Invention Application
- Patent Title: COMPOUNDS AND METHODS FOR MODULATING CLN3 EXPRESSION
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Application No.: US17274981Application Date: 2019-09-10
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Publication No.: US20220280545A1Publication Date: 2022-09-08
- Inventor: Michelle L. Hastings , Frank Rigo
- Applicant: Ionis Pharmaceuticals, Inc. , Rosalind Franklin University of Medicine & Science
- Applicant Address: US CA Carlsbad; US IL North Chicago
- Assignee: Ionis Pharmaceuticals, Inc.,Rosalind Franklin University of Medicine & Science
- Current Assignee: Ionis Pharmaceuticals, Inc.,Rosalind Franklin University of Medicine & Science
- Current Assignee Address: US CA Carlsbad; US IL North Chicago
- International Application: PCT/US2019/050476 WO 20190910
- Main IPC: A61K31/712
- IPC: A61K31/712 ; A61K31/7125 ; A61K31/713 ; A61K47/02

Abstract:
Provided are compounds, methods, and pharmaceutical compositions for modulating the expression of CLN3 RNA in a cell or animal, and in certain instances modulating the expression of CLN3 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 N symptoms and hallmarks include poor motor function, seizures, vision loss, poor cognitive function, psychiatric problems, accumulation of autofluorescent ceroid lipopigment, brain tissue dysfunction or cell death, accumulation of mitochondrial ATP synthase subunit C, accumulation of lipofuscin, or astrocyte activation in brain tissue.
Information query