Compositions and methods for treating ornithine transcarbamylase deficiency
US11685906B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Dec 5, 2019 |
| Grant date | Jun 27, 2023 |
| Priority date | — |
| Expiry date | Jul 9, 2041 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2800/22
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present disclosure provides a modified human OTC protein having improved properties for the treatment of OTC deficiency in a patient. Preferably, the protein of the disclosure is produced from a codon optimized mRNA suitable for administration to a patient suffering from OTC deficiency wherein upon administration of the mRNA to the patient, the protein of the disclosure is expressed in the patient in therapeutically effective amounts to treat OTC deficiency. The present disclosure also provides codon optimized mRNA sequences encoding wild type human OTC comprising a 5′ UTR derived from a gene expressed by Arabidopsis thaliana for use in treating OTC deficiency in a patient.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.