siRNA Molecules Targeting ATXN3 Gene for Spinocerebellar Ataxia Treatment

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COMPOSITIONS AND METHODS FOR TREATMENT OF SPINOCEREBELLAR ATAXIAS

Application US20260098265A1 Kind: A1 Apr 09, 2026

Inventors

Corrie GALLANT-BEHM, Wimalanathan KOKULAPALAN, Matthew HASSLER, Daniel CURTIS, Bruno Miguel DA CRUZ GODINHO

Abstract

The present disclosure provides single- or double-stranded interfering RNA molecules (e.g., siRNA) that target an ataxin-3 (ATXN3) gene. The interfering RNA molecules may contain specific patterns of nucleoside modifications and internucleoside linkage modifications, as pharmaceutical compositions including the same. The siRNA molecules may be branched siRNA molecules, such as di-branched, tri-branched, or tetra-branched siRNA molecules. The disclosed siRNA molecules may further feature a 5′ phosphorus stabilizing moiety and/or a hydrophobic moiety. Additionally, the disclosure provides methods for delivering the siRNA molecule of the disclosure to the central nervous system of a subject, such as a subject identified as having a spinocerebellar ataxia (e.g., spinocerebellar ataxia type 3).

CPC Classifications

C12N 15/113 C12N 2310/14 C12N 2310/52

Filing Date

2023-09-28

Application No.

19115439

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