Modified siRNA Targeting C9orf72 Gene for Neurological Disorder Treatment
Summary
USPTO published patent application US20260098264A1 for modified single- or double-stranded siRNA molecules targeting the C9orf72 gene for treatment of neurological disorders including amyotrophic lateral sclerosis and frontotemporal dementia. The application covers branched siRNA structures with nucleoside modifications, phosphorus stabilizing moieties, and methods for CNS delivery.
What changed
USPTO published patent application US20260098264A1 for compositions and methods related to modified siRNA molecules targeting the C9orf72 gene for neurological disorder treatment. The application claims single- or double-stranded interfering RNA molecules with nucleoside modifications, internucleoside linkage modifications, and optional 5′ phosphorus stabilizing or hydrophobic moieties, including di-, tri-, or tetra-branched configurations. Methods for delivering siRNA to the central nervous system of subjects with ALS or frontotemporal dementia are also disclosed.
Implications: This patent application publication establishes prior art in the RNA therapeutics field for C9orf72-targeted neurological disease treatments. Pharmaceutical and biotechnology companies developing similar RNA-based therapies should evaluate potential freedom-to-operate considerations and assess whether design-around strategies may be needed.
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Apr 12, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
COMPOSITIONS AND METHODS FOR TREATMENT OF NEUROLOGICAL DISORDERS
Application US20260098264A1 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 a chromosome 9 open reading frame 72 (C9orf72) 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, ortetra-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 amyotrophic lateral sclerosis or frontotemporal dementia.
CPC Classifications
C12N 15/113 C12N 2310/14
Filing Date
2023-09-28
Application No.
19115144
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