775304-57-9 Ataluren - Watsonnoke Scientific Ltd

Identification
Properties
Safety Data
Specifications & 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

Structure of Ataluren CAS 775304-57-9

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

Ataluren CAS 775304-57-9 HPLC

Ataluren CAS 775304-57-9 HNMR-1

Ataluren CAS 775304-57-9 HNMR-2

Ataluren CAS 775304-57-9