	US 11,718,659 B2
	CD33 exon 2 deficient donor stem cells for use with CD33 targeting agents
Siddhartha Mukherjee, New York, NY (US); Abdullah Mahmood Ali, New York, NY (US); and Florence Borot, New York, NY (US)
Assigned to THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK, New York, NY (US)
Appl. No. 16/640,086
Filed by The Trustees of Columbia University in the City of New York, New York, NY (US)
PCT Filed Aug. 28, 2018, PCT No. PCT/US2018/048317 § 371(c)(1), (2) Date Feb. 19, 2020, PCT Pub. No. WO2019/046285, PCT Pub. Date Mar. 7, 2019.
Claims priority of provisional application 62/551,075, filed on Aug. 28, 2017.
Prior Publication US 2020/0262891 A1, Aug. 20, 2020
Int. Cl. C07K 14/705 (2006.01); A61P 35/00 (2006.01); C07K 16/28 (2006.01); C07K 16/30 (2006.01); A61K 39/00 (2006.01); C12N 5/0789 (2010.01); A61K 35/12 (2015.01)

CPC C07K 14/70596 (2013.01) [A61K 39/0011 (2013.01); A61K 39/001111 (2018.08); A61P 35/00 (2018.01); C07K 14/70578 (2013.01); C07K 16/2809 (2013.01); C07K 16/30 (2013.01); C12N 5/0647 (2013.01); A61K 2035/124 (2013.01); C07K 2317/24 (2013.01); C07K 2317/53 (2013.01); C07K 2319/02 (2013.01); C07K 2319/03 (2013.01); C07K 2319/09 (2013.01); C07K 2319/33 (2013.01)]

17 Claims

1. A method, comprising administering to a subject in need thereof:

(i) an effective amount of an agent targeting CD33, wherein the agent comprises an antigen-binding fragment that binds CD33, and

(ii) a population of hematopoietic cells that are genetically engineered to comprise a mature transcript of CD33 having a mutated exon 2 and wherein the hematopoietic cells are engineered by a CRISPR-Cas system comprising one or more guide nucleic acids comprising a sequence according to any one of SEQ ID NO: 52-61.