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The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Patent No.:

US 11840497 B1

PDFFull Text
Date of Patent:
Dec. 12, 2023

Filed:

Feb. 22, 2021

4-(p-quinonyl)-2-hydroxybutanamide derivatives for treatment of mitochondrial diseases

Applicant:

Ptc Therapeutics, Inc., South Plainfield, NJ (US);

Inventors:

Orion D. Jankowski, Burlingame, CA (US);

Kieron E. Wesson, Burlingame, CA (US);

Paul Mollard, Saratoga, CA (US);

William D. Shrader, Belmont, CA (US);

Assignee:

PTC Therapeutics, Inc., South Plainfield, NJ (US);

Attorney:

Squire Patton Boggs (US) LLP

Primary Examiner:

Erich A Leeser

Int. Cl.
CPC ...
C07C 235/80 (2006.01); C07C 235/32 (2006.01); C07C 235/78 (2006.01); C07C 317/28 (2006.01); C07D 207/27 (2006.01); C07D 211/46 (2006.01); C07D 213/40 (2006.01); C07D 233/61 (2006.01); C07D 295/13 (2006.01); C07D 295/192 (2006.01); C07D 265/30 (2006.01); C07D 311/66 (2006.01); C07D 207/26 (2006.01); C07D 213/50 (2006.01); C07D 295/116 (2006.01); C07D 295/185 (2006.01); A61P 21/00 (2006.01); A61P 25/00 (2006.01); A61P 25/16 (2006.01); A61P 27/00 (2006.01); C07D 213/51 (2006.01);
U.S. Cl.
CPC ...
C07C 235/80 (2013.01); A61P 21/00 (2018.01); A61P 25/00 (2018.01); A61P 25/16 (2018.01); A61P 27/00 (2018.01); C07C 235/32 (2013.01); C07C 235/78 (2013.01); C07C 317/28 (2013.01); C07D 207/26 (2013.01); C07D 207/27 (2013.01); C07D 211/46 (2013.01); C07D 213/40 (2013.01); C07D 213/51 (2013.01); C07D 233/61 (2013.01); C07D 265/30 (2013.01); C07D 295/116 (2013.01); C07D 295/13 (2013.01); C07D 295/185 (2013.01); C07D 295/192 (2013.01); C07D 311/66 (2013.01); C07C 2601/02 (2017.05); C07C 2601/16 (2017.05);
Abstract

Methods of treating or suppressing mitochondrial diseases, such as Friedreich's ataxia (FRDA), Leber's Hereditary Optic Neuropathy (LHON), mitochondrial myopathy, encephalopathy, lactacidosis, and stroke (MELAS), Kearns-Sayre Syndrome (KSS), are disclosed, as well as compounds useful in the methods of the invention, such as 4-(p-quinolyl)-2-hydroxybutanamide derivatives. Methods and compounds useful in treating other disorders such as amyotrophic lateral sclerosis (ALS), Huntington's disease, Parkinson's disease, and pervasive developmental disorders such as autism are also disclosed. Energy biomarkers useful in assessing the metabolic state of a subject and the efficacy of treatment are also disclosed. Methods of modulating, normalizing, or enhancing energy biomarkers, as well as compounds useful for such methods, are also disclosed.

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