Compounds and methods for treating or preventing disease conditions associated with α-1-antitrypsin

US8993525B2 · US · B2

Patent metadata
FieldValue
Publication numberUS-8993525-B2
Application numberUS-201113879160-A
CountryUS
Kind codeB2
Filing dateOct 14, 2011
Priority dateOct 14, 2010
Publication dateMar 31, 2015
Grant dateMar 31, 2015

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  1. Title

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  2. Abstract

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  5. First independent claim

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Abstract

Official abstract text for this publication.

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.

First claim

Opening claim text (preview).

The invention claimed is: 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. Th…

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What does patent US8993525B2 cover?
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 …
Who is the assignee on this patent?
Sjoberg Eric Richard, Lee Gary, Amicus Therapeutics Inc
What technology area does this patent fall under?
Primary CPC classification C07C231/06. Mapped technology areas include Chemistry & Metallurgy.
When was this patent published?
Publication date Tue Mar 31 2015 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
What related patents are in patentsdb?
We list 8 related publications on this page (citations in our corpus or others sharing the same primary CPC).