Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
US11939601B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Nov 21, 2018 |
| Grant date | Mar 26, 2024 |
| Priority date | — |
| Expiry date | Feb 13, 2040 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2830/50
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
This disclosure relates to mRNA therapy for the treatment of hyperphenylalaninemias such as phenylketonuria (PKU). mRNAs for use in the invention, when administered in vivo, encode human phenylalanine hydroxylase (PAH), functional fragments thereof (e.g., those comprising the catalytic domain or the catalytic domain and the tetramerization domains), and fusion proteins comprising PAH. 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 PAH expression and/or activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of phenylalanine associated with deficient PAH activity in subjects.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.