Ataluren CAS 775304-57-9

Iden­ti­fi­ca­tion Prop­er­ties Safe­ty Data Spec­i­fi­ca­tions and Oth­er Information Links

Iden­ti­fi­ca­tion

CAS Number

775304-57-9

Name

Ataluren

Syn­onyms

3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid [ACD/IUPAC Name] 3-[5-(2-Fluorphenyl)-1,2,4-oxadiazol-3-yl]benzoesäure [Ger­man] [ACD/IUPAC Name] 775304-57-9 [RN] Acide 3-[5-(2-fluorophényl)-1,2,4-oxadiazol-3-yl]benzoïque [French] [ACD/IUPAC Name] Ataluren [INN] [USAN] [Wiki] Ataluren [French] [INN] Atalureno [Span­ish] [INN] atalurenum [Latin] [INN] Ben­zoic acid, 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]- [ACD/​Index Name] [775304-57-9] 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]-benzoic acid 38275-56-8 [RN] Ataluren (PTC124) Ataluren ; PTC-124 ; PTC 124 EC-000.2051 MFCD09864996 [MDL num­ber] NCGC00168759-02 PTC-124 PTC124, Ataluren Translar­na UNII :K16AME9I3V UNII-K16AME9I3V

Mol­e­c­u­lar Structure

SMILES

C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F

Std­InChI

InChI=1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20)

Std­InChIKey

OOUGLTUL­B­SNHNF-UHF­F­­FAOYSA-N

Mol­e­c­u­lar Formula

C15H9FN2O3

Mol­e­c­u­lar Weight

284.24

MDL Number

MFCD09864996

Prop­er­ties

Appear­ance

White Pow­der

Spec­i­fi­ca­tions and Oth­er Infor­ma­tion of Our Ataluren CAS 775304-57-9

Stan­dard

Enter­prise stan­dard

Iden­ti­fi­ca­tion Methods

HNMR/HPLC

Puri­ty

99%min

Pack­age

Accord­ing to cus­tomer require­ments to pack­age.

Stor­age

Under the room tem­per­a­ture and away from light

Appli­ca­tion

PTC124 (Ataluren) selec­tive­ly induces ribo­so­mal read-through of pre­ma­ture but not nor­mal ter­mi­na­tion codons, with EC50 of 0.1 μM, may pro­vide treat­ment for genet­ic dis­or­ders caused by non­sense muta­tions (e.g. CF caused by CFTR non­sense muta­tion). IC50 val­ue : Tar­get : Non­sense muta­tion in vit­ro : Com­pared with Gen­tam­icin which is only active at much high­er con­cen­tra­tions, PTC124 is a more potent non­sense-sup­­press­ing agent and exhibits 4- to 15-fold stim­u­la­tion of read-through rel­a­tive to con­trols. PTC124 (0.01-3 μM) pro­motes dose-depen­­dent read-through of all three non­sense codons in HEK293 cells har­bor­ing LUC-190 non­sense alle­les with the high­est read-through at UGA, fol­lowed by UAG and then UAA, but it does not sup­press mul­ti­ple prox­i­mal non­sense codons. Like Gen­tam­icin, PTC124 is most active when a pyrim­i­dine (in par­tic­u­lar cyto­sine, C) fol­lows the non­sense codon. Con­sis­tent with the sta­ble cell line reporter assay, PTC124 (17 μM) pro­motes sig­nif­i­cant pro­duc­tion of dys­trophin in pri­ma­ry mus­cle cells from Duchenne mus­cu­lar dys­tro­phy (DMD) patients or MDXMDX mice express­ing dys­trophin non­sense alle­les. PTC124 selec­tive­ly pro­motes ribo­so­mal read-through of pre­ma­ture ter­mi­na­tion but not nor­mal ter­mi­na­tion codons, even at con­cen­tra­tions sub­stan­tial­ly greater than the val­ues achiev­ing max­i­mal activ­i­ty. in vivo : Due to func­tion­al recov­ery of dys­trophin pro­duc­tion, oral, intraperi­toneal or com­bined dos­ing of PTC124 for 2-8 weeks par­tial­ly res­cues func­tion­al strength deficit in dys­troph­ic mus­cles of MDX mice, and results in par­tial pro­tec­tion against con­­trac­­tion-induced injury in the exten­sor dig­i­to­rum longus (EDL) mus­cles, as well as sig­nif­i­cant reduc­tions in serum cre­a­tine kinase val­ues. In Cftr-/- mice express­ing a human CFTR-G542X trans­gene, sub­cu­ta­neous or oral admin­is­tra­tion of PTC124 (~60 mg/​kg) sup­press­es the G542X non­sense muta­tion in a dose-depen­­dent man­ner, lead­ing to a sig­nif­i­cant restora­tion of human (h)CFTR pro­tein expres­sion and func­tion with­out any effect on non­sense-medi­at­ed mRNA decay (NMD) or oth­er aspects of mRNA sta­bil­i­ty. PTC124 treat­ment (60 mg/​kg) restores 29% of the nor­mal intesti­nal transep­ithe­lial cAMP-stim­u­lat­ed short­cir­cuit cur­rents observed in Cftr+/+ mice, dis­play­ing a sig­nif­i­cant advan­tage com­pared with Gen­tam­icin

Gen­er­al View of Documents

Links

This prod­uct is devel­oped by our RandD com­pa­ny Cam­ing Phar­ma­ceu­ti­cal Ltd(http://​www​.cam​ing​.com/), and here is the cor­re­spond­ing linkhttp://​www​.cam​ing​.com/​a​t​a​l​u​r​e​n​-​c​a​s​-​7​7​5​3​0​4​-​5​7​-9/

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