US 12,460,205 B2
Compositions and methods for treating alpha-1 antitrypsin deficiency
Shobu Odate, Arlington, MA (US); Walter Strapps, Dedham, MA (US); and Reynald Michael Lescarbeau, Medford, MA (US)
Assigned to Intellia Therapeutics, Inc., Cambridge, MA (US)
Filed by Intellia Therapeutics, Inc., Cambridge, MA (US)
Filed on Dec. 9, 2022, as Appl. No. 18/063,817.
Application 18/063,817 is a division of application No. 16/446,764, filed on Jun. 20, 2019, granted, now 11,549,107.
Application 16/446,764 is a continuation of application No. PCT/US2017/067800, filed on Dec. 21, 2017.
Claims priority of provisional application 62/438,219, filed on Dec. 22, 2016.
Prior Publication US 2023/0212575 A1, Jul. 6, 2023
Int. Cl. C12N 15/113 (2010.01); A61K 9/14 (2006.01); A61K 9/51 (2006.01); A61K 47/24 (2006.01); A61K 47/28 (2006.01); C12N 15/86 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 9/14 (2013.01); A61K 9/5146 (2013.01); A61K 47/24 (2013.01); A61K 47/28 (2013.01); C12N 15/86 (2013.01); C12N 2310/20 (2017.05)] 18 Claims
 
1. A composition comprising a guide RNA or a vector encoding the guide RNA, wherein the guide RNA comprises a guide sequence selected from SEQ ID NOs: 29 and 38 or a guide sequence that is at least 95% or 90% identical to a sequence selected from SEQ ID NOs: 29 and 38, wherein the guide RNA is a single guide RNA comprising, from 5′ to 3′, (1) the guide sequence and (2) the sequence of SEQ ID NO: 141.