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IP Update: Dec 23, 2010

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Title: siRNA Targeting TIE-2

Patent Number: 7,855,186

Filed: Sept. 17, 2009

Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent's abstract states. "By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed."


Title: RNA Interference-Mediated Inhibition of Checkpoint Kinase-1 Gene Expression Using Short Interfering Nucleic Acid

Patent Number: 7,855,284

Filed: Dec. 17, 2009

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

"This invention relates to compounds, compositions, and methods useful for modulating checkpoint kinase gene expression using short interfering nucleic acid molecules," the patent's abstract states. "This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of checkpoint kinase gene expression and/or activity by RNA interference using small nucleic acid molecules."


Title: Compositions and Methods for Inhibiting Viral Replication

Application Number: 20100316699

Filed: Dec. 4, 2009

Lead Inventor: John Matthias, Alnylam Pharmaceuticals

The invention, the patent application's abstract states, "relates to a double-stranded ribonucleic acid having a nucleotide sequence which is less that 30 nucleotides in length and which is substantially identical to at least a part of a 3'-untranslated region of a [positive-strand] RNA virus, such as HCV, as well as pharmaceutical compositions comprising the dsRNA, together with a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for treating infections and diseases caused by the replication or activity of the [positive-strand] RNA virus, as well as methods for inhibiting viral replication."


Title: RNA Interference-Mediating Small RNA Molecules

Application Number: 20100316703

Filed: July 13, 2010

Lead Inventor: Thomas Tuschl, Rockefeller University (Max Planck Institute)

"Double-stranded RNA induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference," the patent application's abstract states. "Using a Drosophila in vitro system, we demonstrate that 19-23 [nucleotide long] short RNA fragments are the sequence-specific mediators of RNAi. The short interfering RNAs are generated by an RNase III-like processing reaction from long dsRNA. Chemically synthesized siRNA duplexes with overhanging 3' ends mediate efficient target RNA cleavage in the lysate, and the cleavage site is located near the center of the region spanned by the guiding siRNA. Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the produced siRNP complex."


Title: Methods and Compositions for Enhancing Delivery of Double-Stranded RNA or a Double-Stranded Hybrid Nucleic Acid to Regulate Gene Expression in Mammalian Cells

Application Number: 20100316707

Filed: Aug. 30, 2010

Lead Inventor: Mohammad Ahmadian, MDRNA (Marina Biotech)

The invention, the patent application's abstract states, is a "delivery-enhancing peptide comprising [a specific] amino acid sequence … or salt thereof. This invention is directed towards methods and compositions to administer a double-stranded ribonucleic acid to a mammal so as to effectuate transfection of the double-stranded RNA into a desired tissue of the mammal."


Title: Molecular Approaches for the Optimization of Biofuel Production

Application Number: 20100317073

Filed: Dec. 4, 2008 PCT Filed: Dec. 4, 2008

Lead Inventor: Richard Sayre, Ohio State University

The invention involves the use of "rational genetic- and chemical-engineering strategies to achieve even greater efficiencies in biofuel production from microalgae," the patent application's abstract states. "These increased efficiencies may be achieved through the application of targeted and well-designed chemical and genetic engineering methods disclosed herein. The exemplary embodiments focus on increasing single cell oil yields, increased algal culture densities, and increased efficiencies in oil production. Individually or in combination, exemplary embodiments may reduce the cost to produce a barrel of biofuel to enable commercial viability."

The application specifically claims the use of siRNAs to inhibit expression of specific genes within algae.


Title: Methods and Compositions for Controlling Efficacy of RNA Silencing

Application Number: 20100317105

Filed: June 7, 2010

Lead Inventor: Phillip Zamore, University of Massachusetts Medical School

"Based at least in part on an understanding of the mechanisms by which small RNAs mediate RNA silencing in plants, rules have been established for determining, for example, the degree of complementarity required between an RNAi-mediating agent and its target, i.e., whether mismatches are tolerated, the number of mismatches tolerated, the effect of the position of the mismatches, etc.," the patent application's abstract states. "Such rules are useful, in particular, in the design of improved RNAi-mediating agents which allow for more exact control of the efficacy of RNA silencing."


Title: Complex Molecule Interfering the Expression of Target Genes and Its Preparing Methods

Application Number: 20100317714

Filed: Nov. 28, 2008 PCT Filed: Nov. 28, 2008

Lead Inventor: Zhen Xi, Suzhou Ribo Life Science

The invention provides a "complex molecule interfering [with] the expression of target genes and the methods for preparing the complex molecule wherein the complex molecule contains two siRNA strands," the patent application's abstract states. "After being administered, the Dicer enzyme in the cells is utilized to release the locked siRNAs from the complex molecules, and after unwinding, the antisense strand of the siRNA is released from the double-stranded siRNA to inhibit the expression of the target genes."


Title: RNA Interference-Mediated Inhibition of TNF and TNF Receptor Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20100317716

Filed: May 11, 2010

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

"This invention relates to compounds, compositions, and methods useful for modulating tumor necrosis factor and/or tumor necrosis factor receptor gene expression using short interfering nucleic acid molecules," according to the patent application's abstract. "This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of tumor necrosis factor and/or tumor necrosis factor receptor gene expression and/or activity by RNA interference using small nucleic acid molecules."


Title: RNA Interference-Mediated Inhibition of Platelet-Derived Growth Factor and Platelet-Derived Growth Factor Receptor Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20100317717

Filed: May 11, 2010

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

"This invention relates to compounds, compositions, and methods useful for modulating platelet derived growth factor and/or platelet derived growth factor receptor gene expression using short interfering nucleic acid molecules," the patent application's abstract states. "This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of platelet derived growth factor and/or platelet derived growth factor receptor gene expression and/or activity by RNA interference using small nucleic acid molecules."


Title: Methods and Compositions for the Treatment of Intestinal Conditions

Application Number: 20100317720

Filed: Aug. 30, 2010

Lead Inventor: Ana Jimenez, Sylentis

The patent application, its abstract states, claims "methods and compositions for the treatment of intestinal disorders, such as IBD and Crohn's disease. ... Also disclosed is a method of specifically targeting [short interfering nucleic acids] to treat intestinal disorders by intrarectal administration of siNA compounds."


Title: Compositions and Methods for Treating Tumors, Fibrosis, and Pulmonary Alveolar Proteinosis

Application Number: 20100317721

Filed: July 15, 2008 PCT Filed: July 15, 2008

Inventor: Gera Neufeld, Technion Research & Development Foundation

The invention comprises "pharmaceutical compositions and methods useful for modulating angiogenesis and for inhibiting metastasis, tumors, pulmonary alveolar proteinosis, and fibrosis in a mammalian tissue," the patent application's abstract states. "Pharmaceutical compositions and methods include inhibitors of LOXL2 expression and activity, such as shRNA targeting LOXL2."


Title: Multi-Targeted RNAi Therapeutics for Scarless Wound Healing of Skin

Application Number: 20100319074

Filed: Nov. 6, 2008 PCT Filed: Nov. 6, 2008

Lead Inventor: Patrick Lu, Sirnaomics

The invention, the patent application's abstract states, "provides small interfering RNA molecules, compositions containing them, and methods of using them for improvement of skin scarless wound healing and other skin conditions, such as psoriasis and lupus-caused cutaneous lesions. The invention includes siRNA molecules and compositions containing them that inhibit the expression of one or more genes that promote pathological or undesired processes in wound healing and methods of using them."

The Scan

Interfering With Invasive Mussels

The Chicago Tribune reports that researchers are studying whether RNA interference- or CRISPR-based approaches can combat invasive freshwater mussels.

Participation Analysis

A new study finds that women tend to participate less at scientific meetings but that some changes can lead to increased involvement, the Guardian reports.

Right Whales' Decline

A research study plans to use genetic analysis to gain insight into population decline among North American right whales, according to CBC.

Science Papers Tie Rare Mutations to Short Stature, Immunodeficiency; Present Single-Cell Transcriptomics Map

In Science this week: pair of mutations in one gene uncovered in brothers with short stature and immunodeficiency, and more.