Ribozyme Pharmaceuticals, Incorporated

Sirna Therapeutics, Inc.

Bharat Chowrira

James A McSwiggen

James D Thompson

Dan T Stinchcomb

Kenneth G Draper

Leonid Beigelman

Chandra Vargeese

Nassim Usman

Weimin Wang

James Mcswiggen

Shawn Patrick Zinnen

Peter Haeberli

Pamela Pavco

Tongqian Chen

Kathy L Fosnaugh

David Morrissey

Dennis G Macejak

Narendra K Vaish

Sharon F Jamison

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)

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

Hairpin ribozyme lacking a substrate moiety, comprising atleast six bases in helix 2 and able to base-pair with a separate substrate RNA, wherein the said ribozyme comprises one or more bases 3' of helix 3 able to base-pair with the said substrate RNA to form a helix 5 and wherein the said ribozyme can cleave and/or ligate said separate RNA(s) in trans.

Hairpin ribozymes

HIV nef targeted ribozymes

Hairpin ribozyme lacking a substrate moiety, comprising at least six bases in helix 2 and able to base-pair with a separate substrate RNA, wherein the said ribozyme comprises one or more bases 3' of helix 3 able to base-pair with the said substrate RNA to form a helix 5 and wherein the said ribozyme can cleave and/or ligate said separate RNA(s) in trans.

HIV targeted ribozymes

Hairpin ribozyme lacking a substrate moiety, comprising at least six bases in helix 2 and able to base-pair with a separate substrate RNA, wherein the ribozyme comprises one or more bases 3' of helix 3 able to base-pair with the substrate RNA to form a helix 5 and wherein the said ribozyme can cleave and/or ligate the separate RNA(s) in trans.

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Boulder, CO, United States of America

Bharat Chowrira