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:
Jun. 24, 2025

Filed:

Jan. 03, 2020
Applicant:

Ultragenyx Pharmaceutical Inc., Novato, CA (US);

Inventors:

Christine Livingston, Lambertville, NJ (US);

Samuel Wadsworth, Shrewsbury, MA (US);

Assignee:
Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 15/86 (2006.01); A61K 48/00 (2006.01); A61P 43/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/86 (2013.01); A61K 48/0058 (2013.01); A61P 43/00 (2018.01);
Abstract

This application relates to adeno-associated viral vectors encoding a truncated yet functional ATP7B for use in gene therapy for treating Wilson disease (WD). The truncated ATP7B described herein has several advantages over the wild-type ATP7B such as higher efficacy and improved manufacturing yield.


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