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AssigneeModernaTX, Inc.
InventorsPaolo Martini, Stephen G. Hoge, Kerry Benenato, Vladimir Presnyak, Iain McFadyen, Ellalahewage Sathyajith Kumarasinghe, Xuling Zhu, Lin Tung Guey, Staci Sabnis
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The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
A messenger RNA (mRNA) comprising:
An mRNA comprising an ORF encoding a human GLA polypeptide of SEQ ID NO:
An mRNA comprising an ORF encoding a human GLA polypeptide of SEQ ID NO:
An mRNA comprising an ORF encoding a human GLA polypeptide of SEQ ID NO:
Dependencies are inferred from the claim text.