Methods and compositions for the treatment of cytoplasmic glycogen storage disorders
US12036190B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Aug 21, 2021 |
| Grant date | Jul 16, 2024 |
| Priority date | — |
| Expiry date | Dec 26, 2041 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y302/01076
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present disclosure is directed to methods of treating a steatosis-associated disorder and methods of treating a cytoplasmic glycogen storage disorder, including glycogen storage disease I, glycogen storage disease III, glycogen storage disease IV, and/or conditions associated with a PRKAG2 mutation, by administering a therapeutic agent selected from a lysosomal enzyme, an autophagy-inducing agent, or a combination thereof. Steatosis-associated disorders discussed herein include GSD Ia, GSD Ib, GSD Ic, NAFLD, and NASH. Other embodiments are directed to methods of reversing steatosis, modulating autophagy, inducing autophagy, and reversing glycogen storage. Methods of treating a cytoplasmic glycogen storage disorder by administering a lysosomal enzyme and a second therapeutic agent are also described. Other embodiments are directed to methods of treating a cytoplasmic glycogen storage disorder by administering a therapeutic agent as an adjunctive therapy to lysosomal enzyme replacement therapy.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.