Art. -Nr.: HY-108753

Eteplirsen

(Synonyms: AVI 4658)

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Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research.

Nur für Forschungszwecke. Wir verkaufen nicht an Patienten.

Reinheit: 98.50%
CAS. Nr.: 1173755-55-9
Formel: C₃₆₄H₅₆₉N₁₇₇O₁₂₂P₃₀
Molecular Weight:10305.74

Speicherung:

-20°C, stored under nitrogen, away from moisture

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

Eteplirsen Chemical Structure

CAS. Nr.: 1173755-55-9

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Biologische Aktivität
- Beschreibung
- In Vitro
- Clinical Trial
Chemical Information
Lösungsmittel & Löslichkeit
- In Vitro
- In Vivo
Reinheit & Dokumentation
Verweise
Help & FAQs
Classifications
Bioz

Biologische Aktivität

Beschreibung

Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research^[1].

In Vitro

Eteplirsen acts to promote dystrophin production by restoring the translational reading frame of Duchenne muscular dystrophy (DMD) through specific skipping of exon 51 in defective gene variants^[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Clinical Trial

NCT Number	Sponsor	Condition	Start Date	Phase A549
NCT01329991	Plexxikon\|		2011-05	PHASE1

Chemical Information

CAS. Nr. 1173755-55-9
Appearance Solid
Molecular Weight 10305.74
Formel C₃₆₄H₅₆₉N₁₇₇O₁₂₂P₃₀
Color White to off-white
SMILES

O=C1NC(N)=NC2=C1N=CN2[C@H]3CNC[C@H](O3)COP(N4C[C@H](O[C@@H](N5C(N=CN=C6N)=C6N=C5)C4)COP(N7C[C@@H](COP(N8C[C@@H](COP(N9C[C@@H](COP(N%10C[C@@H](COP(N%11C[C@@H](COP(N%12C[C@@H](COP(N%13C[C@@H](COP(N%14C[C@@H](COP(N%15C[C@@H](COP(N%16C[C@@H](COP(N%17C[C@@H](COP(N%18C[C@@H](COP(N%19C[C@@H](COP(N%20C[C@@H](COP(N%21C[C@@H](COP(N%22C[C@@H](COP(N%23C[C@@H](COP(N%24C[C@@H](COP(N%25C[C@@H](COP(N%26C[C@@H](COP(N%27C[C@@H](COP(N%28C[C@@H](COP(N%29C[C@@H](COP(N%30C[C@@H](COP(N%31C[C@@H](COP(N%32C[C@@H](COP(N%33C[C@@H](COP(N%34C[C@@H](COP(N%35CCN(C(OCCOCCOCCO)=O)CC%35)(N(C)C)=O)O[C@@H](N%36C(N=C(N)C=C%36)=O)C%34)(N(C)C)=O)O[C@@H](N%37C(NC(C(C)=C%37)=O)=O)C%33)(N(C)C)=O)O[C@@H](N%38C(N=C(N)C=C%38)=O)C%32)(N(C)C)=O)O[C@@H](N%39C(N=C(N)C=C%39)=O)C%31)(N(C)C)=O)O[C@@H](N%40C(N=CN=C%41N)=C%41N=C%40)C%30)(N(C)C)=O)O[C@@H](N%42C(N=CN=C%43N)=C%43N=C%42)C%29)(N(C)C)=O)O[C@@H](N%44C(N=C(N)C=C%44)=O)C%28)(N(C)C)=O)O[C@@H](N%45C(N=CN=C%46N)=C%46N=C%45)C%27)(N(C)C)=O)O[C@@H](N%47C(NC(C(C)=C%47)=O)=O)C%26)(N(C)C)=O)O[C@@H](N%48C(N=C(N)C=C%48)=O)C%25)(N(C)C)=O)O[C@@H](N%49C(N=CN=C%50N)=C%50N=C%49)C%24)(N(C)C)=O)O[C@@H](N%51C(N=CN=C%52N)=C%52N=C%51)C%23)(N(C)C)=O)O[C@@H](N%53C(N=C(N)NC%54=O)=C%54N=C%53)C%22)(N(C)C)=O)O[C@@H](N%55C(N=C(N)NC%56=O)=C%56N=C%55)C%21)(N(C)C)=O)O[C@@H](N%57C(N=CN=C%58N)=C%58N=C%57)C%20)(N(C)C)=O)O[C@@H](N%59C(N=CN=C%60N)=C%60N=C%59)C%19)(N(C)C)=O)O[C@@H](N%61C(N=C(N)NC%62=O)=C%62N=C%61)C%18)(N(C)C)=O)O[C@@H](N%63C(N=CN=C%64N)=C%64N=C%63)C%17)(N(C)C)=O)O[C@@H](N%65C(NC(C(C)=C%65)=O)=O)C%16)(N(C)C)=O)O[C@@H](N%66C(N=C(N)NC%67=O)=C%67N=C%66)C%15)(N(C)C)=O)O[C@@H](N%68C(N=C(N)NC%69=O)=C%69N=C%68)C%14)(N(C)C)=O)O[C@@H](N%70C(N=C(N)C=C%70)=O)C%13)(N(C)C)=O)O[C@@H](N%71C%72=C(N=C%71)C(N)=NC=N%72)C%12)(N(C)C)=O)O[C@@H](N%73C(NC(C(C)=C%73)=O)=O)C%11)(N(C)C)=O)O[C@@H](N%74C(NC(C(C)=C%74)=O)=O)C%10)(N(C)C)=O)O[C@@H](N%75C(NC(C(C)=C%75)=O)=O)C9)(N(C)C)=O)O[C@@H](N%76C(N=C(N)C=C%76)=O)C8)(N(C)C)=O)O[C@@H](N%77C(NC(C(C)=C%77)=O)=O)C7)(N(C)C)=O)(N(C)C)=O
Synonyms

AVI 4658
Sequence

RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), 5'-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate]
Versand

Room temperature in continental US; may vary elsewhere.
Speicherung

-20°C, stored under nitrogen, away from moisture

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

Lösungsmittel & Löslichkeit

In Vitro:

DMSO : 50 mg/mL (4.85 mM; Need ultrasonic; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO)

View the Complete Stock Solution Preparation Table

Preparing Stock Solutions

View the Complete Stock Solution Preparation Table

Concentration Solvent Mass

1 mg

5 mg

10 mg

Concentration

Solvent

Mass

1 mM

0.0970 mL

0.4852 mL

0.9703 mL

5 mM

---

10 mM

---

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 (stored under nitrogen, 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.

Molaritätsrechner

Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

Mass Mass

Konzentration Konzentration

Volumen Volumen

Molecular Weight Molecular Weight

Verdünnungsrechner

Konzentration _{(Stammlösung)} × Volumen _{(Stammlösung)} = Konzentration _{(Ziellösung)} × Volumen _{(Ziellösung)}

Konzentration _{(Stammlösung)} Konzentration _{(Stammlösung)}

Volumen _{(Stammlösung)} Volumen _{(Stammlösung)}

Konzentration _{(Ziellösung)} Konzentration _{(Ziellösung)}

Volumen _{(Ziellösung)} Volumen _{(Ziellösung)}

In Vivo:

Select the appropriate dissolution method based on your experimental animal and administration route.

Stock Solution Working Solution

For the following dissolution methods, please ensure to first prepare a clear stock solution using an In Vitro approach and then sequentially add co-solvents:
To ensure reliable experimental results, the clarified stock solution can be appropriately stored based on storage conditions. As for the working solution for In Vivo experiments, it is recommended to prepare freshly and use it on the same day.
The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.

Protocol 1

Add each solvent one by one: 10% DMSO 40% PEG300 5% Tween-80 45% Saline

Solubility: ≥ 1.25 mg/mL (0.12 mM); Clear solution

This protocol yields a clear solution of ≥ 1.25 mg/mL (saturation unknown).

Taking 1 mL working solution as an example, add 100 μL DMSO stock solution (12.5 mg/mL) to 400 μL PEG300, and mix evenly; then add 50 μL Tween-80 and mix evenly; then add 450 μL Saline to adjust the volume to 1 mL.

Protocol 2

Add each solvent one by one: 10% DMSO 90% (20% SBE-β-CD in Saline)

Solubility: ≥ 1.25 mg/mL (0.12 mM); Clear solution

This protocol yields a clear solution of ≥ 1.25 mg/mL (saturation unknown).

Taking 1 mL working solution as an example, add 100 μL DMSO stock solution (12.5 mg/mL) to 900 μL 20% SBE-β-CD in Saline, and mix evenly.

Protocol 3

Add each solvent one by one: 10% DMSO 90% Corn Oil

Solubility: ≥ 1.25 mg/mL (0.12 mM); Clear solution

This protocol yields a clear solution of ≥ 1.25 mg/mL (saturation unknown). If the continuous dosing period exceeds half a month, please choose this protocol carefully.

Taking 1 mL working solution as an example, add 100 μL DMSO stock solution (12.5 mg/mL) to 900 μL Corn oil, and mix evenly.

Working Solution

For the following dissolution methods, please prepare the working solution directly:

It is recommended to prepare fresh solutions and use them promptly within a short period of time.

The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.

Protocol 1

Add each solvent one by one: PBS

Solubility: 33.33 mg/mL (3.23 mM); Clear solution; Need ultrasonic

In Vivo Dissolution Calculator

Please enter the basic information of animal experiments:

Dosage Dosage
mg/kg
Animal Weight (Per Animal) Animal Weight (Per Animal)
g
Dosing Volume (Per Animal) Dosing Volume (Per Animal)
μL
Number of Animals Number of Animals

Recommended: Prepare an additional quantity of animals to account for potential losses during experiments.

Recommended: Prepare an additional quantity of animals to account for potential losses during experiments.

Please enter your animal formula composition:

%
DMSO +

Recommended: Keep the proportion of DMSO in working solution below 2% if your animal is weak.
%
+
+
%
Tween-80 + +
%
Saline +

The co-solvents required include: DMSO, . All of co-solvents are available by MedChemExpress (MCE). , Tween 80. All of co-solvents are available by MedChemExpress (MCE).

Working solution concentration: 0.22 mg/mL

Reinheit & Dokumentation

Select Batch:

Reinheit: 99.98%

Assay: 99.98%

ee.: 99.98%

Data Sheet (270 KB)
SDS (254 KB)
- English - EN (254 KB)
- Français - FR (254 KB)
- Deutsch - DE (254 KB)
- Norwegian - NO (254 KB)
- Español - ES (254 KB)
- Swedish - SV (254 KB)
- Italian - IT (254 KB)
- Korean - KR (254 KB)
- Portuguese - PT (254 KB)
COA (2659 KB)
Elemental Analysis Report (2659 KB)
HNMR (2659 KB)
CNMR (2659 KB)
RP-HPLC (2659 KB)
NP-HPLC (2659 KB)
MS (2659 KB)
LCMS (2659 KB)
IR (2659 KB)
GC (2659 KB)
OR (2659 KB)
SDS-PAGE (2659 KB)
SEC-HPLC (2659 KB)
Other Report (2659 KB)
Handling Instructions (2242 KB)

Verweise

[1]. Kenji Rowel Q Lim, et al. Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug Des Devel Ther. 2017 Feb 28;11:533-545. [Content Brief]

Help & FAQs

How should lyophilized recombinant proteins be reconstituted and stored?

1. Before opening the cap, centrifuge the vial at 13000 rpm for 20-30 seconds. This step will ensure that any lyophilized powder that may have adhered to the cap or walls is collected at the bottom of the vial, minimizing the risk of product loss.

Complete Stock Solution Preparation Table

Optional Solvent	Concentration Solvent Mass	1 mg	5 mg	10 mg	25 mg
DMSO	1 mM	0.0970 mL	0.4852 mL	0.9703 mL	2.4258 mL

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Eteplirsen

Biologische Aktivität

Chemical Information

Lösungsmittel & Löslichkeit

Reinheit & Dokumentation

Verweise

Help & FAQs

Complete Stock Solution Preparation Table

Eteplirsen Related Classifications

HY-108753 Related Classifications