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AssigneeTranslate Bio, Inc.
InventorsLianne Boeglin, Christian Cobaugh, Frank DeRosa, Michael Heartlein, Kim Askew
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The present invention provides, among other things, methods and compositions for treating methylmalonic academia (MMA) based on mRNA therapy. The compositions used in treatment of MMA comprise an mRNA comprising a methymalonyl-CoA mutase (MUT) coding sequence and are administered at an effective dose and an administration interval such that at least one symptom or feature of MMA is reduced in intensity, severity, or frequency or has a delayed onset. mRNAs with optimized MUT coding sequences are provided that can be administered without the need for modifying the nucleotides of the mRNA to achieve sustained in vivo function.
A method of treating methylmalonic acidemia (MMA) comprising administering to a subject in need of treatment a composition comprising an mRNA comprising a codon-optimized methylmalonyl-CoA mutase (MUT) coding sequence at an effective dose and an administration interval such that at least one symptom or feature of MMA is reduced in intensity, severity, or frequency or has delayed in onset, wherein the mRNA is encapsulated within a liposome that 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 nucleotides of the mRNA are unmodified.
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A composition comprising an mRNA encoding a codon-optimized methylmalonyl-CoA mutase (MUT) coding sequence, wherein the mRNA is encapsulated within a liposome and the nucleotides of the mRNA are unmodified,
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Dependencies are inferred from the claim text.