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:
Jul. 05, 2022

Filed:

May. 31, 2018
Applicant:

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

Inventors:

Kiyoshi Tachikawa, San Diego, CA (US);

Carlos Gustavo Perez-Garcia, San Diego, CA (US);

Padmanabh Chivukula, La Jolla, CA (US);

Hari Bhaskaran, Oceanside, CA (US);

Christian W. Cobaugh, Boston, MA (US);

Sean Christopher Daugherty, Petaluma, CA (US);

Assignee:
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 9/10 (2006.01); A61P 3/00 (2006.01); C12N 9/44 (2006.01); C12N 15/11 (2006.01); A61K 48/00 (2006.01);
U.S. Cl.
CPC ...
C12N 9/1051 (2013.01); A61P 3/00 (2018.01); C12N 9/2451 (2013.01); C12N 15/11 (2013.01); C12Y 204/01025 (2013.01); C12Y 302/01033 (2013.01); A61K 48/00 (2013.01); C12N 2310/334 (2013.01); C12N 2310/335 (2013.01);
Abstract

This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL), or a fragment thereof having AGL activity. The polynucleotide and oligomer molecules are expressible to provide the human AGL or a fragment thereof having AGL activity. The molecules can be used as active agents to express an active polypeptide or protein in cells or subjects. The agents can be used in methods for ameliorating, preventing, delaying onset, or treating a disease or condition associated with reduced activity of amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL) in a subject.


Find Patent Forward Citations

Loading…