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IP Update: Oct 28, 2010

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Title: Methods for Producing Interfering RNA Molecules in Mammalian Cells and Therapeutic Uses for Such Molecules

Patent Number: 7,820,632

Filed: Feb. 13, 2003

Lead Inventor: John Rossi, City of Hope

The invention, the patent's abstract states, comprises "methods for producing interfering RNA molecules in mammalian cells. … Therapeutic uses for the expressed molecules, including inhibiting expression of HIV, are also provided."


Title: RNAi Probes Targeting Cancer-Related Proteins

Patent Number: 7,820,635

Filed: May 6, 2008

Lead Inventor: Martin Gleave, University of British Columbia

The patent, its abstract states, claims "RNAi sequences that are useful as therapeutics in the treatment of cancers of various types, including prostate cancer, sarcomas such as osteosarcoma, renal cell carcinoma, breast cancer, bladder cancer, lung cancer, colon cancer, ovarian cancer, anaplastic large cell lymphoma, and melanoma, [as well as] Alzheimer's disease.

"These sequences target clusterin, IGFBP-5, IGFBP-2, both IGFBP-2 and -5 simultaneously, Mitf, and B-raf," the abstract adds. "The invention further provides for the use of these RNAi sequences in the treatment of cancers of various types … and Alzheimer's disease, and a method of treating such conditions through the administration of the RNA molecules with RNAi activity to an individual, including a human individual in need of such treatment."


Title: Functional and Hyperfunctional siRNA Directed Against Bcl-2

Patent Number: 7,820,809

Filed: March 18, 2005

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."


Title: microRNAs as Markers of the Functional State of a Dendritic Cell

Application Number: 20100266555

Filed: Nov. 14, 2008 PCT Filed: Nov. 14, 2008

Lead Inventor: Simon Skjode Jensen, Dandrit Biotech

The invention "relates to the use of specified microRNAs as markers of the functional state of a dendritic cell," the patent application's abstract states. "In one aspect, the invention relates to a method for producing a quality-controlled therapeutic composition comprising dendritic cells. In another aspect, the invention relates to a method of in vitro screening of immunomodulatory compounds."


Title: Oligonucleotides and Methods of Use Thereof for Treatment of Fibrotic Conditions and Other Diseases

Application Number: 20100266574

Filed: June 8, 2006 PCT Filed: June 8, 2006

Lead Inventor: Orna Mor, Quark Pharmaceuticals

"The invention relates to a double-stranded compound, preferably an oligoribonucleotide, which down-regulates the expression of a gene of the TGase family at post-transcriptional level," the patent application's abstract states. "The invention also relates to a pharmaceutical composition comprising the compound, or a vector capable of expressing the oligoribonucleotide, and a pharmaceutically acceptable carrier. The … invention also contemplates a method of treating a patient suffering from fibrotic disease such as kidney and liver fibrosis and ocular scarring comprising administering to the patient the pharmaceutical composition in a therapeutically effective amount so as to thereby treat the patient. The invention also relates to treatment of fibrotic and other diseases by use of antibodies to TGase polypeptides."


Title: Treatment of Inflammatory Diseases

Application Number: 20100266579

Filed: Oct. 21, 2008 PCT Filed: Oct. 21, 2008

Lead Inventor: Terence Cook, Imperial College London

The invention, the patent application's abstract states, is based on the "involvement of JunD in the activation of macrophages and the association of JunD in inflammatory diseases and conditions."

The application specifically claims the use of an siRNA to inhibit JunD activity.


Title: Method for Expression of Small Antiviral RNA Molecules with Reduced Cytotoxicity within a Cell

Application Number: 20100267146

Filed: April 28, 2010

Lead Inventor: Carlos Lois-Caballe, California Institute of Technology

The invention comprises "methods and compositions for the expression of small RNA molecules within a cell using a retroviral vector," the patent application's abstract states. "Small interfering RNA can be expressed using the methods of the invention within a cell."

Another aspect of the invention comprises "methods for producing siRNA encoding lentivirus where the siRNA activity may interfere with the lentiviral life cycle," it adds. "In yet a further aspect, the invention provides methods for expression of a small RNA molecule within a cell, such as an siRNA capable of down-regulating CCR5, wherein expression of the small RNA molecule is relatively non-cytotoxic to the cell. The invention also includes small RNA molecules, such as an siRNA capable of down-regulating CCR5, that are relatively non-cytotoxic to cells."


Title: Methods of Determining Potency of Chemically Synthesized Oligonucleotides

Application Number: 20100267149

Filed: Oct. 18, 2007 PCT Filed: Oct. 18, 2007

Lead Inventor: Walter Lima, Isis Pharmaceuticals

The patent application, its abstract states, claims "methods for determining potency of RNAi agents. Such methods include, but are not limited to, cell-based and cell-free assays that measure binding of an RNAi agent with Ago2 or that measure Ago2 activity in the presence of such RNAi agents.

"Also provided are assays that determine potency of RNAi agents by assessing their ability to compete with other RNAi agents, including control RNAi agents, for binding and/or activation of Ago2," it adds.


Title: Methods for the In Vivo Identification of Endogenous mRNA Targets of microRNAs

Application Number: 20100267573

Filed: Aug. 24, 2007 PCT Filed: Aug. 24, 2007

Lead Inventor: Jack Keene, Duke University

The patent application, its abstract states, claims "a method of generating a gene expression profile of non-coding regulatory RNA in a cell in vivo, [which] is carried out by partitioning from a cell at least one mRNA-protein complex, the RNP complex comprising an RNA binding protein or RNA associated protein [and] at least one mRNA bound to or associated with said protein, and at least one ncRNA bound to or associated with said protein; and then identifying at least one ncRNA in at least one RNP complex, thereby to produce a gene expression profile comprising the identity of an ncRNA in an RNP complex."


Title: siRNA Targeting KRAS

Application Number: 20100267586

Filed: May 3, 2010

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

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent application's abstract states. "By selecting particular siRNAs directed to silencing KRAS, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes."


Title: siRNA Targeting Cyclin-Dependent Kinase 11

Application Number: 20100267587

Filed: May 5, 2010

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

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent application'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 including those directed to CDK11."


Title: Delivery Method

Application Number: 20100267802

Filed: Nov. 1, 2009

Inventor: Bruce Sullenger, Duke University

The invention, the patent application's abstract states, "relates, in general, to RNA silencing and, in particular, to a method of effecting targeted delivery of an RNA silencing moiety using a targeting moiety that binds to a cell surface receptor and mediates internalization of the RNA silencing moiety to be accessible to Dicer. Also provided is a chimeric nucleic acid molecule comprised of a targeting moiety and an RNA silencing moiety, wherein the targeting moiety is an aptamer and the RNA silencing moiety comprises a Dicer substrate."


Title: Targeting Opposite-Strand Replication Intermediates of Single-Strand Viruses by RNAi

Application Number: 20100267805

Filed: March 1, 2010

Lead Inventor: David McCallus, Alnylam Pharmaceuticals

"The invention relates to methods and compositions for modulating viral replication through double-stranded RNA-mediated gene silencing, wherein the antiviral methods and compositions preferentially target opposite-strand replication intermediates of single-stranded RNA viruses," the patent application's abstract states.


Title: Lipid-Formulated Compositions and Methods for Inhibiting Expression of Eg5 and VEGF Genes

Application Number: 20100267806

Filed: March 12, 2010

Lead Inventor: David Bumcrot, Alnylam Pharmaceuticals

"This invention relates to compositions containing double-stranded ribonucleic acid in a lipid formulation, and methods of using the compositions to inhibit the expression of the human kinesin family member 11 and vascular endothelial growth factor, and methods of using the compositions to treat pathological processes mediated by Eg5 and VEGF expression, such as cancer," the patent application's abstract states.


Title: Double-Stranded Nucleic Acid Targeting Low Copy Promoter-Specific RNA

Application Number: 20100267809

Filed: May 3, 2010

Lead Inventor: John Rossi, City of Hope

According to the patent application's abstract, the invention "relates to transcriptional gene silencing in mammalian, including human, cells that is mediated by small interfering RNA molecules. The … invention also relates to a double-stranded nucleic acid that directs methylation of histones associated with target genes that produce low copy promoter-specific RNA. It has been found that siRNAs can be used to direct methylation of histones in mammalian, including human, cells."


Title: Methods and Compositions for Treating Neurological Disease

Application Number: 20100267810

Filed: June 25, 2010

Lead Inventor: Neil Aronin, University of Massachusetts

"This invention relates to methods and compositions for treating neurological disease, and more particularly to methods of delivering iRNA agents to neural cells for the treatment of neurological diseases," according to the patent application's abstract.


Title: RNAi-Related Inhibition of TNFa Signaling Pathway for Treatment of Ocular Angiogenesis

Application Number: 20100267811

Filed: June 29, 2010

Lead Inventor: Jon Chatterton, Alcon

"RNA interference is provided for inhibition of tumor necrosis factor-alpha by silencing TNF-alpha cell surface receptor TNF receptor-1 mRNA expression, or by silencing TNF-alpha-converting enzyme mRNA expression," the patent application's abstract states. "Silencing such TNF-alpha targets, in particular, is useful for treating patients having a TNF-alpha-related condition or at risk of developing a TNF-alpha-related condition, such as ocular angiogenesis, retinal ischemia, and diabetic retinopathy."


Title: Oligomeric Compounds for Use in Modulation of Small Non-Coding RNAs

Application Number: 20100267813

Filed: Dec. 31, 2008

Lead Inventor: Christine Esau, Regulus Therapeutics

The invention, the patent application's abstract states, comprises "compounds, compositions, and methods … for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs, as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs, are also provided."


Title: PFKFB4 Inhibitors and Methods of Using the Same

Application Number: 20100267815

Filed: April 16, 2010

Lead Inventor: Sucheta Telang, University of Louisville

The patent application, its abstract states, claims "methods of reducing expression of 6-phosphofructo-2-kinase/fructose-2,6-bisphosphatase 4 and methods of treating a cancer in a cell … [by] contacting a cell with an effective amount of a PFKFB4 inhibitor. Short hairpin RNA and small interfering RNA inhibitors of PFKFB4 and their methods of use are also provided."


Title: iRNA Agents with Cleavable Tethers

Application Number: 20100267941

Filed: March 15, 2010

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

"The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group," the patent application's abstract states. "The tether in turn can be connected to a selected moiety … or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent, coupled by way of the cleavable linking group to a targeting agent to target cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent."

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