Alnylam Pharmaceuticals, Inc.

Arrowhead Madison, Inc.

Hoffmann-La Roche Inc.

Arrowhead Pharmaceuticals, Inc.

Axolabs Gmbh

F. Hoffmann-La Roche Ag

Ingo Roehl

Philipp Hadwiger

Hans-Peter Vornlocher

Peter Mohr

David B Rozema

David L Lewis

Torsten Hoffmann

Eric Argirios Kitas

Pamela Tan

Hans Martin Mueller

Guenther Ott

Darren H Wakefield

Dinah Wen-Yee Sah

Kerstin Jahn-Hofmann

David A Bumcrot

Birgit Bramlage

Eric A Kitas

Muthiah Manoharan

Kallanthottathil G Rajeev

Jon Asher Wolff

Akin Akinc

Andrei V Blokhin

Tracy Stage Zimmermann

Jeffrey Carlson

Jonathan D Benson

Maria Frank-Kamenetsky

Anke Geick

Linda Valis

Jeffrey C Carlson

Dinah Sah

Markus Schuster

Kerstin Jahn-Hoffmann

Stephan Seiffert

Torsten Hoffman

Jon A Wolff

Nadine Doerfler

The invention relates to a method for the detection of oligonucleotides using anion exchange high performance liquid chromatography. Fluorescently labelled peptide nucleic acid oligomers, complementary to the oligonucleotide are hybridized to the oligonucleotides. Anion exchange high performance liquid chromatography is then performed and the hybridized moieties detected and quantitated. The invention also relates to a method for the simultaneous detection of both strands of an oligonucleotide in parallel from one sample, and a kit for use in qualitative and quantitative detection of one or two strands of an oligonucleotide.

Oligonucleotide detection method

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 present invention is directed compositions for targeted delivery of RNA interference (RNAi) polynucleotides to cell in vivo. The pharmacokinetic modulator improve in vivo targeting compared to the targeting ligand alone. Targeting ligand-pharmacokinetic modulator targeting moiety targeted RNAi polynucleotides can be administered in vivo alone or together with co-targeted delivery polymers.

Galactose cluster-pharmacokinetic modulator targeting moiety for siRNA

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

This invention relates to modified double-stranded oligoribonucleic acid (dsRNA) having improved stability in cells and biological fluids, and methods of making and identifying dsRNA having improved stability, and of using the dsRNA to inhibit the expression or function of a target gene.

Nuclease resistant double-stranded ribonucleic acid

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

The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.

Compositions and methods for inhibiting expression of huntingtin gene

Compositions and methods for inhibiting expression of Huntingtin gene

The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Nav1.8 gene (Nav1.8 gene), comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Nav1.8 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the Nav1.8 gene using the pharmaceutical composition; and methods for inhibiting the expression of the Nav1.8 gene gene in a cell.

Compositions and methods for inhibiting expression of Nav1.8 gene

Growing community of inventors

Memmelsdorf, Germany

Ingo Roehl