	US 11,685,906 B2
	Compositions and methods for treating ornithine transcarbamylase deficiency
Carlos G. Perez-Garcia, San Diego, CA (US); Kiyoshi Tachikawa, San Diego, CA (US); Daiki Matsuda, San Diego, CA (US); and Padmanabh Chivukula, San Diego, CA (US)
Assigned to ARCTURUS THERAPEUTICS, INC., San Diego, CA (US)
Filed by Arcturus Therapeutics, Inc., San Diego, CA (US)
Filed on Dec. 5, 2019, as Appl. No. 16/705,102.
Claims priority of provisional application 62/776,302, filed on Dec. 6, 2018.
Prior Publication US 2020/0181584 A1, Jun. 11, 2020
Int. Cl. C07H 21/02 (2006.01); C07H 21/04 (2006.01); C12N 9/10 (2006.01); A61P 9/00 (2006.01); A61K 48/00 (2006.01)

CPC C12N 9/1018 (2013.01) [A61P 9/00 (2018.01); C12Y 201/03003 (2013.01); A61K 48/00 (2013.01)]

25 Claims

1. A polynucleotide encoding an ornithine transcarbamylase (OTC) protein comprising an amino acid sequence of SEQ ID NO: 4 and having OTC enzymatic activity, wherein the polynucleotide comprises an optimized coding region encoding SEQ ID NO: 4, and wherein the polynucleotide is an mRNA comprising a sequence of SEQ ID NO: 119.