US 11,851,690 B2
Systems and methods for the treatment of hemoglobinopathies
Jennifer Leah Gori, Jamaica Plain, MA (US); Luis A. Barrera, Somerville, MA (US); Edouard Aupepin De Lamothe-Dreuzy, Boston, MA (US); and Jack Heath, Boston, MA (US)
Assigned to EDITAS MEDICINE, INC., Cambridge, MA (US)
Filed by EDITAS MEDICINE, INC., Cambridge, MA (US)
Filed on Sep. 12, 2019, as Appl. No. 16/569,336.
Application 16/569,336 is a continuation of application No. PCT/US2018/022516, filed on Mar. 14, 2018.
Claims priority of provisional application 62/471,342, filed on Mar. 14, 2017.
Prior Publication US 2020/0157515 A1, May 21, 2020
Int. Cl. C07H 21/02 (2006.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01); A61K 31/7105 (2006.01); A61K 38/46 (2006.01)
CPC C12N 9/22 (2013.01) [A61K 31/7105 (2013.01); A61K 38/465 (2013.01); C12N 15/111 (2013.01); C12N 2310/20 (2017.05); C12N 2320/30 (2013.01)] 12 Claims
 
1. A genome editing system, comprising:
an RNA-guided nuclease;
a first guide RNA;
a second guide RNA, and
a nucleic acid template encoding a deletion of a 13 nt region of a human HBG1 or HBG2 gene,
wherein the first and second guide RNAs comprise first and second targeting domains complementary to first and second sequences on opposite sides of positions of the 13 nt target region of a human HBG1 or HBG2 gene,
wherein one or both of the first and second sequences optionally overlaps the 13 nt target region of the human HBG1 or HBG2 gene, and
wherein at least one of the first and second targeting domains differs by no more than 3 nucleotides from a targeting domain selected from the group consisting of SEQ ID NOs: 277, 338, 327, 299, 276, 333, 278, 339, 310, 340, 940, 941, and 942.