Hoffmann-La Roche Inc.

Arrowhead Madison, Inc.

F. Hoffmann-La Roche Ag

Arrowhead Pharmaceuticals, Inc.

Guenther Ott

Peter Mohr

David B Rozema

Philipp Hadwiger

David L Lewis

Torsten Hoffmann

Eric Argirios Kitas

Ingo Roehl

Hans Martin Mueller

Kerstin Jahn-Hofmann

Jon Asher Wolff

Darren H Wakefield

Andrei V Blokhin

Jeffrey Carlson

Jonathan D Benson

Torsten Hoffman

Kerstin Jahn-Hoffmann

Eric A Kitas

Jeffrey C Carlson

The invention provides novel compounds and conjugates of these compounds useful for the delivery of biologically active substances. Further novel design criteria for chemically stabilized siRNA particular useful when covalently attached to a delivery polymer to achieve in vivo mRNA knock down are disclosed therein.

Small molecule conjugates for intracellular delivery of biologically active compounds

The invention provides use of novel compounds for delivery of nucleic acids, and conjugates of these compounds with nucleic acids. Further novel design criteria for chemically stabilized siRNA particular useful when covalently attached to said compounds and co-administered with a delivery polymer to achieve mRNA knock down in vivo are disclosed therein.

Small molecule conjugates for intracellular delivery of nucleic acids

The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.

In vivo polynucleotide delivery conjugates having enzyme sensitive linkages

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.

Peptide-based in vivo siRNA delivery system

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Bayreuth, Germany

Guenther Ott