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.
Cited by 14 Publications
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Effect of systemic ataluren treatment on mice with the Pax6Sey+/- phenotype. The black arrowhead indicates the lenticular stalk; the black arrow indicates the cornea; and the asterisk indicates the ciliary margin. WT, Pax6+/+; Mt, Pax6Sey/+; L, lens; r, retina. Original magnification, x5.
J Clin Invest 2014 124(1), 111-6. Ataluren (PTC124) purchased from Selleck.
The effect of PTC124, compared with DMSO vehicle, was tested on the subunit c storage in TPP1 patient NPCs. Representative micrograph images are shown of cells immunostained to detect subunit c storage, following 72 h of treatment with 0.5% DMSO or 18 uM PTC124.
Hum Mol Genet 2014 23(8), 2005-22. Ataluren (PTC124) purchased from Selleck.
Control and R120X RPE cells were stained for endogenous Gβ1 (green) and the membrane marker WGA (red). Gβ1 plasma membrane localization is disrupted in R120X RPE cells, but restored with treatment of either G418 or PTC124. N = 3 independent experiments with 300 cells per experiment.
Hum Mol Genet 2014 10.1093/hmg/ddu42. Ataluren (PTC124) purchased from Selleck.
Endogenous full-length RP2 cDNA and protein can be restored in R120X fibroblasts by treatment with G418 and PTC124. (A) Treatment of R120X fibroblasts with a single 24 h dose of 500 uM G418 significantly increased RP2 mRNA levels compared with untreated cells. *P ≤ 0.05, values are mean ± 2 SEM, N = 3. (B) Agarose gel showing products of RT-qPCR amplification. GAPDH was used as an amplification control. (C) Treatment of R120X fibroblasts with a single 24 h dose of 750 uM, 500 uM G418 or 10 ug/ml PTC124 significantly increased RP2 protein levels compared with untreated R120X cells. *P ≤ 0.05, values are mean ± 2 SEM, N = 2. (D) Western blot showing increased RP2 protein levels in G418 and PTC124 treated R120X fibroblasts compared with untreated R120X cells.
Hum Mol Genet 2014 10.1093/hmg/ddu42. Ataluren (PTC124) purchased from Selleck.
Mesenchymal stromal cells (MSCs) were freshly isolated from bone marrow biopsies and incubated with 2.5-5 μM ataluren for 48, 72, or 96 hours or vehicle alone (DMSO).
Am J Hematol, 2018, 93(4):527-536. Ataluren (PTC124) purchased from Selleck.
Quantification by qRT-PCR of PCCA (for P1 and P2) and PCCB (P3 and P4) mRNA levels in fibroblast samples untreated or cultured with different concentrations of readthrough drugs. The y-axis represents the results of the relative quantification expressed as percentages. Data represent the mean ?SD of two experiments performed in triplicate. DMSO, dimethyl sulfoxide.
Hum Mutat 2012 33, 973-80. Ataluren (PTC124) purchased from Selleck.
Tissue-dependent effectiveness of the read-through drug, ataluren (PTC124), in Cln1 R151X mice. Two-month old Cln1 R151X male mice were treated with ataluren (10 mg/kg, i.p.) four times a day for 2 days. Control mice were treated with the vehicle of the drug. Immediately following the last injection on the second day, tissues were collected, and PPT1 enzyme activity was measured in the brain (cerebellum, cortex, striatum/thalamus, brainstem) and peripheral tissues (liver, heart, lung, kidney, skeletal muscle). Ataluren only increased PPT1 activity in the liver and muscle. Columns and bars represent mean ± S.E.M. (eight wild-type and eight Cln1 R151X mice). Statistical significance was determined by unpaired t-test.: *p=0.004 for liver, *p=0.009 for skeletal muscle.
J Cell Mol Med, 2016, 20(2):381-5 . Ataluren (PTC124) purchased from Selleck.
Quantitative RT-PCR analysis of CPT1A in normal and patient fibroblasts treated with various concentration of PTC124 (0, 1, 3 and 5 μM) for 72 hours. The relative contents of mRNA were calculated by using the ΔΔCt method. Expression was normalized to human GAPDH and no significant fold difference was observed in the patient mRNA levels after treatment with PTC124. The normal control cells showed approximately 10-fold higher expression than patient cells. Data are presented as mean; Error bar indicates SEM
Dr. Lu Tan of Children’s Hospital of Philadelphia. Ataluren (PTC124) purchased from Selleck.
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Choose Selective CFTR Inhibitors
|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.|
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. |
|In vitro||DMSO||57 mg/mL (200.53 mM)|
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1% DMSO+30% polyethylene glycol+1% Tween 80
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Clinical Trial Information
|NCT Number||Recruitment||Conditions||Sponsor/Collaborators||Start Date||Phases|
|NCT02758626||Recruiting||Epilepsy||New York University School of Medicine|PTC Therapeutics||November 2016||Phase 2|
|NCT02819557||Recruiting||Duchenne Muscular Dystrophy||PTC Therapeutics||June 2016||Phase 2|
|NCT02647359||Recruiting||Aniridia||PTC Therapeutics||January 2016||Phase 2|
|NCT02456103||Enrolling by invitation||Cystic Fibrosis||PTC Therapeutics||July 2015||Phase 3|
|NCT02409004||Not yet recruiting||Healthy||PTC Therapeutics||March 2015||Phase 1|
|NCT02139306||Active, not recruiting||Cystic Fibrosis||PTC Therapeutics|Cystic Fibrosis Foundation Therapeutics|European Cystic Fibrosis Society-Clinical Trial Network (ECFS-CTN)||June 2014||Phase 3|
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