Is a
Patent attributes
Current Assignee
Patent Jurisdiction
Patent Number
Date of Patent
January 8, 2019
Patent Application Number
15073163
Date Filed
March 17, 2016
Patent Citations Received
Patent Primary Examiner
Patent abstract
The present invention provides, among other things, methods of treating Pompe disease, including administering to a subject in need of treatment a composition comprising an mRNA encoding acid alpha-glucosidase (GAA) at an effective dose and an administration interval such that at least one symptom or feature of Pompe disease 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.
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