Compositions and methods for the treatment of ornithine transcarbamylase deficiency
US12351834B2 · kind B2 · utility
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37Claims
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Key dates
| Filing date | Mar 3, 2021 |
| Grant date | Jul 8, 2025 |
| Priority date | — |
| Expiry date | Sep 29, 2043 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y201/03003
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present disclosure describes compositions and methods for treating ornithine transcarbamylase (OTC) deficiency. The compositions include a lipid formulation and messenger RNA (mRNA) encoding an OTC enzyme. The lipid formulations can comprise an ionizable cationic lipid in a lipid nanoparticle encapsulating the mRNA.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.