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US 11,491,243 B2Granted
Gene therapy constructs and methods of use
AssigneeAmicus Therapeutics, Inc.
InventorsHung Do, Steven Tuske, Russell Gotschall, Ce Feng Liu
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Abstract
Provided herein are improved gene therapy vectors and methods of use, in some embodiments, comprising sequences for improved expression and cellular targeting of a therapeutic protein.
Claims18
A method for treating a genetic disorder comprising, administering to a subject in need thereof a gene therapy vector or a pharmaceutical composition comprising the gene therapy vector, wherein the gene therapy vector comprises a nucleic acid construct encoding a polypeptide comprising:
- (a) a therapeutic protein, wherein the therapeutic protein is a lysosomal enzyme;
- (b) a peptide that binds to the cation-independent mannose 6-phosphate (M6P) receptor (CI-MPR), wherein the peptide is a variant (vIGF2) peptide comprising the sequence of SEQ ID NO:
- 31;
- (c) a linker between the therapeutic protein and the peptide that binds CI-MPR;
- and (d) a signal peptide selected from a binding immunoglobulin protein (BiP) signal peptide and a Gaussia signal peptide;
- wherein the genetic disorder is a lysosomal storage disorder.
3 more elements
Dependencies are inferred from the claim text.