Modified polynucleotides
US-2015064235-A1 · Mar 5, 2015 · US
MRNA therapy for phenylketonuria
US10208295B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-10208295-B2 |
| Application number | US-201615349720-A |
| Country | US |
| Kind code | B2 |
| Filing date | Nov 11, 2016 |
| Priority date | Oct 22, 2013 |
| Publication date | Feb 19, 2019 |
| Grant date | Feb 19, 2019 |
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Abstract
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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.
First claim
Opening claim text (preview).
We claim: 1. A composition for treating phenylketonuria (PKU), comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose amount encapsulated within a liposome, wherein the liposome comprises one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids and wherein the liposome has a size less than 100 nm, wherein serum phenylalanine levels are reduced as compared to baseline serum phenylalanine levels before treatment, wherein the mRNA comprises SEQ ID NO:3. 2. A composition for treating phenylketonuria (PKU), comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose amount encapsulated within a liposome, wherein the mRNA comprises SEQ ID NO:3, and further wherein the liposome comprises one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids and wherein the liposome has a size less than 100 nm. 3. A composition for treating phenylketonuria (PKU), comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose amount encapsulated within a liposome, wherein the liposome comprises one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids and wherein the liposome has a size less than 100 nm, wherein serum phenylalanine levels are reduced as compared to baseline serum phenylalanine levels before treatment, wherein the mRNA encoding phenylalanine hydroxylase (PAH) has a coding sequence 100% identical to SEQ ID NO: 3.
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Classifications
Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00) · CPC title
Drugs for disorders of the nervous system · CPC title
of urine or of the urinary tract, e.g. urine acidifiers · CPC title
- A61K48/005Primary
characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered · CPC title
Oxidoreductases (1) · CPC title
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- What does patent US10208295B2 cover?
- 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 ons…
- Who is the assignee on this patent?
- Translate Bio Inc
- What technology area does this patent fall under?
- Primary CPC classification A61K48/005. Mapped technology areas include Human Necessities.
- When was this patent published?
- Publication date Tue Feb 19 2019 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 12 related publications on this page (citations in our corpus or others sharing the same primary CPC).