Compounds and method for treating or preventing disease conditions associated with alpha-1-antitrypsin
AssigneeAmicus Therapeutics, Inc.
InventorsEric Richard Sjoberg, Gary Lee
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Abstract
The present invention provides compounds and methods for the treatment of an individual having or at risk of having a condition associated with alpha-1-antitrypsin by using a pharmacological chaperone. In particular, such methods are useful for the treatment and/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. The present invention also provides an assay for determining whether a test compound modulates alpha-1-antitrypsin activity.
Claims8
A method of treating an individual having a disease condition associated with α-1-antitrypsin comprising administering to the individual an effective amount of a pharmacological chaperone, wherein the pharmacological chaperone is:
- wherein:
- R1 is —(CH2)2-COOH or —CH2-OH;
- R2 is —CH3 or —CH—(CH3)2;
- wherein:
- R3 is —H or —CH3;
- R4 is —H or —OCH3;
- R5 is —H or —OH;
- or combinations of two or more thereof.
A method of treating an individual having a disease condition associated with α-1-antitrypsin comprising administering to the individual an effective amount of a pharmacological chaperone, wherein the pharmacological chaperone is:
- Ac-Thr-Glu-Ala-Ala-NH2 (SEQ ID NO:
- 4),
- Ac-Thr-Glu-Ala-Ala-Gly-NH2 (SEQ ID NO:
- 1),
- Ac-Thr-Ser-Ala-Ala-NH2 (SEQ ID NO:
- 2),
- Ac-Thr-Glu-Val-Ala-NH2 (SEQ ID NO:
- 3), or combinations of two or more thereof.
A method of treating an individual having a disease condition associated with α-1-antitrypsin comprising administering to the individual an effective amount of a pharmacological chaperone, wherein the individual has a T339C or S292C missense mutation in α-1-antitrypsin in at least one allele.
Dependencies are inferred from the claim text.