	US 12,064,485 B2
	Disulfide bond stabilized polypeptide compositions and methods of use
Hung Do, Edmond, OK (US); and Ce Feng Liu, Brooklyn, NY (US)
Assigned to Amicus Therapeutics, Inc., Philadelphia, PA (US)
Filed by Amicus Therapeutics, Inc., Philadelphia, PA (US)
Filed on Jun. 30, 2021, as Appl. No. 17/363,333.
Application 17/363,333 is a division of application No. 16/598,960, filed on Oct. 10, 2019, granted, now 11,097,015.
Claims priority of provisional application 62/744,069, filed on Oct. 10, 2018.
Prior Publication US 2022/0023443 A1, Jan. 27, 2022
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/79 (2006.01); A61K 38/46 (2006.01); A61K 38/47 (2006.01); A61K 48/00 (2006.01); A61P 43/00 (2006.01); C07H 21/04 (2006.01); C12N 15/86 (2006.01)

CPC A61K 48/0058 (2013.01) [A61K 38/465 (2013.01); A61K 38/47 (2013.01); A61K 48/0033 (2013.01); A61P 43/00 (2018.01); C12N 15/86 (2013.01); C07H 21/04 (2013.01); C12N 15/79 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/00 (2013.01); C12Y 301/02022 (2013.01); C12Y 302/01022 (2013.01)]

20 Claims

1. 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).