| Field | Specification |
|---|---|
| Alternative names | AVI 4658 |
| CAS no. | |
| Applications | |
| Molecular weight | |
| Molecular formula | C364H569N177O122P30 |
| Purity | |
| SMILES | |
| Form | Solid |
| Storage | |
| Shipping | |
| Catalog no. (Mfr.) | |
| Main SKU |
Compound Overview
Eteplirsen, also known as AVI 4658, is a phosphorylated diamine morpholino oligonucleotide that targets exon 51 of the human Duchenne muscular dystrophy (DMD) gene. It induces skipping of exon 51 during splicing, restoring the translation reading frame and yielding a shortened, functional dystrophin, and it can be used in Duchenne muscular dystrophy research[1][2][3][4]. It is supplied as a white to off-white solid (C364H569N177O122P30, MW 10305.74) at 98.50% purity.
Physical & Chemical Properties
| CAS Number | 1173755-55-9 |
|---|---|
| Molecular Formula | C364H569N177O122P30 |
| Molecular Weight | 10305.74 g/mol |
| Purity | 98.50% |
| Appearance | Solid |
| Color | White to off-white |
| 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] |
| 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 |
| Signaling Pathway | Cytoskeleton |
| Solubility | In Vitro: DMSO: 50 mg/mL (4.85 mM; Requires sonication; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO) |
| Storage | -20°C, stored under nitrogen, away from moisture. In solvent: -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture). |
| Shipping | Room temperature in continental US; may vary elsewhere. |
Literature Cited
Sources cited in this description and in the In Vitro & In Vivo Data tab. Peer-reviewed publications that used this product are listed under References.
Safety
For Research Use Only. Not for use in diagnostic or therapeutic procedures, and not for human or veterinary use. Handle in accordance with the Safety Data Sheet and your institution's chemical hygiene plan.
In Vitro
| Solvent | Solubility | Notes |
|---|---|---|
| DMSO | 50 mg/mL (4.85 mM) | requires sonication; use freshly opened DMSO (absorbed moisture lowers solubility) |
Aliquot the stock solution and store it at -80°C (up to 6 months) or -20°C (up to 1 month); stored under nitrogen, away from moisture; avoid repeated freeze-thaw cycles.
In Vivo
Choose the formulation that suits the animal model and route of administration; percentages are volume ratios of the final working solution. Start from a clear DMSO stock (see In Vitro above), add the co-solvents one at a time in the order listed, mixing after each addition, and prepare the working solution fresh on the day of dosing. If precipitation or phase separation occurs, gentle warming or sonication can help.
Protocol 1
| Composition | 10% DMSO + 40% PEG300 + 5% Tween-80 + 45% saline |
|---|---|
| Result | ≥ 1.25 mg/mL (0.12 mM); clear solution |
| How to prepare | Gives a clear solution at ≥ 1.25 mg/mL (saturation not determined). For 1 mL of working solution: add 100 μL DMSO stock (12.5 mg/mL) to 400 μL PEG300; then 50 μL Tween-80; then 450 μL saline to bring the volume to 1 mL. Saline: dissolve 0.9 g sodium chloride in ddH2O and make up to 100 mL. |
Protocol 2
| Composition | 10% DMSO + 90% (20% SBE-β-CD in saline) |
|---|---|
| Result | ≥ 1.25 mg/mL (0.12 mM); clear solution |
| How to prepare | Gives a clear solution at ≥ 1.25 mg/mL (saturation not determined). For 1 mL of working solution: add 100 μL DMSO stock (12.5 mg/mL) to 900 μL 20% SBE-β-CD in saline. 20% SBE-β-CD in saline: dissolve 2 g SBE-β-CD powder in 10 mL saline until clear (4°C, store up to one week). |
Protocol 3
| Composition | 10% DMSO + 90% Corn Oil |
|---|---|
| Result | ≥ 1.25 mg/mL (0.12 mM); clear solution |
| How to prepare | Gives a clear solution at ≥ 1.25 mg/mL (saturation not determined). Use with caution if continuous dosing will exceed two weeks. For 1 mL of working solution: add 100 μL DMSO stock (12.5 mg/mL) to 900 μL corn oil. |
Direct preparation of the working solution
These formulations are prepared directly, without a DMSO stock; use them promptly after preparation.
Protocol 4
| Composition | PBS |
|---|---|
| Result | 33.33 mg/mL (3.23 mM); clear solution; requires sonication |
Data provided by the manufacturer.
In Vitro
In the tri-chromatic reporter cell line (Flp-In CHO cells that harbor the DMD exon 51 minigene), Eteplirsen (AVI 4658) (100 nM; 24 h) induces exon 51 skipping[4]. In differentiated human rhabdomyosarcoma (RD) cells, Eteplirsen (10 μM; 24 h) also induces exon 51 skipping[4]. In the tri-chromatic reporter cell line, Eteplirsen (100 nM; 24 h) promotes exon 51 skipping, with TagRFP-conjugated protein expression serving as a readout of successful splicing modulation[4].
Cell Proliferation Assay[4]
| Cell Line | Flp-In CHO cells harboring DMD exon 51 minigene |
|---|---|
| Concentration | 100 nM |
| Incubation Time | 24 h |
| Result | Induced exon 51 skipping. |
RT-PCR[4]
| Cell Line | Differentiated human rhabdomyosarcoma (RD) cells |
|---|---|
| Concentration | 10 μM |
| Incubation Time | 24 h |
| Result | Induced exon 51 skipping in endogenous DMD transcript. |
In Vivo
Eteplirsen (AVI 4658) (i.v. or s.c.; up to 320 mg/kg; once weekly for 12 weeks) is well tolerated in cynomolgus monkeys, with no drug related adverse impacts observed[2]. In cynomolgus monkeys, Eteplirsen (once weekly for 12 weeks; i.v. or s.c.; up to 320 mg/kg) induces dose-dependent microscopic renal effects, including minimal basophilic granules, minimal to moderate basophilic tubules, and minimal to mild tubular vacuolation; the renal effects induced by Eteplirsen (AVI 4658) sodium are reversible after a 28-day recovery period[2]. At the maximum feasible dose, Eteplirsen (single dose i.v. at up to 320 mg/kg, or s.c. at up to 320 mg/kg once weekly for 4 weeks) shows no test article-related effects on the cardiovascular, respiratory, global neurological, renal, or liver parameters of cynomolgus monkeys[3]. In the mouse bone marrow erythrocyte micronucleus test, Eteplirsen (up to 2000 mg/kg; i.v.; single dose) shows no mutagenic potential[3].
| Animal Model | Repeat-dose toxicity study in cynomolgus monkeys (Chinese origin, 2.7-3 years old)[2] |
|---|---|
| Dosage | 0, 5, 40, 320 mg/kg |
| Administration | i.v. or s.c.; once weekly for 12 weeks |
| Result | Showed no drug-related effects on survival, clinical observations, body weight, food consumption, ophthalmoscopic or electrocardiographic evaluations, hematology, clinical chemistry, urinalysis, organ weights, or macroscopic evaluations. Exhibited dose-dependent microscopic renal effects: basophilic granules (minimal), basophilic tubules (minimal to moderate), tubular vacuolation (minimal to mild); findings were partially reversible after 28-day recovery. |
| Animal Model | Safety pharmacology evaluation in male cynomolgus monkeys[3] |
|---|---|
| Dosage | 0, 40, 160, 320 mg/kg |
| Administration | i.v. or s.c.; single dose |
| Result | Showed no test article-related effects on cardiovascular (arterial blood pressure, heart rate, ECG), respiratory (respiratory rate, inspiratory/expiratory time, tidal volume), global neurological, renal, or liver parameters at doses up to 320 mg/kg. |
Data provided by the manufacturer. Numbered citations refer to the Literature Cited list in the product description.
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