	US 12,070,509 B2
	Nucleic acids and methods of treatment for cystic fibrosis
Carlos G. Perez-Garcia, San Diego, CA (US); Kiyoshi Tachikawa, San Diego, CA (US); Daiki Matsuda, San Diego, CA (US); Padmanabh Chivukula, San Diego, CA (US); Priya Prakash Karmali, San Diego, CA (US); Yanjie Bao, San Diego, CA (US); Jerel Boyd Lee Vega, San Diego, CA (US); Rajesh Mukthavaram, San Diego, CA (US); and Amit Sagi, San Diego, CA (US)
Assigned to Arcturus Therapeutics, Inc., San Diego, CA (US)
Filed by Arcturus Therapeutics, Inc., San Diego, CA (US)
Filed on Apr. 30, 2021, as Appl. No. 17/246,558.
Claims priority of provisional application 63/082,997, filed on Sep. 24, 2020.
Claims priority of provisional application 63/019,170, filed on May 1, 2020.
Prior Publication US 2022/0023442 A1, Jan. 27, 2022
Int. Cl. A61K 48/00 (2006.01); A61P 11/00 (2006.01); C07K 14/47 (2006.01)

CPC A61K 48/0033 (2013.01) [A61K 48/005 (2013.01); A61P 11/00 (2018.01); C07K 14/4702 (2013.01)]

34 Claims

1. An mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA comprises an open reading frame (ORF) having 100% sequence identity with SEQ ID NO: 100, at least 94% sequence identity with SEQ ID NO: 101, or at least 95% sequence identity with one of SEQ ID NOs: 102-105.