MRNA therapy for Fabry disease
AssigneeShire Human Genetic Therapies, Inc.
InventorsBraydon Charles Guild, Frank DeRosa, Michael Heartlein
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Abstract
Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.
Claims8
A method of treating Fabry disease, comprising administering to a human subject in need of treatment an mRNA encoding a human alpha-galactosidase (hGLA) protein such that the hGLA protein is secreted extracellularly after expression and systemically distributed, and such that serum hGLA protein level is increased for at least 72 hours as compared to the baseline serum hGLA level before treatment,
- wherein the mRNA is administered via intravenous or pulmonary administration, and
- wherein the Fabry disease is treated.
Dependencies are inferred from the claim text.