Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
US11859215B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Nov 21, 2018 |
| Grant date | Jan 2, 2024 |
| Priority date | — |
| Expiry date | Jun 19, 2040 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y201/03003
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
This disclosure relates mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC), isoforms thereof, functional fragments thereof, and fusion proteins comprising OTC. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of OTC expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.