Ataluren (PTC124)
- CAS No.
- 775304-57-9
- Chemical Name:
- Ataluren (PTC124)
- Synonyms
- Ataluren;Ptc124;CS-749;Ptc-124;Ptc 124;Atarulon;Ataluren [usan];Ataluren, >=98%;PTC-124,ataluren;Ataluren (PTC124)
- CBNumber:
- CB02128667
- Molecular Formula:
- C15H9FN2O3
- Molecular Weight:
- 284.24
- MOL File:
- 775304-57-9.mol
- MSDS File:
- SDS
- Modify Date:
- 2024/4/24 17:21:39
| Melting point | 241 - 242°C |
|---|---|
| Boiling point | 503.7±60.0 °C(Predicted) |
| Density | 1.379 |
| storage temp. | Refrigerator |
| solubility | DMSO (Slightly) |
| form | White to off-white solid. |
| pka | 3.58±0.10(Predicted) |
| color | White to Off-White |
| InChI | 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) |
| InChIKey | OOUGLTULBSNHNF-UHFFFAOYSA-N |
| SMILES | C(O)(=O)C1=CC=CC(C2N=C(C3=CC=CC=C3F)ON=2)=C1 |
| CAS DataBase Reference | 775304-57-9 |
SAFETY
Risk and Safety Statements
| Symbol(GHS) |   GHS07,GHS08 |
|---|---|
| Signal word | Warning |
| Hazard statements | H336-H373 |
| Precautionary statements | P260-P271-P304+P340+P312-P314-P403+P233-P405 |
| HS Code | 2933998090 |
Ataluren (PTC124) Chemical Properties,Uses,Production
Description
Ataluren is a drug marketed under the trade name Translarna® which was developed by PTC Therapeutics and approved by the European Union in May 2014 for the treatment of Duchenne’s muscular dystrophy (DMD) and potentially other genetic disorders. Ataluren renders ribosomes less sensitive to premature stop or ‘read-through’ codons, which are thought to be beneficial in diseases such as DMD and cystic fibrosis.
Uses
Nonsense mutations create a premature termination of mRNA translation and have been implicated in various genetic disorders, including muscular dystrophy and cystic fibrosis. PTC-124 is a nonaminoglycoside that has been reported to selectively induce ribosomes to read through premature nonsense stop signals on mRNA, thus allowing the production of full-length, functional proteins. In a mouse model of cystic fibrosis caused by nonsense mutations, PTC-124 treatment (60 mg/kg s.c. injection or 0.3-0.9 mg/ml orally) has been shown to restore cystic fibrosis transmembrane conductance regulator (CFTR) protein expression and function. The target activity of PTC-124 was initially evaluated by firefly luciferase reporter cell-based nonsense codon assay (IC50 = 7 nM); however, subsequent assessments using a Renilla reniformis luciferase reporter have failed to produce nonsense codon suppression activity. Thus, while PTC-124 is in clinical testing in patients with nonsense mutations within the CFTR or dystrophin genes, controversy surrounds its exact mechanism of action.[Cayman Chemical]
Synthesis
The sequence to construct ataluren, which was described by the authors at PTC Therapeutics, commenced with commercially available methyl 3-cyanobenzoate (38). This ester was exposed to hydroxylamine in aqueous tert-butanol and warmed gently until the reaction was deemed complete. Then this mixture was treated with 2-fluorobenzoyl chloride dropwise and subsequently triethylamine dropwise. To minimize exotherm and undesired side products, careful control of the addition of reagents was achieved through slow dropwise addition of these liquid reagents. Upon complete consumption of starting materials and formation of amidooxime 39, the aqueous reaction mixture was then heated to 85 ?? to facilitate 1,2,4-oxadiazole formation, resulting in the tricyclic ester 40 in excellent yield across the three steps. Finally, saponification of ester 40 through the use of sodium hydroxide followed by acidic quench gave ataluren (V) in 96% over the two-step sequence.
Ataluren (PTC124) Preparation Products And Raw materials
Raw materials
1of2
chevron_rightPreparation Products
| Supplier | Tel | Country | ProdList | Advantage | Inquiry |
|---|---|---|---|---|---|
| A.J Chemicals | 91-9810153283 | New Delhi, India | 6124 | 58 | Inquiry |
| CLEARSYNTH LABS LTD. | +91-22-45045900 | Hyderabad, India | 6351 | 58 | Inquiry |
| Shanghai Famo Biotech Co Ltd | +8618550473860 | China | 513 | 58 | Inquiry |
| shandong perfect biotechnology co.ltd | +86-53169958659; +8618596095638 | China | 294 | 58 | Inquiry |
| Henan Bao Enluo International TradeCo.，LTD | +86-17331933971 +86-17331933971 | China | 2503 | 58 | Inquiry |
| Hangzhou ICH Biofarm Co., Ltd | +86-0571-28186870; +undefined8613073685410 | China | 985 | 58 | Inquiry |
| Capot Chemical Co.,Ltd. | 571-85586718 +8613336195806 | China | 29798 | 60 | Inquiry |
| ATK CHEMICAL COMPANY LIMITED | +undefined-21-51877795 | China | 32760 | 60 | Inquiry |
| career henan chemical co | +86-0371-86658258 +8613203830695 | China | 29901 | 58 | Inquiry |
| Chongqing Chemdad Co., Ltd | +86-023-6139-8061 +86-86-13650506873 | China | 39916 | 58 | Inquiry |
775304-57-9(Ataluren (PTC124))Related Search:
1of4
chevron_right