2. Immunology/Inflammation Epigenetics
  3. Complement System Small Interfering RNA (siRNA)
  4. Cemdisiran

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.

For research use only. We do not sell to patients.

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)

Cemdisiran Chemical Structure

CAS No. : 1639264-46-2

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Based on 1 publication(s) in Google Scholar

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Description

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[1][2].

In Vivo

Cemdisiran (subcutaneous injection; single dose, 5-25 mg/kg) exhibits a dose-dependent inhibitory effect on total C5 concentrations in male cynomolgus monkeys, with the 25 mg/kg dose achieving a maximum inhibition rate of >90%[1].
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.

Animal Model: Male cynomolgus monkeys (aged 2-4 years, weighing 2-4.9 kg)[1]
Dosage: 5 mg/kg; 25 mg/kg
Administration: s.c.; single dose
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).
Molecular Weight

16782.53

Formula

C542H711F17N169O330P45S6
CAS No.
Appearance

Solid

Color

White to off-white

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)
SMILES

[Cemdisiran]
Shipping

Room temperature in continental US; may vary elsewhere.
Storage

4°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

Solvent & Solubility
In Vitro: 

H2O : ≥ 20 mg/mL (1.19 mM)

*"≥" means soluble, but saturation unknown.

Preparing
Stock Solutions
Concentration Solvent Mass 1 mg 5 mg 10 mg
1 mM 0.0596 mL 0.2979 mL 0.5959 mL
5 mM --- --- ---
View the 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.

* 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.

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Working solution concentration: mg/mL
This product has good water solubility, please refer to the measured solubility data in water/PBS/Saline for details.
The concentration of the stock solution you require exceeds the measured solubility. The following solution is for reference only.If necessary, please contact MedChemExpress (MCE).
Purity & Documentation

Purity: 98.43%

SDS (252 KB)

COA (251 KB) LCMS (380 KB)

Handling Instructions (2242 KB)
References

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.

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Cemdisiran Related Classifications

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  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

Cemdisiran1639264-46-2ALN-CC5Complement SystemSmall Interfering RNA (siRNA)RNA interference pathwaycomplement component C5pozelimabcomplement-mediated diseasesparoxysmal nocturnal hemoglobinuriaC5 proteincynomolgus monkeysclassical pathway hemolytic activityC5 mRNAInhibitorinhibitorinhibit

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