Compositions and methods for treating pompe disease
AssigneeGenzyme Corporation
InventorsKristina AN HAACK, Patrick Finn, Catherine Wilson
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Abstract
The present application provides methods of treating Pompe disease such as infantile-onset Pompe disease (IOPD) using a pharmaceutical composition comprising an oligosaccharide-acid α-glucosidase (GAA) conjugate, such as avalglucosidase alfa. Also provided are formulations of the oligosaccharide-GAA conjugates.
Claims33
A method for treating an infantile-onset Pompe disease (IOPD), comprising administering to a human individual in need thereof a pharmaceutical composition comprising an oligosaccharide-protein conjugate and a pharmaceutically acceptable carrier, wherein the oligosaccharide-protein conjugate has a structure of Formula I:
- wherein GAA is acid α-glucosidase, L is a chemical linker connecting the oligosaccharide and the GAA, and n is 1 to 10, and
- wherein the pharmaceutical composition is administered at a dose of about 20 mg/kg to about 40 mg/kg.
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A formulation comprising:
- (a) an oligosaccharide-protein conjugate;
- and (b) one or more cryoprotectants comprising a sugar that is not degraded by acid α-glucosidase;
- wherein the oligosaccharide-protein conjugate has a structure of Formula I:
- wherein GAA is acid α-glucosidase, L′ is a chemical linker, and n is 1 to 10.
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Dependencies are inferred from the claim text.