US 12,251,403 B2
Genetically engineered hematopoietic stem cells and uses thereof
Joseph Bolen, Boston, MA (US); Aleksandar Filip Radovic-Moreno, Evanston, IL (US); and John Lydeard, Sharon, MA (US)
Assigned to VOR BIOPHARMA INC., Cambridge, MA (US)
Filed by VOR BIOPHARMA INC., Cambridge, MA (US)
Filed on Mar. 10, 2020, as Appl. No. 16/813,958.
Application 16/813,958 is a continuation of application No. PCT/US2019/048662, filed on Aug. 28, 2019.
Claims priority of provisional application 62/809,202, filed on Feb. 22, 2019.
Claims priority of provisional application 62/789,440, filed on Jan. 7, 2019.
Claims priority of provisional application 62/728,061, filed on Sep. 6, 2018.
Claims priority of provisional application 62/723,993, filed on Aug. 28, 2018.
Prior Publication US 2020/0318071 A1, Oct. 8, 2020
This patent is subject to a terminal disclaimer.
Int. Cl. C07K 14/705 (2006.01); A61K 35/28 (2015.01); A61K 38/17 (2006.01); A61K 39/00 (2006.01); A61K 39/395 (2006.01); A61P 35/00 (2006.01); A61P 35/02 (2006.01); C12N 5/074 (2010.01); C12N 5/0789 (2010.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01); C12N 15/113 (2010.01); C12N 15/90 (2006.01); A61K 35/12 (2015.01)
CPC A61K 35/28 (2013.01) [A61K 38/1774 (2013.01); A61K 38/178 (2013.01); A61K 39/3955 (2013.01); A61K 39/4611 (2023.05); A61K 39/4631 (2023.05); A61K 39/464402 (2023.05); A61K 39/464411 (2023.05); A61K 39/464412 (2023.05); A61P 35/00 (2018.01); A61P 35/02 (2018.01); C07K 14/7056 (2013.01); C07K 14/70596 (2013.01); C12N 5/0647 (2013.01); C12N 9/22 (2013.01); C12N 15/11 (2013.01); C12N 15/907 (2013.01); A61K 2035/124 (2013.01); A61K 2039/505 (2013.01); A61K 2239/48 (2023.05); C12N 2310/20 (2017.05); C12N 2510/00 (2013.01); C12N 2800/80 (2013.01)] 18 Claims
 
1. A genetically engineered human hematopoietic stem cell (HSC), or
a descendant thereof, comprising a modified gene encoding a CD33 antigen and a modified gene encoding a CD123 antigen, wherein the modified gene encoding the CD33 antigen comprises a modified exon 2 or a modified exon 3 and wherein the modified gene encoding the CD123 antigen comprises a modified exon 5 or a modified exon 6;
and reduced or eliminated expression of the CD33 antigen and the CD123 antigen relative to a wild-type human HSC.