Ribozyme Pharmaceuticals, Incorporated

Sirna Therapeutics, Inc.

James D Thompson

Kenneth G Draper

James A McSwiggen

Bharat Chowrira

Dan T Stinchcomb

Leonid Beigelman

Peter Haeberli

Chandra Vargeese

Tod M Woolf

Nassim Usman

Jasenka Matulic-Adamic

Alexander Karpeisky

Weimin Wang

Thale Jarvis

James Mcswiggen

Shawn Patrick Zinnen

Pamela Pavco

Tongqian Chen

Laurent Bellon

David Morrissey

Kathy L Fosnaugh

Mark Reynolds

Dennis G Macejak

Narendra K Vaish

Sharon F Jamison

Michael Zwick

The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)

A transcribed non-naturally occurring RNA molecule comprising a desired RNA molecule, wherein the 3' region of the RNA is able to base-pair with at least 8 bases at the 5&#x2032; terminus of the same RNA molecule.

Polymerase III-based expression of therapeutic RNAS

This invention relates to novel chemically-modified nucleic acid molecules having specific formulae that exhibit increased resistance to nucleases and increased binding affinity to target nucleic acid molecules. The invention further relates to methods of modulating gene expression using the novel chemically modified nucleic acid molecules, and compositions and cells comprising said molecules.

Nucleic acid molecules with novel chemical compositions capable of modulating gene expression

An enzymatic RNA molecule which cleaves mRNA associated with development or maintenance of Burkitt's lymphoma or acute lymphocytic leukemia.

Method and reagent for treatment of diseases by expression of the c-Myc gene

An enzymatic RNA molecule which cleaves mRNA associated with development or maintenance of lung cancer.

Method and reagent for treatment of lung cancer and other malignancies caused by the deregulation of L-MYC gene expression

Nucleic acid catalysts, method of screening/selection for nucleic acid catalysts, synthesis of ribozyme libraries and discovery of gene sequences involved in a biological process are described.

Method for target site selection and discovery

An enzymatic nucleic acid molecule which cleaves an immunodeficiency virus RNA in a gene required for viral replication, e.g., the nef or tat gene regions.

Method and reagent for inhibiting human immunodeficiency virus

A transcribed non-naturally occuring RNA molecule comprising a desired RNA molecule, wherein the 3' region of the RNA is able to base-pair with at least 8 bases at the 5' terminus of the same RNA molecule.

RNA polymerase III-based expression of therapeutic RNAs

An enzymatic RNA molecule which specifically cleaves mRNA encoded by an mdr-1 gene.

Method and reagent for inhibiting P-glycoprotein (mdr-1-gene)

HIV nef targeted ribozymes

An enzymatic RNA molecule which cleaves mRNA associated with development or maintenance of colon carcinoma.

Enzymatic RNA with activity to RAS

An enzymatic RNA molecule which cleaves bcl.2 mRNA associated with development or maintenance of follicular lymphoma.

Method and reagent for treatment of diseases caused by expression of the

An enzymatic RNA molecule which cleaves mRNA associated with development or maintenance of CML.

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Boulder, CO, United States of America

James D Thompson