US 12,084,693 B2
Codon-optimized acid alpha-glucosidase expression cassettes and methods of using same
Xavier Anguela, Barcelona (ES); Sean Armour, Holland, PA (US); and Jayme Nordin, Philadelphia, PA (US)
Assigned to SPARK THERAPEUTICS, INC., Philadelphia, PA (US)
Appl. No. 17/055,523
Filed by SPARK THERAPEUTICS, INC., Philadelphia, PA (US)
PCT Filed May 15, 2019, PCT No. PCT/US2019/032502
§ 371(c)(1), (2) Date Nov. 13, 2020,
PCT Pub. No. WO2019/222411, PCT Pub. Date Nov. 21, 2019.
Claims priority of provisional application 62/672,419, filed on May 16, 2018.
Claims priority of provisional application 62/734,454, filed on Sep. 21, 2018.
Prior Publication US 2021/0222141 A1, Jul. 22, 2021
Int. Cl. C12N 9/26 (2006.01); A61K 48/00 (2006.01); C12N 15/86 (2006.01)
CPC C12N 9/2408 (2013.01) [A61K 48/005 (2013.01); C12N 15/86 (2013.01); C12Y 302/0102 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14152 (2013.01); C12N 2800/22 (2013.01); C12N 2830/001 (2013.01); C12N 2830/50 (2013.01)] 40 Claims
OG exemplary drawing
 
1. A nucleic acid encoding an acid α-glucosidase (GAA) protein, wherein said nucleic acid has greater than 90% sequence identity to a sequence selected from the group consisting of any of the sequences set forth as SEQ ID NOs:1-5, and wherein the encoded GAA protein has α-glucosidase activity.