Messenger RNA therapy for treatment of articular disease
AssigneeTranslate Bio, Inc.
InventorsFrank DeRosa, Michael Heartlein
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Abstract
The present invention provides, among other things, a method of intra-articular delivery of messenger RNA (mRNA), comprising administering into a joint of a subject in need of delivery a composition comprising an mRNA encoding a protein, such that the administering of the composition results in expression of the protein encoded by the mRNA in the joint.
Claims18
A method for treating an articular disease, disorder or condition in a subject in need thereof, the method comprising administering into a joint of the subject a composition comprising one or more mRNAs encoding one or more polypeptides deficient in the articular disease, disorder, or condition, such that the administering of the composition results in increased expression or activity of the one or more proteins encoded by the one or more mRNAs in the joint sufficient to treat the protein deficiency,
- wherein the one or more mRNAs are modified RNAs that comprise a backbone modification, a sugar modification, or a base modification,
- wherein the mRNA encodes a wild-type or naturally occurring protein sequence that is normally present in a healthy joint, and
- wherein the one or more mRNAs are encapsulated within one or more nanoparticles.
Dependencies are inferred from the claim text.