Compositions and methods for internalizing enzymes
AssigneeRegeneron Pharmaceuticals, Inc.
InventorsAndrew BAIK, Katherine CYGNAR
Life of this patent
Estimating the term…
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.
Claims36
A composition comprising an enzyme covalently linked to an antigen-binding protein, wherein the enzyme is associated with a lysosomal storage disease (LSD) and the antigen-binding protein binds an internalization effector.
A method of treating a subject suffering from a lysosomal storage disease (LSD) comprising administering to the subject a biotherapeutic complex, wherein the biotherapeutic complex enters a lysosome of a cell of the subject and provides an enzyme activity (“replacement enzyme”) that replaces the enzymatic activity that is associated with the LSD (“endogenous enzyme”).
Dependencies are inferred from the claim text.