Ataluren (PTC124)

For research use only.

Catalog No.S6003

30 publications

Ataluren (PTC124) Chemical Structure

Molecular Weight(MW): 284.24

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 mutation). Phase 3.

Size Price Stock Quantity  
10mM (1mL in DMSO) USD 97 In stock
USD 75 In stock
USD 170 In stock
USD 270 In stock
USD 370 In stock
Bulk Discount

Free Overnight Delivery on orders over $ 500
Next day delivery by 10:00 a.m. Order now.

Selleck's Ataluren (PTC124) has been cited by 30 publications

Purity & Quality Control

Choose Selective CFTR Inhibitors

Biological Activity

Description 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 mutation). Phase 3.
Features Demonstrates oral bioavailability, and an appropriate safety toxicology profile.
Targets
CFTR (nonsense mutant) [1]
(HEK293 cells)
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. [1]

Cell Data
Cell Lines Assay Type Concentration Incubation Time Formulation Activity Description PMID
BAC_MMA∗EGFP MWHGeY5kfGmxbjDBd5NigQ>? NIDNSWozOMLizszN NGrPXmY4OsLiaNMg Mlz0SG1UVw>? NYT5bZNTemW|dXz0d{BqdiCjIIPp[45q\mmlYX70JIlv[3KnYYPlJIlvKG2nZHnhckBx\WGtIH\seY9z\XOlZX7j[S=> MYiyN|A1OTF6OR?=
Mut−/−MUTStop+/− MXnGeY5kfGmxbjDBd5NigQ>? NX3BTYVYOjEEoN88US=> NEnvXnM4OsLiaNMg NXPkZ5Z4TE2VTx?= NUWwZ4RHcW6lcnXhd4V{KHSqZTDhcY92dnRib3dCpG1WXMLibWLORS=> MoT5NlMxPDFzOEm=
HEK293T NFniSGJHfW6ldHnvckBCe3OjeR?= NVj1TlMyOTBiwsXnM41t MnrmSG1UVw>? NXjWZY1nemW|dH;y[ZMh\nWubD3s[Y5ofGhiaHHycY9vcW5iYUGgLQKJxDhyIHvEZUkhcW5icD7SN|FZNXS{YX7z[oVkfGWmIHPlcIx{ NGfPU|IzOzB{N{[0NC=>
ML1 MoLGSpVv[3Srb36gRZN{[Xl? M3OxTVMvOy9zMNMg{txO M3jVd|Q5KGh? MULEUXNQ NIrDcIFqdmO{ZXHz[ZMhSVKVQjDhZ5Rqfmm2eR?= NXKwfJlLOjJ7N{G5OVk>
ML2 NX7rRY1GTnWwY4Tpc44hSXO|YYm= NIT4NGY{NjNxMUFCpO69VQ>? MWG0PEBp MlPPSG1UVw>? Mn2ybY5kemWjc3XzJGFTW0JiYXP0bZZqfHl? NHLQdHIzOjl5MUm1PS=>

... Click to View More Cell Line Experimental Data

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. [1] 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. [2]

Protocol

Animal Research:[2]
- Collapse
  • Animal Models: Cftr-/- hCFTR-G542X transgenic mice
  • Dosages: ~60 mg/kg/day
  • Administration: Subcutaneous injection or oral administration
    (Only for Reference)

Solubility (25°C)

In vitro DMSO 57 mg/mL (200.53 mM)
Water Insoluble
Ethanol Insoluble
In vivo Add solvents to the product individually and in order(Data is from Selleck tests instead of citations):
1% DMSO+30% polyethylene glycol+1% Tween 80
For best results, use promptly after mixing.
30 mg/mL

* Please note that Selleck tests the solubility of all compounds in-house, and the actual solubility may differ slightly from published values. This is normal and is due to slight batch-to-batch variations.

Chemical Information

Molecular Weight 284.24
Formula

C15H9FN2O3

CAS No. 775304-57-9
Storage powder
in solvent
Synonyms N/A
Smiles OC(=O)C1=CC=CC(=C1)C2=NOC(=N2)C3=CC=CC=C3F

In vivo Formulation Calculator (Clear solution)

Step 1: Enter information below (Recommended: An additional animal making an allowance for loss during the experiment)
Dosage mg/kg Average weight of animals g Dosing volume per animal ul Number of animals
Step 2: Enter the in vivo formulation (Different batches have different solubility ratios, please contact Selleck to provide you with the correct ratio)
% DMSO % % Tween 80 % ddH2O
CalculateReset

Bio Calculators

Molarity Calculator

Molarity Calculator

Calculate the mass, volume or concentration required for a solution. The Selleck molarity calculator is based on the following equation:

Mass (mg) = Concentration (mM) × Volume (mL) × Molecular Weight (g/mol)

  • Mass
    Concentration
    Volume
    Molecular Weight

*When preparing stock solutions, please always use the batch-specific molecular weight of the product found on the via label and MSDS / COA (available on product pages).

Dilution Calculator

Dilution Calculator

Calculate the dilution required to prepare a stock solution. The Selleck dilution calculator is based on the following equation:

Concentration (start) x Volume (start) = Concentration (final) x Volume (final)

This equation is commonly abbreviated as: C1V1 = C2V2 ( Input Output )

  • C1
    V1
    C2
    V2

* When preparing stock solutions always use the batch-specific molecular weight of the product found on the vial label and MSDS / COA (available online).

The Serial Dilution Calculator Equation

  • Serial Dilutions

  • Computed Result

  • C1=C0/X C1: LOG(C1):
    C2=C1/X C2: LOG(C2):
    C3=C2/X C3: LOG(C3):
    C4=C3/X C4: LOG(C4):
    C5=C4/X C5: LOG(C5):
    C6=C5/X C6: LOG(C6):
    C7=C6/X C7: LOG(C7):
    C8=C7/X C8: LOG(C8):
Molecular Weight Calculator

Molecular Weight Calculator

Enter the chemical formula of a compound to calculate its molar mass and elemental composition:

Total Molecular Weight: g/mol

Tip: Chemical formula is case sensitive. C10H16N2O2 c10h16n2o2

Instructions to calculate molar mass (molecular weight) of a chemical compound:

To calculate molar mass of a chemical compound, please enter its chemical formula and click 'Calculate'.

Definitions of molecular mass, molecular weight, molar mass and molar weight:

Molecular mass (molecular weight) is the mass of one molecule of a substance and is expressed in the unified atomic mass units (u). (1 u is equal to 1/12 the mass of one atom of carbon-12)
Molar mass (molar weight) is the mass of one mole of a substance and is expressed in g/mol.

Molarity Calculator

Mass Concentration Volume Molecular Weight

Clinical Trial Information

NCT Number Recruitment interventions Conditions Sponsor/Collaborators Start Date Phases
NCT04014530 Recruiting Drug: Ataluren + Pembrolizumab Colorectal Cancer|Endometrium Cancer Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)|Merck Sharp & Dohme Corp.|PTC Therapeutics August 1 2019 Phase 1|Phase 2
NCT02758626 Active not recruiting Drug: ataluren|Drug: Placebo Epilepsy NYU Langone Health|PTC Therapeutics November 2016 Phase 2
NCT02090959 Completed Drug: Ataluren Muscular Dystrophy Duchenne|Muscular Dystrophies|Muscular Disorders Atrophic|Muscular Diseases|Musculoskeletal Diseases|Neuromuscular Diseases|Nervous System Diseases|Genetic Diseases X-Linked|Genetic Diseases Inborn PTC Therapeutics March 31 2014 Phase 3
NCT01826487 Completed Drug: Ataluren|Drug: Placebo Muscular Dystrophy Duchenne|Muscular Dystrophies|Muscular Disorders Atrophic|Muscular Diseases|Musculoskeletal Diseases|Neuromuscular Diseases|Nervous System Diseases|Genetic Diseases X-Linked|Genetic Diseases Inborn PTC Therapeutics March 2013 Phase 3
NCT01557400 Completed Drug: Ataluren Duchenne Muscular Dystrophy|Becker Muscular Dystrophy|Dystrophinopathy PTC Therapeutics May 31 2012 Phase 3
NCT01140451 Completed Drug: Ataluren (PTC124) Cystic Fibrosis PTC Therapeutics|Cystic Fibrosis Foundation August 31 2010 Phase 3

Tech Support

Answers to questions you may have can be found in the inhibitor handling instructions. Topics include how to prepare stock solutions, how to store inhibitors, and issues that need special attention for cell-based assays and animal experiments.

Handling Instructions

Tel: +1-832-582-8158 Ext:3

If you have any other enquiries, please leave a message.

  • * Indicates a Required Field

CFTR Signaling Pathway Map

Related CFTR Products

Tags: buy Ataluren (PTC124) | Ataluren (PTC124) supplier | purchase Ataluren (PTC124) | Ataluren (PTC124) cost | Ataluren (PTC124) manufacturer | order Ataluren (PTC124) | Ataluren (PTC124) distributor
×
Cell Lines Assay Type Concentration Incubation Time Formulation Activity Description PMID