]
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Synonyms
ALN-CC5 sodium
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
4°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Purity & Documentation
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Data Sheet (269 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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Handling Instructions (2242 KB)
References
[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]
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)