The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Mar. 22, 2022

Filed:

May. 03, 2017
Applicant:

Precision Biosciences, Inc., Durham, NC (US);

Inventors:

Derek Jantz, Durham, NC (US);

James Jefferson Smith, Morrisville, NC (US);

Victor Bartsevich, Durham, NC (US);

Clayton Beard, Durham, NC (US);

Michael G. Nicholson, Chapel Hill, NC (US);

Assignee:

Precision BioSciences, Inc., Durham, NC (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/85 (2006.01); A61K 48/00 (2006.01); C07K 14/755 (2006.01); C12N 9/22 (2006.01); C12N 15/00 (2006.01); C12N 15/90 (2006.01); C12N 15/10 (2006.01); A61P 7/04 (2006.01); A61K 38/00 (2006.01); A61K 38/46 (2006.01);
U.S. Cl.
CPC ...
A61K 48/0066 (2013.01); A61K 48/005 (2013.01); A61K 48/0091 (2013.01); A61P 7/04 (2018.01); C07K 14/755 (2013.01); C12N 9/22 (2013.01); C12N 15/00 (2013.01); C12N 15/102 (2013.01); C12N 15/907 (2013.01); C12Y 301/21001 (2013.01); A61K 38/00 (2013.01); A61K 38/465 (2013.01); C12N 2750/14143 (2013.01); C12Y 301/00 (2013.01);
Abstract

The present invention encompasses engineered nucleases which recognize and cleave a recognition sequence within the int22h-1 sequence of a Factor VIII gene. The present invention also encompasses methods of using such engineered nucleases to make genetically-modified cells, and the use of such cells in a pharmaceutical composition and in methods for treating hemophilia A. Further, the invention encompasses pharmaceutical compositions comprising engineered nuclease proteins, nucleic acids encoding engineered nucleases, or genetically-modified cells of the invention, and the use of such compositions for treating of hemophilia A.


Find Patent Forward Citations

Loading…