Drisapersen  (Synonyms: Kyndrisa; GSK2402968A; PRO051)

Drisapersen (Kyndrisa) is a 2^'-O-methyl phosphorothioate RNA antisense oligonucleotide that induces exon 51 skipping. Drisapersen induces skipping of exon 51 during Dystrophin pre-mRNA splicing, allowing the synthesis of partially functional Dystrophin. Drisapersen can be used in research related to Duchenne muscular dystrophy^[1][2].

Drisapersen induces exon 51 skipping during Dystrophin pre-mRNA splicing, leading to the synthesis of Dystrophin, and is used for research related to Duchenne muscular dystrophy^[2].

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

6977.62

C₂₁₁H₂₇₅N₇₆O₁₁₉P₁₉S₁₉

1251830-50-8

Solid

White to off-white

RNA, (P-thio)(Um-Cm-Am-Am-Gm-Gm-Am-Am-Gm-Am-Um-Gm-Gm-Cm-Am-Um-Um-Um-Cm-Um)

CO[C@@H]1[C@H](O)[C@@H](COP(S)(O[C@@H]2[C@@H](COP(S)(O[C@@H]3[C@@H](COP(S)(O[C@@H]4[C@@H](COP(S)(O[C@@H]5[C@@H](COP(S)(O[C@@H]6[C@@H](COP(S)(O[C@@H]7[C@@H](COP(S)(O[C@@H]8[C@@H](COP(S)(O[C@@H]9[C@@H](COP(S)(O[C@@H]%10[C@@H](COP(S)(O[C@@H]%11[C@@H](COP(S)(O[C@@H]%12[C@@H](COP(S)(O[C@@H]%13[C@@H](COP(S)(O[C@@H]%14[C@@H](COP(S)(O[C@@H]%15[C@@H](COP(S)(O[C@@H]%16[C@@H](COP(S)(O[C@@H]%17[C@@H](COP(S)(O[C@@H]%18[C@@H](COP(S)(O[C@@H]%19[C@@H](COP(S)(O[C@@H]%20[C@@H](CO)O[C@H]([C@@H]%20OC)N%21C=CC(NC%21=O)=O)=O)O[C@H]([C@@H]%19OC)N%22C=CC(N)=NC%22=O)=O)O[C@H]([C@@H]%18OC)n%23c%24ncnc(N)c%24nc%23)=O)O[C@H]([C@@H]%17OC)n%25c%26ncnc(N)c%26nc%25)=O)O[C@H]([C@@H]%16OC)n%27cnc%28c%27N=C(N)NC%28=O)=O)O[C@H]([C@@H]%15OC)n%29cnc%30c%29N=C(N)NC%30=O)=O)O[C@H]([C@@H]%14OC)n%31c%32ncnc(N)c%32nc%31)=O)O[C@H]([C@@H]%13OC)n%33c%34ncnc(N)c%34nc%33)=O)O[C@H]([C@@H]%12OC)n%35cnc%36c%35N=C(N)NC%36=O)=O)O[C@H]([C@@H]%11OC)n%37c%38ncnc(N)c%38nc%37)=O)O[C@H]([C@@H]%10OC)N%39C=CC(NC%39=O)=O)=O)O[C@H]([C@@H]9OC)n%40cnc%41c%40N=C(N)NC%41=O)=O)O[C@H]([C@@H]8OC)n%42cnc%43c%42N=C(N)NC%43=O)=O)O[C@H]([C@@H]7OC)N%44C=CC(N)=NC%44=O)=O)O[C@H]([C@@H]6OC)n%45c%46ncnc(N)c%46nc%45)=O)O[C@H]([C@@H]5OC)N%47C=CC(NC%47=O)=O)=O)O[C@H]([C@@H]4OC)N%48C=CC(NC%48=O)=O)=O)O[C@H]([C@@H]3OC)N%49C=CC(NC%49=O)=O)=O)O[C@H]([C@@H]2OC)N%50C=CC(N)=NC%50=O)=O)O[C@H]1N%51C=CC(NC%51=O)=O

Room temperature in continental US; may vary elsewhere.

-20°C, sealed storage, away from moisture

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

* 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.

• [1]. Goemans NM, et al. Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study. PLoS One. 2016;11(9):e0161955. Published 2016 Sep 2.

  [2]. Flanigan KM, et al. Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial. Neuromuscul Disord. 2014;24(1):16-24.  [Content Brief]