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.

Date of Patent:
May. 06, 2025

Filed:

Jul. 07, 2021
Applicant:

University of Pittsburgh—of the Commonwealth System of Higher Education, Pittsburgh, PA (US);

Inventors:

Gerard Vockley, Pittsburgh, PA (US);

Al-Walid A. Mohsen, Pittsburgh, PA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 31/225 (2005.12); A61P 3/00 (2005.12); C07C 69/40 (2005.12); C07C 69/66 (2005.12);
U.S. Cl.
CPC ...
A61K 31/225 (2012.12); A61P 3/00 (2017.12); C07C 69/66 (2012.12);
Abstract

Methods of treating propionic acidemia (PA), methylmalonic acidemia (MMA) and fatty acid oxidation disorders are described. The methods include administering an anaplerotic agent that can directly enter the tricarboxylic acid cycle, such as a succinate derivative or pro-drug, for example trisuccinylglycerol (TSG). Methods of restoring tricarboxylic acid (TCA) cycle function in a cell deficient for propionyl-CoA carboxylase (PCC) or methylmalonyl-CoA mutase (MUT) by contacting the cell with a succinate derivative or pro-drug, such as TSG, are also described.


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