Peptide-based in vivo siRNA delivery system

US8501930B2 · kind B2 · utility

Assignee

• Arrowhead Madison, Inc. · US

Inventors

• David B. Rozema · Madison, US
• Darren H. Wakefield · Fitchburg, US
• David L. Lewis · Madison, US
• Jon Wolff · Madison, US
• Andrei V. Blokhin · Fitchburg, US
• Jonathan D. Benson · Stoughton, US
• Jeffrey C. Carlson · Madison, US
• Philipp Hadwiger · Kulmbach, DE
• Eric A. Kitas · Aesch, CH
• Torsten Hoffmann · Weil am Rhein, DE
• Kerstin Jahn-Hofmann · Neu-Isenburg, DE
• Peter Mohr · Basel, CH
• Hans Martin Mueller · Weilheim, DE
• Guenther Ott · Bayreuth, DE
• Ingo Roehl · Memmelsdorf, DE

Key dates

• Filing date: Dec 15, 2011
• Grant date: Aug 6, 2013
• Expiry date: Dec 15, 2031

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2320/32
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention is directed compositions for targeted delivery of RNA interference (RNAi) polynucleotides to hepatocytes in vivo. Targeted RNAi polynucleotides are administered together with co-targeted melittin delivery peptides. Delivery peptides provide membrane penetration function for movement of the RNAi polynucleotides from outside the cell to inside the cell. Reversible modification provides physiological responsiveness to the delivery peptides.