Ataluren (PTC124) - Moligand™, 10mM in DMSO , 80S Ribosome modulator, CAS No.775304-57-9, 80S Ribosome modulator

CAS: 775304-57-9 Cat. No.: A408140 Molecular Weight: 284.24 EC Number: 695-053-5

GRADE & PURITY Moligand™, 10mM in DMSO

Synonyms

3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid

Storage

Store at -80°C

Shipped In

Dry ice packs + Cold packs

Overview

Information

Ataluren (PTC124) Ataluren (PTC124) selectively induces ribosomal read-through of premature but not normal termination codons, with EC50 of 0.1 μM in HEK293 cells, may provide treatment for genetic disorders caused by nonsense mutations (e.g. CF caused
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.
Cell Data

cell lines：

Concentrations：

Incubation Time：

Powder Purity:≥99%

Specifications

Synonyms

3-(5-(2-fluorophenyl)-1, 2, 4-oxadiazol-3-yl)benzoic acid

Specifications & Purity

Moligand™, 10mM in DMSO

Biochemical and Physiological Mechanisms

Ataluren (PTC124) selectively induces ribosomal read-through of premature but not normal termination codons, with EC50 of 0.1 μM in HEK293 cells, may provide treatment for genetic disorders caused by nonsense mutations (e.g. CF caused by CFTR nonsense mut

Storage

Store at -80°C

Shipped In

Dry ice packs + Cold packs

This product requires cold chain shipping. Ground and other economy services are not available.

Grade

Moligand™

Action Type

MODULATOR

Mechanism of action

80S Ribosome modulator

Product Properties

• ALogP: 3.1

Names and Identifiers

• Isomeric SMILES: C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F
• Molecular Weight: 284.24
• Reaxy-Rn: 11000909
• Reaxys-RN_link_address: https://www.reaxys.com/reaxys/secured/hopinto.do?context=S&query=IDE.XRN=11000909&ln=

Chemical and Physical Properties

• Melt Point(°C): 242°C

References

1. Ran Duan, Hanlin Guo, Zhengxiong Peng, Xinyu Yu, Juping Yu, Hong Tian, Wei Liu.  (2025)  Unraveling the interaction mechanism between the genetic disease drug Ataluren and eukaryotic codon recognition factor 1/ human serum albumin through spectroscopic and nuclear magnetic resonance approaches.  SPECTROCHIMICA ACTA PART A-MOLECULAR AND BIOMOLECULAR SPECTROSCOPY,      [PMID:41308367] [10.1016/j.saa.2025.127240]