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US 10,472,615 B2Granted
Reduced immunogenic proteins for lysosomal storage disorders
AssigneeSaint Louis University
InventorsAdriana Montaño-Suarez, Angela Sosa-Molano
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Abstract
Disclosed are methods and compositions for reduced immunogenic proteins used in enzyme replacement therapy for lysosomal storage disorders. More specifically disclosed are genetically engineered variants of N-acetylgalactosamine-6-sulfatase (GALNS), which are less immunogenetic then wild-type GALNS, but maintain enzymatic activity, and may be used to treat Mucopolysaccharidosis IVA (Morquio A disease, MPS IVA).
Claims14
A method of treating a subject with mucopolysaccharidoses type IVA, the method comprising:
- a) administering, intravenously, an effective amount of a modified N-acetylgalactosamine-6-sulfate sulfatase (GALNS);
- b) the modified GALNS selected from the group consisting of SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4, SEQ ID NO:5, SEQ ID NO:6, SEQ ID NO:7, and SEQ ID NO:8, and secreted from a eukaryote cell.
Dependencies are inferred from the claim text.