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US 12,215,138 B2Granted
Compositions and methods for internalizing enzymes
AssigneeRegeneron Pharmaceuticals, Inc.
InventorsAndrew Baik, Katherine Cygnar, Christopher Schoenherr, Christos Kyratsous, Cheng Wang
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Abstract
Compositions and methods for treating enzyme-deficiency diseases are disclosed. Multidomain therapeutic proteins containing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The multidomain therapeutic proteins are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.
Claims24
A polynucleotide encoding a multidomain therapeutic protein comprising a delivery domain and an enzyme domain,
- wherein the delivery domain is an antigen-binding protein that binds to CD63, and wherein the antigen binding protein is a single-chain variable fragment (scFv),
- wherein the enzyme domain comprises an alpha-glucosidase (GAA) or a portion thereof, and
- wherein the polynucleotide further comprises a virus nucleic acid sequence, wherein the virus nucleic acid sequence is an adeno-associated virus (AAV) nucleic acid sequence.
Dependencies are inferred from the claim text.