MRNA therapy for phenylketonuria
US10208295B2 · kind B2 · utility
18Cited by
139References
3Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Nov 11, 2016 |
| Grant date | Feb 19, 2019 |
| Priority date | — |
| Expiry date | Nov 11, 2036 |
Classification
- Technology area (CPC A)Human Necessities
- CPC primaryA61K48/00
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.