US 12,214,023 B2
Compositions and methods for expressing factor IX
Jonathan Douglas Finn, Cambridge, MA (US); Hon-Ren Huang, Cambridge, MA (US); Moitri Roy, Cambridge, MA (US); KehDih Lai, Tarrytown, NY (US); Rachel Sattler, Tarrytown, NY (US); Christos Kyratsous, Tarrytown, NY (US); and Cheng Wang, Tarrytown, NY (US)
Assigned to Intellia Therapeutics, Inc., Cambridge, MA (US); and Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US)
Filed by Intellia Therapeutics, Inc., Cambridge, MA (US); and Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US)
Filed on Oct. 18, 2019, as Appl. No. 16/657,961.
Claims priority of provisional application 62/829,621, filed on Apr. 4, 2019.
Claims priority of provisional application 62/840,352, filed on Apr. 29, 2019.
Claims priority of provisional application 62/747,509, filed on Oct. 18, 2018.
Claims priority of provisional application 62/829,009, filed on Apr. 3, 2019.
Prior Publication US 2020/0289628 A1, Sep. 17, 2020
Int. Cl. A61K 38/48 (2006.01); A61K 38/46 (2006.01); A61K 48/00 (2006.01); A61P 7/00 (2006.01); C12N 15/113 (2010.01); C12N 15/86 (2006.01); C12N 15/90 (2006.01)
CPC A61K 38/4846 (2013.01) [A61K 38/465 (2013.01); A61K 48/005 (2013.01); A61P 7/00 (2018.01); C12N 15/113 (2013.01); C12N 15/86 (2013.01); C12N 15/90 (2013.01); C12Y 304/21022 (2013.01)] 16 Claims
 
1. A method of expressing Factor IX in a cell or population of cells, comprising administering:
i) a nucleic acid construct comprising a Factor IX protein coding sequence;
ii) an RNA-guided DNA binding agent; and
iii) a guide RNA (gRNA) comprising the sequence of SEQ ID NO: 8,
thereby expressing Factor IX in a cell or population of cells.