IP Library › Granted Patent US 11,083,702
Granted Patent B2
US 11,083,702 · App. 16/094,828 · Granted Aug 10, 2021

Anaplerotic agents for treatment of disorders of propionate and long chain fat metabolism

Inventors: Gerard Vockley (Pittsburgh, PA); Al-Walid A. Mohsen (Pittsburgh, PA)
Assignee: University of Pittsburgh—Of the Commonwealth System of Higher Education
A61K31/225A61P3/00C07C69/66
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Quick Facts
Patent No.
US 11,083,702
App. No.
16/094,828
Granted
Aug 10, 2021
Kind
B2
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.

Claims (1)

1. A method of treating propionic (PA) or methylmalonic acidemia (MMA) in a subject, comprising administering to the subject a therapeutically effective amount of trisuccinylglycerol

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Dec 18, 2018
From: VOCKLEY, GERARD; MOHSEN, AL-WALID A.
To: UNIVERSITY OF PITTSBURGH - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION
Reel/Frame 047810/0993 →
Continuity (2)
Provisional Application 62324537 · Apr 19, 2016
Related Publication 20190117611A1 · Apr 25, 2019