アタルレン
アタルレン 物理性質
- 融点 :
- 241 - 242°C
- 沸点 :
- 503.7±60.0 °C(Predicted)
- 比重(密度) :
- 1.379
- 貯蔵温度 :
- Refrigerator
- 溶解性:
- DMSO (わずかに)
- 外見 :
- 白からオフホワイトの固体。
- 酸解離定数(Pka):
- 3.58±0.10(Predicted)
- 色:
- ホワイトからオフホワイト
- 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 データベース:
- 775304-57-9
安全性情報
- リスクと安全性に関する声明
- 危険有害性情報のコード(GHS)
| 絵表示（GHS） |
 
|
| 注意喚起語 |
警告 |
| 危険有害性情報 |
| コード |
危険有害性情報 |
危険有害性クラス |
区分 |
注意喚起語 |
シンボル |
P コード |
| H336 |
眠気やめまいのおそれ |
特定標的臓器毒性、単回暴露； 麻酔作用 |
3 |
警告 |
|
P261, P271, P304+P340, P312,P403+P233, P405, P501 |
| H373 |
長期にわたる、または反復暴露により臓器の障 害のおそれ |
特定標的臓器有害性、単回暴露 |
2 |
警告 |
|
P260, P314, P501 |
|
| 注意書き |
| P260 |
粉じん/煙/ガス/ミスト/蒸気/スプレーを吸入しないこ と。 |
| P271 |
屋外または換気の良い場所でのみ使用すること。 |
| P314 |
気分が悪い時は、医師の診断/手当てを受けること。 |
| P403+P233 |
換気の良い場所で保管すること。容器を密閉 しておくこと。 |
| P405 |
施錠して保管すること。 |
|
アタルレン 価格
| メーカー |
製品番号 |
製品説明 |
CAS番号 |
包装 |
価格 |
更新時間 |
購入 |
アタルレン 化学特性,用途語,生産方法
効能
デュシェンヌ型筋ジストロフィー治療薬
説明
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.
使用
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]
作用機序
The mechanism of action of Ataluren (PTC124) is to generate functionally normal myotonic dystrophy proteins by facilitating ribosomal read-through of nonsense mutations, thereby bypassing the pathogenic variant and continuing the translation process. Under normal conditions, ribosomes move along the mRNA that links amino acids into proteins until they reach the stop codon. When the ribosome encounters a premature termination codon (PTC) due to a nonsense mutation, eukaryotic release factors [eRF1 (green) and eRF3 (turquoise) complexed with GTP (yellow)] are recruited, and translation is terminated prematurely to produce the truncated protein. Ataluren is thought to interact with the ribosome to promote the recruitment of the proximity-recognition tRNAs, which in turn inhibits the nonsense mutation, allowing the PTC to be read through and synthesised. PTC to be read through and synthesise full-length proteins. The red amino acid on the near-recognition tRNA is incorporated into the read-through protein product. In the Ataluren-mediated nonsense repression model, the X on the tRNA indicates a mispairing at codon position three.
合成
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.
アタルレン 上流と下流の製品情報
原材料
準備製品
アタルレン 生産企業
Global( 230)Suppliers
775304-57-9(アタルレン)キーワード:
クロロスルホニル酢酸エチルエステル
フルシラゾール
3-(トリフルオロメチル)安息香酸
葉酸水和物
4-フェニル安息香酸
rac-1-[(R*)-2-(2,4-ジクロロフェニル)-3-(1,1,2,2-テトラフルオロエトキシ)プロピル]-1H-1,2,4-トリアゾール
エポキシコナゾル
フルキンコナゾール
3-(5-メチル-1,2,4-オキサジアゾール-3-イル)安息香酸
アタルレン
3-(5-メチル-1,2,4-オキサジアゾール-3-イル)ベンズアルデヒド
[3-(5-メチル-1,2,4-オキサジアゾール-3-イル)フェニル]メタノール
- 775304-57-9
- Ptc-124
- PTC-124,ataluren
- Ataluren (PTC124)
- Ataluren PTC 124 Ataluren
- Ataluren, >=98%
- PTC124, 775304-57-9
- 3-[5-(2-Fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid
- Ataluren [usan]
- Benzoic acid, 3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)-
- Ptc 124
- Atarulon
- CS-749
- ATALUREN;PTC-124;PTC 124
- Ataluren (PTC124) USP/EP/BP
- Ptc124
- Ataluren
- 4H-1-Benzopyran-4-one,6-bromo-2,3-dihydro-2,7-dimethyl-
- Estr-4-en-3-one,17-hydroxy-,(21β)-
- アタルレン
- 3-[5-(2-フルオロフェニル)-1,2,4-オキサジアゾール-3-イル]安息香酸
- PTC-124