US 12,251,392 B2
Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial DNA depletion syndromes
Michio Hirano, New York, NY (US); Caterina Garone, Cambridge (GB); and Ramon Marti, Barcelona (ES)
Assigned to The Trustees of Columbia University in the City of New York, New York, NY (US)
Filed by The Trustees of Columbia University in the City of New York, New York, NY (US)
Filed on Apr. 24, 2023, as Appl. No. 18/138,432.
Application 18/138,432 is a division of application No. 17/242,822, filed on Apr. 28, 2021, granted, now 11,666,592.
Application 17/242,822 is a continuation of application No. 16/583,852, filed on Sep. 26, 2019, granted, now 11,110,111, issued on Sep. 7, 2021.
Application 16/583,852 is a continuation of application No. 15/736,092, granted, now 10,471,087, issued on Nov. 12, 2019, previously published as PCT/US2016/038110, filed on Jun. 17, 2016.
Claims priority of provisional application 62/180,914, filed on Jun. 17, 2015.
Prior Publication US 2023/0277577 A1, Sep. 7, 2023
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 31/7072 (2006.01); A61K 9/00 (2006.01); A61K 31/513 (2006.01); A61K 31/7068 (2006.01); A61K 31/7076 (2006.01); A61K 31/708 (2006.01); A61K 45/06 (2006.01); A61P 21/00 (2006.01); A61P 43/00 (2006.01)
CPC A61K 31/7072 (2013.01) [A61K 9/0053 (2013.01); A61K 31/513 (2013.01); A61K 31/7068 (2013.01); A61K 31/7076 (2013.01); A61K 31/708 (2013.01); A61K 45/06 (2013.01); A61P 21/00 (2018.01); A61P 43/00 (2018.01)] 9 Claims
 
1. A method of treating mitochondrial DNA depletion syndrome in a human subject in need thereof comprising administering to the subject a therapeutically effective amount of a composition comprising at least one deoxynucleoside, wherein the mitochondrial DNA depletion syndrome is characterized by at least one mutation in a gene which is DGUOK and wherein the therapeutically effective amount is between about 200 mg/kg/day and 600 mg/kg/day of the at least one deoxynucleoside in the composition and the composition is administered to the subject parenterally.