Nitto-Denko Corporation

Marina Biotech, Inc.

Arcturus Therapeutics, Inc.

Ariz Precision Medicine, Inc.

Sirna Therapeutics, Inc.

Narendra Vaish

Wenbin Ying

Kenjirou Minomi

Jens Harborth

Cima Cina

Jane Zheng

Michael E Houston, Jr

Michael V Templin

Steven C Quay

Vasant R Jadhav

James Mcswiggen

Shaguna Seth

Kathy L Fosnaugh

David Morrissey

Mohammad Ahmadian

Barry A Polisky

Lonnie L Bookbinder

Brad J Niles

Jonathan Flynn

Nicole Nunez

Inhibitory RNA molecules that specifically inhibit mammalian RIZ2 expression, with therapeutic effect in cell proliferative diseases, such as cancer.

Cancer treatment using siRNA to modulate expression of PRDM2/RIZ protein

This invention provides compounds, compositions and methods for modulating the expression of target genes using RNA interference. RNAi structures and molecules of this invention can be used for modulating or silencing the expression of genes, with high levels of RNAi activity and reduced off target actions. Advantageous structures include siRNAs targeted to any gene having one or more 2′-deoxy nucleotides located in the seed region. The RNA interference molecules can be used in methods for preventing or treating diseases.

SiRNA structures for high activity and reduced off target

This invention provides compounds, compositions and methods for modulating the expression of target genes using RNA interference. RNAi structures and molecules of this invention can be used for modulating or silencing the expression of genes, with high levels of RNAi activity and reduced off target actions. Advantageous structures include siRNAs targeted to any gene having one or more 2'-deoxy nucleotides located in the seed region. The RNA interference molecules can be used in methods for preventing or treating diseases.

siRNA structures for high activity and reduced off target

This invention provides single-stranded and multi-stranded compounds that are useful in various therapeutic modalities to regulate the expression of nucleic acid molecules in a cell. A range of compounds is provided, each containing one or more conformationally restricted nucleomonomers (CRN). In addition, compounds can contain one or more conformationally restricted nucleomonomers and one or more hydroxymethyl substituted nucleomonomers (unlocked nucleomonomers, UNA).

Therapeutics with conformationally restricted monomers

This disclosure provides double-stranded RNA complexes having one or more hydroxymethyl substituted nucleomonomer(s) in the passenger strand (or sense strand) of an RNA complex. RNA complexes of the disclosure may be useful for therapeutic applications, diagnostic applications or research applications. RNA complexes include short interfering RNA complexes (siRNA) capable of modulating gene expression comprising an antisense strand and a continuous or a discontinuous passenger strand ('sense strand'). Further, one or more hydroxymethyl substituted nucleomonomer(s) of this disclosure may be positioned at the 3'-end, at the 5&#x2032;-end, at both the 3&#x2032;-end and 5&#x2032;end.

UNA oligomer structures for therapeutic agents

The present disclosure provides meroduplex (nicked or gapped) ribonucleic acid molecules (mdRNA) that decreases or silences target gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a target gene RNA. In addition, the meroduplex may have one or more modifications or substitutions, such as nucleotide base, sugar, terminal cap structure, internucleotide linkage, or any combination of such modifications. Also provided are methods of decreasing expression of a target gene in a cell or in a subject to treat a disease related to altered expression of a target gene.

Nicked or gapped nucleic acid molecules and uses thereof

Growing community of inventors

Kirkland, WA, United States of America

Narendra Vaish