Disease Area, Lung Disease II
Product Name: PTC-124 2(Ataluren) | Avoid early termination caused by nonsense
mutation (#C7124-10)
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PTC-124 (Ataluren) is an oxadiazole-based, orally available agent deisgned to selectively promote ribosomal
readhtrough of premature stop codons, but not normal termination codons. [1] The minimal concentration of
PTC124 showing observable readthrough was 0.01 - 0.1 uM (2.8 - 28 ng/ml), while maximal activity was
observed at 3 uM (852 ng/ml). [2]
PTC-124 has been studied extensively in the treatment of cystic fibrosis and has shown good tolerability and
efficacy in preclinical and clinical settings. [3, 4]
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Details
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Chemical Formula:
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C15H9FN2O3
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CAS No.:
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775304-57-9
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Molecular weight:
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284.24
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Purity:
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> 98%
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Appearance:
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White
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Chemical name:
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3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)benzoic acid
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Solubility:
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Up to 100 mM in DMSO
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Synonyms:
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PTC-124, PTC124, PTC 124
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Storage:
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For longer shelf life, store solid powder at 4oC desiccated, or store DMSO solution
at -20oC
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References
1. Sermet-Gaudelus et al., 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, 182, 1262-1272. Pubmed ID: 20622033
2. Welch et al., PTC124 targets genetic disorders caused by nonsense mutations. Nature, 2007, 447, 87-91.
Pubmed ID: 17450125
3. Du et al., PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-
G542X nonsense allele in a CF mouse model. Proc. Natl. Acad. Sci. 2008, 105(6), 2064-2069
Pubmed ID: 18272502
4. Wilschanski et al., Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. Eur. Respir.
J. 2011, 38, 59-69. Pubmed ID: 17450125
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Product Name: VX-770 (Ivacaftor) | CFTR potentiator (#C8770-5)
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VX-770 (Ivacaftor) is a CFTR potentiator and has been shown to potentiate normal CFTR, as well as CFTR
with G551D and F508-del mutations. Ivacaftor directly binds to the ion channel, causes conformation change
and open the ion channel to improve chloride transportation. In primary cultured human CF bronchial epithelia
(HBE) carrying the G551D and F508del CFTR mutations, Ivacaftor (10 ?M) potently increases the forskolin-
stimulated Cl- secretion with an EC50 of 236 nM. [1]
VX-770 (Ivacaftor) is approved for cystic fibrosis patients with G551D mutation, and has shown efficacy in a
patient with S549N mutation. [2]
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Details
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Chemical Formula:
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C24H28N2O3
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CAS No.:
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873054-44-5
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Molecular weight:
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392.49
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Purity:
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> 98%
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Appearance:
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Off-white
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Chemical name:
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N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide
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Solubility:
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Up to 100 mM in DMSO
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Synonyms:
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VX-770, VX770, lvacaftor, Kalydeco
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Storage:
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For longer shelf life, store solid powder or DMSO solution at -20oC
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1. Van Goor F et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. , Proc
Natl Acad Sci U S A. 2009; 106(44):18825-30. Pubmed ID: 19846789
2. McGarry ME and Nielson DW. Normalization of sweat chloride concentration and clinical improvement with
ivacaftor in a patient with cystic fibrosis with mutation S549N. Chest. 2013; 144(4):1376-8.
Pubmed ID: 24081349
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