US 11,939,600 B2
Compositions and methods for treating phenylketonuria
Kiyoshi Tachikawa, San Diego, CA (US); Carlos Gustavo Perez-Garcia, San Diego, CA (US); Padmanabh Chivukula, San Diego, CA (US); Hari Bhaskaran, San Diego, CA (US); Sean Christopher Daugherty, Petaluma, CA (US); and Christian W. Cobaugh, Newton, MA (US)
Assigned to Arcturus Therapeutics, Inc., San Diego, CA (US)
Appl. No. 16/617,462
Filed by Arcturus Therapeutics, Inc., San Diego, CA (US)
PCT Filed May 31, 2018, PCT No. PCT/US2018/035476
§ 371(c)(1), (2) Date Nov. 26, 2019,
PCT Pub. No. WO2018/222925, PCT Pub. Date Dec. 6, 2018.
Claims priority of provisional application 62/513,363, filed on May 31, 2017.
Prior Publication US 2020/0109375 A1, Apr. 9, 2020
Int. Cl. C12N 9/02 (2006.01); A61K 31/7115 (2006.01); A61K 48/00 (2006.01)
CPC C12N 9/0071 (2013.01) [A61K 31/7115 (2013.01); A61K 48/005 (2013.01); C12Y 114/16001 (2013.01); A61K 48/00 (2013.01)] 14 Claims
 
1. A polynucleotide for expressing a human phenylalanine hydroxylase (PAH), wherein the polynucleotide comprises natural and chemically-modified nucleotides and is expressible to provide the human phenylalanine hydroxylase or a fragment thereof, and wherein the polynucleotide comprises a nucleotide sequence having at least 90% identity to a nucleotide sequence selected from SEQ ID NOs: 8-36 or a nucleotide sequence having at least 94% identity to the nucleotide sequence of SEQ ID NO: 37.