Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
AssigneeREGENXBIO Inc.
InventorsStephen Yoo, Rickey Robert Reinhardt, Curran Matthew Simpson, Zhuchun Wu
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Abstract
Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).
Claims20
Glycosylated recombinant human iduronate-2-sulfatase (IDS) precursor produced by human neuronal or human glial cells.
-15. (canceled)
A method for treating a human subject diagnosed with MPS II, comprising administering to the CSF of said human subject a recombinant nucleotide expression vector encoding human IDS, so that a depot is formed in the human central nervous system that secretes a glycosylated human IDS precursor that is about 90 kDa as measured by polyacrylamide gel electrophoresis, contains a formylglycine, is a2,6-sialylated, does not contain detectable NeuGc, does not contain detectable a-Gal antigen, and/or is mannose-6-phosphorylated.
A method for treating a human subject diagnosed with MPS II, comprising administering to the CSF of said human subject a formulation comprising a recombinant nucleotide expression vector encoding human IDS, wherein the formulation is suitable for administration to the CSF of human brain, so that a depot is formed in the human central nervous system that secretes a glycosylated human IDS precursor that is about 90 kDa as measured by polyacrylamide gel electrophoresis, contains a formylglycine, is a2,6-sialylated, does not contain detectable NeuGc, does not contain detectable a-Gal antigen, and/or is mannose-6-phosphorylated.
Dependencies are inferred from the claim text.