US 12,290,574 B2
Optimized promoter sequences, intron-free expression constructs and methods of use
Xavier Anguela, Barcelona (ES); and Liron Elkouby, Modi'in-Maccabim-Reut (IL)
Assigned to Spark Therapeutics, Inc., Philadelphia, PA (US)
Appl. No. 17/250,712
Filed by SPARK THERAPEUTICS, INC., Philadelphia, PA (US)
PCT Filed Aug. 23, 2019, PCT No. PCT/US2019/048032
§ 371(c)(1), (2) Date Feb. 23, 2021,
PCT Pub. No. WO2020/041773, PCT Pub. Date Feb. 27, 2020.
Claims priority of provisional application 62/784,116, filed on Dec. 21, 2018.
Claims priority of provisional application 62/725,096, filed on Aug. 30, 2018.
Claims priority of provisional application 62/722,547, filed on Aug. 24, 2018.
Prior Publication US 2022/0362408 A1, Nov. 17, 2022
Int. Cl. A61K 48/00 (2006.01); A61K 9/00 (2006.01); A61P 7/04 (2006.01); C07K 14/755 (2006.01); C07K 14/775 (2006.01); C07K 14/81 (2006.01); C12N 7/00 (2006.01); C12N 15/86 (2006.01); A61K 38/00 (2006.01)
CPC A61K 48/0066 (2013.01) [A61K 9/0019 (2013.01); A61K 48/0058 (2013.01); A61P 7/04 (2018.01); C07K 14/755 (2013.01); C07K 14/775 (2013.01); C07K 14/8125 (2013.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); A61K 38/00 (2013.01); C07K 2319/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/22 (2013.01)] 35 Claims
 
1. A polynucleotide comprising a regulatory element nucleic acid sequence at least 98% identical to the sequence of SEQ ID NO: 58 with or without 5′ and/or 3′ flanking 5-7 nucleotides or comprising the sequence of SEQ ID NO: 24.