US 12,290,500 B2
Anaplerotic agents for treatment of disorders of propionate and long chain fat metabolism
Gerard Vockley, Pittsburgh, PA (US); and Al-Walid A. Mohsen, Pittsburgh, PA (US)
Assigned to University of Pittsburgh—Of the Commonwealth System of Higher Education, Pittsburgh, PA (US)
Filed by University of Pittsburgh—Of the Commonwealth System of Higher Education, Pittsburgh, PA (US)
Filed on Jul. 7, 2021, as Appl. No. 17/369,268.
Application 17/369,268 is a division of application No. 16/094,828, granted, now 11,083,702, previously published as PCT/US2017/028100, filed on Apr. 18, 2017.
Claims priority of provisional application 62/324,537, filed on Apr. 19, 2016.
Prior Publication US 2021/0338624 A1, Nov. 4, 2021
Int. Cl. A61K 31/225 (2006.01); A61P 3/00 (2006.01); C07C 69/40 (2006.01); C07C 69/66 (2006.01)
CPC A61K 31/225 (2013.01) [A61P 3/00 (2018.01); C07C 69/66 (2013.01)] 10 Claims
 
1. A method of restoring tricarboxylic acid (TCA) cycle function in a cell deficient for propionyl-CoA carboxylase (PCC) or methylmalonyl-CoA mutase (MUT), comprising contacting the cell with a compound of formula II

OG Complex Work Unit Chemistry
or a pharmaceutically acceptable salt or hydrate thereof, wherein each R1 independently is H or a C1-6alkyl; each R3 independently is a C1-6alkyl; and n is 1;
wherein the compound of formula II is the only therapeutic agent used in the method.