Back to results
US 12,064,485 B2Granted
Disulfide bond stabilized polypeptide compositions and methods of use
AssigneeAmicus Therapeutics, Inc.
InventorsHung Do, Ce Feng Liu
Life of this patent
Estimating the term…
Abstract
Provided herein are polypeptides comprising one or more non-native cysteine residues that form a disulfide bridge between non-native cysteines within the protein or between non-native cysteines of two monomers of the protein. Such modified human polypeptides are useful in treatment of genetic diseases via enzyme replacement therapy and/or gene therapy.
Claims20
A gene therapy vector comprising a nucleic acid construct comprising:
- a nucleic acid encoding a stabilized form of an alpha-galactosidase A (α-GAL) protein comprising one or more non-native cysteine residues that form a disulfide bridge between non-native cysteines within the protein or between non-native cysteines of two monomers of the protein, wherein the α-GAL protein comprises the non-native cysteine residues D233C and 1359C relative to the wildtype α-GAL sequence (SEQ ID NO:
- 1).
Dependencies are inferred from the claim text.