Back to results
US 2022/0023443 A1Published application
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.
Claims30
A gene therapy vector comprising a nucleic acid construct comprising:
- a nucleic acid encoding a stabilized form of a protein for treating a genetic disorder, the stabilized form 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.
Dependencies are inferred from the claim text.