	US 11,690,921 B2
	Delivery of target specific nucleases
Anthony Conway, Richmond, CA (US); Xavier de Mollerat du Jeu, Encinitas, CA (US); and Shikha Mishra, San Diego, CA (US)
Assigned to Sangamo Therapeutics, Inc., Brisbane, CA (US); and Life Technologies Corporation, Carlsbad, CA (US)
Filed by Sangamo Therapeutics, Inc., Richmond, CA (US); and Life Technologies Corporation, Carlsbad, CA (US)
Filed on May 15, 2019, as Appl. No. 16/413,144.
Claims priority of provisional application 62/673,279, filed on May 18, 2018.
Prior Publication US 2019/0365924 A1, Dec. 5, 2019
Int. Cl. A61K 48/00 (2006.01); C12N 15/88 (2006.01); C12N 15/113 (2010.01); C12N 9/22 (2006.01); A61K 9/51 (2006.01); C07K 14/47 (2006.01); A61P 11/00 (2006.01); A61K 38/00 (2006.01)

CPC A61K 48/0033 (2013.01) [A61K 9/5123 (2013.01); A61P 11/00 (2018.01); C07K 14/4712 (2013.01); C12N 9/22 (2013.01); C12N 15/113 (2013.01); C12N 15/88 (2013.01); A61K 38/00 (2013.01); C12N 2320/32 (2013.01); C12N 2800/80 (2013.01)]

24 Claims

1. A cationic lipid nanoparticle (LNP) comprising one or more polynucleotides encoding one or more zinc finger protein (ZFP) transcription factors (ZFP-TFs) or one or more zinc finger nucleases (ZFNs), wherein the ZFP-TFs or ZFNs comprise a ZFP designated 56691 or 56690, wherein

(i) the ZFP designated 56691 comprises 6 zinc finger domains ordered F1 to F6, the zinc finger domains comprising the following recognition helix regions:

F1: DRSHLTR (SEQ ID NO: 6);

F2: QSGDLTR (SEQ ID NO: 7);

F3: DRSNRTT (SEQ ID NO: 8);

F4: RSDALAR (SEQ ID NO: 9);

F5: RSDNLSE (SEQ ID NO: 10); and

F6: ERANRNS (SEQ ID NO: 11); and

(ii) the ZFN designated 56690 comprises 6 zinc finger domains ordered F1 to F6, the zinc finger domains comprising the following recognition helix regions:

F1: DRSALSR (SEQ ID NO: 12);

F2: TSGSLTR (SEQ ID NO: 13);

F3: QSSDLSR (SEQ ID NO: 14);

F4: WRKSLKV (SEQ ID NO: 15);

F5: DRSHLTR (SEQ ID NO: 6); and

F6: RLDWLPM (SEQ ID NO: 16).