Compounds and methods for treating or preventing disease conditions associated with α-1-antitrypsin
View Patent ↗Pharmacological chaperone compounds and methods for the treatment of an individual having, or at risk of having, a disease condition associated with alpha-1-antitrypsin by using said compounds are disclosed. In particular, such methods are useful for the treatment or prevention of lung disorders associated with alpha-1-antitrypsin deficiency as well as liver disorders associated with an excess of alpha-1-antitrypsin. Suitable pharmacological chaperones include peptides and low-molecular weight compounds. Also provided is an assay for determining whether a test compound modulates alpha-1-antitrypsin activity.
1. A compound according to the structure:
wherein:
R1 is —(CH 2 ) 2 —COOH or —CH 2 —OH
R2 is —CH 3 or —CH—(CH 3 ) 2 .
2. The compound of claim 1 , wherein R1 is —CH 2 —OH and R2 is —CH 3 .
3. The compound of claim 1 , wherein R1 is —(CH 2 ) 2 —COOH and R2 is —CH—(CH 3 ) 2 .
4. The compound of claim 1 , wherein R1 is —(CH 2 ) 2 —COOH and R2 is —CH 3 .
5. A compound according to the structure:
wherein
R 3 is —H or —CH 3
R 4 is —H or —OCH 3
R 5 is —H or —OH.
6. The compound of claim 5 having the structure:
7. The compound of claim 5 having the structure:
8. The compound of claim 5 having the structure:
9. The compound of claim 5 having the structure:
10. The compound of claim 5 having the structure:
11. The compound of claim 5 having the structure: