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.
Patent No.:
Date of Patent:
May. 05, 2026
Filed:
Oct. 12, 2023
Applicant:
Ionis Pharmaceuticals, Inc., Carlsbad, CA (US);
Inventor:
Susan M. Freier, San Diego, CA (US);
Assignee:
Ionis Pharmaceuticals, Inc., Carlsbad, CA (US);
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); C12Q 1/68 (2018.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); C12N 2310/315 (2013.01); C12N 2310/322 (2013.01); C12N 2310/3341 (2013.01); C12N 2310/346 (2013.01); C12N 2320/30 (2013.01);
Abstract
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN3 RNA in a cell or animal, and in certain embodiments reducing the amount of ATXN3 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation formation, and neuron death. Such neurodegenerative diseases include spinocerebellar ataxia type 3 (SCA3).