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Eteplirsen (AVI-4658; Exondys 51) is an antisense oligonucleotide approved in 2016 by FDA to treat Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.
Physicochemical Properties
| Exact Mass | 10300.59 |
| CAS # | 1173755-55-9 |
| Appearance | White to off-white solid powder |
| HS Tariff Code | 2934.99.9001 |
| Storage |
Powder-20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment (e.g. under nitrogen), avoid exposure to moisture. |
| Shipping Condition | Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs) |
Biological Activity
| ln Vitro | By specifically skipping exon 51 in the faulty gene variant, eteplirsen restores the translational reading frame in Duchenne muscular dystrophy (DMD) and subsequently increases the production of dystrophin [1]. |
| References |
[1]. Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug Des Devel Ther. 2017 Feb 28;11:533-545. |
Solubility Data
| Solubility (In Vitro) | DMSO : ~50 mg/mL (~4.85 mM) |
| Solubility (In Vivo) |
Solubility in Formulation 1: 33.33 mg/mL (3.23 mM) in PBS (add these co-solvents sequentially from left to right, and one by one), clear solution; with sonication. (Please use freshly prepared in vivo formulations for optimal results.) |