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:
Apr. 21, 2025

Filed:

Nov. 20, 2019
Applicant:

Ionis Pharmaceuticals, Inc., Carlsbad, CA (US);

Inventors:

Susan M. Freier, San Diego, CA (US);

Huynh-Hoa Bui, San Diego, CA (US);

Hien Thuy Zhao, San Diego, CA (US);

Assignee:

Ionis Pharmaceuticals, Inc., Carlsbad, CA (US);

Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2009.12); A61K 31/7088 (2005.12);
U.S. Cl.
CPC ...
C12N 15/113 (2012.12); A61K 31/7088 (2012.12); C12N 2310/14 (2012.12); C12N 2310/31 (2012.12); C12N 2310/315 (2012.12); C12N 2310/321 (2012.12); C12N 2310/322 (2012.12); C12N 2310/3231 (2012.12); C12N 2310/3341 (2012.12); C12N 2310/34 (2012.12); C12N 2310/341 (2012.12); C12N 2310/351 (2012.12); C12N 2320/35 (2012.12);
Abstract

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PRNP RNA in a cell or animal, and in certain instances reducing the amount of PrP 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 spongiform changes in the brain, development of abnormal protein aggregates, neuronal loss, markers of neuronal loss, rapidly progressing dementia, and death. Such neurodegenerative diseases include prion diseases, Creutzfeldt-Jakob disease (CJD), variant Creutzfeldt-Jakob Disease (vCJD), familial Creutzfeldt-Jakob Disease (fCJD), Gerstmann-Straussler-Scheinker syndrome, fatal familial insomnia, kuru, Alzheimer's disease, or Parkinson's disease.


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