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. 28, 2023

Filed:

Sep. 23, 2021
Applicant:

Scribe Therapeutics Inc., Alameda, CA (US);

Inventors:

Benjamin Oakes, El Cerrito, CA (US);

Sean Higgins, Alameda, CA (US);

Hannah Spinner, Boston, MA (US);

Sarah Denny, San Francisco, CA (US);

Brett T. Staahl, Tiburon, CA (US);

Kian Taylor, Atlanta, GA (US);

Katherine Baney, Berkeley, CA (US);

Isabel Colin, Oakland, CA (US);

Maroof Adil, Davis, CA (US);

Cole Urnes, Los Angeles, CA (US);

Assignee:

SCRIBE THERAPEUTICS INC., Alameda, CA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 9/22 (2006.01); C12N 15/11 (2006.01); C12N 15/86 (2006.01); C12N 15/90 (2006.01); A61P 25/28 (2006.01); C12N 9/06 (2006.01); C12N 15/10 (2006.01); C12N 15/113 (2010.01);
U.S. Cl.
CPC ...
C12N 9/22 (2013.01); A61P 25/28 (2018.01); C12N 9/003 (2013.01); C12N 15/101 (2013.01); C12N 15/1051 (2013.01); C12N 15/11 (2013.01); C12N 15/1133 (2013.01); C12N 15/86 (2013.01); C12N 15/907 (2013.01); C12N 2310/20 (2017.05); C12N 2750/14143 (2013.01);
Abstract

Provided herein are CasX:gNA systems comprising CasX polypeptides, guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a SOD1 gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the SOD1 protein or the SOD1 regulatory element. Also provided are methods of using such CasX:gNA systems to modify cells having such mutations and utility in methods of treatment of a subject with a SOD1-related disease.


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