Premature-termination-codons readthrough compounds

Patent metadata

Field	Value
Publication number	US-9255088-B2
Application number	US-201313764748-A
Country	US
Kind code	B2
Filing date	Feb 11, 2013
Priority date	Aug 11, 2010
Publication date	Feb 9, 2016
Grant date	Feb 9, 2016

Abstract

Premature termination codons readthrough compounds, composition thereof, and methods of making and using the same are provided.

First claim

We claim:

1. A compound having the ability to increase read through of premature termination codons (PTCs) in RNA, a pharmaceutically acceptable salt thereof, having a structure of formula (VIII):

wherein:
X is O;
R 3 is an ortho, meta, or para group and is a hydrogen, halo, or nitro; and
R 4 is hydrogen, —C(O)H, halo, or a C1-C6 group.

2. A compound having the ability to read through premature termination codons (PTCs) in RNA, a pharmaceutically acceptable salt thereof, having a structure of formula (IX):

3. The compound of claim 1 where the compound is selected from:

4. The compound of claim 1 , having one of the following formulas:

5. A composition comprising the compound of claim 1 and a pharmaceutically acceptable carrier.

6. A method of treating or ameliorating a medical condition associated with premature termination codons (PTCs) in RNA, comprising: administering to a subject a compound according to claim 1 , wherein the subject has an ATM gene that encodes one or more PTCs, or a DMD gene that encodes one or more PTCs.

Assignees

• Univ California

Inventors

• Gatti Richard
• Du Liutao
• Damoiseaux Robert
• Lai Chih-Hung
• Jung Michael
• Ku Jin-Mo
• Bertoni Carmen

Classifications

• C07C335/16

  having nitrogen atoms of thiourea groups bound to carbon atoms of six-membered aromatic rings of a carbon skeleton · CPC title

• C07D409/06

  linked by a carbon chain containing only aliphatic carbon atoms · CPC title

• C07C215/76

  of the same non-condensed six-membered aromatic ring · CPC title

• C07D221/16

  containing carbocyclic rings other than six-membered · CPC title

• C07C207/04

  the carbon skeleton being further substituted by singly-bound oxygen atoms · CPC title