1,3,4-oxadiazole benzoic acid compounds and their use for nonsense suppression and the treatment of disease
View Patent ↗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.
1. A compound of Formula 1-A:
wherein:
R 3 is absent;
W is selected from the group consisting of:
wherein when W is selected from the group consisting of:
then
shown in Formula 1-A is in the meta or para position; and
wherein when W is selected from the group consisting of
then
shown in Formula 1-A is in the meta position;
and a pharmaceutically acceptable salt of said compound of Formula 1-A, wherein * indicates the only bond of attachment for W, and wherein said bond of attachment is a direct bond of attachment.
2. A compound selected from the group consisting of:
and a pharmaceutically acceptable salt thereof.
3. The compound of claim 2 , wherein said compound is selected from the group consisting of:
and a pharmaceutically acceptable salt thereof.