US 12,404,505 B2
Delivery of a gene-editing system with a single retroviral particle and methods of generation and use
Christos Kyratsous, Irvington, NY (US); Andrew J. Murphy, Croton-on-Hudson, NY (US); and Cheng Wang, White Plains, NY (US)
Assigned to Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US)
Appl. No. 16/644,411
Filed by Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US)
PCT Filed Sep. 5, 2018, PCT No. PCT/US2018/049547
§ 371(c)(1), (2) Date Mar. 4, 2020,
PCT Pub. No. WO2019/050948, PCT Pub. Date Mar. 14, 2019.
Claims priority of provisional application 62/554,500, filed on Sep. 5, 2017.
Prior Publication US 2020/0216860 A1, Jul. 9, 2020
Int. Cl. C12N 15/113 (2010.01); A61K 48/00 (2006.01); C12N 7/02 (2006.01); C12N 9/22 (2006.01); C12N 11/00 (2006.01)
CPC C12N 15/113 (2013.01) [C12N 9/22 (2013.01); A61K 48/0091 (2013.01); C12N 7/02 (2013.01); C12N 11/00 (2013.01); C12N 2310/20 (2017.05); C12N 2740/15043 (2013.01); C12Y 502/01008 (2013.01)] 28 Claims
 
1. A recombinant RNA molecule comprising (i) a sequence of a gene-editing molecule mRNA, or a sequence of a functional fragment thereof, and (ii) a sequence of a non-coding enrichment RNA, wherein said non-coding enrichment RNA comprises 7SL RNA, or a functional fragment thereof, and is capable of enhancing inclusion of said gene-editing molecule mRNA, or functional fragment thereof, into a retroviral particle.