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US 2026/0167944 A1Published application
Mrna therapy for phenylketonuria
AssigneeTranslate Bio, Inc.
InventorsFrank DeRosa, Anusha Dias, Michael Heartlein
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AssigneeTranslate Bio, Inc.
InventorsFrank DeRosa, Anusha Dias, Michael Heartlein
Estimating the term…
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.
A method of treating phenylketonuria (PKU), comprising 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, wherein the mRNA is encapsulated within a liposomal delivery vehicle.
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