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US 2025/0188433 A1Published application
Compositions and methods for internalizing enzymes
AssigneeRegeneron Pharmaceuticals, Inc.
InventorsAndrew Baik, Katherine Cygnar
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Abstract
Compositions and methods for treating lysosomal storage diseases are disclosed. Biotherapeutic complexes containing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The biotherapeutic complexes are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.
Claims10
A method of treating a subject suffering from Pompe disease, comprising administering to the subject a biotherapeutic complex comprising:
- (a) a lysosomal enzyme α-glucosidase (GAA);
- (b) a protein linker;
- and
- (c) an antigen-binding protein that binds CD63,
- wherein the GAA is linked by the protein linker to the antigen-binding protein, and
- wherein the biotherapeutic complex enters a lysosome of a cell of the subject and delivers the GAA to the lysosome).
3 more elements
Dependencies are inferred from the claim text.