Nucleic acids for inhibiting expression of C3 in a cell

US11753642B2 · kind B2 · utility

Assignee

• Sapience Therapeutics, Inc. · DE

Inventors

• Verena Aumiller · Berlin, DE
• Lucas Bethge · Potsdam, DE
• Judith Hauptmann · Berlin, DE
• Marie Wikström Lindholm · Malmö, SE
• Adrien Weingärtner · Berlin, DE

Key dates

• Filing date: May 26, 2022
• Grant date: Sep 12, 2023
• Expiry date: May 26, 2042

Classification

• Technology area (CPC Y): Emerging Cross-Sectional Technologies
• CPC primary: Y02A50/30
• WIPO field: Biotechnology
• WIPO sector: Chemistry

Abstract

The invention relates to nucleic acid products that interfere with complement component C3 gene expression or inhibit its expression. The nucleic acids are preferably for use as treatment, prevention or reduction of risk of suffering from complement component C3 associated diseases, disorders or syndromes, particularly C3 Glomerulopathy (C3G), Paroxysmal Nocturnal Hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS), Lupus nephritis, IgA nephropathy (IgA N), Cold Agglutinin Disease (CAD), Myasthenia gravis (MG), and Primary Membranous Nephropathy.