Compounds and their use for treating somatic mutation-related diseases

A compound, hydrate technology, applied in the treatment or prevention of diseases related to mRNA nonsense mutation, inhibiting premature translation termination related to mRNA nonsense mutation, can solve the problem of no public application of clitocine, no report of development of clitocine analogs or Derivatives etc.

Inactive Publication Date: 2007-11-21
PTC THERAPEUTICS INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

However, the use of clitocine as a therapeutic agent for diseases associated with nonsense mutations has not been disclosed so far
There is also no rep...

Method used

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  • Compounds and their use for treating somatic mutation-related diseases
  • Compounds and their use for treating somatic mutation-related diseases
  • Compounds and their use for treating somatic mutation-related diseases

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[0356] B. Preparation of Compounds of the Invention

[0357] The compounds of the invention may be prepared in any manner known in the art. For example, compounds of the present invention can be prepared according to the general methods described below. For example, certain 1,3,4-oxadiazoles of Formula 1 can be prepared as shown in Scheme A1 below:

[0358]

[0359] Route A1

[0360] According to Scheme A1, benzonitriles of structure Al can be converted to tetrazoles of structure A2 by treatment with eg sodium azide. Treatment of tetrazole A2 with an activated carboxylic acid such as an acid chloride or an acid activated with a dehydrating agent such as dicyclohexylcarbodiimide in a suitable solvent affords the 1,3,4-oxadiazole compound of formula 1-A. Suitable solvents include, but are not limited to, eg toluene or dichloroethane. The reaction can generally be carried out at a temperature in the range of 60-150°C.

[0361]In another embodiment...

Embodiment 1

[0555] Embodiment 1: the preparation of the compound of the present invention

Embodiment A

[0556] Example A: Preparation of 3-[5-(4-isopropylphenyl)-[1,3,4]oxadiazol-2-yl]benzoic acid (Compound No. 6)

[0557]

[0558] Step A: A suspension of methyl 3-cyanobenzoate (5.05 g, 31.4 mmol), sodium azide (3.06 g, 47.0 mmol) and triethylamine hydrochloride (6.47 g, 47 mmol) in 60 mL of toluene was heated Reflux for 12 hours, then cool to room temperature. The heterogeneous mixture was diluted with water and the phases were separated. The organic layer was extracted with saturated sodium bicarbonate, and the aqueous phases were combined and washed with EtOAc. After discarding the organic layer, the combined aqueous phases were acidified to about pH 2 with 6N hydrochloric acid, and the resulting thick paste was extracted with EtOAc (2x). The combined organic layers were washed with saturated NaCl, then dried and concentrated to give 5.30 g (83%) of methyl 3-(1H-tetrazol-5-yl)benzoate as a white solid: mp 180-181 °C; MS m / z 205.1[MH + ].

[0559] Step B: Methyl 3-(1H...

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Abstract

The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.

Description

[0001] related application [0002] This application claims priority and the benefit of US Application 60 / 617,653, filed October 13, 2004, and US Application 60 / 624,170, filed November 3, 2004, under 35 U.S.C. §119. US Application 60 / 624,170, filed November 3, 2004, is hereby incorporated by reference in its entirety. Pursuant to 35 U.S.C. §119, this application claims U.S. Application 60 / 617,655, filed October 13, 2004, U.S. Application 60 / 617,634, filed October 13, 2004, U.S. Application, filed October 13, 2004 60 / 617,633, priority and benefit of US Application 60 / 617,670, filed October 13, 2004, which are hereby incorporated by reference in their entirety. This application also incorporates by reference the entire contents of International Patent Applications filed on October 13, 2005, entitled "Compounds for Nonsense Suppression, and Method for Their Use" and Attorney Docket Nos. identified as 19025.040, 19025.041, 19025.043, and 19025.044. technical field [0003] The p...

Claims

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Application Information

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IPC IPC(8): F28G3/10
Inventor N・阿尔姆斯戴德陈光明G・M・卡普E・韦尔奇R・怀尔德J・A・坎贝尔
Owner PTC THERAPEUTICS INC
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