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:
Nov. 07, 2023

Filed:

Oct. 03, 2018
Applicant:

Prevail Therapeutics, Inc., New York, NY (US);

Inventors:

Asa Abeliovich, New York, NY (US);

Laura Heckman, New York, NY (US);

Herve Rhinn, New York, NY (US);

Assignee:

Prevail Therapeutics, Inc., New York, NY (US);

Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); A61P 25/28 (2006.01); A61K 9/00 (2006.01); C07K 14/005 (2006.01); C12N 7/00 (2006.01); C12N 9/24 (2006.01); C12N 15/86 (2006.01); A61P 25/02 (2006.01); A61P 25/16 (2006.01); A61K 48/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 9/0019 (2013.01); A61K 9/0085 (2013.01); A61K 48/0075 (2013.01); A61P 25/02 (2018.01); A61P 25/16 (2018.01); A61P 25/28 (2018.01); C07K 14/005 (2013.01); C12N 7/00 (2013.01); C12N 9/2402 (2013.01); C12N 15/86 (2013.01); C12Y 302/01045 (2013.01); A61K 48/00 (2013.01); C12N 2310/141 (2013.01); C12N 2310/531 (2013.01); C12N 2710/14043 (2013.01); C12N 2750/14133 (2013.01); C12N 2750/14143 (2013.01); C12N 2770/32022 (2013.01);
Abstract

The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding one or more inhibitory nucleic acids targeting SCNA or a portion thereof, TMEM106B or a portion thereof, or any combination of the foregoing. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.


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