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. 22, 2025

Filed:

Aug. 02, 2019
Applicant:

Genzyme Corporation, Cambridge, MA (US);

Inventors:

Bradford Elmer, Bridgewater, NJ (US);

Brenda Richards, Bridgewater, NJ (US);

Martine Latta-Mahieu, Paris, FR;

Maria Carmen Obinu, Paris, FR;

Véronique Taupin, Paris, FR;

Véronique Blanchard, Paris, FR;

Assignee:

Genzyme Corporation, Cambridge, MA (US);

Attorney:
Assistant Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); C12N 15/86 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); C12N 15/86 (2013.01); C12N 2310/14 (2013.01); C12N 2320/32 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/38 (2013.01); C12N 2830/42 (2013.01); C12N 2830/50 (2013.01);
Abstract

Provided herein are RNAi molecules for treating neurodegenerative synucleinopathies. In some embodiments, the RNAi molecules target expression of alpha-synuclein (SNCA). Further provided herein are expression constructs, vectors (e.g. rAAV), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat neurodegenerative synucleinopathies including Parkinson's disease, multiple system atrophy, and dementia with Lewy bodies.


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