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IP Update: Jul 21, 2011

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Title: Pharmaceutical Compositions and Methods for Delivering Nucleic Acids into Cells

Patent Number: 7,981,446

Filed: Nov. 24, 2008

Lead Inventor: Sang-Kyou Lee, ForHumanTech

The invention, the patent's abstract states, “relates to methods of delivering nucleic acids into cells using a nucleic acid-binding molecule containing a multimeric or spacer-incorporated protein transduction domain. The invention also relates to novel compositions that contain a nucleic acid complexed or conjugated with a nucleic acid-binding molecule.

“The nucleic acid binding molecule may contain a multimeric or spacer-incorporated PTD, and may further contain a nucleic acid-binding region,” the abstract states. “The nucleic acid complexes or conjugations of the … invention may be employed to inhibit expression of a target gene, and/or determine the function of a target gene.“


Title: RNAi Modulation of RSV and Therapeutic Uses Thereof

Patent Number: 7,981,869

Filed: Dec. 2, 2008

Inventor: Rachel Meyers, Alnylam Pharmaceuticals

The invention is based on “the in vivo demonstration that RSV can be inhibited through intranasal administration of iRNA agents, as well as by parenteral administration of such agents,” the patent's abstract states. “Further, it is shown that effective viral reduction can be achieved with more than one virus being treated concurrently. Based on these findings, the … invention provides general and specific compositions and methods that are useful in reducing RSV mRNA levels, RSV protein levels and viral titers in a subject, e.g., a mammal, such as a human. These findings can be applied to other respiratory viruses.”


Title: RNAi-Mediated Inhibition of H1F1A for Treatment of Ocular Angiogenesis

Patent Number: 7,981,870

Filed: Feb. 16, 2010

Lead Inventor: Jon Chatteron, Alcon

The invention, the patent's abstract states, comprises “RNA interference ... for inhibition of HIF1A mRNA expression for treating patients with ocular angiogenesis, particularly for treating retinal edema, diabetic retinopathy, sequela associated with retinal ischemia, posterior segment neovascularization, and neovascular glaucoma, and for treating patients at risk of developing such conditions.”


Title: Lipid-Encapsulated Interfering RNA

Patent Number: 7,982,027

Filed: June 27, 2006

Lead Inventor: Ian MacLachlan, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention provides “compositions and methods for silencing gene expression by delivering nucleic acid-lipid particles comprising a siRNA molecule to a cell,” the patent's abstract states.


Title: microRNA Expression Profile Associated with Pancreatic Cancer

Application Number: 20110171646

Filed: Dec. 7, 2010

Lead Inventor: Thomas Schmittgen, Ohio State University

The invention comprises “methods … for diagnosing whether a subject has, or is at risk of developing, pancreatic cancer,” the patent application's abstract states. “The methods include measuring the level of at least one miR gene product in a biological sample derived from the subject's pancreas. An alteration in the level of the miR gene product in the biological sample as compared to the level of a corresponding miR gene product in a control sample, is indicative of the subject either having, or being at risk for developing, pancreatic cancer.”


Title: Compositions and Systems for the Regulation of Genes

Application Number: 20110172120

Filed: July 6, 2007

Lead Inventor: Didier Trono, Ecole Polytechnique Federale de Lausanne

The invention, the patent application's abstract states, “provides compositions and methods of modulating or regulating eukaryotic gene expression through the controlled or regulated expression of polynucleotide constructs that encode siRNA or other desired exogenous nucleic acids or proteins. Such constructs and additional elements of the system may be transfected into the cells of interest and the expression of the siRNA, and hence the expression of the target gene of the siRNA may be controlled through the administration of a compound to the cell, such as a small molecule or drug. Lentivirus vectors are employed in some embodiments of the invention including the generation of conditional knockdown animals.”


Title: Compositions and Methods for Inhibiting Tumor Growth and Metastasis

Application Number: 20110172287

Filed: May 7, 2010

Inventor: David Waisman, Dalhousie University

The invention, the patent application's abstract states, comprises “compositions and methods useful in the reduction of p11 protein activity in cancer cells. P11 protein is demonstrated to affect plasmin production and activity, MMP activity, plasminogen activation, antiangiogenic plasmin fragment production, cell invasion, tumor development, and metastasis. Compositions that modulate levels of p11 either up or down are demonstrated to be effective in reducing tumor development. Also disclosed are cancer treatment methods that employ compositions that modulate p11 activity and clonal cell lines and assays useful for the identification of compositions that modulate p11 activity.”


Title: Methods and Compositions for the Regulation of microRNA Processing

Application Number: 20110172295

Filed: April 6, 2009

Lead Inventor: Scott Hammond, University of North Carolina at Chapel Hill

The invention, according to the patent application's abstract, comprises “methods and compositions for the regulation of microRNA processing. The invention presents methods for inhibiting the activity of a Lin-28-like polypeptide and methods for treating a subject with cancer comprising administering to a subject with cancer a compound that inhibits the activity of a Lin-28-like polypeptide. Compositions comprise isolated polynucleotides and expression cassettes comprising the same that promote the processing of at least one of a regulated primary microRNA transcript by Drosha and a regulated precursor microRNA by Dicer when introduced into or expressed in a cell. Compositions and methods are also provided for the selective suppression of target polynucleotides by a microRNA based on the level of expression of a Lin-28-like polypeptide within a cell.”


Title: Minor Groove Binder-Oligonucleotide miRNA Antagonists

Application Number: 20110172289

Filed: Nov. 23, 2010

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

The invention, the patent application's abstract states, involves “compositions and methods for inhibiting the actions of non-coding RNAs such as miRNAs and piRNAs. ...The compositions comprise single- or double-stranded oligonucleotides conjugated with minor groove binders. The oligonucleotides can vary in length, can contain nucleotides having one or more modifications, and have regions that are substantially complementary to one or more mature miRNAs or piRNAs.”


Title: RNA Interference for the Treatment of Gain-of-Function Disorders

Application Number: 20110172291

Filed: Dec. 13, 2010

Lead Inventor: Neil Aronin, University of Massachusetts

The invention, the patent application's abstract states, “relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease. The ... invention utilizes RNA interference technology against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.”


Title: Methods and Compositions for Modulating Angiogenesis

Application Number: 20110172293

Filed: July 1, 2009

Lead Inventor: Jason Fish, University of California, San Francisco

The invention “provides compositions comprising antisense nucleic acids that reduce miR-126 levels in an endothelial cell,” the patent application's abstract states. The invention also provides “compositions comprising a target protector nucleic acid … [and] methods of modulating angiogenesis in an individual … [by] administering to the individual an effective amount of an agent that increases or that decreases the level of miR-126 in endothelial cells of the individual.”

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