Composition and methods for treatment of ornithine transcarbamylase deficiency
US11167043B2 · kind B2 · utility
1Cited by
70References
4Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Dec 20, 2018 |
| Grant date | Nov 9, 2021 |
| Priority date | — |
| Expiry date | Dec 20, 2038 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y201/03003
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.