US 12,031,130 B2
Composition for modifying target gene
Akitsu Hotta, Kyoto (JP); Masataka Ifuku, Kyoto (JP); Naoko Fujimoto, Kyoto (JP); Kumiko Iwabuchi, Kyoto (JP); Eriya Kenjo, Kanagawa (JP); Yukimasa Makita, Kanagawa (JP); and Rumiko Ochiai, Kanagawa (JP)
Assigned to TAKEDA PHARMACEUTICAL COMPANY LIMITED, Osaka (JP)
Appl. No. 16/958,006
Filed by TAKEDA PHARMACEUTICAL COMPANY LIMITED, Osaka (JP)
PCT Filed Dec. 27, 2018, PCT No. PCT/JP2018/048034
§ 371(c)(1), (2) Date Jun. 25, 2020,
PCT Pub. No. WO2019/131829, PCT Pub. Date Jul. 4, 2019.
Claims priority of application No. 2017-254798 (JP), filed on Dec. 28, 2017.
Prior Publication US 2021/0371854 A1, Dec. 2, 2021
Int. Cl. C12N 15/11 (2006.01); A01K 67/027 (2006.01); A61K 47/24 (2006.01); C12N 9/22 (2006.01)
CPC C12N 15/11 (2013.01) [A01K 67/027 (2013.01); A61K 47/24 (2013.01); C12N 9/22 (2013.01); A01K 2217/00 (2013.01); A01K 2227/10 (2013.01); C12N 2310/20 (2017.05); C12N 2320/30 (2013.01)] 27 Claims
 
1. A composition for inducing gene modification at a target gene locus in a cell, comprising:
1) A compound of formula (I):

OG Complex Work Unit Chemistry
wherein
n represents an integer of 2 to 5,
R represents a linear C1-5 alkyl group, a linear C7-11 alkenyl group, or a linear C11 alkadienyl group, and
wavy lines each independently represent a cis-type bond or a trans-type bond,
or a salt thereof;
2) a structural lipid; and
3) a guide RNA or a DNA including a sequence encoding the guide RNA, and/or an RNA-guided nuclease or a nucleic acid including a sequence encoding the RNA-guided nuclease, wherein the guide RNA is a chimeric RNA.