MRNA therapy for phenylketonuria
AssigneeTranslate Bio, Inc.
InventorsFrank DeRosa, Anusha Dias, Michael Heartlein
Life of this patent
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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.
Claims21
A method of treating phenylketonuria (PKU), comprising administering to a subject in need of treatment a composition comprising a codon optimized 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, wherein the mRNA is encapsulated with 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.
A composition for treating phenylketonuria (PKU), comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose amount encapsulated within 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, wherein the mRNA comprises a coding sequence at least 90% identical to SEQ ID NO:3, SEQ ID NO:7 or SEQ ID NO:
- 8, a 5′ UTR sequence of SEQ ID NO:4, and a 3′ UTR sequence of SEQ ID NO:5 or SEQ ID NO:
- 6.
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:7 or SEQ ID NO:8, and further wherein the liposome comprises one or both of a cationic lipid and a non-cationic lipid, one or more cholesterol-based lipids and one or more PEG-modified lipids.
Dependencies are inferred from the claim text.