Ataluren (PTC124)
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MedKoo CAT#: 205786

CAS#: 775304-57-9

Description: Ataluren, also known as PTC124, is an investigational new drug designed to enable the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. Ataluren is currently being investigated for use in patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) and cystic fibrosis (nmCF)


Chemical Structure

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Ataluren (PTC124)
CAS# 775304-57-9

Theoretical Analysis

MedKoo Cat#: 205786
Name: Ataluren (PTC124)
CAS#: 775304-57-9
Chemical Formula: C15H9FN2O3
Exact Mass: 284.05972
Molecular Weight: 284.24196
Elemental Analysis: C, 63.38; H, 3.19; F, 6.68; N, 9.86; O, 16.89

Price and Availability

Size Price Availability Quantity
100.0mg USD 550.0 2 Weeks
200.0mg USD 950.0 2 Weeks
500.0mg USD 1450.0 2 Weeks
1.0g USD 2450.0 2 Weeks
2.0g USD 3850.0 2 Weeks
5.0g USD 5950.0 2 Weeks
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Synonym: PTC124; PTC 124; PTC-124; Ataluren.

IUPAC/Chemical Name: 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid

InChi Key: OOUGLTULBSNHNF-UHFFFAOYSA-N

InChi Code: 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)

SMILES Code: O=C(O)C1=CC=CC(C2=NOC(C3=CC=CC=C3F)=N2)=C1

Appearance: Solid powder

Purity: >98% (or refer to the Certificate of Analysis)

Shipping Condition: Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.

Storage Condition: Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).

Solubility: Soluble in DMSO, not in water

Shelf Life: >5 years if stored properly

Drug Formulation: This drug may be formulated in DMSO

Stock Solution Storage: 0 - 4 C for short term (days to weeks), or -20 C for long term (months).

HS Tariff Code: 2934.99.9001

Preparing Stock Solutions

The following data is based on the product molecular weight 284.24196 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

Recalculate based on batch purity %
Concentration / Solvent Volume / Mass 1 mg 5 mg 10 mg
1 mM 1.15 mL 5.76 mL 11.51 mL
5 mM 0.23 mL 1.15 mL 2.3 mL
10 mM 0.12 mL 0.58 mL 1.15 mL
50 mM 0.02 mL 0.12 mL 0.23 mL

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1: Koopmann TT, Verkerk AO, Bezzina CR, de Bakker JM, Wilde AA. The Chemical Compound PTC124 Does Not Affect Cellular Electrophysiology of Cardiac Ventricular Myocytes. Cardiovasc Drugs Ther. 2012 Feb;26(1):41-5. PubMed PMID: 22065308.

2: Sarkar C, Zhang Z, Mukherjee AB. Stop codon read-through with PTC124 induces palmitoyl-protein thioesterase-1 activity, reduces thioester load and suppresses apoptosis in cultured cells from INCL patients. Mol Genet Metab. 2011 Nov;104(3):338-45. Epub 2011 Jun 13. PubMed PMID: 21704547; PubMed Central PMCID: PMC3220191.

3: Tan L, Narayan SB, Chen J, Meyers GD, Bennett MJ. PTC124 improves readthrough and increases enzymatic activity of the CPT1A R160X nonsense mutation. J Inherit Metab Dis. 2011 Apr;34(2):443-7. Epub 2011 Jan 21. PubMed PMID: 21253826.

4: Goldmann T, Overlack N, Wolfrum U, Nagel-Wolfrum K. PTC124-mediated translational readthrough of a nonsense mutation causing Usher syndrome type 1C. Hum Gene Ther. 2011 May;22(5):537-47. Epub 2011 Mar 25. PubMed PMID: 21235327.

5: Wilschanski M, Miller LL, Shoseyov D, Blau H, Rivlin J, Aviram M, Cohen M, Armoni S, Yaakov Y, Pugatsch T, Cohen-Cymberknoh M, Miller NL, Reha A, Northcutt VJ, Hirawat S, Donnelly K, Elfring GL, Ajayi T, Kerem E. Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. Eur Respir J. 2011 Jul;38(1):59-69. Epub 2011 Jan 13. Erratum in: Eur Respir J. 2011 Oct;38(4):996. Pugatch, T [corrected to Pugatsch, T]. PubMed PMID: 21233271.

6: Sermet-Gaudelus I, Boeck KD, Casimir GJ, Vermeulen F, Leal T, Mogenet A, Roussel D, Fritsch J, Hanssens L, Hirawat S, Miller NL, Constantine S, Reha A, Ajayi T, Elfring GL, Miller LL. Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis. Am J Respir Crit Care Med. 2010 Nov 15;182(10):1262-72. Epub 2010 Jul 9. PubMed PMID: 20622033.

7: Finkel RS. Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124). J Child Neurol. 2010 Sep;25(9):1158-64. Epub 2010 Jun 2. Review. PubMed PMID: 20519671.

8: Auld DS, Lovell S, Thorne N, Lea WA, Maloney DJ, Shen M, Rai G, Battaile KP, Thomas CJ, Simeonov A, Hanzlik RP, Inglese J. Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124. Proc Natl Acad Sci U S A. 2010 Mar 16;107(11):4878-83. Epub 2010 Mar 1. PubMed PMID: 20194791; PubMed Central PMCID: PMC2841876.

9: Peltz SW, Welch EM, Jacobson A, Trotta CR, Naryshkin N, Sweeney HL, Bedwell DM. Nonsense suppression activity of PTC124 (ataluren). Proc Natl Acad Sci U S A. 2009 Jun 23;106(25):E64; author reply E65. Epub 2009 Jun 8. PubMed PMID: 19506240; PubMed Central PMCID: PMC2700894.

10: Goodier JL, Mayer J. PTC124 for cystic fibrosis. Lancet. 2009 Apr 25;373(9673):1426; author reply 1426-7. PubMed PMID: 19394530.



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